School of Pharmacy and Pharmaceutical Sciences, Cardiff University, Cardiff, UK.
Pharmacoepidemiol Drug Saf. 2013 Sep;22(9):1004-12. doi: 10.1002/pds.3464. Epub 2013 Jun 5.
To explore the current status and need for a universal benefit-risk framework for medicines in regulatory agencies and pharmaceutical companies.
A questionnaire was developed and sent to 14 mature regulatory agencies and 24 major companies. The data were analysed using descriptive statistics, for a minority of questions preceded by manual grouping of the responses.
Overall response rate was 82%, and study participants included key decision makers from agencies and companies. None used a fully quantitative system, most companies preferring a qualitative method. The major reasons for this group not using semi-quantitative or quantitative systems were lack of a universal and scientifically validated framework. The main advantages of a benefit-risk framework were that it provided a systematic standardised approach to decision-making and that it acted as a tool to enhance quality of communication. It was also reported that a framework should be of value to both agencies and companies throughout the life cycle of a product. They believed that it is possible to develop an overarching benefit-risk framework that should involve relevant stakeholders in the development, validation and application of a universal framework. The entire cohort indicated common barriers to implementing a framework were resource limitations, a lack of knowledge and a scientifically validated and acceptable framework.
Stakeholders prefer a semi-quantitative, overarching framework that incorporates a toolbox of different methodologies. A coordinating committee of relevant stakeholders should be formed to guide its development and implementation. Through engaging the stakeholders, these outcomes confirm sentiments and need for developing a universal benefit-risk assessment framework.
探索监管机构和制药公司中用于药品的通用获益-风险框架的现状和需求。
制定了一份问卷，并发送给 14 个成熟的监管机构和 24 家大公司。使用描述性统计方法对数据进行分析，对于少数问题，在对回复进行手动分组之前进行了分析。
总体回复率为 82%，研究参与者包括机构和公司的主要决策制定者。没有一个机构完全使用定量系统，大多数公司更喜欢定性方法。这组人不使用半定量或定量系统的主要原因是缺乏通用且经过科学验证的框架。获益-风险框架的主要优势在于它为决策提供了系统的标准化方法，并且可以作为增强沟通质量的工具。据报道，该框架对于机构和公司在产品整个生命周期中都具有价值。他们认为，可以开发一个总体的获益-风险框架，该框架应让相关利益相关者参与通用框架的制定、验证和应用。整个队列都表示，实施框架的共同障碍是资源限制、知识匮乏以及缺乏科学验证和可接受的框架。
利益相关者更喜欢包含不同方法工具箱的半定量、总体框架。应成立一个相关利益相关者的协调委员会来指导其开发和实施。通过与利益相关者合作，这些结果证实了开发通用获益-风险评估框架的意见和需求。
