Impact of FDA’s flexibility on orphan drugs

In Uncategorized by Pinky Fadullon

UnicitàNORD released a report last week that two of every three orphan drugs approved show FDA‘s historic flexibility in its review of effectiveness data on orphan drug therapies," Sasinowski said. 

What is the impact of this?

Sasinowski continued to say that "Therefore, FDA has demonstrated in its actions on orphan products that it recognizes the importance of therapies for persons with rare disorders.  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."

Saltonstall, President & CEO of NORD added that they undertook this study because, of the nearly 7,000 known rare diseases, only about 200 have FDA-approved treatments.  "Better understanding the regulatory process and the pathways most likely to lead to increased study of rare diseases and safe, effective treatments for patients is very important to us," he said.

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This seems to be FDA commitment right here and right now. Where do we go from here? What do other stakeholders say to this though? Is there enough ease for orphan drug manufacturers and developers? What other challenges and stakeholder commitments can there be had? What do patients say?

For continuing discussions from global regulators, industry, patients and payers check out the 2nd Annual World Orphan Drug Congress USA in April 2012.