Investigation Details FDA’s Flawed Oversight Efforts For Orphan Drug Program That’s Particularly Vulnerable To Manipulation
The program is part of congressional efforts to motivate pharmaceutical companies to develop drugs for people who lack treatments for their rarer conditions, and it can be very lucrative for the companies. But the GAO uncovered inconsistent and often incomplete reviews by FDA employees early in the process of designating medicines as an orphan drug.
FDA Fails To Assess Important Needed Info For Orphan Status, Report Finds
As more drug makers race to win orphan designations, a new report finds the Food and Drug Administration fails to consistently record and evaluate needed information, which suggests the agency may not always include critical data for granting the coveted orphan status. Specifically, the FDA granted the designation to 26 applications that were missing required information, such as verified estimated patient populations, and 102 of 148 so-called review templates were missing background information. Moreover, FDA guidance was not always clear in instructing agency staff on how to use the recorded information, according to the U.S. Government Accountability Office. (Silverman, 11/30)
Kaiser Health News:
Government Investigation Finds Flaws In The FDA’s Orphan Drug Program
The Government Accountability Office, which spent more than a year investigating the FDA’s orphan drug program, said “challenges continue” in the program that was created to spur development of drugs for diseases afflicting fewer than 200,000 patients. The investigation began after a request from three high-profile Republican senators last year, in the wake of a KHN investigation. KHN found that the program was being manipulated by drugmakers to maximize profits and to protect niche markets for medicines being taken by millions. (Tribble and Lupkin, 11/30)