ICER and NEWDIGS at Tufts Medical Center released a white paper on the challenges and potential policy options for paying for gene therapies. The report analyzes the intersecting challenges of ensuring patient access for gene therapies, including determining fair prices, managing clinical uncertainty, and mitigating financial shocks. 

This paper draws upon the body of work on gene therapy issues by ICER and the NEWDIGS FoCUS (Financing and Reimbursement of Cures in the US) Project. ICER and NEWDIGS obtained feedback from experts from ICER’s Policy Leadership Forum, payer organizations, large and small biopharmaceutical manufacturers, patient advocacy groups, providers, and ancillary vendors offering or providing necessary execution support for potential solutions.

Date of publication: April 2024

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Final Document

ICER’s President and CEO Sarah K. Emond, MPP stated:

“Gene therapies have the possibility to transform thousands of lives, but only if we ensure sustainable access to them for all patients. It is incumbent upon the entire health care system to focus on innovative payment solutions that can address the challenges of paying for high-cost, one-time treatments. This paper should help stakeholders understand the menu of options available to move beyond responding to the high costs with reduced access, while meaningfully addressing the concerns around clinical uncertainty and budget impact.”

Mark Trusheim, Strategic Director of NEWDIGS stated:

“Each condition, therapy and payer is unique, so a single solution cannot satisfy all situations. With a menu of options which can be combined in a single contract or assembled from existing market offerings, each community can construct the most effective approach to ensuring appropriate patient access in their area.”