CDER Office of New Drug Update. Here an update on developments in OND and what is currently in the pipeline for 2018.

Transcription

1 DAY 1-05/12/2017 Registration 7:15am - 8:00am Informal Breakfast Roundtable Discussion 8:00am - 8:45am Panelist: Matthew Might, PhD - Director, Hugh Kaul Precision Medicine Institute, University of Alabama at Birmingham Panelist: Sarfaraz Niazi, PhD - Adjunct Professor, Department of Biopharmaceutical Sciences, UIC College of Pharmacy Panelist: Janet Woodcock, MD - Director, Center for Drug Evaluation & Research (CDER), U.S. Food and Drug Administration (FDA) Chairman s Opening Remarks 8:45am - 9:00am Chairman: Nielsen Hobbs - Editor, The Pink Sheet Opening Keynote Address 9:00am - 9:30am Keynote Addresses Keynote Speaker: Scott Gottlieb, MD - Commissioner of Food and Drugs, The Food and Drug Administration (FDA) Keynote Address: The Center for Drug Evaluation and Research in 2017 and the Year Ahead 9:30am - 10:00am Keynote Addresses Here an update from CDER on current initiatives, challenges and accomplishments of 2017 and what s in store for the year ahead Keynote Speaker: Janet Woodcock, MD - Director, Center for Drug Evaluation & Research (CDER), U.S. Food and Drug Administration (FDA) CDER Office of New Drug Update 10:00am - 10:30am Here an update on developments in OND and what is currently in the pipeline for Patrick Frey - Senior Advisor to the Director, Office of New Drugs CDER, Food & Drug Administration Networking Refreshment Break 10:30am - 11:00am Update on Drug Safety and Post Market Surveillance 11:00am - 11:30am Here an update from the director on the latest developments from the office of Surveillance and Epidemiology. Gerald Dal Pan, MD, MHS - Director, Office of Surveillance and Epidemiology, U.S. Food and Drug Administration (FDA) Keynote Panel: Industry Follow up on Morning Keynotes, Office of New Drug Updates 11:30am - 12:30pm Sit in on a panel discussion in follow up to the morning s keynotes and updates. What are the current trends and developments affecting the industry, key industry implications of regulatory policies and updates, as well as industry insight on what is anticipated for the year ahead. Moderator: Meg Tirrell - Biotech and Pharma Reporter, CNBC Panelist: Karen Hauda - Senior Director; Clinical, Medical and Regulatory Affairs, Novo Nordisk Inc. Panelist: John Maraganore, PhD - CEO, Alnylam Pharmaceuticals, Inc Panelist: Peter Honig - Senior Vice President, Worldwide Safety and Regulatory, Pfizer Understanding the Emerging Role of Patient Reported Outcomes (PRO) and FDA Use in Drug Programs 1:30pm - 2:15pm Patient reporting has not historically held much sway in FDA considerations, but the tide has been turning. As the FDA more seriously considers including patientreported outcomes (PRO) as part of its approval processes, examine the potential impact on drug manufacturers as well as what this will mean for the approval process moving forward and current developments and considerations for individual diseases and treatments. Panelist: Cynthia Grossman, PhD - Associate Director, Science of Patient Input, FasterCures Milken Institute Panelist: Christine McSherry - Executive Director, Jett Foundation Panelist: Carmen Bozic, MD - Vice President, Global Development, Biogen Moderator: Kay Holcombe - Senior Vice President, Science Policy, Biotechnology Innovation Organization (BIO) (retd.) Update: Current Political and Regulatory Proposals and Developments Impacting Pharma and Healthcare 2:15pm - 2:45pm Discuss the latest DC developments, what is coming down the pipeline? Identify and examine current proposals that are circulating the Hill which would impact the Bio/Pharma and healthcare industries. Hector De La Torre - Executive Director, Transamerica Center for Health Studies Networking Refreshment Break 2:45pm - 3:15pm Networking Lunch 12:30pm - 1:30pm

2 DAY 1-05/12/2017 The Opportunities and Challenges of Expanded Access 3:15pm - 4:00pm Cocktail Reception 5:00pm - 6:00pm This session examines best practices and modern approaches to integrating well-designed large group access programs into the drug development process. Examine the current requirements of all expanded access uses and its evolution, as well as those specifically for investigational drugs and biologics What are the practical, ethical and legal issues to be considered? Discuss the drivers of commercial feasibility including cost recovery, regulatory considerations, and pragmatic design. Panelist: Beth Roxland, JD - Senior Consultant on Law, Ethics and Policy Associate,, NYU Langone Medical School Moderator: Jess Rabourn - Managing Director, Wide Trial Panelist: Jonathan Jarow - Senior Medical Advisor, Center for Drug Evaluation and Research (CDER), Food and Drug Administration (FDA) Panelist: Shimere Sherwood PhD - Associate Director, NIH and FDA Advocacy, Policy and Advocacy, American Society of Clinical Oncology Panelist: Martin Naley - General Manager, My Tomorrows Keynote Fireside Chat : The Future of Precision Medicine 4:00pm - 5:00pm Sit in on a discussion on the intersection of ethics, innovation and precision medicine, from patient to pill. Matthew Might, Ph.D., a strategic leader appointed to the White House Precision Medicine Initiative by former President Barack Obama, has been named the inaugural director of the Hugh Kaul Personalized Medicine Institute at the University of Alabama at Birmingham School of Medicine.Might comes to UAB from the University of Utah, where he is a Presidential Scholar and an associate professor in both computer science and pharmaceutical chemistry, and from Harvard Medical School, where he is a visiting professor of biomedical informatics. Might s research interests focus on the intersection of computation and medicine to advance precision medicine through personalized therapeutics. Keynote Speaker: Matthew Might, PhD - Director, Hugh Kaul Precision Medicine Institute, University of Alabama at Birmingham Moderator: Nicole Fisher - Contributor; Founder & CEO, Forbes Magazine; HHR Strategies

3 SCHEDULE DAY 1-05/12/2017 TIME 7:00AM 8:00AM 9:00AM 10:00AM 11:00AM 12:00PM 1:00PM 2:00PM 3:00PM 4:00PM 5:00PM KEYNOTE ADDRESSES 7:15am - Registration 8:00am - Informal Breakfast Roundtable Discussion 8:45am - Chairman s Opening Remarks 9:00am - Opening Keynote Address 9:30am - Keynote Address: The Center for Drug Evaluation and Research in 2017 and the Year Ahead 10:00am - CDER Office of New Drug Update 10:30am - Networking Refreshment Break 11:00am - Update on Drug Safety and Post Market Surveillance 11:30am - Keynote Panel: Industry Follow up on Morning Keynotes, Office of New Drug Updates 12:30pm - Networking Lunch 1:30pm - Understanding the Emerging Role of Patient Reported Outcomes (PRO) and FDA Use in Drug Programs 2:15pm - Update: Current Political and Regulatory Proposals and Developments Impacting Pharma and Healthcare 2:45pm - Networking Refreshment Break 3:15pm - The Opportunities and Challenges of Expanded Access 4:00pm - Keynote Fireside Chat : The Future of Precision Medicine 5:00pm - Cocktail Reception

4 DAY 2-06/12/2017 Registration & Breakfast 7:15am - 7:45am Round-table Breakfast Discussion: Sustainable Health Care Funding 7:45am - 8:30am Chairman s Opening Remarks 8:40am - 8:45am 1. Chairman: Nielsen Hobbs - Editor, The Pink Sheet Opening Keynote Address and Fireside Chat: Partnering Opportunities with Industry to Solve Market Challenges 8:45am - 9:30am CMS Administrator Verma's address will be immediately followed by a fireside chat with FDA/CMS Summit chair, Nielsen Hobbs Speaker: Seema Verma - Administrator, Centers for Medicare and Medicaid Services (CMS) Moderator: Nielsen Hobbs - Editor, The Pink Sheet Pricing, Value and Leverage: Addressing the Ever Growing Cost of Drugs Through New Channels 9:30am - 10:15am Continuing the ongoing discussion around pricing, hear industry perspectives on the driving forces behind the continual increase in drug prices, the role of value in controlling cost as well as what, if any, tools and leverage federal agencies like FDA and CMS have in addressing the issue. Moderator: Meg Alexander - Head, Reputation & Risk Management Practice, InVentiv Health Panelist: Hervé Hoppenot - Chairman, President, and Chief Executive Officer, Incyte Panelist: Lindsay Rosenwald, MD - Chairman, President & Chief Executive Officer, Fortress Biotech Panelist: Jonathan Jarow - Senior Medical Advisor, Center for Drug Evaluation and Research (CDER), Food and Drug Administration (FDA) Networking Refreshment Break 10:15am - 10:45am Obstacles, Reimbursement, and Leverage Opportunities for Boosting Competition and Adoption of Biosimilars in US Markets 10:45am - 11:30am This presentation will examine the 2017 developments surrounding biosimilars in the United States like the Supreme Court decision in June, and the recent CMS reversal on Biosimilar coding and reimbursement policy for Medicare. What are the key mistakes that biosimilar developers are making repeatedly? What are some cost effective approaches for the development of new biosimilars? Is the stage set to change in the US Markets for Biosimilars in 2018 and beyond? Moderator: Nicholas Florko - Associate Editor, Inside Health Policy Panelist: Aaron Hakim - Researcher, Yale School of Medicine Panelist: Sarfaraz Niazi, PhD - Adjunct Professor, Department of Biopharmaceutical Sciences, UIC College of Pharmacy Panelist: Joe Franklin JD, PhD - Associate Director for PolicyTherapeutic Biologics and Biosimilars Staff (TBBS),, Office of New Drugs, CDER, FDA Panelist: Robert Popovian - Senior Director, US Government Relations, Pfizer Pharmaceuticals Fast Track vs Breakthrough Therapy Designation - Small Business Perspective 11:30am - 12:00pm Hear perspective from a a small biotech company that has been granted fast track designation and breakthrough therapy designation in some of its programs on the differences, challenges, and opportunities presented by these two designations. Catherine Melfi, PhD - Chief Regulatory Officer, Omeros Corporation Lunch 12:00pm - 1:00pm Rare Diseases: Trends, Developments and PRVs 1:00pm - 1:45pm Panelist: Jonathan Goldsmith, MD - Associate Director Rare Disease Program, Center for Drug Evaluation and Research (CDER) Food and Drug Administration (FDA) Panelist: Ron Cooper - President and CEO, Albireo Pharma Innovations in the Science of Patient Input 1:00pm - 4:00pm Half-Day Workshop Stakeholders across R&D have a significant interest in collecting data directly from patients to understand their unmet medical needs, perspectives on meaningful outcomes, and preferences when making medical decisions as well as to understand how medical products behave in the real world. This halfday workshop brings together patient organizations, regulators, payers, researchers, and medical product developers interested in advancing the science of patient input. The sessions will highlight current innovations in the collection and use of patientgenerated data (e.g., patient-reported outcomes, preferences, information from wearables, etc.) and identify approaches to advance patient-centered innovation in medical device development and delivery. Workshop participants will: Hear directly from patients and patient organizations about their efforts to innovate on patient-generated data and what value these innovations will bring to patients. Learn about how real-world evidence can be derived from patient-generated data and influence decision-making by regulators, payers, and the research community. Identify opportunities for innovations from within the device industry to collect and use patientgenerated data in decision-making. Panelist: Kim McCleary - Managing Director, Acting Executive Director, FastCures Panelist: Gregory Daniel PhD - Deputy Director and Clinical Professor, Duke-Margolis Center for Health Policy Panelist: M. Suzanne Schrandt - Director, Patient Engagement, Arthritis Foundation Panelist: Jeff Shuren MD - Director, Center for Devices and Radiological Health, Food and Drug Administration (FDA) Panelist: Josh Seidman - Senior Vice President, Avalere Health

5 DAY 2-06/12/2017 Off Label Developments: The Future of Product Communications 1:45pm - 2:30pm Discuss the current standing on FDA product communications and the implications for Address best practices, education and process: Ensure your representatives are utilizing known and understood information about your product. Understand why the need for fair balance is still appropriate Discuss the need for compliance training for traditional representatives. Address the credibility of the HCP/Patient interaction and why each company has an obligation to make sure the HCP understands the total picture Panelist: Kevin Ryan - Senior Director, Compliance & Ethics, Novo Nordisk Panelist: Peter Marchesini - Chief Operating Officer, Alamo Pharma Services The Impact and Implications of the "right to try" Movement 2:30pm - 3:15pm Recent legislation has passed in various states regarding right to try an issue also widely represented in news media and the political landscape of the past year. This session will focus on the recent RTT movement and its impact (if any) on industry developments, and will also explore the wider implications of increased public and political scrutiny on specific areas and issues within the drugs and biologics industry. Moderator: Meg Tirrell - Biotech and Pharma Reporter, CNBC Panelist: Alison Bateman-House, PhD, MPH, MA - Division of Medical Ethics, NYU Langone Health Panelist: Christina Sandefur - Executive Vice President, Goldwater Institute Panelist: Tom Watson - Executive Director, TW Consulting Group Main Conference Concludes 3:15pm - 3:20pm Workshop Concludes 4:00pm - 4:05pm Half-Day Workshop

6 SCHEDULE DAY 2-06/12/2017 TIME HALF-DAY WORKSHOP PLENARY SESSIONS 7:00AM 8:00AM 9:00AM 10:00AM 7:15am - Registration & Breakfast 7:45am - Round-table Breakfast Discussion: Sustainable Health Care Funding 8:40am - Chairman s Opening Remarks 8:45am - Opening Keynote Address and Fireside Chat: Partnering Opportunities with Industry to Solve Market Challenges 9:30am - Pricing, Value and Leverage: Addressing the Ever Growing Cost of Drugs Through New Channels 10:15am - Networking Refreshment Break 10:45am - Obstacles, Reimbursement, and Leverage Opportunities for Boosting Competition and Adoption of Biosimilars in US Markets 7:15am - Registration & Breakfast 7:45am - Round-table Breakfast Discussion: Sustainable Health Care Funding 8:40am - Chairman s Opening Remarks 8:45am - Opening Keynote Address and Fireside Chat: Partnering Opportunities with Industry to Solve Market Challenges 9:30am - Pricing, Value and Leverage: Addressing the Ever Growing Cost of Drugs Through New Channels 10:15am - Networking Refreshment Break 10:45am - Obstacles, Reimbursement, and Leverage Opportunities for Boosting Competition and Adoption of Biosimilars in US Markets 11:00AM 11:30am - Fast Track vs Breakthrough Therapy Designation - Small Business Perspective 11:30am - Fast Track vs Breakthrough Therapy Designation - Small Business Perspective 12:00PM 12:00pm - Lunch 12:00pm - Lunch 1:00PM 1:00pm - Innovations in the Science of Patient Input 1:00pm - Rare Diseases: Trends, Developments and PRVs 1:45pm - Off Label Developments: The Future of Product Communications 2:00PM 3:00PM 2:30pm - The Impact and Implications of the "right to try" Movement 3:15pm - Main Conference Concludes 4:00PM 4:00pm - Workshop Concludes