Regulatory Issues of Propensity Score Methodology Application to Drug and Device Safety Studies

Abstract

While randomized, well-controlled, clinical trials have been viewed as the gold standard in the evaluation of medical products, including drugs, biological products, and medical devices, it is not uncommon for safety assessment to be performed using observational studies, for ethical or practical reasons. In observational studies, various biases could be introduced in every stage and aspect of study, and consequently the resulting statistical inference may carry a lower level of scientific assurance, compared to randomized trials. To ensure the objectivity of study design and interpretability of the results, it is critical to address the challenges of such studies. In this paper, we share regulatory considerations on the prospective design of observational studies to address safety issues using propensity score methodology.

Key Words:

• Devices
• Drugs
• Observational study
• Propensity scores
• Safety evaluation

ACKNOWLEDGMENTS

The authors thank Drs. Greg Campbell, Aloka Chakravarty, and Bradley McEvoy for their valuable comments during the preparation of this article. The authors are also grateful to the referees for their great suggestions and comments, which helped to improve the article.

Notes

Notes. CABG, coronary artery bypass graft surgery; CPB, cardiopulmonary bypass.

Note. PS, propensity score; Tran., tranexamic.

This article not subject to US copyright law.

The co-authors made equal contributions. No official support or endorsement by the Food and Drug Administration of this presentation is intended or should be inferred. The views presented in this article do not necessarily reflect those of the Food and Drug Administration.