FDA identifies regulatory science needs

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Improving access to postmarket data sources was one of the needs noted by the FDA’s Center for Drug Evaluation and Research (CDER) in a 28-page report identifying science and research needs published July 25 in the Federal Register.

Determining the accuracy of available postmarket data sources is "critical" to assess the outcomes of drug therapy, according to the report authors, also noting a need to explore the feasibility of using postmarket and premarket data in safety analyses in a number of scenarios.

“Following approval of a drug, the real-world patient population that uses that drug is rarely the same as the population evaluated in clinical trials,” the authors wrote. “Even when the patient populations are similar, real-world use may differ markedly from use in the highly monitored setting of a controlled clinical trial. Furthermore, clinical trials do not usually address long-term safety.”

Postmarket data should be assessed for their strengths and weaknesses for use in regulatory decisions, according to the authors. Additionally, they cited a need to continue to improve the quality and quantity of postmarket spontaneous reports.

“CDER continues to receive reports with incomplete or otherwise limited information, which often hampers the effective use of these reports in detecting signals and making regulatory decisions,” the authors wrote. “Efforts need to continue to improve the quality, quantity and utility of these reports to enhance the detection and understanding of rare adverse events.”

The report authors stated that risk assessment and management strategies for the safe use of drugs need to be enhanced, noting a need for better assessment with: compounded drugs, dietary supplements fraudulently containing active pharmaceutical ingredients, counterfeit versions of approved drugs and marketed unapproved drugs not medically necessary.

“If we understand these risks better, we can focus our resources of risk evaluation and mitigation strategies and medication error prevention strategies,” the authors wrote.

Other categories of identified needs in the report include: risk management strategies; scientific approaches to regulatory communications; product quality and performance; predictive models; design, analysis and monitoring of clinical trials; and individualization of patient treatment.

The report involved interviewing 200 representatives from various offices to determine their perspectives on scientific questions or needs that, if addressed, would enhance the scientific decision-making in CDER. Interviewees addressed current scientific challenges on a case-to-case basis, recurring science issues across teams, and emerging scientific challenges, according to the report.

The FDA is aiming to solicit input to gather information about ongoing external research which may address some of the regulatory science needs established in the report, and identify possible collaborations with external parties and stakeholders.

“Communicating our science and research needs represents an important step in stimulating research and fostering collaborations with our external partners,” said CDER director Janet Woodcock, MD. “We look forward to hearing from and working with our stakeholders.”

The full report will be open to a 60-day review and comment period.