Module 1 2021

Breakthrough Designation

• Program since 2012 with final guidance 2014 with aim: – to expedite the development and review of drugs intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s)

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Other considerations: Orphan Drugs • To treat a rare disease or condition which affects fewer than 200,000 people nationwide (e.g., cystic fibrosis, Lou Gehrig’s, Tourette’s syndrome, certain cancers) • Congress passed the Orphan Drug Act (ODA) in 1983 − Applies to drugs and biologics − Provides financial incentives, including tax credits for cost of clinical research − Seven years exclusivity if approved first (from approval of NDA/BLA) − FDA Assistance with Protocol Development, through Office of Orphan Products Development • Application for Orphan Drug Designation can be filed at anytime during the drug development process (60 days review) – joint submission with EMA • For devices: Humanitarian Use Device (HUD) − For treatment or diagnosing a disease or condition that affects or is manifested in not more than 8,000 individuals in the United States

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