Over the last few years there’s been a big increase in Big Pharma’s interest in rare diseases. With Genzyme’s success with drugs like Myozyme helping light the way, giants like GlaxoSmithKline, Pfizer, Merck and Novartis have been organizing their own rare disease drug shops. And the FDA’s more flexible attitude in how it judges the data from clinical trials for these drugs – as well as the 7 years of marketing exclusivity they earn along with some hefty tax credits – hasn’t hurt.
In a new study, the National Organization for Rare Diseases decided to put the FDA’s professed flexibility to the test, examining 135 non-cancer orphan drugs which had been approved by the agency. And the FDA comes out with a clap on the back, earning NORD’s seal of approval for being willing to demonstrate its flexibility in 90 of those rare drug programs. Writes NORD: “The study supports the FDA assertion that it exercises flexibility when reviewing applications for orphan drugs.”
The study also notes that the FDA can demonstrate its flexibility in a number of ways, most notably by regulators’ willingness to accept a far more limited set of clinical trial data for an orphan drug, including instances where a single trial was enough to warrant an approval. In 58 cases regulators were willing to customize the approval process to the therapy.
The Wall Street Journal notes that not everyone is happy with the FDA’s approach to these orphan drugs. Public Citizen’s Sidney Wolfe, an outspoken critic of the pharma industry, had this to say: “There are all kinds of other benefits already written into law. Why should the standard of approval be different if you have a rare disease than for non-rare diseases?”
NORD does want the FDA to go a step further in spelling out its approach to orphan indications.
“It is time for that policy to be clearly enunciated as a formal FDA policy, and for FDA medical reviewers to incorporate and recognize this flexibility in a systematic way into their evaluations of each new therapy in development and under FDA review for Americans with any rare disease.”