Expanded access, or “compassionate use,” allows patients and their physicians to request from pharmaceutical medical products (drugs, devices, biologics) that have not yet been approved by the U.S. Food and Drug Administration (FDA). There are several steps you and your doctor must take to get access to an unapproved medication, according to the FDA’s Expanded Access Program.
First, talk to your healthcare provider to determine if you are a good candidate for an unapproved medical product. You will need to get a physician’s approval to use an investigational medical product before a company will grant you access. There may be approved medications already on the market for your condition or nearby clinical trials testing a drug for your condition—both of which you ought to try (if eligible) before seeking expanded access to an unapproved medical product.
If you and your doctor decide to request an unapproved medication, your doctor must request it from the company developing the product. The company is ultimately responsible for deciding whether to give you access. The company may or may not have a program in place for such requests. If the company does not, your doctor can still ask if the company is willing to give you access on a compassionate use/expanded access/pre-approval access basis.
A common complaint from patients and advocates is that it is difficult to even reach the appropriate person in a company. Thus, the fact that there is currently legislation under consideration dealing with just this problem is welcome news. The 21st Century Cures Act (which was passed by the House of Representatives and is being debated in the Senate) includes provisions requiring certain pharmaceutical companies to make public their policies on expanded access; if a company has no expanded access policy, that must be stated as well. The statements must include to whom to submit requests, along with contact information; procedures for making requests; the general criteria the companies use in approving or denying requests; and how long it may take to get a response from the company.
If the company approves your request, your doctor can then submit the required paperwork to the FDA for approval. A persistent myth is that it takes up to 100 hours for doctors to fill out the required FDA form to request approval to use an experimental product. That is incorrect; the 100 hours refers of the amount of time estimated for a drug company to complete a full Investigational New Drug (IND) application, required when any new compound will first be tested in humans (prior to beginning a Phase 1 clinical trial). Embedded in this form was a short section to be completed by physicians requesting to use an unapproved drug.
To eliminate confusion, the FDA released a new form in June 2016 (Form FDA 3926), which is estimated to take 45 minutes to complete. This new form contains only those questions to be completed by physicians.
Your doctor will fill out FDA Form 3926 and submit this paperwork, along with written approval from the drug manufacturer, to the FDA. By law, the FDA must respond to your request within 30 days. However, the FDA normally gives its decision much sooner: for instance, in case of emergency requests, the agency has been known to respond in 24 hours or less, sometimes granting approval immediately. The FDA approves more than 99% of the requests it receives.
The FDA will consider several factors when reviewing your request. First, the agency must make sure that your condition is life-threatening or that it has a “serious” substantial impact on your day-to-day functioning. Second, the FDA must determine that there aren’t any approved medications for your condition and that you are ineligible or unable to enroll in a clinical trial for the drug requested. Finally, the FDA will consider whether the possible risks of the investigational treatment are reasonable for you, given your situation.
If your request is approved by the FDA, federal law requires that the institutional review board (IRB) or ethics committee at the site where you will be treated approve your treatment. IRBs work to protect patients who are receiving unapproved treatments. You (or your legal representative) will have to sign an informed consent document stating that the drug has not yet been shown to be effective for your condition and that you understand the possible risks in taking it. Once the IRB approves your informed consent form and treatment plan, your doctor can begin administering the investigational medical product. If it is an emergency, your doctor may administer the product without IRB approval; however, s/he must notify the IRB of this within 5 days.
For more information on the FDA’s Expanded Access policies, visit: http://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/default.htm
The NYU Langone Medical Center’s Working Group on Compassionate Use and Pre-Approval Access has a relevant FAQ: http://www.med.nyu.edu/pophealth/divisions/medical-ethics/compassionate-use/nyu-working-group-compassionate-use-pre-approval-access