About the Author
Proxima CRO Team
Isabella Schmitt, MBA, RAC
Director of Regulatory Affairs
Ms. Schmitt has also served in additional regulatory affairs and clinical research roles in which she contributed to multiple regulatory submissions and clinical affairs projects across a wide range of indications.

The "Clinical Studies" section should present those endpoints that establish the effectiveness of the drug or show the limitations of effectiveness. This includes endpoints the Agency has accepted as evidence of effectiveness, or closely related endpoints that may be more easily understood. When it would be informative, the "Clinical Studies" section can also discuss other endpoints shown to be affected by the drug and endpoints that might have been expected to be influenced by the drug, but were not.

  • Composite Endpoints: In general, the results for all components of a composite endpoint should be presented. Presentation of all components reveals which components are driving the result and which components may be unaffected, or even adversely affected, by treatment with the drug. When there is a range of effects on the components of a composite endpoint, selectively presenting only a single component of the composite endpoint, or presenting only the change in the composite endpoint, can be misleading. In most cases, discussion of a component of a composite endpoint should be only descriptive (i.e. not be presented with statistical analyses) unless the component has been assessed as a separate endpoint with a prospectively defined hypothesis and analysis plan.
  • Primary and Secondary Endpoints: The terms primary endpoint and secondary endpoint are used so variably that they are rarely helpful. The appropriate inquiry is whether there is a well-documented, statistically and clinically meaningful effect on a prospectively defined endpoint, not whether the endpoint was identified as primary or secondary.
  • Closely Related Endpoints: If two or more endpoints are closely related and convey essentially the same information, only one should generally be presented.

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