TY - JOUR
T1 - Quantitative Benefit–Risk Assessment
T2 - State of the Practice Within Industry
AU - Smith, Meredith Y.
AU - van Til, Janine
AU - DiSantostefano, Rachael L.
AU - Hauber, A. Brett
AU - Marsh, Kevin
N1 - Funding Information:
The authors would like to acknowledge the editorial assistance provided by Fritz Hamme.
Publisher Copyright:
© 2020, The Author(s).
PY - 2021/3/1
Y1 - 2021/3/1
N2 - Background: Benefit–risk assessments for medicinal products and devices have advanced significantly over the past decade. The purpose of this study was to characterize the extent to which the life sciences industry is utilizing quantitative benefit–risk assessment (qBRA) methods. Methods: Semi-structured interviews were conducted with a sample of industry professionals working in drug and/or medical device benefit–risk assessments (n = 20). Questions focused on the use, timing, and impact of qBRA; implementation challenges; and future plans. Interviews were recorded, transcribed, and coded for thematic analysis. Results: While most surveyed companies had applied qBRA, application was limited to a small number of assets—primarily to support internal decision-making and regulatory submissions. Positive impacts associated with use included improved team decision-making and communication. Multi-criteria decision analysis and discrete choice experiment were the most frequently utilized qBRA methods. A key challenge of qBRA use was the lack of clarity regarding its value proposition. Championing by senior company leadership and receptivity of regulators to such analyses were cited as important catalysts for successful adoption of qBRA. Investment in qBRA methods, via capability building and pilot studies, was also under way in some instances. Conclusion: qBRA application within this sample of life sciences companies was widespread, but concentrated in a small fraction of assets. Its use was primarily for internal decision-making or regulatory submissions. While some companies had plans to build further capacity in this area, others were waiting for further regulatory guidance before doing so.
AB - Background: Benefit–risk assessments for medicinal products and devices have advanced significantly over the past decade. The purpose of this study was to characterize the extent to which the life sciences industry is utilizing quantitative benefit–risk assessment (qBRA) methods. Methods: Semi-structured interviews were conducted with a sample of industry professionals working in drug and/or medical device benefit–risk assessments (n = 20). Questions focused on the use, timing, and impact of qBRA; implementation challenges; and future plans. Interviews were recorded, transcribed, and coded for thematic analysis. Results: While most surveyed companies had applied qBRA, application was limited to a small number of assets—primarily to support internal decision-making and regulatory submissions. Positive impacts associated with use included improved team decision-making and communication. Multi-criteria decision analysis and discrete choice experiment were the most frequently utilized qBRA methods. A key challenge of qBRA use was the lack of clarity regarding its value proposition. Championing by senior company leadership and receptivity of regulators to such analyses were cited as important catalysts for successful adoption of qBRA. Investment in qBRA methods, via capability building and pilot studies, was also under way in some instances. Conclusion: qBRA application within this sample of life sciences companies was widespread, but concentrated in a small fraction of assets. Its use was primarily for internal decision-making or regulatory submissions. While some companies had plans to build further capacity in this area, others were waiting for further regulatory guidance before doing so.
KW - Benefit–risk framework
KW - Discrete choice experiment
KW - Medical devices
KW - Medicinal products
KW - Multi-criteria decision analysis
KW - Structured benefit–risk assessment
UR - http://www.scopus.com/inward/record.url?scp=85094150088&partnerID=8YFLogxK
U2 - 10.1007/s43441-020-00230-3
DO - 10.1007/s43441-020-00230-3
M3 - Article
AN - SCOPUS:85094150088
VL - 55
SP - 415
EP - 425
JO - Therapeutic Innovation and Regulatory Science
JF - Therapeutic Innovation and Regulatory Science
SN - 2168-4790
ER -