Module 1 2021

Other Considerations: Animal Rule

• Human clinical trials are not ethical or feasible, approval based on nonclinical evidence of efficacy − Biodefense − Poisoning antidotes • A type of surrogate endpoint − Requires postmarketing studies to confirm efficacy observed in animals

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General Strategic Considerations

• Specific wording for the indication: Claims MUST be derived from well controlled studies and supported by primary endpoints • Generally required to have conducted at least two (2) adequate and well- controlled studies to support proposed indication, each convincing on its own, to establish effectiveness. − Single study is a review issue; the study must be well designed, well conducted, internally consistent and provide persuasive efficacy findings − Studies do not have to be identical in design • ICH Guidelines versus FDA Guidances • Standards of medical practice (e.g. US versus RoW) • Placebo versus active comparators • Ethnicity (US population versus others) • Timing and use of FDA meetings/interactions to optimize drug development and successful reviews (Pre-IND, EOP 1 / 2, SPA)

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