Will the Institute for Clinical and Economic Review's Shared Savings Approach Decrease Value-Based Prices Most for the Most Severe Diseases?

Abstract

Objectives: To identify the types of disease most likely to be affected by the Institute for Clinical and Economic Review's (ICER) shared savings assumptions.

Methods: For diseases with treatments that were Food and Drug Administration approved between 2019 and 2023, annual direct and indirect economic burden and characteristics of each disease were extracted from peer-reviewed literature. ICER's shared savings methodology was applied 2 ways: 50/50 shared savings and $150 000 cost-offset cap. The primary outcome was the difference in eligible cost savings provided by a hypothetical disease cure under ICER's 2 shared savings methods. Characteristics of diseases most impacted by these 2 methods were evaluated descriptively.

Results: Food and Drug Administration approved 260 therapies for 89 unique diseases between 2019 and 2023. Shared savings reduced value of a hypothetical cure for hemophilia A most (50/50 method: -$367 670 per year; cap method: -$585 340 per year), followed by acute hepatic porphyria (50/50 method: -$333 948; cap method: -$517 896) and paroxysmal nocturnal hemoglobinuria (50/50 method: -$291 997; cap method: -$433 993). Compared with diseases with annual burdens <$150 000, those ≥$150 000 had earlier disease onset by 22.0 years (age 12.3 vs 34.3), lower life expectancy by 10.6 years (55.8 vs 66.4 years), and lower disease prevalence (4.7 vs 1981.5 per 100 000). Shared savings' impact on health-benefit price benchmarks was projected to be larger for diseases with shorter life expectancy (ρ = -0.319; p =.005), worse quality of life (ρ = -0.263; P =.020), and lower prevalence (ρ = -0.418; P < .001).

Conclusions: ICER's shared savings assumptions would most likely have the largest negative impact on health-benefit price benchmarks for rare, severe, and pediatric diseases.