UCL Discovery
UCL home » Library Services » Electronic resources » UCL Discovery

Acceptability of using Real-World Data to Estimate Relative Treatment Effects in Health Technology Assessments: Barriers and Future Steps

Gomes, Manuel; Turner, Alex J; Sammon, Cormac; Dawoud, Dalia; Ramagopalan, Sreeram; Simpson, Alex; Siebert, Uwe; (2024) Acceptability of using Real-World Data to Estimate Relative Treatment Effects in Health Technology Assessments: Barriers and Future Steps. Value Health 10.1016/j.jval.2024.01.020. (In press).

[thumbnail of 1-s2.0-S1098301524000706-main.pdf] Text
1-s2.0-S1098301524000706-main.pdf - Accepted Version
Access restricted to UCL open access staff until 29 August 2024.

Download (2MB)

Abstract

OBJECTIVES: Evidence about the comparative effects of new treatments is typically collected in randomized controlled trials (RCTs). In some instances, RCTs are not possible, or their value is limited by an inability to capture treatment effects over the longer-term or in all relevant population subgroups. In these cases, non-randomized studies (NRS) using real-world data (RWD) are increasingly used to complement trial evidence on treatment effects for health technology assessment (HTA). However, there have been concerns over a lack of acceptability of this evidence by HTA agencies. This paper aims to identify barriers to the acceptance of NRS and steps that may facilitate increases in the acceptability of NRS in the future. METHODS: Opinions of the authorship team based on their experience in real-world evidence research in academic, HTA, and industry settings, supported by a critical assessment of existing studies. RESULTS: Barriers were identified that are applicable to key stakeholder groups including HTA agencies (e.g., the lack of comprehensive methodological guidelines for using RWD), evidence-generators (e.g., avoidable deviations from best practices), and external stakeholders (e.g., data controllers providing timely access to high-quality RWD). Future steps that may facilitate future acceptability of NRS include improvements in the quality, integration, and accessibility of RWD, wider use of demonstration projects to highlight the value and applicability of non-randomized designs, living and more detailed HTA guidelines, and improvements in HTA infrastructure relating to RWD. CONCLUSION: NRS can represent a crucial source of evidence on treatment effects for use in HTA when RCT evidence is limited.

Type: Article
Title: Acceptability of using Real-World Data to Estimate Relative Treatment Effects in Health Technology Assessments: Barriers and Future Steps
Location: United States
DOI: 10.1016/j.jval.2024.01.020
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: Real-world data, causal inference, comparative effectiveness, health technology assessment, non-randomised studies
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Institute of Epidemiology and Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Institute of Epidemiology and Health > Applied Health Research
URI: https://discovery.ucl.ac.uk/id/eprint/10188023
Downloads since deposit
1Download
Download activity - last month
Download activity - last 12 months
Downloads by country - last 12 months

Archive Staff Only

View Item View Item