Cost and Compliance Dominate Agenda for 2011

Top priorities for manufacturers include user fees, new health initiatives, and regulatory compliance.
Jan 02, 2011
Volume 35, Issue 1

The political tensions wrought by last November's mid-term elections will affect pharmaceutical manufacturers in many ways in the coming year. Congressional Republicans will seek to cut the federal deficit by squeezing resources to government agencies, including the US Food and Drug Administration. New House leaders may also seek opportunities to criticize lax regulatory oversight and drug quality failures that compromise product safety. Pressure on federal agencies to appear as tough regulators and to obtain additional revenues will intensify government efforts to fine violative companies and to ratchet up industry user fees.

Jill Wechsler
Challenges to the Obama administration's healthcare reform program are likely to create uncertainties about whether new fees and policies will be implemented as planned. The alternative is that manufacturers may have to pay stiffer rebates and taxes without the benefit of gaining millions of additional customers as part of a reformed affordable-access health-insurance system. The conventional wisdom is that President Obama has one year to enact any new legislation or establish new programs before the political infighting becomes even more intense leading up to the 2012 Presidential elections.

Fees first

The Prescription Drug User Fee Act (PDUFA) has to be reauthorized by Oct. 1, 2012, in order for FDA to continue collecting nearly $700 million in fees to support the review process for drugs and biologics. Although that legislative deadline may seem far away, FDA wants to have a PDUFA V plan ready for public review by fall in order to transmit it to Congress early 2012. Failure to reauthorize user fees by next summer theoretically would force FDA to lay off hundreds of staffers and shut down its drug review process.

In April 2010, FDA's Center for Drug Evaluation and Research (CDER) launched a two-year process for revising PDUFA. CDER Director Janet Woodcock noted that 65% of human drug review funding comes from user fees, a situation that some critics claim makes the agency overly dependent on industry. Yet, despite some qualms about the current program, no one suggests that FDA curtail any activities or cancel the fees.

Bio/pharmaceutical companies offered general support for user fees, while pointing out that multiple postmarketing requirements are slowing down the drug-review process and undermining approval timeframes. Patient advocates, pharmacists, and doctors agreed that the proliferation of risk evaluation and mitigation strategies (REMS) made drug development more costly and complicated prescribing and dispensing. Consumer groups focused more on direct-to-consumer (DTC) drug advertising, seeking user-fee support for mandatory pre-review of DTC advertisements, clearer prescribing information, better protection of patients in clinical trials, and more comparative studies to ensure that new drugs are superior to those already on the market.

FDA has been discussing these and other issues at meetings with manufacturers, patient and consumer groups, healthcare professionals and academic experts, as part of a broad, transparent consultative process required by the FDA Amendments Act (FDAAA) of 2007. A key FDA goal for PDUFA V is to gain more flexibility in meeting review timeframes and responding to sponsor meeting requests. The agency has proposed extending the review clock for more complex applications such as those with REMS, those that require advisory committee meetings, or those that involve inspections of foreign manufacturing facilities. But manufacturers fear that most new drug application (NDAs) would qualify for extensions under these criteria.

The overarching issue is to what extent industry fees should fund FDA initiatives to improve drug development and regulatory science. FDA proposes to tap fees to increase staff consultations on quality-by-design and other complex manufacturing issues, on innovative clinical trial designs, on using biomarkers in drug development and for standardizing electronic submissions. Additional resources also could support the Sentinel active surveillance system, standards for meta-analysis, biomarker qualification, development of treatments for rare diseases, and improved dose selection and drug-safety assessments. But this year's fee to process an NDA with clinical data already exceeds $1.5 million, up from $1.17 million in 2008, and manufacturers are wary that expanding the pool of activities supported by PDUFA would boost industry outlays disproportionately.

Many of these issues will be addressed by Congress as it crafts a broader FDA reform bill that includes PDUFA reauthorization. As with FDAAA, this "must-pass" legislation will be a prime candidate to carry measures establishing a host of new FDA policies and programs: curbs on drug advertising, expanded drug reimportation, ban on pay-for-delay deals between innovator and generic-drug makers, refinements to the REMS program, new requirements that drugs demonstrate comparative superiority, and enhanced FDA authority to pull drugs off the market and to issue subpoenas are some of the possibilities.

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