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# FDA Issues for Orphan Drug Approvals
- URL: https://www.fdaweb.com/fda-issues-for-orphan-drug-approvals/
- Published: 2017-09-07T12:00:00.000Z
- Updated: 2026-09-14T22:47:31.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5139574

Three FDAers write in an online *Nature* [comment](http://www.nature.com/nrd/journal/vaop/ncurrent/full/nrd.2017.151.html?foxtrotcallback=true&ref=fdaweb.com) that since approval of the Orphan Drug Act in 1983, the agency has approved more than 600 orphan drug indications. The article discusses three key issues for maintaining that approval trajectory for rare disease therapies: 

- a science-based rationale for granting orphan drug designation;
- striking a balance between incentives for rare disease drug development and generic or biosimilar competition; and
- using the totality of the evidence when evaluating the efficacy and safety of medicines for rare diseases.

As advances in genomics and precision medicine continue to be made, the authors say, FDA will need to take such new developments into account as it considers what constitutes a “disease or condition” for the purposes of orphan designation and approval. “Fortunately,” they write, “the current statutory and regulatory framework provides FDA with the flexibility needed to make these science-driven decisions.”

The post also says that using real-world evidence takes account of small patient populations available for randomized clinical trials in rare diseases. And it also can be useful in studying therapies and outcomes, designing clinical trials, and developing care guidelines, it adds. “FDA looks forward to continuing to work with patients, patient advocacy groups, academia, and industry to incorporate prospectively planned and designed observational studies to aid in the understanding of the natural history of a rare disease or condition and in the development of a new therapy,” the article concludes.