This is Revision 1 to the May 1998 guidance "Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products", and is applicable to new drug applications (NDAs), biologics license applications (BLAs), or supplements to NDAs or BLAs that require the demonstration of substantial evidence of effectiveness".
The Introduction notes:
"This guidance highlights that there are various ways to provide substantial evidence and emphasizes the many factors that can impact the strength of evidence of effectiveness for a drug. These factors include important elements of the design, conduct, and analysis of the adequate and well-controlled clinical investigation(s), the clinical and statistical persuasiveness of results, and the characteristics of the overall development program. Whether sponsors have demonstrated substantial evidence will depend on the strength of the evidence provided."
But then immediately notes that substantial evidence is not sufficient:
"The finding of substantial evidence of effectiveness is necessary but not sufficient for FDA approval. The approval decision also requires a determination that the drug is safe for the intended use and that the benefits outweigh the risks. As such, there must be sufficient safety data for FDA to consider the drug's safety profile. As all drugs have adverse effects, evaluating whether a drug is safe involves determining whether the benefits of the drug outweigh its risks under the conditions of use defined in labeling. Uncertainties about benefits and risks are also considered when making an approval determination; a drug with greater risks may require a greater magnitude and certainty of benefit to support approval. This benefit-risk assessment and other determinations necessary for approval are outside the scope of this guidance."
Along with the guidance on demonstrating substantial evidence of effectiveness, the draft includes a section on "Regulatory Flexibility" before closing on the other primary factor "Safety Considerations".
You can download the full document here:
Demonstrating Substantial Evidence of Effectiveness for Drugs
| U.S. Food and Drug Administration |
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| Demonstrating Substantial Evidence of Effectiveness for Drugs |
| Draft Guidance for Industry: Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products |
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#fda #SubstantialEvidence #RevisionDraft
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Mark Arnold Ph.D., FAAPS
Westampton, NJ
[email protected]Bioanalytical Solution Integration
LinkedIn:
https://www.linkedin.com/in/markearnoldphd/Website & Blog: Bioanalysis & Biomarkers <bioanalysisandbiomarkers.blogspot.com>
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