Patient and publicinvolvement in the benefit riskassessment of medicines:developing a semi quantitativeframework toincorporate patient views askey criteria in decision making

During a drug’s lifecycle, evidence must demonstrate that the benefits of the product continue to outweigh the risks. Benefit-risk (B-R) assessment is a vital stage of the drug approval process and is an important task for regulators. Involving patients in B-R assessment is a recent development. Patients may view benefits and risks very differently when compared with the views of pharmaceutical companies or regulatory assessors. The aim of this research was to investigate this topic to ultimately propose a framework for involving patients in this process. The research strategy involved three phases. In phase I, a survey was submitted to (1) pharmaceutical companies and regulatory agencies and (2) patient advocacy groups across Europe, to obtain their opinions on involving patients in B-R assessment. Phase I of the research identified several challenges, including: how to ensure adequate representation of patients and a lack of an established method. It was also identified that to date, only patient advocacy groups were directly involved in B-R assessment discussions. However, some companies were developing initiatives to involve patients. Based on these findings, phase II was implemented, where individuals from regulatory agencies, pharmaceutical companies and patient advocacy groups participated in semi-structured interviews to identify themes around patient involvement and to establish rich data around the current challenges. This data was used to inform the development of a novel framework, one element of which was tested in phase III of the research; where qualitative focus groups were conducted with patients. The framework proposed from this research, therefore, involves qualitative focus groups, enabling patients to provide insight into their disease and treatment. The information obtained, when presented alongside quantitative preference elicitation data, may then be used to contribute to B-R discussions by regulators, to ultimately support their decision-making.