Robert M. Califf was commissioner of the Food and Drug Administration from 2016 to 2017. He is a professor of medicine and vice chancellor for health data science at Duke University School of Medicine and an adviser for Verily Life Sciences.
Scott Gottlieb’s unexpected resignation as commissioner of the Food and Drug Administration caused widespread concern that we are losing steady hands on a crucial government agency. Those concerns were allayed by the announcement last week that Norman “Ned” Sharpless, director of the National Cancer Institute, will succeed him as acting commissioner.
Sharpless, no doubt, has the scientific knowledge and leadership skills needed to defend public health and advance science while overseeing and regulating industries that together account for nearly 25 percent of the nation’s economic activity. Nevertheless, strong headwinds await anyone who takes the reins of the FDA in the coming years. The public health community is wading into uncharted territories on many fronts, and it needs strong leadership to guide the way.
Because the FDA’s decisions have enormous downstream effects, they will inevitably be criticized. Gottlieb was savvy in tapping the blend of science, policy and law that has evolved over more than a century to advance the FDA’s mission. His adroit use of social media has provided lucid, accessible explanations of the agency’s purpose, accomplishments and challenges, helping to demystify the factors that shape the FDA’s work. He has enhanced the generation of medical evidence by strongly supporting concepts embedded in the 21st Century Cures Act , including the use of “real-world” digital information gathered outside of the clinic to assess the safety and effectiveness of medical products.
Although the FDA’s ability to affect drug prices is limited, Gottlieb also closed loopholes and supported a systematic approach to evaluating generic drugs that accelerated rates of approval. His forthright approach in attempting to stem the growth of vaping among young people has been refreshing. And he has been an outspoken advocate for the FDA’s workforce and worked to improve hiring and retention efforts for key staff.
Despite these significant achievements, we are also witnessing a perilous erosion in public trust in science as a basis for policy and decision-making. The recent surge in anti-vaccination sentiment, stoked and spread through online misinformation, offers just one example.
We must also come to grips with the fact that while most of our peer nations continue to experience improvements in longevity and health status, the United States — which already has the worst life expectancy among peer nations — has seen declines in life expectancy for three years in a row. These alarming statistics are driven by opioid overdose and suicide, but we are also seeing worsening burdens of heart disease, obesity and diabetes among people with lower incomes and people with less education, with an especially alarming deterioration unfolding in rural communities. Although research investments in cancer and rare diseases, supported by the FDA’s streamlined evaluation policies for new drugs and devices, are yielding dramatic results, we cannot neglect the chronic diseases that account for the bulk of our health problems.
When I interviewed for the job of FDA commissioner with President Barack Obama, he pointed out that the same scientific advances that offer opportunities to improve the well-being of Americans also carry significant risks. The growing list of critical issues facing the agency spans both biological science and digital infrastructure. The recent gene editing in babies in China made headlines, but less attention has been paid to applications of genetic engineering that could dramatically improve nutrition and cure disease — or result in horrendous effects on humans. The physical interdependence enabled by modern transportation continues to raise the specter of antibiotic resistance and new disease outbreaks, threats that demand coordinated action on a global scale. Vaping holds promise for reducing the toll of death and disability from tobacco use, but a massive increase in teenage vaping could open the door to lifelong addiction and a transition to lethal tobacco products. Finally, the misuse of powerful painkillers has led to rampant opioid addiction, devastating families and entire communities.
Meanwhile, the public health field will need to adjust to the digital “fourth industrial revolution.” Ubiquitous networked communications have enabled direct involvement by consumers and patients so that products can be tailored to individual needs and patient advocates and the public can have input into policies. We are also better able to measure the risks and benefits of products in the context of people’s everyday lives. As our country moves toward new systems designed to pay clinicians and health systems based on the value and quality of care provided, the FDA must lead the way to incorporate rich sources of real-world data to measure directly the impact of technologies and products on longevity, quality of life and cost, while ensuring safety and privacy.
Fortunately, FDA staffers and leadership offer a tremendous resource of knowledge in science, policy and legal issues. Sharpless has impeccable integrity and deep scientific insight, and I believe he also has the wisdom to follow Gottlieb’s lead in supporting the work of the FDA’s experienced, knowledgeable staff. So long as he and his successors do that, the FDA will continue to create an ecosystem that shapes technological and scientific innovation to serve human good.