Advancing patient access to cell & gene therapies: partnerships, pilots, & psyche

Cell & Gene Therapy Insights 2022; 8(1), 43–51


Published: 1 February 2022
Heather McDonald, PhD

Cell and gene therapies offer patients the hope of durable treatment with curative potential in as little as a single dose. Having demonstrated unprecedented efficacy in a number of diseases, including lifelong disorders previously deemed incurable, success of these transformative therapies heralds a new era of personalized medicines. While still in their infancy, cell and gene therapies are projected to expand to multiple disease areas within the next decade. Although showing tremendous promise, there are also challenges in ensuring that patients can access these new modalities. Payers, for example, have articulated concerns related to affordability and uncertainty around long-term clinical benefit. Manufacturers, meanwhile, face challenges navigating payer evaluation frameworks, given that cell and gene therapies are typically one-time treatments with a lifetime value proposition studied in comparatively small, sometimes single-arm clinical trials of a finite duration. Patient access for cell and gene therapies will therefore depend on collaborative efforts within the healthcare ecosystem and a willingness to share not only in the benefits, but also the risks associated with their reimbursement.