A new online tool called the Expanded Access Navigator aims to help seriously ill patients obtain access to potentially life-saving investigational drugs.
The tool is meant for patients with serious or immediately life-threatening diseases or conditions who have no comparable or satisfactory alternative therapy. The development of the Expanded Access Navigator was led by the Reagan-Udall Foundation in collaboration with patient advocacy groups, the pharmaceutical industry, the FDA, and others in the federal government, according to an FDA Voice blog post by Richard A. Moscicki, MD, the FDA’s Deputy Center Director for Science Operations, Center for Drug Evaluation and Research.
The FDA Expanded Access process permits a product’s manufacturer, with FDA authorization, to provide an investigational drug to a patient with a serious or immediately life-threatening disease or condition for which there is no FDA-approved treatmen, even if the drug is still in development or under FDA review. Each year FDA receives more than 1,000 applications requesting authorization of expanded access through individual patient, intermediate-size, or large-size expanded access programs.
While the FDA has worked to streamline the process—particularly with a simplified application for individual patient expanded access—it can still be challenging and time-consuming for patients, family members, caregivers, and health care professionals to navigate the system.
The Expanded Access Navigator helps to educate patients and physicians about the process. Most of what they need to seek expanded access is now available in this single online location, including a directory where companies can submit public links to their expanded access policies, criteria used by companies to determine whether to make a drug available through expanded access, and contact information.
The tool is not a portal for applications; it is the first consolidated starting point for researching available investigational therapies. To help users find information quickly, the Navigator is separated into one section for patients and caregivers, and another for physicians.
The patient and caregiver section provides links to resources such as how to determine if a patient can participate in a clinical trial, the difference between an intermediate-size or larger expanded access program and a single-patient expanded access program, and their physician’s role in helping them obtain an investigational drug. Patients also can reach out to FDA’s Office of Health and Constituent Affairs’ Expanded Access Team.
Physicians can use the tool to identify investigational treatment options for their patients, explore clinical trials on behalf of their patients, learn how to engage with FDA and pharmaceutical companies as part of the process, and read about important factors to discuss with patients when considering expanded access. After using the Navigator and deciding on an investigational drug treatment option, physicians may contact FDA’s Division of Drug Information for assistance with their expanded access application.
The partners who contributed to the website’s development, in addition to FDA, include the American Cancer Society, the American Society of Clinical Oncology, the Biotechnology Innovation Organization, the Pharmaceutical Research and Manufacturers of America, Foundation Medicine, Susan G. Komen, Bristol-Myers Squibb, Genentech, Janssen, Lilly, Merck, and Pfizer.