The Orphan Drug Act at 40: Legislative Triumph and the Challenges of Success

Policy Points The Orphan Drug Act (ODA) was the result of patient advocacy and by many measures has been strikingly successful. However, approximately 95% of the more than 7,000 known rare diseases still have no US Food and Drug Administration–approved treatment. The ODA's success led to sustai...

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Bibliographic Details
Published in:The Milbank quarterly 2024-03, Vol.102 (1), p.83-96
Main Authors: SALTONSTALL, PETER, ROSS, HEIDI, KIM, PAUL T.
Format: Article
Language:English
Subjects:
Advocacy
Clinical research
Criticism
Disease
Drug abuse
Drug Approval
Drug prices
FDA
Humans
Morality
orphan drug
Orphan Drug Production
Patients
Prescription drugs
Rare diseases
Rare Diseases - drug therapy
United States
United States Food and Drug Administration
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Summary:Policy Points The Orphan Drug Act (ODA) was the result of patient advocacy and by many measures has been strikingly successful. However, approximately 95% of the more than 7,000 known rare diseases still have no US Food and Drug Administration–approved treatment. The ODA's success led to sustained criticism of high drug prices, often for products that have orphan drug indications. Critics misconstrue the ODA's intent and propose reducing its incentives instead of pursuing policies focused on addressing broader prescription drug price challenges that exist in both the orphan and nonorphan drug market. Patients and their families will continue to defend the purpose and integrity of the ODA and to drive investments into rare disease research and clinical development.
ISSN:0887-378X
1468-0009
DOI:10.1111/1468-0009.12680