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In searching for a clinical trial, there may be instances in which a patient will not be allowed to enroll due to an inclusion/exclusion requirement. If this happens there may be other options to explore that might enable access to the treatment.

Compassionate use or expanded access (both names are used) is a potential pathway for a patient with an immediate life-threatening illness to gain access to an investigational medication when no other clinical trials are available. It is understood that, due to the seriousness of a DIPG/DMG tumor, the patient’s family willing to accept risk in using compassionate use medications before the FDA provides approval

Outside of qualifying for a traditional clinical trial, the most common way for a patient to gain access to experimental medications is through an established compassionate use/expanded access clinical trial. Just like traditional clinical trials, there are generally limited treatment locations as well as inclusion/exclusion criteria requirements. Discuss with your medical team and/or nurse navigator if interested.

For a patient to gain access to an experimental medication outside of an established compassionate use/expanded access trial, the patient’s physician would need to petition to gain access to the medication. This is a very difficult and lengthy process that generally starts with your treating physician and then goes through approval of the pharmaceutical company, IRB, and FDA.  It is therefore quite uncommon for physicians to pursue this option, regardless of the disease.

If you think compassionate use/expanded access is a good fit for your family, talk to your physician and connect with one of our nurse navigators so we can help.

The Right-to-Try Act was approved by Congress on May 30, 2018. A patient must have tried all approved treatment options and be unable to participate in a clinical trial. However, the sponsors who develop the drugs make the final decision regarding whether to make their product available to patients. 

Please refer to the following URL for additional information: https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/right-try