The time when you decide to seek compassionate use medicine for your child is very stressful, and we are sorry that you are facing the prospect of trying to access an unapproved drug. As you take steps in this process, please contact us if you have any questions or seek support for your compassionate use application.
What is compassionate use?
In search of new or more effective treatments, kids with cancer often enroll in studies of new medications called clinical trials. But some kids with in life-threatening situations cannot participate in a trial, or there are no trials for them. These kids can apply to access new medications through compassionate use, sometimes also called expanded access.
The idea behind a compassionate use of a drug is to let a seriously ill patient try a medicine which is still being studied before it receives government approval and becomes available for purchase. Usually this is done because the patient can’t wait, and early research shows that the medicine is promising. For kids with cancer, compassionate use requests are sometimes for medicines that are studied in adults only but have the same molecular target as the child’s cancer, and therefore might help. These not-yet-approved drugs are also called investigational, or experimental drugs.
How to find compassionate use drugs?
Families often learn about experimental drugs from their doctor. Some conduct research on the Internet themselves. A good place to start is the database of clinical trials, www.ClinicalTrials.gov. Many disease-specific patient organizations and patient forums on the Internet can provide guidance. Finally, patients often hear about new drugs from other patients.
Application process summary
Ask your child’s doctor if compassionate use is the right path for your child.
Have your doctor visit our Information for Physicians page.
Ask your doctor to contact the drug company and apply for compassionate use.
If the drug company agrees to provide the drug, have your physician submit the paperwork to the Food and Drug Administration (FDA) and the Institutional Review Board (IRB) of the hospital.
Wait for the FDA’s approval and for an assignment of an investigational new drug number (IND) to the application. For children with cancer, it usually takes a few days.
Have your doctor submit the IND received from the FDA to the drug company.
Your doctor will receive the shipment of the drug directly from the drug company.
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Application steps explained
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Help us help future kids – share your story
We want kids to have access to as many cancer treatment options as adults have. If we know the numbers and the outcomes of compassionate use applications for kids, it will help our advocacy work aimed at changing public policies. Right now, we only know the number of compassionate use requests that were granted by the companies, but the number of denied requests is not known.
Let us know to which companies you have made compassionate use requests. Let us know the point of contact in the company that you used, whether the company responded and in how much time, and whether your request was ultimately granted. And, please let us know how your child did – was there any possible benefit from the drug?  Were there unexpected toxicities?
Please help us build a registry of compassionate use application outcomes by answering a few questions for our database, or email Elena Gerasimov at elena@kidsvcancer.org.