Consumer Protection: Drug Shortages, Import Safety Dominate Discussions

Lawmakers are expected to resume deliberations regarding reauthorization of the Prescription Drug User Fee Act (PDUFA) early this year. The law, which allows the Food & Drug Administration to collect fees from pharmaceutical manufacturers to help speed up drug reviews, is set to expire on Sept. 30.

Industry-paid user fees are critical for FDA because they make up about 60% of the agency’s drug review budget. PDUFA was first signed into law in 1992 and has been reauthorized every five years since.

Drug companies and FDA reached a deal last fall to increase user fees by 6% above fiscal 2012 levels, bringing the projected total amount to $693 million for fiscal 2013, in exchange for increased communication and more consistent drug approvals (C&EN, Sept. 12, 2011, page 17). It is now up to Congress to decide whether to go along with those recommendations. Industry groups and FDA are urging Congress to reauthorize PDUFA quickly, without tacking on additional FDA-related mandates to the legislation.

Additional provisions “could create excessive regulatory burdens” or “delay the legislative process,” said David E. Whea­don in testimony at an FDA public meeting last fall. Wheadon is a senior vice president for scientific and regulatory affairs at the Pharmaceutical Research & Manufacturers of America (PhRMA). “Failure to reauthorize PDUFA in a timely manner would have an extraordinarily disruptive effect on the agency and impede patients’ access to new treatments,” he stressed.

A clean bill without additional provisions, however, is unlikely. Lawmakers in both the House and Senate have already signaled their intent to use PDUFA reauthorization to address other FDA-related concerns.

For example, at a hearing last month held by the Senate Health, Education, Labor & Pensions (HELP) Committee to address the growing problem of prescription drug shortages in the U.S., Sen. Barbara A. Mikulski (D-Md.) called for measures to alleviate drug shortages as part of PDUFA reauthorization. “President [Barack] Obama issued an executive order on Oct. 31 to immediately take action to reduce shortages; but we can and must do more,” she said.

Mikulski is a cosponsor of the Preserving Access to Life-Saving Medications Act (S. 296), introduced last year by Sen. Amy Klobuchar (D-Minn.). The bill would require all drug manufacturers to give FDA at least six months’ notice when they plan to discontinue or interrupt drug production or make adjustments that could lead to drug shortages of medically necessary drugs. Currently only companies that are the sole manufacturer of a drug are required to notify FDA when they make such changes.

S. 296 would also require FDA to establish monetary penalties for companies that fail to notify the agency of production changes. Currently there is no penalty if a company does not comply.

Members of the Senate HELP Committee have also hinted at introducing initiatives into the PDUFA reauthorization that would help secure the safety of the pharmaceutical supply chain, which is becoming increasingly globalized. After a hearing last fall on government oversight of the drug supply chain, Mikulski and HELP Committee Chairman Tom Harkin (D-Iowa) suggested using PDUFA as a vehicle to pass provisions that would modernize the drug supply system.

Some members of the House Energy & Commerce Committee are also eyeing PDUFA reauthorization as an opportunity to examine and improve FDA’s regulatory process. At a subcommittee hearing last summer on PDUFA reauthorization, lawmakers discussed how FDA regulations affect innovation, job creation, and access to critical drugs. They also brought up concerns that FDA is focusing too heavily on risk in its risk-benefit analysis of drug approvals and that current conflict-of-interest provisions limit who can serve on FDA drug review advisory committees.

“These problems at FDA appear to be stifling American innovation, costing American jobs, and hurting American patients,” said Fred Upton (R-Mich.), chairman of the committee. The committee is expected to examine such concerns again this year as PDUFA reauthorization moves ahead.

For the first time, Congress is also set to take up the Generic Drug User Fee Act (GDUFA) this year; a hearing is already scheduled for next month in the House Energy & Commerce Committee. Although the last several budgets have contained a generic drug user fee program, new legislation is needed to put such fees into place, according to FDA.

At next month’s hearing, the Energy & Commerce Committee is also planning to address user fees for biosimilars, generic-like versions of complex biological drugs. A consideration of biosimilars fees is also a first for Congress.