Professor of Biostatistics
Vanderbilt University School of Medicine
Professor of Psychiatry and, by courtesy, of Medicine (Cardiovascular Medicine) and of Biomedical Data Science
Stanford University School of Medicine
Revised July 17, 2017 It is often said that randomized clinical trials (RCTs) are the gold standard for learning about therapeutic effectiveness. This is because the treatment is assigned at random so no variables, measured or unmeasured, will be truly related to treatment assignment.
Randomized clinical trials (RCT) have long been held as the gold standard for generating evidence about the effectiveness of medical and surgical treatments, and for good reason. But I commonly hear clinicians lament that the results of RCTs are not generalizable to medical practice, primarily for two reasons:
Patients in clinical practice are different from those enrolled in RCTs Drug adherence in clinical practice is likely to be lower than that achieved in RCTs, resulting in lower efficacy.