Structured Benefit-Risk Assessment Across the Product Lifecycle: Practical Considerations

Smith, Meredith Y.; Benattia, I.; Strauss, Carmit; Bloss, Laura; Jiang, Qi

doi:10.1177/2168479017696272

Cited by 11 publications

(8 citation statements)

References 13 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Recently, a survey assessed the usage of quantitative methods in the bene t-risk assessment, based on semi-structured interviews from 20 selected individuals working in drug and/or medical device companies [13]. Additionally, a literature review of published quantitative bene t-risk approaches was conducted [14] and two published case studies reported the particular approaches of individual companies to sBRA [15]- [17].…”

Section: Introductionmentioning

confidence: 99%

A survey to assess the current status of structured Benefit-Risk assessment in the global drug and medical device industry

Gebel

Renz²,

Rodríguez

et al. 2023

Preprint

View full text Add to dashboard Cite

Background This industry survey was conducted to gain insight into the ways structured Benefit-Risk assessment (sBRA) of medical products is approached across drug or medical device developing companies, including frameworks and methods that are currently used and areas where future work is being planned. Methods A survey containing 28 questions covering five key areas of sBRA was set-up and shared with representatives from the participating companies. Each company was asked to complete a single survey response including inputs across the company’s multidisciplinary key representatives involved in benefit-risk assessment. Results Of the 26 participating companies, 21 are conducting sBRA. Qualitative frameworks were used by almost every company (19), while only 12 have used a quantitative method. Many companies have sBRA training (17), document templates (16), Standard Operating Procedures (SOPs)/checklists (13), and /or best practice manuals/examples (12) available. Software tools (15/26) and BR planning documents (11/26) were identified as areas into which many companies intend to put effort. Conclusions The industry survey confirmed a wide usage of sBRA by many companies involved in research and development. Nevertheless, sBRA is evolving and several future opportunities like the implementation of visualization tools were identified by the representatives of the pharmaceutical companies. Finally, challenges like the cross-functional comprehension of the added value of sBRA are still seen.

show abstract

Section: Introductionmentioning

confidence: 99%

A survey to assess the current status of structured Benefit-Risk assessment in the global drug and medical device industry

Gebel

Renz²,

Rodríguez

et al. 2023

Preprint

View full text Add to dashboard Cite

show abstract

“…A second, subsequent survey [ 6 ] demonstrated similar findings and further emphasized a trend towards companies initiating BR assessments earlier in drug development. Other recent industry case studies have illustrated how BR assessment frameworks can be effectively integrated into an organizational context [ 7 , 8 ].…”

Section: Introductionmentioning

confidence: 99%

A Structured Benefit-Risk Assessment Operating Model for Investigational Medicinal Products in the Pharmaceutical Industry

Sullivan

Zorenyi

Feron

et al. 2023

Ther Innov Regul Sci

View full text Add to dashboard Cite

Robust and transparent formal benefit-risk (BR) analyses for medicinal products represent a means to better understand the appropriate use of medicinal products, and to maximize their value to prescribers and patients. Despite regulatory and social imperatives to conduct structured BR (sBR) assessments, and the availability of a plethora of methodological tools, there exists large variability in the uptake and execution of sBR assessments among pharmaceutical companies. As such, in this paper we present an sBR assessment framework developed and implemented within a large global pharmaceutical company that aims to guide the systematic assessment of BR across the continuum of drug development activities, from first-time-in-human studies through to regulatory submission. We define and emphasize the concepts of Key Clinical Benefits and Key Safety Risks as the foundation for BR analysis. Furthermore, we define and foundationally employ the concepts of sBR and a Core Company BR position as the key elements for our BR framework. We outline 3 simple stages for how to perform the fundamentals of an sBR analysis, along with an emphasis on the weighting of Key Clinical Benefits and Key Safety Risks, and a focus on any surrounding uncertainties. Additionally, we clarify existing definitions to differentiate descriptive, semi-quantitative, and fully quantitative BR methodologies. By presenting our framework, we wish to stimulate productive conversation between industry peers and health authorities regarding best practice in the BR field. This paper may also help facilitate the pragmatic implementation of sBR methodologies for organizations without an established framework for such assessments.

show abstract

“…More recently, 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]. Collectively, these efforts have enhanced the scientific rigor, transparency, and consistency with which regulatory agencies conduct benefit-risk assessments [21][22][23][24][25].…”

Section: Introductionmentioning

confidence: 99%

“…Indeed, despite expanding literature on benefit-risk assessment [2,3,21,22,31,32], most of the published studies in this area have focused on novel, "one-off" applications of benefit-risk assessment frameworks and/or qBRA methods [31]. Notable exceptions to this include two case studies that describe how benefit-risk assessment frameworks and toolkits have been integrated into the drug development process within two different biopharmaceutical companies [21,22,24,33].…”

Section: Introductionmentioning

confidence: 99%

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

Smith

Til

DiSantostefano

et al. 2020

Ther Innov Regul Sci

Self Cite

View full text Add to dashboard Cite

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.

show abstract

Structured Benefit-Risk Assessment Across the Product Lifecycle: Practical Considerations

Cited by 11 publications

References 13 publications

A survey to assess the current status of structured Benefit-Risk assessment in the global drug and medical device industry

A survey to assess the current status of structured Benefit-Risk assessment in the global drug and medical device industry

A Structured Benefit-Risk Assessment Operating Model for Investigational Medicinal Products in the Pharmaceutical Industry

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

Contact Info

Product

Resources

About