Policy Summit

Policy Summit Takeaways Include Advice for Action on Access

ASGCT Staff - September 27, 2021

During sessions on Medicare and Medicaid payment policies, speakers on the first day of the Policy Summit provided potential solutions for stakeholders working toward patient access to gene and cell therapies.

The third annual ASGCT Policy Summit last week included sessions on Medicaid and Medicare payment policies, complete with insights on what stakeholders can do to support patient access.

Session speakers provided context on the scope of the problem. Mark Trusheim, strategic director of MIT NEWDIGS, pointed out that while the number of durable therapies likely to be approved by 2030, at 54 to 74, is enough to cause concern, the impact on the Medicaid system is remarkably small. This overall small impact is partially because these therapies are meant to treat rare diseases. Similarly, Josh Trent, principal at Leavitt Partners, noted that when Sovaldi was approved for the treatment of hepatitis C, there was concern about budget impact and access, yet the patient population normalized, so payers were able to handle reimbursement.

Gene therapies do pose some risk to payers, however. To mitigate that risk, policymakers have begun to utilize tools such as value-based payments (VBPs). Some state Medicaid programs allow VBPs through state plan amendments, and a federal rule, expected to go into effect in July 2022, will allow these arrangements within Medicaid through a new mechanism called multiple best price reporting. But more stakeholder efforts are necessary to address gene therapy patient access challenges, according to several speakers.

A high-level view of potential solutions, and how stakeholders can work toward them, include the following:

  • Demetrios Kouzoukas, JD, former director of the Center for Medicare and principal deputy administrator of the Centers for Medicare & Medicaid Services, commented that the cost of gene and cell therapies make Medicare a promising area for value-based payment, though the low Medicare spending to date may require more time for consideration of this solution.
  • Providing state Medicaid programs with resources to inform decisions and additional federal funding to support payment is a potential policy opportunity, according to Trent.
  • Diane Berry, PhD, senior vice president of global health policy, government and patient affairs at Sarepta Therapeutics, advocated for states to cover gene therapies immediately upon approval and to cover all FDA-labeled populations.
  • Anne Schwartz, PhD, executive director of the Medicaid and CHIP Payment and Access Commission (MACPAC), indicated a need for a national repository of outcomes data.
  • Berry, a member of ASGCT’s Government Relations Committee, announced that ASGCT has embarked on a project to assess a sampling of Medicaid program coverage and reimbursement policies to further inform stakeholders on the scope of access challenges, examples of best practices, and potential policy strategies.
  • Trent suggested that stakeholders provide private payer examples of VBP agreements to state Medicaid programs for consideration of how to adopt them.
  • Berry and Trent both noted that education of policymakers—on topics such as the science of gene therapy, disease progression for the diseases it treats, treatment expectations, FDA regulations, the pipeline, and Medicaid coverage—may facilitate policy development over time.
  • Schwartz invited stakeholders to read MACPAC’s recent report and propose design considerations for a potential new framework for gene and cell therapies under Medicaid.

For a deeper dive on the public payment policies discussed at the Policy Summit, watch for or sign up to receive our next policy newsletter, The Advocate, coming out on October 20. Registered attendees can also view all Policy Summit content on demand through Oct. 24. If you haven’t registered, it’s not too late; do so now to get access!

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