Aisling Mcmahon, Professor of Law at Maynooth University explored the need to develop better systems for sustainable access to CAR T and other cell therapies. She kicked off the discussion by suggesting that the cost of these therapies is so great that many public health systems cannot provide them, therefore we must look at mechanisms around this and how they can be reshaped. Mcmahon added: “Increasingly we’re seeing and hearing conversations around investors considering reimbursement pathways.”
This complicated challenge does not have one particular definitive solution, but to tackle it, Mcmahon posited that a patient-centric approach is the most essential outlook. To provide context for the CAR T landscape, the first two CAR T therapies (Kymriah and Yescarta) were approved in 2018. According to the European Society for Blood and Marrow Transplant, these two therapies have reached over 10,000 patients, thus demonstrating their applicability to large population sets. Furthermore, there have been positive results from CAR T therapies for lupus and other autoimmune conditions.
There is a mismatch between the reimbursement and approval processes for approved CAR T cell therapies which have posed major ethical and policy considerations. Mcmahon stated: “These (Kymriah and Yescarta) were EMA approved in 2018 but in Ireland, they weren’t actually provided or reimbursed in the healthcare system until 2021 and 2022.” The 3-4-year gap between EMA approval and actual provision in Ireland is a concern. Mcmahon explained that there were some alternative pathways that patients could take such as the Treatment Abroad Scheme yet this still presented extra obstacles for patients.
The growing number of patients in need of these treatments means that there is now a higher demand for them. Researchers are investigating novel reimbursement pathways: within the Irish context there is an emphasis on outcome-based models where one traces evidence over time and pays on the basis of evidence. Mcmahon also advocates for rebate models and instalment-based plans evidence and outcomes are measured over time. These approaches are also being tested in countries like Germany, Spain, and Italy.
From a legal policy standpoint, there is an issue of transparency. Mcmahon elaborated on this: “So some states, as we know, many of the prices are not available and that's for commercial reasons and sensitivity reasons that we don't have those and legal requirements indeed NDAs, etcetera that have been signed to that effect.” Mcmahon said that decentralizing production near clinical sites could reduce costs and improve timelines, reducing the current four-week turnaround for Irish patients.
Overall, expanding partnerships between academic and commercial entities for innovation remains a priority. Leveraging decentralized manufacturing and hospital exemptions is predicted to enhance affordability and accessibility. Confronting these issues requires collaborative efforts among public health systems, commercial entities, and academic researchers.
