Managing the Challenges of Paying for Gene Therapy: New ICER / NEWDIGS whitepaper 

Eric NormanFoCUS, News

ICER and NEWDIGS Release White Paper Analyzing the Challenges and Potential Policy Options for Paying for Gene Therapies

— Paper outlines policy reforms and market actions to support innovation and access while managing uncertainty, affordability, and equity concerns; developed as part of ICER’s Policy Leadership Forum with input from diverse stakeholders —

Boston, April 23, 2024 – The Institute for Clinical and Economic Review (ICER) and NEWDIGS at Tufts Medical Center have collaborated to publish a new white paper today that provides clear policy and market solutions to help manage the challenges of paying for gene therapies. The paper, entitled, “Managing the Challenges of Paying for Gene Therapy: Strategies for Market Action and Policy Reform,” was developed with input from a diverse set of patient advocacy groups, payer organizations, large and small biopharmaceutical manufacturers, providers, and others.

The white paper analyzes the intersecting challenges of ensuring patient access for gene therapies, including determining fair prices, managing clinical uncertainty, and mitigating financial shocks.  

The specific policy reforms and market-based tools ICER and NEWDIGS identify in the paper are intended to help the US health system address these challenges to achieve more equitable and affordable access for patients to the growing number gene therapies expected to be approved in the coming years. Options such as value-based pricing, value-based outcomes contracts, warranties, subscription insurance models, installment payment arrangements, and a federal carve-out benefit program are explored.  For each option, the white paper analyzes the relative advantages and potential unintended consequences, along with suggestions for implementation, and an exploration of unique opportunities for combination or layered approaches.

“Gene therapies have the possibility to transform thousands of lives, but only if we ensure sustainable access to them for all patients” said Sarah K. Emond, MPP, President and CEO of ICER. “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.”

“Each condition, therapy and payer is unique, so a single solution cannot satisfy all situations” said Mark Trusheim, Strategic Director of NEWDIGS. “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.”

This paper relies on data and perspectives gathered from a targeted literature review and 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 twenty-two experts from payer organizations, large and small biopharmaceutical manufacturers, patient advocacy groups, providers, and ancillary vendors offering or providing necessary execution support for potential solutions.

This white paper went through several iterations following ICER’s Policy Summit in December 2023. At that meeting, policy leaders from patient advocacy groups, purchasers, and employers joined with ICER’s Policy Leadership Forum (PLF) to discuss a preliminary draft, debate the policy recommendations, and provide suggestions for revisions to the paper. None of the individuals involved in this meeting, nor their organizations, should be assumed to support any part of this report, which is solely the work of the ICER and NEWDIGS teams.

ICER develops annual white papers in collaboration with the ICER Policy Leadership Forum.  Previous papers have addressed topics such as Strengthening the FDA’s Accelerated Approval PathwayCornerstones of ‘Fair’ Drug CoverageReforms to Orphan Drug Development, Pricing, and CoverageAlternative Models for Pharmaceutical Rebates, and White Bagging, Brown Bagging, and Site of Service Policies.

About ICER

The Institute for Clinical and Economic Review (ICER) is an independent non-profit research institute that produces reports analyzing the evidence on the effectiveness and value of drugs and other medical services. ICER’s reports include evidence-based calculations of prices for new drugs that accurately reflect the degree of improvement expected in long-term patient outcomes, while also highlighting price levels that might contribute to unaffordable short-term cost growth for the overall health care system.

About NEWDIGS at Tufts Medical Center

NEW Drug Development ParadIGmS (NEWDIGS) is dedicated to improving health outcomes by accelerating appropriate and timely access for patients to biomedical products, in ways that work for all stakeholders. NEWDIGS designs, evaluates, and catalyzes the real-world implementation of system innovations that are too complex and cross-cutting to be addressed by a single organization or market sector. Its members include global leaders from patient advocacy, payer organizations, biopharmaceutical companies, regulatory agencies, clinical care, academic research, and investment firms. For more information, visit