An abstract was presented as a poster at the DIA 2012 48th Annual Meeting: DIA 2012 48th Annual Meeting Poster Abstracts. Drug Information Journal 2012; 46: 494–504. DOI:10.1177/0092861512449813.
Is there a need for a universal benefit–risk assessment framework for medicines? Regulatory and industry perspectives†
Article first published online: 5 JUN 2013
Copyright © 2013 John Wiley & Sons, Ltd.
Pharmacoepidemiology and Drug Safety
Volume 22, Issue 9, pages 1004–1012, September 2013
How to Cite
Leong, J., McAuslane, N., Walker, S. and Salek, S. (2013), Is there a need for a universal benefit–risk assessment framework for medicines? Regulatory and industry perspectives. Pharmacoepidem. Drug Safe., 22: 1004–1012. doi: 10.1002/pds.3464
- Issue published online: 20 AUG 2013
- Article first published online: 5 JUN 2013
- Manuscript Accepted: 29 APR 2013
- Manuscript Revised: 2 APR 2013
- Manuscript Received: 5 SEP 2012
- benefit–risk assessment;
- benefit–risk methodologies;
- pharmaceutical industry;
- regulatory agency;
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. Copyright © 2013 John Wiley & Sons, Ltd.