Seeking Expanded Access

What does it take for patients with cancer who have exhausted their other medical options to gain access to late-stage investigational drugs?

What does it take for patients with cancer who have exhausted their other medical options to gain access to late-stage investigational drugs? It’s the mission of CancerActionNow.org, produced by the Marti Nelson Cancer Foundation, to help share that information. For patients who have learned about experimental treatments that sound promising for them, that organization and other experts suggest the following steps:

A. Determine if you might be eligible for a clinical trial of the drug. Search for the drug on ClinicalTrials.gov, and on the website of the drug’s developer.

B. If there are no appropriate clinical trials enrolling, search ClinicalTrials.gov and drug company websites for open expanded access programs (EAPs). Searching online for press releases or news reports about EAPs that have opened may also bear fruit. Or, you can check with the FDA by calling 301-827-4460.

C. If you find an EAP, call the drug developer to check your eligibility.

D. If there is no EAP available for the drug of interest, ask your doctor to help you open one. The doctor will need backing from a hospital or independent institutional review board before asking the drug’s developer to provide the treatment. If the company is willing, either it or the physician can act as the sponsor of the potential EAP, formally asking the FDA to approve it.

E. Once your doctor has agreed to help, try to smooth the path to approval. Call the FDA at 301-827-4460 for advice on how to approach the drug’s developer. Then, call that company’s medical director for oncology clinical development and arrange for him or her to speak with your doctor. Try to be with your doctor during the meeting so that you’re sure it has happened and will know the results.

F. Make the process easier for your doctor by supplying the EAP form required by the FDA, available at: www. fda.gov/downloads/AboutFDA/ReportsManualsForms/ Forms/UCM083533.pdf. If your doctor is interested, also share the FDA’s guidance on “What Physicians Need to Know Before Requesting Expanded Access/ Compassionate Use for Patients,” available at www.fda.gov/ForPatients/Other/ExpandedAccess/ ucm416812.htm. Your doctor can also call the FDA at 301-827-4460 for help with the application process.