Definition
Accelerated Approval is an FDA pathway allowing approval of drugs for serious conditions based on surrogate endpoints or intermediate clinical endpoints reasonably likely to predict clinical benefit. Post-approval confirmatory trials are required to verify the predicted benefit.
How Accelerated Approval Works
Drugs receive approval based on surrogate markers rather than clinical outcomes. Sponsors must then conduct Phase 4 confirmatory trials to verify the surrogate truly predicts meaningful benefit.
Eligibility Criteria
- Treats a serious or life-threatening condition
- Provides meaningful advantage over available treatments
- Uses surrogate endpoint reasonably likely to predict clinical benefit
- Sponsor commits to confirmatory trials
Accelerated Approval Process
| Phase | Requirement |
|---|---|
| Pre-Approval | Demonstrate effect on surrogate endpoint |
| Approval | FDA grants based on surrogate data |
| Post-Approval | Conduct confirmatory trials |
| Conversion | Full approval upon confirmation |
| Withdrawal | If benefit not confirmed |
Common Surrogate Endpoints
| Therapeutic Area | Surrogate Endpoint |
|---|---|
| Oncology | Tumor response rate, progression-free survival |
| HIV | CD4 count, viral load |
| Cardiovascular | LDL cholesterol reduction |
| Hematology | Hemoglobin levels |
Why BD Teams Track Accelerated Approval
For business development professionals, Accelerated Approval presents unique considerations:
- Deal Implication: Accelerated Approval provides early market access but carries confirmatory trial risk
- Due Diligence Focus: Assess confirmatory trial design and probability of verifying clinical benefit
- Opportunity Signal: Post-approval confirmatory trials may require partnership support