Multiple Perspectives on the Need for Real-World Evidence to Inform Regulatory and Health Technology Assessment Decision-Making: Scoping Review and Stakeholder Interviews.
Marieke S Jansen, Olaf M Dekkers, Saskia le Cessie, Lotty Hooft, Helga Gardarsdottir, Anthonius de Boer, Rolf H H Groenwold
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引用次数: 0
Abstract
Purpose: Real-world evidence (RWE) is increasingly considered in regulatory and health technology assessment (HTA) decision-making, though perspectives on its relevance may vary. Expanding on a recent review regarding regulatory decisions, this study aimed to identify factors influencing the need for RWE in HTA decision-making, confirm and enrich factors with stakeholder views, and evaluate similarities and differences between regulatory and HTA needs.
Methods: Previous scoping review methodology was used to identify factors influencing the need for RWE in HTA decision-making. Semi-structured interviews with stakeholders were conducted to confirm and enrich literature-derived factors for both regulatory and HTA contexts. Insights from the reviews and interviews were combined to explore similarities and differences in RWE needs across these domains.
Results: The HTA review, featuring 118 articles, revealed two major themes and six subthemes, encompassing 45 factors. The need for RWE depended on (1) questions addressable with RWE, and (2) contextual factors. Stakeholder interviews confirmed literature-derived factors. While contextual factors aligned between regulatory and HTA decision-making, question-related factors partly differed. Unlike the benefit-risk assessment in regulatory decision-making, RWE serves as direct input for the HTA, and involves specific details and a broader scope. Regulators require RWE for orphan status submissions, alternative approval pathways and to evaluate the impact of risk minimization measures, whereas HTA uses RWE to guide comparator selection, evaluate treatment implementation, quality of care and general healthcare impacts.
Conclusion: Contextual factors that influence the need for RWE are similar between regulatory and HTA decision-making, with variations seen in questions addressable with RWE.
期刊介绍：
The aim of Pharmacoepidemiology and Drug Safety is to provide an international forum for the communication and evaluation of data, methods and opinion in the discipline of pharmacoepidemiology. The Journal publishes peer-reviewed reports of original research, invited reviews and a variety of guest editorials and commentaries embracing scientific, medical, statistical, legal and economic aspects of pharmacoepidemiology and post-marketing surveillance of drug safety. Appropriate material in these categories may also be considered for publication as a Brief Report.
Particular areas of interest include:
design, analysis, results, and interpretation of studies looking at the benefit or safety of specific pharmaceuticals, biologics, or medical devices, including studies in pharmacovigilance, postmarketing surveillance, pharmacoeconomics, patient safety, molecular pharmacoepidemiology, or any other study within the broad field of pharmacoepidemiology;
comparative effectiveness research relating to pharmaceuticals, biologics, and medical devices. Comparative effectiveness research is the generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat, and monitor a clinical condition, as these methods are truly used in the real world;
methodologic contributions of relevance to pharmacoepidemiology, whether original contributions, reviews of existing methods, or tutorials for how to apply the methods of pharmacoepidemiology;
assessments of harm versus benefit in drug therapy;
patterns of drug utilization;
relationships between pharmacoepidemiology and the formulation and interpretation of regulatory guidelines;
evaluations of risk management plans and programmes relating to pharmaceuticals, biologics and medical devices.
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