subject to change
All Times are in Eastern Time

Required Pre-Course Work Advocacy Education Course
(90 minutes)
An online advocacy education program and community working to help patients, advocates, and caregivers acquire the necessary tools to effectively communicate with drug researchers, developers, and regulators.

Tuesday, November 29

11:30–11:40 AM
Welcome and Announcements

11:40 AM–12:40 PM
Incorporating Patient Experience into Drug Development and FDA Policy (Part 1)
Joseph F. Lamendola, Senior Director, Faegre Drinker Consulting
David R. Zook, Chair, Faegre Drinker Consulting

  • FDA Advisory Committees, Workshops, and Meetings
  • Speaking as a Patient
  • Patient Representatives
  • Role of Patient Advocates
  • Commenting on Draft Guidances, Proposed Regulations, and Other FDA Publications

12:45–1:30 PM
Incorporating Patient Experience into Medical Product Development: Fireside Chat
Robyn Bent
, Director, Patient-Focused Drug Development Program, Center for Drug Evaluation and Research (CDER), FDA
Elizabeth Hart, Branch Chief, Division of Clinical Evaluation and Pharmacology/Toxicology (DCEPT), Office of Tissues and Advanced Therapies (OTAT), Center for Biologics Evaluation and Research (CBER), FDA
Kerry Jo Lee, Associate Director for Rare Diseases, Office of New Drugs, Center for Drug Evaluation and Research (CDER), FDA
Vasum Peiris
, Chief Medical Officer and Director, Pediatrics and Special Populations, Center for Devices and Radiological Health (CDRH), FDA
Moderated by Peter J. Pitts, President, Center for Medicine in the Public Interest

  • Changes in Patient Voice in Drug and Device Development
  • Influence of the Patient Voice on Clinical Trial Design and Recruitment
  • The Role of Real-World Evidence (RWE), Quality of Life Data (QoL), and Patient Reported Outcomes (PROs) on the Way Reviewers Consider End Points and Other Regulatory milestones
  • Engaging the Patient Voice in More Robust Post-Market Surveillance
  • Steps FDA Can Take to Integrate the Patient Voice More Fully into Review Protocols

1:30–1:45 PM

1:45–2:30 PM
What Patient Organizations Should Know About Regulation of Cell & Gene Therapies
Eva A. Temkin, Partner, King & Spalding LLP
Elaine H. Tseng, Partner, King & Spalding LLP

  • Overview of Product Regulation
  • Increasing Role of Targeted Therapies and Immunotherapies
  • Stem Cell Therapies: Overlap and Differences
  • RMAT Designation
  • End of Enforcement Discretion Period

2:302:45 PM

2:45–3:45 PM
Patient Access to Other Agencies and Lawmakers
Karin Hoelzer, Director, Policy and Regulatory Affairs, National Organization for Rare Disorders (NORD)
Cara Tenenbaum, Principal, Strathmore Health Strategy

  • Congressional Jurisdiction and Oversight
  • The Relationship Between NIH, FDA, and CMS
  • Impact on Supply Shortages, Infant Formula, and More
  • The Relationship Between Industry and Agencies
  • OTC Hearing Aid, An Example of Working Together to Get It Accomplished

3:50–4:50 PM
Accelerated Approval Expedited Pathways
Amy Abernethy, President, Clinical Studies Platform, Verily Life Sciences LLC
Joe Franklin, Head of Strategic Affairs, Clinical Studies Platform, Verily Life Sciences LLC

  • History and Development of Accelerated Approval Pathway
  • Approvals Based on Surrogate and Immediate Endpoints versus Direct Evidence of Clinical Benefit
  • Confirmatory Studies and Post-market Authorities
  • FDA: Regulator of and Partner with Industry
  • June 2021 Accelerated Approval of Alzheimer’s Drug
  • Patient Reported Outcomes and Real-World Evidence

Wednesday, November 30

12:00–1:00 PM
Engaging with FDA: Opportunities and Boundaries
Andrea Furia-Helms, Director, Office of Patient Affairs, Office of the Commissioner, FDA
Michelle Tarver, Director, Patient Science and Engagement, Center for Devices and Radiological Health (CDRH), FDA

  • Patient Initiatives
  • FDA Patient Engagement Activities
  • Patient Focused Drug Development
  • Resources for Patients
  • Patient Engagement in Medical Devices

1:05–2:05 PM
Patient Access to Treatments
Richard Klein, Director, Expanded Access Programs & Policy, GE2P2 Global Foundation

  • FDA Guidance and Changes for Clinical Trials
  • Overview and How to Navigate
  • Diversity and Inclusion in Clinical Trials
  • Traveling for Patient Access to Therapies
  • Informed Consent
  • Safety Reporting
  • Expanded Access and the Right to Try
  • Withdrawn Drugs
  • Off-Label Use
  • Drug Repurposing

2:05–2:20 PM

2:20–3:20 PM
Lessons Learned from COVID-19
Melissa Barhoover, Senior Regulatory Affairs Manager, Becton Dickinson (BD)
Anne Zavertnik, Vice President, Regulatory Affairs, Integrated Diagnostic Solutions, Becton Dickinson (BD)

  • EUA Process and Vaccines
  • Device Clearance Process during Public Health Emergencies
  • Remote Inspection Tools
  • Patient Advocacy through Remote Clinical Trials
  • Post-COVID Plans and the Potential for Upcoming Legislation
  • Beyond Therapeutic Developments into Cross-Functional Communications
  • Supply Chain and Logistics Concerns
  • Monkeypox Outbreak

3:25–4:25 PM
Hot Topics
Remy Brim, Principal, BGR Group
Daniel A. Kracov, Partner, Arnold & Porter LLP and Chair, FDLI Board of Directors
Kate Rawson, Senior Editor, Prevision Policy LLC
Moderated by Ryan M. Hohman, Vice President, Public Affairs, Friends of Cancer Research

  • CURES Act, PDUFA, other policies that are moving
  • Executive Order on Promoting Competition in the American Economy
  • Valid Act
  • FDA Inspection Backlog: Possible Approval Delays and Supply Chain Capability
  • Patient Registries
  • The Role of FDA and State Initiatives in Transformative Medicines
  • R. 3: Lower Drug Costs Now Act
  • Q&A Opportunity

4:25 PM
Course Adjournment