2016: Statement from FDA Commissioner Robert Califf, M.D.
on release of final individual patient expanded access form
Overview - How it works
Expanded Access for an Individual Patient
FDA may permit expanded access to an investigational new drug outside of a clinical investigation, or to an approved drug where availability is limited by a REMS, for an individual patient when the applicable criteria in § 312.305(a) (which apply to all types of expanded access) and § 312.310(a) (which apply specifically to individual patient expanded access, including for emergency use) are met.
Under the applicable criteria in § 312.305(a), FDA must determine that:
• The patient to be treated has a serious or immediately life-threatening disease or condition, and there is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition;
• The potential patient benefit justifies the potential risks of the treatment use and those potential risks are not unreasonable in the context of the disease or condition to be treated; and
• Providing the investigational drug for the requested use will not interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval of the expanded access use or otherwise compromise the potential development of the expanded access use.
Under the applicable criteria in § 312.310(a):
• The patient’s physician must determine that the probable risk to the person from the investigational drug is not greater than the probable risk from the disease or condition; and
• FDA must determine that the patient cannot obtain the investigational drug under another IND or protocol.
Source: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM432717.pdf
FDA Q&A 2016: PDF
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* FDA video on Expanded Access, 2014: YouTube
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The requested treatment protocol must be considered reasonable by your oncologist and you must be ineligible or unable to participate in a clinical trial that is testing the protocol.
The protocol must be deemed reasonable for your treatment circumstance, relative to available treatment options.
NOTE: Safety issues are not always published and available to the patient or the doctor, leading to unrealistic expectations. Efficacy can be overstated in early reports.)
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Your doctor must then contact the sponsor of the investigational agent, who must agree to provide the drug - they may not be able or willing.
This step can be a significant obstacle. The drug sponsor has little to gain and possible liabilities (financial, insurance, legal) when providing investigational drugs outside the structure and close supervision of a clinical trial.
"When a physician would like to request an Investigational New Drug (IND) application to use an unapproved drug or other product for a single patient, the first step is to obtain permission from the manufacturer.
Without the consent of the manufacturer, the unapproved product will not be available to the patient. After the manufacturer agrees to provide the product, the recommended procedure is to submit the following information to the appropriate review division. ... " FDA.gov
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Your doctor completes forms for FDA review & consent as described in the checklist below. The FDA will probably not stand in the way if the requested protocol is reasonable, relative to other available treatment options.
The rate limiting circumstance might depend on how your doctor communicates the urgency.
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Finally, your doctor must obtain IRB (Institutional Review Board) approval and draw up a consent form. This step is easier if your doctor is also a clinical investigator and is familiar with the process.
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Your doctor will be responsible for monitoring, and reporting and oversight. Cost to physician might not be compensated. Doctor might have to assume some liability.
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