Robert Califf, M.D., the U.S. commissioner of food and drugs from 2016 to 2017, was at the UNC Eshelman School of Pharmacy Monday, Feb. 27, to discuss data sharing and diversity in clinical trials and to host an open forum with faculty and students.
At a luncheon held at the Carolina Club on the University of North Carolina at Chapel Hill campus, Califf spoke about his experiences as head of the Food and Drug Administration and about the need for ensuring diversity in clinical trials, pointing out that the United States accounts for only 4 percent of the world’s population yet is developing drugs and medical products for a worldwide market. Trials need to be faster, less expensive and more relevant to patient populations. Often the participants enrolled in clinical trials are younger and healthier with fewer comorbidities than the patients who will be taking the treatment, he said.
Califf said researchers must work to balance diversity of subjects with scientific need. Diversity may not be as important in a phase I trial when you’re just trying to show that something works at all, he said. Given that over 90 percent of drugs entering clinical trials have unexpected toxicity or fail to have benefit, simply showing some benefit is a “high hurdle,” he said. But once the early phases are positive, the goal should be to conduct highly credible studies designed to be more relevant to the population that will be treated in practice.
The key to improving clinical trials and addressing the hyperinflation of their costs will be the curation, aggregation and sharing of digital data already collected in health care delivery so that the enormous expense of creating stand-alone clinical research system can be avoided. He emphasized the need for health-care organizations to gather and share data in collaboration with their patients, pointing to the Precision Medicine Initiative led by NIH with support from FDA, as a model for aggregating and analyzing shared data sets.
“We’re just getting to the point where we can look at many different data sets as a single entity,” Califf said. “The winning game is going to change from who owns the data to who can analyze the data.”
The aggregation of data collected from treating individuals is also good for making decisions about the health care of populations, Califf said, and we desperately need better data to make better decisions. Less than 15 percent of health-care decisions are supported by high quality evidence, and our goal should be to get to the point where patients and their health-care providers have access to the information they need to make decisions about their own health, he said.
“Active participation of patients is going to be the emancipating factor for the data we need,” Califf said. “Health-care CEOs are going to have to buy in to sharing patient data because the vast majority of American patients want their data to be used to improve health care.”
Second-year Doctor of Pharmacy student Frank Tillman introduced Califf at the luncheon and spoke about the need for diversity in both clinical trials and in the pharmacy and pharmaceutical professions.
“As a student pharmacist, I have learned that being a minority or of a specific gender are risk factors in themselves that oftentimes lead to higher incidences of morbidity mortality,” Tillman said. “The question for us to think about, as leaders within our respective professions, is where are we currently in terms of providing holistic care to a diverse, global population?”
In addition to his presentation at UNC, Califf toured the Biomanufacturing Research Institute and Technology Enterprise facility at North Carolina Central University and met with BRITE faculty and NCCU Acting Chancellor Johnson Akinleye, Ph.D. At UNC, he met with Provost James Dean, Ph.D., and Vice Chancellor for Research Terry Magnuson, Ph.D., and hosted an open forum with pharmacy faculty, students and staff at the pharmacy school.
Califf was head of the U.S. Food and Drug from February 2016 to January 2017. Before joining the FDA, he was a cardiologist at Duke University from 1980 to 2015 and served as Duke’s vice chancellor of clinical and translational research.