Real-World Evidence-Driven Modeling Methods to Inform Payment Approaches for Cell and Gene Therapies

Speaker(s)

Discussion Leader: Robert Brett McQueen, PhD, Department of Clinical Pharmacy, Center for Pharmaceutical Outcomes Research (CePOR), Skaggs School of Pharmacy and Pharmaceutical Sciences, University of Colorado Anschutz Medical Campus, Denver, CO, USA
Discussants: Julia F. Slejko, PhD, School of Medicine, University of Maryland Baltimore, Baltimore, MD, USA; Antal Tamas Zemplenyi, PhD, University of Colorado Skaggs School of Pharmacy and Pharmaceutical Sciences, Denver, CO, USA; Andrew York, PharmD, JD, Maryland Prescription Drug Affordability Board, Bowie, MD, USA

Presentation Documents

PURPOSE:

Cell and gene therapies (CGTs) can offer considerable life extension and quality of life improvements for patients with previously untreatable conditions. The European Medicines Agency and the Food and Drug Administration have each approved over 20 CGTs to date. Post approval, manufacturers face reimbursement challenges given many CGTs are up-front high-priced technologies combined with uncertainties around the durability of benefit. Associated financial risk as a function of these uncertainties have led to proposed alternative payment approaches that may improve access while creating predictable budget scenarios. However, reimbursement negotiations may be limited by using short-term regulatory approval evidence alone. The value of real-world evidence (RWE) combined with conventional simulation modeling methods can reduce the aforementioned uncertainties. This session will use case examples from different country-specific settings to discuss how RWE combined with simulation modeling approaches can help reimbursement agencies understand trade-offs of paying for CGTs.

DESCRIPTION:

Workshop attendees will obtain a working knowledge of how RWE and simulation modeling can inform payment strategies for up-front high-cost CGTs. Brett McQueen will provide an overview of global reimbursement strategies and examples of novel payment approaches for CGTs. Julia Slejko will present examples of how payment approaches have been implemented for CGTs in mainland Europe and the role of RWE. Antal Zemplenyi will present a case study illustrating the utilization of real-world data to investigate the association between eligibility criteria, the proportion of eligible patients, and the financial impact for the United States public Medicaid insurance program paying for gene therapies in hemophilia A and B. Finally, Andrew York will provide a policy perspective on the use of RWE to complement clinical trial data when considering different payment models for CGTs. Workshop participants will be asked about their own experience using RWE for payment approaches using ISPOR polling software.

Code

127

Topic

Health Policy & Regulatory