Abstract
BACKGROUND: Clinical outcome assessments (COAs) are tools widely used in clinical trials to evaluate treatment efficacy by capturing patient experience. However, little is known about how COAs are used in specialty drug coverage decisions. OBJECTIVE: To describe the frequency, type, and role of COAs in US commercial health plans' specialty drug coverage policies. METHODS: We analyzed coverage data from the Tufts Medical Center Specialty Drug Evidence and Coverage (SPEC) Database, which tracks specialty drug coverage policies from 18 large US commercial health plans. Researchers reviewed each policy to identify disease-specific COAs, excluding tools assessing objective physiological data or clinical algorithms (eg, biomarkers, risk prediction scores). We categorized COAs by (1) type (ie, patient-reported outcomes [PROs], clinician-reported outcomes [ClinROs], etc), (2) time point (initial approval vs reauthorization), and (3) application (ie, supporting diagnosis, documenting baseline, limiting access for initial criteria; or defining meaningful treatment response for reauthorization criteria). RESULTS: As of April 2024, SPEC included 13,933 coverage policies for 440 specialty drugs for 386 indications. These policies incorporated COAs for 169 (43.8%) indications. In total, 2,188 (15.7%) policies referenced at least 1 COA, and of these 763 (34.9%) referenced multiple COAs. Because policies can incorporate COAs into both initial and reauthorization criteria, we identified 4,305 total COA references. Inclusion of COAs varied across plans, ranging from 5% to 27.2% of coverage decisions. COAs appeared 2,077 times in initial criteria, most commonly as ClinROs (69.0%) and infrequently as PROs (5.7%). In initial criteria, plans primarily used COAs to limit access (50.5%), whereas in reauthorization criteria, plans used COAs to determine treatment response. CONCLUSIONS: COA inclusion varied across plans, with most using them to limit access. ClinROs were the most frequently used COA type, underscoring the pivotal role of providers on behalf of patients and health plans in shaping treatment access decisions. Plans used PROs relatively infrequently, with some including PROs in their policies more than others, suggesting a missed opportunity to consistently incorporate patients' voices to complement the assessment of treatment outcomes when determining coverage. Future research should explore the rationale behind COA use and its implications for treatment access.