Key Opinion Leaders’ Interviews to Inform the Future of Benefit–Risk Planning in the Medical Total Product Life Cycle of Global Pharmaceutical and Medical Device Organizations

Arianna Simonetti (), Susan Colilla, Brian Edwards, Jürgen Kübler, Leila Lackey, Lisa Rodriguez, Susan Talbot, Hong Yang, William Wang, Danae Williams and James Matthew Higginson
Additional contact information
Arianna Simonetti: US Food and Drug Administration
Susan Colilla: Teva Pharmaceuticals
Brian Edwards: Husoteria Ltd. Ashtead
Jürgen Kübler: Quantitative Scientific Consulting
Leila Lackey: US Food and Drug Administration
Lisa Rodriguez: US Food and Drug Administration
Susan Talbot: Amgen Inc
Hong Yang: US Food and Drug Administration
William Wang: Merck Sharp & Dohme LLC
Danae Williams: GlaxoSmithKline
James Matthew Higginson: Merck KGaA

Drug Safety, 2024, vol. 47, issue 9, No 4, 853-868

Abstract: Abstract Background and Objectives Key opinion leader (KOL) interviews were conducted by the Benefit–Risk Assessment Planning (BRAP) Taskforce to seek expert opinion mainly from industry and regulatory bodies, about the current status and future direction of benefit–risk assessment (BRA) planning in the lifecycle of medical product development. The findings from these interviews are intended to help communication concerning planning for BRA between industry and regulators and shape future guidance. Methods Key opinion leader interviews consisted of 5 questions related to BRA planning, which were administered to volunteers (mainly clinicians and statisticians) within a pool of experienced pharmaceutical and medical device professionals representing academia, industry, regulatory agencies and a patient group. The interviewees’ responses to the 5 questions were summarized. To analyze the qualitative data, a Coding System was developed to label themes arising from the interviews. The key findings from the interviews were summarized into a Master Template. A quantitative analysis based on descriptive statistics was also conducted. Results Of the 27 interviewees, there were 11 professionals from regulatory agencies, 11 from industry, 4 from academia and 1 from a patient advocacy group. Key findings based on the comments provided by 48% of the interviewees indicated the need of incorporating BRA into other (e.g., existing) processes with the importance of alignment between processes being stressed in the comments provided by 59% of the interviewees. Commencing BRA early in the product lifecycle was emphasized in comments provided by 44% of the interviewees. Among other needs identified were an appropriate contextualization of benefits and risks (based on comments provided by 41% of interviewees) through adoption of an integrated approach with structured support by regulatory agencies and a need for understanding the audience with better communication of benefit–risk (BR) among all stakeholders (based on comments provided by 44% of the interviewees). Almost all comments provided by interviewees (96%) highlighted the importance of utilizing patient experience/preference to guide new product development and BRA. Comments provided by 74% of the interviewees expressed the need to understand patient tolerance for risk and trade-offs, with a majority (78%) of interviewees highlighting how to gather information, and 59% stressing the need for the selection and development of appropriate methodologies as important considerations for enhancing the quality and relevance of the data collected from patients. Conclusions Interviewees indicated that BRA should commence early in the medical product development and inform decision-making throughout the product lifecycle. Better planning and integration of BRA into existing processes within industry would be valuable. The importance of incorporating the patient voice into BRA and medical product development was emphasized. Other key findings from the KOL interviews included a need for improved communication of BR information, and establishment of methodologies for performing BRA and soliciting patient input.

Date: 2024
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DOI: 10.1007/s40264-024-01442-4

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