FDA’s 5-Year Plan For User Fees Puts Post- And Pre-Market On Equal Footing

Over the next five years, FDA will shift its regulatory focus to ensure that the safe and effective use of drugs is of equal importance as ushering safe and effective medicines to market.

The agency laid out a strategy to change its regulatory paradigm before the next round of Prescription Drug User Fee Act negotiations in a five-year spending ¹ plan released by the agency April 1.

"With the goal of maintaining a systematic and scientific approach to the evaluation of benefit/risk throughout the product lifecycle, FDA must build the scientific and administrative capacity needed to become active and collaborative players in the U.S. health care delivery system," the draft plan states.

Drug safety elements were first introduce to the user fee program under PDUFA III, which authorized additional resources for risk management plan activities at pre-NDA and pre-BLA meetings, during the review of applications, and in the first two-to-three years after approval.

PDUFA IV and the FDA Amendments Act significantly expand the agency's post-market focus, and the report describes how FDA intends to allocate user fee funds to meet its commitments under the user fee program.

An Abundance Of Safety Funding

"Under the PDUFA IV program, $29.29 million (plus an annual inflation factor) will be allocated annually to enhance the Agency's drug safety capabilities. With these funds, FDA will be able to increase the number of employees dedicated to safety evaluation of marketed medications," the report states.

Along with an increase in user fees negotiated with industry, FDAAA gives FDA the authority to collect additional monies from pharma and biotech firms to carry out the increased safety oversight powers created under the law. Those fees would help FDA implement Risk Evaluation and Mitigation Strategies, mandate post-approval studies and label changes and actively monitor risk.

The proposed plan does not address FDA's intended use of those funds, set at $25 million for fiscal 2008, but the agency expects to include a description in a subsequent update of the plan.

As part of the plan, "FDA will also be able to add resources for adopting new scientific approaches to drug safety, reducing the risk of medication errors, improving the utility of existing tools for detection and prevention of adverse events, and incorporating the new approaches into the agency's drug safety program."

The proposed plan represents a compilation of planned and ongoing safety activities at the agency and reiterates FDA's new Safety First initiative aimed at bringing PDUFA-style management to post-market oversight (² (Also see "Safety First: FDA Unveils New Postmarketing Management Process For Safety" - Pink Sheet, 3 Mar, 2008.), p. 4).

FDA is currently straining to implement the new authorities and tools granted by FDAAA, and CDER officials recently were granted permission to miss PDUFA goals until fully trained staff can handle the workload (³ (Also see "FDA Misses User Fee Goals As Resources Tighten For Application Reviews, SPAs" - Pink Sheet, 17 Mar, 2008.), p. 11). CDER is adding safety evaluators, epidemiologists, risk management experts, medication error experts, and regulatory project managers, largely to the Office of Surveillance and Epidemiology (⁴ (Also see "FDA’s Drug Safety Office Reorganizing To Accommodate Influx Of PDUFA Staff" - Pink Sheet, 25 Feb, 2008.), p. 5).

"Similarly, CBER has taken steps to support and strengthen its post-market safety activities. CBER is increasing the staff in the Office of Biostatistics and Epidemiology for safety activities ... and will be expanding resources and FTEs to access and analyze health care databases in 2008," the report states.

With People Come Workshops And Guidances

FDA is working to improve its ability to detect and analyze safety signals from post-market data. To improve its collection and analysis of adverse event data, the agency recently held a public workshop and is planning its first systematic evaluation of its passive surveillance system (⁵ (Also see "What Does AERS Mean? FDA Takes A Close Look At Spontaneous Reporting" - Pink Sheet, 11 Feb, 2008.), p. 31).

FDA also is working to establish and implement epidemiology best practices. The agency is planning a May 2008 public workshop to receive input on a guidance that is expected to be issued in fiscal year 2010. FDA will be focusing significant resources toward increasing access to large electronic databases for targeted post-market surveillance and epidemiology, including collaborations with other agencies to share electronic health record data and administrative claims data.

Funding also will be allocated to help FDA continue to strengthen risk management and communication tools. "FDA will develop a plan to identify, develop and validate RiskMAPs and REMS elements including risk communication tools and will also conduct an assessment of their effectiveness," the plan says. The agency will hold a public workshop to help prioritize which tools should be evaluated.

Also using PDUFA funds, FDA will upgrade its information technology infrastructure. Among other changes, FDA is working to create a single Internet portal for product safety submissions and standardize adverse event reporting (⁶ , p. 8).

The agency also is exploring the possibility of centralizing its adverse event reporting systems. And FDA is continuing work on assembling the Sentinel network (⁷ (Also see "Rx Safety Gets More Attention From CERTs With Addition Of Health IT Center" - Pink Sheet, 16 Apr, 2007.), p. 20).

FDA also committed under PDUFA IV to reducing review times for new drug trade names, an issue that is becoming increasing acute for drug firms (see ⁸ (Also see "Drug Name Battles May Be Waged More Publicly; FDA Petitioned On Zentase" - Pink Sheet, 7 Apr, 2008.)).

In addition to establishing review goals and timelines and a working group to ensure FDA meets those goals, the agency is developing new guidance on the contents of a complete submission package for a proposed name, slated for publication in FY 2010. The agency also expects to publish guidance on name evaluation best practices by end of FY 2012.

FDA is in the process of preparing for a public meeting in the first half of 2008 to discuss and plan a pilot program under which sponsors evaluate proposed trade names and submit the data from those evaluations to FDA (⁹ (Also see "Brand Names In 2008: Can Industry Pilot Its Way Out Of Trouble?" - Pink Sheet, 14 Jan, 2008.), p. 15). FDA expects to publish a concept paper by the end of FY 2008 and conduct the program, which will run at least two years, in FY 2009.

FDA will also make the code for its computer system that analyzes drug trade names available to the public for further development this year.

Epidemiology Is A Growth Category

Overall, FDA plans to spend $13 million to support and strengthen post-market activities, $7 million on epidemiology best practices and data acquisition, $4 million on risk management and risk communication and $5.3 million on new drug trade name review in FY 2008.

The agency would allocate user fees fairly consistently across those categories during the next five years if the proposal is made final (see chart: " ¹⁰ APPLICATION OF PDUFA IV DRUG SAFETY ENHANCEMENT RESOURCES "). Factoring in increases for inflation, FDA expects spending on post-market activities, risk management and communication and drug name reviews to increase by about 25 percent by FY 2012.

Spending to improve epidemiologic tools and methods would rise slightly faster; the agency projected a 36 percent increase, to $9.5 million, for FY 2012.

- Brian Marson ([email protected])