Understanding payer perceptions of a novel trial design
Analysis of the opportunities for performing basket studies across global markets in the context of rare epilepsy syndromes
EU4 + UK, Australia, Brazil, Japan and US
Therapy Area:
Medium sized pharmaceutical company
The Challenge
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
Project objectives
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
The client was provided with a comprehensive assessment of the clinical and commercial opportunity associated with utilising a basket study design where traditional clinical trial designs may present challenges

Strategic recommendations focussed on key recommendations for additional opportunities/workstreams to maximise the viability of a novel approach to clinical studies for rare epilepsy syndromes

The market access and clinical teams were able to gain a consensus on the potential limitations of conducting basket studies from a payer perspective