Essentials of the FDA Landscape for Patient Organizations

February 7–8, 2024 I Virtual Course
For 501(c)(3) Patient or Disease Organizations Only

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.

Preliminary Agenda

Agenda Subject to Change
All Times Listed in Eastern Time

Wednesday, February 7

11:00–11:10 AM
Welcome and Announcements
Paige Samson, Director, Innovative Programs and Publications, FDLI

11:10 AM–12:10 PM
Navigating FDA as a Patient Advocate
Joseph F. Lamendola, Senior Director, Faegre Drinker Consulting
David R. Zook, Chair, Faegre Drinker Consulting

  • Role of Patient Advocates
  • FDA Advisory Committees
  • Commenting on Draft Guidances, Proposed Regulations, and Other FDA Publications
  • PFDD Meetings, Listening Sessions, Patient Experience Data

12:10–12:30 PM

12:30–1:10 PM
Engaging with FDA: Opportunities and Boundaries
Shawn Shermer, FDA Patient Listening Session Program Lead, Office of the Commissioner, FDA

  • Patient Initiatives
  • FDA Patient Engagement Activities
  • Examples of Practical Applications
  • Patient-Focused Drug Development
  • Resources for Patients
  • Patient Engagement in Medical Devices, Drugs, and Biologics

1:10–1:40 PM

1:40–2:40 PM
Understanding the Regulation of Cell and 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
  • Digital Health/Emerging Technologies
  • Pharmacovigilance
  • Pilot to Accelerate Review of Biologic Therapies for Rare Diseases

2:402:50 PM

2:50–3:50 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
  • State Substitution Laws, Payor Responsibility

3:50–4:00 PM

4:00–5:00 PM
Accelerated Approval Expedited Pathways
Daniel A. Kracov, Partner, Arnold & Porter LLP

  • 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
  • Patient Reported Outcomes and Real-World Evidence
  • Regulatory Flexibility
  • Options if No Pathway Applies

Thursday, February 8

11:00 AM–12:00 PM
Incorporating Patient Experience into Medical Product Development: Fireside Chat
Celia M. Witten, Deputy Director, Center for Biologics Evaluation and Research (CBER), 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
  • Federal Preemption and FDA Authority
  • Shifting Regulatory Environment and What it Means for Patients Judicially and Legislatively

12:00–12:15 PM

12:15–1:45 PM
Patient Access to Treatments
Richard Klein, Director, Expanded Access Programs & Policy, GE2P2 Global Foundation

  • Part I: Expanded Access and the Right to Try
    • Traveling for Patient Access to Therapies
  • Part II: FDA Efforts to Expand Access to Clinical Trials
    • FDA Guidance and Changes for Clinical Trials
      • Diversity, Inclusion, and Design
      • Decentralized Clinical Trials
    • Diversity and Inclusion in Clinical Trials
      • New IRB rules
    • Informed Consent & Patient Compensation
    • Withdrawn Drugs
    • Off-Label Use
    • Expansion of Telehealth Services
    • Remote Inspection Tools

1:45–2:00 PM

2:00–3:00 PM
Interactions with Industry: Opportunities and Boundaries
Susan Lee, Partner, Goodwin Proctor LLP
Tiffany Patrick, VP, Head of Patient Advocacy, Alnylam Pharmaceuticals

  • How Patient Experience can Influence Clinical Trial Design and Other Development Decisions
  • How Patient Organizations Can Support Industry Development
  • Pre-Approval Promotion, Unblinding, Inducements, and Other Legal/Regulatory Basics
  • What Patients Can (and Can’t) Expect When Interacting with Industry
  • Q&A

3:00–3:10 PM

3:10–4:00 PM
Hot Topics
Remy Brim, Principal, BGR Group
Kate Rawson, Senior Editor, Prevision Policy LLC
Moderated by Ryan M. Hohman, Vice President, Public Affairs, Friends of Cancer Research

  • Digital Health/Emerging Technologies
  • Panelists will discuss the most late-breaking and relevant topics
  • Q&A Opportunity

4:00 PM
Course Adjournment