HHS issues highly critical report of FDA's risk evaluation and mitigation strategies (REMS) program

It’s going on five years since FDA initiated its REMS program, with some fanfare, in early 2008. The program, set up to enable drugs that had significant health risks to be approved and marketed—as long as ongoing assessments were made of safety measures imposed with the approval, came out of the FDA Amendments Act of 2007 (FDAAA). Prior to that Act, FDA had a voluntary program, part of which went under the name RiskMAPS, to accomplish more or less the same goals. But inconsistencies and uneven performance of RiskMAPS led to the legislative mandate. Now it’s looking as if the situation hasn’t changed very much.

In its report, FDA Lacks Comprehensive Data to Determine Whether Risk Evaluation and Mitigation Strategies Improve Drug Safety, The Office of Inspector General (OIG) of HHS looked at REMS programs through December 2011. Of 99 programs in place by the end of 2011, OIG reviewed 49, finding that 21 were not meeting the mandated goals; and 21 had uncertain or un-evaluated assessments. Only 7 of the 49 met all goals. Further, only one of the REMS with the added “elements to assure safe use” (ETASU)—a more comprehensive safety program—met all its goals; 18 others either did not or the assessments were undefined. Finally, FDA has an obligation to Congress under FDAAA to perform one assessment of REMS with ETASUY annually, but has only performed one since the program inception (and that one assessment was done just as OIG was closing its review period in December 2011).

REMS—especially REMS with ETASU—impose a burden on manufacturers and healthcare providers, and can also involve distributors and pharmacies. By mid-2011, healthcare providers were complaining to FDA about the administrative burden, and FDA revamped some of its procedures later that year. OIG says that it has heard of the difficulties that program administrators have in assessing, for example, whether patient or physician training has been adequately performed, or that data on how drugs are being prescribed is collected. (Of course, elements like these are exactly what the REMS is supposed to do.) Providers are supposed to report data to manufacturers, and manufacturers are supposed to collect and assess the data, and then forward to FDA. (Interestingly, FDAAA set substantial financial penalties—up to $1 million—for noncompliance, but FDA has not imposed any such penalty. FDA is also authorized to withdraw the approval of the drug if REMS is not being carried out, but that hasn’t happened either.)

OIG had several recommendations, most of which boil down to telling FDA to do what it’s supposed to, and FDA agreed. One recommendation—to seek additional legislative authority to enforce reporting all necessary data—is one that FDA notes was partially addressed by last year’s FDA Safety and Innovation Act. Scott Gottlieb, a fellow at the American Enterprise Institute (and former FDA assistant administrator) wrote in a Forbes blog that REMS and other FDA initiatives expand FDA’s regulatory scope into healthcare itself—physicians’ and pharmacists’ practices. “The agency wants to make sure that doctors conform to the FDA’s judgment about how new products should be used. But FDA’s control over medical practice is tenuous, and will remain so. Efforts to exert more leverage will only alienate the providers that FDA relies on,” he wrote. In the meantime, FDA will be holding more public workshops during this year, and beefing up its internal operations.