Washington Editor

NEW YORK - In the background of Tuesday's full slate of investor presentations and one-on-one business meetings among the 3,000 attendees at the BIO CEO and Investor Conference, there's plenty of talk about the Prescription Drug User Fee Act (PDUFA). And while advocates at BIO acknowledge that some in Congress would like to tack on any number of attachments to the bill, they're pushing for the renewal process to move forward independently, given its time-sensitive nature.

"We are urging everybody not to add extra pieces onto PDUFA, irrespective of their merits," Sara Radcliffe, BIO's vice president of science and regulatory affairs, told BioWorld Today at the conference. "There may be worthy legislation that should be pursued, but we think it just ought to be done separately so that PDUFA can move through and get done."

That's because the clock is ticking. After an ongoing public comment period on reauthorizing the law, which includes a meeting Friday to solicit public recommendations, the FDA will move its final draft to Capitol Hill, where both chambers' relevant committees pick up the issue.

In reality, Congress needs to renew PDUFA well ahead of the end of the government fiscal year, Sept. 30, to satisfy timelines related to the FDA's staff because employees would need to be given notice a few months ahead of them in the unlikely event that the law wasn't reauthorized. So the process needs to wrap up in late May or early June.

"I think people, as always with must-pass legislation, will try to put a lot of things onto it," Radcliffe said. "But in this particular case, the ramifications for FDA's staff and for approvals are very severe."

Since Friday's meeting is being run by the FDA, it's not necessarily a venue for vetting legislative proposals that some want wrapped into PDUFA. After all, the agency isn't in a position to make such recommendations.

Radcliffe expects Congress to receive a PDUFA proposal similar to what will be discussed Friday, given that the agency developed it in consultation with broad input from a range of stakeholders such as drugmakers, patient groups and medical and scientific professionals.

"The package of proposals already incorporates a lot of the suggestions that have been brought to them," she said. "We can only hope that there wouldn't be substantial changes."

After that point, though, many industry watchers expect congressional members to work in some element of new drug safety regulations, or at least advance such efforts in parallel legislation. And it would seem that a bill introduced by Sens. Edward Kennedy (D-Mass.) and Mike Enzi (R-Wyo.) would be the starting point.

In a policy discussion held during the meeting here, Alex Azar, who recently stepped down as deputy secretary of the Department of Health and Human Services, predicted that "some form of" their measure would get incorporated into PDUFA. And the lawmakers, who run the Senate's Health, Education, Labor and Pensions (HELP) Committee, have said they would take up the drug safety issue in early spring when considering PDUFA.

Called the "Enhancing Drug Safety and Innovation Act," the bill includes multiple provisions related to reshaping the FDA's authorities on its drug safety oversight functions, both before and after products are approved. Among its proposals is a post-approval mandate for so-called risk evaluation and mitigation strategies (REMS), which are designed to lessen safety risks once drugs are in wide use.

Radcliffe said the bill includes some thoughtful proposals, but goes too far in taking a "one-size-fits-all" approach to post-approval safety requirements for all drugs on the market instead of recognizing the variations between one drug and another. "You want to give the agency the ability to use different tools for different situations," she added.

BIO, which takes the position that the FDA's existing safety oversight framework isn't a failure, nevertheless advocates for improving the system. For example, the agency could improve its use of information technology and mining large databases for epidemiological purposes, Radcliffe said, noting that the PDUFA proposal outlines such initiatives. It "lays the groundwork," she added, for building a better safety system and developing a life-cycle management approach to drug regulation.

In addition to new safety regulations, some believe that newly reintroduced follow-on biological legislation could get attached to PDUFA, but Radcliffe said the time frame is untenable for such an add-on, especially given lawmakers' general lack of understanding on the issue. That's her point regarding any potential attachments related to safety or biosimilars. There needs to be more time for crafting such legislation than PDUFA's renewal allows. In addition, it's worth noting that no one in Congress would want to be the reason the FDA has to lop its review staff in half because the reauthorization stalled over attachments.

"This is not something that's politically must-pass," Radcliffe added, noting that in the alternate, "things will come to a standstill. In that sense, if people are being realistic, they will understand that loading things onto PDUFA is not the best idea."