For patients facing terminal or debilitating illnesses, time is not on their side. The FDA’s Expanded Access (Compassionate Use) program and the more recent Right to Try (RTT) pathway both offer hope when no approved therapies remain. As the FDA’s 2025 guidance reaffirms, decades of experience with compassionate use and the emergence of RTT have together reshaped how patients access potentially life-saving experimental drugs.
The Expanded Access (EA) or Compassionate Use program allows patients with serious or immediately life-threatening diseases to obtain investigational drugs outside of clinical trials. Created under 21 CFR 312 Subpart I, this framework has evolved since the 1980s and was formalized in 2009, with the latest FDA revision, released this week, reflecting lessons from more than three decades of practice. The FDA’s updated guidance details the three primary EA categories: Individual patient access, including emergency use; Intermediate-size population access for small groups; and Treatment INDs for widespread access.
These programs rely on FDA authorization, Institutional Review Board (IRB) oversight, and informed consent—ensuring safety and ethical accountability. Sponsors must also agree to provide the drug, as FDA authorization does not compel them to do so. While this may slow access, it creates a balance between patient need and preserving the scientific integrity of ongoing clinical trials. The new guidance, shaped by the 21st Century Cures Act and FDARA, also requires sponsors to publicly post their expanded access policies, improving transparency for patients and physicians seeking investigational therapies.
The Right to Try (RTT) pathway was created as an alternative to the compassionate use pathway.
Enacted in 2018, the Right to Try Act was designed to complement—not replace—the FDA’s compassionate use system. RTT allows patients with life-threatening or severely debilitating conditions to directly request investigational drugs from manufacturers without FDA pre-approval. Drugs eligible for RTT must have completed at least Phase I trials and remain under FDA investigation.
Unlike compassionate use, RTT removes the requirement for IRB review and FDA authorization, cutting administrative delays and offering a faster route for those in desperate need. Importantly, RTT may also apply in non-life-threatening but serious conditions, expanding the ethical conversation around patient autonomy and early therapeutic access. However, RTT lacks FDA oversight on dosing, monitoring, and safety reporting. This freedom increases speed—but also risk. Manufacturers are not required to supply the drug or report outcomes, and data collected through RTT cannot typically support drug approval.
Compassionate Use vs. Right to Try: A Side-by-Side Look
| Compassionate Use (Expanded Access) | Right to Try (RTT) | |
| Oversight | FDA and IRB review required | No FDA or IRB pre-approval |
| Eligibility | Life-threatening or serious disease only | Life-threatening and certain non-life-threatening conditions |
| Speed of Access | Moderate—requires FDA and IRB review | Faster—bypasses regulatory approval |
| Safety Measures | Robust—FDA monitoring, safety reports, informed consent | Limited oversight; relies on physician discretion |
| Sponsor Obligation | Voluntary participation | Voluntary participation |
| Data Use | May support regulatory filings | Not used for FDA submissions |
The FDA’s Expanded Access Guidance reaffirms the strength of a system that has safely served patients for over 30 years. Yet, the Right to Try Act represents a new era of patient-driven access—faster, more flexible, and empowering those who have run out of time or options. Together, these two pathways form a complementary ecosystem: one rooted in safety and structure, the other in speed and autonomy—both offering hope to patients who cannot wait.
