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 kept entirely separate from clinical practice.”
Califf said that electronic health records for each patient would help researchers analyze data and identify trends that physicians could use in practice. He applauded the ACC and AHA for creating registries that gather clinically relevant information and inform practice, but he said more needed to be done to help cardiologists fit into integrated health systems.
In May 2008, the FDA created a database known as the “Sentinel System” that now includes claims data, prescriptions and clinical outcomes from more than 190 million people. Califf said the FDA uses the database every day to monitor safety issues with medications. Califf also said that starting next year, pharmaceutical companies would be required to submit clinical trial data to the FDA with common data standards.
Califf said the next step for the FDA is to combine the elements into a unified national system. As of now, there are a few initiatives examining evidence-based research, including the Patient-Centered Outcomes Research Institute, which is funded by taxpayers and insurance companies and encompasses 40 percent of U.S. health systems.
President Barack Obama and both political parties have endorsed a precision medicine initiative. Over the next year, U.S. residents will be asked to volunteer their electronic healthcare data into a system, which will be made available to all researchers who have proper credentials.
In the future, the healthcare industry will focus on integrated health systems, better data and the ability of physicians to engage patients in a learning system with minimal work and costs, according to Califf.
He also would like to see no more advertisements for medications and in health systems, which he said doesn’t provide useful information to patients. In the idealized system, he said physicians could rely on quantitative information to give people the best choices and tailor their treatments instead of being influenced by advertisements.
“If we do the right things over the next several years, you and your colleagues, when you see patients, will be able to focus on their decisions, how to implement the knowledge that you already know exists instead of so often having to guess and just do the best you can without really knowing what the best thing to do would be,” Califf said.
Califf delivered the Eugene Braunwald Lecture, which is named after the famous cardiologist from Harvard Medical School and Brigham and Women’s Hospital in Boston. Before joining the FDA in February 2015, Califf was a cardiologist, vice chancellor of clinical and translational research at Duke University and founder of the Duke Clinical Research Institute. The Senate confirmed Califf as FDA commissioner on Feb. 24, five months after President Barack Obama nominated him to lead the FDA.