In the opening congressional hearing to reauthorize PDUFA, the discussion Wednesday ranged from the dangers of the Brazilian Blowout hair straightener to the Tobacco Control Act.

Despite the rabbit trails that are likely to continue as Congress considers the user fee agreement, Rep. Joseph Pitts (R-Pa.), chairman of the House Health Subcommittee, said his goal is to have PDUFA V approved by the end of June, well in advance of the Sept. 30, 2012, expiration of PDUFA IV.

Both Republican and Democrat lawmakers stressed the importance of reauthorization and urged bipartisanship, but Rep. Henry Waxman (D-Calif.) took a swipe at the Republican leadership of the committee, chiding it on the name of the hearing, "Reauthorization of PDUFA: What It Means for Jobs, Innovation, and Patients."

"The FDA should not take jobs into consideration," he said, adding that PDUFA should not be held hostage to a wish list of lawmakers wanting to use the reauthorization to advance their own drug-related legislation.

In the next breath, Waxman promoted the Drug Safety Enhancement Act, H.R. 1483, which he's co-sponsoring. The bill, which has been stuck in the subcommittee since it was introduced in April 2011, would expand the drug establishment registration requirements to overseas firms; give the FDA the authority to recall, detain, destroy or seize drugs; and require inspections at least once every two years for high-risk facilities and at least once every four years for those with less risk.

The bill also would require drugmakers to list, on the product website, country-of-origin labeling for each active pharmaceutical ingredient in the drug along with the place of manufacture of the finished product.

The lack of inspections of foreign manufacturing facilities was a major safety issue addressed at Wednesday's hearing. Several congressmen once again urged the FDA to adopt a risk-based approach to inspections.

FDA commissioner Margaret Hamburg said the agency is ramping up inspections through use of its foreign offices and partnerships with other regulators as it seeks parity between domestic and foreign inspections. However, she backed away from earlier testimony in which she cited U.S. union restrictions as a factor in the low number of foreign inspections. Under the union contract, U.S.-based inspectors have to volunteer for overseas duty.

The last time PDUFA came up for reauthorization, it was approved just days before its expiration due to congressional concerns that the FDA was favoring quick approvals over safety. (See BioWorld Today, Sept. 4, 2007, and Sept. 7, 2007.)

Drug safety is still a crucial concern, with Waxman insisting that it should be the only issue involved in PDUFA reauthorization. While other congressmen, from both sides of the aisle, agreed that safety is key, they stressed that the FDA's approval process must be straightforward and predictable to encourage the development of innovative treatments, especially for unmet health needs.

Varying Levels of Risk

In striking a balance between speedy approvals and degree of risk, Rep. Edolphus Towns told Hamburg the agency must consider that the tolerable levels of risk differ with patient groups. For example, patients with a life-threatening illness that has no approved treatment would accept higher risks than those with a chronic condition with approved treatments.

Hamburg agreed, saying PDUFA V sets up a formal way for the FDA to engage with the patient community. Meanwhile, she said, the agency wants to ensure that a drug offers real benefits, not just a minor improvement.

In his testimony, David Gollaher, president and CEO of the California Healthcare Institute, praised PDUFA V's "concentration on patient-focused drug development" and acknowledged the importance of building a better shared understanding of benefits and risks.

"Virtually all medicines bear some capacity for harm," he noted. "A zero-risk approach would shut down the development of beneficial drugs," which could be happening in some sectors, such as diabetes, as investment dries up because of FDA data requirements and review times.

In determining the risk-benefit profile of a drug, the FDA "focuses almost exclusively on the direct risks of drugs," Gollaher said. But what about the indirect risks to public health, he asked, "if investors avoid an important disease because the FDA's demands for data are so extensive and its standards for drug approval so uncertain?"

Testifying on behalf of the Biotechnology Industry Organization, Richard Pops, chairman and CEO of Alkermes plc, noted the average time between treatment discovery and availability to patients is 10 to 15 years. "That is much too long," he said.

"Unpredictability and inconsistency in the review process, suboptimal communication with sponsors and decreased FDA performance not only hinder patient access to new treatments, but also negatively affect the ability of biotechnology companies to raise funding to support clinical development and ongoing innovation," Pops added.

By increasing transparency and scientific dialogue, advancing regulatory science and strengthening postmarket surveillance, Pops said PDUFA V could enhance the drug development and review process and speed access to breakthrough technologies.

As Congress takes up PDUFA reauthorization, the biopharma industry is keeping its fingers crossed that the provisions it negotiated with the FDA don't get bogged down in policy squabbles. Several questions popped up during the subcommittee hearing that suggested Congress might try to interject its concerns on such areas as accelerated approval, conflicts of interest, drug pedigrees and orphan drug incentives. (See BioWorld Today, Oct. 26, 2011.)

The subcommittee will hold a hearing Feb. 7 on the new user fees for generic drugs and biosimilars.