Presentation (CTI)

OBJECTIVES: Different routes (e.g., oral/subcutaneous/IV) and methods (HCP/patient) of administration are generally associated with higher or lower prices given varied economic incentives across sites of care, manufacturing costs, and adherence/convenience value propositions. The extent to which this holds true for non-oncology orphan products (where convenient RoAs may be relatively less significant to manufacturers and payers) is unclear. This research aims to evaluate the impact of product administration on WAC price alongside disease prevalence (an established significant price factor) and the extent to which this impact has increased or diminished with the previous two years of orphan drug launches.
METHODS: All FDA CDER new molecular entity approval lists from 2015-2024 were documented, then non-orphan and oncology products were removed (CDER excludes cellular and gene therapy products, blood products, plasma derivatives, allergenic products, and vaccines). US prevalence and WAC at launch were calculated for each therapy. For products with multiple indications, only the first-approved indications were considered. Estimates of US prevalence, and WAC per patient per year/course of therapy at launch were calculated. Product administrator and RoA were classified. Multivariable linear regressions were performed evaluating WAC as a dependent variable of US prevalence, route of administration, and administrator.
RESULTS: A total of n=108 orphan drug launches were assessed, 32 of which were launched in the previous two years. Average prices varied across oral ($314k), subcutaneous ($449k), and intravenous ($517k) routes of administration. HCP-administered drugs ($515k) were costlier than patient-administered therapies ($346k).
CONCLUSIONS: HCP-administered products launch at higher prices relative to patient-administered products, and IV products remain costlier than subcutaneous therapies, which are both costlier than oral therapies. Pricing differences have generally persisted with recent launches, but further research may examine more closely how administration relates to other pricing factors (e.g., indication severity).