Dataset Information

Quantitative Benefit-Risk Assessment: State of the Practice Within Industry.

ABSTRACT:

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.

SUBMITTER: Smith MY

PROVIDER: S-EPMC7864811 | biostudies-literature | 2021 Mar

REPOSITORIES: biostudies-literature

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Publications

Quantitative Benefit-Risk Assessment: State of the Practice Within Industry.

Smith Meredith Y MY van Til Janine J DiSantostefano Rachael L RL Hauber A Brett AB Marsh Kevin K

Therapeutic innovation & regulatory science 20201027 2

<h4>Background</h4>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.<h4>Methods</h4>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 ...[more]

PMID: 33111177

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Project description:IntroductionUnderstanding the balance between the benefits and risks of vaccination is essential to ensure informed and adequate public health decision making. Quantitative benefit-risk models (qBRm) represent useful tools to help decision makers with supporting benefit-risk assessment throughout the lifecycle of a medical product. However, few initiatives have been launched to harmonise qBRm approaches, specifically for vaccines.ObjectivesThe aim of this paper was to identify publications about qBRm applied to vaccines through a systematic literature review, and to describe their characteristics.MethodsMedline, Scopus and Institute for Scientific Information Web of Knowledge databases were searched to identify articles in English, published from database inceptions up to December 2019. The search strategy included the combination of three key concepts: 'benefit-risk', 'modelling' and 'vaccines'. Data extracted included the modelling context and the methodological approaches used.ResultsOf 3172 publications screened, 48 original publications were included. Most of the selected studies were published over the past decade and focused on rotavirus (15), dengue (10) and influenza (6) vaccines. The majority (30) of studies reported analyses related to high-income countries. The methodology of the studies differed, particularly in modelling techniques, benefit-risk measures, and sensitivity analyses. The present work also pointed out a high level of variability in the quality of reporting across studies, with particular regard to input parameters and methodological approaches.ConclusionsThis review provides an extensive list of qBRm applied to vaccines. Discrepancies across studies were identified during our review. While the number of published qBRm studies is increasing, no reporting guidance for qBRm applied to vaccines is currently available. This may affect decision makers' confidence in the results and their benefit-risk assessment(s); therefore, the development of such reporting guidance is highly needed.

Project description:BACKGROUND:Health risks linked with dioxin in fish remain a complex policy issue. Fatty Baltic fish contain persistent pollutants, but they are otherwise healthy food. We studied the health benefits and risks associated with Baltic herring and salmon in four countries to identify critical uncertainties and to facilitate an evidence-based discussion. METHODS:We performed an online survey investigating consumers' fish consumption and its motivation in Denmark, Estonia, Finland, and Sweden. Dioxin and methylmercury concentrations were estimated based on Finnish studies. Exposure-response functions for several health endpoints were evaluated and quantified based on the scientific literature. We also quantified the infertility risk of men based on a recent European risk assessment estimating childhood dioxin exposure and its effect on sperm concentration later in life. RESULTS:Baltic herring and salmon contain omega-3 fatty acids and vitamin D, and the beneficial impact of these fishes on cardiovascular diseases, mortality, and the risk of depression and cancer clearly outweighs risks of dioxins and methylmercury in people older than 45?years of age and in young men. Young women may expose their children to pollutants during pregnancy and breast feeding. This study suggests that even in this critical subgroup, the risks are small and the health benefits are greater than or at least similar to the health risks. Value of information analysis demonstrated that the remaining scientific uncertainties are not large. In contrast, there are several critical uncertainties that are inherently value judgements, such as whether exceeding the tolerable weekly intake is an adverse outcome as such; and whether or not subgroup-specific restrictions are problematic. CONCLUSIONS:The potential health risks attributable to dioxins in Baltic fish have more than halved in the past 10 years. The new risk assessment issued by the European Food Safety Authority clearly increases the fraction of the population exceeding the tolerable dioxin intake, but nonetheless, quantitative estimates of net health impacts change only marginally. Increased use of small herring (which have less pollutants) is a no-regret option. A more relevant value-based policy discussion rather than research is needed to clarify official recommendations related to dioxins in fish.

Dataset Information

Quantitative Benefit-Risk Assessment: State of the Practice Within Industry.

Background

Methods

Results

Conclusion

Publications

Quantitative Benefit-Risk Assessment: State of the Practice Within Industry.

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