Alternative payment models for durable and potentially curative therapies: The case of gene therapy for haemophilia A

Abstract

Introduction: The emergence of durable and potentially curative cell and gene therapies (also known as advanced therapy medicinal products) with high price tags is challenging conventional health care payment systems. Among these are gene therapies in various phases of development for haemophilia A and B. The emergence of these therapies comes with clinical and economic uncertainties for payers, providers, patients, and manufacturers. These include uncertainties about expression of the intended physiological response, patient outcomes, and duration of treatment effect, along with potentially high upfront costs, variable additional costs, and uncertainties about uptake of the therapy among indicated patient populations. These clinical and economic uncertainties raise interest among payers, providers, and manufacturers in risk-sharing arrangements, including alternative payment models (APMs).

Summary: APMs differ from traditional fee-for-service payment for health care. For example, some APMs are designed to reward clinicians and provider organizations for delivering high-quality and cost-effective care. The APMs described here, outcomes-based models (or value- or performance-based models) and finance-based models, are designed to mitigate or otherwise manage financial risks associated with health care payment, including instances in which patient outcomes for costly therapies are uncertain. We examine how such APMs may be applicable in the context of emerging gene therapies for haemophilia A, including considerations in determining whether any particular APM may be most suitable.

Key points of consideration: Gene therapies for haemophilia are among the emerging durable and potentially curative cell and gene therapies with high price tags that are challenging conventional payment systems. These therapies present clinical and economic uncertainties for payers, providers, patients, and manufacturers, including regarding expression of the intended physiological response, duration of treatment effect, patient outcomes, potentially high upfront costs and variable additional costs, and uptake among potentially indicated, diverse patient subgroups. These uncertainties raise interest among payers, manufacturers, and providers in risk-sharing arrangements, including alternative payment models (APMs) such as various outcomes-based and finance-based models. This paper describes a taxonomy of APMs. Then, for the particular context of gene therapy for haemophilia A, we present a set of factors to be considered when determining whether an APM risk-sharing arrangement may be appropriate for a given gene therapy, and, if so, which APM may be most suitable. [Correction added on 21 February 2022, after first online publication: the 'Key Points of Consideration' have been added in this version.].

Keywords: factor VIII deficiency; gene therapy; haemophilia A; insurance design; value-based purchasing.