the global orphan drug conference and expo · the global gathering for orphan drugs join these...

April 25–27, 2018Gaylord National Harbor Hotel, Oxon Hill, Maryland

www.terrapinn.com/orphandrugsUSA

THE GLOBAL ORPHAN DRUG CONFERENCE AND EXPO

Brought to you by:

Together with:

1000+ ATTENDEES 250+ SPEAKERS

7 TRACKS

http://www.terrapinn.com/orphandrugsUSA

THE GLOBAL GATHERING FOR ORPHAN DRUGS

Join these speakers at World Orphan Drug Congress USA by booking your ticket today at www.terrapinn.com/WODC2017

ASIA & AUSTRALASIA

INDIA PHILIPPINES

JAPAN SINGAPORE

CHINA SOUTH KOREA

MALAYSIA TAIWAN

AUSTRALIA THAILAND

EUROPE

SPAIN

NORWAY

POLAND

SLOVENIA

FRANCE

RUSSIA

GERMANY

MALTA

AUSTRIA

BELGIUM

UNITED KINGDOM

BULGARIA

FINLAND

DENMARK

SWEDEN

SWITZERLAND

IRELAND

CROATIA

ITALY

PORTUGAL

NEW ZEALAND

NETHERLANDS

CZECH REPUBLIC

HUNGARY

ICELAND

AFRICA & MIDDLE EAST

SOUTH AFRICA

UNITED ARAB EMIRATES

ISRAEL

TURKEY

AMERICAS

USA

COLOMBIA

MEXICO

CANADA

BRAZIL

PANAMA

http://www.terrapinn.com/WODC2017

OUR STORYSince 2011, the World Orphan Drug Congress USA has been the go to global meeting for stakeholders to meet, dialogue and brainstorm about the challenges and opportunities of orphan drug development.

The 8th Annual World Orphan Drug Congress USA in 2018 will continue to expand the reach of key stakeholders from around the world coming together to discuss ideas and showcase how innovation can expedite rare disease research and bring therapies to patients faster. Key topics include: How to build up successful Expanded Access Programs? How RWE can be effectively incorporated into orphan drug development? How do you price cell and gene therapies? What are the advances in gene editing for rare diseases? Who to partner with to launch orphan drugs in Japan, Latin America, Australia and the Middle East? What is the benefit of genetic testing beyond patient identification and enrollment?

In a sector where collaboration is the key to success, there’s no better opportunity than hearing and networking altogether with leaders from industry, patient advocacy community, researchers, government & regulators, payers, investors and innovative solution providers helping expedite orphan drugs to patients in need.

In 2018, WODC USA will be held at the impressive Gaylord National Harbor Resort & Convention Center in Oxon Hill, MD, in the Washington D.C. metro area. The 250+ speakers and 1,000 attendees from over 50 countries will be spread around 3 days of full content and the following 7 streams:

• Gene Track

• Global Market Access

• Regulatory, Clinical Development

• Commercial

• Rare Disease Advocacy World

• Pitch & Partner

• Patient Groups Seminar

On the Pre-conference Day, 5 Workshops will be held covering topics such as: achieving success with a high performance community of Patients, Advocates and Caregivers; Expanded Access Programs; Business Development & Forecasting; Innovation in rare disease R&D; and Regulatory & Global Market Access success. Along with the workshops the same day will feature 4 seminars covering Next Generation Therapies, Personalized Medicine, Diagnostics, and the successful Payers Seminar, which will once again bring over 15 payers from the US, Europe and around the world to discuss clear guidelines and opportunities to improve pricing & reimbursement negotiations.

We look forward to meeting you in Oxon Hill, MD this April!

Sincerely,

André SingerGeneral Manager, World Orphan Drug Congress USAT/ +1 646 619 1797E/ [email protected]

NEW

NEW

mailto:[email protected]

2018 SPEAKERS 250+ CONFIRMED INCLUDINGPHARMA AND BIOTECHS

Katherine HighCo-founder, President and Head of Research and Development, Spark Therapeutics

Sue WasherChief Executive Officer, AGTC

Charlie AlbrightChief Scientific Officer, Editas Medicine

Mark BaglinVice President of Global Marketing, Alnylam Pharmaceuticals

Tim MillerChief Executive Officer, Abeona Therapeutics

Cristina KlafehnAssociate Director, Development Sciences Patient Advocacy, Biomarin

François NaderChairman, Acceleron Pharma

Douglas IngramChief Executive Officer, Sarepta Therapeutics

Rodger NovakChief Executive Officer and Founder, CRISPR Therapeutics

Olivier DanosChief Scientific Officer, REGENXBIO

Mark RotheraChief Executive Officer, Orchard Therapeutics

Ayse Deniz OzgerVice President, Healthcare Division, Eczacibasi

Sandy MacraePresident and Chief Executive Officer, Sangamo Therapeutics

Beverly HarrisonHead, Patient Support, Janssen Office of the Chief Medical Officer, J&J

Frederic RevahChief Executive Officer, Genethon

Yvette VenableGlobal Head of Public Affairs, Kyowa Kirin Pharmaceutical

Ken MillsChief Executive Officer, REGENXBIO

Lara SullivanPresident and Founder, SpringWorks Therapeutics

Tricia MullinsBoard Member, NDF

Francesca CookDirector, Pricing and Market Access, REGENXBIO

Dana LevasseurAssociate Director of Research, Bioverativ

Barbara WuebbelsVice President of Patient Advocacy, Audentes Therapeutics

Carole Ben-MaimonPresident & Chief Executive Officer, Chondrial Therapeutics

Jayne GershkowitzSenior Vice President and Chief Patient Advocate, Amicus Therapeutics

Rylan HanksGlobal R&D and Regulatory Policy, Product Regulatory Strategy and Intelligence, Amgen

Dawn BirChief Commercial Officer, Reata Pharmaceuticals

Christopher MisslingCEO, Anavex Life Sciences

Chandler D. RobinsonChief Executive Officer, Monopar Therapeutics Inc

Tom SabiaVice President, Haemophilia Marketing and Operations, SOBI

Jay MadanFounder & President, Innovate Biopharmaceuticals

Christer RosénChief Executive Officer, Jupiter Orphan Therapeutics

Ron AlfaVice President, Discovery & Product, Recursion Pharmaceuticals

Bill HornungCBO, Diffusion Pharmaceuticals

Igor LokotChief Executive Officer, Double Bound Pharmaceuticals

Stephen FranklinChief Executive Officer, Evgen Pharma

Saira BatesChief Executive Officer, Escend Pharmaceuticals

Donnie McGrathHead of Corporate Strategy & Business Development, Biohaven Pharmaceuticals

Philipp NovakCEO & Founder, Orphanix GmbH

Matthew RitterHead of Business Development, La Jolla Pharmaceutical

Philippe GuedatCEO and Founder, Inflectis Bioscience

Franz ObermayrChief Executive Officer, Panoptes Pharma

Alan RobertsonChief Executive Officer, Alsonex Pharmaceuticals

Fred JacobsChief Executive Officer, TYG Oncology

Laxminarayan BhatCEO, Reviva Pharmaceuticals

Brian SchwartzChief Medical Officer, ArQule

Lynnet KohCEO, Targazyme

Martin WelschofChief Executive Officer, Opsona Therapeutics

David A. CoryChief Executive Officer, Eiger Pharma

Ray TakigikuCEO, Bexion Pharmaceuticals

Scott SchliebnerVice President, Scientific Affairs, Rare Diseases, PRA Health Sciences

Arthur TzianabosPresident and Chief Executive Officer, Homology Medicines

Eric LiChief Scientific Officer, Luxena Pharmaceuticals

John LeeChief Medical Officer, Phase Bio

Mehran MoghaddamChief Executive Officer, OROX Biosciences

Prasad GabbitaChief Executive Officer, P2D Bioscience

Samantha ParkerChief Patient Access Officer, Lysogene

Aldar BourinbaiarChief Executive Officer, Immunitor

Roberto TonelliPresident, Chairman of the Board, Co-founder, Biogenera

Gregory FondDirector, External Innovation, Rare Diseases, Sanofi

Daniel de BoerFounder and Chief Executive Officer, ProQR Therapeutics

André ChoulikaChairman & Chief Executive Officer, Cellectis

Alex La CroixSenior Director, Rare Genetic Disease Marketing, Agios Pharmaceuticals

Jude SamulskiVice President, Gene Therapy, Pfizer Rare Disease

Alvin ShihChief Executive Officer, Enzyvant Therapeutics

Richard MuruveChief Executive Officer, Director, Co-founder, Arch Biopartners

Karen AiachFounder, Chief Executive Officer, Lysogene

Anita AppiusGlobal Development Team Leader, F.Hoffmann-La Roche AG

Diego ArdigòATMP Project Leader, Corporate Drug Development, R&D, Chiesi Pharmaceuticals

Aoife BrennanChief Medical Officer, Synlogic Therapeutics

Suma KrishnanCOO and Founder, Krystal Biotech

Betsy RickettsSenior Director, Government Affairs, Ultragenyx Pharmaceutical

Timothy WalbertChairman, President and Chief Executive Officer, Horizon Pharma plc

2018 SPEAKERS 250+ CONFIRMED INCLUDING

Ronald RenaudChief Executive Officer, Translate Bio

Hans BishopPresident & CEO, Juno Therapeutics

Stephen WrightChief Medical Officer, GW Pharmaceuticals

Tim Van HauwermeirenChief Executive Officer, Argenx

Meghan MillerProject Leader/Discovery Scientist, Rare Diseases, Roche

Brian BronkHead of External Innovation, Rare Diseases, Sanofi

Tuyen OngChief Development Officer, Nightstar Therapeutics

Christopher AnzalonePresident and Chief Executive Officer, Arrowhead Pharmaceuticals

Frederic ChereauChief Executive Officer, LogicBio Therapeutics

PATIENT ADVOCACY

Catherine PajakSenior Director, Marketing, North America, Lysosomal Storage Disorders, BioMarin Pharmaceutical

Christine CharmanExternal Asset Lead, TAK-celerator, Centre for External Innovation (CEI), Takeda Pharmaceuticals

Chad PulliamDirector, Channel Operations, Jazz Pharmaceuticals

Jordanna MoraAssociate Director, Patient Advocacy & Engagement, Alnylam Pharmaceuticals

John RippleChief Executive Officer, Exonics

Martine ZimmermannExecutive Director Global Regulatory Affairs, Alexion

Yann Le CamChief Executive Officer, EURORDIS

Peter SaltonstallPresident and Chief Executive Officer, NORD

Kenneth HobbyPresident, Cure SMA

Preston CampbellPresident, Cystic Fibrosis Foundation

Pamela GavinChief Strategy Officer, NORD

Pat FurlongFounding President and CEO, Parent Project Muscular Dystrophy (PPMD)

Jeanne BarnettPresident and Founder, Cysticfibrosis.com

Carmencita D PadillaFounding Chairman, Philippine Society for Orphan Disorders

Rob LongDirector of Strategic Development, Uplifting Athletes

Angela ChavezPresident, Colombia Rare Disorders Society (FECOER)

Andrew McFadyenExecutive Director, The Isaac Foundation

Durhane Wong-RiegerPresident & CEO, Canadian Organization for Rare Disorders

Ron BartekPresident, Friedreich’s Ataxia Research Alliance (FARA)

Gustavo San MartinExecutive Director, AME - Amigos Múltiplos pela Esclerose

Kelly du PlessisCEO, Rare Diseases South Africa

Steven KamiskyChief Scientific Officer, International Rett Syndrome Foundation

Harsha RajasimhaCo-Founder, Organization for Rare Diseases, India

Kevin HuangPresident, Chinese Organization for Rare Disorders

Birthe Byskov HolmPresident, Rare Disorders Denmark

Yukiko NishimuraPresident, ASrid Japan

Beata FerenczProject Manager, Rare Diseases Sweden

Katri AsikainenChairperson, HARSO Rare Diseases Alliance of Finland

Vladimir TomovPresident National Alliance of Rare Diseases, Bulgaria

Denis BelyakovChief Executive Officer, Union of Patients and Patients’ Organizations with Rare Diseases in Russia

Alastair KentAmbassador, Genetic Alliance UK

Kim HollanderExecutive Director, Oxalosis and Hyperoxaluria Foundation

Terri KleinInterim President and CEO, National MPS Society

Susan Fenters LerchPresident, The Hemophilia Foundation of Michigan

Catherine MartinExecutive Director, The Huntington’s Disease Youth Organization

Isabel LousadaChief Executive Officer, Amyloidosis Research Consortium

Chris MoenPresident, Choroideremia Research Foundation

Anne BrunsExecutive Director, The Atypical HUS Foundation

Hugh M. O’NeillPresident, Autoimmune & Rare Disease, Mallinckrodt Pharmaceuticals

Bob WardChairman of the Board and Chief Executive Officer, Eloxx Pharmaceutical

Gary GeipelGlobal Director, Global Brand Communications, Ultragenyx Pharmaceutical

John MaslowskiChief Executive Officer, Fibrocell

Raquel CaboVice President, Global Market Access, Ovid Therapeutics

Mariana FagnilliHead of Global Patient Advocacy, bluebird bio

Holly MayVice President, Sales and Marketing, United States, Sobi

Trista MorrisonVice President, Communications, North America, Sobi

Alexander StephanDiscovery Research Project Leader, Rare Diseases, Roche Pharma Research and Early Development

Martin GiedlinVice President, Technical Operations, Poseida Therapeutics

Rodolphe HajjChief Pharmacology Officer, Pharnext

Kevin WilliamsMD, Chief Medical Officer, Rare Disease, Pfizer Innovative Health at Pfizer

Sonal BhatiaMD, Vice President, Rare Disease, Pfizer

Mary Chi MichaelVice President, Patient Advocacy and Stakeholder Management, Otsuka

Mary Ellen Carroll Director, Global Community Engagement, Pfizer

Krupa SivamurthyMD - Medical Director, Pfizer

Bert BruceVice President, Global Marketing, Rare Disease at Pfizer

http://Cysticfibrosis.com


2018 SPEAKERS 250+ CONFIRMED INCLUDINGSOLUTION PROVIDERS

Nadea LeavittPredictive Analytics Principal, Real-World Insights, IQVIA

Robert DonellDirector of Business Development, Durbin

Marie TradExecutive Medical Director, IQVIA

Lynne HughesVice President & Head CNS Growth Strategy, IQVIA

Dan DonovanFounder and Chief Executive Officer, rareLife solutions

Tony HowellCo-Founder & COO, rareLife solutions

Scott GrayChief Executive Officer, Clincierge

David LapidusPresident, Lapidus Data

Scott SchliebnerVice President, Scientific Affairs, Rare Diseases, PRA Health Sciences

Joseph B. MusumeciSenior Advisor, BluePrint Orphan

Michael MurphyChief Medical and Scientific Officer, Worldwide Clinical Trials

Adam HutchingsManaging Director, Dolon

Thomas GossSenior Vice President, Boston Healthcare Associates

Matt ComstockVice President of US Business Development, WEP Clinical

Daniel AndersonHead of Commercial Partnerships, Invitae

Tom SmithBusiness Development Specialist and Project Developer, Durbin PLC

Jack MyckaGlobal President, CEO & Partner, Medical Marketing Economics

Mariah BaltezegarExecutive Director, Clinical Development, Rare Diseases, Syneos Health

Kevin KisslingVice President, 3PL Services, McKesson Specialty Health

Jaswinder Singh KheraManaging Director Americas, WEP Clinical

Douglas PaulVice President & Partner, Medical Marketing Economics

Karen KaucicSenior Vice President, Early Development, Rare Disease and Pediatric Center of Excellence, PPD

Sonja BanksMPA, MBA, President/Chief Operating Officer, Sickle Cell Disease Association of America (SCDAA)

Amanda Vassall Community Manager oneSCDvoice, Communications Assistant, Sick Cells

Stephanie RoyPrincipal, Real-World Evidence Solutions, IQVIA

Adam SohnVP, Consulting Services, IQVIA

Stella BlackburnVice President, Global Head of Early Access and Risk Management, RWES, IQVIA

Anne CroppChief Scientific Officer, Early Access Care

Simone SeiterVP, IQVIA

Kathryn LaschExecutive Director of Patient Reported Outcomes, Pharmerit International

Bob DesaiVice President, Technical, PAREXEL International

Angi RobinsonExecutive Director, Strategic Development, Rare Diseases & Pediatrics, Premier Research

Tero-Pekka AlastaloChief Medical Officer, Presidentand a co-founder, Blueprint Genetics

Walter ColasanteVice President, Charles River Associates

Jean CampbellARG Strategic Advisory Board Member, JF Campbell Associates, LLC

Carol Greve-PhillipsStrategic Advisory Board Member, Atlantic Research Group

Marilee NewmanDirector, Data Management, Atlantic Research Group

Rhonda RobertsAssociate Director, Biostatistics, Atlantic Research Group

Brad WhitlowSenior Project Manager, Atlantic Research Group

Erin FarrisDirector, Project Management, Atlantic Research Group

Tim GulliamsChief Executive Officer, Healx

Alain LamproyeChief Executive Officer, Yposkesi

Colin OrfordEVP Drug Development, ICON

James ValentineAttorney, Hyman Phelps McNamara

Abe GittermanAttorney, Arnold & Porter Kaye Scholer LLP

Gretchen GollerVP, Patient Recruitment, ICON

Cindy DukesVP, Global Project Management, Vaccines, Rare Disease, ICON

Kelly FranchettiVP, Patient Insights, legacy Mapi

Donald CreightonDivisional Principal, ICO, ICON

Will MaierChief Scientific Officer, Head Rare Disease Research, ICON

Laura WuerthrareLife solutions

Howard WoolleyPresident, Howard Woolley Group LLC

REGULATORS

Kaye RobertsonAssistant Director, Prescription Medicines Clinical Evaluation Unit 3, Therapeutic Goods Administration (TGA)

Jacqueline Corrigan-CurayDirector, Office of Medical Policy, Center for Drug Evaluation and Research, FDA

Leandro SafatleSecretário-Executivo, Câmara de Regulação do Mercado de Medicamentos (CMED), ANVISA

David LitwackPersonalized Medicine Officer, OIR/CDRH/FDA

Mark DantFounder Executive Director, The Ryan Foundation

Mark DunningChairman, Usher Syndrome Foundation

Bruce BloomChief Executive Officer, Cures Within Reach

Bryan ViauChairperson, The Huntington’s Disease Youth Organization

Henry MoehringPresident & CEO, Alpha – 1 Foundation

Julie RaskinExecutive Director, Congenital Hyperinsulinism International

Sook Yee LimCo-founder, We CARE Journey

Melissa HoganPresident, Project Alive

Join these speakers at World Orphan Drug Congress USA by booking your ticket today at www.terrapinn.com/WODC2017

INVESTORS

Kush ParmarManaging Partner, 5AM Ventures

David MottGeneral Partner, NEA

Ari BrettmanPrincipal, Clarus Ventures

David BonitaPrivate Equity Partner, OrbiMed Advisors

Ken KengatharanManaging General Partner, Atheneos Ventures

OTHER

Dr. Luiz Fernando Lima ReisDirector of Education and Research, Hospital Sírio-Libanês

Pamela TenaertsExecutive Director, Clinical Trials Transformation Initiative (CTTI)

Alison Bateman HouseAssistant Professor, Department of Population Health, NYU Langone Health

Rick ChapmanDirector of Health Economics, ICER

Gregory DanielDeputy Director, Duke-Margolis Center for Health Policy

Timothy J. McMahonProfessor of Pulmonary, Allergy, and Critical Care Medicine, Duke University School of Medicine

Scott SutherlandDirector, Portals Group, Data Sciences Platform, Broad Institute

Steven BrennerProfessor, Plant & Microbial Biology, Berkeley University

Bill RichPast President, American Academy of Ophthalmology, Chair, IRIS Registry Executive Committee

2018 SPEAKERS 250+ CONFIRMED INCLUDING

Anil G JeggaAssociate Professor Biomedical Informatics, Cincinnati Children’s Hospital

Sam EmaminejadAssistant Professor, Electrical Engineering, UCLA

Cynthia TifftDeputy Clinical Director, National Human Genome Research Institute (NHGRI)

Heather AscaniBusiness Operations, Applied Systems Biology Core Internal Medicine – Nephrology, University of Michigan

Matthias KretzlerProfessor of Internal Medicine, Computational Medicine and Bioinformatics, University of Michigan

Kelly EastGenetic Counselor, Hudson Alpha Institute for Biotechnology

David CareyAssociate Chief Research Officer, Geisenger Institute

Jarbas Barbosa da Silva JrDirector-President, ANVISA

Diane KleinermansAdvisor to the Ministry of Health and Social Affairs, Belgium Ministry of Public Health and Social Security

Makoto SuematsuPresident, Japan Agency for Medical Research and Development

Edmund JessopMedical Advisor, NHS England

Nora YangDirector, Portfolio and Project Management, Strategic Operations, NCATS, NIH

Trevor RichterDirector, Common Drug Review and Optimal Use of Drugs, CADTH

Arnaldo HossepianMember, Conselho Nacional de Justiça (Brazil)

Philip J. BrooksProgram Director, Office of Rare Diseases Research and Division of Clinical Innovation, NCATS/NIH

David ElviraDirector, Servei Català de la Salut (CatSalut)

Francis ArickxHead of the Directorate Reimbursement of Medicines and Pharmaceutical Policy, National Institute for Health and Disability Insurance (NIHDI RIZIV/INAMI)

Jeff MyersCEO, Medicaid Health Plans of America

Leung-Ming YuSenior Advisor, Norwegian Medicines Agency

Tanya PotashnikDirector, Policy and Economics Analysis Branch, Patented Medicine Prices Review Board, Government of Canada

Chris AustinDirector, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH)

Silke BaumannHead of Division, Medicinal Product Supply in the Statutory Health Insurance, Federal Ministry of Health, Germany

Jan LiliemarkProfessor, Program Manager, Swedish council for Health Technology Assessment (SBU)

GOVERNMENT INSTITUTIONS AND PAYERS


The IRIS Registry as an effective tool to gain insight into real-world diagnostic, preventive, and treatment patterns of ophthalmic conditions in the United StatesBill Rich, Past President, American Academy of Ophthalmology, Chair, IRIS Registry Executive Committee

NEXT GEN THERAPIES SEMINAR PAYERS SEMINAR DIAGNOSTICS SEMINAR

Read-through therapy as a treatment strategy for genetic diseases caused by nonsense mutationsRobert Ward, Chief Executive Officer, Eloxx Pharma

Evaluating highly priced medicines in a changing payer landscape – preparing for the introduction of next generation therapies and looking into more evidence based reimbursement

9:00 9:00 9:00

Using comprehensive phenotyping and next generation sequencing to diagnose pediatric patients with rare undiagnosed diseases Cynthia, Tifft, Deputy Clinical Director, National Human Genome Research Institute (NHGRI)

11:00

Skin TARgeted Delivery platform using engineered viral vector for gene transfer as an off-the-shelf treatment for rare dermatological diseasesSuma Krishnan, COO and Founder, Krystal Biotech

10:30

Pricing & Reimbursement Simulation Presented by Dolon

How much? Orphan drug pricing simulation workshop Pharmaceutical pricing is complex, opaque and sometimes controversial. In this simulation workshop, participants will be put in the shoes of a biotech CEO making a pricing decision for an innovative new rare disease medicine.

Learn: How do biopharmaceutical executives make pricing decisions for orphan drugs? Which factors are relevant to pricing decisions and which are not? What is the relationship between R&D investment and price?

Gain: Expert knowledge about how to make pricing decisions for orphan drugs in a way that is transparent, fair, and supportive of future innovation in rare diseases.

Who: Pharmaceutical industry executives, payers, patient representatives and anyone interested in better understanding how drug pricing and investment decisions are made in rare diseases.

Themes: Therapeutic value, benchmarking, return on investment, and willingness-to-pay

11:00

Genetic counseling’s key role in the rare disease diagnostics ecosystemKelly East, Genetic Counselor, Hudson Alpha Institute for Biotechnology

11:25

Next generation in vivo and ex vivo gene editing using homologous recombination to treat rare diseasesArthur Tzianabos, President and Chief Executive Officer, Homology Medicines

11:25

9:20

Transformative RNA therapies for the treatment of cystic fibrosis (CF) and ornithine transcarbamylase (OTC) Ronald Renaud, Chief Executive Officer, Translate Bio

9:20

Medicaid update on drug coverage and reimbursement in the current healthcare landscapeJohn Coster, Director, Division of Pharmacy, Center for Medicare and Medicaid Services (CMS)

9:20

Overcoming challenges of genome sequencing interpretation from newborn genome sequencesSteven Brenner, Professor, Plant & Microbial Biology, Berkeley University

9:40

Innovative approaches to treat Spinal Muscular Atrophy by correcting the splicing of SMN2 genes Alexander Stephan, Discovery Research Project Leader, Rare Diseases, Roche Pharma Research and Early Development

9:40

Access and pricing for drugs in Medicaid: how to reform the program to address both priorities?Jeff Myers, President & CEO, Medicaid Health Plans of America

9:40

IRUD Beyond - a new approach to find undiagnosed patients in Japan Makoto Suematsu, President, Japan Agency for Medical Research and Development

APRIL 25, 2018

NETWORKING BREAK10:00

IS YOUR COMPANY IS INTERESTED IN SPONSORING/ HOSTING A WORKSHOP?Contact Andre Singer at [email protected] or +1 646 619 1797

NETWORKING LUNCH12:00

11:40

Novel genome editing approach to develop lifelong cures for early-onset rare diseases Frederic Chereau, Chief Executive Officer, LogicBio Therapeutics

PERSONALIZED MEDICINE SEMINAR

3:00

A cannabinoid-based medicine offering relief and meeting the needs of patients with a rare childhood-onset epilepsyStephen Wright, Chief Medical Officer, GW Pharmaceuticals

3:00

Belgium, Netherlands, Luxembourg and Austria, collaborating in horizon scanning, HTA, pricing and reimbursement to ensure patients’ access to expensive medicinesFrancis Arickx, Head of the directorate Reimbursement of Medicines and Pharmaceutical Policy, National Institute for Health and Disability Insurance (NIHDI RIZIV/INAMI), Belgium

3:00

Wearable molecular diagnostic platforms for large-scale and longitudinal rare disease clinical investigation and patient monitoringSam Emaminejad, Assistant Professor, Electrical Engineering, UCLA

3:50

Gene therapy and CAR-T product candidates for orphan diseases and cancerMartin Giedlin, Vice President, Technical Operations, Poseida Therapeutics

3:40

Ensuring fair pricing of pharmaceuticals and looking into the Canadian landscape for highly priced medicinesTanya Potashnik, Director, Policy and Economics Analysis Branch, Patented Medicine Prices Review Board, Government of Canada

3:40

The use of biomarkers as a form of personalized medicine to help gauge response to therapy in patients with Pulmonary arterial hypertension (PAH) Timothy J. McMahon, Professor of Pulmonary, Allergy, and Critical Care Medicine, Duke University School of Medicine

3:25

Differentiated antibody-based therapies for the treatment of severe autoimmune rare diseases and cancerTim Van Hauwermeiren, Chief Executive Officer, Argenx

3:20

Strengthening evidence-based decisions and the benefit assessment of orphan drugs in GermanySilke Baumann, Head of Division, Medicinal Product Supply in the Statutory Health Insurance, Federal Ministry of Health, Germany

3:20

Applicability and shareability of gene sequencing data in rare disease research Scott Sutherland, Director, Data Sciences Platform, Broad Institute

1:25

SingleCut CRISPR for the treatment of neuromuscular diseasesJohn Ripple, Chief Executive Officer, Exonics

1:25

Precision medicine and the impact of information on drug repurposing Bruce Bloom, Chief Executive Officer, Cures Within Reach

1:40

Gene therapy of inherited retinal diseaseTuyen Ong, Chief Development Officer, Nightstar Therapeutics

1:00

Synthetic biotic medicines for the treatment of rare genetic diseases including Urea Cycle Disorder (UCD) and Phenylketonuria (PKU)Aoife Brennan, Chief Medical Officer, Synlogic Therapeutics

1:00

Updates on new legislation and guidelines on how to conduct pharmacoeconomic analysis in Norway – Quantifying severity and the willingness to pay for new drugsLeung-Ming Yu, Senior Advisor, Norwegian Medicines

1:00

Update on The FDA’s role in moving towards a global precision medicine industry: Impact on rare diseasesDavid Litwack, Personalized Medicine Officer, OIR/CDRH/FDA

1:50

Brazil’s landscape for the recognition of rare diseases and the evaluation of orphan drugs to ensure access and continuous supply Leandro Safatle, Secretário-Executivo, Câmara de Regulação do Mercado de Medicamentos (CMED), ANVISA

NETWORKING COFFEE BREAK2:20

Improving access to rare disease therapies: lessons learned from the Catalan experienceDavid Elvira, Director, Servei Català de la Salut (CatSalut)

1:25

1:50

The DiscovEHR Project: the role of genetic variation in rare and common diseasesDavid J. Carey, Associate Chief Research Officer, Geisenger Institute

2:00

Genetically-modified fibroblast platform for rare skin diseasesJohn Maslowski, Chief Executive Officer, Fibrocell

PRE-CONFERENCE SEMINARS 1/3 PRE-CONFERENCE SEMINARS 2/3




WORKSHOPS

Unleashing the Power of the PAC:Achieving Success with a High-Performance Community of Patients, Advocates and Caregivers

WEDNESDAY APRIL 25TH 2018 – 9AM TO 12PM4:15

Next generation RNAi-based therapies for the treatment of rare diseases Christopher Anzalone, President and Chief Executive Officer, Arrowhead Pharmaceuticals

4:15

Ethical and health economic aspects on introduction of new orphan drugs in SwedenJan Liliemark, Professor, Program Manager, Swedish council for Health Technology Assessment (SBU)

4:00

Data-driven approaches to identify candidate therapeutics for rare lung disordersAnil Jegga, Associate Professor, Biomedical Informatics, Cincinnati Children’s Hospital

4:20

Orphan drug reimbursement and HTA in Poland the second largest market in Europe

4:20

Integrating structured and unstructured data and digital health technologies to accelerate biopharma R&D Harsha K. Rajasimha, Co-Director, Rare Diseases and Systems Biology Initiative, George Mason University

4:40

Next generation drug repurposing using network pharmacology to develop new therapeutic entities for rare diseasesRodolphe Hajj, Chief Pharmacology Officer, Pharnext

4:40

Precision medicine based treatment strategies in rare disease in public private partnerships via platform clinical trial approachesHeather Ascani, Business Operations, Applied Systems Biology Core Internal Medicine – Nephrology, University of MichiganMatthias Kretzler, Professor of Internal Medicine, Computational Medicine and Bioinformatics, University of Michigan

END OF SEMINARS5:00

PRE-CONFERENCE SEMINARS 3/3


Hosted by rareLife Solutions WORKSHOP 1

WHO IS IT FOR?Interact with expert panelists from rare disease advocacy, pharma and compliance who discuss their perspectives on the importance of building online high-performing, multi-stakeholder PAC communities. Novel technology frameworks are being customized by the PAC to meet their specific needs and thereby ushering in a new generation of online communities. The PAC works in collaboration with a coalition of leaders from their rare disease ecosystem of patients, advocates, caregivers, social workers, healthcare professionals, researchers and others to design their own online experience which in turn meets their particular needs for their constituency: emotional support, curated knowledge, access to new treatments, clinical trial information, insight generation through patient experience data collection and analysis and many other solutions.

TOPICS COVERED1. defining: (a) high performance communities, (b) value propositions to all stakeholders, and (c) success measurements including Social ROI

2. identifying and addressing considerations involving: (a) experiences of the patient and caregiver, (b) patients as partners, (c) community engagement,

3. a case study of high performance community success in sickle cell disease, and

4. planning for FDA compliance and generating insights, data and potential new endpoints to support possible labeling expansion benefitting the PAC.

Engage directly in the workshop using real-time live polls and pose questions to the participants!

AGENDA

Welcome, Opening Remarks & slido.com InstructionsDan Donovan, Founder and Chief Executive Officer, rareLife solutions

9.00–9.10

COFFEE AND DISCUSSION BREAK10.10-10.25

Defining the Value of a High-Performance CommunityDan Donovan, Founder and Chief Executive Officer, rareLife solutions

9.10–9.20

Panel: Creating Environments for Real World Change: How the PAC Community Contributes to Treatment Research, Development and CommercializationModerated by: Laura Wuerth, EVP and Rare Strategies Leader, rareLife solutionsHoward Woolley, President, Howard Woolley Group LLCBert Bruce, Vice President, Global Marketing, Rare Disease at PfizerSonal Bhatia, MD, Vice President, Rare Disease, PfizerJodie Gillon, Global Medical Lead, Patient Engagement, Pfizer Rare Disease

9.10–9.20

Panel: Pfizer & Advocacy Collaboration Case Study: Setting the Stage for Real World Change in Sickle Cell Disease Moderated by: Tony Howell, Co-Founder & COO, rareLife solutionsSonja Banks, MPA, MBA, President/Chief Operating Officer, Sickle Cell Disease Association of America (SCDAA)Mary Ellen Carroll, Director, Global Marketing, PfizerKrupa Sivamurthy, MD - Medical Director, PfizerAmanda Vassall, Community Manager, oneSCDvoice, Communications Assistant, Sick Cells

10.25–11.10

Panel: FDA & High-Performance Communities: Addressing Compliance Considerations and Utilizing Patient Experience Data Moderated by: Tony Howell, Co-Founder & COO, rareLife solutionsJames Valentine, Attorney, Hyman Phelps McNamaraAbe Gitterman, Attorney, Arnold & Porter Kaye Scholer LLP

11.10–11.50

CLOSING REMARKSDan Donovan, Founder and Chief Executive Officer, rareLife solutions

11.50-12.00


WORKSHOPS

Expanded Access Programs and Data Collection

WEDNESDAY APRIL 25TH 2018 – 9AM TO 12PM

Hosted by WEP ClinicalWORKSHOP 2

WHO IS IT FOR?Project Managers and Executives in Pharma/Biotech/Advocacy interested in learning how data can be captured and used in an Expanded Access Program

TOPICS COVEREDThis interactive presentation by WEP Clinical will provide you with an opportunity to learn more about how Expanded Access Programs can be used to collect and analyze Real World patient data. You will learn from the experiences gained in delivering multiple projects across many therapeutic areas. We will share best practices in collecting data, data collection tools, management of the data, regulations around the collection of data in an EAP environment, and the benefits of collecting this type of data.

The first part of the session will focus on the regulations around collecting and protecting Real World Data. The second part will look at the methods and tools that can be used to collect and house the data, as well as the value of this data. This workshop will be extremely beneficial to those individuals considering an EAP as part of their drug development strategy.

AGENDA

Regulations & HIPAAJas Khera, Managing Director, WEP ClinicalMatt Comstock, Vice President, WEP Clinical

9.00–9.10

NETWORKING COFFEE BREAK10.15–10.45

Data and CollectionJas Khera, Managing Director, WEP ClinicalMatt Comstock, Vice President, WEP Clinical

9.00–9.10


WORKSHOPS

Ensuring success of rare disease drug development

WEDNESDAY APRIL 25TH 2018 – 9AM TO 12PM

Hosted by ICON plcWORKSHOP 3

WHO IS IT FOR?Pharma and biotech companies involved in rare disease development

• Patient advocacy groups looking to set up registries and clinical trials• Job titles:

• Medical director

• Therapy area director

• Clinical trial director/manager

• Regulatory

• Market Access

WHAT IS THE PROBLEM THAT YOUR APPROACH CAN HELP SOLVE?• Orphan drug development has several unique challenges. This workshop provides expert guidance and practical case studies to help ad-

dress the major challenges of drug candidate early development, regulatory strategy, trial design, patient engagement and market access.

WHAT ARE THE BENEFITS OF ATTENDING YOUR WORKSHOP?• Meet experts with unique and varied experience across all aspects of orphan and rare disease drug development and commercialization• Understand the lifecycle of product development and rapidly gain insights to help maximize your investments to increase chances of

success.

AGENDA

Endpoint design and selection in the current regulatory and HTA environment in the US and Europe Rory Graham, Regulatory, Mapi

Early drug development advice and guidance to optimize the pre-clinical profileColin Orford, EVP Drug Development, ICON

Patient-based clinical trial designs that are scientifically robust and address the unique challenges of the diseaseCindy Dukes, Vice President, Global Project Management, Vaccines, Rare Disease, ICON

Engagement and retention of patients in clinical trials, expanded access programs and patient registriesGretchen Goller, VP, Patient Recruitment, ICON

Pricing strategies for market access of rare disease treatmentsDonald Creighton, Divisional Principal, ICO, ICON

Communication with patients, health care providers and other disease community stakeholder groups to build support and advocacy for the develop-ment of new rare disease treatmentsKelly Franchetti, Vice President, Patient Insights, Mapi




WORKSHOPS

Predicting and influencing the market potential of orphan drugs: Embracing the interdependence of forecasts and early development decisions

WEDNESDAY APRIL 25TH 2018 – 1PM TO 5PM

Hosted by MME and Lapidus DataWORKSHOP 4

MME and LapidusData will share their expertise in developing market assessments/forecasts for orphan drugs. We will demonstrate how the end goal—a broadly-accessible, high-value orphan drug—requires early attention to clinical decision-making as well as traditional mar-ket analyses. We will discuss the US, EU5, and other key markets as needed.

WHO IS IT FOR?• CCO, CEO, CBO (start-ups)• Business Development, Forecasting and Portfolio• Product Management, Marketing, Global Branding, Product Development• Pricing, Reimbursement and Market Access• Clinical Development, R&D

WHAT IS THE PHARMA FACES THAT YOUR EXPERTISE/APPROACH CAN HELP RESOLVE?Rare diseases have a unique set of challenges for market sizing, pricing and reimbursement throughout the globe. We help identify and capture value by quantifying market sizes, assessing the value of an opportunity, and planning commercial strategies.

WHAT ARE THE BENEFITS OF YOUR APPROACH?By reducing uncertainty, you can better establish the value of a product/company and guide commercial/clinical planning. A stronger understanding of the quality of available data can:

• Highlight weak points in your present-day valuation and forecast• Identify specific market research and clinical activities that can support a stronger valuation

Our long history in the orphan drug space gives us unparalleled experience overcoming the challenges and pitfalls of rare diseases and orphan drugs. We use a unique combination of manufacturer and customer perspectives combined with academic theory to develop an understanding of the value perceptions of all key stakeholders.

In addition to work in oncology and orphan markets, we have helped >40 ultra-orphan drugs to quantify, frame, communicate and capture the value of their therapies.

WHAT ARE THE BENEFITS OF ATTENDING YOUR WORKSHOP?• Build a stronger rationale for the valuation of an orphan drug or company• Understand how the factors behind a valuation influence each other: epidemiology, unmet need, trial design, and pricing/reimbursement• Identify which valuation factors can be improved and when these activities must be planned• Learn best practices for orphan drug market assessments, including clinical activities that have commercial impact• Review specific resources that can provide data to support market assessments and valuation: literature, insurance data, electronic medi-

cal records, and payor research

AGENDA

A holistic view on how clinical and commercial choices affect/reinforce each other

a. How many patients exist (market size)? Is our therapy valuable to them (market share)? How can we provide them with access to our therapy (pricing and reimbursement)?

b. Does market size/share set the terms of pricing/reimbursement—or does pricing/reimbursement set the terms for market size/share?David Lapidus, President, Lapidus DataDoug Paul, Vice President and Partner, Medical Marketing Economics Jack Mycka, Global President and Chief Executive Officer, Medical Marketing Economics

Market potential: treatable population and subsets

a. Identify key drivers: Prevalence, diagnosis rates, disease severity

b. Assess key drivers: Evaluate today’s epidemiology and generate new data

c. Does history repeat itself? Selecting analog diseases/products when orphan markets are changing

d. The conundrum of disparate indications: using a value perspective to prioritize indications

e. Formulation: impact on competition, reimbursement, and value proposition David Lapidus, President, Lapidus DataDoug Paul, Vice President and Partner, Medical Marketing Economics Jack Mycka, Global President and Chief Executive Officer, Medical Marketing Economics

Market share: population and subsets likely to receive treatment

a. Using the treatment decision to define market segmentation

b. Unmet need

c. Competition David Lapidus, President, Lapidus Data=

Setting a price: What does “value” mean?

a. Unmet need versus clinical endpoints

b. Competition / price frames

c. Dosing

i. Defining the “average” patient and assessing outliers

d. Market access opportunities and challenges in major orphan markets

i. Impact of single use products (e.g., gene therapies)

e. Gross-to-net implicationsDoug Paul, Vice President and Partner, Medical Marketing Economics Jack Mycka, Global President and Chief Executive Officer, Medical Marketing Economics





WORKSHOPS

Innovation in Rare Disease R&D

WEDNESDAY APRIL 25TH 2018 – 1PM TO 5PM

Hosted by Atlantic Research GroupWORKSHOP 5

Atlantic Research Group, a rare-disease focused CRO, is pleased to host today’s workshop at the World Orphan Drug Congress. These interactive sessions are geared to clinical research professionals and advocacy representatives.

WHO IS IT FOR?• Company profiled: pharma and biotechs• Positions: CEO, C-level Management, VPs, Heads, Directors• Departments: Clinical Operations, Clinical Development• Therapeutic areas: hematology, immunology, and rare diseases

AGENDA

The promise of gene therapy in rare disease

A discussion on this emerging field of research, and provide insight for clinical research professionals and patient advocacy representatives. Ms. Greve-Philips will present the challenges and benefits of gene therapy and leave participants with the wonder of “what can be”.Carol Greve-Phillips, ARG Strategic Advisory Board Member, Consultant


1.00

The importance of advocacy in rare disease clinical trial development

A discussion on the role that Patient Advocacy groups play in rare disease clinical trials. ARG recognizes the value of each subject enrolling in a clinical trial, and our success has been built on this model - without advocacy groups linking industry to patient communities, rare disease clinical trials may be significantly delayed.Jean Campbell, ARG Strategic Advisory Board Member, JF Campbell Associates

1.45

AFTERNOON COFFEE BREAK 2.30

Data-Driven Trial Management (DDTM) in rare disease trials

A presentation of the ideas and discipline related to DDTM and how ARG puts them into practice in rare disease research.Rhonda Roberts, Associate Director, Biostatistics, Atlantic Research GroupMarilee Newman, Director, Data Management, Atlantic Research Group

3.00

Challenges in rare disease trials

A discussion on the challenges in implementing and running rare disease trials, and provide insight on what works and how ARG has overcome obstacles.Erin Farris, Director, Project Management, Atlantic Research GroupBrad Whitlow, Senior Project Manager, Atlantic Research Group

3.45

Q&A session and networking 4.30

END OF WORKSHOP5.00


CELL THERAPIES

GENE THERAPIES

INTERESTED IN SPONSORING OR EXHIBITING?Contact Andre Singer at [email protected] or +1 646 619 1797

In 2017, CAR-T cell therapies became a reality with the first 2 approvals of such therapies by the FDA, and increasing number of similar programs being developed and advancing to late stage trials. At the World Orphan Drug Congress USA 2018, some of the biotechs developing the most advanced CAR-T therapies will be presenting their data.

As gene therapies reach the market and gene editing clinical development becomes a reality, the Gene Track will feature what’s most advanced in applying genetics to diagnose and treat rare disease patients.

NEXT GENERATION THERAPIES


Rare disease research is evolving. So does the types of therapies that are being developed to treat and cure these rare and ultra-rare disorders. From gene editing to microbiome therapies, the World Orphan Drug Congress USA 2018 will be featuring a variety of presentations by innovative biotechs who are disrupting R&D.

Gene therapy and CAR-T product candidates for orphan diseases and cancer

Martin GiedlinVice President, Technical Operations

Cell and gene therapy innovation and their role in the future of rare disease research

Hans BishopPresident & CEO

Next-generation CAR T-cells to cure cancer – paving a new way for R&D, value-assessment and pricing and reimbursement of treatments for genetic diseases

André ChoulikaChairman & Chief Executive Officer

A cannabinoid-based medicine offering relief and meeting the needs of patients with a rare childhood-onset epilepsy

Stephen WrightChief Medical Officer

Gene therapy and CAR-T product candidates for orphan diseases and cancer

Martin GiedlinVice President, Technical Operations

Differentiated antibody-based therapies for the treatment of severe autoimmune rare diseases and cancer

Tim Van HauwermeirenChief Executive Officer

Moderator:

Jude SamulskiVice President, Gene Therapy

Tim MillerChief Executive Officer

Mark RotheraChief Executive Officer

Ken MillsChief Executive Officer

Frederic RevahChief Executive Officer

Karen AiachFounder, Chief Executive Officer

Keynote CEO Panel: Gene therapy – a new paradigm for patients, new challenges for industry

NEXT GENERATION THERAPIES

Synthetic biotic medicines for the treatment of rare genetic diseases including Urea Cycle Disorder (UCD) and Phenylketonuria (PKU)

Aoife BrennanChief Medical Officer

Rodolphe HajChief Pharmacology Officer

Next generation drug repurposing using network pharmacology to develop new therapeutic entities for rare diseases

Transformative RNA therapies for the treatment of cystic fibrosis (CF) and ornithine transcarbamylase (OTC)

Ronald RenaudChief Executive Officer

Frederic ChereauChief Executive Officer

Novel genome editing approach to develop lifelong cures for early-onset rare diseases



PARTICIPATING PATIENT ADVOCACY GROUPS INCLUDE


EURORDIS Cure SMA Cystic Fibrosis Foundation NORD Parent Project Muscular Dystrophy (PPMD),

Philippine Society for Orphan Disorders Cysticfibrosis.com Uplifting Athletes We CARE Journey Canadian

Organization for Rare Disorders, Rare Diseases South Africa Colombia Rare Disorders Society (FECOER)

Friedreich’s Ataxia Research Alliance (FARA) International Rett Syndrome Foundation The Isaac Foundation

AME - Amigos Múltiplos pela Esclerose Organization for Rare Diseases India Chinese Organization for

Rare Disorders ASrid Japan HARSO Rare Diseases Alliance of Finland Rare Disorders Denmark Rare

Diseases Sweden Genetic Alliance UK National Alliance of Rare Diseases Bulgaria Union of Patients and

Patients’ Organizations with Rare Diseases in Russia RARE Science Oxalosis and Hyperoxaluria Foundation

National MPS Society Amyloidosis Research Consortium Choroideremia Research Foundation The Atypical

HUS Foundation The Hemophilia Foundation of Michigan The Huntington’s Disease Youth Organization

Foundation Fighting Blindness Usher Syndrome Foundation ADNP Kids Research Foundation The

Sturge-Weber Foundation Osteogenesis Imperfecta Foundation Facioscapulohumeral Muscular Dystrophy

Society Canadian Pituitary Patient Network Foundation for Ichthyosis and Related Skin Types Chiari and

Syringomyelia Foundation Phelan-McDermid Syndrome Foundation NBIA Disorders Association Platelet

Disorder Support Association National Blood Clot Alliance Sarcoidosis of Long Island MLD Foundation

Myotonic Dystrophy Foundation CHERUBS Canadian Association of Pompe Bardet Biedl Syndrome Family

Association Mickie’s Miracles, Cure HHT FamilieSCN2A Foundation National Down Syndrome Congress

American Behcet’s Disease Association The LAM Foundation Ben’s Friends, Tuberous Sclerosis Alliance

Association for Frontotemporal Dementias (AFTD) Sickle Cell Thalassemia Patients Network Angioma Alliance

International Waldenstrom’s Macroglobulinemia Foundation Interstitial Cystitis Association Alagille Syndrome

Alliance Accord Alliance Syncope Trust and Reflex Anoxic Seizures Foundation for Prader Willi Research

Intracranial Hypertension Research Foundation Circadian Sleep Disorders Network PKD Foundation

Neuromuscular Disease Foundation Kids v Cancer A Twist of Fate A.T.S. Myasthenia Gravis Foundation

of America SMA Foundation Cure CMD Relapsing Polychondritis Awareness and Support Foundation

Wilson Disease Association American Autoimmune Related Diseases Association Jett Foundation Costello

Syndrome Family Network EveryLife Foundation for Rare Diseases Organic Acidemia Association Glut1

Deficiency Foundation PBCers.org Osteogenesis Imperfecta Foundation Fibrous Dysplasia Foundation

A-T Children’s Project MLD Foundation Jonah’s Just Begun The Jansens Foundation Caregiver Action

Network Kennedy Krieger Institute Cures Within Reach Alpha – 1 Foundation E.C.D Global Alliance

Fight ALD Muscular Dystrophy Association The National Alliance for Rare Diseases Support – Malta



http://PBCers.org

KATHERINE HIGHPresident and Head, Research & DevelopmentSpark Therapeutics

Opening keynote address: A new era in medicine – introducing gene therapy

HANS BISHOPPresident & CEOJuno Therapeutics

Keynote address: Cell and gene therapy innovation and their role in the future of rare disease research

GENE TRACK


As gene therapies reach the market and gene editing clinical development becomes a reality, the Gene Track will feature what’s most advanced in applying genetics to diagnose and treat rare disease patients.

ADVANCED GENE EDITING BIOTECHS

Developing genomic medicines and the first ever in- vivo human genome editing trials using zinc finger nucleases

Sandy MacraePresident and Chief Executive Officer

Novel genome editing approach to develop lifelong cures for early-onset rare diseases

Frederic ChereauChief Executive Officer

Translating CRISPR/Cas9 technology into transformative gene-based medicines for patients with serious diseases

Rodger NovakPresident, CRISPER Therapeutics AD and Director of Board

SingleCut CRISPR for the treatment of neuromuscular diseases

John RippleChief Executive Officer

Advancing innovative therapies to treat genetically-defined diseases using CRISPR-based genome editing

Charlie AlbrightChief Scientific Officer

Next generation in vivo and ex vivo gene editing using homologous recombination to treat rare diseases

Arthur TzianabosPresident and Chief Executive Officer

The NIH Common Fund Somatic Cell Genome Editing Program: Developing tools and technologies tso enable safe and effective genome editing in humans

Christopher AustinDirector

Partnerships between industry and patient groups to advance gene therapy research to treat inherited retinal diseases

Sue WasherChief Executive OfficerAGTC

Keynote address - A new era in medicine- introducing gene terhapy

Katherine HighPresident and Head of Research and DevelopmentSpark Therapeutics

Creating value in gene therapy clinical development programs, achieving trial optimization and supporting the path to commercialization

Olivier DanosChief Scientific OfficerREGENXBIO

Keynote CEO Panel – Gene therapy: a new paradigm for patients, new challenges for industry

Moderated by Pfizer

Tim MillerChief Executive OfficerAbeona Therapeutics

Mark RotheraChief Executive OfficerOrchard Therapeutics

Jude SamulskiVice President, Gene TherapyPfizer Rare Disease

Ken MillsChief Executive OfficerREGENXBIO


GLOBAL MARKET ACCESS After an orphan drug is approved in the US or Europe the next logical commercial step is to find patients in other markets who can access and benefit from the therapy.

The Global Market Access track will showcase regulators, government and other key stakeholders from 10+ countries on how to successfully navigate the regulatory, pricing and reimbursement frameworks for a successful launch.

CANADA: Orphan drug evaluation and decision making in Canada in the context of international HTA decisions Trevor Richter

Director, Common Drug Review and Optimal Use of DrugsCADTH

Turkey: Access to orphan drugs in a large market outside the European Union Ayse Deniz Ozger

Vice President, Healthcare DivisionEczacibasi

ITALY: The Italian experience on market access for rare disease drugs

Martine ZimmermannExecutive Director Global Regulatory AffairsAlexion

Market access challenges and opportunities for orphan and ultra-orphan drugs Thomas Goss

Senior Vice PresidentBoston Healthcare Associates

The Philippines: How a newly passed Rare Diseases Act is unlocking an emerging market’s commercial potential Carmencita D. Padilla

Founding ChairmanPhilippine Society for Orphan Disorders

BENELUX-A Belgium, Netherlands, Luxembourg and Austria joining forces to improve access and to orphan drugs and paving the way for more collaborations

Diane KleinermansAdvisor to the Ministry of Health and Social AffairsBelgium Ministry of Public Health and Social Security

BRAZIL: Accelerating the review and approval of orphan drugs in Latin America’s largest market Jarbas Barbosa da Silva Jr.

Director-PresidentANVISA

Australia: ‘Orphan drug program reforms: changes in the eligibility criteria of orphan drug designations Kaye Robertson

Assistant Director Prescription Medicines Clinical Evaluation Unit 3Therapeutic Goods Administration (TGA)

COMMERCIAL

Collaborative mission-driven approach to developing innovative potential new treatments for unserved and underserved patient communities Lara Sullivan

President and Founder

Value Assessment is different for Rare Diseases, requiring new thinking to build consensus around access decisions Walter Colasante

Vice President

Ensuring commercial success through lifelong innovation: evolving alongside patients to meet their changing needs through the product lifecycle

Holly MayVice President, Sales and Marketing, United States

Development and execution of commercial strategies for clinical and commercial stage RNAi products for serious, life-threatening diseases

Mark BaglinVice President of Global Marketing

TAK-celeratorTM - An incubator-like model to accelerate the development of transformative therapies in rare diseases

Christine CharmaExternal Asset Lead, TAK-celerator, Centre for External Innovation (CEI),

Designing your distribution channel to optimize your orphan drug commercialization strategy

Kevin KisslingVice President, 3PL Services

Chad PulliamDirector, Channel Operations

What drives orphan drug commercial launch success?

A mature and more competitive orphan drug industry requires more innovative business models, commercialization strategies, and particularly forecasting and epidemiology analysis. Join the sessions on the Commercial Track to learn how rare disease biotechs have successfully planned and executed their business models.

Gregory FondDirector, External Innovation

Navigating the rare disease innovation ecosystem and partnering for the long-term

Launching a commercial function from scratch – developing capabilities to enable rare disease product launches

Alex La CroixSenior Director, Rare Genetic Disease Marketing

Adam SohnVice President, Consulting Services

Simone SeiterVice President, Global Lead Launch Excellence

REGULATORY & CLINICAL While rare disease clinical development becomes increasingly complex, the opportunities from successful approaches in Expanded Access Programs, Real World Evidence and patient centricity put in practice are poising huge opportunities to accelerate the pace of new and better orphan drugs reaching patients in need. Join the Regulatory & Clinical Development Track to find out how to benefit from those!

ROUNDTABLE: Incentives ecosystem - current political, regulatory and legislative climate in the EU and US, around orphan product incentives

Rylan HanksGlobal R&D and Regulatory Policy, Product Regulatory Strategy and IntelligenceAmgen

ROUNDTABLE: Gene Therapy trials- Overcoming the challenges of conducting gene therapy studies and navigating the regulatory landscape

Scott SchliebnerVice President, Scientific Affairs, Rare DiseasesPRA Health Sciences

The evolution of a partnership to help decision making and improve patient access to investigational medicines

Beverly HarrisonHead, Patient Support, Janssen Office of the Chief Medical OfficerJ&J

Alison Bateman HouseAssistant Professor, Department of Population HealthNYU Langone Health

The virtual opportunity in rare disease trialsKaren KaucicSenior Vice President, Early Development, Rare Disease and Pediatric Center of ExcellencePPD

From real world data to real world evidence: FDA’s perspective on the enhanced use of RWE Jacqueline Corrigan-Curay

Director, Office of Medical Policy, Center for Drug Evaluation and ResearchFDA

Early development planning for orphan drugs: tasks, timelines, and takeaways

Angi RobinsonExecutive Director, Strategic Development, Rare Diseases & PediatricsPremier Research



IRDiRC goals 2017-2027 – new research goals to accelerate rare disease therapies and diagnostics globally Christopher Austin

ChairmanInternational Rare Disease Research Consortium



PRICING & REIMBURSEMENT


‘Value’ is the term of the moment for how orphan drugs are prices and reimbursed. Payers are increasingly looking into real value offered by new and innovative therapies reaching the market and their cost-benefit ration to both the healthcare system and patients. As cell and gene therapies reach the market, this discussion is not only imminent, but crucial. Make sure to attend the sessions on this track to hear from the most innovative value-based pricing & reimbursement approaches.


Drug pricing in Europe, improving the role of HTA agencies and looking into the sustainability of managed access agreements Edmund Jessop

Medical AdvisorNHS England

Proposed Value based frameworks for pricing and reimbursement of orphan drugs - can a new value framework help ease friction over orphan drug prices? Rick Chapman

Director of Health EconomicsICER

Medicaid update on drug coverage and reimbursement in the current healthcare landscape

John CosterDirector, Division of PharmacyCenter for Medicare and Medicaid Services (CMS)

Improving access to rare disease therapies: lessons learned from the Catalan experience

David ElviraDirector, Servei Català de la Salut (CatSalut)

Brazil’s landscape for the recognition of rare diseases and the evaluation of orphan drugs to ensure access and continuous supply Leandro Safatle

Secretário-Executivo, Câmara de Regulação do Mercado de Medicamentos (CMED), ANVISA

Access and pricing for drugs in Medicaid: how to reform the program to address both priorities Jeff Myers

President & CEOMedicaid Health Plans of America

Belgium, Netherlands, Luxembourg and Austria, collaborating in horizon scanning, HTA, pricing and reimbursement to ensure patients’ access to expensive medicines

Francis ArickxHead of the directorate Reimbursement of Medicines and Pharmaceutical Policy, National Institute for Health and Disability Insurance (NIHDI RIZIV/INAMI), Belgium

Ensuring fair pricing of pharmaceuticals and looking into the Canadian landscape for highly priced medicines

Tanya PotashnikDirector, Policy and Economics Analysis Branch, Patented Medicine Prices Review Board, Government of Canada

Developing new payment and alternative financing models to ensure higher value and better outcomes for the money spent in highly priced medical products Gregory Daniel

Deputy DirectorDuke-Margolis Center for Health Policy





VC Panel: Investing in the orphan drug space – derisking commercial models and driving biotechs to successful launchesModerated by Silver Sponsor

Kush ParmarManaging Partner5AM Ventures

David MottGeneral Partner NEA

Brian BronkHead of External Innovation, Rare Diseases Sanofi

David BonitaPrivate Equity PartnerOrbiMed Advisors

PITCH & PARTNER Funding is the common and constant gap impeding more rare disease therapies to be developed. Often ‘funding’ comes in the form of co-development, partnering, grants and licensing opportunities that can transform a promising pre-clinical candidate into a life-saving therapy for rare disease therapies.

At Pitch & Partner, we provide a platform for SMEs rare disease biotechs to present their clinical programs to potential investors, while these showcase their own criteria and risk-assessment frameworks.

Case study: Enzyvant’s approach to developing RVT-802, a novel tissue-based therapeutic for complete DiGeorge Syndrome Alvin Shih

Chief Executive OfficerEnzyvant Therapeutics

Case Study: What Sucampo’s acquisition by Mallinckrodt Pharmaceuticals tells about investing in ultra-rare diseases with unmet medical needs Hugh M. O’Neill

President, Autoimmune & Rare Disease Mallinckrodt Pharmaceuticals

ROUNDTABLE: Unorthodox funding strategies - alternate but complimentary paths to funding orphan drug companies Ken Kengatharan

Managing General Partner Atheneos Ventures

Maximizing the impact of public investment to accelerate rare disease therapeutic development

Nora YangDirector, Portfolio and Project Management Strategic Operations, NCATS, NIH

VC funding

Ari BrettmanPrincipal, Clarus Ventures

Pitch & Partner presenters include:

Jay MadanFounder & PresidentInnovate Biopharmaceuticals

Bill HornungCBODiffusion Pharmaceuticals

Chandler D. RobinsonChief Executive OfficerMonopar Therapeutics Inc

Igor LokotChief Executive OfficerDouble Bound Pharmaceuticals

Christer RosénChief Executive OfficerJupiter Orphan Therapeutics

Stephen FranklinChief Executive OfficerEvgen Pharma

Saira BatesChief Executive OfficerEscend Pharmaceuticals

Martin WelschofChief Executive OfficerOpsona Therapeutics

Philippe GuedatCEO and FounderInflectis Bioscience

Ray TakigikuCEOBexion Pharmaceuticals

Laxminarayan BhatCEOReviva Pharmaceuticals

Alan RobertsonChief Executive OfficerAlsonex Pharmaceuticals

Brian SchwartzChief Medical OfficerArQule

Donnie McGrathHead of Corporate Strategy & Business DevelopmentBiohaven Pharmaceuticals

Joseph E. PayneFounder & CEOArcturus Therapeutics

Philipp NovakCEO & FounderOrphanix GmbH

Eric LiChief Scientific OfficerLuxena Pharmaceuticals

Lynnet KohCEOTargazyme

Erik RichardsonResearch & Development Project LeadertAcucela

Matthew RitterHead of Business DevelopmentLa Jolla Pharmaceutical

David A. CoryChief Executive OfficerEiger Pharma

Fred JacobsChief Executive OfficerTYG Oncology

Franz ObermayrChief Executive OfficerPanoptes Pharma

Mehran MoghaddamChief Executive OfficerOROX Biosciences

Prasad GabbitaChief Executive OfficerP2D Bioscience

John LeeChief Medical OfficerPhase Bio

Richard MuruveChief Executive Officer, Director, Co-founderArch Biopartners

Roberto TonelliPresident, Chairman of the Board, Co-founderBiogenera

Aldar BourinbaiarChief Executive OfficerImmunitor

YANN LE CAMChief Executive OfficerEURORDIS

Lunch keynote address: Advanced therapies call for advanced collaborative processes to ensure patients access and market viability

PETER SALTONSTALLPresident and Chief Executive OfficerNORD

Lunch keynote address: Evaluating the incentives of the Orphan Drug Act to ensure sustainability and fair drug pricing

RARE DISEASE ADVOCACY WORLD

Empowered educated patients influencing pre-clinical development and working with industry to optimize drug discovery

Steven KamiskyChief Scientific Officer, International Rett Syndrome Foundation

Christopher MisslingCEO, Anavex Life Sciences

It is no news to the orphan drug industry that the rare disease patients and community have contributed incredibly to accelerate research and drug development. As their efforts evolve from patient enrollment and identification, the Rare Disease Advocacy World features what’s best in patient centricity put in practice.

Carole Ben-MaimonPresident & Chief Executive OfficerChondrial Therapeutics

Ron BartekPresidentFriedreich’s Ataxia Research Alliance (FARA)

Beyond “patient focused drug development” buzzwords- let’s get practical

Cross-functional education of internal stakeholders while maximizing patients’ opportunities through the product lifecycle Cristina Klafehn

Associate Director, Development Sciences Patient AdvocacyBiomarin

35 Years: Where have we come and where are we going

Pamela GavinChief Strategy Officer, NORD

Scaling up rare disease patient engagement in Europe

Yann Le CamChief Executive Officer, EURORDIS

Collaboration of board members & rare disease non-profits Tricia Mullins

Board MemberNeuromuscular Disease Foundation (NDF)

The holy grail of rare disease research – Framework for effective engagement with patient groups in clinical trials

Pamela TenaertsExecutive DirectorClinical Trials Transformation Initiative (CTTI)

The post market patient and patient advocacy- Acknowledging that with approval, the work is not done

Kenneth HobbyPresident, Cure SMA

Patient-centered qualitative research to inform potential outcome measures for Angelman syndrome clinical trials

Meghan MillerProject Leader/Discovery Scientist, Rare Diseases, Roche

Community-based patient advocacy

Mariana FagnilliHead of Global Patient Advocacybluebird bio

Patient advocates taking a real stand in drug development: how the Cystic Fibrosis Foundation hired pharma to find a cure Preston Campbell

PresidentCystic Fibrosis Foundation

PATIENT GROUPS SEMINARFollowing the steps and achievements of NORD and EURORDIS, patient advocacy organizations from other countries are centralizing their efforts or collaborating to advance their own policies, expand access and educate physicians, caregivers and patients about their rare diseases.

For the first time ever, 13+ patient organization from different countries come together to present their initiatives and outcomes to the global orphan drug industry.

#thinkSMALLactBIG – getting people to think about SMA and rare diseases, the SMALL ones who are affected by it, and to do their part to make a BIG difference

Sook Yee LimCo-founderWeCareJourney

Bulgarian Patients with rare diseases in focus of World and EC orphan drug development

Vladimir Tomov ChairmanNational Alliance of People with Rare Diseases in Bulgaria

Challenges of patients with rare diseases in India and recent progress

Harsha RajasimhaCo-FounderOrganization for Rare Diseases, India

Rare Diseases Sweden – Challenges and opportunities

Beata Ferencz Project ManagerRare Diseases Sweden

State of the art for the rare people in Denmark - Europa

Birthe Byskov HolmPresidentRare Disorders Denmark

Overcoming the lack of medical infrastructure, research and support to improve the quality of life of rare disease patients in South Africa

Kelly du PlessisChief Executive OfficerRare Diseases South Africa

The challenges of Brazil’s third sector in the participation of health promotion, access and regulation of public policies in rare and chronic diseases

Gustavo San MartinExecutive DirectorAME - Amigos Múltiplos pela Esclerose

The global challenges of rare diseases and the role of China (2016)

Kevin HuangPresidentChinese Organization for Rare Disorder

Reviewing dynamics of public policies for orphan drugs in Latin America – perspectives of patients and access situation

Angela ChavezPresidentColombia Rare Disorders Society (FECOER)

Making rare and intractable diseases an Asian health priority

Yukiko NishimuraPresidentASrid Japan

Resetting the clock: Giving patients and families a voice in developing innovative therapies for rare diseases

Alastair KentAmbassadorGenetic Alliance UK

The situation with rare diseases and patients’ organizations in Russia. Local legislation and it pitfalls, sharing experience

Denis BelyakovChief Executive OfficerUnion of Patients’ and Patients’ Organizations with Rare Diseases in Russia

Is there a roadmap to rare diseases and orphan drugs in Canada?

Durhane Wong-RiegerPresident & Chief Executive OfficerCanadian Organization for Rare Disorders

Rare diseases in a universal healthcare system – experiences in medical treatments and rehabilitation from patients’ perspective

Katri AsikainenChairpersonHARSO Rare Diseases Alliance of Finland

PATIENT GROUPS SEMINAR AGENDA


INTERNATIONAL PATIENT GROUPS SEMINAR

DAY 1 – APRIL 26TH, 2018

CANADA: Is there a roadmap to rare diseases and orphan drugs in Canada?

Durhane Wong-Rieger, President & Chief Executive Officer, Canadian Organization for Rare Disorders

10:30

SOUTH AFRICA: Overcoming the lack of medical infrastructure, research and support to improve the quality of life of rare disease patients in South Africa

Kelly du Plessis, Chief Executive Officer, Rare Diseases South Africa

10:50

COLOMBIA: Reviewing dynamics of public policies for orphan drugs in Latin America – perspectives of patients and access situation

Angela Chavez, President, Colombia Rare Disorders Society (FECOER)

11:10

BRAZIL: The challenges of Brazil’s third sector in the participation of health promotion, access and regulation of public policies in rare and chronic diseases

Gustavo San Martin, Executive Director, AME - Amigos Múltiplos pela Esclerose

11:30

INDIA: Challenges of patients with rare diseases in India and recent progress

Harsha Rajasimha, Co-Founder, Organization for Rare Diseases, India

11:50

CHINA: The global challenges of rare diseases and the role of China (2016)

Kevin Huang, President, Chinese Organization for Rare Disorders

12:10

JAPAN: Collaboration with/among multi-stakeholders as an intermediary organization in Japan

Yukiko Nishimura, President, ASrid Japan

12:30


FINLAND: Rare diseases in a universal healthcare system – experiences in medical treatments and rehabilitation from patients’ perspective

Katri Asikainen, Chairperson, HARSO Rare Diseases Alliance of Finland

2:30

DENMARK: State of the art for the rare people in Denmark - Europa

Birthe Byskov Holm, President, Rare Disorders Denmark

2:50

SWEDEN: Rare Diseases Sweden – Challenges and opportunities

Beata Ferencz, Project Manager, Rare Diseases Sweden

3:10

Resetting the clock – giving patients and families a voice in developing innovative therapies for rare diseases

Alastair Kent, Ambassador, Genetic Alliance UK

3:30

BULGARIA: Bulgarian patients with rare diseases in focus of World and EC orphan drug development

Vladimir Tomov, Chairman, National Alliance of People with Rare Diseases in Bulgaria

3:50

RUSSIA: The situation with rare diseases and patients’ organizations in Russia. Local legislation and it pitfalls, sharing experience

Denis Belyakov, Chief Executive Officer, Union of Patients’ and Patients’ Organizations with Rare Diseases in Russia

4:10

MALAYSIA: #thinkSMALLactBIG – getting people to think about SMA and rare diseases, the SMALL ones who are affected by it, and to do their part to make a BIG difference

Sook Yee Lim, Co-founder, We CARE Journey

4:30

END OF SEMINAR4:50

Collaboration between patient advocacy groups and industry - creating a lasting partnershipAnne Bruns, Executive Director, The Atypical HUS Foundation

Advancing Choroideremia research and therapy development through industry partnershipsChris Moen, President, Choroideremia Research Foundation

12:10


Building community advocacy and patient engagement for individuals with bleeding disorders to educate legislators, care providers and other stakeholders

Susan Fenters Lerch, Executive Director, The Hemophilia Foundation of Michigan

2:00

Driving treatments in Hyperoxaluria through collaborations with industry, FDA and patients

Kim Hollander, Executive Director, Oxalosis and Hyperoxaluria Foundation

2:20

Engaging the patient community to drive innovative and solution based collaborations to cure Alpha-1 Antitrypsin Deficiency

Henry R. Moehring, President and CEO, Alpha-1 Foundation

3:20

New born screening and registries, a foundation’s approach to change the future

Terri Klein, Interim President and CEO, National MPS Society

3:40

HIStories: sharing the patient experience to advance the scientific understanding of congenital hyperinsulinism

Julie Raskin, Executive Director, Congenital Hyperinsulinism International

4:00

END OF SEMINAR4:20

PATIENT ADVOCACY ORGANIZATIONS

Following the steps and achievements of NORD and EURORDIS, patient advocacy organizations from other countries are centralizing their efforts or collaborating to advance their own policies, expand access and educate physicians, caregivers and patients about their rare diseases.

For the first time ever, 13+ patient organization from different countries come together to present their initiatives and outcomes to the global orphan drug industry.

For the second day of the conference, patient groups from the US discuss the different initiatives that have enabled them to advance scientific research and benefit their communities including the tight work with industry stakeholders.

DAY 2 – APRIL 27TH, 2018

Advancing Choroideremia research and therapy development through industry partnerships

Chris Moen, President, Choroideremia Research Foundation

10:50

Roadmap for collaboration – accelerating the development of and access to new and innovative treatments for systemic amyloidosis

Isabelle Lousada, Chief Executive Officer, Amyloidosis Research Consortium

11:10

Using the power of community to advance treatments in Usher syndrome

Mark Dunning, Chairman, Usher Syndrome Coalition

11:30

Achieving our goals together – caregivers and patients’ partnerships with industry to advance Huntington’s disease therapies

Catherine Martin, Executive Director, The Huntington’s Disease Youth Organization

Bryan Viau, Chairperson, The Huntington’s Disease Youth Organization

11:50

Using a private digital platform to accelerate R&D - enabling patients to collect, share and monetize their data

Jeanne Barnett, President and Founder, Cysticfibrosis.com

2:40

Inspiring the rare disease community with hope through the power of sport – an athlete’s rare disease story

Rob Long, Executive Director, Uplifting Athletes

3:00



THE EARLIER YOU BOOK, THE MORE YOU SAVE!Register now at www.terrapinn.com/orphandrugsUSA

CONFERENCE DAY ONE (Thursday, April 26th, 2018)

1/8 2/8

Opening remarks – Scott Gray, Chief Executive Officer, Clincierge8:20

Keynote address: Cell and gene therapy innovation and their role in the future of rare disease research

• Current state of cell and gene therapies and why this field is becoming more and more significant • Looking into scalability and centralized approaches for cell and gene therapy development and commercialization• Novel approaches and how these therapies benefit the continuum of research for rare diseasesHans Bishop, President & Chief Executive Officer, Juno Therapeutics

8:55

Keynote CEO Panel: Gene therapy - a new paradigm for patients, new challenges for industry

• Looking back at the commercial performance of the first generation of gene therapies- from proof of concept to the road to approval • How will gene therapy innovation look in the next 10 years?• Considering potential new value propositions for patients, payers and the health care system• Strengthening clinical development, exploring novel technologies while maintaining safety- what needs to be improved

Moderated: Jude Samulski, Vice President, Gene Therapy, Pfizer Rare Disease

Tim Miller, Chief Executive Officer, Abeona Therapeutics

Mark Rothera, Chief Executive Officer, Orchard Therapeutics

Ken Mills, Chief Executive Officer, REGENXBIO

Frederic Revah, Chief Executive Officer, Genethon

Karen Aiach, Founder, Chief Executive Officer, Lysogene

9:45

Keynote address: Next- generation CAR T-cells to cure cancer - paving a new way for R&D, value-assessment and pricing and reimbursement of treatments for genetic diseases

• Learnings from CAR-T development that can be applied to rare disease drug development • Will CAR therapy pricing impact pricing schemes of other orphan drugs and gene therapies? • Exploring aspects of these different therapeutic areas that share common goals, personalization and pricing challenges André Choulika, Chairman & Chief Executive Officer, Cellectis

9:20

Keynote address: A new era in medicine- introducing gene therapy

• What we’ve learned – lessons science has taught us as we create the path for the first gene therapy for an inherited disease in the U.S.• Staying true to our roots while preparing the patient, medical and policy maker communities for a new chapter in the retinal disorders

textbook• Assessing the impact of potential one-time treatments like gene therapies for patients with rare diseasesKatherine High, President and Head, Research & Development, Spark Therapeutics

8:30

A NEW ORPHAN DRUG ERA

Clinical Development & Regulatory Track

Gene TrackCommercial TrackRare Disease Advocacy

WorldGlobal Market Access

TrackPitch & Partner

Access & TrialsGenetic Testing,

Genome SequencingBusiness Model

Influencing Drug Development

Established MarketsInvestment landscape

& 10min Biotech Pitches

The evolution of a partnership to help decision making and improve patient access to investigational medicines

• Exploring ethical issues surrounding access to investigational products before FDA approval

• Working with patient groups to help demystify pre-approval access and create community-specific resources

• Developing a group approach to ethical decision medicine – pharma and ethicists working together

Beverly Harrison, Head, Patient Support, Janssen Office of the Chief Medical Officer, J&J

Alison Bateman House, Assistant Professor, Department of Population Health, NYU Langone Health

Genetic testing and patient engagement: faster time to diagnosis and access to research and treatments

• Removing barriers to genetic testing can help patients secure an accurate diagnosis more quickly

• Genetic testing programs drive patient identification, expedite trial enrollment and accelerate time to market for new therapeutics

• Creates engaged patient communities that partner biopharma and patient advocacy groups through Patient Insights Networks (PIN)

Moderator:

Daniel Anderson, Head of Commercial Partnerships, Invitae

Catherine Pajak, Senior Director, Marketing, North America, Lysosomal Storage Disorders, BioMarin Pharmaceutical

Jordanna Mora, Associate Director, Patient Advocacy & Engagement, Alnylam Pharmaceuticals

Mark Dant, Founder and Executive Director, The Ryan Foundation

Collaborative mission-driven approach to developing innovative potential new treatments for unserved and underserved patient communities

• Addressing Industry challenges of too much good science and insufficient resources to fully advance pipelines

• Aligning biopharma innovators with investigators, patient groups, and funders to advance promising investigational therapies

• Concept in Action: Big Pharma expertise and capabilities leveraged to enable company launch pipeline of four mid- and late -stage clinical assets, enabling strong Series A funding

Lara Sullivan, President and Founder, SpringWorks Therapeutics

Empowered educated patients influencing pre-clinical development and working with industry to optimize drug discovery

• Patient groups role guiding research, disease understanding, supporting pre-clinical development and follow up experiments

• Driving translational research and the influence on formulation, routes to delivery and overall clinical development

• Effective partnerships between advocacy and industry to expand collaborations that can support enrollment and physician engagement

Steven Kamisky, Chief Scientific Officer, International Rett Syndrome Foundation

Christopher Missling, Chief Executive Officer, Anavex Life Sciences

CANADA: Orphan drug evaluation and decision making in Canada in the context of international HTA decisions

• Orphan drug evaluation at CADTH to reimburse, reimburse with clinical criteria/conditions and not reimburse

• HTA challenges when there is no regulatory framework for orphan drugs in Canada and no international consensus for OD

• Balancing evidence requirements with treatment’s cost

Trevor Richter, Director, Common Drug Review and Optimal Use of Drugs, CADTH

VC Panel: Investing in the orphan drug space – derisking commercial models and driving biotechs to successful launches

• Partnering with key KOLs and advocacy groups at the outset to help facilitate a physician/patient company dynamic to help inform both regulatory and commercial strategy

• Type of work and diligence to obtain comfort with the ultimate market opportunity

• Assessing barriers to entry including therapeutic modality and IP

• Regulators role in incentivizing or impeding the ability to develop products both domestically and abroad. What can be different?

Moderated by Silver Sponsor

Kush Parmar, Managing Partner, 5AM VenturesDavid Mott

General Partner, NEA

David Bonita, Private Equity Partner, OrbiMed Advisors

Brian Bronk, Head of External Innovation, Rare Diseases, Sanofi

11:30

The virtual opportunity in rare disease trials

• Solving specific challenges with virtual trials and their supporting technologies

• Reducing patient burden through siteless data capture and evidence generation

• Exploring the promise and pitfalls of telemedicine

Karen Kaucic, Senior Vice President, Early Development, Rare Disease and Pediatric Center of Excellence, PPD

Navigating the rare disease innovation ecosystem and partnering for the long-term

• Looking into new technology platforms and transformative new products

• Nurturing relationships with academia, incubators, startups and biotechs to develop fruitful partnerships

• From due diligence to alliance management, realizing the potential of scientific innovation, and going beyond ROI

Gregory Fond, Director, External Innovation, Rare Diseases, Sanofi

Patient advocates taking a real stand in drug development: how the Cystic Fibrosis Foundation hired pharma to find a cure

• Research funds and initiatives coming from advocacy and not from pharma

• Uniting the CF community with drug developers through transparency and the pursuit of a common goal

• Fueling motivation from all the involved parties to continue with the co-development of drugs

• What is the applicability of this model to other rare diseases?

Preston Campbell, President, Cystic Fibrosis Foundation

ITALY: The Italian experience on market access for rare disease drugs

• What is needed to achieve regional access? How does Italy compare to other European countries?

• Looking into new rules to gain ‘innovative status’

• Opportunities to market access through a more formalized process for early access and compassionate use requests

Martine Zimmermann, Executive Director Global Regulatory Affairs, Alexion

11:55

SPEED NETWORKING FOLLOWED BY MORNING NETWORKING BREAK 10:30


Expediting rare disorder patient identification and a genetic testing-based diagnostics ecosystem to drive orphan drug adoption

• Development of sustainable diagnostic systems to help find rare disease patients pre-and post-approval

• Molecular technologies for prenatal, newborn and adult testing and what this means in cost-savings for pharma

• Educating and enabling physicians to recognize and diagnose rare diseases

If you’re interested in sponsoring this session contact André Singer at [email protected] or +1 646 619 1797

The role of commercial in early development through launch

• Leading market access strategies, KOL development, engagement and mapping out all scenarios for a drug’s entire life cycle prior to launch

• Driving lifecycle expansion into other indications and geographies

• Managing and growing global commercial teams in rare disease

Alex La Croix, enior Director, Rare Genetic Disease Marketing, Agios Pharmaceuticals

Inclusion of patient reported outcomes (PROs) in rare disease trial design

• Capturing patients’ bona-fide needs, preferences, and value drivers through systematic investigation

• Adoption of end- to-end patient evidence generation pathways to increase the probability of success

• Enabling payers and providers to better understand patients’ values

• Developing meaningful patient reported outcomes and producing further evidence to support approval

If you’re interested in sponsoring this session contact André Singer at [email protected] or +1 646 619 1797

Market access challenges and opportunities for orphan and ultra-orphan drugs

• Looking into the global demand for an evidence-based approach toward orphan drug pricing and reimbursement

• Current landscape on pharmaceutical practices: undertaking burden of Illness studies, focusing on baseline quality of life, and direct and indirect costs to evaluate orphan drugs and assist with reimbursement

• Tools to overcome the challenges faced by industry when identifying relevant data sources to document the cost and burden of orphan diseases

• Strengths and limitations of available methods of data capture

Thomas Goss, Senior Vice President, Boston Healthcare Associates

Validive for the treatment of severe oral mucositis

Chandler D. Robinson, Chief Executive Officer, Monopar Therapeutics Inc

JOTROL™ for treatment of MELAS syndrome, Friedreich’s ataxia and MPS I

Christer Rosén, Chief Executive Officer, Jupiter Orphan Therapeutics

12:40

12.50

Early development planning for orphan drugs: tasks, timelines, and takeaways

• Organizing orphan drug designations: Where do they fit in and how long do they take?

• Understanding natural history studies: purpose, design, and obtaining meaningful data

• Preparing for an IND/IMPD and related clinical studies

Angi Robinson, Executive Director, Strategic Development, Rare Diseases & Pediatrics, Premier Research

Validating and optimizing NGS-based diagnostics to improve diagnostic yield and clinical utility

• Challenges in the field of genetic diagnostics and current technologies used to address them

• Introducing the concept and importance of transparent, traceable, and comprehensive analytic validation of an NGS platform for clinical diagnostics

• Patient examples which demonstrate the importance of high quality and comprehensive genetic diagnostics for improved outcomes for rare disease patients

• Controlling costs by utilizing high quality WES derived sequencing data for rare disease patient

Tero-Pekka Alastalo, Chief Medical Officer, President, and co-founder, Blueprint Genetics

Development and execution of commercial strategies for clinical and commercial stage RNAi products for serious, life-threatening diseases

• Pre-launch strategies and building a rare disease commercial team from scratch

• Launching successfully: different challenges, different approaches, same result

• Achieving market access by placing a focus on prescribers, patients and payers

• Global vs. local strategies

Mark Baglin, Vice President of Global Marketing, Alnylam Pharmaceuticals

The post market patient and patient advocacy- Acknowledging that with approval, the work is not done

• How can patient groups keep encouraging orphan disease research and investments after approval?

• Rare disease clinical development when approved treatments are available.

• Opportunities to reduce delays to access prior to approvals

• Post-marketing engagement to improve and expand patient access – engaging with multiple new stakeholders.

• Working synergistically with industry counterparts to educate the physician community, delivery centers, and payers.

Kenneth Hobby, President, Cure SMA

BENELUX-A Belgium, Netherlands, Luxembourg and Austria joining forces to improve access and to orphan drugs and paving the way for more collaborations

• Joint collaboration to negotiate with pharmaceutical companies the price of orphan drugs

• Looking into data exchange, sharing of registries and coordinated assessment of new technologies and innovation

• Advantages in patient access and dossier submission for pharmaceutical companies

Diane Kleinermans, Advisor to the Ministry of Health and Social Affairs, Belgium Ministry of Public Health and Social Security

Case study: Enzyvant’s approach to developing RVT-802, a novel tissue-based therapeutic for complete DiGeorge Syndrome

• Leveraging expedited regulatory pathways, including breakthrough designation and Regenerative Medicine Advanced Therapy (RMAT) designation

• Collaborating with academic partners

• Fostering advocacy relationships and aligning corporate and patient priorities

• Developing a commercial model for a transformative, one-time therapy in the pre-launch phase

Alvin Shih, Chief Executive Officer, Enzyvant Therapeutics

12:20


NETWORKING LUNCH RESUMES1:50

Breakthrough therapies call for breakthrough processes to ensure sustainable patient access

Yann Le Cam, Chief Executive Officer, EURORDIS

1:30

3/8 4/8IRDiRC goals 2017-2027 – new research goals to accelerate rare disease therapies and diagnostics globally

• Spearheading patient diagnosis by having currently undiagnosable individuals enter a globally coordinated diagnostic and research pipeline

• Working toward the approval of 1000 new rare diseases therapies focused mainly on diseases without approved options

• Development of methodologies to assess the impact of diagnoses and therapies on rare disease patients

Christopher Austin, Chairman, International Rare Disease Research Consortium


ROUNDTABLES (1-hour roundtable sessions split between 2 rounds of 30min – attendees can select two roundtables to attend)

2:30

ROUNDTABLE 1: Pediatric trials - reducing the financial, logistical and emotional burden for patients and families participating in pediatric trialsPat Furlong, Founding President and Chief Executive Officer, Parent Project Muscular Dystrophy (PPMD)

ROUNDTABLE 2: Compassionate Use Programs - helping patients get access to medication, educating the community, physicians and investors to ensure safe access to experimental treatments while staying true to corporate strategy Andrew McFadyen, Executive Director, The Isaac Foundation

Temodex, a locally acting form of temozolomide, for treatment of glioblastomaIgor Lokot, Chief Executive Officer, Double Bound Pharmaceuticals








END OF ROUNDTABLE SESSIONS 3.30

ROUNDTABLE 7: Gene therapy - new payment and financing models are needed to facilitate accessFrancesca Cook, Director, Pricing and Market Access, REGENXBIO

ROUNDTABLE 9: Managed Access Programs - How MAPs (pre-commercial supply to patients with unmet medical needs) can be a valuable component of launch strategy for orphan drugs Robert Donell, Director of Business Development, Durbin

ROUNDTABLE 5: BD & Forecasting - pricing and access considerations and understanding how to build the patient forecast based on available epidemiological information Joseph B. Musumeci, Senior Advisor, BluePrint Orphan

ROUNDTABLE 11: Scientific innovation - platform approaches to evaluate multiple genetic diseases at the same timePhilip J. Brooks, Program Director, Office of Rare Diseases Research and Division of Clinical Innovation, NCATS/NIH

ROUNDTABLE 13: Government affairs - developing a company’s footprint and contributions to impact policy, reimbursement and patient engagementBetsy Ricketts, Senior Director, Government Affairs, Ultragenyx Pharmaceutical

ROUNDTABLE 8: Clinical development - When the sum is greater than the parts through academic, pharmaceutical and CRO collaboration in orphan disease drug development Michael Murphy, Chief Medical and Scientific Officer, Worldwide Clinical Trials

ROUNDTABLE 10: Gene therapy- Optimizing gene therapy clinical development through education, collaboration, patient input and involvement in advisory boardsSamantha Parker, Chief Patient Access Officer, Lysogene

ROUNDTABLE 6: Strategy - patient engagement and pipeline building for the commercialization and access of rare disease productsYvette Venable, Global Head of Public Affairs, Kyowa Kirin Pharmaceutical

ROUNDTABLE 12: Epidemiology and forecasting - using epidemiological data to determine market viability, niche populations and commercial differentiation David Lapidus, President, Lapidus Data

ROUNDTABLE 14: Patient identification - realizing the full potential of machine learning algorithms to search for undiagnosed patients with rare diseasesNadea Leavitt, Predictive Analytics Principal, Real-World Insights, IQVIA

Stephanie Roy, Principal, Real-World Evidence Solutions, IQVIA

A bis-benzylisoquinoline alkaloid for use with chemotherapy for refractory and relapsed acute myeloid leukemia (AML)Saira Bates, Chief Executive Officer, Escend Pharmaceuticals

2:50

OPN-305, A first in class toll like receptor 2 (TLR2) antibody inhibitorMartin Welschof, Chief Executive Officer, Opsona Therapeutics

3:00

IFB-088 a potential new therapeutic option to treat demyelinating charcot-marie-tooth diseasesPhilippe Guedat, Chief Executive Officer and Founder, Inflectis Bioscience

3:10

5-(4-carboxymethyl-phenylazo)-2-hydroxybenzoic acid disodium salt (INN-108) for treatment of ulcerative colitis in pediatric/adult patientsJay Madan, Founder & President, Innovate Biopharmaceuticals

3:20

ROUNDTABLE 15: Expanded Access – drug development strategy considerations for open label extension or expanded access protocolsAnne B. Cropp, Chief, Scientific Officer, Early Access Care, LLC

ROUNDTABLE 16: Disease awareness - best practices and tips for smaller companies to tell a corporate or rare disease story and raise awarenessTrista Morrison, Vice President, Communications, North America, Sobi

ROUNDTABLE 3: Virtual Trials - enabling clinical trials to be more virtual, reducing distance times, overcoming geographic barriers and increasing data collection through the use of use of wearablesScott Schliebner, Vice President, Scientific Affairs, Rare Diseases, PRA Health Sciences

ROUNDTABLE 4: Expanded access - incorporating the patient advocacy perspective to develop a review process and a formalized procedure on expanded access Jayne Gershkowitz, Senior Vice President and Chief Patient Advocate, Amicus Therapeutics

SFX-01 for the treatment of subarachnoid haemorrhageStephen Franklin, Chief Executive Officer, Evgen Pharma

2:40

ROUNDTABLE 17: VC funding – successfully addressing investors interests and challenges when assessing companies of all sizes to guarantee funding of orphan drug biotechsAri Brettman, Principal, Clarus Ventures


5/6Clinical Development &

Regulatory TrackGene TrackCommercial Track

Rare Disease Advocacy World

Global Market Access Track

Pitch & Partner

Data, Big Data and Real-World Evidence

Clinical & Manufacturing

Forecasting Let’s get practicalEmerging Markets Biotech Pitches

From real world data to real world evidence: FDA’s perspective on the enhanced use of RWE

• RWE goals to have regulatory decisions incorporate data/evidence from settings that more closely reflect clinical practice

• RWE expectations within 21st Century Cures and PDUFA VI

• FDA’S experience with RWE, collaborations and the assessment of data fitness and standards

• Impact of the inclusion of RWE in regulatory decision-making and the assessment of orphan drugs

Jacqueline Corrigan-Curay, Director, Office of Medical Policy, Center for Drug Evaluation and Research, FDA

Creating value in gene therapy clinical development programs, achieving trial optimization and supporting the path to commercialization

• Investigating novel vector technologies that can improve efficacy while reducing immunogenicity and potential re-dosing

• Thinking about end to end clinical development including trial design, enrollment, execution, supply chain and manufacturing

• How can clinical development be optimized to support scalability? What are the current limitations faced by this field despite a decade of improvements?

Olivier Danos, Chief Scientific Officer, REGENXBIO

Value Assessment is different for Rare Diseases, requiring new thinking to build consensus around access decisions

• Addressing divergent priorities from stakeholders in response to potentially curative gene and cell therapies, including physicians, regulators, patients, payers and manufacturers

• Unblocking the affordability conundrum by bringing stakeholders together to rethink the measurement of value and how this impacts wider societal and economic factors

• The need for tailored planning that differs from more mainstream disease areas given the very distinct role that patients and advocacy groups play in rare diseases

Walter Colasante, Vice President, Charles River Associates

Beyond “patient focused drug development” buzzwords - let’s get practical

• Industry and patient partnerships for clinical trial design and execution, funding discussions and community outreach

• Patients as board members through the product lifecycle to impact the needs of the community

• Partnering with patients in a compliant way and achieving support from government and academic institutions to drive scientific research

Carole Ben-Maimon, President & Chief Executive Officer, Chondrial Therapeutics

Ron Bartek, President, Friedreich’s Ataxia Research Alliance (FARA)

AUSTRALIA: Orphan drug program reforms: changes in the eligibility criteria of orphan drug designations

• Expansion of the orphan disease prevalence threshold to allow more conditions to classify as orphan

• Changes including the validity period of orphan drug designation before seeking approval, fee waivers, and additional criteria

• Transition period considerations for sponsors

Kaye Robertson, Assistant Director Prescription Medicines Clinical Evaluation Unit 3, Therapeutic Goods Administration (TGA)

RP5063: A novel investigational drug to treat pulmonary arterial hypertension (PAH)

Laxminarayan Bhat, Chief Executive Officer, Reviva Pharmaceuticals

BXQ-350, a novel approach for the potential treatment of glioblastoma multiforme

Ray Takigiku, Chief Executive Officer, Bexion Pharmaceuticals

ALS205 for the treatment of amyotrophic lateral sclerosis (ALS)

Alan Robertson, Chief Executive Officer, Alsonex Pharmaceuticals

3:40 3:30

3.40

3.50

6/8




Real World Evidence 2.0 – from basic evidence collection to its inclusion in drug review applications

• What is usable Real World Evidence?

• Simplifying collection, incorporating data into drug applications, and leveraging its potential to drive drug effectiveness

• What are the necessary steps to incorporate RWE in the regulatory approval process? How can these be incorporated early on?

Achieving patient retention in gene therapy follow up

• Strategies for optimizing patient retention throughout gene therapy programs (Phase 1-3, follow-up studies, registries)

• New ways to better understand and navigate patient challenges first hand (logistical hurdles presented by moving from the initial “treatment” site/country (HCP) to potentially new follow-up sites/HCPs)

• Gene therapy trials vs. other trials and what needs to be considered

Mariah Baltezegar, Executive Director, Clinical Development, Rare Diseases, Syenos Health

What drives orphan drug commercial launch success?

• Launch excellence: how orphan drugs performance is associated with pre-launch preparedness and developed commercial awareness

• Performance: the impact of multiple levels of orphan drug differentiation and unmet medical needs

• Not all orphan drugs are the same – how to leverage pre-approval initiatives, forecasting and epidemiology for commercial success

Adam Sohn, Vice President, Consulting Services, IQVIA

Simone Seiter, Vice President, Global Lead Launch Excellence, IQVIA

Collaboration of board members & rare disease non-profits

• NDF strategic partnerships to expand research Successful completion of International Symposium 2017

• Certification of NDF Patient Advocates

• Growing advocacy initiatives globally Improving education and awareness

• Best practices of Board Members

Tricia Mullins, Board Member, Neuromuscular Disease Foundation (NDF)

TURKEY: Access to orphan drugs in a large market outside the European Union

• Legislative efforts to define rare diseases and promote the development and commercialization of orphan drugs

• Channels available to obtain unapproved drugs in Turkey but available in the EU (off-label use, named- patient imports, compassionate use)

• Working towards establishing national networks for the prevention, surveillance, treatment of rare disease

Ayse Deniz Ozger, Vice President, Healthcare Division, Eczacibasi

4:20

A novel mechanism for treating ipf using small molecules

Mehran Moghaddam, Chief Executive Officer, OROX Biosciences

Hepcidin for treatment of iron overload disorders

Matthew Ritter, Head of Business Development, La Jolla Pharmaceutical

4:20

4:30

Filling post market evidence gaps to support approval decisions and reimbursement

• Thinking about post approval data collection before approval

• Designing new studies and looking into real world evidence, patient reported outcomes and natural history studies

• Developing an evidence strategy that assesses the current reimbursement, regulatory and commercial landscape

Catherine Stehman-Breen, Senior Vice President and Chief Medical Officer, Sarepta Therapeutics

Gene editing approaches in rare blood disorders

• Unmet need for transformative therapies in hemoglobinopathies

• Disrupting an erythroid specific BCL11A enhancer with zinc finger nucleases for the treatment of sickle cell disease and beta-thalassemia

• The potential to reactivate fetal hemoglobin to treat patients with SCD and beta-thalassemiai

Dana Levasseur, Associate Director, Research, Bioverativ

Spotlight on ultra-rare: commercializing therapies for very small patient populations

• Lessons from the development and launch of Mepsevii for a disease affecting <200 patients

• Accelerating approval, finding the patients, building a value story, and assuring access

• Commercialization challenges posed in the ultra-rare- versus rare-disease space.

Gary Geipel, Senior Director, Global Brand Communications, Ultragenyx Pharmaceutical

The holy grail of rare disease research – Framework for effective engagement with patient groups in clinical trials

• Understanding current practices in patient groups-sponsors collaborations

• Filling knowledge gaps regarding best practices for patient groups engagement in every phase of drug development

• From fundraising to trial operations to helping with access- understanding the ecosystem

Pamela Tenaerts, Executive Director, Clinical Trials Transformation Initiative (CTTI)

BRAZIL: accelerating the review and approval of orphan drugs in Latin America’s largest market

• Resolution to establish a procedure for clinical trials consent and good manufacturing practices

• Guidelines for registration of new drugs for treatment, diagnosis or prevention of rare diseases

• What this means for patients, drug manufacturers and the healthcare system

Jarbas Barbosa da Silva Jr., Director-President, ANVISA

4:00

TZ 101/TZ 102: Transforming cancer patient survival and quality of life outcomes by improving the delivery of therapeutic cells to tumors and sites of inflammation

Lynnet Koh, Chief Executive Officer, Targazyme

Human rhodospin optogenetics for treatment of retinitis pigmentosa

Erik Richardson, Research & Development Project Leader, Acucela

4:00

4:10

AFTERNOON NETWORKING BREAK4:40

NETWORKING COCKTAIL PARTY – RESERVED FOR GOLD SPONSOR6:20

Keynote address: Fostering innovation in rare disease and advancing the development of drugs for the rarest of diseases

• Developing a global vision for the commercialization of rare disease therapeutics• Maintaining a clinical stage portfolio and working on nurturing pre-clinical stage programs• Working hand in hand with advocacy and policy makers to support rare disease development efforts

5:20

Keynote panel: How rare disease companies are ramping up innovation to boost orphan drug development and patient centricity

• Incorporating patients in the drug development process and working towards commercial viability- how to fully engage? • How do you view disruptive therapies such as (gene therapy/gene editing) impacting the broader orphan disease development paradigm? How

are you innovating within your company?• Responding to the changes in the political, regulatory and reimbursement landscape while guiding the direction of rare disease companies • How to achieve industry collaboration and idea exchanges that can better serve the patient communityFrançois Nader, Board Chairman, Acceleron Pharma (XLRN), Former President & Chief Executive Officer, NPS Pharma

Douglas Ingram, Chief Executive Officer, Sarepta Therapeutics

Daniel de Boer, Founder a Chief Executive Officer, ProQR Therapeutics

Timothy Walbert, Chairman, President and Chief Executive Officer, Horizon Pharma plc

5:40

INNOVATION

7/8 8/8





REGISTER NOWwww.terrapinn.com/orphandrugsusa

CONFERENCE DAY TWO

Opening remarks 8:30

Keynote address: Developing genomic medicines

• Gene therapy, genome editing, cell therapy, gene regulation: choosing the best therapeutic platform to address patients’ needs• Editing liver cells with ZFNs to potentially cure rare diseases, including MPS I, MPS II and hemophilia B• Zinc finger nuclease technology: Precision, Efficiency, Specificity -- what’s important when developing genomic medicines• Zinc finger nucleases as a best-in-class T-cell editing tool for cell-based therapies• The next frontier: what does the future of genome editing look like?Sandy Macrae, President and Chief Executive Officer, Sangamo Therapeutics

8:35

Keynote address: Advancing innovative therapies to treat genetically-defined diseases using CRISPR-based genome editing

• Momentum toward making medicines – a potentially transformative new category of medicines that aim to repair “broken” genes• Strategy focused on diseases with few or no available treatments and exploring a Regulatory framework for CRISPR-based medicines• New data demonstrating advancements for translating CRISPR technologies into medicines• Clinical natural history study of Leber Congenital Amaurosis to understand the disease course and potential clinical endpoints • Advancing toward the clinic with potential to transform therapy for retinal disease LCA10-CEP290Charlie Albright, Chief Scientific Officer, Editas Medicine

9:00

Keynote address: Translating CRISPR/Cas9 technology into transformative gene-based medicines for patients with serious diseases

• In vivo and ex vivo treatments using CRISPR/Cas9 for different therapeutic indications• CRISPR/Cas9 potential to correct DNA changes in somatic (non germ line) cells in patients with serious disease and the road to clinical trials• Partnerships with other pharmaceutical companies to develop gene-editing-based therapeuticsRodger Novak, Chief Executive Officer and Founder, CRISPR Therapeutics

9:25

The NIH Common Fund Somatic Cell Genome Editing Program: Developing tools and technologies to enable safe and effective genome editing in humans

• Overview of the NIH Common Fund Somatic Cell Genome Editing Program• Program components including creating better animal models, tools to detect adverse events, technologies to deliver genome editing and

developing capabilities for knowledge sharing • Looking into the future of genome editing technologies and strengthening academic and industry collaborations to achieve wider adoption Christopher Austin, Director, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH)

9:50

Proposed Value based frameworks for pricing and reimbursement of orphan drugs - can a new value framework help ease friction over orphan drug prices?

• Methods to recommend fair prices to allow for broader insurance coverage for innovative new treatments

• Different proposals for gene therapy pricing and possible routes for implementation

• Impact of willingness to pay thresholds in the US and the orphan market space

Rick Chapman, Director of Health Economics, ICER

Orphan drug designations in an era of novel technologies including CAR-T-cell, stem cells and gene therapy

• Case studies of recent FDA approvals with orphan drug designation

• Challenges for the newer modality of therapies such as CAR-T intended to treat diseases with higher prevalence including cancers

• Different requirements and challenges faced by pharmaceutical companies to obtain global orphan drug designations

Bob Desai, Vice President, Technical, PAREXEL International

TAK-celeratorTM - An incubator-like model to accelerate the development of transformative therapies in rare diseases • Taking assets through

translation into novel therapies utilising externalisation paths such as creation of NewCo’s or partner collaborations to increase the chance of reaching patients

• Internal and external funding opportunities and the benefits of a global pharma company

• Building mutually beneficial collaborations across academia, patient organisations and industry through a flexible working and agile style

Christine Charman, External Asset Lead, TAK-celerator, Centre for External Innovation (CEI), Takeda Pharmaceuticals

Community-based patient advocacy

• Different approaches to patient advocacy

• Health disparities and the role of stakeholders in complex healthcare ecosystems

• Interaction with patient advocacy groups and patient advocates

Mariana Fagnilli, Head of Global Patient Advocacy, bluebird bio

THE PHILIPPINES: How a newly passed Rare Diseases Act is unlocking an emerging market’s commercial potential

• Two years after the introduction of a Rare Disease Act covering registries, patient care, R&D, patient care and screenings

• Introduction of a national strategy specifically for rare disease management facilitating coverage and access

• How the Philippine society for orphan disorders pushed for the approval of this key legislation

Carmencita D. Padilla, Founding Chairman, Philippine Society for Orphan Disorders

Maximizing the impact of public investment to accelerate rare disease therapeutic development

• Private public partnerships with NCATS to bridge translational gaps – funding and technical expertise

• Establishing platform technologies at TRND to catalyze rare disease therapeutic development

• Exploring innovative approaches to unblock regulatory bottlenecks

Nora Yang, Portfolio and Project Management, Strategic Operations, NCATS, NIH

Drug pricing in Europe, improving the role of HTA agencies and looking into the sustainability of managed access agreements

• Looking into voluntary price joint price negotiations as a means to assess highly priced drugs and increase access

• Can HTA assessment be improved across European countries?

• What will be the future of managed access agreements as more drugs require them and more resources are needed? Can this model be replicated around Europe?

Edmund Jessop, Medical Advisor, NHS England

Pioneering partnerships between industry and patient groups to advance gene therapy research to treat inherited retinal diseases

• Supporting research initiatives like My Retina Tracker registry and studies to identify barriers that prevent patients use of genetic testing

• Leveraging partnerships and funding opportunities to increase gene therapy clinical trial enrollment

• Working toward common objectives and maximizing patient groups’ and industry’s capabilities

Sue Washer, Chief Executive Officer, AGTC

Ensuring commercial success through lifelong innovation: evolving alongside patients to meet their changing needs through the product lifecycle

• Reviewing opportunities for biopharma companies to continue listening to patients long after a drug has launched, particularly as pediatric patients become adults and their needs evolve

• Turning patient and physician insights into new formulations, new doses, and new storage options

• Going beyond scientific innovation to address changing patient needs through ambassador and other programs

Holly May, Vice President, Sales and Marketing, United States, Sobi

Cross-functional education of internal stakeholders while maximizing patients’ opportunities through the product lifecycle

• Creating a strategic plan to address patient advocacy across the spectrum of pharmaceutical functions

• Presenting opportunities for patients to engage with regulatory agencies at preclinical and clinical stages

• Incorporating the patient perspective to influence endpoints, recruitment, retention, etc.

Cristina Klafehn, Associate Director, Development Sciences Patient Advocacy, Biomarin

BRAZIL: Qualifying and standardizing the legal process for access to non-approved orphan drugs in Brazil

• Working towards better access for orphan drugs to surpass the common route of legal action by patients

• Navigating Brazil’s complex payer landscape and leveraging SUS’ guidelines for improved diagnosis and treatment

• Strategies for launching orphan drugs in LatAm’s most promising orphan drug market

Dr. Luiz Fernando Lima Reis, Director of Education and Research, Hospital Sírio-Libanês

Arnaldo Hossepian, Member, Conselho Nacional de Justiça (Brazil)

Case study: What Vtesse’s acquisition by Sucampo Pharmaceuticals tells about investing in ultra-rare diseases with unmet medical needs

Peter Greenleaf, Chairman and Chief Executive Officer, Sucampo Pharmaceuticals

11:40

11:15

Pricing & Reimbursement Track

Gene TrackCommercial TrackRare Disease Advocacy

WorldGlobal Market Access

TrackPitch & Partner

Value Based Reimbursement

Gene Therapies/Editing

Strategy Collaboration Emerging Case studies & 10min

Biotech Pitches

SPEED NETWORKING FOLLOWED BY MORNING NETWORKING BREAK10:15

(Friday, April 27th, 2018)

1/5 2/5

http://www.terrapinn.com/orphandrugsusa

Developing new payment and alternative financing models to ensure higher value and better outcomes for the money spent in highly priced medical products

• Adapting health system reimbursement models to encourage innovation in curative gene therapies and other innovative therapies

• Obstacles and opportunities for the increased implementation of value-based payment arrangements in the United States

• Gene therapies vs. other highly priced treatments- looking into the sustainability of the healthcare system

Gregory Daniel, Deputy Director, Duke-Margolis Center for Health Policy

Scaling up viral vector development and manufacturing for cell and gene therapies

• Quality, analytical, and validation characteristics unique to gene therapy products

• Early stage commercial planning to enable scale up

• In-house, outsourcing or hybrid models: adapting viral vector developing and manufacturing to your gene therapy program needs

• Selecting and managing external manufacturers effectively in a suppliers’ shortage scenario

Alain Lamproye, Chief Executive Officer, Yposkesi

Designing your distribution channel to optimize your orphan drug commercialization strategy

• Taking the right steps to maintain inventory control

• Ensuring access to patient site of care

• Increase speed to market through strong channel footprint

• Gain valuable business insights through data and analytics

Kevin Kissling, Vice President, 3PL Services, McKesson Specialty Health

Chad Pulliam, Direct, Channel Operations, Jazz Pharmaceuticals

Patient-centered qualitative research to inform potential outcome measures for Angelman syndrome clinical trials

• Identifying and developing the best outcome measures and biomarkers for future clinical trials

• Working with doctors and patients globally to determine what to measure, how to measure it, and how to interpret findings

• The Angelman Biomarkers and Outcome Measures Alliance (A-BOM)

Meghan Miller, Project Leader/Discovery Scientist, Rare Diseases, Roche

Tailoring a market access strategy to include the patient voice and effectively engage with key stakeholders locally and globally

• Including the patient and payer perspective early on in clinical development and understanding the different levels of unmet medical need

• Health economics and outcomes research- capturing the PROs around quality of life and linking that to the disease, patients and caregivers

Raquel Cabo, Vice President, Global Market Access, Ovid Therapeutics

12:05

AGENDA UPDATED AS OF JANUARY 1, 2018For the most up to date agenda, visit www.terrapinn.com/orphandrugsUSA

REGISTER NOWwww.terrapinn.com/orphandrugsusa

NETWORKING LUNCH RESUMES1:30


Lunch keynote address: Evaluating the role of the Orphan Drug Act and looking into incentives, orphan drug usage and costs to ensure sustainability

Peter Saltonstall, President and Chief Executive Officer, NORD

1:00

ROUNDTABLES (1-hour roundtable sessions split between 2 rounds of 30min – attendees can select two roundtables to attend)

If you are interested in sponsoring a roundtable contact André Singer at [email protected] or +1 646 619 1797

END OF ROUNDTABLE SESSIONS

2:00

3.30

ROUNDTABLE 1: Incentives ecosystem - current political, regulatory and legislative climate in the EU and US, around orphan product incentivesRylan Hanks, Global R&D and Regulatory Policy, Product Regulatory Strategy and Intelligence,Amgen

ROUNDTABLE 7: Cognitive endpoints – designing trials for success and strategies for rescuing a failed studyMelissa Hogan, President, Project Alive

ROUNDTABLE 9: Unorthodox funding strategies - alternate but complimentary paths to funding orphan drug companiesKen Kengatharan, Managing General Partner, Atheneos Ventures

ROUNDTABLE 3: Gene therapy - patient advocacy in gene therapy vs. traditional programs, achieving adherence and long-term engagement Barbara Wuebbels, Vice President of Patient Advocacy, Audentes Therapeutics

ROUNDTABLE 5: Startup building - building and leading start-up biotechs into successful companies and building the necessary platforms to support commercial goals Dawn Bir, Chief Commercial Officer, Reata Pharmaceuticals

ROUNDTABLE 2: Cell and gene therapy - post-approval challenges for orphan advanced therapies Diego Ardigò, ATMP Project Leader, Corporate Drug Development, R&D, Chiesi Pharmaceuticals

ROUNDTABLE 8: RWD – utilizing real-world data throughout the product life-span for rare disease treatmentsStella Blackburn, Vice President, Global Head of Early Access and Risk Management, RWES, IQVIA

ROUNDTABLE 4: Gene Therapy trials - Overcoming the challenges of conducting gene therapy studies and navigating the regulatory landscapeScott Schliebner, Vice President, Scientific Affairs, Rare Diseases, PRA Health Sciences

ROUNDTABLE 6: Clinical development- incorporating the patient voice in endpoint design and designing trials with all stakeholders in mindAnita Appius, Global Development Team Leader, F.Hoffmann-La Roche AG

Retinol palmitate for prevention of bronchopulmonary dysplasia (BPD) and retinopathy of prematurity (ROP) in preterm babiesPhilipp Novak, Founder and Chief Executive Officer, Orphanix

Orally Inhaled Ondansetro for the prevention of chemotherapy-induced nausea and vomiting due to highly emetogenic chemotherapy in pediatric patientsEric Li, Chief Scientific Officer, Luxena Pharmaceuticals

2.25

Enabling a New Era of RNA medicinesMark Herbert, Interim President, Arcturus Therapeutics

2.35

The Use of AKT Inhibitors in rare overgrowth syndromesBrian Schwartz, Chief Medical Officer, ArQule Pharmaceuticals

2.45

Multiple clinical-staged programs for the treatment of rare diseasesDavid A. Cory, Chief Executive Officer, Eiger Pharma

2.55

Cancer and Thyroid hormone deprivation - a novel oncological approachOffer Fabian, Chief Executive Officer, Musli Thyropeutics

3.05

Glutamate modulating medicines for the treatment of rare neurologic diseases

Donnie McGrath, Head of Corporate Strategy & Business Development, Biohaven Pharmaceuticals

12.20

PP-001 a novel small molecule for the treatment of noninfectious uveitis

Franz Obermayr, Chief Executive Officer, Panoptes Pharma

12.30

Better treatments for cancer including GBM and metastatic brain cancer

Bill Hornung, Chief Business Officer, Diffusion Pharmaceuticals

12:40

Active checkpoint control therapeutics will supersede most monoclonal antibodies

Fred Jacobs, Chief Executive Officer, TYG Oncology

12.50

2.15

3/5 4/5

ROUNDTABLE 10: Topic to be confirmedModerated by Covance

ROUNDTABLE 11: PROs in clinical trials – what have we accomplished and what is the new frontier in orphan drug treatments?Kathryn Lasch, Executive Director of Patient Reported Outcomes, Pharmerit International


http://www.terrapinn.com/orphandrugsusa


Discovery Track

Machine learning/AI

Rare Disease Advocacy World

National Patient Organizations

Pitch & Partner

Biotech Pitches

The value of computer vision, machine learning and robotic automation to expedite rare disease research

• Running experiments and performing analysis on hundreds of diseases simultaneously using powerful software and analytics

• Finding new indications for existing drugs using augmented high-throughput drug research and technology

• Creating high-resolution, comprehensive biological data sets

• Academia-industry partnerships to foster rare disease research

Ron Alfa, Vice President, Discovery & Product, Recursion

Using artificial intelligence to match rare disease treatments

• Leveraging drug repurposing and combination therapies to accelerate therapeutic outcomes

• Applying genomic drug-matching• Partnering effectively with patient foundations

Tim Gulliams, Chief Executive Officer, Healx

35 Years: Where have we come and where are we going

• A look at the progress the rare disease community has made over the last 35 years, since the founding of NORD and the passage of the Orphan Drug Act

• NORD’s POV on the current state of affairs in the United States in health care, policy, research, and awareness

• Where are science and medicine heading, and where does the rare disease community need to focus its attention and efforts to ensure sustainability for the next 35 years

Pamela Gavin, Chief, Strategy Officer, NORD

Scaling up rare disease patient engagement in Europe

• The EURORDIS new structured approach on patients’ engagement in research, drug development and life cycle and healthcare

• Creating value for patients and working through partnership to create value to all stakeholders

Yann Le Cam, Chief Executive Officer, EURORDIS

Safety, tolerability and efficacy of a novel sustained release analog of vasoactive intestinal peptide, PB1046, in patients with pulmonary arterial hypertension

John Lee, Chief Medical Officer, PhaseBio

3:10

3:35

3.15

Treatment of hepatocellular carcinoma (HCC) and tuberculosis (TB) with oral tableted immunotherapeutics, hepcortespenlisimut-L and tubimod

Aldar Bourinbaiar, Chief Executive Officer, Immunitor Pharmaceuticals

3.55

Acidified Sodium Nitrite and EDTA for the Treatment of Pseudomonas aeruginosa infections in the lungs of cystic fibrosis patients

Richard Muruve, Chief Executive Officer, Director, Co-founder, Arch Biopartners

3.45

BIOGENERA: A new era of DNA personalized drugs

Roberto Tonelli, President, Chairman of the Board, Co-founder, Biogenera

3.35

Oral, centrally acting TNF inhibitors for CNS inflammatory diseases including Frontotemporal Dementia (FTD)

Prasad Gabbita, Chief Executive Officer, P2D Bioscience

3.25

END CONFERENCE 4:05


5/5


COFFEE

NETWORKING LOUNGE

NET

WOR

KIN

G ZO

NE

COFFEE

BUFFET

TO MAIN EXPO HALL

TO CONFERENCE ROOMS

BUFF

ET

FLOORPLAN 2018Collaboration. Science. Technology. This motto has enabled the orphan drug industry and rare disease community to collaboratively develop communication programs with the patients’ voice in mind.

Find out from how to build customized programs rooted in science and online platforms in rare diseases.

If your company struggles to develop and implement an Expanded Access Program or is willing to improve it, make sure to consult with

Running clinical trials for rare disease presents an enormous burden for patients and caregivers

provide clinical concierge solutions for trial participants and families that can lessen that burden while improving trial performance.

Want to learn how genetic testing can address your patients’ needs?

Stop by booth and attend their panel session on April 26th to learn more!

and are key partners of the World Orphan Drug Congress USA! Engage with their executive teams to learn about their policy, advocacy, and research initiatives helping expedite orphan drug development.

For a complete and comprehensive product lifecycle planning that tackles drug safety, patient loyalty and access, leverage the expertise of

No orphan drug is successfully developed,

launched and commercialize without an effective

business development and commercialization plan.

Forecasting and epidemiology are some of the key

solutions provided by , ,

and to

achieve commercial success!

Specialty Pharmacy is a key to success in orphan drug commercialization.

can leverage a strong physical and intellectual access to hospitals and communities globally to improve your patient access strategies. For medical supply solutions, you would want to meet with to get the best out of your supply chain approach to orphan drugs to patients in need.

Looking for a global powerhouse rare disease CRO? Make sure to meet with:

Meeting with will give you an insight into excellence in providing dosage form development and cGMP manufacturing services!

Pro�lesPro�les

AgendaP

Agenda

MeetingsAg

Meetings

SpeakersM

Speakers

SponsorsSp

Sponsors

Floor PlanSp

Floor Plan

Messages

NETWORKING & EVENT PARTNERS

Event Partners


TOP 10 REASONS TO ATTEND

1. GENE THERAPY Join discussions on how to develop and advance gene therapy programs from those who have done it, including Spark Therapeutics, REGENEXBIO, Abeona Therapeutics, Pfizer and AGTC

6. PRICING & REIMBURSEMENT Delve into new pricing and reimbursement frameworks and guidelines for orphan drugs from institutions and government organizations such as ICER, ANVISA (Brazil) and NHS (England)

7. COMMERCIAL Learn about successful commercialization strategies including pre-launch, launch, forecasting, competitiveness and key models for launching rare disease companies from SpringWorks Therapeutics, Alnylam Pharmaceuticals, Ablynx and SOBI

10. WORKSHOPS & SEMINARS Attend 4 insightful workshops on forecasting techniques for orphan & ultra-orphan drugs, the integration of the community voice, expanded access programs and 5 seminars on digital health, data collections, global pricing and reimbursement, next generation therapies and precision medicine

2. GENE EDITING Learn how the 4 most prolific gene editing biotechs like Editas Medicine, CRISPR Therapeutics, Intellia Therapeutics and Sangamo Therapeutics are advancing their therapies into trials

3. GLOBAL Meet with 1,000+ attendees, including market access and patient advocacy leaders from from over 50 countries including: US, Canada, Japan, Australia, South Africa, France, Sweden, UK, Italy, Netherlands, New Zealand, Colombia, Brazil, India, China, Bulgaria, Finland, and Russia

9. MARKET SUSTAINABILITY Support NORD and EURORDIS in their articulation for the sustainability of orphan drug policies, collaboration and market viability for rare disease therapeutics

BONUS REASONNETWORKING – Engage in 3 days of learning through 150+ talks and networking sessions, including over 1000 one-on-one meetings

4. PITCH AND PARTNER Analyze over 30 orphan drug biotechs presenting new data as they looking for funding and partnerships

5. PATIENT CENTRICITY Understand how patient centric drug development has been put into practice from research to launch, from cases such as Cystic Fibrosis Foundation, Friedreich’s Ataxia Research Alliance, and Biomarin

8. REAL WORLD EVIDENCE Hear about the inclusion of RWE into the regulatory approval process as well as how to fill evidence gaps to support approval, access and reimbursement decisions

JUJAMAJUJAMA is our full service app that allows you to manage your time efficiently and effectively at the event, while ensuring you meet all the right people.

You can use the app to set meetings, explore the agenda, familiarize yourself with speakers and exhibitors and much, much more. Log into your profile, update your details and get started. It’s that simple!

Networking with Jujama is available 24/7, enabling you to plan and get the most out of the event well in advance. Accessible via numerous devices, you can:

• Review the latest exhibitor list and see where booths are on the floorplan

• Contribute to specific groups and start conversations long before the event

• Share relevant content with your social networks

As an event attendee, you’ll get access to your FREE event management profile and will get personalized login details to use the app.

Business Matching at the event goes live four weeks before the show.

Note: Jujama respects your privacy and assures you that all of your personal contact information will be kept confidential. Rest assured that no contact information will be shared via our platform.”


PRICING SPONSORS


This year we are mixing things up! We have 100 passes per tiered price. As soon as we have sold 100, the price will go up. So the earlier you book the more you’ll save – really!

It’s really easy to book your place online. And our online calculator will ensure you take advantage of the best deal.

Go to and book now on www.terrapinn.com/WODC2018

DELEGATE BOOKING

TIER 4book by April 6

Final PriceTIER 3book by March 16

TIER 2book by February 26

TIER 1book by February 6

2 DAY CONFERENCE PASS

2 DAY CONFERENCE PASS + HALF DAY WORKSHOP PASS

2 DAY CONFERENCE PASS + FULL DAY WORKSHOP PASS

$3,060SAVE $340

$3,400

$3,510SAVE $390

$3,900

$3,735 SAVE $415

$4,150

$2,890SAVE $510

$3,315SAVE $585

$3,530 SAVE $620

$2,720SAVE $680

$3,120 SAVE $780

$3,320 SAVE $830

$2,550SAVE $850

$2,925 SAVE $975

$3,115SAVE $1,035

BOOK NOWGo to www.terrapinn.com/WODC2018 or call +1 212 379 6320

PLATINUM

PATIENT ADVOCACY

SILVER

ASSOCIATE

GOLD

EXHIBITOR




the global orphan drug conference and expo · the global gathering for orphan drugs join these...

Documents