OBJECTIVES:

The high up-front cost of potentially curative cell and gene therapies, combined with the lack of long-term outcomes data at launch, places barriers to these treatments gaining market access. This article sets out to review how performance-linked payment models, or value based contracts (VBC), have been used as solutions, and what is the likely future for such models.

METHODS:

This study involved primary and secondary research. Firstly, a targeted online literature review of VBC for innovative therapies in the US and Europe was performed. Subsequently, hypotheses were generated during roundtable discussions. Finally, expert insights were derived from in-depth interviews with payer stakeholders across the US and top-five European markets.

RESULTS:

Three relevant therapies have recently been approved in the US; two CAR-T therapies for hematological cancers (tisagenlecleucel and axicabtagene ciloleucel) and a gene transfer technology for inherited retinal disease (voretigene neparvovec). Two of these have VBC in place. In Europe, another high-cost innovative therapy (adenosine deaminase cDNA transduced CD34+ cells for ADA-SCID) is reimbursed via VBC, but only in Italy. In all cases, payment is linked to outcomes at pre-defined times post-treatment. During interviews, payers show strong interest in VBC, given the relationship between payment and outcomes; however, they cite concerns with implementing VBC more broadly. Their experience has shown that VBC are administratively burdensome and do not provide the desired certainty on durability of responses.

CONCLUSIONS:

VBC provide a great opportunity for enabling timely patient access to innovative and potentially curative treatments. To maximize the successful implementation of VBC across markets, there are several key payer recommendations for innovators: firstly, support healthcare providers with delivery and logistics; secondly, make payments related to longer-term outcomes or stagger smaller payments over time; and lastly, collaborate on new funding models to allow affordable patient access as the number of these treatments increase.