Presentation (CTI)

OBJECTIVES: In response to Independence Blue Cross’ recent drug coverage policy update in Pennsylvania to exclude non-oncology drugs from reimbursement within 18 months of a drug’s accelerated approval (AA), we sought to explore how this policy impacts the current landscape of ongoing AA non-oncology drugs.
METHODS: We analyzed the FDA AA program drug list at different timeframes to understand the ongoing AA drug mix by therapeutic area for non-oncology drugs. We included all oncology and non-oncology AA drug indications that have not been withdrawn from the market as of January 1^st, 2025, and have yet to convert to full FDA approval. In addition, the FDA’s Orphan Drug Product Designation database was used to determine if a disease was classified as rare.
RESULTS: Since 1996, we found 96 total ongoing drug-indication accelerated approvals, of which 29% were non-oncology indications (28/96). In the past 18 months, there was a similar proportion of non-oncology drug indications (29% or 8/28). When focusing on non-oncology approvals, almost two-thirds of non-oncology indications are within rare diseases (18/28, or 64%). After applying a 5-year lookback period, from 2019-2024, we found 17 ongoing non-oncology drug-indication accelerated approvals; most of these drug-indications were for rare diseases (9/17, or 53%), and the remaining non-rare indications were for infectious, kidney, hepatic, or neurological diseases.
CONCLUSIONS: These results demonstrate that non-oncology drugs make up approximately one-third of total accelerated approvals and that rare diseases make up the majority of non-oncology accelerated approvals. Payer exclusions to non-oncology accelerated approval drugs may have devastating consequences for patients with rare disease and others with high unmet need.