Using RWE to Inform the Value and Affordability Assessment of Cell and Gene Therapies

Separate registration required.

This short course explores the role of real-world evidence (RWE) in supporting the economic evaluation of cell and gene therapies (CGTs). Many CGTs are one-time therapies that have the potential to offer transformative sustained benefits for patients with severe conditions. In many situations, the ‘do-nothing’ alternative is the norm, so the need for a control arm may be futile yet reducing the options for patients to be included in allegedly active arms. However, at launch there is often resistance from payers to reimburse these potentially transformative therapies due to the limited validity of the supporting evidence (small, single arm trials, etc) which leads to uncertainty regarding: the size and heterogeneity of the patient population eligible for CGTs; the definition of standard of care and natural disease progression, given CGTs may unlock treatment possibilities of previously deemed untreatable and rare diseases; the novel therapy’s duration of benefit; and the relative effectiveness of the novel therapy compared to the current standard of care, particularly, as cell and gene therapies are often only supported by a single arm trial.

Payer concerns with these uncertainties in the evidence are heightened by the typically high up-front costs associated with cell and gene therapies and the consequence for their affordability. While there is generally not a good understanding of the effect sizes and costs of standard of care.

This course will provide an overview of the potential contribution, planning and use of RWE to help address these concerns. We will assume payer archetypes that are focused on one or more of relative effectiveness and cost-effectiveness (value), and budget impact (affordability). Within these archetypes, we will discuss the role and acceptability of RWE to inform payment and policy with emphasis on eligibility, appropriate comparators, durability of benefit, spending for patients that meet criteria for eligibility and the development of appropriate outcome-based agreements. A strong focus will be brought on validity (internal and external) of the existing data. We will further provide participants with real-world examples of using RWE to inform policy making. This course will target a wide range of participants, from medical operations to HEOR, interested in understanding the depth of issues to consider when balancing access with payment for CGTs.

PREREQUISITE: This course requires familiarity with basic economic evaluation and HTA concepts and methodologies of pharmaceuticals.