WASHINGTON Hopes for a mean, lean PDUFA bill that would cruise speedily through Congress are being dashed as lawmakers, and industry, continue to add their pet extras.
As a House subcommittee held its final hearing on the user fee package last week and a Senate committee prepared for a markup session Wednesday, lawmakers introduced more legislation they want to add to the design of the PDUFA reauthorization, ranging from reforming the FDA to advancing pediatric trials. And now there's talk of adding cosmetics to the mix. (See BioWorld Today, April 19, 2012.)
One of the more expansive bills introduced last week, the PATIENTS' FDA Act, S. 2292, is intended to complement the user fee/performance goal agreements negotiated between industry and the FDA by ensuring transparency and accountability in the agency's review and decision processes, according to the bill's sponsors, Sens. Richard Burr (R-N.C.) and Tom Coburn (R-Okla.)
The bill would require the FDA to submit extensive annual reports to Congress, showing that it is meeting its PDUFA goals. Citing a recent Government Accountability Office report that criticized the agency's device performance standards, the bill's sponsors blamed that failure on "a complete lack of congressional oversight. Meaningful Congressional oversight will ensure that the FDA is held accountable for its performance goals and is fulfilling its public health mission on behalf of patients in a predictable and timely manner, including decisions on life-saving drugs and devices," Burr and Coburn said. "This will help keep medical innovation and job creation from going overseas."
But the FDA is wary of adding more measurements to its workload. Testifying before the House Energy and Commerce Subcommittee on Health last week, Janet Woodcock, director of the agency's drug center, warned that adding provisions to PDUFA would have tradeoffs.
In the last PDUFA reauthorization, Congress added substantial policy changes and provisions, including risk evaluation and mitigation strategies, that significantly expanded FDA's responsibilities and activities. Subsequently, the FDA fell behind on its drug approval goals because it was trying to implement the new provisions. (See BioWorld Today, Oct. 26, 2011.)
In addition to the annual performance reports, S. 2292 would require the FDA to:
document all significant decisions, providing that documentation upon request;
have legal counsel review all warning letters, including untitled letters, before they're issued (this used to be common practice, but Commissioner Margaret Hamburg discontinued it as a way to expedite the warning letter process);
recalibrate risk-benefit considerations;
reduce unnecessary delays and the regulatory burdens of the current clinical trial system;
restore an appropriate balance between conflict-of-interest considerations and necessary experts on advisory committees;
develop an integrated strategy and management plan for each center;
contract for an independent assessment of its drug review program.
Another bill that could be included in PDUFA is the bipartisan Better Pharmaceuticals and Devices for Children Act, S. 2289. Like the House draft PDUFA bill, S. 2289 would do away with the five-year sunsets for the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act (PREA), making them permanent.
But S. 2289 would strengthen those laws by requiring the submission of a pediatric study plan at the end of Phase II in the drug development process and ensuring that the pediatric studies are completed. Both measures were recommended by the American Academy of Pediatrics (AAP). (See BioWorld Today, Feb. 3, 2012.)
As currently written, PREA doesn't require a sponsor to submit a plan for pediatric studies until it submits its biologic or new drug application/supplement for the adult use. Submitting the pediatric plan that late in the process can lead to delays of important pediatric data and delay the development of pediatric drugs.
PREA also doesn't give the FDA enforcement tools to ensure pediatric trials are completed on time. As a result, 78 percent of PREA studies in the FDA's drug center and 54 percent in its biologics center that were due after Sept. 27, 2007, are still pending or were completed after their due date. S. 2289 would give the agency the tools it needs to enforce timely completion of the trials.
Additionally, the bill would increase the transparency of completed, pending and declined studies in children. Prior to 2007, written requirements submitted to the FDA were not made public. Since this information could be critical to researchers and clinicians, S. 2289 would make written requests issued between 2002 and 2007 public.
Since PDUFA, which expires Sept. 30, is considered a "must-pass" bill, sponsors of other pieces of drug- and device-related legislation are looking at it as a vehicle for their bills. For instance, Rep. Marsha Blackburn (R-Tenn.) is reportedly considering it as a way to force the FDA to approve cosmetic products containing prostaglandin analogues as drugs.
Whether it's added to PDUFA or passed as a standalone bill, such legislation would benefit Allergan Inc., which makes Latisse (bimatoprost), the only product approved by the FDA to treat people with sparse lashes. Several other companies are selling similar, less-expensive products as cosmetics, without going through the clinical trials required for Latisse.
Bill Aims at Making Generics Safer
Looking to undo the consequences of a recent Supreme Court ruling, Sen. Patrick Leahy (D-Vt.) last week introduced the Patient Safety and Generic Labeling Improvement Act, S. 2289, to give generic drug companies the ability to improve the warning information for their products in the same way that brand manufacturers can under existing law.
The Supreme Court ruled 5-4 last year in Pliva v. Mensing that generic-drugmakers can't be held liable under state tort law for "failure to warn," even if they know their label is inadequate. The decision was based on a federal law requiring generics to have the same label as the brand drug.
In the 10 months since the Supreme Court decision, more than 40 judges have dismissed cases alleging inadequate labeling by generic manufacturers, according to Leahy's office. A companion bill to S. 2289 is expected to be introduced in the House.