(17) Does time on market influence the evidence cited in U.S. commercial health plan specialty drug coverage policies?

Background: : Specialty drug coverage is a critical component of commercial health plans in the United States, providing access to treatments that address complex medical conditions. The type of evidence cited in support of coverage decisions may change over time.

Objective: : This study explores the relationship between the duration a drug has been on the market and the types of evidence cited in coverage policies.

Methods: : We used the Tufts Medical Center Specialty Drug Evidence and Coverage (SPEC) database, which includes publicly available coverage decisions for 469 drugs from 18 large US commercial health plans, as well as the evidence cited in support of these decisions. We categorized evidence as (1) randomized controlled trial (RCT), (2) other clinical study (e.g., phase 2 clinical trial), (3) real-world evidence (RWE), (4) systematic review/meta-analysis, or (5) health technology assessment (HTA) or economic evaluation. For each study, we identified the drugs and FDA-labeled indications assessed, along with years since FDA approval for each drug-indication pair. We then assessed how the types of evidence cited varied by time on the market, grouped by years since FDA approval. Data were current as of August 2024.

Results: : A total of 8,490 citations were identified from coverage decisions active in August 2024. For treatments on the market for 1-5 years, RCTs and other clinical studies collectively accounted for 82.5% of citations (1,441 out of 1,746), with RWE comprising 3.7%, systematic reviews/meta-analyses 10.3%, and HTAs or economic evaluations representing 3.5%. For treatments on the market 10-15 years, RCTs and other clinical studies constituted 69.5% of citations (1,372 out of 1,975), with RWE accounting for 14.1% of citations, systematic reviews/meta-analyses 13.1%, and HTAs or economic evaluations representing 3.4%. For treatments on the market 25+ years, RCTs and other clinical studies comprised 51.1% of citations (283 out of 554), with RWE accounting for 21.8% of citations, systematic reviews/meta-analyses 19.7%, and HTAs or economic evaluations constituting 7.4%.

Conclusions: : U.S. commercial health plans initially rely on RCTs and clinical studies for coverage decisions of new specialty drugs. Over time, they increasingly refer to RWE, systematic reviews/meta-analyses, HTAs, and economic evaluations. Product manufacturers should anticipate these shifting evidence needs and focus on generating varied types of evidence as their products mature.

Sponsorship:: EMD Serono.