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Open Access 01-12-2020 | Commentary

Can real-world data really replace randomised clinical trials?

Authors: Sreeram V. Ramagopalan, Alex Simpson, Cormac Sammon

Published in: BMC Medicine | Issue 1/2020

Excerpt

Classically, randomised controlled trials (RCTs) are considered the gold standard for demonstrating product efficacy for the regulatory approval of medicines. However, as personalised medicine becomes increasingly common, patient recruitment into RCTs is affected and – sometimes – it is not possible to include a control arm [1]. …

Moscow JA, Fojo T, Schilsky RL. The evidence framework for precision cancer medicine. Nat Rev Clin Oncol. 2018;15:183.CrossRef

McDonald L, Lambrelli D, Wasiak R, Ramagopalan SV. Real-world data in the United Kingdom: opportunities and challenges. BMC Med. 2016;14:97.CrossRef

Eichler HG, Koenig F, Arlett P, Enzmann H, Humphreys A, Pétavy F, et al. Are novel, nonrandomised analytic methods fit for decision-making? The need for prospective, controlled, and transparent validation. Clin Pharmacol Therapeut. 2019. https://doi.org/10.1002/cpt.1638.

Cave A, Kurz X, Arlett P. Real-world data for regulatory decision making: challenges and possible solutions for Europe. Clin Pharmacol Ther. 2019;106:36.CrossRef

Anglemyer A, Horvath HT, Bero L. Healthcare outcomes assessed with observational study designs compared with those assessed in randomized trials. Cochrane Database Syst Rev. 2014;4:MR000034.

Hemkens LG, Contopoulos-Ioannidis DG, Ioannidis JP. Agreement of treatment effects for mortality from routinely collected data and subsequent randomized trials: meta-epidemiological survey. BMJ. 2016;352:i493.CrossRef

Dickerman BA, García-Albéniz X, Logan RW, Denaxas S, Hernán MA. Avoidable flaws in observational analyses: an application to statins and cancer. Nat Med. 2019:1–6.

Carrigan G, Whipple S, Capra WB, Taylor MD, Brown JS, Lu M, et al. Using electronic health records to derive control arms for early phase single-arm lung cancer trials: proof-of-concept in randomized controlled trials. Clin Pharmacol Ther. 2019. https://doi.org/10.1002/cpt.1586.CrossRef

RCT DUPLICATE. Projects. Effectiveness research with Real World Data to support FDA’s regulatory decision making: A Real World Evidence demonstration project. https://www.rctduplicate.org/projects.html. Accessed 21 Nov 2019.

Title: Can real-world data really replace randomised clinical trials?
Authors: Sreeram V. Ramagopalan
Alex Simpson
Cormac Sammon
Publication date: 01-12-2020
Publisher: BioMed Central
Published in: BMC Medicine / Issue 1/2020
Electronic ISSN: 1741-7015
DOI: https://doi.org/10.1186/s12916-019-1481-8

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Can real-world data really replace randomised clinical trials?

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Keynote webinar | Spotlight on medication adherence

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