WASHINGTON – The reauthorization of PDUFA could lead to a change in mission for the FDA that would require the agency to couple economic growth and job creation with its public health duties.

The House discussion draft for PDUFA V expands the agency's mission statement to reflect the realities of the 21st century, acknowledging a dual role of protecting the public health and enabling patient access to novel products while "promoting economic growth, innovation, competitiveness and job creation among the industries" it regulates.

The proposal drew fire at a House subcommittee hearing Wednesday on the user fee reauthorization. "Those changes concern me very much," Rep. Jan Schakowsky (D-Ill.) said. She feared that adding economic factors to the agency's mission would take away from its public health focus.

FDA officials testifying at the hearing agreed. The promotion of economic growth and job creation "are things scientists shouldn't be dealing with," Jeffrey Shuren, director of the FDA's device center, told Schakowsky.

"Whose jobs are we talking about?" he asked. The sponsor of a product? Its competitors? Foreign companies?

Noting that a few lawsuits have already been filed against the FDA on the basis of its current mission statement, which is aimed solely at protecting and promoting public health, Shuren said including economic factors in the statement could lead to more lawsuits.

Janet Woodcock, director of the FDA's drug center, testified that the FDA doesn't have the economic expertise that mission expansion would require. "I see this could have negative consequences," she said.

For instance, it could lead to challenges of the agency's approval decisions that go beyond safety and efficacy issues, she said, adding that the FDA's mission should focus on the impact to patients – not jobs for the industry.

While industry witnesses testifying at the hearing didn't dwell on the mission statement, they recognized the dual role the agency has. In her written testimony, Sara Radcliffe, executive vice president for health at the Biotechnology Industry Organization, said, "The agency has a critical role in facilitating health care innovation, but this fact is not formally and forcefully recognized in FDA's legislative mandate."

The draft also expands the mission statement to emphasize the need for transparency, public participation, regulatory predictability and flexibility, and measures of the actual results of the FDA's regulatory requirements.

While the primary purpose of PDUFA is to enact the user fee/performance goal agreements negotiated between industry and the FDA, Congress typically uses the five-year reauthorization to address other issues at the agency.

Since those other issues can delay a new bill, David Wheadon, senior vice president for scientific and regulatory affairs at the Pharmaceutical Research and Manufacturers of America, once again urged Congress to pass a PDUFA package based on the original negotiated provisions with minimal add-ons. (See BioWorld Today, April 2, 2012.)

The House draft has a number of add-ons, some of which are similar to provisions in the Senate discussion draft bill that's heading to markup next week. (See BioWorld Today, April 6, 2012.)

While some of the details differ, both the House and Senate drafts would require early notification of events that could lead to a drug shortage, provide incentives for the development of new antibiotics and enhance the accelerated approval path. The Senate version also includes provisions to ensure the safety of the drug supply chain. The House draft the Energy and Commerce Health Subcommittee released Tuesday has yet to flesh out a proposal for securing the supply chain.

Removing BPCA, PREA Sunset

Other provisions are unique to the House draft. It adds permanent reauthorization of the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) to the user fee package. Like the user fees, BPCA and PREA have been on five-year reauthorization cycles. And each time they come up for renewal, the requirements for pediatric drug development change. (See BioWorld Today, Feb. 3, 2012.)

As a result, the lack of permanency for BPCA and PREA has hindered the development of pediatric drugs. Since the average pediatric clinical trial spans six years, the five-year sunsets on the programs lead to changes in the ground rules part way through a trial, Radcliffe told the subcommittee Wednesday.

Besides making it difficult for industry to invest in pediatric infrastructure, the five-year changes in statutes and regulations make it practically impossible for the FDA to issue guidance about the pediatric regulatory framework, Radcliffe said.

Wheadon noted that no current guidance exists for pediatric drug development. The lack of that guidance is creating additional challenges in developing needed therapies for pediatric use, he said.

Controversy in the Details

While making BPCA and PREA permanent seems to have broad support, some of the other provisions being discussed in the PDUFA context may be more controversial, if Wednesday's hearing is any indication.

Rep. Henry Waxman (D-Calif.) questioned the incentives for new antibiotics and the language defining which antibiotics would qualify. Like the Senate bill, the House draft would grant qualifying antibiotics five years of market exclusivity, plus an additional six months if they have companion diagnostics.

Waxman pushed for a narrower definition of qualifying drugs, stressing the need to target serious or life-threatening infections. Instead of exclusivity, he urged consideration of an expedited limited population antibacterial drug approval path based on smaller, more rapid clinical trials.

Woodcock agreed that such a narrow development program would be a stronger incentive than exclusivity.

Securing the drug supply chain also may generate some controversy – if it involves a track-and-trace system at the unit level. An industry coalition has proposed RxTEC, a lot-level system, but Woodcock testified that while RxTEC could be used after the fact, it would do nothing to prevent or identify fake drugs entering the U.S. market. (See BioWorld Today, March 12, 2012.)