Presentation (CTI)

OBJECTIVES: Orphan drugs are defined by the FDA as drugs treating diseases with a prevalence <200,000 or which will not be profitable within 7 years post-approval. In 2023, our team produced a tracking system summarizing the significant characteristics (e.g., prevalence, price, route of administration) of approved orphan drugs, but data did not reflect recent orphan approvals. This research aims to further articulate the ‘typical’ orphan drug, segment approvals by their attributes, and identify any new trends in orphan product characteristics.
METHODS: We referenced the CDER database to collect all FDA approvals from 2015-2024 (CDER excludes cellular and gene therapy products, blood products, plasma derivatives, allergenic products, and vaccines). All oncology and non-orphan products were then removed by comparison with the orphan drug database. Some products under exceptional circumstances (e.g., humanitarian applications) were excluded. Secondary sources were referenced to collect the launch annual WAC, prevalence, treatment duration, route of administration, and level of competition for products.
RESULTS: n=108 orphan drugs were evaluated, n=31 being new approvals (2023/2024). Overall, products had an average annual WAC of $403,569 and average US prevalence of 17,175. 91% of products were for chronic use and 60% were first in class therapies (the average number of indicated competitors across all products was 1.3). Most products were orals (42%), followed by subcutaneous products (29%), IVs (26%), and topicals (2%); 66% of products are self-administered.
CONCLUSIONS: Recent orphan approvals have increased the average annual WAC of orphan drugs by ~$10k while also slightly increasing average prevalence, reflecting industry trends towards higher-cost therapies. Subcutaneous therapies have overtaken IV therapies as the second most-common route of administration, indicating investment in patient convenience and adherence.