Evaluating the Impact of Disease Prevalence and Product Administration on Launch Pricing Trends for Non-Oncology Orphan Products

OBJECTIVES: This research aims to evaluate the relationships between disease prevalence, therapy administration, and duration of therapy on 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. All CDER NME approval lists from 2015-2022 were documented, from which non-orphan and oncology 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. Multivariable linear regressions were performed evaluating WAC as a dependent variable of US prevalence, therapy administration, and duration of therapy.

RESULTS: A total of n=76 orphan approvals were assessed. There was a highly statistically significant correlation between prevalence and launch WAC for orphan therapies (p=0.001). Segmenting the data into ultra (≤1,000 US prevalence) and non-ultra (≥1,000 US prevalence) orphan products resulted in P-values of 0.917 and 0.001 respectively. Among the 76 products, 27 were healthcare professional (HCP)-administered and 49 were patient-administered. There was a statistically insignificant higher expected price for HCP-administered drugs, as the analysis suggested an expected price $148,180 higher than patient-administered products (p=0.089). The dataset was then segmented by duration of therapy, of which 69 were administered chronically and 7 were administered for a limited period. There was a statistically insignificant higher expected price for chronically administered drugs as the data suggested an expected price $220,170 higher than limited duration products (p=0.122).

CONCLUSIONS: This analysis strongly suggests that prevalence is the driving factor in manufacturer pricing decisions. Therapy administration and duration of therapy appear to be somewhat related with price, but do not achieve statistical significance in the analysis. Non-ultra orphan therapies have a stronger price/prevalence correlation than ultra-orphan products.