Given that reimbursement frameworks are obsolete when it comes to these newer therapies, payors acknowledge the need for specific clinical evidence in order to assess product value and are seeking alternative payment models. The lack of matured efficacy and safety data, which are generally guiding reimbursement principles, casts doubt on the durability of clinical benefit. Payors are posed with a significant financial risk if they provide the full payment upfront and a patient stops responding to one-time treatment. On the other hand, if insurers were to stagger payments over time in order to monitor treatment effect, a risk arises with patient healthcare plan portability. With either approach, the stakes remain high.
Value-based and outcomes-based contracting models are the predominant reimbursement approaches currently being undertaken in the US by gene therapy manufacturers to increase the rebate willingness of payors. Novartis, Gilead, and Spark have all tried different and creative variations to contracting approaches, but the need remains for a reimbursement model specifically tailored towards gene therapy products.
Thus, the FDA is currently working on six disease specific gene-therapy guidelines to provide manufacturers and payors with alternative data assessment recommendations, set the groundwork for future treatments and galvanize the development of methodical payment models.