Italian Medicines Agency (AIFA), Rome, Italy.
Ann Oncol. 2014 Jan;25(1):265-9. doi: 10.1093/annonc/mdt512.
The process leading to a regulatory outcome is guided by factors both related and unrelated to the data package, defined in this analysis as 'formal and informal factors', respectively. The aim of this qualitative study was to analyse which formal and informal factors drive the decision-making process of the European Medicines Agency (EMA) and Food and Drug Administration (FDA) regulators with regard to anticancer drugs, using in-depth semi-structured interviews with regulators of the two agencies.
In line with the theory and practice of qualitative research, no set sample size was defined a priori. Respondent enrolment continued until saturation and redundancy were reached. Data were collected through means of in-depth semi-structured interviews conducted either in a face-to-face setting or via Skype(®) with each regulator. The interviews were audio-recorded and verbatim transcribed. The analysis was manually carried out on the transcribed text. Data were independently coded and categorized by two researchers. Interpretation of the findings emerged through a process of triangulation between the two.
Seven EMA and six FDA regulators, who had extensive experience with making decisions about anticancer medicines, were interviewed between April and June 2012. There is an open dialogue between the FDA and EMA, with the two moving closer and exchanging information, not opinions. Differences in decision-making between the agencies may be due to a different evaluation of end points. Different interaction modalities with industry and patients represent an additional source of divergence with a potential impact on decision-making. The key message of our respondents was that the agencies manage uncertainty in a different way: unlike the EMA, the FDA has a prevailing attitude to take risks in order to guarantee quicker access to new treatments.
Although formal factors are the main drivers for regulatory decisions, the influence of informal factors plays an important role in the drug evaluation process.
导致监管结果的过程受到与数据方案相关和不相关的因素的影响，在本分析中分别定义为“正式因素”和“非正式因素”。本定性研究的目的是分析在抗癌药物方面，欧洲药品管理局（EMA）和美国食品药品监督管理局（FDA）监管机构的正式和非正式因素如何驱动其决策过程，方法是对这两个机构的监管人员进行深入的半结构化访谈。
根据定性研究的理论和实践，事先没有确定固定的样本量。在达到饱和和冗余时，继续招募受访者。通过面对面或通过 Skype（®）与每位监管人员进行深入的半结构化访谈收集数据。访谈进行录音并逐字记录。分析手动对转录文本进行。两位研究人员独立对数据进行编码和分类。通过两者之间的三角关系来解释研究结果。
2012 年 4 月至 6 月期间，采访了 7 名 EMA 和 6 名 FDA 监管人员，他们在制定抗癌药物决策方面拥有丰富的经验。FDA 和 EMA 之间有公开的对话，两个机构越来越接近并相互交流信息，而不是观点。机构之间决策的差异可能归因于终点评估的不同。与行业和患者的不同互动方式是另一个潜在的决策分歧的来源。我们受访者的主要信息是，各机构以不同的方式管理不确定性：与 EMA 不同，FDA 有一种普遍的态度，愿意冒险以保证更快地获得新的治疗方法。
尽管正式因素是监管决策的主要驱动因素，但非正式因素的影响在药物评估过程中起着重要作用。
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