Quantifying Impact of Alternative Payment Agreements on Research and Reimbursement Decisions for Gene Therapies

Abstract:

Gene Therapies (GTs) provide new treatment options for disease with high unmet medical need. To address economic challenges for GTs, an increasing body of literature discusses payment in instalments with or without conditions, as opposed to traditional on-off upfront payment. Uptake of such alternative payment agreements models are explored by healthcare systems and payers across the globe. To move from theory to practice systematic assessment of these schemes, their design and impact on population health and costs is needed. 

Traditionally, reimbursement decisions are binary: Approve or Reject. However, newer policies like "Only in Research" (OIR) and "Approval with Research" (AWR) offer earlier access while allowing for the collection of evidence to reduce uncertainty. OIR allows access within a defined research population, while AWR gathers more evidence with wider access. Claxton, Palmer et al. were the first to develop a transparent framework making the key principles, assessments, and decisions explicit. In this framework, under traditional one-off upfront payment models GTs are expected to be mostly guided towards an OIR policy decision. This hampers widespread access to these new technologies. Combining the systematic assessment population health and value of additional information of the OIR/AWR-framework with alternative payment agreements is a novel approach which could move application and design of alternative payment agreements forward and increase patient access.

Therefore, the aim of this research is to assess impact of applying alternative payment agreements between manufacturer and payers on Approve, AWR, OIR or Reject policy choices. This is done via a case study of recently authorized gene therapy indicated for severe Hemophilia A.