As possible cuts in appropriations loom on the horizon, FDA is looking for outside help in meeting the regulatory science needs it has identified at the Center for Drug Evaluation and Research.

The Identifying CDER's Science and Research Needs Report, issued July 25, is an intermediate step for the agency on the road to releasing "a cross-cutting strategic plan for regulatory science," according to the report itself. It is part of a longer process that includes the development of the Critical Path Initiative and the release of a report on regulatory science last October (Also see "FDA Has Regulatory Science Plan; Now It Needs Funding" - Pink Sheet, 6 October, 2010.).

FDA wants to know if any outside groups are already researching the areas it is interested in, or if any would be interested in partnering with the agency. The Science Board Advisory Committee will weigh in with additional suggestions when it meets Aug. 19.

After interviewing staff in several CDER offices, the CDER Science Prioritization and Review Committee said seven main regulatory science areas need to be addressed: data and methods for analyzing post-marketing data, risk management strategies, scientific approaches to regulatory communications, product quality and performance, predictive models, personalized medicine, and the design, analysis and monitoring of clinical trials.

Regulatory science long has been a watchword for FDA Commissioner Margaret Hamburg, who focused on it in the agency's five-year plan (Also see "FDA's Revised Strategic Plan Emphasizes Countermeasures, Biosimilars" - Pink Sheet, 21 April, 2011.).

A Wide-Ranging Agenda

One particularly rapidly changing area for regulatory science is clinical trials. The report says the agency is developing several guidances in this field, including one on multiplicity adjustments.

"We need to evaluate existing statistical approaches to multiplicity (multiple endpoints, multiple treatment comparisons) and make recommendations for their use in particular situations. In particular, additional methodologies to assess complex situations such as assessment of both primary and secondary endpoints, as well as sequential and 'gatekeeper' approaches, are needed."

Other guidance in the works will cover histopathology, biomarker qualification and imaging in clinical trials (Also see "Biomarkers, PRO Tools Warrant Separate Qualification Guidelines, Groups Tell FDA" - Pink Sheet, 21 February, 2011.). At the same time, the agency plans to examine the use of statistical methods to deal with missing data and adaptive trials;

Other specific items mentioned in the CDER regulatory science report include:

# Analysis of post-marketing data - Determining the accuracy of electronic health records and spontaneous reports while searching for new sources of information. The Sentinel Initiative is expected to play a role in this;

# Risk management strategies - Determining how well the current system of Risk Evaluation and Mitigation Strategies, "Dear Health Care Provider" letters and Medication Guides works, using behavioral science as a guide. The Safe Use Initiative is expected to play a role here, as the agency works on such issues as unapproved or counterfeit drugs and medication errors;

# Regulatory communications - Evaluating drug labels, effectiveness of FDA emergency communications and drug safety communications ('FDA's Risk Communication Strategic Plan, At A Glance,' 'The Pink Sheet,' May 11, 2009);

# Product quality and performance - Getting drug manufacturers to adopt Quality by Design principles;

# Predictive models - Developing preclinical trials, pharmacometric models and the Animal Rule; and

# Personalized medicine - Investigating new biomarkers and the effect of drugs on specific populations.

-Martin Berman-Gorvine ( m.berman-gorvine@elsevier.com )

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