“This review of FDA actions concludes that two of every three orphan drugs approved show FDA’s historic flexibility in its review of effectiveness data on orphan drug therapies,” Frank Sasinowski, the NORD chairman.
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.
He adds that the FDA should memorialize its approach to provide greater certainty. “It would be helpful for such flexibility and importance to be recognized in a formal FDA policy, and for FDA officials to incorporate and recognize that flexibility in a systematic way in their evaluations of each new therapy in development and under FDA review for Americans with any rare disease,” he says.
Interestingly, orphan drugs already seem to have greater odds of winning FDA approval according to a 2010 study by the Tufts Center for the Study of Drug Development. Sponsors engaged in clinical development between 2000 and 2009 funded through orphan grants reported that 22 percent of their programs led to approvals, compared to a clinical approval success rate of 16 percent among other drug developers.
Further, orphan drugs have faster development time lines, lower R&D expenses, and a higher likelihood of clinical and regulatory success. Once on the market, they tend to have less competition, lower marketing costs and a longer life-cycle with less risk of generic erosion. Orphan drug products are also generally more innovative than non-orphan drugs as evidenced by a high number of new molecular entities.
The study seems to show their are plenty of reasons for drug companies to now pursue more orphan indications. In fact, several major players -- among them GSK, Pfizer, Merck and Novartis -- have announced orphan programs at least partially based on historic successes realized by comapnies like Genzyme and Shire. See Fierce Biotech.
Posted by Bruce Lehr Oct 12th 2011.