DATELINE: WASHINGTON D.C. - MARCH 22, 2018

Right-To-Try law not a shortcut to investigational drugs – Expanded Access Navigator can reduce confusion.

Serious or terminally ill patients hoping House passage of federal Right-To-Try legislation will bypass hurdles to unapproved medical treatments need accurate, neutral information now more than ever.

Even with the new legislation, biopharmaceutical companies still decide whether patients can “try” their investigational products – a process sometimes called compassionate use – not the Food and Drug Administration. Which is why the Reagan-Udall Foundation for the FDA created a Company Directory on its Expanded Access Navigator.

The Navigator’s Directory pre-dates the federal legislation and is a first-of-its-kind clearinghouse for patients and physicians to research expanded access policies and criteria. It also includes company-specific expanded access links to the clinicaltrials.gov database and a plain-language guide to using that resource. This is important information because companies generally don’t grant single patient compassionate use access if alternatives, like clinical trials or group expanded access programs, are available.  

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 clinical results are reported. Under the pre-existing system, the FDA approves more than 99% of all expanded access requests after manufacturers agree to provide the drug.

The Navigator breaks down the entire process with a step-by-step tutorial for both physicians and patients/caregivers. Resources include downloadable document checklists, FDA forms, safety reports and reporting requirements so that physicians have the tools they need to request expanded access.

“As the Congressionally-mandated nonprofit foundation for the FDA, we do not lobby or take a position on Right-To-Try legislation,” said June S. Wasser, Executive Director. “Our goal is to make the existing process more accessible to all patients without sacrificing safety or jeopardizing clinical trials already underway.”

The Foundation’s Navigator leverages the commitment of 38 drug manufacturers and the FDA by creating a single location to begin exploring expanded access.

“Because the federal Right-To-Try legislation does not establish new pathways for obtaining unapproved drugs, the up-to-date criteria and contact information listed on the Expanded Access Navigator’s Company Directory is more critical than ever,” said Dr. Richard L. Schilsky, Chief Medical Officer of the American Society of Clinical Oncology and Vice-Chair of the Reagan-Udall Foundation for the FDA. “Doctors treating patients with life-threatening illness don’t have time for false starts. They need complete, accurate and easily accessible information”

The American Cancer Society, the American Society of Clinical Oncology, the Biotechnology Innovation Organization, FDA, Foundation Medicine, the Pharmaceutical Research and Manufacturers of America and Susan G. Komen helped develop the Navigator along with the support of Bristol-Myers Squibb, Genentech, Janssen, Eli Lilly and Company, 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.