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, James Matthew Higginson
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引用次数: 0
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.
背景和目标:效益风险评估规划(BRAP)工作组进行了关键意见领袖(KOL)访谈,主要从行业和监管机构征求专家意见,了解医疗产品开发生命周期中效益风险评估(BRA)规划的现状和未来方向。这些访谈的结果旨在帮助行业和监管机构就 BRA 的规划进行沟通,并形成未来的指导意见:关键意见领袖访谈包括 5 个与 BRA 规划相关的问题,访谈对象为代表学术界、工业界、监管机构和患者团体的经验丰富的制药和医疗器械专业人士中的志愿者(主要是临床医生和统计学家)。对受访者对 5 个问题的回答进行了总结。为分析定性数据,开发了一个编码系统,用于标注访谈中出现的主题。访谈的主要结论被归纳到主模板中。此外,还进行了基于描述性统计的定量分析:在 27 位受访者中,11 位专业人士来自监管机构,11 位来自行业,4 位来自学术界,1 位来自患者权益组织。根据 48% 的受访者提供的意见得出的主要结论表明,有必要将生物风险评估纳入其他流程(如现有流程),59% 的受访者强调了流程之间保持一致的重要性。44% 的受访者在评论中强调了在产品生命周期的早期开始 BRA 的重要性。其他已确定的需求包括:在监管机构的结构化支持下,通过采用综合方法,对效益和风险进行适当的背景分析(根据 41% 的受访者提供的意见);需要了解受众,在所有利益相关者之间更好地沟通效益-风险(BR)(根据 44% 的受访者提供的意见)。几乎所有受访者(96%)都强调了利用患者经验/偏好指导新产品开发和 BRA 的重要性。74% 的受访者在评论中表示需要了解患者对风险和权衡的承受能力,大多数受访者(78%)强调了如何收集信息,59% 的受访者强调需要选择和开发适当的方法,作为提高从患者处收集的数据质量和相关性的重要考虑因素:受访者表示,生物风险评估应在医疗产品开发早期开始,并在整个产品生命周期中为决策提供信息。更好地规划 BRA 并将其纳入行业内的现有流程将非常有价值。受访者强调了将患者意见纳入 BRA 和医疗产品开发的重要性。从 KOL 访谈中得出的其他重要结论包括:有必要改进生物风险评估信息的沟通,并建立执行生物风险评估和征求患者意见的方法。
期刊介绍:
Drug Safety is the official journal of the International Society of Pharmacovigilance. The journal includes:
Overviews of contentious or emerging issues.
Comprehensive narrative reviews that provide an authoritative source of information on epidemiology, clinical features, prevention and management of adverse effects of individual drugs and drug classes.
In-depth benefit-risk assessment of adverse effect and efficacy data for a drug in a defined therapeutic area.
Systematic reviews (with or without meta-analyses) that collate empirical evidence to answer a specific research question, using explicit, systematic methods as outlined by the PRISMA statement.
Original research articles reporting the results of well-designed studies in disciplines such as pharmacoepidemiology, pharmacovigilance, pharmacology and toxicology, and pharmacogenomics.
Editorials and commentaries on topical issues.
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