A Dolon Institute Publication
CAR-T therapies have the potential to deliver life-changing, potentially lifelong benefits for patients, but their novel characteristics create challenges that traditional healthcare and reimbursement systems are not always equipped to address. This ISPOR Europe 2023 panel brought together perspectives from clinical development, market access and policy to explore how Europe can ensure CAR-T therapies remain sustainable, affordable and available to the patients who need them.
The panel highlighted that CAR-Ts differ fundamentally from conventional medicines. Their complex manufacturing and delivery processes, specialised infrastructure and expertise, and requirement for continuous investment create significant economic and operational challenges. These complexities also mean that economies of scale are difficult to achieve, while clinical development can require alternative trial designs, such as single-arm or crossover studies, because of the transformative benefits of these therapies and resulting ethical considerations.
These characteristics create particular challenges for pricing and reimbursement (P&R). Although CAR-Ts may be highly cost-effective because of their potential long-term effects, their one-off payment model can create greater affordability challenges for payers than treatments delivered over multiple years. At the same time, increasingly stringent cost-containment measures, price erosion and limited willingness to engage with innovative payment models are creating greater uncertainty around the commercial sustainability of CAR-T development.
From a policy perspective, the panel argued that the current environment is not sufficiently tailored to the complexity of novel therapies such as CAR-Ts. Adequate funding is required not only to support continued innovation but also to equip hospitals with the infrastructure and resources needed to deliver these treatments. Despite CAR-Ts being available in some settings, only around one-third of eligible patients are currently receiving treatment, underlining the gap between availability and actual access.
The discussion points towards the need for greater collaboration and new approaches to P&R, rather than relying solely on existing frameworks. Priorities include mechanisms that can manage long-term clinical and affordability uncertainty, earlier engagement between stakeholders, and payment approaches that better reflect the distinctive characteristics of one-off therapies.
Importantly, the panel highlighted emerging efforts to address these challenges. Policymakers and other stakeholders are working through initiatives including the WHO Europe Novel Medicines Platform and working groups focused on conditional reimbursement, with demonstration projects intended to test potential approaches in practice.