Recent advances in cell and gene therapies are rapidly evolving care for many life-threatening conditions with high unmet need. The introduction of durable and potentially curative new treatment options is changing the prospects for providers, patients, and their families. Regulatory agencies are accounting for these innovative treatments with new programs and guidelines. Under the FDA Cures Act, cell and gene therapies can receive a regenerative medicine advanced therapy (RMAT) designation, which accelerates access to market. Similarly, the EMA has developed a number of scientific guidelines specific to gene therapy products to keep pace with clinical and scientific innovations.

As regulators put policies in place to prepare for the coming wave of cell and gene therapies, significant challenges exist to assess long-term value using conventional metrics. Lack of effective comparators and heterogeneity of the patient population in clinical trials introduce uncertainties when assessing the value of these medicines. High upfront costs and limited evidence of long-term outcomes also highlight the need for new reimbursement models. A new value assessment framework is needed to incorporate broader elements of value into decision-making. In light of this landscape, presenters will discuss common considerations and potential alternative approaches to assess value-based pricing for cell and gene therapies. They will facilitate a discussion on how to prepare the healthcare system for the advancement of cell and gene therapies, from the perspectives of manufacturers, policy makers, and payers.