PURPOSE:

The objective of this workshop is to discuss the evolving role of real-world evidence (RWE) in pricing and reimbursement evaluations of innovative one-time treatments, such as CAR-Ts and gene therapies across global markets.

DESCRIPTION:

Innovative, single or short-term treatments, have shown exciting potential for translating early response into durable long-term outcomes. Due to high unmet need, these therapies are currently being granted marketing authorization based on non-comparative evidence packages with limited duration of follow-up. While these novel approaches may offer potentially curative outcomes, the lack of robust supporting data at launch creates uncertainty in the duration of clinical benefit and payer hesitation for covering their unprecedented costs. To mitigate the uncertainties, payers are increasingly turning to RWE to evaluate whether these therapies are fulfilling their initial curative promise. While reassessments based on local post-marketing data are becoming an increasing point of interest across global markets, the magnitude of impact from RWE in influencing pricing and access decisions is not yet certain.

During the workshop, panelists and participants will discuss key issues and considerations regarding the collection and evaluation of RWE in the context of single or short-term treatments, including what procedures are currently in place for such assessments and how these are expected to evolve. Drawing on recent examples, the discussion will explore the current climate and payer perceptions of post-marketing data and innovative payment models as levers to drive positive assessments and pricing negotiations. Additionally, the role of the manufacturers from an evidence generation perspective will be evaluated.

The final outputs of the panel will provide a critical look at the future use of RWE for evaluation of innovative single or short-term treatments. Current challenges from the payer and manufacturer perspectives will be outlined and potential targeted mitigation strategies will be proposed based on panel and audience feedback.