Randomized controlled trials (RCTs) are widely considered the gold standard for inference on the comparative effects of 2 or more treatment strategies. Randomization is the most powerful technique available in causal inference, and the controlled setting of carefully designed experiments is a necessity in evaluating most treatments. Still, as currently designed, conducted, and analyzed, clinical trials are not without limitations and might be susceptible to some of the biases present in observational studies (1, 2). Strict inclusion and exclusion criteria, as well as trials often being conducted “in parallel” with regular clinical care, have led to concerns that the enrolled ...
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Author, Article, and Disclosure Information
Duke University School of Medicine, Durham, North Carolina (M.J.P.)
Duke Clinical Research Institute, Durham, North Carolina (F.W.R.)
Boston University, Boston, Massachusetts (R.B.D.)
Disclosures: Disclosures can be viewed at www.acponline.org/authors/icmje/ConflictOfInterestForms.do?msNum=M18-1093.
Corresponding Author: Michael J. Pencina, PhD, Duke University School of Medicine, 2400 Pratt Street, Duke Box 3850, Durham, NC 27710; e-mail, michael.
Current Author Addresses: Dr. Pencina: Duke University School of Medicine, 2400 Pratt Street, Duke Box 3850, Durham, NC 27710.
Dr. Rockhold: Duke Clinical Research Institute, 2400 Pratt Street, Duke Box 3850, Durham, NC 27710.
Dr. D'Agostino: Boston University, 111 Cummington Mall, Boston, MA 02215.
Author Contributions: Conception and design: M.J. Pencina, F.W. Rockhold, R.B. D'Agostino.
Analysis and interpretation of the data: M.J. Pencina, F.W. Rockhold, R.B. D'Agostino.
Drafting of the article: M.J. Pencina.
Critical revision for important intellectual content: F.W. Rockhold, R.B. D'Agostino.
Final approval of the article: M.J. Pencina, F.W. Rockhold, R.B. D'Agostino.
Statistical expertise: M.J. Pencina, F.W. Rockhold, R.B. D'Agostino.
This article was published at Annals.org on 24 July 2018.
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