As expected, the Reagan-Udall Foundation for the FDA has opened its Expanded Access Navigator, meant to streamline information-gathering for patients and physicians who want access to drugs still in the development phase. “Expanded access” (aka, in other contexts, as “compassionate use,” “named patient” and other terms) has become something of a political hot potato as legislators in the US argue, under the “right to try” claim, for reducing or removing access barriers to drugs after Phase I safety trials have been successfully concluded. FDA Commissioner Scott Gottlieb isn’t a proponent of such a drastic loosening of the drug-approval process, but has made statements that the process should be less expensive and time-consuming.
The new Navigator site has a list of drug manufacturers’ expanded-access programs (currently, 34 are listed); there were some indications early on that it would include a comprehensive list of drug trials, but that is not a part of the service as currently structured. (Users are directed to the ClinicalTrials.gov site for that information.) In general, requests for expanded access need to begin with a patient’s physician and subsequently get reviewed by an institutional review board; FDA has a say, but it notes that it has approved over 99% of recent requests, and has streamlined its emergency-status review process for life-or-death situations. On the manufacturer side, companies have the option of including an expanded-access process as part of a clinical trial protocol, or handling requests on a one-off basis. Manufacturers are not obligated to fulfill a request, and many do not, in part because of concerns over how the outcome of the expanded-access use might affect the drug approval process.
In an FDA announcement, Richard Moscicki, MD, FDA deputy center director for science operations, notes that the new site “also offers a great deal of useful information about participation in clinical trials, which is the preferred option. Clinical trials help to ensure adequate patient protection and may provide the evidence of safety and effectiveness required to support approval of a marketing application.”
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. The Reagan-Udall Foundation for the FDA is an independent, nonprofit corporation created by Congress to advance the mission of FDA.