Perspectives on Payment Policies for Gene Therapies

Tuesday, November 5 | 9 a.m.–5 p.m. | The Westin City Center, Washington, D.C.

This program will address the status of legislative and regulatory solutions to the primary challenges in paying for gene therapies for different payer types (Medicaid, Medicare, and commercial payers), as well as the current state of Medicaid reimbursement policies for these therapies.


  1. Discuss recent progress in efforts to enable and utilize novel payment models for gene therapies
  2. Explore the status of Medicaid policies for gene therapies and opportunities for enhancing reimbursement mechanisms
  3. Identify the implications of recent decisions on Medicare coverage and reimbursement of CAR T-cell therapies

Full Agenda

Value of Gene Therapy & Significance of the Issues
9-10:35 a.m.

  • David Barrett, J.D.—Introduction and Keeping Current on the Pipeline
    Executive Director, American Society of Gene & Cell Therapy
  • Colin Young, Ph.D.—Preparing the System: Pipeline Prediction Update
    Director of Drug Development Pipeline Research, MIT NEWDIGS
  • Jenn McNary—Parent and Patient Advocate Perspective on Value for Duchenne Muscular Dystrophy
    Parent and advocate for people with DMD; Manager, J McNary Consulting
  • Khrystal Davis, J.D.Parent and Patient Advocate Perspective on Value for Spinal Muscular Atrophy
    Parent and advocate for people with SMA; Founder & President, Texas Rare Disease Alliance
  • Francis Pang—Market Access and Value Philosophy for Gene Therapies
    Vice President of Global Market Access, Orchard Therapeutics
  • Panel Q&A
    Moderator: Paul Melmeyer, Director of Regulatory Affairs, Muscular Dystrophy Association

Advances in Novel Payment Solutions for Public & Private Payors
10:50 a.m. - 12:15 p.m.

  • Rob Osborne—Updates on Outcomes-Based and Innovative Payment Solutions
    Vice President of Trade Relations, Accredo Specialty Pharmacy and CuraScript SD
  • Tresja Bolt—Novel Payment Approaches: The Experience from Europe
    Head of European Government Affairs and Policy, bluebird bio
  • Dan Mytelka—Managing Gene Therapies: The Orphan Reinsurer and Benefit Manager (ORBM)
    Director of Policy and Simulations, MIT NEWDIGS
  • Panel Q&A
    Moderator: Janet Lambert, CEO, Alliance for Regenerative Medicine

Legislative Solutions: Novel Payment Mechanisms for Gene Therapies
1:15-2 p.m.

  • Anne Dwyer, J.D.
    Senior Health Counsel, Senate Committee on Finance
  • Stuart Portman
    Health Policy Advisor, Senate Committee on Finance
  • Panel Q&A
    Moderator: Mark Battaglini, J.D., Vice President, Global Government Affairs & Public Policy, bluebird bio

Deep Dive I: Perspectives on State and Federal Medicaid Policies
2-3:30 p.m.

  • Mark McClellan, M.D., Ph.D.—Opportunities and Barries for Advancing Value-Based Payment 
    Founding Director, Margolis Center for Health Policy at Duke University
  • Ross Margulies, J.D.—Medicaid Landscape: A Look at State Medicaid Approaches to Gene Therapy Coverage and Reimbursement
    Associate, Foley Hoag
  • Kaha Hizanishvili—The Massachusetts Medicaid Approach to Gene Therapy Reimbursement
    Chief, Office of Provider & Pharmacy Programs, MassHealth
  • Chethan Bachireddy, M.D.—A Developing Approach to Break-through Therapies: The Virginia Medicaid Experience
    Chief Medical Officer, Virginia Department of Medical Assistance Services
  • Panel Q & A
    ModeratorMark McClellan

Deep Dive II: A Fresh Look at Medicare Reimbursement Policies
3:45-5 p.m.

  • Anna Griffin—Developer View: Current and Future Patient Access to CAR T-cell Therapy Under Medicine
    President & CEO, Griffin Consulting
  • Jugna Shah—Proposals for CAR T-cell Therapy Reimbursement Beyond the NTAP Period
    President, Nimitt Consulting
  • Panel Q & A
    Moderator: Jugna Shah


  • Member: $200
  • Non-Member: $250

Register today for the Tuesday, November 5 session, Perspectives on Payment Policies for Gene Therapies, or all of the ASGCT 2019 Policy Summit (a PDF registration form is also available).

Cancellation Policy: Refund requests must be submitted in writing to ASGCT prior to October 21, 2019. A $35 processing fee will be charged for all refunds. We regret refunds cannot be allowed for requests postmarked or received after October 21, 2019.

Special Accommodations: If you require special accommodations under the ADA in order to fully participate in the meeting, please send a written description of your needs to the ASGCT Executive Office. Please notify us of your needs by October 28, 2019.


ASGCT Policy Summit

November 4 – 6 | Washington D.C.