OBJECTIVES: This research aims to evaluate the relationships between disease prevalence, first-in-indication (FII) products, first-in-class (FIC) products, and launch wholesale acquisition cost (WAC).

METHODS: The FDA’s Center for Drug Evaluation and Research (CDER) publishes a list of new molecular entities (NMEs) and biological products approved each year. The list excludes cell and gene therapies. CDER NME approval lists from 2015-2022 were documented, from which oncology and non-orphan products were removed. Only the first-approved indications were considered. Estimates of US prevalence and WAC per patient per year/course of therapy at launch were calculated. FII/FIC therapies were classified. Multivariable linear regressions were performed evaluating WAC as a dependent variable of US prevalence and FII/FIC classifications.

RESULTS: A total of n=76 orphan approvals were assessed, of which 41 were FII and 35 were approved after at least 1 on-label alternative. Additionally, n=41 products were classified as FIC by the FDA. There was a higher expected price for FII orphan products compared to non-FII products ($210,565, p=0.010). There was also a higher expected price for FIC products compared to non-FIC products ($36,617, p=0.661). Performing a non-ultra orphan (≥1,000 US prevalence) subgroup analysis similarly resulted in a higher expected price for FII products ($70,243, p=0.251) and FIC products ($24,066, p=0.699). Performing an ultra-orphan (≤1,000 US prevalence) subgroup analysis yielded a higher expected price for FIC products compared to non-FIC ($235,091, p=0.437). All ultra-orphan products in the dataset were FII.

CONCLUSIONS: Analysis strongly suggests that FII and FIC non-oncology rare disease therapies have significantly higher prices relative to non-FII and non-FIC orphan products. FII appears to be more impactful on manufacturer pricing than FIC.