Dateline: Washington, D.C.
The Reagan-Udall Foundation for the FDA launches Navigator website to share resources and policies on expanded access to investigational therapies.
The nonprofit Reagan-Udall Foundation for the FDA released a new, online resource called the Expanded Access Navigator. The website features the first pharmaceutical company directory of expanded access policies and criteria, as well as educational resources, guides and downloadable forms.
The Navigator guides patients and physicians through the complicated process often called compassionate use, beginning with assessing whether all approved therapeutic options for patients with serious or life-threatening conditions are exhausted. If no clinical trials or expanded access programs (EAPs) are available or accessible, the Navigator acts as a clearinghouse for the single-patient expanded access policies of dozens of leading biopharmaceutical companies.
Many patients don’t know that their physicians must first receive permission from the drug manufacturer before FDA can approve expanded access requests. To date, FDA authorized 99% of expanded access requests received and for the launch posted an FDA VOICE blog explaining the Navigator.
“We know how important it is for patients to be able to access investigational treatments when there are no other options to treat their serious disease or condition,” said Dr. Janet Woodcock, Director of the Center for Drug Evaluation and Research at the FDA. “The Navigator is an important tool developed under this public-private partnership and will help make this process easier and faster.”
While drug manufacturers are not required to grant expanded access to investigational therapies, the 21st Century Cures Act stipulates public availability of expanded access policies and contact information. Those publically disclosed policies must include links or references to applicable expanded access clinical trial records on the National Institutes of Health’s website www.clinicaltrials.gov and the Navigator includes a downloadable user guide for that resource.
“Until now, patients with serious or life-threating illnesses had the added burden of becoming their own treatment detectives and social media campaigners,” said June S. Wasser, Executive Director of the Reagan-Udall Foundation for the FDA. “The Navigator makes searching for information more equitable for patients and demystifies the process.”
It also helps physicians, who initiate all formal requests to companies for investigational drugs.
“This is an extraordinary resource that is rich with helpful information,” said Dax Kurbegov, MD, Vice President & Physician-in-Chief, Clinical Programs at Sarah Cannon, the Cancer Institute of HCA Healthcare. “I’m thrilled that an effort has been made to empower physicians to navigate this complex area and to serve a previously unmet need in patient care.”
Because investigational therapies are unproven, they may have serious side effects and risks. The FDA and Institutional Review Boards play essential roles in ensuring that patient protections are in place, that medicines are administered by licensed practitioners and that all adverse events and results are reported. The Navigator makes it easier for patients and their caregivers to understand acceptance criteria, find clinical trials, research existing expanded access programs and download forms for physicians to complete.
“It is increasingly important for patients and their caregivers to have access to information that could affect their treatment and to feel empowered to participate in the decision-making process with their physicians,” said Ellen Sigal, PhD, the Foundation’s Board Chair and Chairperson & Founder of Friends of Cancer Research. “The Navigator more easily provides that critical information, which is why patient advocacy groups, such as Friends, are supporting this important new tool.”
The American Cancer Society, the American Society of Clinical Oncology, the Biotechnology Innovation Organization (BIO), FDA, Foundation Medicine, PhRMA and Susan G. Komen helped develop the Navigator along with the support of Bristol-Meyers Squibb, Genentech, Janssen, Lilly, Merck and Pfizer.
About the Reagan-Udall Foundation for the FDA
The Reagan-Udall Foundation for the FDA is an independent, nonprofit corporation created by Congress to advance the mission of the Food and Drug Administration. It does not participate in regulatory matters or offer advice to FDA on policy and the Navigator does not encourage or discourage requests for expanded access.