Variation in Commercial Coverage of Accelerated Approval Drugs


LaMountain F, Beinfeld M, Chambers J
Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, MA, USA

OBJECTIVES: The FDA-Accelerated Approval (AA) program speeds patients access to drugs that: (i) treat a serious condition, (ii) provide a meaningful advantage over available therapies, and (iii) demonstrate an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit. However, uncertainties about the underlying evidence supporting AA drugs challenges healthcare payers. We assessed commercial health plan coverage for 60 AA drugs.

METHODS: We used the Tufts Medical Center Specialty Drug Evidence and Coverage Database to identify drugs granted FDA-AA status 2016-2021. We identified 60 drugs with at least 10 coverage policies issued by 18 large US commercial payers (current April 2023). We categorized policies according to step therapy protocol (patients must first try an alternative therapy) and subgroup restrictions (patients must meet certain clinical criteria, e.g., disease severity) beyond the FDA label. We stratified the results by oncology and orphan drug designation.

RESULTS: Our sample included 1,206 coverage policies for 60 AA drugs, the majority of which were for oncology (87%) and orphan (72%) indications. Among available coverage policies, 28% included restrictions beyond the FDA label (most often subgroup restrictions). Payers did not impose step therapy requirements in 92% coverage policies. Payers covered oncology drugs more often than non-oncology drugs (non-coverage was <1% for oncology and 20% for non-oncology drugs, e.g., drugs indicated for muscular dystrophy) and with fewer restrictions than non-oncology drugs (21% vs 82%). Payers included restrictions on coverage for orphan drugs more often than for non-orphan drugs (32% vs 20%).

CONCLUSIONS: Commercial plans cover AA drugs in a manner largely consistent with the FDA label, however coverage between oncology and non-oncology drugs varies. Payers are less likely to cover AA drugs for non-oncology conditions that are associated with high-costs and limited evidence without restrictions.

Conference/Value in Health Info

2024-05, ISPOR 2024, Atlanta, GA, USA

Value in Health, Volume 27, Issue 6, S1 (June 2024)




Health Policy & Regulatory

Topic Subcategory

Insurance Systems & National Health Care, Reimbursement & Access Policy


Drugs, Rare & Orphan Diseases

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