Expanded Access (EA) is a program run by the Reagan-Udall Foundation to provide some patients with serious, life-threatening illnesses access to investigational treatments not yet approved by the FDA. Also known as compassionate use, named-patient use, or single-patient access, information about program is available on the Foundation’s web site through the EA Navigator page. Patients and caregivers are directed to work with their physician and use various websites to first identify clinical trials for which they may be eligible to participate. If the patient is not eligible for or able to participate in a trial, there are often Expanded Access Programs (EAP) that may offer a treatment approach. If the EAP are not appropriate, the physician may finally make a request for single-patient expanded access. This involves multiple steps that are analogous to enrollment in a clinical trial but are tailored for the specific individual case:

1. The physician requests permission from the pharmaceutical company to use the treatment. The company will review the request and decide whether to grant access based on a weighing of potential risks versus benefits. If access is granted, the company will issue required documentation to the physician and may work with them on the treatment plan.
2. The physician next requests permission from the FDA by submitting a Form 3926 and the required documentation. The FDA will again weigh the potential risks and benefits and will respond within 30 days. If permission is granted, the physician will receive an IND number.
3. The physician will next request approval from an Institutional Review Board (IRB) by submitting the FDA forms, the required information from the company, a treatment protocol, and an informed consent form to the IRB. The board is composed of medical and ethics experts as well as legal and community representatives, and they are responsible for ensuring the risks and benefits are clearly communicated in the documentation and the patient’s rights and safety are protected.
4. Following IRB approval, the physician will obtain the patient’s informed consent using the approved form, which should clearly explain the proposed protocol, the expected outcomes and foreseeable risks, and any alternative options available, along with an explanation of patient rights and record confidentiality.
5. Once the informed consent is obtained, the treatment will be shipped to the physician who will administer it and report serious and unexpected adverse events and outcomes to the pharmaceutical company, the FDA, and the IRB.

Similar steps are required as part of the normal clinical trials process for a new drug product, which is why review of existing trials is the first step of EA. The steps are important, firstly to protect the patient, and secondly to enable EAP and single-patient EA data to potentially be used in support of a regulatory application. This latter topic is the subject of an upcoming conference on November 19, 2018. Participants include the FDA, members of the Reagan-Udall Foundation, doctors from industry and academia, and representatives of patient groups. Members of the public are welcome and should register here. The Foundation is a non-profit organization created by Congress to advance regulatory science, and you can read more about their activities here.

Text Copyright © 2018 Katrina Rogers