january 29-30, 2018 hilton crystal city arlington, va ... · endpoints, and endpoints that reflect...
TRANSCRIPT
Drug Development Breakthrough
Therapy
Accelerated Approval
Regulatory Policy
Expedited Review
Priority Review
January 29-30, 2018 // Hilton Crystal City // Arlington, VA
Innovative Regulatory PathwaysRegulatory Strategies to Expedite Patient Care
SUMMIT
Jennifer Dudinak, Pharm.D., Vice President, Global Regulatory Affairs, Head, Pharma Regulatory Therapeutic Team, GLAXOSMITHKLINE
Stephen Hoffman,CEO, SANARIA INC
John Billington, Director, U.S. Public Policy, Vaccines, GLAXOSMITHKLINE PLC
Jayne C. Gershkowitz, Senior Vice President and Chief Patient, Advocate, AMICUS THERAPEUTICS
Mark Stewart, Ph.D., Senior Science Policy Analyst, FRIENDS OF CANCER RESEARCH
Dr. Henrietta Ukwu, Senior Vice President, Head of Global Regulatory Affairs, OTSUKA PHARMACEUTICAL COMPANIES
Alejandro D. Ricart, M.D., Executive Medical Director, Oncology, Early Development Strategy and Innovation, NOVARTIS
Andrew Storey, Vice President, Global Regulatory Strategy, ABBVIE
Conference Chair: Keynote Speaker:
WHO SHOULD ATTENDThis conference is designed for representatives from pharmaceutical, biotechnology, and medical device companies and advocacy groups with responsibilities in the following areas:
• Regulatory Affairs/Strategy/Operations• Regulatory Policy• Business Development/Strategy• Clinical Operations/Development/Affairs• Product Development• R&D/Drug Development• Patient Advocacy• Federal Government Relations• HEOR and Outcomes Research• Strategic Planning• Competitive Intelligence• Oncology Program Management• Rare Disease Program Management• Pipeline/Portfolio Management• Pharmacovigilance• Medical Information/Affairs
This conference is also of interest to:• Regulatory Advisors/Service Providers/
Consultants • Clinical/Contract Research
Organizations• Drug Development Service Providers• Law Firms• Preclinical/Analytical Research
Organizations• Strategic/Management Consultants
VENUEHilton Crystal City at Washington Reagan National Airport2399 Jefferson Davis Hwy Arlington, VA 22202To make reservations, please call 1-800-695-7551 and request the negotiated rate for ExL’s January meetings. You may also make reservations online using the following weblink: http://bit.ly/2ulR93E. The group rate is available until January 9, 2018. Please book your room early, as rooms available at this rate are limited. *ExL Events, Inc. is not affiliated with Exhibition Housing Management (EHM)/ Exhibitors HousingServices (EHS) or any third-party booking agencies, housing bureaus, or travel and events companies. In the event that an outside party contacts you for any type of hotel or travel arrangements, please disregard these solicitations and kindly email us. ExL has not authorized these companies to contact you and we do not verify the legitimacy of the services or rates offered. Please book your guest rooms through ExL’s reserved guest room block using the details provided.
SPONSORSHIP AND EXHIBITION OPPORTUNITIESDo you want to spread the word about your organization’s solutions and services to potential clients attending this event? Take advantage of the opportunity to exhibit, underwrite an educational session, host a networking event or distribute promotional items to attendees. ExL Events will work closely with you to customize a package that will suit all of your needs.
Dear Colleague,
There are regulatory mechanisms in place to accelerate the review and/or approval of treatments for patients. In the United States, Accelerated Approval, Breakthrough Therapy Designation, Fast Track Designation, Priority Review, and the Orphan Drug Act are designed to expedite treatment availability for medications that provide an increased benefit over existing treatments.
Breakthrough Therapy Designations and other expedited pathways encourage sponsors to increase communication and collaboration with the FDA, to enable efficient drug development. Accelerated drug development options are also available globally in ICH countries through programs like PRIME and Sakigake. Understanding how to best utilize these regulatory options is imperative to make an informed decision about the best ways to accelerate drug development.
ExL’s Innovative Regulatory Pathways Summit will bring together leading regulatory experts and thought leaders to discuss breakthrough therapy designations, accelerated approvals, and global regulatory strategies to expedite review/approval of drugs that have promise in treating serious or life–threatening conditions.
Sincerely,
Dario Dario CavaliereConference Production Director ExL Events
Drug Development
Breakthrough Therapy Expedited
Review
8:00 Open Registration and Continental Breakfast
8:50 Conference Chair Opening Remarks Alejandro D. Ricart, M.D., Executive Medical Director, Oncology, Early Development Strategy and Innovation, NOVARTIS
9:00 AbbVie’s Experience With Breakthrough Therapy Designation• Understand the type of data required for BTD eligibility• Optimize the application process and communication with the
FDA• Utilize increased communication to effectively expedite drug
developmentAndrew Storey, Vice President, Global Regulatory Strategy, ABBVIE
9:45 Breakthrough Therapy Designations in Oncology• Provide safe and effective novel oncology drugs to market
quickly for life-threatening diseases and unmet needs• Identify the importance of clinical trial design and clinical
setting: Pivotal study vs. confirmatory study• Discuss surrogate endpoints, well-established surrogate
endpoints, and endpoints that reflect patient benefit• Understand concern over the timely conduct of postmarketing–
study commitmentsAlejandro D. Ricart, M.D., Executive Medical Director, Oncology, Early Development Strategy and Innovation, NOVARTIS
10:30 Networking Break
11:00 Impact of Breakthrough Therapy Designation on Clinical Trial Design • Understand the implications of receiving a designation on
Phase II and Phase III• Collaborate with the FDA to obtain the best clinical data• Clinical data management for expedited drug development• Find the pathway for generating evidence needed about safety
and efficacy in a narrow populationJoseph A. Sliman, M.D., MPH, Chief Medical Officer, SYNTHETIC BIOLOGICS
11:45 Harmonize Manufacture Readiness Plans With Accelerate Drug Development • Discuss the benefits and impacts of increased communication
with the FDA on clinical trial design and CMC plans• Implement and review CMC plans during early clinical
development• Accelerate manufacturing plans in pace with expedited clinical
trial design• Discuss safety and regulatory considerations for accelerated
manufacturingUsha Ramesh, Ph.D., PMP, Executive Director, Regulatory Affairs, CMC, PHARMACYCLICS, AN ABBVIE COMPANY
12:30 Luncheon
1:30 Sakigake Designation System: A Japanese Strategy for Expedited Drug Development• Review a brief history of why the Sakigake Designation System
was derived• Understand the benefits of receiving Sakigake designation• Discuss global development timelines for Sakigake submission• Implement regulatory strategies to obtain clinical data for
Sakigake application• Incorporate Sakigake/PMDA in global development:
engagement, clinical studies, and submission• Regulatory considerations for applying and maintaining
Sakigake designationColin Vechery, Manager, U.S. Regulatory Affairs–Strategy, BIOGEN
2:15 Global Regulatory Options for Accelerated Drug Development • Discuss the challenges and benefits of utilizing FDA and EMA
expedited review pathways• Review the expedited regulatory options in non–ICH regions• Compare the eligibility requirements for breakthrough therapies
and PRIMEPanelists: Jennifer Dudinak, Pharm.D., Vice President, Global Regulatory Affairs, Head, Pharma Regulatory Therapeutic Team, GLAXOSMITHKLINEDr. Henrietta Ukwu, Senior Vice President, Head of Global Regulatory Affairs, OTSUKA PHARMACEUTICAL COMPANIESJerry Stewart, JD, MS, RPh, Deputy Vice President, Scientific & Regulatory Advocacy, PHARMACEUTICAL RESEARCH AND MANUFACTURERS OF AMERICA (PHRMA)Lawrence Liberti, MSc, Executive Director, CENTER FOR INNOVATION IN REGULATORY SCIENCE (CIRS)
3:15 Networking Break
3:45 Coordinating Global Submissions: How to Reuse Content for Emerging Markets• Harmonize regulatory operations to submit to multiple global
regulatory bodies simultaneously• Discuss Eisai’s experience reducing delays by implementing
global strategies• Learn timesaving options to maximize submission efficiency to
the FDASophia A. Kourliouros, Senior Manager, Global Regulatory Operations, EISAI INC.
4:30 Interact Effectively With the FDA to Accelerate Development of Novel Malaria Vaccine Candidates for a Global Unmet Need• Discuss the global impact of a whole-parasite malaria vaccine• Collaborate with regulatory bodies to enable clinical
development in the developing world• Communicate with the FDA regarding global health
considerationsStephen Hoffman, CEO, SANARIA INC
5:15 Day One Concludes
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Day One Monday, January 29, 2018
12:30 Luncheon
1:30 Integrate Patient Advocacy Groups Into Drug Development Programs to Navigate the Expedited Regulatory Options• Compare eligibility requirements across available expedited
pathways• Examine the benefits and challenges of creating patient-
focused product development plans• Discuss best practices for engaging advocacy groups in early
stages of development• Consider the impact of BTD on the FDA resource allocationBrian E. Harvey, M.D., Ph.D., Executive Vice President, Scientific and Regulatory Affairs, GLOBAL LIVER INSTITUTE; Former Vice President U.S. Regulatory Strategy, PFIZER
2:15 Effective Collaboration Between Drug Developers and Patient Advocates • Utilize data-driven insights to understand the impact of the
patient perspective on regulatory strategies• Establish endpoints that are both clinically significant and
meaningful for the patient• Maximize clinical development efficiency through patient-
focused drug developmentModerator: Brian E. Harvey, M.D., Ph.D., Executive Vice President, Scientific and Regulatory Affairs, GLOBAL LIVER INSTITUTE; Former Vice President U.S. Regulatory Strategy, PFIZER Panelists: Jayne C. Gershkowitz, Senior Vice President and Chief Patient Advocate, AMICUS THERAPEUTICSRon Bartek, President and Co-Founder, FRIEDREICH’S ATAXIA RESEARCH ALLIANCE (FARA)Stacy Woeppel, Advocate in Science, SUSAN G. KOMEN
3:15 Networking Break
3:45 Utilize Expedited Review Pathways in Precision Medicine• Identify clinical endpoints based on available treatments and
disease progression• Demonstrate non–progression of the disease to gain BTD
eligibility• Discuss regulatory strategies to earn conditional approval to
expedite treatment availability• Understand the options for expedited review for targeted
oncology therapiesChristopher Adams, CEO, ANDARIX PHARMACEUTICALS
4:30 Implications of Personalized Medicine on Clinical Trial Design and Expedited Review Pathways • Understand the potential role of personalized medicine in
clinical development• Discuss accelerated solutions for patients with unmet needs
using personalized methodologies• Empathize the impact of personalized medicine on regulatory
strategiesChitra Edwin, Ph.D., RAC, Senior Vice President of Regulatory Affairs and Compliance, SPOTLIGHT INNOVATION, INC
5:15 Conference Concludes
PAN
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8:00 Open Registration and Continental Breakfast
8:50 Conference Chair Opening Remarks Alejandro D. Ricart, M.D., Executive Medical Director, Oncology, Early Development Strategy and Innovation, NOVARTIS
9:00 Development and Implementation of Whole-Parasite Malaria Vaccine • Discuss the benefits and challenges of working with U.S. and
foreign regulatory officials• Consider the impact of fast-track designation on the review
process• Analyze the eligibility requirements for breakthrough therapy
designationTooba Murshedkar, Senior Director, Regulatory Affairs, SANARIA INC.
9:45 A Public Policy Perspective on Regulatory Policies and Market-Shaping Considerations for Emerging Infectious Diseases • Compare the protocols in place for expedited access to
vaccines during a public health emergency• Discuss the impact of ACIP recommendations on portfolio
decisions and market access for vaccines• Evaluate existing policies to maximize market access in
uncertain markets for infectious disease products• Consider innovative business model proposals for emerging
infectious disease and how they might interact with existingregulatory policies
John Billington, Director, U.S. Public Policy, Vaccines, GLAXOSMITHKLINE
10:30 Networking Break
11:00 Expedited Options for Competitive Generic Therapies • Consider the impact of GDUFA II on earlier access and
increased competition between generic therapies• Understand updated fee structures and effects on small
businesses• Identify the Impact on prioritization and approval of First
Generics• Discuss the effectiveness of the updated submission protocols
and impact on market dynamicsPanelists: Lisa Parks, R.Ph., Vice President, Sciences and Regulatory Affairs, ASSOCIATION FOR ACCESSIBLE MEDICINESNicholas Tantillo, Head, Policy and Regulatory Strategy, SANDOZ INC
11:45 Utilize Patient–Focused Drug Development to Build Clinical Trial Frameworks for Breakthrough Therapy Applications• Discuss the current and potential applications of breakthrough
therapies in oncology and other disease spaces• Understand the role of patients and advocacy groups in
informing effective clinical development programs• Pathways for patient engagement in clinical development
programsMark Stewart, Ph.D., Senior Science Policy Analyst, FRIENDS OF CANCER RESEARCH
Day Two Tuesday, January 30, 2018PA
NEL
EARLY BIRD PRICINGRegister by Friday, December 15, 2017
Conference $1,895
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Conference $2,095
ONSITE PRICING
Conference $2,295
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January 29-30, 2018 // Hilton Crystal City // Arlington, VA
SUMMIT
20+ Accelerated Approvals
50+ Breakthrough
Therapy Designations
24 Organizations
585+ Years of
Experience
22 Speakers
Combined With
and
Representing
Regulatory Strategies to Expedite Patient Care
Innovative Regulatory Pathways