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What types of clinical studies should be included in your FDA submission?
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About the Author
Proxima CRO Team
Isabella Schmitt, RAC
Director of Regulatory Affairs

Prior to joining Proxima, Isabella served as the Senior Regulatory & Quality Manager at a medical device company, where she outlined the regulatory strategy & put together design controls & design history documentation. She was the Dir. of CMC & Quality at a biopharmaceutical company, where she oversaw all manufacturing and analytical processes and timelines and ensured CMC regulatory strategy was sufficient for filings in Europe and the US.

The following are the types of adequate and well-controlled studies that should usually be included in the "Clinical Studies" section.

(1) Clinical studies that provide primary support for effectiveness

(2) Clinical studies that provide other important information about a drug’s effectiveness not furnished by the studies that provide primary support for effectiveness, such as:

  • Studies that suggest differential effects in population subsets (e.g. women vs. men, presence or absence of concomitant illness or medications)
  • Studies that suggest lack of effectiveness in a clinical situation or lack of effect on a particular endpoint where the drug might have been expected to work
  • Studies that provide information relevant to dose selection or adjustment (e.g. dose-response studies or studies in non-responders to a particular dose)
  • Studies that provide information about the nature and size of the treatment effect, particularly where the effect is small

(3) Clinical studies that prospectively evaluate an important safety endpoint

The following are the types of studies that should usually not be included in the "Clinical Studies" section, unless they also meet one of the factors in above. If an exception is made, the limitations of the study and the reasons for inclusion should be stated.

  • Clinical studies with results that imply effectiveness for an unapproved indication, use, or dosing regimen
  • Active control clinical studies that imply comparative effectiveness or safety claims not supported by substantial evidence
  • Studies that are not adequate and well-controlled within the meaning of 21 CFR 314.126

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