Synthetic and External Controls in Clinical Trials - A Primer for Researchers
Top Cited Papers
Open Access
- 1 January 2020
- journal article
- review article
- Published by Taylor & Francis Ltd in Clinical Epidemiology
- Vol. 12, 457-467
- https://doi.org/10.2147/CLEP.S242097
Abstract
There has been a rapid expansion in the use of non-randomized evidence in the regulatory approval of treatments globally. An emerging set of methodologies have been utilized to provide greater insight into external control data used for these purposes, collectively known as synthetic control methods. Through this paper, we provide the reader with a set of key questions to help assess the quality of literature publications utilizing synthetic control methodologies. Common challenges and real-life examples of synthetic controls are provided throughout, alongside a critical appraisal framework with which to assess future publications.This publication has 28 references indexed in Scilit:
- Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EUEuropean Journal of Clinical Pharmacology, 2012
- State-Transition Modeling: A Report of the ISPOR-SMDM Modeling Good Research Practices Task Force-3Value in Health, 2012
- The prognostic value of baseline CD4+ cell count beyond 6 months of antiretroviral therapy in HIV-positive patients in a resource-limited settingAIDS, 2012
- GRADE guidelines: 9. Rating up the quality of evidenceJournal of Clinical Epidemiology, 2011
- GRADE guidelines: 1. Introduction—GRADE evidence profiles and summary of findings tablesJournal of Clinical Epidemiology, 2011
- New response evaluation criteria in solid tumours: Revised RECIST guideline (version 1.1)European Journal of Cancer, 2009
- Standard of Care Versus Standards of Care in Oncology: A Not So Subtle DistinctionJournal of Oncology Practice, 2007
- Stratification and weighting via the propensity score in estimation of causal treatment effects: a comparative studyStatistics in Medicine, 2004
- Placebo-controlled trials: good science or medical neglect?Western Journal of Medicine, 2000
- New Guidelines to Evaluate the Response to Treatment in Solid TumorsJNCI Journal of the National Cancer Institute, 2000