Benjamin Djulbegovic, M.D., Ph.D.
The unknown can be scary. In life, and in medical care, it is natural to crave definitive answers.
“Medicine is ultimately about decision-making under uncertainty,” said Djulbegovic, an award-winning practitioner who serves as professor and director of research in the Department of Supportive Care Medicine
. He was also appointed as director of a new program on evidence-based medicine and comparative effectiveness research.
Uncertainty is really what shapes and colors our clinical encounters.”
Through his investigations, he aims to improve decisions made during those encounters. A leading investigator in the still-young field of evidence-based medicine, he wields tools more familiar to economists, mathematicians and psychologists. His widely cited results already have helped rewrite guidelines for cancer care.
He says he sees a special opportunity to improve health outcomes for patients around the world.
Djulbegovic’s specialty is research synthesis, looking at multiple studies on the same topic and using that big-picture view to draw conclusions. In most cases, he is in search of “totality evidence” — everything that currently can be known about a topic.
“You very rarely can inform your decision by one study, one piece of evidence,” he said.
Out of this approach, he and his colleagues have created new knowledge that changed the practice of stem cell transplantation. Djulbegovic was part of a collaborative research group that, in 2005, found that stem cells collected from the bloodstream produced better outcomes than those from bone marrow.
He also helped develop influential national guidelines about blood and platelet transfusions, as well as treatment of anemia.
No Perfect Decisions
One aspect of Djulbegovic’s work focuses intently on decisions — the input and influences, the deliberative process itself and the relative success of results.
There, too, is where uncertainty comes in.
“I think people should be aware that perfect decision-making is impossible,” he said. “We only know in retrospect. Instead, the question is finding the optimal, rational decision under uncertainty.”
From Patient Inquiries to Scientific Inquiry
Often, the research questions Djulbegovic addresses start in the clinic — inspired by questions that his patients ask him.
In one case, a comment from a patient inspired Djulbegovic to develop a framework for decision-making in cancer care that takes into account potential for regret. Studying the approach empirically, he found that in end-of-life scenarios, considering regret led patients to be more satisfied with their decisions.
Another time, a patient considering enrolling in a clinical trial asked how often investigational treatments were successful. It turned out that there was no answer.
A Paradox Worth Embracing
Djulbegovic and his colleagues analyzed existing evidence to see how often therapies from randomized phase 3 clinical trials outperform the standard of care.
The public needs to understand that there is no (and should not be a) way to predict the success of a particular trial or patient case, he said. But revealing their findings in the journal Nature, the scientists showed that on average new treatments are successful 50 to 60 percent of the time. Progress in therapeutics has occurred precisely because science and ethics require that the results of individual randomized clinical trials are not predictable.
“You may say, ‘That’s not a super chance. It’s like flipping a coin,’” Djulbegovic said. “But it’s still the best bet. And ultimately, a doctor has to be uncertain in order to enroll someone in a trial. If we knew the answer before undertaking a trial, scientifically nothing new would be learned, and ethically we would be exposing patients to potentially harmful treatments.”
The concept called “equipoise” is fundamental for Djulbegovic.
“It’s a paradox,” he explained. “You put all your effort into being certain to succeed. But being certain that something works can, in fact, preclude testing in humans. In working with human beings, paradoxically, there are ethical considerations that require you to be uncertain of the results up front.”
Across the Lines
In part, Djulbegovic credits his background in basic science for helping him articulate and pursue complicated research questions. As a doctoral student in biomedical sciences he developed a mathematical model for the proliferation of leukemia cells.
His own decision to join City of Hope was motivated largely by the ease of collaborating across the lines between departments and disciplines. He also welcomes the chance to be part of the biomedical center’s critical mass of expertise and to serve and study its large population of patients.
“Here, you can really make a mark on patient care,” he said.
Djulbegovic cultivates key allies and a robust arsenal of research tools because his goal is ambitious. He wants to contribute to improving the entire medical system.
One fundamental issue: In medical care, there is so much overuse (unnecessary tests and therapies) and underuse (tests and treatments that should have occurred but didn’t).
Djulbegovic finds this challenge engaging.
“It stands to reason that if you improve evidence and improve decision-making, that will lead to improved health outcomes,” he said. “You have to be interested in that. For me, it’s a no-brainer.”
In one project under development, he aims to take a new framework for medical decision-making and put it to the test in the clinic.
“At the end of the day, it’s about translating the science into advances that make people’s lives better,” he said.
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