For patients with serious or life-threatening conditions who have exhausted standard treatment options, expanded access (also called compassionate use) may provide access to investigational drugs before they complete clinical trials.
What Is Expanded Access?
Expanded access is an FDA regulatory pathway allowing patients outside of a clinical trial to receive investigational drugs, biologics, or medical devices when no comparable alternative exists and the patient’s condition is serious or life-threatening.
Unlike a clinical trial, expanded access does not generate formal effectiveness data, but it does collect safety information that contributes to the drug’s development dossier.
Types of Expanded Access
Individual patient expanded access, the most common form. A physician submits an application to the FDA for a single patient. The FDA approves or denies, typically within 24–72 hours for emergencies.
Intermediate-size expanded access, for a small group of patients not eligible for the ongoing trial or for whom the trial has closed.
Widespread expanded access, used when a drug is in the final stages of development and there is sufficient evidence of safety, allowing broader access while approval is pending.
How Clinical Trials Differ From Expanded Access
In a clinical trial, participants receive systematic monitoring, physician oversight, and contribute to scientific evidence. In expanded access, safety monitoring is less structured and the patient may be responsible for costs not covered by the sponsor or insurance.
Is It Available for NuLine’s Studies?
Expanded access availability depends on the specific sponsor and drug program. For most of NuLine’s active studies, the preferred path is direct enrollment in the clinical trial, which provides more comprehensive monitoring and no-cost care.
Contact NuLine to discuss whether you may qualify for an active study or whether expanded access may be relevant to your situation.