Driving Evidence-Based Neonatology Forward: A Q&A With Barbara Schmidt, MD

As a student at McMaster University in Canada, Barbara Schmidt, MD, attending neonatologist at Children’s Hospital of Philadelphia, and director of Clinical Research, Neonatology at Penn Medicine, met William A. Silverman, MD, for the first time. Known as the father of neonatal intensive care, Dr. Silverman had given a talk that inspired Dr. Schmidt in its rigorous and questioning approach to newborn research and care: Both Dr. Silverman and Dr. Schmidt believed that nothing but the sharpest evidence should back the decisions we make when treating sick infants. Though she didn’t know it at the time, decades later, Dr. Schmidt would give a lecture on the same subject – one that, in fact, was in Dr. Silverman’s honor.

Earlier this year, the American Academy of Pediatrics recognized Dr. Schmidt with the William A. Silverman Lectureship Award. The award recognizes an individual whose work has significantly advanced neonatal ethics or the field of neonatal evidence-based medicine. As the lead investigator of the International Trial of Caffeine for Apnea of Prematurity (CAP) and a host of other collaborations and clinical trials in newborns, Dr. Schmidt is a natural fit.

CAP demonstrated the important benefits of caffeine therapy for babies with apnea of prematurity, a condition in which premature infants experience difficulty breathing and a higher risk for developing long-term disabilities such as cerebral palsy. Fellow neonatologists praised the study, as exemplified in one such tribute in the journal Neonatology: “It is thanks to the efforts of Barbara Schmidt and the Caffeine for Apnea of Prematurity Trial Group that we now have high-quality and reliable data not only on short-term but also long-term outcomes of caffeine use for apnea of prematurity.”

We sat down with Dr. Schmidt to talk about why the William A. Silverman Lectureship Award means so much to her – and what she hopes to see in the future of both neonatology and neonatal research.

Congratulations on your award! Let’s talk a little bit about Dr. Silverman. How would you describe him?  

People describe William Silverman as the first neonatologist in North America – a pioneer in neonatal medicine who looked after sick and preterm babies before our specialty even existed. He has also been one of the most forceful and earliest proponents of rigorous clinical research, and what we now call evidence-based medicine. That means not just going out and trying something and thinking it will work, but actually doing ethical and proper trials, even in sick babies. Dr. Silverman was a harsh critic of our discipline, but always factual. No fake news here.

Why is receiving the lectureship in his legacy so important to you?

The qualifications of the award are that you have to either have been working academically to move the dial on neonatal ethics, or have been doing something major in evidence-based medicine – something that moves what we know about how to treat babies truly forward. Every award is an honor, but not all awards are created equal. I was particularly happy about this one because of my strong belief in evidence-based medicine and my passion for trying to resolve uncertainty in our management of babies, slowly but surely. I also found it a major responsibility to do justice to Dr. Silverman.

It seems you do justice to him indeed, in the lecture you presented for the award. Your lecture has a very interesting title: “Progress in a Groove?” Tell us about its significance.

“Progress in a Groove?” sounds like an odd title, right? But it’s actually the title of one of the chapters in Dr. Silverman’s book, “Retrolental Fibroplasia,” published in 1980. I borrowed the title, and the only thing I changed was the addition of a question mark. By “Progress in a Groove,” Dr. Silverman meant that tunnel vision, wherein you’re not really seeing the big picture, had characterized our neonatal specialty during its development. Those were the early years of the discipline; it was only around the 1970s that people started to talk about neonatology, and proper neonatal ICUs were set up.

So according to Dr. Silverman, tunnel vision did three things – first, it made clinicians ignore the social consequences of their action: They didn’t pay enough attention to societal issues and the social conditions in which they treated sick preterm babies and into which they released these babies. Second, their research agenda was uncoordinated: Everybody just thought, let me study this or that. And thirdly, the research methods were flimsy, not rigorous enough, and therefore the conclusions were shoddy and often built on sand.

That was basically the outline of my talk. I asked: Have we have improved? What has happened in the nearly 40 years since Dr. Silverman published his critique in 1980? For example, are we better at paying attention to the social consequences of our actions and the way the families live?

Tell us a few examples that you gave about how clinical research has changed since Dr. Silverman’s time.

Well, with respect to the social consequences, the most striking example that Dr. Silverman gave was of a very premature baby in the 1940s or 50s who had finally been sent home to a Harlem flat. Within less than a week, the baby had died at night when a rat bit his throat. Now, I remember reading this as a young person, and it’s a pretty startling example – but not one that seems terribly foreign to me. Babies don’t necessarily die from rat bites these days, but quite frankly, we know that some families we serve here in Philadelphia don’t live in very much better conditions, and that’s concerning. I expanded a little bit on the social consequences in the lecture.

Then, for the coordination of research, I gave some good and bad examples from our field: Here are things we’re doing better, and here are things that we’re still doing in a haphazard way.

And finally, I think the most progress we’ve made is in the third example. Evidence-based medicine is still under constant threat from people who don’t believe in it or just want to push a treatment for fame or profit. But for the most part, there are a lot of good examples nowadays, and we have certainly made progress. I gave some of the better known examples, including one that I’m sure had a lot to do with why I was chosen for this particular award: The International Trial of Caffeine for Apnea of Prematurity. 

That is a wonderful research study. Why do you choose to pursue clinical research, and what keeps you motivated?  

I was always a little bit of a rebel and always questioning something that didn’t make sense to me. So I was very fortunate to come to Canada from Germany to first study, and then teach, at McMaster University at a time when it was the cradle of the evidence-based medicine movement. We had it around us all the time. Doing the Master in Clinical Epidemiology and Research Methods at Mac – and then teaching it and working with very bright people – gave me the tools to ask questions more intelligently.

I always wanted to do the right thing, not just something that the professor said we should do because it was the professor who said it. I wanted to know that, on average, the benefits outweigh the risks, because we have had too many misadventures in medicine and in our specialty. So that was always a passion of mine.