Problem Statement: Evidence generation and synthesis (EGS) is a long-term strategic process required for supporting product value proposition for multiple stakeholders at launch and beyond, and can involve thinking ahead to likely launch needs at the early trial design stages. In this case study, using publicly available data, we highlight two innovative therapies that utilized EGS to achieve market access and reimbursement.

Description: EGS was operationalized to support value propositions for Zolgensma for SMA Type I and Sleepio for dCBT-I. Zolgensma provides an example for rare disease (high price, low volume) while Sleepio provides an example for a high prevalence disease (low price, high volume). Zolgensma’s value proposition for SMA Type I is based on improvement in survival and health-related quality of life (HRQoL) while Sleepio’s value proposition is based on reduction in total cost of care. The EGS strategy for Zolgensma relied on showing milestone maintenance/improvement in the clinical trial and long-term follow-up. In contrast, the EGS strategy for Sleepio was based on showing clinical equivalence to treatment as usual based on results of twenty-eight studies including 12 trials. This enabled the company to focus exclusively on total cost of care reduction in subsequent modelling activities.

Lessons Learned: It is beneficial to craft an EGS strategy concurrently with value proposition development as early as possible. As the value proposition matures, the EGS strategy should be refined to highlight evidence gaps and value drivers via health economic models that are clinically validated and economically sound. Understanding cost offsets and HRQOL for the intended population and comparators is important when gathering evidence to support market access and reimbursement.

Stakeholder Perspective: The case study is from a manufacturer perspective and the EGS strategy development is from multiple stakeholders’ perspective including patients/caregivers, providers, and payers/health technology assessment agencies.