Module 1 2021

Expedited Pathways towards Approval • Various processes in place (guidance)

Guidance “Expedited Programs for Serious Conditions – Drugs and Biologics”: https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ UCM358301.pdf

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Accelerated Approval • Intended to make promising products for life threatening diseases available on the basis of preliminary evidence prior to formal demonstration of patient benefit − Under Subpart H (21CFR314.510) & 21CFR 601.41 • Approval based on a surrogate endpoint or on an effect on a clinical endpoint other than survival or irreversible morbidity − A measurement that is considered likely to predict patient benefit • Considered provisional approval with a written commitment to complete clinical studies that formally demonstrate patient benefit − If studies don’t confirm the initial results, the FDA can withdraw approval − Post-marketing studies should be underway. Sponsor needs to show diligence in completing studies in a timely manner. Status update may be requested to report at Advisory meeting − If confirmatory study shows clinical benefit, FDA will grant ‘regular’ approval

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