Propensity Score Methodology Application to Drug and Device Safety Studies: Regulatory Perspectives

• Activity Number: 167
• Type: Topic Contributed
• Date/Time: Monday, July 30, 2012 : 10:30 AM to 12:20 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Lilly Yue*+ and Mark S Levenson
• Companies: FDA and FDA
• Keywords: propensity scores ; observational study ; safety evaluation ; regulatory perspective

Abstract

While carefully designed and well-controlled randomized 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 present two examples to elucidate some regulatory considerations on the prospective design and appropriate data analysis of observational studies using propensity score methodology.