Using observational data for personalized medicine when clinical trial evidence is limited

When clinical trial evidence is limited, observational data from large and information-rich health care databases combined with modern methods can be used to learn about the effects of treatments.

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Volume 109, Issue 6, Pages 946–951

Authors:

Boris Gershman, M.D., David P. Guo, M.D., Issa J. Dahabreh, M.D., M.S.

Abstract:

Randomized clinical trials are considered the preferred approach for comparing the effects of treatments, yet data from high-quality clinical trials are often unavailable and many clinical decisions are made on the basis of evidence from observational studies. Using clinical examples about the management of infertility, we discuss how we can use observational data from large and information-rich health-care databases combined with modern epidemiological and statistical methods to learn about the effects of interventions when clinical trial evidence is unavailable or not applicable to the clinically relevant target population. When trial evidence is unavailable, we can conduct observational analyses emulating the hypothetical pragmatic target trials that would address the clinical questions of interest. When trial evidence is available but not applicable to the clinically relevant target population, we can transport inferences from trial participants to the target population using the trial data and a sample of observational data from the target population. Clinical trial emulations and transportability analyses can be coupled with methods for examining heterogeneity of treatment effects, providing a path toward personalized medicine.


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Fertility and Sterility

Editorial Office, American Society for Reproductive Medicine

Fertility and Sterility® is an international journal for obstetricians, gynecologists, reproductive endocrinologists, urologists, basic scientists and others who treat and investigate problems of infertility and human reproductive disorders. The journal publishes juried original scientific articles in clinical and laboratory research relevant to reproductive endocrinology, urology, andrology, physiology, immunology, genetics, contraception, and menopause. Fertility and Sterility® encourages and supports meaningful basic and clinical research, and facilitates and promotes excellence in professional education, in the field of reproductive medicine.

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