Who is Amy Abernethy and what does her FDA appointment mean for health data?
Amy Abernethy, M.D., Ph.D., a Duke University professor and chief medical officer of the much-discussed Flatiron Health, will join the U.S. Food and Drug Administration as deputy commissioner, reporting to commissioner Scott Gottlieb, according to an internal memo.
Abernethy is an oncologist and data scientist whose career has been focused on bridging the distance between treatment and research. Her appointment to the second-highest position at the FDA is a good indication of the agency’s position on the importance of data in healthcare.
Dr. Abernethy spent 25 years at Duke University where she directed the university’s cancer research program and cancer institute. She also served as a palliative care physician, prolific researcher and advocate of TK. In 2014, she moved to the health-tech standout Flatiron Health as chief medical and data science officer, leading the company’s efforts to collect and clean real-world cancer data for use in treatment and research.
“Dr. Abernethy brings to the FDA deep experience in evidence generation and clinical trial efficiency and innovation, which will help us advance our clinical policy and programs,“ Gottlieb wrote in the internal memo.
Flatiron was started by two entrepreneurs in 2012 and purchased by pharmaceutical giant Roche in February of this year for $1.9 billion. The company’s stated mission, and Abernethy’s expertise, lies in unlocking the value in real-world data, or data derived during care and treatment of patients rather than carefully designed clinical trials. The challenge is to get the data to a standard where it can generate real-world evidence that’s on par with the results from clinical trials.
Abernethy’s work at Flatiron was in leading the process of collecting and cleaning data produced during oncology care for that purpose. That data have been used in research, and in some cases, duplicated the results of clinical trials.
In fact, Flatiron and the FDA have been collaborating since 2016, using the data to produce research that could potentially satisfy regulatory questions. In one study, researchers from the FDA and Flatiron found that lung cancer patients treated with Opdive and Keytruda tended to be older than the population in the clinical trials.
This is in line with the FDA’s embrace of real-world data and evidence. Early in December, the FDA released a framework for working with real-world evidence, as it was mandated to do in 2016 by the 21st Century Cures Act. The framework outlines new ways for real-world evidence to satisfy regulatory questions, such as whether a drug that’s already on the market can be used on additional populations or in different dosages. It won’t, as yet, be used during the initial approval process.
“As the breadth and reliability of (real-world evidence) increases, so do the opportunities for FDA to make use of this information,” Gottleib said in a statement in the 40-page report.
Abernethy worked closely with the FDA during their collaboration and has referred to Rob Califf, M.D., the former FDA commissioner under President Obama, as her mentor. She has frequently worked with Sean Khozin, M.D., MPH, the head of the FDA Oncology Center of Excellence, who spearheaded INFORMED, a joint initiative at the FDA and Department of Health and Human Services that’s “designed to harness the power of big data” for better health outcomes.
While the FDA’s focus has been on the application of real-world data to research, Abernethy has long advocated for that data to be channeled back into patient care as well. During a TedMed talk in 2013, Abernethy talked about the importance of patients having control over their own data. A melanoma patient she was treating participated in a clinical trial, that later fell apart, but the researchers wouldn’t release her data, locking up information that could have led to better understanding of the disease.
In 2014, Abernethy’s father was diagnosed with a rare type of cancer and declined to accept an experimental treatment, part of a clinical trial, because it would have meant he would be away from family and friends. In an opinion piece for the Wall Street Journal, Abernethy and Khozin argued that better study designs that incorporate real-world clinical care would allow patients like her father to participate from wherever they are.
Abernethy will begin her role at the FDA in early 2019, replacing Rachel Sherman, M.D., MPH.
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