FDA to Launch Expanded Access Pilot ‘Project Facilitate’ by End of May
Posted 16 May 2019 | By
Ahead of the launch of a new pilot aimed at improving patient access to unapproved oncology drugs, dubbed Project Facilitate, the US Food and Drug Administration (FDA) held a public workshop on Thursday explaining its goals for the initiative and to discuss ongoing issues related to expanded access.
According to Richard Pazdur, director of FDA’s Oncology Center of Excellence, the pilot will be rolled out towards the end of May.
“Access to clinical trials and access to novel therapeutics for patients is still a problem. We know that not all oncologists or healthcare providers have the regulatory expertise or resources to navigate the single patient [investigational new drug] IND process, and I know many oncologists just assume the process is cumbersome without even trying it,” said Acting FDA Commissioner Ned Sharpless.
The goal of Project Facilitate is to address those issues by providing a single point of contact for oncology expanded access requests within FDA.
Under Project Facilitate, FDA staff will operate a call center for oncology expanded access requests that will help patients and healthcare providers find expanded access contacts at drug or biotechnology companies, complete the expanded access request form (Form 3296) and identify Institutional Review Board (IRB) resources.
Sharpless also added that FDA will gather information about whether a company provides a drug or not, and if not, try to document the company’s reason for declining a request.
“Project Facilitate staff will also follow up with the healthcare provider, or their designee, to remind them to submit the required summary report on whether the patient received benefit from the treatment and if there were adverse events,” said Jessica Boehmer, a regulatory scientist at the Office of Hematology and Oncology Products.
When asked about FDA’s approach to gathering and reporting data via Project Facilitate, OCE Deputy Director Gideon Blumenthal said the agency wants to “balance being least burdensome for busy practicing oncologists and their teams with capturing good data,” adding that the agency doesn’t have plans for how frequently it will report on data gathered during the pilot.
Responding to several questions asked during the workshop, Pazdur expanded on some of the limitations and potential future areas of development for FDA’s expanded access program.
When asked why medical devices and in vitro
diagnostics are not part of the pilot program, Pazdur said “maybe in the future,” but pointed out that the program is entering its pilot phase and was not designed to manage those products. Pazdur also noted that the program is limited to a single therapeutic area and could be expanded to other life-threatening diseases.
Pazdur also commented that he would like to see the agency develop an electronic version of Form 3296 but emphasized that an electronic form will not be able to replace human interaction when issues arise.
“One cannot underestimate the need—if somebody has a problem—to actually call somebody to walk through the system,” Pazdur said.
Another issue Pazdur raised is that some companies are still not in compliance with the requirement under the 21st Century Cures Act
to publish their expanded access policies on their websites.
“Many of them still don’t have the policies on the website. Maybe it’s because they are too new or start ups … there are a lot of companies that still don’t know about the requirements,” Pazdur said.