Traditional clinical trial designs may be prohibitive to the commercialisation of assets in rare epilepsy syndromes due to small target populations, and lack of a defined standard of care across the population due to the heterogeneity of individual disease

The client wanted to understand potential payer value associated with non-traditional clinical trial designs, particularly basket studies, to demonstrate effect of Product X in these syndromes

Provide an overview of varying “non-standard” trial types that have previously been used for the study of orphan indications

Scope out the opportunities to perform basket studies across global markets in the context of rare epilepsy syndromes

Provide an understanding of the payer value that can be derived from basket studies and how this should be optimised across key global markets

Development of a comprehensive set of recommendations for the optimal conditions for non-registration “non-standard” studies was required

A programme of targeted desk research was completed to provide an overview of varying “non-standard” study types that have supported orphan indications globally, including an analogue analysis

Primary research was completed with 27 payers across 9 markets to validate study designs, including a review of an existing study protocol to highlight challenges/opportunities associated with using basket studies to demonstrate payer relevant value to ensure positive HTA/ coverage outcomes

Research findings were analysed by the Lightning Health team, to inform a final report on the next steps for clinical development