The Joy of Clinical Trials.

Abstract

“But I only received the placebo,” commented a research participant sitting next to me during our trial update and appreciation event. During an evening when the positive results of the active intervention arms were being celebrated and research participants were ecstatically sharing their trial experiences, his disappointment caught me off guard, giving me pause. When it was my turn to take the stage and present the trial update to the group, I took a detour and first asked for a show of hands to demonstrate which arm they believed was most important in the study—the active intervention arm(s) or the placebo arm. The vast majority raised their hands for the active interventions. And that is when I shared the enlightenment inspired by our brief interaction—that those in the placebo arm represent, for patients around the world, the very best care that we have in the present, while those in the active intervention arm are testing our hopes and the unknown. It is only with both, equally, that we can map our collective path forward toward a better future. My introduction to clinical trials began with the most generous mentors, who openly shared their world and provided the freedom to explore with the hope of seeing a spark ignite. I found myself gravitating toward the collaborative spirit embraced in clinical trials. I did not realize that when patients accepted the invitation to become research participants, we would learn as much from them—their individual stories, past struggles, and future hopes—as from their data. It has been transforming to experience the dedication of our research participants, to realize that it is our mutual commitment to each other that enables us to learn the full truth of what we set out to answer. Once we, both investigators and participants, commit to the journey of a clinical trial, there is a certain element of being all in, of committing to learning the answers together, whatever they may be—whether we uncover exciting advances in therapy or come across an unanticipated safety issue that we need to investigate. While research participants represent future patients, as an investigator, I have come to appreciate that I too represent physicians who face the decisional dilemma studied in each trial. Ironically, while our research teams worked ardently to retain our participants, subtly, our participants have reciprocally retained our research teams on this broader journey in clinical research. Acting as an investigator on clinical trials also taught me that careers in clinical research are not a straight path. Each study lands us at a different vantage point from the one where we began, feeding our continual curiosity and learning. Each new therapy and trial has contributed one more piece to an ever-expanding puzzle. Each advance we have collectively been a part of brings greater hope that the best is still yet to come and is within the grasp of our lifetimes, moving us forward with a renewed sense of optimism and confidence. Having served as an investigator on over 100 trials now, I can say this: It changes you. It has changed who I am as a person and how I approach so many other people in my life, including patients, colleagues, trainees, and yes, even my own children. Whereas before I placed a greater emphasis on knowing and demonstrating knowledge of the “best” paths and the “right” answers, I find that now my approach in life is largely inspired by the shared investigator–participant journey, one recognizing the value of nonjudgmental equipoise and unconditional partnership imbued with passion and commitment. To the participant who caught me off guard that evening—thank you. This one, brief moment opened me up to the greatest sense of appreciation; regardless of the treatment arm, role, or results, it is the journey we embark on together, side by side with our participants, teams, and peers, that will contribute to our greater collective knowledge, advances in care, and a better future. And that, is pure joy. Acknowledgments: The author wishes to extend deep personal gratitude to her lifelong clinical research mentors, Robert R. Henry, MD, and Robert E. Ratner, MD; adviser, Jason G. Umans, MD, PhD; John B. Buse, MD, PhD, for encouragement in sharing this personal narrative; and to all the research participants who selflessly share of themselves to better our collective future.