Building trust in real world evidence (RWE): moving transparency in RWE towards the randomized controlled trial standard

References

• US Food & Drug Administration. Framework for FDA’s real-world evidence program 2018 [cited 2023 July 13]. Available from: https://www.fda.gov/media/120060/download.
   Google Scholar
• US Food & Drug Administration. Real-world evidence 2023 [cited 2023 July 20]. Available from: www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence.
   Google Scholar
• US Food & Drug Administration. Considerations for the use of real-world data and real-world evidence to support regulatory decision-making for drug and biological products: guidance for industry 2023 [cited 2023 September 20]. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/considerations-use-real-world-data-and-real-world-evidence-support-regulatory-decision-making-drug.
   Google Scholar
• Arlett P, Kjaer J, Broich K, et al. Real-world evidence in EU medicines regulation: enabling use and establishing value. Clin Pharmacol Ther. 2022 Jan;111(1):21–23. doi: 10.1002/cpt.2479.
   PubMed Web of Science ®Google Scholar
• Nishioka K, Makimura T, Ishiguro A, et al. Evolving acceptance and use of RWE for regulatory decision making on the benefit/risk assessment of a drug in Japan. Clin Pharmacol Ther. 2022 Jan;111(1):35–43. doi: 10.1002/cpt.2410.
   PubMed Web of Science ®Google Scholar
• National Institute for Health and Care Excellence. NICE real-world evidence framework: corporate document [ECD9] 2022 [cited 2023 July 23]. Available from: https://www.nice.org.uk/corporate/ecd9/chapter/overview.
   Google Scholar
• Canadian Agency for Drugs and Technologies in Health. Guidance for reporting real-world evidence 2023 [cited 2023 20 September]. Available from: https://www.cadth.ca/sites/default/files/RWE/MG0020/MG0020-RWE-Guidance-Report-Secured.pdf.
   Google Scholar
• Purpura CA, Garry EM, Honig N, et al. The role of real-world evidence in FDA-approved new drug and biologics license applications. Clin Pharmacol Ther. 2022 Jan;111(1):135–144. doi: 10.1002/cpt.2474.
   PubMed Web of Science ®Google Scholar
• US Food & Drug Administration. FDA roundup: July 21, 2023 [cited 2023 July 28]. Available from: https://www.fda.gov/news-events/press-announcements/fda-roundup-july-21-2023.
   Google Scholar
• Dhruva SS, Zhang S, Chen J, et al. Safety and effectiveness of a catheter with contact force and 6-hole irrigation for ablation of persistent atrial fibrillation in routine clinical practice. JAMA Netw Open. 2022 Aug;5(8):e2227134. doi: 10.1001/jamanetworkopen.2022.27134.
   PubMed Web of Science ®Google Scholar
• Winterstein AG, Ehrenstein V, Brown JS, et al. A road map for peer review of real-world evidence studies on safety and effectiveness of treatments. Diabetes Care. 2023 Aug;46(8):1448–1454. doi: 10.2337/dc22-2037.
   PubMed Web of Science ®Google Scholar
• White R. Building trust in real-world evidence and comparative effectiveness research: the need for transparency. J Comp Eff Res. 2017 Jan;6(1):5–7. doi: 10.2217/cer-2016-0070.
   PubMed Web of Science ®Google Scholar
• Wang SV, Pinheiro S, Hua W, et al. STaRT-RWE: structured template for planning and reporting on the implementation of real world evidence studies. BMJ. 2021 Jan;372:m4856. doi: 10.1136/bmj.m4856.
   PubMed Web of Science ®Google Scholar
• Wang SV, Pottegard A, Crown W, et al. 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 Jan;32(1):44–55. doi: 10.1002/pds.5507.
   PubMed Web of Science ®Google Scholar
• Concato J, Corrigan-Curay J. Real-world evidence – where are we now? N Engl J Med. 2022 May;386(18):1680–1682. doi: 10.1056/NEJMp2200089.
   PubMed Web of Science ®Google Scholar
• US Food & Drug Administration. Oncology Quality, Characterization and Assessment of Real-World Data (QCARD) initiative: facilitating high-quality early RWD study proposals in oncology 2023 [cited 2023 July 27]. Available from: https://www.fda.gov/about-fda/oncology-center-excellence/oncology-quality-characterization-and-assessment-real-world-data-qcard-initiative.
   Google Scholar
• European Network for Health Technology Assessment. REQueST Tool and its vision paper 2019 [cited 2023 July 25]. Available from: https://www.eunethta.eu/request-tool-and-its-vision-paper/.
   Google Scholar
• European Medicines Agency. Data Analysis and Real World Interrogation Network (DARWIN EU) 2023 [cited 2023 July 19]. Available from: https://www.ema.europa.eu/en/about-us/how-we-work/big-data/data-analysis-real-world-interrogation-network-darwin-eu.
   Google Scholar
• Gatto NM, Campbell UB, Rubinstein E, et al. The structured process to identify fit-for-purpose data: a data feasibility assessment framework. Clin Pharmacol Ther. 2022 Jan;111(1):122–134. doi: 10.1002/cpt.2466.
   PubMed Web of Science ®Google Scholar
• Gatto NM, Vititoe SE, Rubinstein E, et al. A structured process to identify fit-for-purpose study design and data to generate valid and transparent real-world evidence for regulatory uses. Clin Pharmacol Ther. 2023 Jun;113(6):1235–1239. doi: 10.1002/cpt.2883.
   PubMed Web of Science ®Google Scholar
• National Institute for Health and Care Excellence. NICE real-world evidence framework Appendix 1 – Data Suitability Assessment Tool (DataSAT) 2022 [cited 2023 July 20]. Available from: https://www.nice.org.uk/corporate/ecd9/chapter/appendix-1-data-suitability-assessment-tool-datasat.
   Google Scholar
• Berger ML, Sox H, Willke RJ, et al. Good practices for real-world data studies of treatment and/or comparative effectiveness: recommendations from the joint ISPOR-ISPE Special Task Force on real-world evidence in health care decision making. Pharmacoepidemiol Drug Saf. 2017 Sep;26(9):1033–1039. doi: 10.1002/pds.4297.
   PubMed Web of Science ®Google Scholar
• Orsini LS, Berger M, Crown W, et al. 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 Sep;23(9):1128–1136. doi: 10.1016/j.jval.2020.04.002.
   PubMed Web of Science ®Google Scholar
• International Coalition of Medicines Regulatory Authorities. ICMRA statement on international collaboration to enable real-world evidence (RWE) for regulatory decision-making 2022 [cited 2023 July 25]. Available from: https://www.icmra.info/drupal/sites/default/files/2022-07/icmra_statement_on_rwe.pdf.
   Google Scholar
• Mahmoud AN, Gad MM, Elgendy AY, et al. Efficacy and safety of aspirin for primary prevention of cardiovascular events: a meta-analysis and trial sequential analysis of randomized controlled trials. Eur Heart J. 2019 Feb;40(7):607–617. doi: 10.1093/eurheartj/ehy813.
   PubMed Web of Science ®Google Scholar
• Wang SV, Verpillat P, Rassen JA, et al. Transparency and reproducibility of observational cohort studies using large healthcare databases. Clin Pharmacol Ther. 2016 Mar;99(3):325–332. doi: 10.1002/cpt.329.
   PubMed Web of Science ®Google Scholar
• Langan SM, Schmidt SA, Wing K, et al. The reporting of studies conducted using observational routinely collected health data statement for pharmacoepidemiology (RECORD-PE). BMJ. 2018 Nov;363:k3532. doi: 10.1136/bmj.k3532.
   PubMed Web of Science ®Google Scholar
• von Elm E, Altman DG, Egger M, et al. The Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) statement: guidelines for reporting observational studies. J Clin Epidemiol. 2008 Apr;61(4):344–349. doi: 10.1016/j.jclinepi.2007.11.008.
   PubMed Web of Science ®Google Scholar
• Dreyer NA, Bryant A, Velentgas P. The GRACE checklist: a validated assessment tool for high quality observational studies of comparative effectiveness. J Manag Care Spec Pharm. 2016 Oct;22(10):1107–1113. doi: 10.18553/jmcp.2016.22.10.1107.
   PubMed Web of Science ®Google Scholar
• Sterne JA, Hernan MA, Reeves BC, et al. ROBINS-I: a tool for assessing risk of bias in non-randomised studies of interventions. BMJ. 2016 Oct;355:i4919. doi: 10.1136/bmj.i4919.
   PubMed Web of Science ®Google Scholar
• Crown W, Dahabreh IJ, Li X, et al. Can observational analyses of routinely collected data emulate randomized trials? Design and feasibility of the observational patient evidence for regulatory approval science and understanding disease project. Value Health. 2023 Feb;26(2):176–184. doi: 10.1016/j.jval.2022.07.003.
   PubMed Web of Science ®Google Scholar
• Merola D, Campbell U, Gautam N, et al. The Aetion coalition to advance real-world evidence through randomized controlled trial emulation initiative: oncology. Clin Pharmacol Ther. 2023 Jun;113(6):1217–1222. doi: 10.1002/cpt.2800.
   PubMed Web of Science ®Google Scholar
• Wang SV, Schneeweiss S; RCT-DUPLICATE Initiative Emulation of randomized clinical trials with nonrandomized database analyses: results of 32 clinical trials. JAMA. 2023 Apr;329(16):1376–1385. doi: 10.1001/jama.2023.4221.
   PubMed Web of Science ®Google Scholar