The past two decades have witnessed the evolution of patient engagement as a priority at the Food and Drug Administration (FDA) in the US. Since the first patient group was established for HIV/AIDs patients in 1988, the FDA has been slowly welcoming patients into the fold. Highlights include the first Patient Council meeting held in 2016, and last year’s inaugural patient advisory committee.

Those developments continued this year with an unequivocal statement from FDA Commissioner Scott Gottlieb, M.D., on new agency efforts to advance the patient voice in medical product development and FDA regulatory decision-making. The statement also announced the publication of new draft guidance on patient-focused drug development.

“We learn through scientific advances, but also by listening to patients,” said Gottlieb. The FDA’s work demands that they continue to reflect on how they can make the science of drug development and review more modern and more patient-centered, he noted, “so that approved products impact the metrics that real-world patients and families value most”. This will require ongoing engagement with the patient community, he added.

“Done well, the result is more and more of our review of new medical products benefits from a better understanding of the patient’s experience, providing our reviewers with the critically-important context of a disease, and helping them to understand what’s most important to patients related to treatment benefits, risks and disease burden.”

Gottlieb outlined how the FDA has made it a priority to work with companies and other stakeholders on gathering information from patients about their views and needs, and on building the tools that are required to capture patient input in a way that provides meaningful data.

“The Administration has to date held Patient Focused Drug Development (PFDD) meetings in more than 20 disease areas where we’ve heard directly from those impacted by diseases, including opioid use disorder, autism, HIV, Parkinson’s disease and various conditions involving pain.

“These meetings have given the FDA’s professional staff a deeper understanding of patient and caregiver experiences. We have gained from this experience,” wrote Gottlieb.

He also stressed the importance of educating companies about rigorous approaches to obtaining and incorporating this “important and unique input into product development”, and explaining how the FDA will incorporate this information into our regulatory decision-making.

The new draft guidance stems from what was learned at these intensive meetings. It is just the first of a series of four methodological PFDD guidance documents that the FDA is developing. The document will address how stakeholders, including patients, researchers, medical product developers and others, can collect and submit patient experience data and other relevant information from patients and caregivers for medical product development and regulatory decision making.

This newly-structured approach to patient engagement and the incorporation of patient insight into clinical trials and product development in a truly meaningful way mirrors what PFMD has been working towards since its inception. PFMD endorses the work of the FDA in bringing patients into the regulatory fold, and also their bids to liaise with all stakeholders in the drug development process.