Early Value Assessment in Gene Therapy Clinical Trial Design
A global biotechnology company engineering precision genetic medicine for rare diseases was in need of expertise in early assessment of proposed clinical trial designs and their potential payer coverage. They engaged with Certara’s Cell & Gene Therapy practice because of its proven experience in tailoring commercialization support to the needs of gene therapy drug development.
Certara Evidence and Access commercial strategists assessed potential coverage policies for the company’s gene therapy in development in several clinical trial designs and at several fixed prices. Our key finding was that with the proposed trial design and criteria, the payer access would have narrowed the population such that the revenue objectives were impossible to meet.
Certara Evidence and Access provided the evidence that the company’s commercial team used to build the case to change the clinical trial design.
Clinical trial in/ex criteria for gene therapies are often very specific to ensure positive outcomes in few patients. However, commercial aspirations, rooted in the breadth of payer access, may consider vastly different patient populations.
