Principles and practices for successful gene therapy innovative contracting: Insights from a multistakeholder...

Gene therapies (GTs) offer potentially 1-time, curative treatment approaches for inherited and other rare diseases, but their often high upfront costs and small patient populations challenge traditional reimbursement models. In response, US payers and manufacturers have increasingly implemented innovative contracts (ICs) tied to value and outcomes.

To share key stakeholder experiences and identify opportunities for efficient and effective innovative contracting solutions for GTs.

We used a 6-step multimethod approach, including a literature review, stakeholder interviews, framework development, a virtual convening of stakeholders, and a pre- and postconvening survey. We defined "ICs" as agreements between payers and manufacturers that use real-world outcomes to determine the total price paid for GTs, encompassing various contracting models (eg, rebates, warranties, and performance designs) and terminologies that link outcomes to pricing. We defined "principles" as elements of ICs for successful GT innovative contracting and "practices" as processes and steps for stakeholders to explore feasibility, negotiation, and execution of an IC for a GT. The consensus principles and practices include those that received at least 75.0% of the votes (9 of a possible 12 stakeholders) for minimal or no revisions, prioritizing the top 8 principles and practices with the highest consensus.

Eight principles of a possible 18 (44.4%) achieved 75.0% consensus with minimal to no revisions, and 7 of 26 possible practices (26.9%) achieved 100.0% consensus with minimal to no revisions. Principle topics included outcomes, duration, data collection, types of contracts, and patient costs. Practice topics included a focus on feasibility, negotiation, contract term, and evaluation. Stakeholders reviewed additional topics that did not ultimately reach consensus, including the role of patients and providers in innovative contracting.

The mixed methods approach enabled the development of nuanced and contemporary principles and practices in innovative contracting for GTs. As more GTs are approved, there may be a need to refresh the principles and practices.