(2286) world orphan drug congress usa 2016 a4 32 page brochure 1.5 screen spread

6th Annual

April 21-22, 2016Washington Hilton in Washington, D.C.

Pre-conference workshops on April 20, 2016

THE GLOBAL ORPHAN DRUG CONFERENCE AND EXPO

Created by

Together with:

THE GLOBAL GATHERING FOR ORPHAN DRUGS

Stephen Rose Chief Research Officer, Foundation Fighting Blindness IRELAND

FOR MORE INFORMATION contact Pinky Fadullon at [email protected] or +1 646 619 1785

David Pena President, FEMEXER, Mexican Federation of Rare Diseases MEXICO

Agnes V. Klein Director, Centre for Evaluation of Radiopharmaceuticals and Biotherapeutics, Health Canada CANADA

Guisela Zurich Head of Registries, ANAMED, National Medicines Agency, Chilean Public Health institute CHILE

Yann Le Cam CEO, EURORDIS – FRANCE

Segolene AymeCoordinator, Scientific Secretariat, IRDiRC – FRANCE

Marc de GaridelChariman and CEO, Ipsen FRANCE

Bruno Hatschbach President, National Association of Rare Diseases, BRAZIL

Bruno Sepodes Chair, Committee for Orphan Medicinal Products (COMP), European Medicines Agency (EMA)PORTUGAL

Virginia Llera President, Geiser Foundation, ARGENTINA

Adam Barak Managing Director, PPi Healthcare Consulting UK

Yukiko Nishimura Chief Secretariat of International Relations, Japan Patients Association JAPAN

Hatijah Ayob President, Malaysian Rare Disorders Society, MALAYSIA

Tomoko Kodama National Institute of Public Health, JAPAN

Abida Syed Director, Pharmacy Practice & Development, Pharmaceutical Services Division, Ministry of Health, MALAYSIA

Carmencita Padilla Chancellor, University of the Philippines, Chair, Philippine Society for Orphan Disorders PHILIPPINES

OUR STORY EVENT AT A GLANCEThe World Orphan Drug Congress was conceptualized to provide a platform for pharma and biotech to come together and discuss ways to expedite orphan drug access to rare disease patients. Patient groups were already well-organized at that time but there were no groups organized specifically for industry. We at Terrapinn saw this as important because although Orphan Drug Acts existed in US, Europe, Japan and Australia, the process from R&D to patient posed many challenges. Each year the conference brings together key industry stakeholders to discuss the latest strategies to overcome these challenges.

When the World Orphan Drug Congress USA was launched five years ago, it had 200 participants. Shire, Genzyme, Amicus, Ultragenyx, SOBI and a few other leaders in the sector were there, of course, but there was also a noticeable representation from big pharma talking about their business approach in orphan drugs.

As the sector grew, the conference grew now bringing together pharma and biotech; regulators, government agencies, payers, academia, patient groups, KOLs and investors. From 200 to 600 participants last April in 2015, and a representation from 29 countries worldwide, the World Orphan Drug Congress USA is indeed the largest global gathering of the orphan drug sector.

For 2016, the event starts on April 20th, with a pre-conference workshop day. As an attendee you can choose from 8 half day workshops or 2 full-day seminars. Workshops range in topic from market forecasting to pricing & reimbursement, R&D, commercialization, marketing and treatment.

But this won’t be an event the Terrapinn way without some light fun and networking. We have at least ten hours of networking including a Welcome Drinks Reception, Networking Cocktails, 1-2-1 Meetings and Speed Networking.

We look forward to welcoming you to DC next April.

*Please note that access to the pre-conference workshop day is only available to those who purchase a 3-Day or a 2.5-Day ticket

The two full-day seminars are for KOLs and payers:

The main two-day event kicks of on April 21st with keynote plenary sessions and there after the agenda splits into three streams:

1. KOLs: training strategies, patient registries, collaboration, diagnosis and improving patient outcomes.

2. Payers: cross-country, to discuss different approaches to funding treatments for rare disease. Payers will talk about the approaches adopted in their own markets, and then exchange views, from a multi-stakeholder audience to provide an interesting, informative and insightful discussion on best practice.

1. MAIN CONFERENCE FOR INDUSTRY

2. RARE DISEASE ADVOCACY WORLD FOR PATIENT GROUPS

3. PITCH & PARTNER SESSION FOR BIOTECHS RAISING CAPITAL

FRIDAY, APRIL 22

Networking coffee break

Networking lunch break

Chairpersons’ closing and end of conference

MAIN CONFERENCE RARE DISEASE ADVOCACY WORLD

Medical Affairs

Pricing & Reimbursement National patient organizations

Patient groups from around the world

PITCH & PARTNER

8 slots

7 slotsRoundtable Sessions

Innovation: processes & drug discovery development approach

10:40

1:50

11:40

9:00

10:00

12:20

3:00

THURSDAY, APRIL 21

Chairpersons’ closing remarks Networking cocktails




MAIN CONFERENCE RARE DISEASE ADVOCACY WORLD PITCH & PARTNER

EAP & the ‘Right to Try’

Roundtable Sessions

Marketing trends

International policy trends

Putting patients in the center of drug development

Regulatory, stats, toxicology,

Collaborations & partnerships

Bioethics & patients’ rights

7 slots

8 slots

8 slots

State of the Industry

11:00

1:50

4:10

4:30

2:30

2:50

9:00

5:10 5:35

10:30

12:20

3:10

10:00 Speed networking

Full day seminar:Diagnosing rare

diseases and orphan drugs 101 for KOLs

WEDNESDAY, APRIL 20 (PRE-CONFERENCE DAY)


Welcome drinks reception End of pre-conference day

Market forecastingBy: Lapidus Consulting

Raising capital

Full day seminar:Best practice cross-

country market access policies

Marketing orphan drugs

By: ZS Associates

Addressing the Why Behind Treatment Nonadherence:

Leveraging Health Psychology to Design

Effective Interventions in Rare Disease

By:Atlantis Healthcare

Gene Therapy and Personalized medicine

Overcoming challenges in rare disease

health research and Orphan Drug Program

CommercializationBy: MAPI

Pricing & reimbursement

By: Medical Marketing Economics

Specialty pharmacy

9:00

1:00

12:00

5:00 6:00

BOOK YOUR TICKET TODAYSimply visit www.terrapinn.com/WODC2016 to get your ticket

INTERESTED IN HOSTING A WORKSHOP?Contact Pinky Fadullon at [email protected] or +1 646 619 1785

MORNING WORKSHOPS (9am - 12pm)

PRE-CONFERENCE WORKSHOP AGENDA (April 20th, 2016) PRE-CONFERENCE WORKSHOP AGENDA (April 20th, 2016)

AFTERNOON WORKSHOPS (1pm - 5pm)

WORKSHOP A: Market forecastingSo you got the science right, but have you worked out the numbers? Will you be able to recoup the cost to develop your orphan drug?

Learn how to forecast for an orphan drug and how to factor in market conditions such as epidemiology, natural history, treatable population, diagnosis, uptake and price

MODERATED BY: David Lapidus, President, Lapidus Data Consulting

WORKSHOP C: Gene therapy and personalized medicine We are at the cusp of a gene therapy and personalized medicine era, so we cannot not talk about the challenges biopharma/biotech face developing gene therapy.

Do policies and regulations change when we consider these to be transformational therapies? How do payers perceive the value of gene therapy?

Join this workshop to learn more about this up and coming sector.

WORKSHOP D: Achieving commercial success for orphan drugs Commercializing and launching orphan drugs is a vastly different challenge with different needs and requiring different approaches – so what does it take to succeed?

With success stories and perspectives from industry leaders, this workshop will focus on key areas including launch excellence, customer experience models, patient and HCP targeting, field force effectiveness, and incentive compensation design

MODERATED BY: Michael Thomas, Principal, ZS Associates

WORKSHOP B: Pricing & ReimbursementWe’ve heard instances where the marketing approval is celebrated for an orphan drug which is assumed to achieve great market access with a significant price, only later to find out those earlier assumptions were wrong.

Medical Marketing Economics (MME) who has completed more than 125 price strategies in the last three years will take you through value-based marketing strategies. We have helped >40 ultra-orphan drugs to identify, frame, communicate and capture the value of their therapies

Gain from our unique combination of:

Manufacturer and customer economic and clinical perspectives combined with solid academic theory

Strategy development and tactical execution to support informed decision making

Assessment and planning of opportunities and competitive situations at every stage of the product life cycle.

SPONSORED BY:

WORKSHOP E: Raising capital Whether you’re looking for seed capital with angels or about ready to go into bigger clinical trials, this workshop is for you.

Join this workshop to learn more about how to raise capital through M&As, private placements and access to equity and debt capital markets.

WORKSHOP F: Specialty PharmacyThis workshop will uncover solutions that sponsors face when delivering orphan drugs to their rare disease patients.

How do you make sure patients get the orphan drug in time? What are the policies in a country regarding importation? What are the ethical standards? How do you choose which specialty pharmacy to work with?

Join this workshop to iron-out issues you have with making sure your patients get access to your orphan drug.

WORKSHOP G: Overcoming challenges in rare disease health and orphan drug program commercialization This workshop will take you through the nitty gritty of developing an orphan drug from understanding the patient’s physical needs and social influences to efficiently deliver what payers require by first understanding the market access challenges sponsors commonly experience.

Hear MAPI’s experts discuss the following topics:

The process of understanding the person with the indication Kelly Franchetti, Executive Director, Global Patients Insights and Engagement, MAPI

Market access challenges for rare diseases Charles Makin, Vice President, MAPI

Recent developments in HTA and reimbursement decision-making considerations for orphan drugs in Europe Carina Righetti, Associate Director, MAPI

Orphan drug and rare diseases - requirements for Real World Data: generating data to gain and keep market access (3 - 5 years post launch) Will Maier, Chief Scientific Officer, MAPI

Regulatory Hurdles Orphan Drug - Are They Increasing? Patricia Anderson, Vice Preseident, MAPI

Lunch is provided for pre-conference day participants only 12:00

WORKSHOP H: Addressing the why behind treatment non adherence Leveraging health psychology to design effective interventions in rare disease. Learn how health psychology frameworks are being applied to understand barriers and motivators in addressing treatment adherence, a key area to improving self-management long-term

MODERATED BY: Laura Moore, Ed.D., CHES, Health Psychology Specialist, Atlantis Healthcare

SEMINAR AGENDA (April 20th, 2016) SEMINAR AGENDA (April 20th, 2016)

Training in clinical investigation: partnerships between clinical researchers and corporate liaisons to advance orphan trials and product development

• Training programs for clinicians to more effectively identify and recruit patients for rare disease trials• Positively impact trial design and execution through training clinicians on product development at pharmaceutical

companies • Academic vs. monetary reimbursement to strengthen the commitment to develop and complete trialsFrederick Kaskel, Vice Chair of Pediatrics, Director of Child Health, Einstein-Montefiore Institute for Clinical and Translational Research, Albert Einstein College of Medicine

Putting pemphigus and pemphigoid on the radar of dental professionals: an early diagnosis campaign

• Importance of early diagnosis of pemphigus and pemphigoid to accelerate the start of treatment• Designing a tailored campaign to spread awareness on dental professionals through the creation of an emotional

connection with the rare disease • Preliminary data on the impact of the campaign in pemphigus and pemphigoid diagnosis and ways to spread the

outreach of the campaign moving forward Kate Frantz, Awareness Program Manager, International Pemphigus & Pemphigoid Foundation (IPPF)

Advancing research in rare diseases through tools that build rare disease registries: how this not only benefits patients but also clinicians and researchers

• Providing opportunities for improved understanding of the widely variable disease manifestations • Using registries to improve quality of life and accelerate development of effective therapies • The NIH/NCATS Global Rare Diseases Registry Data Repository program as a unique platform to integrate and

accelerate rare disease research benefiting patients, clinicians and researchers

Robert Haws, Doctor of Pediatric Nephrology, Marshfield Clinic

Involving physicians, patients and advocacy groups in early phases of orphan drug development

• How can pharma help build patient registries and strengthen interpharma collaboration?

• Partnering with early phase development teams and involving patients and other stakeholders in clinical trial design

• Industry’s role in identifying, educating and equipping physicians in the rare disease spaceMauricio Silva de Lima, Vice President, US Medical Affairs, Head, Spectrum Medical Unit, Genentech

How to foster collaboration between the NIH, industry and researchers to develop robust clinical trials for rare diseases

• Achieving funding for Investigator-initiated trials (IITs) from government institutions and pharmaceutical companies• Educating stakeholders in order to design better trials, find robust endpoints and strengthen patient recruitment • Putting rare diseases on the radar of decision-makers to advance research initiatives Roham Zamanian, Associate Professor of Medicine, Director, Adult Pulmonary Hypertension Service, Stanford University School of Medicine

9:00

Exome sequencing to diagnose pediatric diseases and how this can be used to gain insight into more common disorders

• Diagnosis strategies for pediatric rare diseases and why this can help entire family’s generations

• Efficiently integrate clinical expertise into rare disease research

• Using greater public recognition of rare genetic diseases to improve research and diagnostic tools

Virginia Kimonis, Professor, Department of Pediatrics, Division of Genetics and Genomic Medicine, University of California-Irvine Medical Center

Brain biomarkers in urea cycle disorders to improve diagnosis and prediction of disease outcomes

• Techniques to measure changes in brain biochemistry, white matter integrity and neural circuitry and their relation to disease severity

• Influencing treatment decisions by detecting subtle changes in brain biochemistry through improved diagnostic techniques

• Strengthening trial recruitment by reducing the burden on children with rare diseases through less invasive and more efficient diagnostic tools

Andrea Gropman, Chief, Division of Neurogenetics and Developmental Pediatrics, Professor in Neurology and Pediatrics, Children’s National Medical Center

Genetic mutation analysis and PET scan imaging to predict disease prognosis in pediatric patients with hyperinsulinism

• Determining which pediatric patients will have a curable form of hyperinsulinism through rapid genetic testing

• Using diagnostic tools that are not FDA approved. What are the different protocol options?

• How to help clinicians do genetic testing quickly in any place of the world and how do you get rare disease patients to specialized diagnostic centers?

Charles Stanley, Medical Director, Congenital Hyperinsulinism Center, The Children’s Hospital of Philadelphia, Professor Emeritus of Pediatrics, Perelman School of Medicine, University of Pennsylvania

3:00

3:45

4:30

9:45

11:00

1:00

1:30

Strengthening stakeholder relationships: Industry perspective on working with KOLs in the rare disease space

• Integrating physicians and researchers in the drug development process to develop clinical programs relevant to rare disease patients

• Pre-launch strategies: non-branded disease education awareness sites, KOL identification, finding the right patient communities/advocates

• Maintaining relationships when dealing with larger territories and less number of KOLsMauricio Berdugo, Associate Director, Health Science Executive, Boehringer Ingelheim

2:30

TRAINING THE NEXT GENERATION OF EXPERTS

ACCURATE DIAGNOSIS AND IMPROVING PATIENT OUTCOMES

PATIENT REGISTRIES

COLLABORATION BETWEEN MULTIPLE STAKEHOLDERS

Networking coffee break 10:30

Networking Lunch 11:45

Welcome drinks reception 5:00

End of Pre-conference day 6:00

Networking coffee2:00

1/2 2/2

FOR MORE INFORMATIONContact Pinky Fadullon at [email protected] or +1 646 619 1785

KOLS SEMINAR - ADDRESSING THE NEEDS OF PHYSICIANS IN THE RARE DISEASE SPACE

April 21-22, 2016Full details of the Main Conference agenda and speaking faculty

can be found over the next few pages.

Keep reading for information on Rare Disease Advocacy World 2016.

World orphan drug congress is the place to be for the leaders in the rare disease community

Hans Schikan, Chief Executive Officer, Prosensa Therapeutics Bv

BOOK YOUR TICKET TODAY at www.terrapinn.com/WODC2016

WORLD ORPHAN DRUG CONGRESS USA SPEAKERS

Nancy Silliman Vice President, Biostatistics, Epidemiology, Data Management, and Programming, Alexion Pharmaceuticals

Bruno Hatschbach President, National Association of Rare Diseases, Brazil

Kyle Brown Founder & CEO, Patient Crossroads

Roham Zamanian Associate Professor of Medicine, Director, Adult Pulmonary Hypertension Service, Stanford University School of Medicine

Virginia Kimonis Professor, Department of Pediatrics, Division of Genetics and Genomic Medicine, University of California-Irvine Medical Center

Frederick KaskelVice Chair of Pediatrics, Director of Child Health, Einstein-Montefiore Institute for Clinical and Translational Research, Albert Einstein College of Medicine

Robert Haws Doctor of Pediatric Nephrology, Marshfield Clinic

Andrea Gropman Chief, Division of Neurogenetics and Developmental Pediatrics, Professor in Neurology and Pediatrics, Children’s National Medical Center

Charles Stanley Medical Director, Congenital Hyperinsulinism Center, The Children’s Hospital of Philadelphia, Professor Emeritus of Pediatrics, Perelman School of Medicine, University of Pennsylvania

Art Caplan Head, Division of Medical Ethics, NYU Langone Medical Center

Alison Bateman-HousePostdoctoral Fellow, Division of Medical Ethics, New York University Langone Medical Center

Ian Phillips Director, Center for Rare Disease Therapies, Faculty Director, Postbaccalaureate Premedical Certificate Program, Keck Graduate Institute of Applied Life Sciences

Gigi Hirsch Executive Director, NEWDIGS and CBI, MIT

David PenaPresident, FEMEXER, Mexican Federation of Rare Diseases

Virginia LleraPresident, Geiser Foundation, Argentina

Yukiko Nishimura Chief Secretariat of International Relations, Japan Patients Association

Max Bronstein Senior Director of Public & Government Relations, EveryLife Foundation

Deborah SittigFounder, The Soft Bones Foundation

Tomoko Kodamanvited Researcher, Department of International Health and Collaboration, National Institute of Public Health, Japan

Jorge Venâncio Coordinator of the National Research Ethical Board (CONEP), Member of the National Health Council (CNS), Brazil

Rashmi Gopal-Srivastava Director, Extramural Research Program, Office of Rare Diseases Research, NCATS, National Institutes of Health

Richard Moscicki Deputy Center Director for Science Operations, Center for Drug Evaluation and Research, FDA

Bruno Sepodes Chair, Committee for Orphan Medicinal Products (COMP), European Medicines Agency (EMA)

Guisela ZurichHead of Registries, ANAMED, National Medicines Agency, Chilean Public Health institute

Agnes V. KleinDirector, Centre for Evaluation of Radiopharmaceuticals and Biotherapeutics, Health Canada

Carmencita PadillaChancellor, University of the Philippines, Chair, Philippine Society for Orphan Disorders

Abida SyedDirector, Pharmacy Practice & Development, Pharmaceutical Services Division, Ministry of Health, Malaysia

Dan Donovan Founder & CEO, Clear Pharma

Wendi LeVigne Vice President Manufacturer Relations, One-World, Inc

ACADEMIC MEDICAL CENTERS/HOSPITALS

Emil KakkisPresident & CEO, Ultragenyx

Laurie LetvakVP, Clinical Development Policy, Novartis

Mary Ellen Carroll Director, Global Commercial Development, Rare Diseases, Pfizer

Charles Stark Senior VP, Research & Development, Emmaus Life Sciences

Marc de Garidel Chariman and CEO, Ipsen

Samit Hirawat Senior Vice President, Novartis Oncology

Ron Guido SVP, Global Regulatory Affairs and Chief Compliance Officer, Veloxis Pharmaceuticals

INDUSTRY

Mark Rothera Chief Commercial OfficerPTC Therapeutics

Alvin ShihEVP, Head of Research and Development, Retrophin

Rob Scott Head Global CV, Metabolic & Neuroscience Development and Development Design Center, Amgen

Steven Ryder Senior Vice President and Chief Development Officer, Alexion

Mauricio Silva de LimaVice President, US Medical Affairs, Head, Spectrum Medical Unit, Genentech

Mark Baglin Vice President, Product Strategy Team Leader, Angioedema Franchise, Rare Disease Business Unit, Shire

Mauricio Berdugo Associate Director, Health Science Executive, Ofev, Boehringer Ingelheim

Elizabeth White Assistant Vice President, Rare Disease Commercial Development, Global Innovative Pharma Business, Pfizer

Colin FosterPresident & CEO, BioBlast

Peter Saltonstall President and CEO, NORD

Yann Le CamCEO, EURORDIS

Kate Frantz Awareness Program Manager, International Pemphigus & Pemphigoid Foundation (IPPF)

Stephen Rose Chief Research Officer, Foundation Fighting Blindness

Claudia Hirawat Chair, International Circle of Ambassadors, EURORDIS

Pat Furlong President & CEO, Parent Project Muscular Dystrophy

Hatijah AyobPresident, Malaysian Rare Disorders Society

PATIENT (ADVOCACY) GROUPS

GOVERNMENT INSTITUTIONS REGULATORS SOLUTION PROVIDERS/EXPERTS

Scott SchliebnerVice President of Scientific Affairs, Rare Diseases, Federal Work, PRA Health Sciences

Eric GemmenSenior Director, Scientific Affairs, Biostatistics & Outcomes Research, Quintiles

Benoit Arnould Senior Director, Patient-Centered Outcomes, MAPI Group

Keith Watts Executive Vice President Strategic Services, One-World, Inc

Mary SeidemanManaging Partner, Arcus Medica

Adam Barak Managing Director, PPi Healthcare Consulting

Malcolm Mackenzie SVP, Strategic Planning/New Business Development, Juice Pharma Worldwide

Michael ThomasPrincipal, ZS Associates

Abbe Steel CEO, HealthiVibe

Laure BurkeFounder, LORA GROUP

Laura Moore Health Psychology Specialist, Atlantis Healthcare

Stella BlackburnVice President, Global Head of Risk Management, Real-World & Late Phase Research, Quintiles

Edmund PezallaVice President, National Medical Director, Pharmacy Policy and Strategy, Aetna

James GreenwoodCEO, Biotechnology Industry Organization

Segolene Ayme Coordinator, Scientific Secretariat, IRDiRC

Steve UsdinSenior Editor, BioCentury

PAYERS ASSOCIATION

JOIN THEM IN WASHINGTON IN APRIL visit www.terrapinn.com/WODC2016

Registration & networking breakfast

SPONSORED BY:

Terrapinn welcome remarks

Chairperson’s opening remarks

8:00

8:50

8:55

CONFERENCE DAY ONE (Thursday, April 21st, 2016) CONFERENCE DAY ONE (Thursday, April 21st, 2016)

21st Century Cures and Innovations Acts: Opening the door to faster treatment development for devastating diseases or increasing the risk of approving drugs that are not effective or safe?

• What are the specific provisions for orphan drug development in the 21st Century Cures Act?• What are the general provisions that can be applied to orphan diseases and aid in drug development?• Is the act setting overly optimistic expectations to think that drugs can be approved easily? • What will be the benefits for patients and drug manufacturers?Richard Moscicki, Deputy Center Director for Science Operations, Center for Drug Evaluation and Research, FDA Peter Saltonstall, President and CEO, NORD Laurie Letvak, VP, Clinical Development Policy, NovartisEmil Kakkis, President & CEO, Ultragenyx

Keynote: Creating medicines with real value for orphan diseases

• The 21st century cures and Innovations acts: Progressing the science of treatment development for the rarest patient

• Approaches to development for the rarest of diseases• Directing treatment to the disease burden of patients to optimize the development of meaningful drugsEmil Kakkis, President & CEO, Ultragenyx

The ethics of expanded access and compassionate use in the USA and beyond

• What do ‘Right to Try’ (RTT) laws do to enhance patient access to unapproved drugs and treatments?• What is the compassionate use advisory committee that J&J/Janssen has formed with NYU• Can expanded access and clinical trials co-exist? • Morally what do companies owe to those who cannot qualify for clinical trials of their drugs and treatments?Art Caplan, Head, Division of Medical Ethics, NYU Langone Medical Center

Pursuing accelerated approval for rare disease therapeutics

• Overview of the accelerated approval (subpart H) pathway and its relevance to rare disease drug development• What are the optimal attributes of an acceptable surrogate endpoint?• Making it happen: lessons learned through the Sparsentan experience to dateAlvin Shih, EVP, Head of Research and Development, Retrophin

Panel discussion: navigating drug development. Identifying biomarkers and surrogate endpoints when there is no known natural history

• Where to begin to find fundamental information of a disease and patient population?• Biomarker identification and developing diagnostic tests to select patients for clinical trials • How to effectively work with physicians and advocacy groups to increase access to patient data Rob Scott, Head Global CV, Metabolic & Neuroscience Development and Development Design Center, Amgen Steven Ryder, Senior Vice President and Chief Development Officer, Alexion Mauricio Silva de Lima, Vice President, US Medical Affairs, Head, Spectrum Medical Unit, Genentech

Europe’s focus on re-engineering the process rather than new legislations to get more, better, faster, cheaper orphan drugs

• Medicines Adaptive Pathways to Patients (MAPPs)

• Implications of a more flexible framework on regulatory processes, health technology assessments (HTA), pricing, reimbursement and delivery of treatment

• Is Europe and the rest of the world ready to operate on a new risk trade-off, increased uncertainties and the need for real world evidence?

Yann Le Cam, CEO, EURORDIS

9:20

9:00

Roundtable sessions: 1:50

11:00

11:20

11:40 2:30

STATE OF THE INDUSTRY

CLINICAL DEVELOPMENT

INTERNATIONAL POLICY TRENDS

Speed networking10:00


Networking lunch break 12:20

1/3 2/3

ROUNDTABLE 1: Super-charging online communities: Potential “outputs” of online communities such as data, insights, action and advocacyDan Donovan, Founder & CEO, Clear Pharma

ROUNDTABLE 5: Developing a registry for successfully meeting multi-stakeholder evidence requirements Eric Gemmen, Senior Director, Scientific Affairs, Biostatistics & Outcomes Research, Quintiles

ROUNDTABLE 3: Integrating electronic health records into patient opt-in registriesKyle Brown, Founder & CEO, Patient Crossroads

ROUNDTABLE 7: Effectively engaging physicians in rare diseases: from disease awareness to advisory boardsMary Seideman, Managing Partner, Arcus Medica

ROUNDTABLE 9: Seeking commercial and development guidance for orphan drugs to support market access in EuropeAdam Barak, Managing Director, PPi Healthcare Consulting

ROUNDTABLE 11: The unique patient journey in orphan and its impact on commercialization and promotionMichael Thomas, Principal, ZS Associates

ROUNDTABLE 13 Preparing for commercialization: timing, challenges and solutionsJoel Schindler, Senior Vice President, Market Vision Research

ROUNDTABLE 2: Pre-license sales and compassionate use: two models of expanded access programsWendi LeVigne, Vice President Manufacturer Relations, One-World, Inc.

ROUNDTABLE 6: Digital strategies to optimize patient engagementMary Ellen Carroll, Director, Global Commercial Development, Rare Diseases, Pfizer

ROUNDTABLE 4: Innovative clinical solutions for rare disease clinical trialsScott Schliebner, Vice President of Scientific Affairs, Rare Diseases, Federal Work, PRA Health Sciences

ROUNDTABLE 10: Early-stage commercialization of orphan drugsMalcolm Mackenzie, SVP, Strategic Planning/New Business Development, Juice Pharma Worldwide

ROUNDTABLE 12: Measuring Patient-Centered Treatment BenefitAbbe Steel, CEO, HealthiVibe Laure Burke, Founder, LORA GROUP

ROUNDTABLE 8: Clinical trials in Brazil and the path for rare diseases treatments: Preserving the ethical aspects in defense of the integrity and dignity of the research subjectsJorge Venâncio, Coordinator of the National Research Ethical Board (CONEP), Member of the National Health Council (CNS), Brazil, Hosted by: Expressa Group

Interested in moderating a roundtable? Contact Pinky Fadullon at +1 646 619 1785 or email her at [email protected]

CONFERENCE DAY ONE (Thursday, April 21st, 2016)

Panel discussion: Rare disease policy updates in Japan, Canada, Malaysia, Chile and the Philippines

• Lack of health policies that recognize the social importance of rare diseases• Limited designated funding for reimbursement/coverage of orphan drugs• Application of health technology assessment (HTA) models that are not appropriate for rare diseases• Less developed patient advocacy than in other regionsGuisela Zurich, Head of Registries, ANAMED, National Medicines Agency, Chilean Public Health institute Agnes V. Klein, Director, Centre for Evaluation of Radiopharmaceuticals and Biotherapeutics, Health CanadaCarmencita Padilla, Chancellor, University of the Philippines, Chair, Philippine Society for Orphan DisordersAbida Syed, Director, Pharmacy Practice & Development, Pharmaceutical Services Division, Ministry of Health, Malaysia Tomoko Kodama, Invited Researcher, Department of International Health and Collaboration, National Institute of Public Health, Japan

Panel discussion: Success measures in the launch and lifecycle of orphan drugs

• Pre-launch strategies: non branded awareness, KOL identification, communication with patient advocates• Addressing treatment adherence in rare disease, including challenges of adolescent to adult transitions• Developing direct-to-patient, HCP and caregiver connections• Establishing measurements and metrics to define successMauricio Berdugo, Associate Director, Health Science Executive, Ofev, Boehringer Ingelheim Deborah Sittig, Founder, The Soft Bones Foundation Antoun Nabhan, Vice President, Corporate Development, Raptor Pharmaceuticals

MODERATOR: Laura Moore, Health Psychology Specialist, Atlantis Healthcare

Rare is different: commercializing drugs for orphan disease populations

• Clinical development and commercialization strategy in rare diseases• Achieving market access by placing a focus on prescribers, patients and payers• Differential challenges in the US and internationally and the critical role of the patient at all pointsMark Baglin, Vice President, Product Strategy Team Leader, Angioedema Franchise, Rare Disease Business Unit, Shire

3:50

4:30

4:50

The ‘significant benefit’ concept in the European regulatory framework for orphan medicinal products

• How is ’significant benefit’ assessed at time of orphan drug designation and at the time of market authorization? Why is it so important in the European orphan regulation?

• Importance of having proper clinical development to show a ‘significant benefit’ at the time of marketing authorization

• What has Europe learned from international collaboration on orphan drugs and contributed to the international discussions?

Bruno Sepodes, Chair, Committee for Orphan Medicinal Products (COMP), European Medicines Agency (EMA)

2:50

MARKETING TRENDS


3/3

Networking cocktails sponsored by5:35

Chairperson’s closing remarks5:30

End of day one 6:35





8:00

8:50

8:55

CONFERENCE DAY TWO (Friday, April 22nd, 2016) CONFERENCE DAY TWO (Friday, April 22nd, 2016)

Keynote: Adaptive biomedical innovation: re-engineering how we innovate

• The era of blockbusters, RCTs-only, and average patients is over!• What is required to drive meaningful value for patients as innovation is “orphanized”? • View this evolution through the design lens of MIT NEWDIGS – a global “think and do” tank for collaborative

systems engineering across the traditional siloes of the innovation chain. Gigi Hirsch, Executive Director, NEWDIGS and CBI, MIT

Keynote: A systematical approach to orphan drug discovery: starting with the unmet needs of payers and patients

• Has anything changed in orphan drug reimbursement?• Moving away from target discovery: how to meet payer’s and patient’s needs• How can pharma justify the high prices of orphan drugs to payers? Edmund Pezalla, Vice President, National Medical Director, Pharmacy Policy and Strategy, Aetna

Targeting ‘bigger’ targets: increase nonsense mutation read through applicable to other rare diseasesMark Rothera, Chief Commercial Officer, PTC Therapeutics

How IRDiRC is establishing and providing access to harmonized data and samples

• De-risking R&D and making it cheaper to produce affordable drugs• Making databases interoperable:“IRDiRC recommended” label as a standard for databases that contribute to

accelerate R&D • How to incentivize research for the remaining rare diseases for which there is no available treatment?Segolene Ayme, Coordinator, Scientific Secretariat, International Rare Diseases Research Consortium (IRDiRC)

Maintaining a relationship based on trust: finding common goals with a KOL and its institution, and maneuvering communications between people internally and externally

• Relationship initiation, development and maintenance including identifying key stakeholders outside KOLs• Finding mutual areas of expertise, needs, and deliverables for goals • Types of business relationships: patents, rights to publications, partnership, grants payments for services, etc.Charles Stark, Senior VP, Research & Development, Emmaus Life Sciences

Screening tools to capture the perspectives of patients and caregivers in rare diseases

• The development of the PKU-QoL screening tool to measure the severity of Phenylketonuria and support clinical development

• Addressing cross-cultural variations, working across different age groups, and optimizing research in small samples

• Initiatives aiming at providing adapted guidance to the conduct of patient- centered outcomes research in the context of orphan drug development

Benoit Arnould, Senior Director, Patient-Centered Outcomes, MAPIAntoine Regnault, Research Director, HEOR & Strategic Market Access, MAPI

9:20

9:40

10:40

11:00

11:20

9:00

INNOVATION: PROCESSES & DRUG DISCOVERY DEVELOPMENT APPROACH

MEDICAL AFFAIRS

The high cost of rare disease drugs: is the pricing sustainable?

• How are different stakeholders determining the price of orphan drugs?• Will gene therapy pricing models test payers tolerance for the high costs of drugs? • Why are rare disease drug prices high and climbing higher?Interested in speaking? Contact Pinky Fadullon at +1 646 619 1785 or email her at [email protected]

Panel discussion: Appropriate pricing models for a one-time transformational gene therapy vs. orphan drugs for life

• Exploring original methods to overcome limitations of value-based pricing by adjusting prices according to the number of potential patients

• Comparative Effectiveness Research: higher payments for services demonstrated by adequate evidence to provide superior health benefits compared to alternative options

• Developing a common language of the components of value across pharma, payers and other stakeholdersJames Greenwood, CEO, Biotechnology Industry Organization Ian Phillips, Director, Center for Rare Disease Therapies, Faculty Director, Postbaccalaureate Premedical Certificate Program, Keck Graduate Institute of Applied Life SciencesSteve Usdin, Senior Editor, BioCentury

1:50

2:10

PRICING & REIMBURSEMENT


Networking lunch12:20

End of conference 3:00


1/2 2/2

Roundtable sessions: 11:40

ROUNDTABLE 1: Expanded Access Programs: meeting (market) expectations and managing riskKeith Watts, Executive Vice President Strategic Services, One-World, Inc.

ROUNDTABLE 7: Pre-promotion vs. patient education: how does the greater good weigh against a potential risk?

ROUNDTABLE 3: Multi-stakeholder collaboration to accelerate rare disease clinical developmentScott Schliebner, Vice President of Scientific Affairs, Rare Diseases, Federal Work, PRA Health Sciences

ROUNDTABLE 9: Digitalizing collaborations because it doesn’t have to be that difficult

ROUNDTABLE 5: Drugmaker accountability: learning how J&J’s Velcade and Bayer’s Levitra were able to do this and survived

ROUNDTABLE 11: Public acclamation vs. science: guarding the orphan drug industry’s credibility

ROUNDTABLE 2: Balancing safety versus access for patients with rare diseasesStella Blackburn, Vice President, Global Head of Risk Management, Real-World & Late Phase Research, Quintiles

ROUNDTABLE 8: Markets other than US & Europe: where can I take my orphan drug next?

ROUNDTABLE 4: The complexities of healthcare, innovation and societal value for rare disease medicines Elizabeth White, Assistant Vice President, Rare Disease Commercial Development, Global Innovative Pharma Business, Pfizer

ROUNDTABLE 10: “Dream big detailed sales aid” early to identify the nitty gritty and not miss anything at Phase III

ROUNDTABLE 6: Generic/biosimilar play in orphan drugs: how to prepare for it

ROUNDTABLE 12: Expanded use vs. ‘Right to Try’: aren’t they the same?

Interested in moderating a roundtable? Contact Pinky Fadullon at +1 646 619 1785 or email her at [email protected]

April 21-22, 2016Full details of the agenda for Rare Disease Advocacy World 2016 and its impressive

speaking faculty can be found over the next few pages.

It was an impressive collection of stakeholders from both industry & patient

groups and I very much appreciated the opportunity to attend with a patient pass.

The roundtables featured great discussions

Stephanie Fischer, Patient

GROUP DISCOUNTS AVAILABLE at www.terrapinn.com/WODC2016

RARE DISEASE ADVOCACY WORLD SPEAKERS

Marc de Garidel Chariman and CEO, Ipsen

Claudia Hirawat Chair, International Circle of Ambassadors, EURORDIS

David Pena President, FEMEXER, Mexican Federation of Rare Diseases

Stephen Rose Chief Research Officer, Foundation Fighting Blindness

Bruno Hatschbach President, National Association of Rare Diseases, Brazil

Yann Le CamCEO, EURORDIS

Charles O’Neill VP, Pharmacological Sciences, BioMarin Pharmaceutical

Hatijah Ayob President, Malaysian Rare Disorders Society

Peter SaltonstallPresident and CEO, NORD

Nancy Silliman Vice President, Biostatistics, Epidemiology, Data Management, and Programming, Alexion Pharmaceuticals

Rashmi Gopal-Srivastava Director, Extramural Research Program, Office of Rare Diseases Research, NCATS, National Institutes of Health

Max Bronstein Senior Director of Public & Government Relations, EveryLife Foundation

Ron Guido SVP, Global Regulatory Affairs and Chief Compliance Officer, Veloxis Pharmaceuticals

Pat FurlongPresident & CEO, Parent Project Muscular Dystrophy

Yukiko Nishimura Chief Secretariat of International Relations, Japan Patients Association

Samit Hirawat Senior Vice President, Novartis Oncology

Alison Bateman-House Postdoctoral Fellow, Division of Medical Ethics, New York University Langone Medical Center

Virginia Llera President, Geiser Foundation, Argentina

FOR MORE INFORMATION contact Pinky Fadullon at [email protected] or +1 646 619 1785





8:00

8:50

8:55

11:00

CONFERENCE DAY ONE (Thursday, April 21st, 2016) CONFERENCE DAY ONE (Thursday, April 21st, 2016) 1/3 2/3

STATE OF THE INDUSTRY

PUTTING PATIENTS IN THE CENTER OF DRUG DEVELOPMENT

Keynote: Creating medicines with real value for orphan diseases

• The 21st century cures and Innovations acts: Progressing the science of treatment development for the rarest patient

• Approaches to development for the rarest of diseases

• Directing treatment to the disease burden of patients to optimize the development of meaningful drugs

Emil Kakkis, President & CEO, Ultragenyx

Nothing about us without us: driving research in retinal degenerative diseases through the patient’s eyes

• Putting the patient’s voice in the center of policy development

• Working with patients to gain consensus of issues that will affect them, their families and future

• Bringing all stakeholders to the table in order to educate patients on the complexities of drug development

Stephen Rose, Chief Research Officer, Foundation Fighting Blindness

Ipsen’s journey to develop a full range of products & services for the orphan drug population

• How do you address an orphan population with a wide range products?

• Complete disease management: from diagnostic tools, orphan drugs and second line treatments for disease progression

• Working with a patient social network to strengthen the measurement of treatment outcomes based on patient-generated data

Marc de Garidel, Chariman and CEO, Ipsen

9:00

REGULATORY, STATS, TOXICOLOGY

Current status and changing provisions for accelerated pre-approval drug access and other regulatory developments in orphan drug development

• Review of current and planned mechanisms of access to a drug prior to its approval; “right-to-try” statutes, established pathways for Compassionate Use, Expanded Access, Single Patient INDs and Conditional Approval

• Differences in dossier submissions in different regulatory pathways (US, EU and other key markets)

• Provisions for expedited marketing approvals and associated post market commitments

Ron Guido, SVP, Global Regulatory Affairs and Chief Compliance Officer, Veloxis Pharmaceuticals

Statistics as the only way you can extract meaning from clinical data and interpret its REAL value

• How developers design clinical trials and how developers and regulators interpret their results

• Hear about adaptive and Bayesian clinical trials and the impact on trial design, statistical analyses and duration

• Why this science does mean so much for patients

Nancy Silliman, Vice President, Biostatistics, Epidemiology, Data Management, and Programming, Alexion Pharmaceuticals

1:50

2:10

Nonclinical studies: use of normal animals and disease animal models for nonclinical safety programs supporting rare disease therapeutics

• Background on the regulatory landscape for nonclinical programs for rare diseases

• Nonclinical development strategies considerations for rare diseases

• Integrating normal animal and animal model toxicology with other disciplines to streamline rare disease drug development

Charles O’Neill, VP, Pharmacological Sciences, BioMarin Pharmaceutical

2:30

11:05

DRUG DISCOVERY & DEVELOPMENT

Drug development process from preclinical to commercialization and how this works for orphan drugs

• How are drugs discovered and how can this be optimized for rare diseases?

• Moving from compound discovery to preclinical phases to clinical trials to post-approval studies and final approval

• Running clinical trials with few patients and limited pathophysiological knowledge of the disease

Chistopher Trotta, Senior Director of Biology, PTC Therapeutics

11:45

How can we adapt drug development to address the urgent needs of orphan disorder populations?

• When it comes to safety and efficacy, are Phases 1, 2 and 3 absolutely necessary?

• Applying pathophysiologic knowledge and developing disease biomarkers early in a drug development program

• Optimizing logistics and infrastructure requirements when having few clinical sites

Samit Hirawat, Senior Vice President, Novartis Oncology

12:05

11:25

21st Century Cures and Innovations Acts: Opening the door to faster treatment development for devastating diseases or increasing the risk of approving drugs that are not effective or safe?

• What are the specific provisions for orphan drug development in the 21st Century Cures Act?

• What are the general provisions that can be applied to orphan diseases and aid in drug development?

• Is the act setting overly optimistic expectations to think that drugs can be approved easily?

• What will be the benefits for patients and drug manufacturers?

Richard Moscicki, Deputy Center Director for Science Operations, Center for Drug Evaluation and Research, FDA

Peter Saltonstall, President and CEO, NORD

Laurie Letvak, VP, Clinical Development Policy, Novartis

Emil Kakkis, President & CEO, Ultragenyx

9:20

Speed networking10:00


Networking lunch break 12:25


CONFERENCE DAY ONE (Thursday, April 21st, 2016) 3/3

BIOETHICS AND PATIENTS’ RIGHTS

COLLABORATIONS & PARTNERSHIPS

Ethical considerations: Patient’s rights when participating in rare disease trials

• Overview of key ethical issues in rare disease trials such as eligibility to participate, access to medication and receiving a placebo

• Post-trial considerations: Will you have access to your information? Will you be switched to the treatment group after the trial?

• How to minimize risk for patients while delivering treatment?

Claudia Hirawat, Chair, International Circle of Ambassadors, EURORDIS

When you don’t qualify for a trial: is ‘Right to Try’ (RTT) the solution to access?

• What ‘Right to Try’ (RTT) laws give patients and what they take away

• How ‘Right to Try’ (RTT) laws differ from the FDA’s compassionate use/expanded access program

• Pre-approval access issues, what are the key ethical considerations?

Alison Bateman-House, Postdoctoral Fellow, Division of Medical Ethics, New York University Langone Medical Center

Moving from patient centricity into patient partnerships

• How and where patients should intersect in the drug development process

• What is the language/ channels patients should use to interact with industry stakeholders?

• Building long-lasting partnerships with all the stakeholders in mind

Pat Furlong, President & CEO, Parent Project Muscular Dystrophy

Working with patients in the rare disease space to accelerate translation from discovery to health benefits

• Collaboration between patients and government institutions to support patient-centered research

• Improving collaboration, study enrollment and data sharing to benefit patients

• Helping physicians and scientists work together with patient advocacy groups to advance rare disease research

Rashmi Gopal-Srivastava, Director, Extramural Research Program, Office of Rare Diseases Research, NCATS, National Institutes of Health

2:50

3:50

4:10

4:30

Networking coffee break3:10

Networking cocktails sponsored by5:35


End of day one 6:35




8:00

8:50

8:55

1/2 2/2

INNOVATION: PROCESSES & DRUG DISCOVERY DEVELOPMENT APPROACH

RARE DISEASE PATIENT ADVOCACY ORGANIZATIONS OFFERING

PATIENT GROUPS FROM AROUND THE WORLD

CONFERENCE DAY TWO (Friday, April 22nd, 2016) CONFERENCE DAY TWO (Friday, April 22nd, 2016)

Targeting ‘bigger’ targets: increase nonsense mutation read through applicable to other rare diseases

Mark Rothera, Chief Commercial Officer, PTC Therapeutics

Addressing patient groups’ main challenges: the North American and European patient advocacy organizations perspective

Three of the main rare disease patient advocacy organizations will address some of the major challenges faced by patients. They will discuss their initiatives to help patients get involved with industry, regulators and other government institutions. In addition, they will talk about ways to mobilize resources in order to help patients get faster access to treatments and support them in having a louder collective voice that can push for better diagnosis and data sharing

How IRDiRC is establishing and providing access to harmonized data and samples

• How are patient groups working with regulators and other government institutions to strengthen policy-making and the focus on rare diseases?

• What are the different legal avenues to get access to treatment when treatment is not covered by insurance plans?

• Importance of patient groups to strengthen access to patients and the understanding of disease epidemiology

Hatijah Ayob, President, Malaysian Rare Disorders Society

Bruno Hatschbach, President, National Association of Rare Diseases, Brazil

David Pena, President, FEMEXER, Mexican Federation of Rare Diseases

Virginia Llera, President, Geiser Foundation, Argentina

Yukiko Nishimura, Chief Secretariat of International Relations, Japan Patients Association, ASrid

10:40

9:40

National Organization for Rare Disorders: identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services

Peter Saltonstall, President & CEO, NORD

1:30

European Organization for Rare Diseases: improving the quality of life of people living with rare through the promotion of health policies and patient empowerment and training

Yann Le Cam, Chief Executive Officer, EURORDIS

1:50

EveryLife Foundation for Rare Diseases: accelerating biotech innovation for rare disease treatments through science-driven public policy

Max Bronstein, Senior Director of Public & Government Relations, EveryLife Foundation

2:10

Networking coffee break10:00

Networking lunch 12:00

End of conference 2:40



Keynote: Adaptive biomedical innovation: re-engineering how we innovate

• The era of blockbusters, RCTs-only, and average patients is over!

• What is required to drive meaningful value for patients as innovation is “orphanized”?

• View this evolution through the design lens of MIT NEWDIGS – a global “think and do” tank for collaborative systems engineering across the traditional siloes of the innovation chain.

Gigi Hirsch, Executive Director, NEWDIGS and CBI, MIT

Keynote: A systematical approach to orphan drug discovery: starting with the unmet needs of payers and patients

• Has anything changed in orphan drug reimbursement?

• Moving away from target discovery: how to meet payer’s and patient’s needs

• How can pharma justify the high prices of orphan drugs to payers?

Edmund Pezalla, Vice President, National Medical Director, Pharmacy Policy and Strategy, Aetna

9:30

9:00

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THE EXHIBITION

*Correct as of November 11, 2015

INTERESTED IN SPONSORING OR EXHIBITING? Contact Pinky Fadullon at [email protected] or +1 646 619 1785

• Pharma/Biotech

• Market Research consultants

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• CROs

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• Marketing & Communications Consultants

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• Keeping your brand front of mind

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OUR PACKAGES OFFER HIGH VALUE INCLUDING

• The chance to show over 200 pharma and

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expedite orphan drug development

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meetings for you

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establish thought leadership

WHY EXHIBIT? PURSUE AND PARTNER

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PHARMA - Head, SVP, VP, Director of:

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BIOTECH:• CEO

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UNIVERSITIES - Professors and Heads

Academic medical center chiefsPayers & regulators

• 300+ of partnering meetings in 2 days

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• 5+ hours of assisted networking during 40-minute coffee breaks and 1 ½ hour lunch breaks

• Over 30 intimate roundtable, focus-group discussions to participate in

• 1-2-1 Speed Networking session

• An end of day one Cocktail Hour!

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Join us once the conference sessions are finished on Day One for Networking Cocktails.

TOP 11 REASONS TO ATTEND


1. Discuss international orphan drug policy developments from regulators and stakeholders in the U.S., Europe, Malaysia, Chile, Japan, Canada and the Philippines.

3. Understand the ethics of Expanded Access in the US and what ‘Right To Try’ really is from Art Caplan, Head of the Division of Medical Ethics at the NYU Langone Medical Center.

6. Join James Greenwood, from the Biotechnology Industry Organization, Ian Phillips from the Keck Graduate Institute of Applied Life Sciences and Steve Usdin from BioCentury in the pricing debate for orphan drugs and hear about appropriate pricing models for a one-time transformational gene therapy vs.orphan drugs for life.

9. Explore how stakeholders are looking to improve the processes behind orphan drug development when Yann Le Cam shares Europe’s Medicine Adaptive Pathways to Patients (MAPPs).

4. Learn about the challenges faced by physicians in the rare disease space and how industry can engage Key Opinion Leaders strengthening patient outreach and clinical trial performance.

7. Hear Alvin Shih from Retrophin explain the accelerated approval (subpart H) pathway and its relevance to rare disease drug development.

10. Join Biomarin, Alexion, PTC, Veloxis, Novartis, EURORDIS and the NIH at the Rare Disease Advocacy World discuss the nitty gritty of R&D, regulatory, patients’ rights, bioethics and collaboration.

2. Hear about the advocacy work done by patient groups from Argentina, Mexico, Brazil, Japan and Malaysia.

5. Understand how the the 21st Century Cures and Innovations Act can facilitate faster treatment development for devastating diseases or increase the risk of approving drugs that are not effective or safe from Novartis, NORD, Ultragenyx and the FDA.

8. Uncover the latest innovations in our Pitch & Partner session. We’re bringing even more companies than last year. Meet with 35 biotechs looking for partners.

11. Examine how Aetna is creating a systematic process to orphan drug discovery by starting with the unmet needs of payers and patients.

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