FDA's Califf Tells Fellow Cardiologists How Precision Medicine Can Reshape Clinical Trials

In recent months, his duties have spanned high-priced cancer drugs to food safety, but on Monday FDA Commissioner Robert M. Califf, MD, MACC, enjoyed a warm welcome from the community he knows best—his fellow cardiologists.

“It’s great to come back to cardiovascular medicine after a few months in the world of all medicine,” Califf said, as he began the Eugene Braunwald, MD, Lecture at the 65^th Scientific Session of the American College of Cardiology, which concluded in Chicago, Illinois. In February 2016, a year after he was nominated by President Barack Obama, the founding director of the Duke Clinical Research Institute was confirmed by the Senate in an 89-4 vote.

Califf was introduced by Mount Sinai’s Valentin Fuster, MD, PhD, editor of the Journal of the American College of Cardiology, and enjoyed an onstage greeting with the 86-year-old Braunwald, who fled Hitler’s arrival in his native Vienna and went on to mentor to generations of cardiologists, including Califf. It was clear the nation’s top food and drug regulator enjoyed this moment among friends.

Still, he used the lecture to describe key components of FDA’s agenda: to promote precision medicine, and to use patient medical records to reshape a core component of cardiology—the clinical trial.

He started by laying out the principles of his mentor: Braunwald, he said, was devoted to clinical practice at a time when the stars of his generation often were not; he advocated taking time to scrutinize problems and looking intensely for solutions, then communicating findings and developing allies who carry forth the ideas.

That philosophy is needed today as medicine looks for new ways to advance evidence-based practice. Solutions can be found in precision medicine, Califf said, which isn’t as new as some believe.

Clinical trials have become so expensive that more and more of them are happening outside the United States, Califf said. This raises questions of whether researchers can bring down the cost and time involved by harnessing the information being gathered in electronic health records (EHRs). Social media also gives researchers the ability to gather patient feedback, he said.

“We are going to be swamped in data—the question is, who can make intelligent information and knowledge out of that data?” Califf asked.

The future of evidence-based practice, he said, will involve integrating these elements of clinical expertise with patient values and research evidence, to create the best possible decision-making process for patient care.

FDA laid the groundwork for this, Califf said, when it created the Mini-Sentinel system, which gathered information on 100 million lives to act as an early warning system on drug and device safety. This system now has data on 190 million lives.

Together with NIH, FDA has just proposed a draft clinical trial template, which would standardize the presentation of data for phase 2 and 3 trials and make it easier to use data within them. And, Califf said, “We’re asking Americans to put their data into a system that will have a number of measurements that will be made available to researchers.”

Rather than have this system organized centrally, he explained, more than one coordinating center would be involved in managing the data, depending on the question that needs to be answered. This way researchers could tap data gathered not only by NIH or FDA, but also healthcare giants like Kaiser Permanente, he said.

With virtually everyone possessing a cellphone, he said, information sharing has the potential for physicians to gain access to data to make decisions, and deploy it just as quickly. “There’s the ability for the practitioner, with minimal cost and minimal work, to engage patients.”

Direct information sharing, he said, could lead to the end of drug advertising, he said. “The role of opinion and expertise would be even more important, but it would be put to a much higher purpose.”

During a question-and-answer session, Califf was asked how to balance calls for data transparency with concerns that some will misuse it. He said an open system is very important, even though there will be a “rocky period” as academics and industry professionals establish methods to police themselves.

“If data is too protected,” Califf said, it can take years to find out it is being misused. “When people are doing bad things with data, we need to hold them publicly accountable.”