E9(R1) statistical principles for clinical trials: addendum : estimands and sensitivity analysis in clinical trials
- Collection:
- Health Policy and Services Research
- Series Title(s):
- Guidance for industry
- Contributor(s):
- United States. Department of Health and Human Services, issuing body. United States. Food and Drug Administration, issuing body. Center for Drug Evaluation and Research (U.S.), issuing body. Center for Biologics Evaluation and Research (U.S.), issuing body.
- Publication:
- Silver Spring, MD : Center for Drug Evaluation and Research, May 2021
- Language(s):
- English
- Format:
- Text
- Subject(s):
- Clinical Trial Protocols as Topic Clinical Trials as Topic Statistics as Topic United States United States. Department of Health and Human Services United States. Food and Drug Administration
- Genre(s):
- Technical Report
- Abstract:
- To properly inform decision-making by pharmaceutical companies, regulators, patients, physicians, and other stakeholders, clear descriptions of the benefits and risks of a treatment (medicine) for a given medical condition should be made available. Without such clarity, there is a concern that the reported treatment effect will be misunderstood. This addendum presents a structured framework to strengthen the dialogue between disciplines involved in the formulation of clinical trial objectives, design, conduct, analysis and interpretation, as well as between sponsor and regulator regarding the treatment effect or effects of interest that a clinical trial should address. Precision in describing a treatment effect of interest is facilitated by constructing the estimand (see Glossary; section III (A.3)) corresponding to a clinical question of interest. Clarity calls for a thoughtful envisioning of intercurrent events (see Glossary; section III.A (A.3.1)) such as discontinuation of assigned treatment, use of an additional or alternative treatment, and terminal events such as death. The description of an estimand should reflect the clinical question of interest in respect to these intercurrent events, and this addendum introduces strategies to reflect different questions of interest that might be posed. The choice of strategies can influence how more conventional attributes of a trial are reflected when describing the clinical question, for example, the treatments, population, or the variable (endpoint) of interest. The statistical analysis of clinical trial data should be aligned to the estimand. This addendum clarifies the role of sensitivity analysis (see Glossary) to explore robustness of conclusions from the main statistical analysis.
- Copyright:
- The National Library of Medicine believes this item to be in the public domain. (More information)
- Extent:
- 1 online resource (1 PDF file (i, 19 pages))
- Illustrations:
- Illustrations
- NLM Unique ID:
- 9918350580906676 (See catalog record)
- Permanent Link:
- http://resource.nlm.nih.gov/9918350580906676