2022 Policy Summit

September 29-27, 2023

Policy Summit Co-Chairs: Diane Berry, PhD, Sarepta Therapeutics & Keith Wonnacott, PhD, LEXEO Therapeutics

Monday September 26, 2022

9:00 – 10:30 a.m.: Supporting Innovative CGT Development

• RMAT in 2022: Updates and Learnings
  Wilson Bryan, MD, FDA OTAT

• Health Canada Oversight of Advanced Cell and Gene Therapies and Approaches to Regulatory Innovation     
  Emily Griffiths, PhD, Health Canada

• New Endpoints and Biomarkers in Supporting CGT Development
  Adora Ndu, PharmD, JD, BridgeBio

• The Bespoke Gene Therapy Consortium: Increasing Accessibility of Gene Therapies for Rare Diseases
  Courtney Silverthorn, PhD, Foundation for the National Institutes of Health

• Moderator: Daniela Drago, PhD, NDA Partners

10:30 – 10:45 a.m.: Networking coffee break

10:45 a.m. – 12:00 p.m.: Panel on PDUFA Reauthorization

• PDUFA VII: Overview of Statutory Updates and New Commitments
  Khushboo Sharma, Accumulus Synergy  

• PDUFA VII: CBER Updates
  Wilson Bryan, MD, FDA OTAT

• PDUFA VII: Industry Perspective and Implications for Biotechs
  Brad Glasscock, PharmD, BioMarin

• Moderator: Keith Wonnacott, PhD, LEXEO Therapeutics

12:00 – 1:00 p.m.: Lunch

1:00 – 1:30 p.m.: Keynote Address

• Medicaid Coverage of Gene & Cell Therapies
  John Coster, PhD, Division of Pharmacy, Center for Medicaid and CHIP Services, Centers for Medicare and Medicaid Services

• Moderator: Dan Farmer, BGR Group

1:30 – 2:45 pm: Panel on Diversity in Cell and Gene Therapy Development

• Clinical Trial Access and Health Disparities for Minority Populations
  Rayne Rouce, MD, Texas Children’s Hospital & Baylor College of Medicine 

• Legislative Efforts to Improve Clinical Trial Participation in Underrepresented Populations 
  Aisling McDonough, Office of Chairwoman Anna G. Eshoo 

• FDA Draft Guidance on Clinical Trial Diversity
  Asha Das, MD, Office of Tissues and Advanced Therapies

• NASEM Report on Improving Representation in Clinical Trials and Research
  Alex Helman, PhD, National Academy of Sciences, Engineering, and Medicine

• Moderator: Rayne Rouce, MD, Texas Children's Hospital & Baylor College of Medicine

2:45 – 3:15 p.m.: Networking coffee break

3:15 – 4:30 p.m.: Panel on Accelerated Approval

• Current Hill Discussions on Accelerated Approval
  Remy Brim, PhD, BGR Group

• Medicaid Program Experiences with Cell and Gene Therapy Products: Lessons and Future Considerations
  Matt Salo, formerly National Association of Medicaid Directors

• Optimizing the Use of the Accelerated Approval Pathway for Rare Disease and Gene Therapies  
  Annie Kennedy, EveryLife Foundation 

• Developer Viewpoint on Accelerated Approval
  Diane Berry, PhD, Sarepta Therapeutics

• Moderator: Jeremy Allen, Spark Therapeutics

5:00 – 6:30 p.m.: Networking reception for all attendees at the JW Marriott

Tuesday September 27, 2022

9:00 – 10:15 a.m.: Panel on Coverage and Reimbursement for Cell and Gene Therapies

• Coverage and Reimbursement Policies: Trends and Innovation 
  Mark Trusheim, Tufts NEWDIGS Initiative 

• Managing High-Cost, High-Value Therapies: Oklahoma's Experience
  Terry Cothran, Oklahoma Health Care Authority

• Learnings from European Payment Systems 
  Andrew Olaye, PhD, Orchard Therapeutics 

• Payer Decisions that Impact Patient Access
  Kollet Koulianos, National Hemophilia Foundation

• Moderator: Francesca Cook, REGENXBIO

10:15 – 10:30 a.m.: Networking coffee break

10:30 a.m. – 12:00 p.m.: CMC Applications and Hurdles for Gene and Cell Therapies

• Current CMC Expectations for Gene Therapy Products Incorporating Genome Editing
  Zhaohui Ye, PhD, Office of Tissues and Advanced Therapies

• ASGCT’s Recommendations on CMC at the 2021 FDA Liaison Meeting 
  Jan Thirkettle, PhD, Transine Therapeutics 

• Comparability Considerations for Advanced Therapy Medicinal Products Subject to Changes in their Manufacturing Process
  Andrew Chang, PhD, Novo Nordisk

• Applying Benefit/Risk to CMC Considerations in Development 
  Stacey Frisk, Sarepta Therapeutics 

• Moderator: Megan Zoschg Canniere, PharmD, Spark Therapeutics

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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.