WASHINGTON -- As expiration of the Prescription Drug and User Fee Act (PDUFA) nears, the Senate is preparing to focus on legislation that would reauthorize the act and enact reforms at FDA.
The legislation, released last week and introduced into the Senate this week by Sen. Jim Jeffords (R-Vt.), chair of the Senate Labor and Human Resources Committee, fulfills Jeffords' April promise to use the expiration of PDUFA to spur FDA modernization and to submit a bill by the end of May. In addition, the bill fails to include some of the more contentious issues that came up last year.
"I am very pleased about this bill," said Carl Feldbaum, president of the Biotechnology Industry Organization (BIO). "The substance is solid, and it incorporates everything that we discussed with the committee."
First and foremost, Jeffords' bill includes the language that FDA, BIO and the Pharmaceutical Research and Manufacturers of America (PhRMA) agreed upon for the reauthorization of PDUFA. Those provisions establish a five-year reauthorization of PDUFA, written protocol agreements and a 10-month review goal.
"We strongly support this bill," said Anne Oman, assistant vice president of communications at PhRMA. "We consider the five-year renewal of PDUFA to be the most important provision."
Feldbaum also noted that the bill codifies the biologics rewrite that was established last year. Under that administrative action, the agency decided to regulate biologics based upon their potential threat to public health. This rewrite also streamlines reporting of manufacturing changes that previously required FDA approval of even the slightest change in manufacturing a biologic.
"We have been actively supporting the codification of this rewrite," Feldbaum said. "It had a good reason when it was put in place -- we didn't have much experience with biologics -- but that reason is gone."
In addition, Jeffords' bill clearly states that the agency may use "one or more clinical trials" as the basis of approval. Under current law, the agency may approve a drug should it prove safe and effective in well-controlled clinical trials. Some reviewers have interpreted that language to mean that a sponsor must conduct at least two clinical trials.
The legislation also clearly states that the agency is permitted to contract with third parties: to conduct clinical reviews and clinical investigations into new uses for pharmaceuticals; for surveillance of rare and unforeseen side effects; and to conduct research on the comparative effectiveness. All outside consultation still will be at the discretion of the agency.
In addition to provisions discussed with industry, Feldbaum noted that Jeffords had "put his own mark on the bill." For one, missing from the legislation are the hammers and provisions for dissemination of off-label uses of drugs. Jeffords' bill also includes a mission statement that declares the agency's mission to protect and promote the public health.
"It may just be symbolic," Feldbaum told BioWorld Today, "but he highlights the fact that the agency has two goals. By giving the promotion of the public health more visibility, it may play a role in changing the culture that emphasizes safety above all else."
The bill will enter mark-up on June 11. Jeffords originally had intended for the Labor and Human Resources committee to mark up the bill today; however, he postponed it because members of the Committee requested more time to review the bill, which Jeffords released during the Memorial Day recess.
Once the bill makes it through the committee, it will need to be approved on the Senate floor before the House can take action on it. The Health and Environment Subcommittee of the House is expected to introduce its own version of FDA modernization in the next couple of weeks. Nevertheless, both chambers of Congress will have to approve a conference bill before the president can sign the bill into law. With the pending expiration of PDUFA forcing the issue, legislators will have to continue to press forward at a generous clip in order to have a bill signed into law before the end of September.
"Almost more than anything else, I am pleased about the timing of this bill," Feldbaum said. "Jeffords said that he would have the bill out by the end of May and he did. It shows they are serious about moving it through the Senate."
"It would be a great loss to see PDUFA expire -- a real step backward," Feldbaum added.
When Jeffords stated his intention to combine the PDUFA reauthorization with the broader FDA reforms, some in industry, patient organizations and the agency itself expressed concern that piling other reforms onto PDUFA could jeopardize its reauthorization. Feldbaum noted that the add-ons to PDUFA don't appear to be "game-breakers;" however, the fiscal year 1998 budget could very well be the Achilles heel of this legislation.
The Clinton administration has proposed a budget that relies on a slew of new user fees that have little likelihood of being authorized in order to supplement an 8 percent cut in appropriations for the agency. Industry estimates by both BIO and PhRMA indicate that the agency will need approximately $68 million more than the administration proposed to meet the PDUFA requirement that the agency receive level funding.
"The administration's budget is illusory," Feldbaum said. "And everybody -- the FDA, industry and patients -- is concerned that it could jeopardize PDUFA."
However, Feldbaum noted that finding that $68 million is going to be difficult because the agency will have to compete for money with well-established agricultural programs and the Women's, Infants' and Children's aid program. "The tricky part is that jurisdictionally, the FDA appropriations come from the same subcommittee that appropriates moneys to agriculture -- it's a tough area," he said.
Nevertheless, industry will meet with the Office of Management and Budget (OMB) in order "to reduce any resistance at OMB to congressional action that would restore funds to the agency," Feldbaum said.
"Jeffords' bill displays a moderateness and reasonableness and it should have a high chance of passing," Feldbaum said. "And we intend to do everything we can to keep it on the fast track." *