Hemophilia Gene Therapy Value Assessment: Methodological Challenges and Recommendations

Area of Science:

• Medical economics
• Gene therapy
• Hematology

Background:

• Gene therapy for hemophilia offers long-term health gains but involves high upfront costs.
• Assessing the clinical and economic value of these one-time treatments at launch is challenging due to uncertainties in long-term benefits.

Purpose of the Study:

• To identify and discuss methodological challenges in evaluating the value of hemophilia gene therapies.
• To propose recommendations for a robust framework for assessing gene therapy value.

Main Methods:

• Review of existing frameworks, specifically the Institute for Clinical and Economic Review's framework for single or short-term therapies (ICER-SST).
• Identification of key challenges: durability of benefits, defining cure, trial design limitations, quality of life measures, cost-savings, and perspective.

Main Results:

• Uncertainty in durability of benefits and lack of cure definition hinder value assessment.
• Limitations exist in traditional trial designs and quality of life measures for hemophilia.
• Current frameworks may not fully capture the value of innovative gene therapies.

Conclusions:

• Leverage expert opinion for benefit durability assumptions.
• Employ external synthetic controls and modified utility measures.
• Consider outcome-based contracting and multiple perspectives (societal, healthcare system) for comprehensive value assessment.