Rare diseases: Although limited, available evidence suggests medical and other costs can be substantial : report to congressional committees
- Collection:
- Health Policy and Services Research
- Alternate Title(s):
- Although limited, available evidence suggests medical and other costs can be substantial
- Contributor(s):
- United States. Government Accountability Office, issuing body. United States. Congress, issuing body.
- Publication:
- Washington, DC : United States Government Accountability Office, October 2021
- Language(s):
- English
- Format:
- Text
- Subject(s):
- Government Regulation Rare Diseases -- economics United States
- Genre(s):
- Technical Report
- Abstract:
- Why GAO did this study. By definition, few people have any specific rare disease. But there are many different rare diseases—about 7,000—and as a result, an estimated 30 million people in the United States have one or more of them. About half of those with a rare disease are children. Often genetic, many rare diseases are chronic, progressive (they worsen over time), and life-threatening. Relatively little is known about the costs of rare diseases, either individually or collectively. The Joint Explanatory Statement for the Further Consolidated Appropriations Act, 2020, includes a provision for GAO to study the costs of rare diseases within the U.S. GAO examined, among other things, the challenges to diagnosing rare diseases and what is known about their costs. GAO reviewed documents from two agencies in HHS—the National Institutes of Health (NIH) and Food and Drug Administration (FDA) and published literature, including studies on the costs of rare diseases in the United States and elsewhere published from 2000 through 2021. GAO also interviewed NIH and FDA officials; selected researchers and experts on rare diseases, health care, and health economics; and officials of organizations representing those with rare diseases. The organizations included two devoted to rare diseases in general and six representing those with a specific rare diseases or sets of related rare diseases.
- Copyright:
- The National Library of Medicine believes this item to be in the public domain. (More information)
- Extent:
- 1 online resource (1 PDF file (ii, 103 pages))
- Illustrations:
- Illustrations
- NLM Unique ID:
- 9918383083506676 (See catalog record)
- Permanent Link:
- http://resource.nlm.nih.gov/9918383083506676