2023 Policy Summit
Sept. 18-19, 2023
Monday September 18, 2023
8 – 9 a.m.: Registration and breakfast (provided for in-person attendees)
Fireside Chat: Office of Therapeutic Products
9 - 9:45 a.m.
The director of the FDA CBER Office of Therapeutic Products (OTP), Dr. Nicole Verdun, will kick off the Summit by discussing the new super office, including plans to accommodate substantial growth in the CGT field and how to continue supporting development of innovative, novel products under their regulatory jurisdiction.
-
Updates on the Transition from Office of Tissues and Advanced Therapies (OTAT) to Office of Therapeutic Products (OTP)
Nicole Verdun, MD, FDA CBER Office of Therapeutic Products
-
Moderator: Kristin Van Goor, PhD, Takeda
Standards for Cell and Gene Therapies
9: 45 - 10:15 a.m.
The role of regulatory standards to help ensure efficient, safe cell and gene therapy development and learn how to navigate the evolving world of CGT standards. Join this session to hear how standards are drafted and applied, who is setting them, and how you can get involved.
-
The Role of SCB in Setting Standards for CGT Development
Dawn Henke, PhD, Standards Coordinating Body
-
The Role of USP in Setting Standards for CGT Development
Fouad Atouf, PhD, United States Pharmacopeia
-
The Role of NIST in Setting Standards for CGT Development
Samantha Maragh, PhD, National Institute of Standards and Technology
-
Moderator: Judith Arcidiacono, FDA CBER
Break
10:15 - 10:45 a.m.
Fireside Chat: Center for Medicare & Medicaid Innovation
10:45 - 11:30 a.m.
Discuss the recent Cell and Gene Therapy Access Model from CMMI and other federal policy efforts on payment policy, with audience Q&A to round out the session. Hear about CMS’ role in negotiating outcomes-based arrangements and how the agency is thinking about access to innovative therapies.
-
CMMI’s Cell & Gene Therapy Access Model
Laura McWright, JD, Center for Medicare & Medicaid Innovation
-
Moderator: Dan Farmer, BGR Group
Public-Private Partnerships for CGT Development
11:30 a.m. - 12:15 p.m.
Public-private partnerships can help fuel the transformative potential of cell and gene therapies, propelling innovation, accelerating accessibility, and reshaping the future of healthcare. Discuss current efforts in this space with your colleagues.
-
Bespoke Gene Therapy Consortium: Updates and Future Plans
Courtney Silverthorn, PhD, Foundation for the National Institutes of Health
-
Bill & Melinda Gates Foundation Efforts Towards Development and Global Delivery of Curative Interventions for HIV and Sickle Cell Disease
Mike McCune, MD, PhD, Bill & Melinda Gates Foundation
-
Advocacy in the Public-Private Space: Launching the NCATS Alliance
Kristin Stephenson, JD, St. Jude Children’s Research Hospital
-
Moderator: Jennifer Wellman, Akouos
Lunch (provided for in-person attendees)
12:15 - 1:15 p.m.
Coding and Reimbursement 101
1:15 - 2 p.m.
This session will explore the systems underpinning the American healthcare system. Panelists will discuss how coding is tied to reimbursement, DRG mapping and implementation, why the CMS coding system is so critically important for CGT success - and how all of those questions play into access challenges on the ground.
-
Introduction to Coding and Special Considerations for CGTs
Amy Rinkle, Nimitt Consulting
-
How Coding is Tied to Reimbursement
Preeya Noronha Pinto, JD, King & Spalding
-
Moderator: Ashley Hume, Emerging Therapy Solutions
Fireside Chat: Congressional Staff
2 - 2:30 p.m.
Discuss key health policy issues in 2023 and beyond with Hill staff. You'll hear about recent Congressional activity in the healthcare space and dive into value-based payments and patient access considerations, regulatory challenges for innovative therapies, and more.
-
Aisling McDonough, Office of Congresswoman Anna G. Eshoo (CA-16)
-
Moderator: Margarita Valdez Martínez, ASGCT
Break
2:30 - 3 p.m.
Cell and Gene Therapy Manufacturing: Policy Realities
3 - 4:30 p.m.
CGTs hold immense promise for patients, but their transformative potential can be hindered by manifold challenges of manufacturing, scaling up, and meeting CMC requirements. Discuss current efforts to overcome today’s challenges and help patients realize the full potential of CGTs.
-
Overview of Manufacturing Considerations for CGTs: Key Challenges and Future Pathways
Kim Benton, PhD, Dark Horse Consulting
-
The Advanced Manufacturing Technology Designation: Application and Challenges for CGTs
Kit Shaw, PhD, Boston Children’s Hospital
-
FDA's CMC Development & Readiness Program (CDRP): Background and Goals for the Agency and Industry
Anne-Virginie Eggiman, PhD, Tessera Therapeutics
-
Cell Therapy Manufacturing Challenges
John Tomtishen, Cellares
-
Moderator: Katy Spink, PhD, Dark Horse Consulting
Networking Reception
5:00 – 6:30 p.m.
Opening Remarks (in-person attendees only)
Congressman Scott Peters (CA-50), United States House of Representatives
Congressman Brett Guthrie (KY-02), United States House of Representatives
Light hors d'oeuvres and refreshments provided.
Tuesday September 19, 2023
8 – 9 a.m.: Registration and breakfast (provided for in-person attendees)
Fireside Chat: Advanced Research Projects Agency for Health
9 - 9:30 a.m.
Director of the Advanced Research Projects Agency for Health (ARPA-H), Dr. Renee Wegrzyn, will discuss the Agency’s role in advancing high-potential, high-impact biomedical and health research that cannot be readily accomplished through traditional channels—and how these projects can empower CGT development.
Considerations for Patient-centered Development
9:30 - 10: 45 a.m.
Incorporate patient perspectives and the principles of Patient-focused Drug Development (PFDD) for cell and gene therapies. Discuss the role of patient input in developing novel endpoints for CGTs, patient preference studies in regulatory development and decision-making, and what patient advocates want regulators and product developers to know.
-
Considerations for Patient-centered Development
Anne Rowzee, PhD, FDA CBER Office of Therapeutic Products
-
Patient-centric Development from the Patient Perspective
Mary Booth Dwight, Cystic Fibrosis Foundation
-
Developing Novel Endpoints for CGTs: Challenges, Regulatory Principles, and the Role of Patient Input
Snehal Naik, PhD, Spark Therapeutics
-
Using Patient Preference Studies in Regulatory Development and Decision Making
Juan Marcos Gonzalez Sepulveda, PhD, Duke University
-
Moderator: Rasika Kalamegham, PhD, Genentech
Break
10:45 - 11 a.m.
Patient Access to CGTs: Realities on the Ground
11 a.m. - 12 p.m.
Assess patient access and examine the role of Medicaid in ensuring equitable patient care. Discuss issues across the public payor space, including reimbursement challenges and policy considerations, health system readiness for CGTs, and how to navigate access challenges from the patient perspective.
-
Reimbursement for CGTs: Overview, Challenges, and Policy Considerations
John Feore, JD, Institute for Gene Therapy
-
Health System Readiness for CGTs: Hospital System Perspective
Michael Storey, PharmD, Nationwide Children’s Hospital
-
Supporting Patients for Wider Access
Beth Halpern, JD, Hogan Lovells
-
Navigating Access Challenges from the Patient Perspective
Kelly Maynard, Little Hercules Foundation
-
Moderator: Diane Berry, PhD, Sarepta
You may consider subscribing to The Advocate, our monthly policy and advocacy newsletter.
ASGCT is a 501(c)3 tax-exempt organization and does not participate in any political activity, either directly or indirectly, including support for or against campaigns for public office. The participation of speaker(s) who are elected officials, members of their staff, members of the governing administration, and/or former members of these offices (“government officials”), does not indicate support for or against any political party, political party agenda, or candidate for public office. Any government official who is also a speaker at an ASGCT-sponsored event is permitted to discuss topics related exclusively to the mission of the Society and is expressly prohibited from discussing any campaign for public office or other political activity.