HEALTH CARE RESOURCE USE IN RARE DISEASE CLINICAL TRIALS: A SYSTEMATIC REVIEW
Author(s)
Hema K. Gandhi, PhD, MPH1, Dorothee Oberdhan, PhD2, Jialu He3, Precious Nchekwube, MPH4, Onur Baser, PhD, MS, MA5, Samuel Wagner, PhD6;
1Otsuka Pharmaceuticals Development & Commercialization, Inc., Associate Director, Newtown, PA, USA, 2Otsuka Pharmaceutical Development & Commercialization, Inc., Alexandria, VA, USA, 3USA, 4CDA, New York, NY, USA, 5Columbia Data Analytics, New York, NY, USA, 6Columbia Data Analytics, Newtown, PA, USA
1Otsuka Pharmaceuticals Development & Commercialization, Inc., Associate Director, Newtown, PA, USA, 2Otsuka Pharmaceutical Development & Commercialization, Inc., Alexandria, VA, USA, 3USA, 4CDA, New York, NY, USA, 5Columbia Data Analytics, New York, NY, USA, 6Columbia Data Analytics, Newtown, PA, USA
OBJECTIVES: The burden of health care resource utilization (HCRU) in rare diseases can be substantial with complex treatment regimens and need for on-going care. Measuring HCRU during trials could provide critical data on the economic impact of therapies. We examined (HRCU) metrics reported from rare disease clinical trials across immunology, rheumatology, dermatology, and nephrology to identify current practices and potential opportunities.
METHODS: A systematic review of clinical trial registries, bibliographic databases, grey literature, and health technology assessment reports was conducted following PRISMA guidance. Eligible interventional or observational studies enrolled rare disease populations in the four therapeutic areas and reported at least one quantifiable HCRU metric; standardized extraction captured trial characteristics, HCRU endpoints, data sources, timing, and analytic methods, and findings were summarized descriptively by therapeutic area and metric type.
RESULTS: Twenty trials reported HCRU: Explicit HCRU for immunology=12, dermatology=4, rheumatology=3, nephrology=0; one chronic kidney disease trial (EMPA-KIDNEY) contributed contextual hospitalization outcomes. All 20 trials (100%) reported ≥1 direct HCRU metric: hospitalizations and length of stay in 15 trials (75%), emergency or acute visits in 12 (60%), outpatient visits in 9 (45%), and medication or treatment utilization in 11 (55%). Indirect HCRU was less frequent: 13 trials (65%) reported work or productivity loss, 3 (15%) caregiver burden, and 3 (15%) patient-level cost or socioeconomic burden, typically as secondary endpoints captured via electronic case report forms, self-reported questionnaires, or claims data, with substantial heterogeneity in definitions, measurement windows, and units and relatively few studies translating resource use into standardized cost outcomes.
CONCLUSIONS: HCRU metrics are rarely incorporated into rare disease trials in immunology, rheumatology, and dermatology. When incorporated, they are largely secondary, variably defined, and infrequently linked to cost. Prospective adoption of a core HCRU assessment with standardized definitions, data collection tools, and costing approaches could improve comparability and support payer-relevant evidence generation.
METHODS: A systematic review of clinical trial registries, bibliographic databases, grey literature, and health technology assessment reports was conducted following PRISMA guidance. Eligible interventional or observational studies enrolled rare disease populations in the four therapeutic areas and reported at least one quantifiable HCRU metric; standardized extraction captured trial characteristics, HCRU endpoints, data sources, timing, and analytic methods, and findings were summarized descriptively by therapeutic area and metric type.
RESULTS: Twenty trials reported HCRU: Explicit HCRU for immunology=12, dermatology=4, rheumatology=3, nephrology=0; one chronic kidney disease trial (EMPA-KIDNEY) contributed contextual hospitalization outcomes. All 20 trials (100%) reported ≥1 direct HCRU metric: hospitalizations and length of stay in 15 trials (75%), emergency or acute visits in 12 (60%), outpatient visits in 9 (45%), and medication or treatment utilization in 11 (55%). Indirect HCRU was less frequent: 13 trials (65%) reported work or productivity loss, 3 (15%) caregiver burden, and 3 (15%) patient-level cost or socioeconomic burden, typically as secondary endpoints captured via electronic case report forms, self-reported questionnaires, or claims data, with substantial heterogeneity in definitions, measurement windows, and units and relatively few studies translating resource use into standardized cost outcomes.
CONCLUSIONS: HCRU metrics are rarely incorporated into rare disease trials in immunology, rheumatology, and dermatology. When incorporated, they are largely secondary, variably defined, and infrequently linked to cost. Prospective adoption of a core HCRU assessment with standardized definitions, data collection tools, and costing approaches could improve comparability and support payer-relevant evidence generation.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
EE277
Topic
Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies, Work & Home Productivity - Indirect Costs
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Rare & Orphan Diseases