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Real-World Evidence to Reinforce Clinical Trial Evidence in Health Technology Assessment: A Critical Review of Real-World Evidence Requirements from Seven Countries and Recommendations to Improve Acceptance.

作者信息

Thokagevistk Katia, Coppo Céline, Rey Laetitia, Carelli Amanda, Díez Veronica, Vaselenak Sarah, Oliveira Liana, Patel Ajay, Sicari Emilia, Ramos Teresa, Schach Susanne, Schirghuber Erika, Simpson Alex, Choquet Remy, Le Lay Katell

机构信息

Roche, 4 Cr de l'Île Seguin, 92100 Boulogne-Billancourt, France.

IQVIA, 17 bis Tsse. des Reflets, 92400 Courbevoie, France.

出版信息

J Mark Access Health Policy. 2024 May 20;12(2):105-117. doi: 10.3390/jmahp12020009. eCollection 2024 Jun.


DOI:10.3390/jmahp12020009
PMID:38808313
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11130860/
Abstract

BACKGROUND: Real-world evidence (RWE) can reinforce clinical trial evidence in health technology assessment (HTA). OBJECTIVES: Review HTA bodies' (HTAbs) requirements for RWE, real uses, and acceptance across seven countries (Brazil, Canada, France, Germany, Italy, Spain, and the United Kingdom) and outline recommendations that may improve acceptance of RWE in efficacy/effectiveness assessments and appraisals processes. METHODS: RWE requirements were summarized based on HTAbs' guidelines. Acceptance by HTAbs was evaluated based on industry experience and case studies. RESULTS: As of June 2022, RWE methodological guidelines were in place in three of the seven countries. HTAbs typically requested analyses based on local data sources, but the preferred study design and data sources differed. HTAbs had individual submission, assessment, and appraisal processes; some allowed early meetings for the protocol and/or results validation, though few involved external experts or medical societies to provide input to assessment and appraisal. The extent of submission, assessment, and appraisal requirements did not necessarily reflect the degree of acceptance. CONCLUSION: All the countries reviewed face common challenges regarding the use of RWE. Our proposals address the need to facilitate collaboration and communication with industry and regulatory agencies and the need for specific guidelines describing RWE design and criteria of acceptance throughout the assessment and appraisal processes.

摘要

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本文引用的文献

[1]
Across-Country Variations of Real-World Data and Evidence for Drugs: A 5-European-Country Study.

Value Health. 2023-4

[2]
HARmonized Protocol Template to Enhance Reproducibility of hypothesis evaluating real-world evidence studies on treatment effects: A good practices report of a joint ISPE/ISPOR task force.

Pharmacoepidemiol Drug Saf. 2023-1

[3]
Comparing clinical trial population representativeness to real-world populations: an external validity analysis encompassing 43 895 trials and 5 685 738 individuals across 989 unique drugs and 286 conditions in England.

Lancet Healthy Longev. 2022-10

[4]
Reproducibility of real-world evidence studies using clinical practice data to inform regulatory and coverage decisions.

Nat Commun. 2022-8-31

[5]
Marketing Authorization Applications Made to the European Medicines Agency in 2018-2019: What was the Contribution of Real-World Evidence?

Clin Pharmacol Ther. 2022-1

[6]
The Past, Present, and Future of Economic Evaluations of Precision Medicine at the Committee for Economic Analyses of the Canadian Cancer Trials Group.

Curr Oncol. 2021-9-21

[7]
Use of External Comparators for Health Technology Assessment Submissions Based on Single-Arm Trials.

Value Health. 2021-8

[8]
Common Problems, Common Data Model Solutions: Evidence Generation for Health Technology Assessment.

Pharmacoeconomics. 2021-3

[9]
Improving Transparency to Build Trust in Real-World Secondary Data Studies for Hypothesis Testing-Why, What, and How: Recommendations and a Road Map from the Real-World Evidence Transparency Initiative.

Value Health. 2020-9

[10]
Real-world evidence use in assessments of cancer drugs by NICE.

Int J Technol Assess Health Care. 2020-7-10

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