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GUIDANCE DOCUMENT

E9(R1) Statistical Principles for Clinical Trials: Addendum: Estimands and Sensitivity Analysis in Clinical Trials October 2017

Draft

Not for implementation. Contains non-binding recommendations.

This guidance is being distributed for comment purposes only.

E9(R1) Statistical Principles for Clinical Trials: Addendum: Estimands and Sensitivity Analysis in Clinical Trials

Issued by:
Guidance Issuing Office
Center for Drug Evaluation and Research
Center for Biologics Evaluation and Research

To properly inform the choices that are made by patients and prescribing physicians, clear 2 descriptions of the effects of a medicine should be available. These descriptions are complicated by the different ways in which each individual patient responds to treatment.  Some subjects will tolerate a medicine and adhere to its administration schedule, others will not. Some subjects will require changes in dose of concomitant medication or administration 6 of additional medication, others will not. Multiple ways to quantify treatment effects can be envisaged based on how to take into account, for example, tolerability, adherence and  whether or not additional medication is required. Without a precise understanding of the treatment effect that is being described, there is a risk that its magnitude and meaningfulness will be misunderstood.


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Submit comments on this guidance document electronically via docket ID: FDA-2013-S-0610 - Specific Electronic Submissions Intended For FDA's Dockets Management Staff (i.e., Citizen Petitions, Draft Proposed Guidance Documents, Variances, and other administrative record submissions)

If unable to submit comments online, please mail written comments to:

Division of Dockets Management (HFA- 305)
Food and Drug Administration
5630 Fishers Lane, Rm. 1061
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All comments should be identified with the title of the guidance.