Compassionate Use: An Option Beyond Standard of Care


In the world of pancreatic cancer, time is the patient’s most precious commodity. Delays in treatment—for whatever reason—can have life-threatening consequences.

Several factors make pancreatic cancer a challenge for doctors to treat—perhaps the most obvious being that pancreatic cancer is often not caught until the later stages. By that time, patients will likely not respond, or will stop responding, to the few treatment options available.

Doctors then face the dilemma of few options to offer their patients hope when the future looks grim. In these cases, “compassionate use”—treating with drugs (or medical devices) different from the standard of care—can bring hope to an otherwise dark situation. Compassionate use (or “expanded access”) describes the use of medications that are not approved by the Food and Drug Administration (FDA) but are currently being studied in clinical trials; such medications are known as investigational drugs.

“Whenever there’s a disease that requires medical treatment, there’s a standard of care considered to be the most effective treatment to date,” says Allyson Ocean, M.D., a medical oncologist, associate professor of clinical medicine at Weill Cornell Medicine in New York, and co-founder of Let’s Win Pancreatic Cancer. “These are drugs and regimens that became standards of care because they went through the rigors of clinical trials and have the data to support the safety and efficacy.”

Ocean explains that clinical trials also include standards of care, as studies often compare the standard drugs to other treatments. This holds true for phase III clinical trials. In those trials, the new regimen is usually compared to the standard of care or included with the standard to see if the new drug works better than what is already in use.

Off-Label Compassionate Use

Compassionate use comes into play when trying to help the patient live longer. Take the drug trastuzumab (Herceptin), for example. Commonly used to treat breast and stomach cancers, the FDA has not approved the drug for pancreatic cancer. However, a doctor may use trastuzumab in a patient with pancreatic cancer who has received several other pancreatic cancer treatments if their tumor contains the protein that trastuzumab is targeting. The practice of using drugs to treat a condition other than the one for which they are FDA-approved is known as off-label compassionate use. Even though trastuzumab is not indicated for pancreatic cancer, the rationale is that the drug may help that pancreatic cancer patient live a little bit longer. So having other treatment options helps.

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