As a physician, you must have wished there would be more treatment options for patients who have not responded to available therapies. Thank you for your willingness to seek access to an investigational drug for your patient through compassionate use (expanded access). [What is it?]
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See the list of precision medicine clinical trials.
Application process overview
Identify the investigational drug and the company that makes it.
Apply to a drug manufacturer: Ask the company to provide the drug for your patient. Secure a Letter of Authorization from the company.
Apply to the U.S. Food and Drug Administration for authorization of a compassionate use request for your patient.
Apply for IRB approval.
Submit required safety reports.
Help us build a registry of requests – share your case
To develop effective compassionate use policies, it is necessary to understand the unmet need. Currently, only the number of requests that were granted by both the companies and FDA is known. FDA authorizes over 99% of requests it receives. There is no data on the numbers of applications submitted for pediatric patients, or applications that were denied by the manufacturers.
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 patient 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, Program Director, at elena@kidsvcancer.org.
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Apply to a Drug Manufacturer | Apply for FDA Authorization | Apply to IRB |
Safety Reports Requirements | Request Our Help