FDA further announced at a public workshop on May 16, 2019 that as part of its new program FDA will start contacting drug sponsors to collect metrics on whether a request for expanded access was granted, and, if not, the rationale for any denial. When a sponsor grants an expanded access request, FDA stated it will also collect data from physicians to determine if their patient benefited from the investigational therapy or whether it caused adverse effects.
In opening remarks to the workshop, Acting FDA Commissioner Dr. Ned Sharpless said that he hoped the new pilot will create more clarity and transparency around the expanded access process and will encourage drug sponsors to provide access to investigational treatment options.
There are a number of questions that companies should consider with respect to FDA’s new pilot. Importantly, it remains unclear what FDA intends to do with the data it will be collecting from this pilot and what implications, if any, this data will have on the expanded access process and new drug reviews more broadly. When asked about this at the public workshop, Gideon Blumenthal, Deputy Director of OCE, noted that FDA does not have a strategy yet as to how frequently FDA would report the data publicly and that the Agency is open to receiving feedback on this point. Additionally, during his general remarks, Director of OCE, Richard Pazdur, stated that the FDA is looking to bring equity into the expanded access process to understand why some patients are denied access while others are granted access. Specifically, he pointed to an example where 100 patients might be denied for a specific therapy but the 101st request could be granted. He also pointed out that many companies are still not compliant with the requirements under the 21st Centuries Cures Act (Cures Act) and do not have expanded access policies publicly available.
Brief Background on the Cures Act
As we have previously reported, under section 3032 of the Cures Act, manufacturers and distributors of investigational drugs for serious diseases or conditions must publicly post their expanded access policies for individual patient access. The policies must include procedures for the public to submit expanded access requests to the company and the company’s general criteria for evaluating and responding to such requests. The deadline for compliance with the Cures Act was on February 11, 2017, and applies to investigational products upon initiation of a Phase II or Phase III study for that drug.
FDA has stated that “[p]osting this information on the Reagan-Udall Foundation’s Expanded Access Navigator, including links to a company’s own web page describing its policy for the investigational drug, can be used to satisfy this requirement.”
We note at the outset that nothing under current law forces companies to make their investigational drugs available in response to individual requests for expanded access. However, drug manufacturers of investigational oncology products should be on notice that FDA has indicated that it will call companies directly to document whether these requests have been granted and why a request has been denied. While it is unclear how this data will be used and whether it will be publicly available, it is likely FDA is taking this action to put pressure on companies to have clear expanded access policies in place to enhance compliance with the Cures Act and to increase access to investigational products. We also note that while the pilot is limited to oncology drugs only, FDA noted at the workshop that it is possible that the pilot call center would expand to include other therapeutic categories. Accordingly, companies should anticipate and consider preparing for how to handle these conversations with FDA and whether potential revisions to their expanded access policies are warranted.
If you have any questions about the new pilot program or need assistance with drafting a compliant expanded access policy, please contact one of the authors of this alert or the Hogan Lovells attorney with whom you regularly work.
Authored by Robert F. Church and Jane Kalinina