GAO finds flaws in FDA's orphan drug program: 6 things to know

The FDA failed to consistently record and evaluate necessary information to ensure drugs given the prized rare-disease designation met the legal standards required by the Orphan Drug Act, according to a new report from the Government Accountability Office.

Here are six things to know:

1. The Orphan Drug Act was implemented 35 years ago to motivate pharmaceutical companies to develop drugs for rare diseases that affect fewer than 200,000 people. Rare diseases were often overlooked by big pharma because they were not expected to be profitable. The law provides waivers from FDA fees, tax incentives for research and seven years of patent protection for an orphan drug designation.   

2. The number of drugmakers seeking orphan drug status grew 85 percent, from 185 in 2008 to 527 in 2017.

3. A federal  probe began after a Kaiser Health News investigation last year found the program was being manipulated by drugmakers to "maximize profits and protect niche markets for medicines."

4. For its yearlong investigation, the GAO analyzed 148 drug applications that were sent to the FDA for orphan drug status last year.  

5. The report found that the FDA granted the designation to 26 applications that were missing required information, and 102 of the 148 reviewed applications were missing pertinent background information. The report also found 80 drugs were given the orphan drug status for more than one disease. 

6. The GAO recommended the FDA review procedures for assessing orphan designations to ensure all required information is consistently recorded and evaluated.

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