The EA Navigator Mission
The Expanded Access Navigator, or EA Navigator, represents a unique partnership between the Reagan-Udall Foundation for the FDA, patient advocacy organizations, the pharmaceutical industry, and the federal government to provide clear, digestible information on single-patient EA.
Single-patient EA has gained attention over the last few years because of high profile social media campaigns, press coverage, and legislative actions. The amount of information available on expanded access has also increased dramatically, including EA information shared by the FDA and many pharmaceutical companies. It is still difficult, however, to find clearly organized, easy-to-understand information on the EA request process in one place.
To address this need for clarity and information about EA, the Navigator was created. This website provides a roadmap to guide patients, caregivers, and physicians through the request process, which involves various decisions, steps, and players, including pharmaceutical companies, the FDA, and Institutional Review Boards (IRBs). The Navigator clarifies the process for people who need information on investigational therapy or treatment options, which include clinical trials and EAPs, as well as single-patient EA. The goal of the Navigator is to increase understanding of the request process, not to encourage or discourage requests for expanded access.
We understand that the FDA is committed to implementing the new Right To Try law, and the Reagan Udall Foundation for the FDA is committed to providing information for patients with serious or life-threatening conditions who have exhausted other treatment options and may feel compelled to explore investigational therapies. The Expanded Access Navigator is intended to make it possible for patients and providers to access resources like: a company directory that leads them to the various biopharmaceutical company policies on expanded access; a guide provided for anyone trying to navigate ClinicalTrials.gov to find clinical trials for which they may be eligible; and, gives companies an opportunity to comply with some of the key provisions of the 21st Century Cures Act, which requires that companies disclose their expanded access policies and contact information.
The EA Navigator provides information and resources for non-emergency situations.
If your patient is in an emergency situation where access is needed in a matter of hours or days and a company is willing and able to provide the needed product, contact the FDA directly through its Division of Drug Information at 855-543-3784 or firstname.lastname@example.org. To request emergency access to investigational biological products regulated by CBER, call 240-402-8010 or 800-835-4709 or email@example.com
Weekends and after 4:30pm EST weekends, physician questions about and requests for expanded access for emergency use for drugs, biologics and medical devices should be directed to the FDA Emergency Call Center, telephone: 866-300-4374 or 301-796-8240.
The FDA also responds to patient questions on investigational drugs, biologics (including gene therapies for rare diseases) and medical devices. Patients or caregivers can contact the FDA’s Office of Health and Constituent Affairs at (301) 796-8460 or email PatientAffairs@fda.hhs.gov
For questions concerning the Navigator website or about the Reagan-Udall Foundation for the FDA, please email firstname.lastname@example.org. Please keep in mind that we are unable to answer questions or comments about expanded access that do not pertain to the Navigator program.
Understanding Single-Patient Expanded Access
For the expanded access process to work effectively, physicians, patients, the FDA, Institutional Review Boards and the pharmaceutical industry need to communicate and collaborate. The Navigator fosters an effective EA process by providing the context, considerations, and practical guidance needed to determine if a clinical trial, existing expanded access program, or single-patient expanded access is an appropriate option.
At launch, the Navigator focused principally on oncology treatment, but the process is similar for patients with other serious or life-threatening diseases or medical conditions, including rare diseases. The 30 million Americans with rare diseases are in particular need: FDA-approved treatments exist for only 5% of known rare conditions. The Navigator includes information and links to help all patients and physicians understand the process and potential of expanded access, and highlights some issues of particular concern for patients with rare diseases.
Because investigational treatments are still under study, it is not known if their potential benefits outweigh their risks. The Navigator is designed to help patients, physicians and caregivers understand this when considering the use of an investigational treatment. Most treatments under investigation do not ultimately demonstrate both effectiveness and that their benefits outweigh their risks, and therefore never come to market.
About the Reagan-Udall Foundation
The Reagan-Udall Foundation for the FDA is a non-profit 501 (c)(3) created by Congress to advance regulatory science critical to helping the U.S. Food and Drug Administration (FDA) meet its mission. The Foundation works collaboratively with stakeholders including academia, patient groups, industry groups, and FDA scientists. The EA Navigator exemplifies the Foundation’s process of working with public and private stakeholders to develop coordinated resources containing clear information for use by patients, healthcare providers, and others.
The Foundation would like to thank the partners who have come together to develop this important resource for patients with unmet medical needs.