By: Mark Terry
Project Facilitate is a pilot project to create a single point of contact for the FDA’s oncology expanded access requests. In order to do so, the FDA will run a call center to help patients and healthcare providers work their way through contacts at drug and biotechnology companies, complete Form 3296, the expanded access request form, and identify Institutional Review Board (IRB) resources.
“Access to clinical trials and access to novel therapeutics for patients is still a problem,” stated Acting FDA Commissioner Ned Sharpless. “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.”
The FDA indicates that it expects the call center to be opened by the end of May.
Part of Project Facilitate is that the agency will gather data as to whether a company actually offers a drug, and if it doesn’t, document the company’s reason for declining to offer the drug. It will also work to make sure that patients or their healthcare providers provide accurate data to the companies about the effectiveness of the drugs.
“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,” stated Jessica Boehmer, a regulatory scientist at the Office of Hematology and Oncology Products.
The FDA is being careful about creating more work for oncologists and hasn’t yet made plans for how often it will report on the data it gathers during the program. OCE Deputy Director Gideon Blumenthal stated that the FDA wants to “balance being least burdensome for busy practicing oncologists and their teams with capturing good data.”
At this time, Project Facilitate is only focused on cancer-related access programs. It is also not currently involved in medical devices and in vitro diagnostics. Because it is a pilot program, it is launching with a limited design, but could be expanded later. The agency also indicated it would like to eventually create an electronic version of Form 3296, but it also feels the human interaction is important.
“One cannot underestimate the need—if somebody has a problem—to actually call somebody to walk through the system,” stated Richard Pazdur, director of FDA’s Oncology Center of Excellence.
At the meeting, Pazdur also mentioned that some companies are not in compliance with the 21st Century Cares Act’s requirement for them 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.”
The FDA, under Commissioner Scott Gottlieb, expanded its “compassionate use”, or expanded access program, offering a possible route for a patient with an “immediate life-threatening condition or serious disease or condition” to gain access to an investigational medical product in May 2018. In the same month, Congress passed the “Right to Try Act,” which amended the Federal Food, Drug and Cosmetic Act. Patients can request the drugs from the drugmakers if they have been part of a small-scale Phase I clinical trial and are still being considered by the FDA.
It’s not as simple as just asking for an experimental drug and being handed a bottle of pills. Biopharma companies in early stages of trials have very limited amounts of the drug which are typically manufactured in very small batches under strict conditions. In the past, companies have been reluctant to offer drugs outside of a clinical trial for fear of it undermining the trial data or exposing the companies to possible litigation, or in some cases, creating a flood of patients demanding the drug.
Overall, though, the industry seemed to believe the FDA’s activities in response to the Right to Try Act were positive, taking steps to reduce the administrative burden and create streamlined processes and a central point of contact.
Barbara Jacoby is an award winning blogger that has contributed her writings to multiple online publications that have touched readers worldwide.