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PDUFA Heads to Markup with a Warning to Leave it Alone

By Mari Serebrov
Washington Editor

WASHINGTON – With more riding on the reauthorization of PDUFA than ever before, a Senate committee is pushing through a package of user fees with a warning against bogging it down with costly and controversial measures.

Patients, jobs and innovation will be on the line if Congress doesn't reauthorize PDUFA before its Sept. 30 expiration date, the Senate Health, Education, Labor and Pensions (HELP) Committee was reminded last week in its final hearing before it begins marking up the package that would reauthorize brand drug and medical device fees and establish new fees for generic drugs and biosimilars.

In opening the hearing, HELP Chairman Tom Harkin (D-Iowa) noted that PDUFA currently funds 60 percent of the FDA's Center for Drug Evaluation and Research (CDER), as well as nearly a third of the device center. If the fee package is not reauthorized on time, the FDA would have to fire 2,000 employees, which would impact the drug and device review process.

"We cannot let that happen," Harkin said, as he urged his colleagues not to yield to outside forces that view the five-year reauthorization as an opportunity to advance their own agendas.

CDER Director Janet Woodcock echoed his urgency. The biopharma industry is in crisis, she said, as it faces a global economic crunch at a time when increased R&D investments are producing fewer new drugs.

However, given the number of new, innovative drugs approved last year, Woodcock said industry may be at a turning point. Failure to reauthorize PDUFA on time could threaten that turnaround, as well as the nation's status as the leader in new drug development. (See BioWorld Today, Nov. 7, 2011.)

Another concern is that Congress would use the fee reauthorization to add to the FDA's workload. Jeffrey Shuren, director of the FDA's device center, reminded the committee that the last time PDUFA was reauthorized, Congress loaded it with new responsibilities for CDER. In trying to implement those requirements, CDER fell behind in its approval of new drugs. (See BioWorld Today, Oct. 26, 2011.)

Besides setting user fees and FDA review performance goals, PDUFA V would provide more support for orphan drug development by expanding agency staff tasked with helping small companies through the regulatory process for orphan drugs.

It also would further open the door for patient involvement in drug development and approval. "Most drugs are not really safe," Woodcock said. "They have liability. They have risks." The FDA needs to know what risks patients are willing to accept.

Toward that end, the agency is bringing patients into the process in 20 different disease areas, including obesity. As part of PDUFA, the FDA hopes to incorporate patient-centered outcomes into future clinical trials.

Despite the pleas for a clean fee bill, Congress is expected to attach other FDA-related bills to the package. During the hearing, Sen. Richard Blumenthal (D-Conn.) was critical of the FDA's handling of a growing drug shortage crisis. He said this problem must be addressed as part of the user fee package. (See BioWorld Today, Sept. 26, 2011, Sept. 27, 2011, Sept. 28, 2011, Nov. 1, 2011, Dec. 16, 2011, and Jan. 20, 2012.)

Other measures that are likely to be added to the fee reauthorization include ones to spur the development of new antibiotics and to improve drug supply chain safety.

In concluding the hearing, Harkin called on industry organizations to commit to ensuring the "must-do" user fee legislation is kept free of controversial provisions that would slow or stall its passage.

In response, Wheadon, senior vice president of scientific and regulatory affairs at the Pharmaceutical Research and Manufacturers of America, said, "Our original goal was a skinny PDUFA. We moved to a lean PDUFA. We now recognize that we have a PDUFA that probably needs to go to Weight Watchers."

A date has yet to be set for the HELP Committee to mark up the legislation. The Senate will not return from a two-week break until April 16.

Breakthrough Drugs Need a Break

Legislation requiring the FDA to mentor sponsors developing breakthrough therapies was introduced in the Senate last week.

S. 2236 would add a new category, similar to fast-track designation, that would speed the development of drugs to treat serious or life-threatening diseases and conditions. To get the breakthrough product designation, a drug would need preliminary clinical evidence indicating that it may offer substantial improvement over existing therapies.

Jeff Allen, executive director of Friends of Cancer Research, suggested the idea last month in a House subcommittee hearing. Under the proposed designation, a sponsor would work closely with the FDA to develop trial designs to abbreviate or combine the traditional phases of drug development. (See BioWorld Today, March 9, 2012.)

"This would shorten the pathway to approval and avoid giving larger numbers of patients a potentially harmful or ineffective drug as part of a control arm, while maintaining current safety and efficacy standards," Allen testified.

The bill also would require the FDA to engage in interactive communication and hold timely meetings with the sponsor throughout the development of a breakthrough product. The agency would have to assign a cross-disciplinary project lead to facilitate efficient review of the product, as well as involve senior management and experienced review staff in the collaborative, cross-disciplinary review.

Bill Makes BPCA, PREA Permanent

The Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) would become permanent under a bill introduced last week in the House.

Instead of simply reauthorizing the programs that have made more drugs available for pediatric use, H.R. 4274 would "encourage continued investment in pediatric research," said Jim Greenwood, president and CEO of the Biotechnology Industry Organization. (See BioWorld Today, Feb. 3, 2012.)

The bipartisan bill also would improve the timing and quality of pediatric research and ensure that required pediatric studies are completed by their due date.