Clinician-Reported Outcomes—A Rare Opportunity for Orphan Labels?
Author(s)
Kristen A. Cribbs, PhD, MPH, Lucas T. Blackmore, MPH, Betsy J. Lahue, MPH;
Alkemi, Manchester Center, VT, USA
Alkemi, Manchester Center, VT, USA
Presentation Documents
OBJECTIVES: FDA and ISPOR guidance (2020-2023) highlights the importance of Clinician-Reported Outcome Measures (ClinROMs) for capturing indirect observations of patient symptoms and functioning, either as a complement to, or substitute for, direct patient self-reporting when the latter is not feasible. This study is the first to characterize ClinROM-based labeling inclusion for FDA-approved orphan drugs.
METHODS: We reviewed FDA orphan drug databases to identify molecular entities (NMEs) and biologic license applications (BLAs) receiving orphan designation between January 2018-October 2024. FDA-approved labels reporting ClinROM study endpoints met inclusion criteria. Trial records, package inserts, and instrument references were reviewed, and data were abstracted on drug approval year, breakthrough status, patient-reported outcome measure (PROM) inclusion, ClinROM endpoint ranking, outcomes measured, and validation status. Results were analyzed descriptively; Year-over-year and overall trends were assessed (chi-square, p-value<0.05).
RESULTS: ClinROM-based labeling was included in 10% (19/198) of FDA-approved orphan NMEs and BLAs. Labels from 2024 (8/19, 42%) had greatest ClinROM inclusion, and 37% (7/19) of labels were for drugs with breakthrough designation. Among labels including ClinROMs, 42% (8/19) also included a PROM. Median 2 ClinROMs were used per label (range: 1-5), with 22 unique ClinROMs included across labels. ClinROMs were primary endpoints in 74% of labels (14/19) and secondary endpoints in 32% (6/19). Five labels (5/19, 26%) included >1 ClinROM (four included 2 tools, one included 5). Most ClinROMs captured symptom-related (13/22, 59%) versus functional (9/22, 41%) outcomes, and all were validated. No significant trends were observed for tool use or ranking, including for breakthroughs.
CONCLUSIONS: ClinROM-based orphan FDA labeling is uncommon. Labels with ClinROMs often included these tools as primary endpoints, to measure symptom-related outcomes, and in addition to patient-reported measures. Greater ClinROM integration in orphan trials may enhance patient-relevant treatment and care, both when PROMs are infeasible, and to complement patient-reported experiences.
METHODS: We reviewed FDA orphan drug databases to identify molecular entities (NMEs) and biologic license applications (BLAs) receiving orphan designation between January 2018-October 2024. FDA-approved labels reporting ClinROM study endpoints met inclusion criteria. Trial records, package inserts, and instrument references were reviewed, and data were abstracted on drug approval year, breakthrough status, patient-reported outcome measure (PROM) inclusion, ClinROM endpoint ranking, outcomes measured, and validation status. Results were analyzed descriptively; Year-over-year and overall trends were assessed (chi-square, p-value<0.05).
RESULTS: ClinROM-based labeling was included in 10% (19/198) of FDA-approved orphan NMEs and BLAs. Labels from 2024 (8/19, 42%) had greatest ClinROM inclusion, and 37% (7/19) of labels were for drugs with breakthrough designation. Among labels including ClinROMs, 42% (8/19) also included a PROM. Median 2 ClinROMs were used per label (range: 1-5), with 22 unique ClinROMs included across labels. ClinROMs were primary endpoints in 74% of labels (14/19) and secondary endpoints in 32% (6/19). Five labels (5/19, 26%) included >1 ClinROM (four included 2 tools, one included 5). Most ClinROMs captured symptom-related (13/22, 59%) versus functional (9/22, 41%) outcomes, and all were validated. No significant trends were observed for tool use or ranking, including for breakthroughs.
CONCLUSIONS: ClinROM-based orphan FDA labeling is uncommon. Labels with ClinROMs often included these tools as primary endpoints, to measure symptom-related outcomes, and in addition to patient-reported measures. Greater ClinROM integration in orphan trials may enhance patient-relevant treatment and care, both when PROMs are infeasible, and to complement patient-reported experiences.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
PT13
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
SDC: Rare & Orphan Diseases