Alexion Pharmaceuticals, Inc., 121 Seaport Boulevard, Boston, MA, 02210, USA.
Department of Regulatory and Quality Sciences, School of Pharmacy, University of Southern California, Los Angeles, CA, 90089, USA.
Ther Innov Regul Sci. 2021 Mar;55(2):415-425. doi: 10.1007/s43441-020-00230-3. Epub 2020 Oct 27.
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.
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.
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.
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.
在过去的十年中，药品和医疗器械的获益-风险评估有了显著进展。本研究的目的是描述生命科学行业利用定量获益-风险评估（qBRA）方法的程度。
对从事药物和/或医疗器械获益-风险评估的行业专业人员（n=20）进行了半结构式访谈。问题集中在 qBRA 的使用、时间安排和影响；实施挑战；以及未来计划。对访谈进行了录音、转录和编码，以进行主题分析。
尽管大多数接受调查的公司都应用了 qBRA，但应用仅限于少数资产-主要是支持内部决策和监管提交。使用 qBRA 的积极影响包括改善团队决策和沟通。多准则决策分析和离散选择实验是最常使用的 qBRA 方法。qBRA 使用的一个关键挑战是其价值主张不明确。公司高层的支持和监管机构对这些分析的接受被认为是成功采用 qBRA 的重要催化剂。在某些情况下，还通过能力建设和试点研究对 qBRA 方法进行了投资。
在这个生命科学公司的样本中，qBRA 的应用很广泛，但集中在一小部分资产上。其使用主要是用于内部决策或监管提交。虽然一些公司有计划在这一领域进一步建立能力，但另一些公司在这样做之前正在等待进一步的监管指导。
