Definition
Breakthrough Therapy Designation is an FDA program for drugs intended to treat serious conditions where preliminary clinical evidence indicates substantial improvement over available therapies on clinically significant endpoints. Created by FDASIA in 2012.
How Breakthrough Therapy Works
Sponsors submit requests with preliminary clinical data demonstrating substantial improvement. FDA evaluates whether evidence supports clear advantage over existing treatments on meaningful endpoints.
Eligibility Criteria
- Intended to treat a serious condition
- Preliminary clinical evidence (not just preclinical)
- Demonstrates substantial improvement over available therapy
- Improvement on clinically significant endpoint(s)
Breakthrough Therapy Benefits
Breakthrough Therapy provides the most intensive FDA support:
- All Fast Track Features: Rolling review, frequent meetings
- Intensive Guidance: FDA helps design efficient development program
- Senior Leadership Involvement: Organizational commitment
- Cross-Disciplinary Meetings: Multiple FDA divisions engaged
- Expedited Review: Often combined with Priority Review
Breakthrough Therapy Statistics
| Metric | Value |
|---|---|
| BTDs Granted (as of 2025) | 600+ |
| Approval Rate | ~65-70% |
| Average Development Time Reduction | 30% |
| Percentage of Novel Approvals with BTD | ~35-40% |
Why BD Teams Track Breakthrough Therapy
For business development professionals, BTD is a strong value signal:
- Deal Implication: BTD assets command premium valuations due to higher approval probability and faster timelines
- Due Diligence Focus: Review the clinical data supporting BTD and FDA’s assessment of substantial improvement
- Opportunity Signal: BTD-designated programs are attractive acquisition targets