New York University School of Medicine.
Center for Outcomes Research and Evaluation, Yale-New Haven Hospital.
Milbank Q. 2019 Sep;97(3):796-819. doi: 10.1111/1468-0009.12403. Epub 2019 Jul 14.
Policy Points Food and Drug Administration (FDA) advisory committee recommendations and the agency's final actions exhibit high rates of agreement, with cases of disagreement tending to reflect the proposed action type and degree of advisory committee consensus. In the case of disagreements, the FDA tended to be less likely than its advisory committees to approve new products, approve new supplemental indications, or enact new safety changes. These findings raise important issues regarding the factors that differentially shape decision making by advisory committees and the FDA as an agency, including institutional or reputational concerns.
The Food and Drug Administration (FDA) convenes advisory committees to provide external scientific counsel on potential agency actions and to inform regulatory decision making. The degree to which advisory committees and their respective agency divisions disagree on recommendations has not been well characterized across product and action types.
We examined public documents from FDA advisory committee meetings and medical product databases for all FDA advisory committee meetings from 2008 through 2015. We classified the 376 voting meetings in that period by medical product, regulatory, and advisory committee meeting characteristics. We used multivariable logistic regression to determine the associations between these characteristics and discordance between the advisory committee's recommendations and the FDA's final actions.
Twenty-two percent of the FDA's final actions were discordant with the advisory committee's recommendations. Of these, 75% resulted in the FDA making more restrictive decisions after favorable committee recommendations, and 25% resulted in the agency making less restrictive decisions after unfavorable committee recommendations. Discordance was associated with lower degrees of advisory committee consensus and was more likely for agency actions focused on medical product safety than for novel approvals or supplemental indications. Statements by public speakers, advisory committee conflicts of interest, and media coverage were not associated with discordance between the committee and the agency.
The FDA disagrees with the recommendation of its advisory committees a minority of the time, and in these cases it tends to be less likely to approve new products or supplemental indications and take safety actions. Deviations from recommendations thus offer an opportunity to understand the factors influencing decisions made by both the agency and its expert advisory groups.
食品和药物管理局（FDA）顾问委员会的建议和机构的最终行动表现出高度的一致性，意见不一致的情况往往反映了拟议的行动类型和顾问委员会共识的程度。在意见不一致的情况下，FDA 倾向于不批准新产品、批准新的补充适应症或实施新的安全变更，其可能性低于顾问委员会。这些发现提出了一些重要问题，涉及到影响顾问委员会和作为机构的 FDA 做出决策的因素，包括机构或声誉方面的考虑。
食品和药物管理局（FDA）召集顾问委员会，就潜在的机构行动提供外部科学建议，并为监管决策提供信息。顾问委员会及其各自的机构部门在推荐方面的分歧程度在不同产品和行动类型中尚未得到很好的描述。
我们查阅了 2008 年至 2015 年期间 FDA 顾问委员会会议的公开文件和医疗产品数据库。我们按医疗产品、监管和顾问委员会会议特征对该期间的 376 次投票会议进行分类。我们使用多变量逻辑回归来确定这些特征与顾问委员会的建议和 FDA 的最终行动之间的差异之间的关联。
FDA 的最终行动中有 22%与顾问委员会的建议不一致。其中，75%的情况是在顾问委员会提出有利建议后，FDA 做出了更具限制性的决策，25%的情况是在委员会提出不利建议后，该机构做出了限制较少的决策。不一致与顾问委员会共识程度较低有关，并且与关注医疗产品安全的机构行动相比，与新药批准或补充适应症的不一致更有可能发生。公开演讲者的发言、顾问委员会的利益冲突以及媒体报道与委员会和机构之间的不一致无关。
FDA 只有少数时候不同意其顾问委员会的建议，在这些情况下，它倾向于不太可能批准新产品或补充适应症，并采取安全措施。因此，建议的偏离为了解影响机构及其专家顾问团体做出决策的因素提供了机会。
