For which diseases do broader value elements matter most? An evaluation across 20 ICER evidence reports

Area of Science:

• Health economics
• Health technology assessment
• Pharmacoeconomics

Background:

• U.S. value frameworks, like those from the Institute for Clinical and Economic Review (ICER), often use a health system perspective in cost-effectiveness analyses.
• These frameworks may not fully capture the societal and broader benefits and costs of healthcare interventions.
• The impact of including broader value elements on quantitative treatment value estimates remains unclear.

Purpose of the Study:

• To evaluate how the relevance of broader value elements in cost-effectiveness analysis varies across different diseases.
• To identify associations between broader value elements, disease rarity, treatment cost, cost-effectiveness, and ICER committee voting outcomes.

Main Methods:

• Identified 32 broader value elements (e.g., caregiver burden, health equity, productivity) through a literature review.
• Analyzed ICER evidence reports (July 2017-January 2020) to determine which broader value elements were discussed for specific diseases.
• Examined correlations between cited broader value elements and factors like rare disease status, treatment cost, cost-effectiveness, and ICER voting results.

Main Results:

• Household and leisure activities (e.g., caregiver burden) were the most frequently cited broader value element category.
• Inherited retinal disease (19 elements) and sickle cell disease (18 elements) had the most cited broader value elements.
• Rare diseases (15.9 elements) and less cost-effective treatments (higher ICERs) were associated with a greater number of cited broader value elements compared to non-rare diseases (11.5 elements).

Conclusions:

• The inclusion and relevance of broader value elements in cost-effectiveness analyses differ significantly across diseases.
• Treatments with less favorable cost-effectiveness ratios tend to involve more broader value considerations.
• Comprehensive inclusion of all relevant value elements is crucial for incentivizing innovation and optimizing research funding allocation.