The US Food and Drug Administration (FDA) determines whether any particular drug may be prescribed by doctors. It’s normally a years-long process to get a drug approved, across multiple phases, first to ensure a drug’s safety, then to determine its efficacy, and finally to assess its effectiveness compared against existing “gold-standard” treatments. Yet in some cases, patients with brain tumors and other serious conditions can get permission to try treatments that haven’t yet gone through this process, thanks to the FDA’s Expanded Access program.

Expanded Access, which is sometimes called “compassionate use,” due to its roots in offering drug options to terminally ill patients, gives patients a way to access investigational or experimental treatments outside of the structure of a clinical trial. In the past, clinical trials were the only way to access experimental treatments. According to the FDA, Expanded Access is for patients “with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device).”

Treatments through Expanded Access remains something that is carried out under the supervision of your doctor, and so discussing all of your options with your neuro-oncologist remains the first step. Whenever possible, your doctor will probably recommend clinical trials before expanded access, because clinical trial drugs may be better understood from the perspective of safety, dosing, side effects, and drug interactions. Furthermore, data collected in clinical trials contributes to global progress against the disease.

Nevertheless, many brain tumor patients may be in a situation where standard treatments have been exhausted and clinical trial eligibility cannot be met. In those cases, Expanded Access might be the best or only way to use a drug that could help.

The program works like this:

  1. A doctor asks a drug company to provide an experimental treatment.
  2. The doctor submits the treatment plan to an Institutional Review Board (IRB), who act as a check on patient welfare and ensure that the patient can properly give their informed consent.
  3. The doctor and the IRB submit the request to the FDA, who approve most requests that have gone through the appropriate steps.

One obstacle to Expanded Access is that many doctors have limited experience going through the process. To remove roadblocks, the FDA launched an initiative in 2019 to make it easier for doctors and patients to take advantage of the program. The FDA’s oncology staff now run a call center for doctors wishing to submit Expanded Access requests on behalf of their patients. FDA staffers will now help the doctor with paperwork, drug company contacts, and IRB proposals.

While no patient hopes to find themselves in a position where throwing a Hail Mary pass seems like the best option, it remains an unfortunate reality that many brain tumor patients reach that point. When other approaches seem exhausted