That is the title of a paper recently accepted for publication by Value in Health with co-authors Shanshan Wang, Khounish Sharma, Kathryn Spurrier, Robert J. Nordyke. The abstract is below.
Objectives
To identify the types of disease most likely to be impacted by the Institute for Clinical and Economic Review’s (ICER) shared savings assumptions.
Methods
For diseases with treatments that were FDA-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 two 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
FDA 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 to 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=0.005), worse quality-of-life (ρ=-0.263; p=0.020), and lower prevalence (ρ=-0.418; p<0.001).
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.
You can read the full paper here.
