PURPOSE: Developing cost-effectiveness models (CEMs) and budget impact models (BIMs) for cell and gene therapies (C&GTs) is inherently challenging due to the lack of consistent control arms, small sample sizes in registrational trials, difficulty in defining durable clinical cures using surrogate endpoints, defining treatment-eligible populations, and high upfront costs. Ongoing changes to the regulatory environments in the EU introduce additional uncertainties. This workshop discusses some of the relevant methodological and organizational challenges in developing value dossiers for C&GTs from multiple perspectives and approaches to addressing these challenges in the context of evolving regulatory environments.

DESCRIPTION: Dr. Steuten (12 minutes) will present a framework for conceptualizing CEMs that incorporates multiple requirements of various EU countries vis-à-vis PICO criteria, building scenarios in the core model to meet these expectations (12 minutes). Mr. Ektare will discuss the framework for constructing simplified BIMs tailored for both national and local policymakers, emphasizing the need for scenarios that account for alternative payment models, such as annuity-based payments, and the integration of Real-World Evidence (RWE) for monitoring treatment effectiveness (12 minutes). Dr. Fameli will discuss what readiness looks like for manufacturers, how manufacturers adapt to the changing landscape of regulations, and what opportunities and critical challenges are faced internally. Finally, Dr. Ghabri will discuss how the current HTA environments can streamline market access for these life-saving treatments by promoting evidence-based decision-making and collaboration between countries, such as opportunities for harmonized modeling practices and designing and sharing registry data and models (12 minutes). The workshop will conclude with a 12-minute Q&A session. This workshop is designed for health economists, policymakers, industry analysts, and other stakeholders in developing and assessing economic models for innovative therapies. The audience will be polled for the most pressing challenges and the types of methods and data they want to see to overcome them.