title: Is there a need for a universal benefit-risk assessment framework for medicines? Regulatory and industry perspectives.
abstract: OBJECTIVE
  To explore the current status and need for a universal benefit-risk framework for medicines in regulatory agencies and pharmaceutical companies.
  
  
  METHODS
  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.
  
  
  RESULTS
  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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: School of Pharmacy and Pharmaceutical Sciences, Cardiff University, Cardiff, UK.
authors.forename: James
authors.initials: J
authors.lastname: Leong
authors.forename: Neil
authors.initials: N
authors.lastname: McAuslane
authors.forename: Stuart
authors.initials: S
authors.lastname: Walker
authors.forename: Sam
authors.initials: S
authors.lastname: Salek
mesh_terms: D003661:Decision Support Techniques; D003663:Decision Trees; D004345:Drug Industry / Q000191:economics / Q000331:legislation & jurisprudence* / Q000639:trends; D064420:Drug-Related Side Effects and Adverse Reactions; D019317:Evidence-Based Medicine / Q000379:methods / Q000639:trends; D033161:Government Regulation; D007880:Legislation, Drug; D008962:Models, Theoretical; D009313:National Health Programs / Q000191:economics / Q000458:organization & administration* / Q000639:trends; D004364:Pharmaceutical Preparations / Q000191:economics; D011358:Product Surveillance, Postmarketing / Q000379:methods* / Q000592:standards / Q000639:trends; D018570:Risk Assessment
chemical_list: D004364:Pharmaceutical Preparations
keywords: benefit-risk assessment; benefit-risk methodologies; framework; pharmaceutical industry; pharmacoepidemiology; regulatory agency; universal