With REMS, what goes up can come down

Abstract

Recent actions by FDA indicate the agency, when shown evidence that a suspected risk does not really exist, will alter a risk evaluation and mitigation strategy (REMS). In the past half year, the agency has lifted a requirement for monthly liver-function tests for one drug and, for another drug, eliminated the REMS program altogether. “I’m not surprised,” said Timothy Lesar, pharmacy director at Albany Medical Center in New York. Having participated in and witnessed numerous meetings of FDA’s advisory committees, Lesar said discussants regularly express concern about “rare, serious” adverse events that occurred in clinical trials of drugs for rare diseases. “People are going to wonder: [The company] only studied 200 patients . . . and 2 of them had anaphylactic reactions,” the former member of FDA’s Drug Safety and Risk Management Advisory Committee said. “But, you’re not sure if that’s a drug effect. “You just sort of . . . say, ‘Well, gee, you could collect data on this.’