In the Age of RTT, FDA Struggles to Keep Its Compassionate Use Program Alive
​[Thursday, November 15, 2018]

Last week FDA announced new ways to strengthen its expanded access program and, in the process, highlighted what an uphill task is has due to competition from the Right-To-Try (RTT) law. FDA released an updated webpage dedicated to the Expanded Access Program. The page describes in detail how “easy” and “fast” it is to request and get approval from FDA for compassionate use of an investigational drug. To be fair, expanded access approval was never hard. FDA has a more than 99% approval rate for requests for expanded access requests such as single-patient treatment INDs and emergency-use INDs. FDA also grants immediate approvals on the phone to many such requests and within a few hours to practically all requests. But RTT law takes out the need for seeking FDA pre-approval for such use of investigational drugs.  The eligibility conditions for the compassionate use program and the RTT are practically identical with one big difference, compassionate use or expanded access requires a physician to formally request permission from FDA, while the under the RTT law such permission is not required. One can argue that if the conditions are the same, RTT offers an easier route to access to an investigational drug. Hence FDA’s dilemma; on one hand it has to follow its time-tested and reliable methods to account for all uses of investigational drugs, while on the other hand it has to abide by the new law that was created explicitly to reduce FDA oversight. Just looking at the expanded access and the RTT pages on FDA’s website show the contrast in FDA’s opinion about the two pathways; the expanded access is “cheerful” and has detailed information and helpful information, while the RTT page is gloomy and has little helpful information other than recitation of the law. Either way, expanded access program is in trouble, thanks to RTT. While currently FDA approval may still be the preferred route for use of investigational drugs in the needy patients, as federal RTT law gain more visibility and patient comfort, RTT will likely be favored by patients.