FDA Widens Expanded Access Navigator as Johnson Presses House to Pass Right-to-Try
Posted 03 November 2017 | By
US Food and Drug Administration (FDA) Commissioner Scott Gottlieb on Thursday announced that a web platform to help patients find information on clinical trials for cancer drugs would be expanded to include orphan disease drugs.
The decision follows a House Energy & Commerce Committee hearing last month on a bill that passed the Senate in August and seeks to speed and increase access to experimental therapies for terminally ill patients.
In written comments to the House committee, Gottlieb said: "If enacted without changes, sponsors and other persons providing eligible investigational drugs to eligible patients under the specified conditions of the bill would not be subject to a number of sections of the FD&C Act and FDA regulations related to clinical trials, premarket approval, and labeling. The current language in S. 204 may therefore preclude FDA from taking enforcement actions based on those provisions."
Critics of the bill also note that it would not require companies developing these investigational treatments to provide them to patients, which is usually the rate-limiting step in accessing the potential drugs, as FDA has approved more than 99% of all expanded access requests it receives.
Gottlieb said in a blog post on Thursday: "FDA is widening the scope of the new Expanded Access Navigator tool, a comprehensive online information resource maintained by the nonprofit Reagan-Udall Foundation to facilitate pre-approval access to drugs. Previously this tool was rolled out for drugs that treat cancer. It will now apply to drugs that treat orphan diseases."
The changes to the Expanded Access Navigator coincide with a push from Sen. Ron Johnson (R-WI) to encourage Speaker Paul Ryan (R-WI) and the House to "promptly take up and pass his bill," S.204. Johnson and Sen. Joe Donnelly (D-IN) also sent a letter on 2 October to the House Energy and Commerce Committee encouraging the House to take up the bill.