Print“In order to prevent similar delays in the future, PDUFA V must pass Congress cleanly, without the addition of unnecessary legislative mandates.”
A Democratic senator is working with the biotech industry to draft legislation that would expand the number of rare disease drugs granted accelerated approval by the U.S. Food and Drug Administration.
The bill, which has yet to be introduced by its author, Senator Kay Hagan (D-North Carolina), would allow more drugmakers to seek early approvals, according to a report by Bloomberg. Hagan was selected by the Biotechnology Industry Organization as Legislator of the Year for 2009-2010. Neither Hagan’s office nor BIO would comment on the bill at this time.
Given the difficult financing environment and the amount of time and resources it takes to develop new drugs, many biotechs view developing drugs for niche rare disease markets too risky. Allowing faster market access and a quicker path to revenue could make the development of needed drugs for rare diseases more attractive.
That has made expanding and improving the accelerated approval pathway a key part of the BIO’s ambitious five-year policy agenda. BIO’s plan, unveiled at its international convention in June, set the goal of establishing a progressive approval mechanism, one which would allow patients with unmet medical needs earlier access to promising experimental therapies.
The FDA already has a pathway for accelerating the approval of such drugs, called Fast Track. More than a third of drugs approved in 2011 were given a Fast Track designation. But accelerated approval can provide faster access to markets because it allows the agency to approve a drug based on clinical data showing that the drug has an effect on a surrogate endpoint likely to predict a clinical benefit—such as evidence that a drug shrinks cancer tumors—but without requiring that it demonstrate the clinical benefit itself.
The FDA has approved 11 drugs for rare diseases in 2011, including the most recent, Incyte’s Jakafi for the treatment of patients with the bone marrow disease myelofibrosis.
Hagan’s bill would be freestanding, not an amendment or add-on to the Prescription Drug User Fee Act, or PDUFA, which establishes industry fees and agency commitments during the drug review process. The industry, which has already reached an agreement with the FDA on the legislation, is reticent to open up the discussion on additions to the bill for fear of delays and the opportunity critics of the industry might see in amending it.
“During the last PDUFA reauthorization (PDUFA IV, in 2007), the FDA was burdened with additional regulations that seriously hindered its ability to meet review goals, almost doubling the review times for priority drugs,” noted The Campaign for Modern Medicines, an industry advocacy group sponsored by Eli Lilly, on November 10. “In order to prevent similar delays in the future, PDUFA V must pass Congress cleanly, without the addition of unnecessary legislative mandates.”
November 17, 2011
http://www.burrillreport.com/article-the_need_for_speed.html
.gif)
