The confirmation of Robert Califf to lead the Food and Drug Administration is among the most consequential recent leadership appointments in Washington. In a polarized Senate, Califf’s past relationships with industry and decisions made under his previous FDA watch were enough to create uncertainty and a close confirmation vote. A parliament of patients, however, would likely have given Califf a more sizable majority based on something in his background that was not openly discussed: He is a believer in elevating the roles of patients in research and the development of new medicines.
A paper trail extending at least as far back as 2014 makes clear that Califf not only shares the view that “patients should be involved in the prioritization, design, conduct, analysis, interpretation, and dissemination of research” — his words in a 2014 paper in the North Carolina Medical Journal — but also recommends prompt action to realize that goal.
Throughout the Covid-19 pandemic, public health officials and regulators — in gaining public acceptance of vaccine safety, for example — struggled with understanding the perspectives of patients. So this is a relevant qualification for an FDA leader, and the sort of detail that should have been considered critical in the nomination process.
No transformation currently on the horizon in drug research and development — and that includes any individual new device or medicine — promises anything like the accelerations, efficiencies, and relevant breakthroughs likely to result from the widespread application of what observers now call “patient centricity.” In a nutshell (albeit a very large nutshell), this means including patients in every significant decision on the road from drug discovery through development and delivery, including their insights about their real-world experiences, needs, and preferences.
In other words, biopharma companies need to listen to patients before they even begin trying to make a new medicine.
By any definition, this is as hard to do as it is important to do.
A certain amount of paternalism likely got in the way of listening to patients in the past — doctors and scientists like to think they know best — but the main reason patient centricity is a new thing and not an old, common-sense thing is the legitimate need to get beyond one-off, anecdotal information. The databases and data analysis tools needed to mine patient data and insights on a population-wide scale simply did not exist. They do now.
In his 2014 article, Califf was endorsing the promise of the National Patient-Centered Clinical Research Network (PCORnet) — a research ecosystem aggregating health data on millions of anonymized patients, early clinical trial results, patient-insights research, and more. The system now demonstrates its impact regularly, not least in a recent study of so-called long Covid involving health records of more than 2 million people.
PCORnet is just one example of the sorts of networked technologies that will drive patient centricity. Others include the FDA’s Sentinel initiative to track the real-world safety of medicines and medical devices, and a growing number of proprietary tools being developed in the private sector.
But the transformation is not just about technology. It has a softer side as well, which is no less important. It will succeed only if drug industry and regulatory mindsets are open to patient-centered decision making. Those changes, too, are happening. In some pharmaceutical companies, the evolution from fielding modest patient advocacy relations teams tasked with seeking patient engagement to restructuring entire R&D and business functions around patient centricity has played out in less than a decade.
The FDA has been active in moving this important change forward. It began holding what it calls patient-focused drug development (PFDD) meetings in 2012, in which regulators hear directly from patients as well as their advocates and caregivers. Concrete examples now abound of how insights from PFDD meetings and other patient-centered research changed R&D trajectories. The agency also is developing guidance on the collection of patient experience data and its use in drug development. When Califf last led FDA in 2016, he said that leveraging such real-world evidence was his “top programmatic priority.”
This revolution is underway, though it needs a few more sparks. Robert Califf as FDA commissioner can be a powerful one. With reauthorization of the federal government’s prescription drug user fee program and a potential 2.0 version of the 21st Century Cures Act in Congress, both on the horizon in 2022, a regulatory leader determined to remove unnecessary barriers to patient-centered drug development will leave a powerful legacy.
Welcome back to the FDA, Dr. Califf. Patients will see you now.
Anthony Yanni is senior vice president and global head of patient centricity at Astellas Pharma, Inc.