Navigating Compassionate Use Options for Your Patients with Cholangiocarcinoma

September/October 2021, Vol 2, No 3
Melinda Bachini
Director of Advocacy
Cholangiocarcinoma Foundation
Billings, MT

I believe that the most powerful and positive force in medicine is that of a self-advocating patient. At the Cholangiocarcinoma Foundation, we strive to connect, educate, and empower patients with cholangiocarcinoma (CCA) to partner with their care team in navigating the course of their treatment. However, we understand that the treatment options are extremely limited for many patients with CCA.

Depending on the specific CCA diagnosis, a patient’s treatment plan may include a combination of surgery, radiation therapy, chemotherapy, immunotherapy, or targeted therapy. For many patients with CCA, the standard of care remains the same, a chemotherapy combination of cisplatin and gemcitabine.

Often, patients may not fully understand their options and may only be offered palliative care, and then later find a clinical trial that could offer a life-extending treatment and contribute to their quality of life.

Because the treatment options are currently so limited, many patients with CCA are motivated to participate in clinical trials. These efforts are intended to help extend the patients’ lives, enhance their quality of life, or help to advance the research that could contribute to the science that will help future patients.

But not all patients are eligible to participate in clinical trials. Factors that may disqualify a patient include1:

  • Autoimmune conditions or use of immunosuppressant therapies
  • Active or acute infection requiring antibiotics
  • Chronic infection or chronic disease, such as HIV, hepatitis B, or hepatitis C
  • Active brain metastases
  • Recent history of another cancer
  • Life expectancy of less than 3 to 6 months
  • Patients who are unable to provide adequate self-care.

The Cholangiocarcinoma Foundation strongly advocates for inclusive eligibility criteria for all clinical trials. Because CCA is a disease that is often diagnosed at a late stage, a large percentage of patients with this malignancy may be sicker than the typical patient with another type of cancer who participate in clinical trials.

As a physician, you can intervene to advocate for the compassionate use of an experimental treatment for a patient who does not qualify for a clinical trial.

Criteria for Compassionate Use or Expanded Access

For patients with CCA, expanded access, also known as “compassionate use,” may offer the only available treatment option. The eligibility criteria for a patient to receive a therapy on a compassionate use basis include2:

  • The patient has a serious disease or a life-threatening condition
  • No comparable alternative therapy is available to diagnose, monitor, or treat the disease or condition, or the patient has not been helped by FDA-approved treatments
  • The potential benefit to the patient justifies the potential risks of the investigational treatment
  • The patient is not eligible for clinical trials of the experimental drug
  • The company that manufactures the drug has a program for compassionate use and agrees to provide that medication to your patient.

Physicians can take several steps to help navigate the expanded access or compassionate use for their patients. The physician must agree to continue to be the treating physician for that patient for the duration of the experimental therapy. The physician must petition the FDA through an expanded access request, as well as ensure that the request is approved through the clinical trial’s Institutional Review Board, and that the drug manufacturer is willing and able to provide the treatment to the patient.

For some patients with CCA, compassionate use may offer the only available treatment option. For more information or support with navigating compassionate use, please contact This email address is being protected from spambots. You need JavaScript enabled to view it.. More information is also available through the FDA’s Project Facilitate Program.


  1. National Cancer Institute. Inclusion/Exclusion Criteria for National Cancer Institute (NCI) Sponsored Clinical Trials. September 26, 2018. Accessed September 8, 2021.
  2. US Food and Drug Administration. Expanded access. Accessed September 8, 2021.

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