RTT Fizzles While FDA Increases Support for Compassionate Use Program 
[Thursday, June 6, 2019]

A year after the federal Right to Try (RTT) Act became law, there have been only 2 reported cases where the law was used to provide treatments to patients. During the same time hundreds of compassionate use INDs were approved by the FDA. It is no secret that the RTT was designed to circumvent FDA review, and that FDA does not “like” it. While FDA has not taken any measures to move an inch beyond what it is forced to do regarding the implementation of the RTT law, it has steadily increased support for its compassionate use program by making it easier to file an application for compassionate use, also known as expanded access, and approving almost all requests received. This week FDA announced a special call center to help oncologists file compassionate use INDs. The initiative, termed “Project Facilitate”, will create a “single point of contact where FDA oncology staff will help physicians treating patients with cancer through the process to submit an Expanded Access request for an individual patient, including follow-up of patient outcomes.” There are no new changes to the expanded access program; only physicians can file such an application and work directly with the manufacturers to get their patients access to experimental drugs, and companies can decide whether to provide the drug or reject the request. FDA has also announced that it will encourage sponsors to relax the entry criteria for trials so more patients can become eligible to enroll in a given trial. For all intents and purposes, the expanded access program and RTT have similar goals, namely providing not yet approved products to patients with life-threatening diseases who have exhausted all other options. But there is one big difference; RTT does not require getting pre-approval from FDA for such uses, and RTT technically allows a manufacturer to charge for the experimental drug. It should be noted that so far, no company has charged for providing its experimental drugs under the RTT laws. The main purpose of the new initiative seems intended to make it so easy to file for compassionate use that RTT becomes an obsolete law, never used. It is surprising that the RTT activists seem to have lost steam after creating an unprecedented momentum to support RTT where almost 40 states passed versions of the law before it was enacted at the federal level. Before the RTT law was passed, it was projected that numerous patients needed and were interested in getting experimental drugs via RTT to avoid the delays caused by the FDA permission process, but it is obvious the demand for RTT use has not materialized. An alternate explanation could be that unlike the compassionate use program where FDA maintains a list of how many requests are received by the Agency, for RTT no such list of requests exists. Patients directly approach companies and we only hear about it when a patient is treated. Similarly, it is possible that while FDA has approved hundreds of applications for compassionate use and will keep doing it, the number of patients actually getting the drug under this program may be a lot lower as companies can still reject the request. Either way, FDA keeps chipping away at the RTT law by creating such practical programs that make RTT unnecessary.