Journal
BMJ EVIDENCE-BASED MEDICINE
Volume 27, Issue 2, Pages 109-119Publisher
BMJ PUBLISHING GROUP
DOI: 10.1136/bmjebm-2020-111493
Keywords
evidence-based practice; health care economics and organisations; health services research
Categories
Ask authors/readers for more resources
This study developed a guidance using a mixed methods approach to direct researchers and healthcare decision-makers on when and how to combine evidence from non-randomised studies (NRS) and randomised controlled trials (RCTs) to enhance transparency and confidence. The framework consists of seven steps and offers recommendations on statistical approaches based on different healthcare decision-making scenarios.
Introduction: High-quality randomised controlled trials (RCTs) provide the most reliable evidence on the comparative efficacy of new medicines. However, non-randomised studies (NRS) are increasingly recognised as a source of insights into the real-world performance of novel therapeutic products, particularly when traditional RCTs are impractical or lack generalisability. This means there is a growing need for synthesising evidence from RCTs and NRS in healthcare decision making, particularly given recent developments such as innovative study designs, digital technologies and linked databases across countries. Crucially, however, no formal framework exists to guide the integration of these data types. Objectives and Methods: To address this gap, we used a mixed methods approach (review of existing guidance, methodological papers, Delphi survey) to develop guidance for researchers and healthcare decision-makers on when and how to best combine evidence from NRS and RCTs to improve transparency and build confidence in the resulting summary effect estimates. Results: Our framework comprises seven steps on guiding the integration and interpretation of evidence from NRS and RCTs and we offer recommendations on the most appropriate statistical approaches based on three main analytical scenarios in healthcare decision making (specifically, 'high-bar evidence' when RCTs are the preferred source of evidence, 'medium,' and 'low' when NRS is the main source of inference). Conclusion: Our framework augments existing guidance on assessing the quality of NRS and their compatibility with RCTs for evidence synthesis, while also highlighting potential challenges in implementing it. This manuscript received endorsement from the International Society for Pharmacoepidemiology.
Authors
I am an author on this paper
Click your name to claim this paper and add it to your profile.
Reviews
Recommended
No Data Available