The Food and Drug Administration (FDA) is to focus on giving guidance for how to collect patient data systematically and use it in clinical trial design, as well as continuing to participate in externally-led patient focused drug development (PFDD) meetings, now that its own PFDD meetings have come to an end.
Launched in response to the Prescription Drug User Fee Act (PDUFA) V as a means of strengthening engagement between the FDA and patients, the FDA ran 24 PFDD meetings over 5 years, with the final one on September 25 focusing on hereditary angioedema.
Like its predecessors, the meeting gave patients a platform to share their perspectives directly to representatives from the FDA, including the impact of their conditions on their lives and what they want from current standard and future therapies. Now that the FDA meetings have finished, the focus is on what the FDA will do with the patient input it has gathered.
Guidance on using patient data
Speaking during a panel session at the recent BIO Patient & Health Advocacy Summit on October 2, Pujita Vaidya, Team Acting Director at the Center for Drug Evaluation & Research Decision and Support Analysis, said that the agency will now look into how patient data can inform four key FDA guidances.
According to Vaidya, these guidances will likely take 4 to 5 years to create but will leave “everyone being able to really understand what FDA wants and where folks need to be focusing.”
The guidances will focus on four areas:
- Who to talk to during the development stages of a new therapy or tool
- What questions to ask patients to identify what has the greatest impact on disease and treatment burden
- What to measure that might be able to go into a clinical trial
- How to include patient information in trial designs
Externally-led meeting attendance
The FDA will continue attending PFDD meetings that are led externally, as well as encouraging the participation of treatment developers.
“Along with the FDA staff, I do see this being an effort where we want drug developers in the room themselves,” explained Vaidya. “The input that we’re hearing and the information that we’re getting from patients is very important. When product developers are hearing this very early on, it helps them to think about them with trial design.”
Also present at the BIO meeting was Theresa Mullin, Director of the FDA Office of Strategic Programs. Speaking during a fireside chat, Mullin emphasized the importance of patient input into the medicine development process:
“One of our takeaways is not only do patients have a crucial expertise and perspective to inform drug development and decision-making, but in addition to inform the therapeutic context [in the risk/benefit framework]. Ideally, we’d be getting these measures of what they care about into the drug development program.”
The FDA’s next immediate focal point though will be on meeting the deadlines for the four guidances, said Mullin. As laid out in PDUFA VI, a draft for the fourth guidance is due by the end of 2021.