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, outcomesbased 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.

Preparing for tomorrow: Defining a future agenda

“…In this supplement issue, Goodman et al provide proposals of alternative payment models (APM), including those based on outcomes (i.e., value-or performance-based) and on finance (i.e., designed to mitigate financial risks when outcomes are uncertain). 12 Considerations in selecting an alternative payment model are described, addressing questions on how to determine the best-fit APM for a particular national or regional health care system, and how to characterize a linked set of specific factors for determining whether to pursue a particular APM in the context of emerging gene therapies for haemophilia. 12 Table 3 presents the key points of consideration described by Goodman et al, along with our commentary regarding those points.…”

“…12 Considerations in selecting an alternative payment model are described, addressing questions on how to determine the best-fit APM for a particular national or regional health care system, and how to characterize a linked set of specific factors for determining whether to pursue a particular APM in the context of emerging gene therapies for haemophilia. 12 Table 3 presents the key points of consideration described by Goodman et al, along with our commentary regarding those points.…”

“…Different healthcare systems can include a single payer market or national procurement model (e.g., United Kingdom, France, Canada, Ireland, Australia), a national/federal government payer system with health budgets devolved to the regions (e.g., Spain, Italy), a government-mandated compulsory insurance market (e.g., Germany, Austria, Switzerland), or a complex insurance-based multi-payer market (e.g., United States) . The 18 factors described by Goodman et al 12 give guidance that may influence which alternative payment models are most appropriate for haemophilia gene therapy within a giving healthcare system. Figure 2 is an illustrative example using two types of payment model and comparing it for a single payer market (Brian O'Mahony and Declan Noone, personal communication).…”

Is the world ready for gene therapy?

“…In this supplement, Goodman et al indicate that the suitability and success of a payment model will require consultation among the multiple stakeholders. 13 The weight of responsibility for realizing the potential of gene therapy does not simply rest with the manufacturer and medical system, as the healthcare system needs to be innovative from a policy and access mindset, in order to incentivize such stepwise innovations to come to market, now and in the future. Only then can we ensure a symbiotic balance between the value of innovation and healthcare budget sustainability.…”

“…Despite uncertainties related to gene therapy, evidence-based decisions with existing data should be undertaken with the understanding that data collection will continue through registries and longitudinal studies, while alternative payment models will be developed to reduce risk. 10,13 As described by O'Hara and Neumann, health technology assessment (HTA) bodies may require greater flexibility in evidence requirements, recognizing that outcomes can vary across patient populations and health systems. 15 Instead of traditional mechanisms used by HTAs, those evaluating haemophilia gene therapies may need to consider the limitations in data collection for rare diseases such as haemophilia and to consider use of intra-patient data and extrapolation of treatment durability based on existing data.…”