Does Expedited Regulatory Approval Status Increase Rare Disease Drug Non-Coverage?
Author(s)
Fraser M1, Clifton K2, Privett B3
1Red Nucleus, Cambridge, MA, USA, 2Red Nucleus, Freedom, NH, USA, 3Red Nucleus, Brookline, MA, USA
Presentation Documents
OBJECTIVES: The FDA uses the Accelerated Approval pathway to allow for earlier approval of drugs that treat serious conditions and fill an unmet medical need based on a surrogate endpoint. Manufacturers are required to release full trial data after launch. The FDA also uses the Fast Track pathway to facilitate development and review of drugs that treat serious conditions with unmet need on an expedited timeline, but with full data. We aim to show that payer coverage/non-coverage and patient access of drugs approved through expedited pathways are not related to their pathway status, but rather the strength of their evidence package. METHODS: We isolated 10 non-oncology, non-infectious rare disease drugs that received Accelerated Approval and 10 that received Fast Track approval. We then analyzed coverage vs. non-coverage across the policies of 10 commercial payers. We used Chi Square testing to analyze the difference in non-coverage between the groups. We also scored the patient access of these drugs, from least restrictive (3) to most restrictive (0) and analyzed the effect of the pathway on coverage restrictiveness using Chi Square testing. RESULTS: Drugs with Accelerated Approval have significantly more non-coverage policies than drugs with Fast Track approval (p < 0.01, df=1). Through our scoring process, payer policies for Accelerated Approval drugs are on average more restrictive on patient access (1.26) than Fast Track drugs (1.51). CONCLUSIONS: Non-oncology, rare disease Accelerated Approval drugs face more non-coverage than non-oncology, rare disease Fast Track drugs. By definition, Accelerated Approval drugs launch with incomplete data. Thus, our research demonstrates that the robustness of data packages, independent of an expedited regulatory status, is influential on payer coverage and patient access.
Conference/Value in Health Info
2024-05, ISPOR 2024, Atlanta, GA, USA
Value in Health, Volume 27, Issue 6, S1 (June 2024)
Code
HPR22
Topic
Health Policy & Regulatory
Topic Subcategory
Approval & Labeling, Coverage with Evidence Development & Adaptive Pathways, Reimbursement & Access Policy
Disease
Rare & Orphan Diseases