Using Mixed Methods to Evaluate Risk Minimisation Programs in Europe and the USA: An Innovative Blueprint.

IF 4 2区医学 Q1 PHARMACOLOGY & PHARMACY

Drug Safety Pub Date : 2025-07-01 Epub Date: 2025-03-12 DOI:10.1007/s40264-025-01533-w

Meredith Y Smith, Rachel Davis, Priya Bahri, Delphine Saragoussi, Viviana Nguyen, Gita A Toyserkani, Alison Hamilton

{"title":"Using Mixed Methods to Evaluate Risk Minimisation Programs in Europe and the USA: An Innovative Blueprint.","authors":"Meredith Y Smith, Rachel Davis, Priya Bahri, Delphine Saragoussi, Viviana Nguyen, Gita A Toyserkani, Alison Hamilton","doi":"10.1007/s40264-025-01533-w","DOIUrl":null,"url":null,"abstract":"Background: Significant methodological shortcomings have been documented to date in risk minimisation program evaluations for medicinal products, including overreliance on survey methods alone. Recently updated guidances from the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) recommend the use of frameworks and mixed methods designs to improve the rigor of these assessments.Objective: The purpose of this paper was to exemplify how a mixed methods approach, guided by an implementation science framework, can be used to design the evaluation of a risk minimisation program.Methods: We selected the Practical, Robust, Implementation and Sustainability Model (PRISM) as the implementation science framework to guide our mixed methods approach. PRISM provides a comprehensive and systematic approach to measuring the key domains relevant to the implementation and outcomes of a risk minimisation program. We mapped the PRISM domains to the evaluation dimensions described in the EMA and FDA guidances. We then specified a mixed methods evaluation design and data collection methods using a fictitious risk minimisation program as a case study for illustrative purposes.Results: On the basis of our case study, we developed quantitative and qualitative measures, including specific items for surveys and interviews, for both formative and summative evaluations. For both the formative and summative evaluations, measures focussed on assessing (1) contextual factors that could affect program implementation and impact and (2) outcomes including implementability and acceptability as well as degree of program reach, adoption, implementation, effectiveness and maintenance.Conclusions: Mixed methods, guided by a well-established implementation science framework, can be applied to ensure comprehensive formative and summative evaluations that provide fit-for-purpose information that may inform regulatory decision-making.","PeriodicalId":11382,"journal":{"name":"Drug Safety","volume":" ","pages":"821-838"},"PeriodicalIF":4.0000,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12174294/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Drug Safety","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s40264-025-01533-w","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/3/12 0:00:00","PubModel":"Epub","JCR":"Q1","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}

引用次数: 0

Abstract

Background: Significant methodological shortcomings have been documented to date in risk minimisation program evaluations for medicinal products, including overreliance on survey methods alone. Recently updated guidances from the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) recommend the use of frameworks and mixed methods designs to improve the rigor of these assessments.

Objective: The purpose of this paper was to exemplify how a mixed methods approach, guided by an implementation science framework, can be used to design the evaluation of a risk minimisation program.

Methods: We selected the Practical, Robust, Implementation and Sustainability Model (PRISM) as the implementation science framework to guide our mixed methods approach. PRISM provides a comprehensive and systematic approach to measuring the key domains relevant to the implementation and outcomes of a risk minimisation program. We mapped the PRISM domains to the evaluation dimensions described in the EMA and FDA guidances. We then specified a mixed methods evaluation design and data collection methods using a fictitious risk minimisation program as a case study for illustrative purposes.

Results: On the basis of our case study, we developed quantitative and qualitative measures, including specific items for surveys and interviews, for both formative and summative evaluations. For both the formative and summative evaluations, measures focussed on assessing (1) contextual factors that could affect program implementation and impact and (2) outcomes including implementability and acceptability as well as degree of program reach, adoption, implementation, effectiveness and maintenance.

Conclusions: Mixed methods, guided by a well-established implementation science framework, can be applied to ensure comprehensive formative and summative evaluations that provide fit-for-purpose information that may inform regulatory decision-making.

查看原文本刊更多论文

使用混合方法评估欧洲和美国的风险最小化项目：一个创新蓝图。

背景：迄今为止，在药品风险最小化规划评估中，已记录了重大的方法学缺陷，包括过度依赖单独的调查方法。美国食品和药物管理局（FDA）和欧洲药品管理局（EMA）最近更新的指南建议使用框架和混合方法设计来提高这些评估的严谨性。目的：本文的目的是举例说明如何在实施科学框架的指导下使用混合方法方法来设计风险最小化计划的评估。方法：我们选择实用、稳健、实施和可持续性模型（PRISM）作为实施科学框架来指导我们的混合方法方法。PRISM提供了一种全面和系统的方法来衡量与风险最小化计划的实施和结果相关的关键领域。我们将PRISM域映射到EMA和FDA指南中描述的评估维度。然后，我们指定了混合方法评估设计和数据收集方法，使用虚构的风险最小化程序作为案例研究，以说明目的。结果：在我们的案例研究的基础上，我们制定了定量和定性的措施，包括调查和访谈的具体项目，用于形成性和总结性评估。对于形成性评估和总结性评估，测量方法集中于评估(1)可能影响计划实施和影响的上下文因素和(2)结果，包括可实施性和可接受性以及计划的范围、采用、实施、有效性和维护程度。结论：在完善的实施科学框架的指导下，可以采用混合方法来确保全面的形成性和总结性评估，从而提供可能为监管决策提供信息的符合目的的信息。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

Drug Safety 医学-毒理学

CiteScore

7.60

自引率

7.10%

发文量

112

审稿时长

6-12 weeks

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. Additional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in Drug Safety Drugs may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances.