A clinical trial is a controlled research study designed to evaluate a medicine’s efficacy and safety, following strict protocols and inclusion criteria.
An Expanded Access Program (EAP), also called Early Access or Compassionate Use, allows patients who are not eligible for a trial and who have serious or life-threatening conditions without alternatives to access an investigational medicine outside the trial setting.
Clinical Trial vs Expanded Access
| Aspect | Clinical Trial | Expanded Access Program (EAP) |
|---|---|---|
| Primary Purpose | Generate scientific evidence on efficacy/safety for regulatory approval | Provide treatment to patients with no alternatives |
| Regulator oversight | Approved & monitored by regulatory authorities & ethics committees | Authorized by regulators (FDA, EMA, national agencies) with less strict protocols |
| Participants | Patients meeting strict inclusion/exclusion criteria | Patients not eligible for trials but with urgent medical need |
| Design | Randomized, controlled, blinded when possible | Non-randomized, no control group |
| Data collection | Comprehensive (efficacy, safety, endpoints) | Limited – mainly safety and real-world use |
| Patient cost | Usually free; sponsored by manufacturer | Often free or reimbursed (EU), or cost-recovery allowed (US) |
| Goal | Regulatory approval & scientific validation | Patient access + supportive safety data |
Key Takeaway
- Clinical trials = research → generate data for approval.
- Expanded Access = treatment → provide access for patients outside trials.
Sources
- EMA – Clinical Trials
https://www.ema.europa.eu/en/human-regulatory/research-development/clinical-trials - EMA – Compassionate Use (EAP in EU)
https://www.ema.europa.eu/en/human-regulatory/research-development/compassionate-use - FDA – Clinical Trials vs Expanded Access
https://www.fda.gov/news-events/public-health-focus/expanded-access - BMJ – Expanded Access in Clinical Practice
https://www.bmj.com/content/371/bmj.m4050
