Clinical Trials and This Practicing Rheumatologist

By: Herbert S. Baraf, MD, FACP, MACR
Featured Article: Sjorgren’s Foundation Quarterly 

With a career spanning almost forty years, it’s time for me to look back upon those things that have given me professional satisfaction and draw advice from those experiences to pass on to my younger colleagues. I have worked in a private practice setting since completing a rheumatic disease fellowship at Duke University in 1978. Duke, a premier center for basic and clinical research for decades, was a great place to be. As a fellow, however, my goal was to take care of patients, eventually in a community practice setting, and not to become an academician or a researcher.

Like many colleagues interested in gaining clinical skills from a premier university whose focus and reputation are based on its research “chops,” I found myself “hiding under the radar. ”My hope was to avoid appearing disinterested in a pathway towards an academic career, all the while clearly focused on leaving to begin a practice. 

In my final year of fellowship, I was required to choose a research project and pursue its goals to completion. As I had no experience or interest in “bench” research, I sought out a clinical project. Duke is, and has been, a leading center in uric acid research and the treatment of gout and hyperuricemia. As a Southern medical institution, it served an interesting clientele with a storied culture, at least from the perspective of a city boy from the Northeast.

There was a tradition in the more rural areas of North Carolina of brewing whiskey locally. This tradition antedated prohibition. The so-called “moonshine” produced in home-fashioned distilleries, or stills, was the favored source of alcohol by some and had at least been sampled at one time or another by a large portion of the population. The distillation process was, and likely remains, not up to the standards of the major commercial distilleries. This was reflected in the presence of certain contaminants that entered the finished product – particularly lead – during distillation.

Lead in moonshine had been imputed to be the reason why so many who consumed moonshine in North Carolina developed gout. The focus of my research project was to determine whether moonshine consumption caused chronic lead intoxication which then led to gout. What I found was that gout, in the 38 patients I studied, was more likely related to the alcohol in moonshine and not the lead. Only 2 of the 38 patients had evidence of elevated lead stores.

Thus began my initially reluctant career in clinical research. In 1978, I entered private practice in 1978. Four years later, I participated in my first clinical trial. It was a study of a new NSAID for rheumatoid arthritis. Ironically, as of this writing and since that first trial, have been involved in more than 400 clinical trials in rheumatology, mostly as a principal investigator. What had been a reluctant first step to engage in clinical research, became something of an obsession, not to mention a major source of satisfaction in my work.

During the ensuing years, I have been witness to the development of important therapies that did not exist at the start of my training. Even more rewarding, I have contributed to the development of many of these treatments.

My years in rheumatology have seen rheumatoid arthritis treatment move from gold salts to methotrexate and then to biologics. Aspirin and indomethacin have given way to safer and more convenient antiinflammatories (the debacle of the Vioxx withdrawal notwithstanding). Revolutionary new treatments for gout and osteoporosis, improved options for lupus, for Sjögren’s, psoriatic arthritis and ankylosing spondylitis have all become available. There is barely an area in rheumatology that has not been affected by the work that thousands of others like myself have done in the clinical trials arena.

The personal rewards of engaging in clinical trials are enormous. My clinical trials experience has expanded my horizons and given me a national and international perspective on rheumatology and the varied work that rheumatologists do in their clinics. Further, I have worked closely with peers from all over this country and can point to friendships I’ve developed with at least one rheumatologist in almost every state of the Union. Rheumatologists who participate in clinical trials are extremely well networked.

Important relationships with scientists, statisticians and investigators in industry have also grown from my years as a clinical trialist as well as productive associations with highly respected academicians in our field. We all learn from one another, clinicians, academicians and physicians and other scientists in industry about the trials process and the science and utility of new treatments. Further, to do clinical trials provides important perspectives on the importance of sound study design –not just for a specific study but for the broader world of therapeutics in rheumatology. Thus, participating in an active clinical trials program has enriched my experiences in rheumatology and broadened by horizons.

Perhaps most importantly, on a day-to-day basis, being involved in clinical trials has improved the care that I provide. When there are no viable existing treatment options, participation in a clinical trial has given patients new and effective therapies. In many instances, the clinical trials program in our practice has provided our patients with access to expensive new treatments which would otherwise be economically prohibitive. In addition, my participation in clinical trials has enabled me to confidently offer newer treatments to my patients, as I have frequently had significant experience with such
treatments prior to their approval.

As a result of my involvement in clinical research, I have had the opportunity to present results at national and international meetings in poster and in oral presentation formats, and I have authored or coauthored several papers that were published in peer-reviewed journals. These activities have been particularly satisfying to me and valuable to the wider medical community.

From a humble start, hoping to just take care of patients, I have participated in the struggle to make my patients’ lives better one at a time. By being part of the clinical trials process, a clinician’s reach extends beyond the lives of his/her patients to the lives of all patients with rheumatic disease.

Given the progress in immunology and rheumatic disease research during the last 40 years, one can only imagine what is to come in the next 40. The Sjögren’s Syndrome Foundation is aware of at least 9 companies developing therapies for Sjögren’s Syndrome alone. Who is going to do that work? Work that is professionally rewarding. Work that amplifies the good we hope to do in our rheumatology practices and extends it to rheumatic disease patients everywhere.

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