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Comment & Response
August 6, 2019

Incentivizing Therapies for Rare Diseases

Author Affiliations
  • 1Department of Population Medicine, Harvard Pilgrim Health Care Institute, Boston, Massachusetts
JAMA. 2019;322(5):464-465. doi:10.1001/jama.2019.6954

To the Editor I offer a few points of disagreement in response to the Viewpoint on the Orphan Drug Act (ODA).1

The authors incorrectly stated that the ODA extends patent protections. In fact, it does not alter patent life; rather, it grants a period of postapproval market exclusivity distinct from patent protection.

A distinction should be drawn between 2 categories of orphan drugs: those that treat rare or ultra-rare conditions and those that are approved for narrow subsets of disease (often advanced cancer with the presence of a genetic mutation and/or after failure of prior therapy). The latter is problematic while the former is appropriately incentivized by the ODA. Existing literature has established the unique problems posed by orphan anticancer drugs,2,3 to which the authors alluded. Rather than a new definition of orphan drugs, the onus should be on the US Food and Drug Administration to exercise greater discretion in its approval of orphan drugs for narrowly defined disease subsets.