The FDA is planning to launch a new program later this year which will streamline the expanded access application and review processes, so that patients can more readily receive access to promising investigational therapies. The program, which has been dubbed Project Facilitate, will see the FDA taking on a more involved role, helping drive the process, from the point an initial request is made, right through to when a drug is delivered and administered.
Under Project Facilitate, the FDA will provide a telephone number which patients and physicians can use to call the agency and request compassionate use access to a given drug. The FDA agent receiving the call will complete the form required for single-patient IND requests and will then send it to the physician for signature, before forwarding it on to the drug manufacturer.
Just like under the current expanded access framework, manufacturers will continue to have the discretion to approve or deny requests as they see fit. However, for the first time, they will now be required to respond to requests within a specified period of time (not yet determined by the FDA) and will need to provide a reason for denying access.
If the manufacturer agrees to provide a drug, the FDA will then be responsible for reviewing the application and sending it on to an Institutional Review Board (IRB). The FDA and IRB review processes, which are no longer required under recently approved Right to Try legislation, are important for ensuring patient safety and wellbeing are protected. Both entities can request changes to be made to the application before granting approval, for example tweaking the prescribed dosing or intended regimen.
Once an application has been approved, the FDA will ensure that the drug is made available to the patient in a timely manner and will contact the physician or patient to get some basic information about outcomes.
The FDA hopes that Project Facilitate will not only make it easier for patients and physicians to request access to unapproved products but will also highlight its support for appropriate and safe pre-approval drug use, which should provide greater incentive for companies to grant access. The agency recognizes that companies may be more reluctant to say no to the FDA than they would to an individual patient or physician. As such, FDA Commissioner, Scott Gottlieb, has said that he hopes this new initiative will “help tip the scale in favor of expanded access.”
Furthermore, being at the center of the expanded access process will give the FDA greater insight into drug company behavior and outcomes. Under the new program, the agency will be able to track the number of compassionate use requests a company receives and the reason(s) it uses for denying access. With this improved insight, if the agency finds a company is receiving numerous requests, it could then recommend that the company open an expanded access protocol, and work with the company to go about doing so in a way that does not hinder ongoing clinical trial efforts.
The FDA hopes to launch a pilot version of the program, which will focus on cancer, in the first half of 2019. The plan is to then review and later expand to all indications.