Abstract
BackgroundBenefit–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.MethodsSemi-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.ResultsWhile 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.ConclusionqBRA 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.
Highlights
Over the past two decades, both the science and practice of benefit–risk assessment have been subjected to heightened scrutiny from regulators, health technology assessment (HTA) agencies, and manufacturing authorization holders (“sponsors”) of medicinal products and devices
It draws upon the Food and Drug Administration (FDA’s) benefit–risk assessment grid and elements of the PrOACT-URL that the EMA uses, and we have, in addition ... a standard grid format which we have as a template
In support of our initial hypotheses, we found that quantitative benefit– risk assessment (qBRA) was being used, especially within companies who had instituted a structured approach to benefit–risk assessment
Summary
Over the past two decades, both the science and practice of benefit–risk assessment have been subjected to heightened scrutiny from regulators, health technology assessment (HTA) agencies, and manufacturing authorization holders (“sponsors”) of medicinal products and devices. Regulators have sought to incorporate the patient’s perspective into benefit–risk assessment, as reflected in the release of new guidance and accompanying templates [16,17,18,19,20]. These efforts have enhanced the scientific rigor, transparency, and consistency with which regulatory agencies conduct benefit–risk assessments [21,22,23,24,25]. 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. While some companies had plans to build further capacity in this area, others were waiting for further regulatory guidance before doing so
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