Drug companies rely on the Food and Drug Administration to review and approve their drugs for sale in the U.S. market. But for the past 20 years, the FDA has also been reliant on drugmakers — using fees paid by industry to bolster its staff, modernize its review process and get treatments to patients more quickly.
The fee system was set up under a program called the Prescription Drug User Fee Act, first implemented in 1992. A Medical Device User Fee Act was created 10 years later. Each act requires congressional reauthorization every five years, and both — PDUFA and MDUFA, respectively — are set to expire Sept. 30 if Congress doesn't take action. Both houses of Congress have approved measures reauthorizing the programs, but differences in those measures must now be reconciled.
Dean J. Paranicas, president and CEO of the HealthCare Institute of New Jersey, said the programs have become essential to help the FDA deal with the increasingly rapid development of new drug and medical device technologies.
"As that technological pace has accelerated, the demands in order to review and approve the products have continued to increase," he said.
According to the FDA, PDUFA funding has allowed the agency to double its full-time employees involved in the review process, but Paranicas said the funding also has helped train federal regulators to keep them up to date with the newest technologies.
Thus far, the reauthorizations of both PDUFA and MDUFA have moved forward with little friction. That's in part because the FDA has been meeting with industry representatives for nearly two years to discuss the reauthorizations and possible improvements going forward.
Among the changes proposed in the PDUFA reauthorization is a plan to retool the process by which the FDA evaluates the new drug compounds aimed at addressing areas of unmet medical need. Historically, about 80 percent of such applications are approved, according to Debbie Hart, president of trade group BioNJ, but fewer than half were approved based on the initial submission.
"That denotes it was just a lack of communication, a lack of transparency and a lack of a logical pathway for communication," Hart said.
The reforms in the PDUFA reauthorization call for increased meetings between the FDA and the applicants both prior to and during the approval process, in order to proactively handle concerns or questions and keep the approval timeline on target.
The PDUFA reauthorization also calls for the permanent reauthorization of two programs aimed at encouraging drug companies to do pediatric clinical trials, as they are generally more difficult and costly than adult trials. One program adds six months to drugmakers' exclusivity periods if they do pediatric trials. Another empowers the FDA to require drugmakers to do pediatric studies for drugs approved for adults, but thought to be appropriate for children. Currently, those programs require five-year reauthorizations.
"Because pediatric trials can take years, we think that permanent reauthorization … will help affirm for companies that the incentive will still be intact by the end of the trial," said Kate Connors, a spokeswoman for the Pharmaceutical Research and Manufacturers of America, the drug industry's main lobby in Washington.
This PDUFA bill also includes provisions aimed at enhancing drug safety and increasing public input in the agency's benefit-risk analysis process. The medical device fee reauthorization also calls for better communication and faster review periods, and would require an independent study of the agency's review process to help facilitate those goals.
Throughout the process, PhRMA has stressed the need to pass a "clean" bill that isn't bogged down by amendments.
Still, David Finegold, senior vice president for lifelong learning and strategic growth at Rutgers University, said a "clean" bill also means a sterile one: PDUFA was a significant innovation 20 years ago, but he said its reauthorization is more about keeping the status quo.
"PDUFA had its one-time bump, but now it's been built into the overall model," Finegold said.
In the same way PDUFA was a breakthrough 20 years ago, Finegold said technological advances have created the need for a broader reimagining of the FDA — one that engages universities and other stakeholders and takes advantage of efficiencies afforded by new technologies.
"That goes much beyond PDUFA," he said.
Still, with Congress' summer recess not far off, a Supreme Court decision on President Barack Obama's health care reform legislation scheduled for June and an election set for the fall, industry representatives are hoping reauthorization of the reconciled bills passes as soon as possible to avoid last-minute snags.
In a statement applauding the Senate's passage of a PDUFA reauthorization bill May 24, Biotechnology Industry Organization President and CEO Jim Greenwood stressed the importance of PDUFA funding to the FDA, and urged Congress not to drag its heels as it reconciles differences between the House and Senate versions.
"Even the threat of a downsizing would be devastating to the agency's public health mission and its ability to review new drugs and biologics," he said.
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