Are We Capturing the Socioeconomic Burden of Rare Genetic Disease? A Scoping Review of Economic Evaluations and Cost-of-Illness Studies

Background and Objectives Rare diseases have a significant impact on patients, families, the health system, and society. Measuring the socioeconomic burden is crucial to valuing interventions for rare diseases. Healthcare system costs are significant, but so are costs to other government sectors, pa...

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Bibliographic Details
Published in:	PharmacoEconomics Vol. 41; no. 12; pp. 1563 - 1588
Main Authors:	Marshall, Deborah A., Gerber, Brittany, Lorenzetti, Diane L., MacDonald, Karen V., Bohach, Riley Jewel, Currie, Gillian R.
Format:	Journal Article
Language:	English
Published:	Cham Springer International Publishing 01-12-2023 Springer Nature B.V
Subjects:	Chronic illnesses Costs Drugs Families & family life Genetic disorders Health Administration Health care expenditures Health care policy Health Economics Medical equipment Medical technology Medicine Medicine & Public Health Pharmacoeconomics and Health Outcomes Productivity Public Health Quality of Life Research Rare diseases Resource allocation Scoping Review Society Socioeconomic factors Canada United States > US
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Summary:	Background and Objectives Rare diseases have a significant impact on patients, families, the health system, and society. Measuring the socioeconomic burden is crucial to valuing interventions for rare diseases. Healthcare system costs are significant, but so are costs to other government sectors, patients, families, and society. To understand the breadth of costs captured in rare disease studies, we examined the cost categories and elements of socioeconomic burden captured in published studies. Methods A scoping review was conducted using five electronic databases to identify English language economic evaluations and cost-of-illness studies of interventions for rare diseases (2011–21). We mapped costs using a previously developed evidence-informed framework of socioeconomic burden costs for rare disease. Results Of 4890 studies identified, 48 economic evaluations and 22 cost-of-illness studies were included. While 18/22 cost-of-illness studies utilized a societal perspective, only 7/48 economic evaluations incorporated societal costs. Most reported cost categories related to medical costs, with medication and hospitalizations being the most common elements for both study designs. Costs borne by patients, families, and society were reported less among economic evaluations than cost-of-illness studies. These included: productivity (10% vs 77%), travel/accommodation (6% vs 68%), government benefits (4% vs 18%), and family impacts (0% vs 50%). Conclusions Contrary to cost-of-illness analyses, most of the included economic evaluations did not account for the hidden burden of rare diseases, that is, costs borne by patients, families, and societies. Including these types of costs in future studies would provide a more comprehensive picture of the burden of disease, providing empirical data to inform how we value and make decisions regarding rare disease interventions, health policy, and resource allocation.
Bibliography:	ObjectType-Article-2 SourceType-Scholarly Journals-1 ObjectType-Feature-3 content type line 23 ObjectType-Review-1
ISSN:	1170-7690 1179-2027
DOI:	10.1007/s40273-023-01308-0