Research With an Eye on Better Choices, Better Outcomes:
Chris Feudtner, MD, PhD, MPH
New treatments and therapeutics must undergo rigorous testing and evaluation before they are approved for use in patients. Historically, however, many of those clinical tests involve comparing the proposed new treatment with a placebo, a substance with no active medication. Rarely are there head-to-head studies to determine if one medication or treatment is better than a competing one.
It’s like comparing apples to oranges, rather than apples to apples. And when it comes to patient care and prescribing the most effective treatment course, how can you be sure one medication is better than another?
“Once a medication is approved and becomes available, post-marketing surveillance typically does not give enough information for hospitals, physicians, and consumers to make wise choices,” says Chris Feudtner, MD, PhD, MPH, director of the Department of Medical Ethics at Children’s Hospital and holder of the Steven D. Handler Endowed Chair in Medical Ethics. “But conducting high-quality head-to-head studies of two competing treatments that may be distinguished by a small difference between their effects it isn’t so easy to do — or even desirable in many cases.”
Furthermore, while one drug may do a little more than another, does the patient really benefit from the small difference? Is a small increased benefit worth a potentially much higher cost? And if one drug turns out to be the best, why use any of the others? It all comes down to what you really want — and need — to know.
“Scientifically, there is a deep issue here about what knowledge we really want to have to guide healthcare choices,” says Dr. Feudtner. “It’s a matter of exploring the differences in the impact of various treatments that will actually make a difference – a difference that really matters to that patient.”
Comparative effectiveness research (CER) offers a unique approach to this conundrum by allowing investigators to compare treatment strategies to one another — not just to a placebo — and to do it in a way that allows physicians to provide the best possible treatment based on a individual’s unique needs.
Through CER, investigators can conduct observational studies of similar patients who have the same underlying medical condition and are affected by the disease to the same degree yet, for no discernable reason, are given two different treatments. Sometimes this happens because doctors in one area of the country or one set of hospitals like to use treatment A, but other doctors prefer to use treatment B, even though their patients are essentially the same. With this data, Dr. Feudtner and other researchers in the field can look back several years and create more of a head-to-head comparison of medications to discern with greater accuracy the outcomes of those different treatments.
“Comparative effectiveness research provides an important, alternative way of obtaining information that is useful for decision-makers,” says Dr. Feudtner, whose primary research focuses on medical ethics and strategies to improve the quality of life for children and families dealing with complex chronic diseases.
Among his current projects, Dr. Feudtner is leading a nationwide study aimed at providing critical data that will guide future efforts to advance pharmaceutical effectiveness and safety. Using several large existing national databases, the research team is looking a multitude of variables, including clinical data, hospitalization records, and drug exposure information — all with an eye on improving patient outcomes through the more informed use of medications.
“This comprehensive project will provide information that is of relevance to consumers, clinicians, and policy makers, and can be translated into targeted programs to improve outcomes,” he says.