Use of Clinical Outcome Assessments in Specialty Drug Coverage Policies
Author(s)
Julia A. Rucker, MPH/MSW1, Jiat Ling L. Poon, PhD2, Fariel C. LaMountain, B.A.1, Yichen Lin, B.A.1, Molly T. Beinfeld, MPH1, Megan N. Klopchin, MPH, MHA2, James D. Chambers, MSc, PhD1;
1Tufts Medicine, Boston, MA, USA, 2Eli Lilly and Company, Indianapolis, IN, USA
1Tufts Medicine, Boston, MA, USA, 2Eli Lilly and Company, Indianapolis, IN, USA
OBJECTIVES: Clinical outcome assessments (COAs) are instruments used to measure how patients feel, function, or survive. While COAs are widely used in clinical trials to assess treatment efficacy, there is limited information on their role in specialty drug coverage. Our objective was to document the frequency, types, and purpose of COAs embedded in US commercial health plans’ specialty drug coverage policies.
METHODS: We analyzed information from the Tufts Medical Center Specialty Drug Evidence and Coverage (SPEC) Database, which tracks coverage decisions from 18 large US commercial health plans. We reviewed coverage decisions to identify disease-specific COAs, excluding assessments that do not involve direct patient input (e.g., biomarkers or risk prediction scores). COAs were categorized by type: ‘patient-reported outcomes’ (PRO), ‘observer-reported outcomes’ (ObsRO), ‘clinician-reported outcomes’ (ClinRO), ‘performance outcomes’ (PerfO) as well as ‘composites’ for assessments that include multiple COAs, and ‘COA, unspecified’ if an outcome is required, but no specific instrument named.
RESULTS: As of April 2024, the SPEC Database included 386 disease indications, with 152 (39%) featuring at least one COA in coverage policies. Nearly half (47%) included multiple COAs. Spinal muscular atrophy (n=12 COAs) and multiple sclerosis (n=11 COAs) were the indications with the most COAs. Frequently cited COAs included the Eastern Cooperative Group assessment (n=62 indications), the 6-Minute Walk Test (n=14 indications), and the New York Heart Association Functional Class (n=12 indications). Of 166 unique COAs, 14% were ‘COA, unspecified’, while 86% had sufficient detail for further classification. Among the classified COAs, 32% were ClinROs, 25% composites, 23% PROs, 18% PerfOs, and 2% ObsROs.
CONCLUSIONS: This study provides important insights into the prevalence and types of COAs health plans embed in specialty drug coverage policies. ClinROs, composites, and PROs were the most common COA types. These findings underscore the role of COAs in informing treatment access decisions.
METHODS: We analyzed information from the Tufts Medical Center Specialty Drug Evidence and Coverage (SPEC) Database, which tracks coverage decisions from 18 large US commercial health plans. We reviewed coverage decisions to identify disease-specific COAs, excluding assessments that do not involve direct patient input (e.g., biomarkers or risk prediction scores). COAs were categorized by type: ‘patient-reported outcomes’ (PRO), ‘observer-reported outcomes’ (ObsRO), ‘clinician-reported outcomes’ (ClinRO), ‘performance outcomes’ (PerfO) as well as ‘composites’ for assessments that include multiple COAs, and ‘COA, unspecified’ if an outcome is required, but no specific instrument named.
RESULTS: As of April 2024, the SPEC Database included 386 disease indications, with 152 (39%) featuring at least one COA in coverage policies. Nearly half (47%) included multiple COAs. Spinal muscular atrophy (n=12 COAs) and multiple sclerosis (n=11 COAs) were the indications with the most COAs. Frequently cited COAs included the Eastern Cooperative Group assessment (n=62 indications), the 6-Minute Walk Test (n=14 indications), and the New York Heart Association Functional Class (n=12 indications). Of 166 unique COAs, 14% were ‘COA, unspecified’, while 86% had sufficient detail for further classification. Among the classified COAs, 32% were ClinROs, 25% composites, 23% PROs, 18% PerfOs, and 2% ObsROs.
CONCLUSIONS: This study provides important insights into the prevalence and types of COAs health plans embed in specialty drug coverage policies. ClinROs, composites, and PROs were the most common COA types. These findings underscore the role of COAs in informing treatment access decisions.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
CO106
Topic
Clinical Outcomes
Topic Subcategory
Clinical Outcomes Assessment
Disease
No Additional Disease & Conditions/Specialized Treatment Areas