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# Orphan Drugs Slower to Develop, Launch: Study
- URL: https://www.fdaweb.com/orphan-drugs-slower-to-develop-launch-study/
- Published: 2018-05-09T12:00:00.000Z
- Updated: 2026-09-15T00:04:07.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5141391

Orphan drugs take on average 2.3 years longer to go from patent to launch than other drugs, according to an analysis by the Tufts Center for the Study of Drug Development reported in the May/June issue of its [*Impact Report*](https://csdd.tufts.edu/csddnews/?ref=fdaweb.com)(subscription or purchase required). The center studied all 46 first-in-class, orphan-designated, new molecular entities approved by FDA from 1999 through 2012\. Average development time for the 46 drugs was 15.1 years, some 18% longer than the 12.8 years for all new drugs, Tufts says.

Some 17% of orphan drug designations resulted in FDA approvals during 1986-2016\. In 2016, FDA granted 333 (57%) of 582 orphan drug designation requests it received, 10 times the number of designations granted in 1986 shortly after the orphan drug program started. However, the number of approvals as a share of designations declined during the last few decades to 12% in 2016, slightly lower than the 17% share for the entire 1986-2016 period.

The analysis found that designations and approvals for orphan oncology drugs outpaced all other therapeutic areas. It also determined that orphan drug developers encountered an average of 4.3 challenges during the period 1999-2012, including variability in expression, severity, and/or course; geographically dispersed population; small population; selecting among multiple pathways; and lack of endpoints, outcome measures, etc.

Therapeutic areas with the most challenges experienced fewer orphan drug successes, the report says. Developers of orphan drugs focused on central nervous system or cardiovascular diseases experienced the greatest number of development challenges and the lowest number of approvals as a share of designations.

While average development time for orphan drugs in the study period was 15.1 years, the development time for drugs to treat ultra-orphan diseases that affect only up to a few hundred patients in the U.S. was 17.2 years or 34% longer than the development time for all new drugs.