CHICAGO – FDA commissioner Robert M. Califf, MD, stressed the importance of relying on data and evidence when approving medications and creating guidelines during a session at the ACC scientific session on April 4.
Califf defined evidence-based practice as the integration of clinical expertise, patient values and the best research evidence into the decision making for patient care. He said FDA labels and ACC/American Heart Association (AHA) clinical practice guidelines should serve as the foundations for evidence-based medicine.
The commissioner also mentioned that precision medicine was similar to evidence-based practice in that they both tailor medical treatment to individual characteristics of patients. However, he noted that doesn’t mean creating medications or medical devices that are unique to each patient. Instead, the goal is to classify patients in small sub-populations that share their susceptibility to diseases, the biology or prognosis of those diseases or their response to a specific treatment. Califf cited a 2011 report from the National Research Council that pointed out the differences between precision medicine and personalized medicine.
“There are many who are advertising that they know exactly the right treatment for each individual,” Califf said. “I would argue that no matter how much data we have, we’ll never be able to do that. But what we can do is recognize the characteristics that groups of people have in common and tailor the treatment to identify the best treatments for those people. That’s what we should be doing.”
Califf also discussed the changing physician model. In the old system, doctors were in charge and worked with patients, but access to the best doctors was limited because of the reimbursement system. In addition, doctors had limited information on medical advances before the Internet and had to rely on print medical journals to learn.
These days, Califf said the FDA decides on the labeling, payers decide what drugs and devices are covered and health systems decide what goes on the formulary or purchasing systems. Doctors also have much more information available on the latest news.
“The question I think before us is, ‘How do we bring up the whole system rather than evolving to the least common denominator?'” Califf said. “There’s a real danger in such a system-based approach that we will go to the lowest common denominator. I would argue if we take advantage of the resources we have now, we can actually optimize what clinicians do by making the best data available at the point care just in time.”
Califf said the ideal system would focus on measurement and education and rely on trials and guidelines. However, he said “we’re falling short with what we could do” and cited a 2009 study that found only 15 percent of major recommendations from the ACC/AHA guidelines were supported by high-quality evidence.
“You’d probably all agree that when it comes to doing clinical research, we have a system that is too slow, too expensive and not reliable enough,” Califf said. “And yet, now we’re surrounded by data. How do we turn this data into information and knowledge?”
Califf reiterated the importance of appropriate FDA labels for medical products based on the best evidence as well clinical practice guidelines based on evidence and input from clinicians on how to use the products in practice.
“These two outcomes should be something that we have completely in mind as we design the research that we do,” he said. “They provide complementary information that in fact are not meant to be identical but should serve each other and then [materials] should be developed along with electronic health records to produce useful ways of people digesting this information as they make decisions, both consumers, patients and practitioners.”
Califf cited a 2014 report that found the price of clinical trials increased at eight times the rate of the general biomedical price index, which is increasing about twice the rate of the general consumer price index. He also said that clinical research sites in the U.S. are working harder even though they’re enrolling fewer patients. More clinical trials are being done overseas, too.
“The way we’re currently doing clinical research is making it way too expensive and way too slow,” Califf said. “We’re making clinical trials more complex than they need to be, largely because the way we’ve thought about doing clinical trials is to create a parallel universe where the study is