The Benefit-Risk Assessment of Medicines: Experience of a Consortium of Medium-Sized Regulatory Authorities

McAuslane, Neil; Leong, James; Liberti, Lawrence; Walker, Stuart

doi:10.1177/2168479017696260

Cited by 15 publications

(22 citation statements)

References 7 publications

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“…The revised International Council for Harmonisation (ICH)'s M4E guidance (revision 2), which was approved in 2015, may have played a facilitating role in this regard, although no respondent specifically referenced it [4]. The M4E revision set forth a standard format for presenting a product's benefit-risk assessment, one that incorporated the key elements found in the FDA's Benefit-Risk Assessment Grid [5]. Thus, companies are required to use this structured format when submitting marketing authorization applications via the electronic Common Technical Document (eCTD).…”

Section: Discussionmentioning

confidence: 99%

“…The comparatively limited use of qBRA within a company's portfolio of products, however, is consistent with recommended practice in this area. qBRA methods are most appropriate for complex decision-making, especially when the tradeoffs between benefits and risks are highly preference sensitive [5]. Results, however, suggest that qBRA may be underutilized to some extent, due to a variety of factors-perceived uncertainty regarding the value proposition, lack of consensus regarding which qBRA methods to use in which circumstances and how to implement them internally, and the absence of regulatory guidance in this area.…”

Section: Discussionmentioning

confidence: 99%

“…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. Commensurate with this, a range of initiatives has been launched, including projects aiming to identify applicable benefit-risk assessment frameworks and methods [1][2][3][4][5], catalog relevant graphics and visualization tools for use in benefit-risk decision-making [1,6,7], generate evidence-based recommendations for the conduct of benefit-risk evaluation [8,9], and incorporate patients' perspectives into such assessments [10,11].…”

Section: Introductionmentioning

confidence: 99%

“…In parallel with this work, several regulatory authorities have sought to apply specific frameworks and tools-first in the context of pilot studies [5,8,10] and then as an integral part of their review process for new drug and medical device marketing authorization applications [12][13][14][15]. The term "structured framework" refers to a systematic and standardized format for presenting the relevant benefit-risk data and the supporting narrative.…”

Section: Introductionmentioning

confidence: 99%

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Quantitative Benefit–Risk Assessment: State of the Practice Within Industry

Smith

Til

DiSantostefano

et al. 2020

Ther Innov Regul Sci

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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.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Quantitative Benefit–Risk Assessment: State of the Practice Within Industry

Smith

Til

DiSantostefano

et al. 2020

Ther Innov Regul Sci

View full text Add to dashboard Cite

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“…The development of the UMBRA (Universal Methodology for Benefit-Risk Assessment) framework makes steps towards this structured regulatory decision-making process as it incorporates several frameworks (PrOACT-URL, PhRMA BRAT and FDA 5-step framework) [ 131 ]. The UMBRA framework uses benefit-risk summary template and corresponding user manual to clearly communicate benefit risk analysis to all stakeholders and upon review was found to be of value by several regulatory agencies [ 132 , 133 ].…”

Section: The Biorima Risk Management Frameworkmentioning

confidence: 99%

Risk Management Framework for Nano-Biomaterials Used in Medical Devices and Advanced Therapy Medicinal Products

et al. 2020

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The convergence of nanotechnology and biotechnology has led to substantial advancements in nano-biomaterials (NBMs) used in medical devices (MD) and advanced therapy medicinal products (ATMP). However, there are concerns that applications of NBMs for medical diagnostics, therapeutics and regenerative medicine could also pose health and/or environmental risks since the current understanding of their safety is incomplete. A scientific strategy is therefore needed to assess all risks emerging along the life cycles of these products. To address this need, an overarching risk management framework (RMF) for NBMs used in MD and ATMP is presented in this paper, as a result of a collaborative effort of a team of experts within the EU Project BIORIMA and with relevant inputs from external stakeholders. The framework, in line with current regulatory requirements, is designed according to state-of-the-art approaches to risk assessment and management of both nanomaterials and biomaterials. The collection/generation of data for NBMs safety assessment is based on innovative integrated approaches to testing and assessment (IATA). The framework can support stakeholders (e.g., manufacturers, regulators, consultants) in systematically assessing not only patient safety but also occupational (including healthcare workers) and environmental risks along the life cycle of MD and ATMP. The outputs of the framework enable the user to identify suitable safe(r)-by-design alternatives and/or risk management measures and to compare the risks of NBMs to their (clinical) benefits, based on efficacy, quality and cost criteria, in order to inform robust risk management decision-making.

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