A Framework for Incorporating Patient Preferences Regarding Benefits and Risks into Regulatory Assessment of Medical Technologies

Ho, Martin; Saha, Anindita; McCleary, Kimberly; Levitan, Bennett; Christopher, Stephanie; Zandlo, Kristen; Braithwaite, R. Scott; Hauber, A. Brett

doi:10.1016/j.jval.2016.02.019

Cited by 113 publications

(116 citation statements)

References 3 publications

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“…PE in the research and development setting especially has received much focus with the development of frameworks or guidance . There are also guidance or frameworks at other milestones such as in health technology appraisal, benefit‐risk assessment and in value determination . The issue of definition and terminology of PE and patient centricity has also highlighted the need for a common understanding to facilitate multistakeholder teamwork .…”

Section: Introductionmentioning

confidence: 99%

What do stakeholders expect from patient engagement: Are these expectations being met?

Boudes

Robinson

Bertelsen³

et al. 2018

Health Expectations

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BackgroundMeaningful patient engagement (PE) in medicines development and during the life cycle of a product requires all stakeholders have a clear understanding of respective expectations.ObjectiveA qualitative survey was undertaken to understand stakeholder expectations.DesignThe survey explored 4 themes from the perspective of each stakeholder group: meaning, views, expectations and priorities for PE. Participants were grouped into 7 categories: policymakers/regulators; health‐care professionals (HCPs); research funders; payers/purchasers/HTA; patients/patient representatives; pharmaceutical/life sciences industry; and academic researchers.ResultsFifty‐nine interviews were conducted across a range of geographies, PE experience and job seniority/role. There was consensus across stakeholders on meaning of PE; importance of promoting PE to a higher level than currently; need for a more structured process and guidance. There was little consensus on stakeholder expectations and roles. Policymakers/regulators were expected by others to drive PE, create a framework and facilitate PE, provide guidelines of good practice and connect stakeholders, but this expectation was not shared by the policymakers/regulators group. HCPs were seen as the link between patients and other stakeholders, but HCPs did not necessarily share this view.Discussion and conclusionsDespite broad stakeholder categories, clear themes emerged: there is no “leader”; no stakeholder has a clear view on how to meaningfully engage with patients; there are educational gaps; and a structure and guidance for PE is urgently required. Given the diversity of stakeholders, there needs to be multistakeholder collaborative leadership. Effective collaboration requires consensus on roles, responsibilities and expectations to synergize efforts to deliver meaningful PE in medicines life cycle.

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Section: Introductionmentioning

confidence: 99%

What do stakeholders expect from patient engagement: Are these expectations being met?

Boudes

Robinson

Bertelsen³

et al. 2018

Health Expectations

View full text Add to dashboard Cite

show abstract

“…They have also released guidance suggesting that patients may provide useful experiential information and that stated and revealed preference methods can be informative for understanding patient preferences . Similar guidance has been published by the public–private Medical Device Innovation Consortium . On the European side of drug regulation, the EMA has conducted a study to investigate elicitation of patient preferences and is participating in the Innovative Medicines Initiative—Patient Preferences in Benefit‐Risk Assessments during the Drug Life Cycle (IMI PREFER), a public–private partnership project that aims to shape the future of patient‐centered benefit‐risk assessment .…”

Section: Discussionmentioning

confidence: 99%

“…7 Similar guidance has been published by the public-private Medical Device Innovation Consortium. 65 On the European side of drug regulation, the EMA has conducted a study to investigate elicitation of patient preferences 57,66 and is participating in the Innovative Medicines Initiative-Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (IMI PREFER), a public-private partnership project that aims to shape the future of patientcentered benefit-risk assessment. 67 These initiatives underline the importance of quantitative preference-based benefit-risk assessments in drug decision making.…”

Section: Discussionmentioning

confidence: 99%

Quantifying Preferences in Drug Benefit‐Risk Decisions

Tervonen

Angelis

Hockley

et al. 2019

Clin Pharma and Therapeutics

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Benefit‐risk assessment is used in various phases along the drug lifecycle, such as marketing authorization and surveillance, health technology assessment (HTA), and clinical decisions, to understand whether, and for which patients, a drug has a favorable or more valuable profile with reference to one or more comparators. Such assessments are inherently preference‐based as several clinical and nonclinical outcomes of varying importance might act as evaluation criteria, and decision makers must establish acceptable trade‐offs between these outcomes. Different healthcare stakeholder perspectives, such as those from patients and healthcare professionals, are key for informing benefit‐risk trade‐offs. However, the degree to which such preferences inform the decision is often unclear as formal preference‐based evaluation frameworks are generally not used for regulatory decisions, and, if used, rarely communicated in HTA decisions. We argue that for better decisions, as well as for reasons of transparency, preferences in benefit‐risk decisions should more often be quantified and communicated explicitly.

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“…16 Patient and user feedbacks are critical in the early and prototyping stages. 34,35 OCT has been paired with surgical microscopes to provide intraoperative monitoring of microsurgical anastomoses, which may improve patient outcomes. 36 One challenge with OCT was that decreasing cost and/or imaging speed came at the expense of imaging resolution; as part of the iterative process of improving OCT, one group designed alternative processing methods and algorithms to solve this issue.…”

Section: Stakeholders and Market Analysismentioning

confidence: 99%

Biomedical device innovation methodology: applications in biophotonics

et al. 2017

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Abstract. The process of medical device innovation involves an iterative method that focuses on designing innovative, device-oriented solutions that address unmet clinical needs. This process has been applied to the field of biophotonics with many notable successes. Device innovation begins with identifying an unmet clinical need and evaluating this need through a variety of lenses, including currently existing solutions for the need, stakeholders who are interested in the need, and the market that will support an innovative solution. Only once the clinical need is understood in detail can the invention process begin. The ideation phase often involves multiple levels of brainstorming and prototyping with the aim of addressing technical and clinical questions early and in a cost-efficient manner. Once potential solutions are found, they are tested against a number of known translational factors, including intellectual property, regulatory, and reimbursement landscapes. Only when the solution matches the clinical need, the next phase of building a "to market" strategy should begin. Most aspects of the innovation process can be conducted relatively quickly and without significant capital expense. This white paper focuses on key points of the medical device innovation method and how the field of biophotonics has been applied within this framework to generate clinical and commercial success.

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A Framework for Incorporating Patient Preferences Regarding Benefits and Risks into Regulatory Assessment of Medical Technologies

Abstract: This article discusses key concepts in patient preference assessment of particular importance for regulators and researchers that are addressed in the MDIC Framework for patient centered benefit-risk assessment as well as the unique public-private collaboration that led its development.

Cited by 113 publications

References 3 publications

What do stakeholders expect from patient engagement: Are these expectations being met?

What do stakeholders expect from patient engagement: Are these expectations being met?

Quantifying Preferences in Drug Benefit‐Risk Decisions

Biomedical device innovation methodology: applications in biophotonics

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