What Is a Compassionate Use Program for Mesothelioma?

What Compassionate Use Means

Compassionate use — formally called expanded access — is an FDA-regulated pathway that allows patients with serious or life-threatening conditions to access investigational drugs outside of clinical trials. According to the FDA, this pathway exists for patients who cannot participate in a clinical trial and have no satisfactory alternative treatments available.

For mesothelioma patients, compassionate use can provide access to promising experimental treatments — such as novel immunotherapy agents, targeted drugs, or combination therapies — that are still in development but have shown encouraging results in early-phase trials. This pathway is particularly relevant for patients who do not meet clinical trial eligibility criteria due to prior treatments, performance status, or other factors.

How to Apply

The compassionate use process involves three parties: the treating physician, the FDA, and the drug manufacturer. Your physician submits a request to the FDA explaining your diagnosis, why existing treatments are inadequate, and why the specific investigational drug is appropriate for your case. The FDA has streamlined this process and typically responds within days for individual patient requests.

The drug manufacturer must also agree to provide the investigational drug. This is not guaranteed — companies may decline if providing the drug could compromise an ongoing clinical trial or if supply is limited. However, many companies have formal expanded access programs and published policies for handling these requests.

Eligibility and Considerations

To qualify for compassionate use, you must have a serious or life-threatening condition (mesothelioma qualifies), no comparable or satisfactory alternative treatment options, inability to enroll in a clinical trial for the drug, and a physician willing to manage your treatment and submit the necessary documentation.

Important considerations include that the treatment is still experimental and may not work, side effects may be significant or unknown, insurance coverage for the drug and associated care is not guaranteed, and the level of monitoring may differ from what is provided in a formal clinical trial with its structured safety oversight.

Taking Action

If you are interested in compassionate use, discuss it with your oncologist. Your doctor can evaluate whether an investigational drug is appropriate for your situation and initiate the application process. The Right to Try Act of 2018 also provides an alternative pathway for accessing certain investigational drugs that have completed Phase 1 trials.

While exploring experimental treatment options, continuing to pursue legal compensation for asbestos exposure ensures financial resources are available to support your care. An experienced mesothelioma attorney can evaluate your case at no cost.