1.5 Regulatory Approval
Regulatory agencies evaluate safety and efficacy evidence to approve new drugs. The FDA (US), EMA (EU), and other agencies have harmonized standards.
NDA/BLA Submission
New Drug Application (NDA)
Small molecule drugs. Contains all preclinical/clinical data, CMC, proposed labeling.
Biologics License Application (BLA)
Biologics (antibodies, vaccines, gene therapies). Additional manufacturing requirements.
Common Technical Document (CTD)
Standardized format for submissions: Quality, Nonclinical, Clinical modules.
Review Process
Standard Review
10-12 months. For drugs with modest clinical benefit.
Priority Review
6 months. Significant improvement over existing therapies.
Fast Track
Serious conditions with unmet need. More FDA meetings, rolling review.
Breakthrough Therapy
Substantial improvement. Intensive FDA guidance, priority review.
Accelerated Pathways
Accelerated Approval
Based on surrogate endpoints (e.g., tumor shrinkage). Confirmatory trials required.
Orphan Drug Designation
Rare diseases (<200,000 US patients). Tax credits, 7-year exclusivity, reduced fees.
Emergency Use Authorization (EUA)
During public health emergencies. Lower evidence bar, temporary authorization.
Global Regulatory Bodies
FDA (USA)
Food and Drug Administration. CDER, CBER divisions.
EMA (EU)
European Medicines Agency. Centralized procedure for EU approval.
PMDA (Japan)
Pharmaceuticals and Medical Devices Agency.
ICH
International Council for Harmonisation. Global guideline development.