Expanded Access Program
Expanded access, sometimes known as compassionate use, is a process regulated by the United States Food and Drug Administration (FDA), which allows pharmaceutical companies such as La Jolla Pharmaceutical Company (“La Jolla”), to provide investigational medicine(s) to patients with serious, life-threatening diseases or conditions, and who otherwise cannot participate in a clinical trial.
La Jolla, in accordance with our core values, supports expanded access programs in the United States for permissible investigational medicine(s).
How Expanded Access Program Works
Making decisions about expanded access requests is always complex. We strive to make these decisions as ethically and appropriately as possible, considering the patient’s case and the available medical and scientific information about the investigational medicine(s). Our primary priority is to ensure the expanded access program is conducted in a manner that minimizes the potential risk of harm to patients, while providing access to investigational medicine(s) outside of clinical trials.
La Jolla will consider granting expanded access to an investigational medicine(s) if all of the following criteria are met (as codified in 21 CFR Part 312.305):
Patient has a serious or life-threatening illness with no comparable or satisfactory alternative therapies
- Patient is ineligible for, or otherwise unable to, participate in a clinical trial
- Patient has a disease for which there is sufficient evidence of a projected benefit from the use of the investigational medicine and the benefit outweighs the known or anticipated risks
- Providing the investigational medicine for the requested use will not interfere with the initiation, conduct, or completion of active clinical trials
- The patient is either being enrolled as a part of a treatment protocol (“cohort” expanded access) or the FDA has approved the use of the investigational medicine(s) for the individual patient.
The investigational medicine(s) made available through expanded access have not yet been approved by the FDA and thus have not been deemed safe and effective. They may be effective in the treatment of a condition, or they may not. They also may have unexpected serious side effects. Doctors and patients should consider all possible benefits and risks when seeking access to investigational medicine(s) prior to regulatory approval.
Once a regulatory agency approves the investigational medicine for commercial use and the medicine is commercially available, existing expanded access programs for that medicine will be phased out.