A Retrospective Review of Center for Biologics Evaluation and Research Advisory Committee Meetings in the Context of the FDA's Benefit-Risk Framework.

Autor: Mutanga JN; Office of Biostatistics and Pharmacovigilance, Center for Biologics Evaluation and Research, US FDA, Silver Spring, Maryland, USA., Nukala U; Office of Biostatistics and Pharmacovigilance, Center for Biologics Evaluation and Research, US FDA, Silver Spring, Maryland, USA., Rodriguez Messan M; Office of Biostatistics and Pharmacovigilance, Center for Biologics Evaluation and Research, US FDA, Silver Spring, Maryland, USA., Yogurtcu ON; Office of Biostatistics and Pharmacovigilance, Center for Biologics Evaluation and Research, US FDA, Silver Spring, Maryland, USA., McCormick Q; Office of Therapeutic Products, Center for Biologics Evaluation and Research, US FDA, Silver Spring, Maryland, USA., Sauna ZE; Office of Therapeutic Products, Center for Biologics Evaluation and Research, US FDA, Silver Spring, Maryland, USA., Whitaker BI; Office of Biostatistics and Pharmacovigilance, Center for Biologics Evaluation and Research, US FDA, Silver Spring, Maryland, USA., Forshee RA; Office of Biostatistics and Pharmacovigilance, Center for Biologics Evaluation and Research, US FDA, Silver Spring, Maryland, USA., Yang H; Office of Biostatistics and Pharmacovigilance, Center for Biologics Evaluation and Research, US FDA, Silver Spring, Maryland, USA. Hong.Yang@fda.hhs.gov.
Jazyk: angličtina
Zdroj: The AAPS journal [AAPS J] 2023 Feb 09; Vol. 25 (1), pp. 24. Date of Electronic Publication: 2023 Feb 09.
DOI: 10.1208/s12248-023-00789-3
Abstrakt: The US FDA Center for Biologics Evaluation and Research (CBER) is responsible for the regulation of biologically derived products. FDA has established Advisory Committees (AC) as vehicles to seek external expert advice on scientific and technical matters related to the development and evaluation of products regulated by the agency. We aimed to identify and evaluate common topics discussed in CBER AC meetings during the regulatory decision-making process for biological products and medical devices. We analyzed the content of 119 CBER-led AC meetings between 2009 and 2021 listed on the FDA AC webpage. We reviewed publicly available meeting materials such as briefing documents, summaries, and transcripts. Using a structured review codebook based on FDA benefit-risk guidance, we identified important considerations within the benefit-risk dimensions discussed at the AC meetings: therapeutic context, benefit, risk and risk management, and benefit-risk trade-off, where evidence and uncertainty are critical parts of the FDA benefit-risk framework. Based on a detailed review of 24 topics discussed in 23 selected AC meetings conducted between 2016 and 2021, the two most frequently discussed considerations were "Uncertainty about assessment of the safety profile" and "Uncertainty about assessment of the benefit based on clinical trial data" (16/24 times each) as defined in our codebook. Most of the reviewed meetings discussed Investigational New Drug or Biologics License Applications of products. This review could help sponsors better plan and design studies by contextualizing how the benefit-risk dimensions were embedded in the AC discussions and the considerations that went into the final AC recommendations.
(© 2023. This is a U.S. Government work and not under copyright protection in the US; foreign copyright protection may apply.)
Databáze: MEDLINE
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