I’ve reported to you on occasion about results emerging from PCORI’s earliest funded studies. Most recently, in April, I called your attention to a number of articles that appeared in leading medical journals. At the time, I predicted a steady stream of additional publications reporting findings from studies we’ve funded, and that’s just what we’re seeing. So, with this blog, we’ll start regularly updating you about the latest reports in the scientific literature. These studies show, better than I ever could have predicted when PCORI began funding studies, what happens when patients’ interests come first in planning and conducting comparative clinical effectiveness research (CER).
The Benefits Accumulate
From our first solicitations for research proposals, we asked applicants to involve patients, clinicians, and other stakeholders in forming research teams and selecting the research questions and outcomes that mattered most from the patient perspective. And with patients, clinicians, and other stakeholders joining scientists on our review panels, we selected the applications that followed these instructions.
We see the benefits of this approach in many of the more than 30 studies with important CER results published to date. It’s clear that some of the study outcomes are far more relevant to patients than the ones researchers alone might have chosen, making the findings particularly useful in helping patients and their clinicians choose among different care options.
Two examples are the PCORI-funded studies I mentioned in April that compare treatment approaches for men with localized prostate cancer. You can hear from the researchers and one of their patients in a recent PCORI Blog post. Another example is a recent article in JAMA Internal Medicine concluding that the daily finger sticks many people with diabetes use to check their blood sugar levels offer little value for those who have type 2 diabetes and are not using insulin. This practice has always posed a dilemma for patients because finger sticks are uncomfortable and can cause anxiety, the self-monitoring supplies are costly, and it’s not very clear what to do with the results.
Now, in a PCORI-funded study, Katrina Donahue, MD, MPH, and colleagues at the University of North Carolina at Chapel Hill found that routine daily testing did not lead to better control of glucose or other benefits—a result not clearly established before. This means that for many diabetic patients who are not taking insulin, clinicians may be able to advise them that self-monitoring can be greatly reduced or stopped altogether.
These studies show, better than I ever could have predicted when PCORI began funding studies, what happens when patients' interests come first in planning and conducting comparative clinical effectiveness research.
We also saw important new secondary findings coming out of a study led by Erik P. Hess, MD, MS, of the Mayo Clinic. That study tested the effectiveness of a simple decision aid in safely reducing how often patients choose to be admitted for further tests after visiting an emergency department for chest pain that proves not to have been caused by a heart attack. Hess had already reported in The BMJ that patients who used the aid in conversations with clinicians were less likely to stay overnight but developed no additional heart problems in the weeks that followed, as compared with patients who received only usual care.
Shortly after that article appeared, my wife and I were having dinner with friends. When I told them about this study, one said, “That happened to me!” She had experienced chest pain and went to the hospital; tests showed no heart attack, but hospital staff urged her to stay overnight for a stress test in the morning. As a nurse, she knew she was at very low risk and refused, signing out of the hospital against medical advice. Two days later, outpatient tests showed no signs of heart disease. “I saved a lot of money and avoided a night in the hospital,” she told me. “This study makes me feel better about that decision.”
Now, in a paper published in Medical Decision Making in May, these Mayo researchers added to that finding. Their new analysis found patients in different sociodemographic groups experienced similar benefit from the decision aid, but those more skilled in using numbers tended to come away more knowledgeable about their heart risk and healthcare options than did people who were less skilled.
It’s clear that some of the study outcomes are far more relevant to patients than the ones researchers alone might have chosen, making the findings particularly useful in helping patients and their clinicians choose among different care options.
Results That Matter
Here are some other recently published articles of note on PCORI-funded studies that we think can help patients and those who care for them make better-informed decisions.
A study led by Elsie M. Taveras, MD, MPH, of Massachusetts General Hospital compared two approaches to helping overweight or obese children reduce their body mass index (BMI) scores. The two appeared to work equally well at improving BMI scores, and they also improved other key health measures and family satisfaction with children’s care, the researchers report in JAMA Pediatrics. On top of usual care for childhood obesity, both approaches linked families to community resources and sent supportive text messages, though half the families also received additional texts and individualized coaching about healthy behaviors. The results suggest that parents and children can consider the approach that best suits their needs. You can learn more about this study from the researchers and participants in a feature article on our website.
Researchers led by Adil Haider, MD, MPH, of Brigham and Women’s Hospital in Boston, studied whether patients would be uncomfortable disclosing their sexual orientation during an emergency department visit. Their work generated important articles that can be helpful to clinicians and patients, in JAMA Internal Medicine and Academic Emergency Medicine, along with a fair bit of media attention, including a feature article in the New York Times.
In the first article, the team reported that 80 percent of providers believed patients in the emergency department would refuse to answer a question about their sexual orientation, while only 10 percent of patients said they would refuse. Routinely collecting such information would boost clinicians’ ability to provide appropriate care to, and identify and address health disparities experienced by, lesbian, gay, and bisexual populations. The second paper reported on a national survey of self-identified transgender patients and found that 90 percent thought primary care providers should know about patients’ gender identity, while more than half said those clinicians should also know sexual orientation.
We’ve been pleased to see more reports coming out on the important results of projects we fund to improve methods for conducting clinical research. For instance, a team led by Kay Dickersin at Johns Hopkins University has been working to develop better methods for performing systematic reviews of research results. In May, the team published an article in the Journal of Clinical Epidemiology documenting a significant barrier: different randomized clinical trials on a single clinical approach can include hundreds of different outcomes and results. This creates a challenge for analysts doing systematic reviews—and perhaps an opportunity to pick and choose results that fit their hypotheses, the team said. PCORI is very interested in encouraging some standardization of the outcomes used in studies for treating a single condition, selecting the outcomes that patients say are most important and those that perform better in comparative tests.
Despite those difficulties, systematic reviews remain a powerful way to bring together published results from clinical research. A PCORI-funded team led by David R. Nelson, MD, of the University of Florida, published such a review in May in Annals of Internal Medicine, reporting that several oral medications all proved safe and showed high viral response for the most common type of hepatitis C infection. Nelson and one of his patient partners talk about the project in a recent PCORI Blog post.
A Growing Collection
These latest articles add to the growing collection of published evidence indicating that even in our earliest days, we were funding meaningful outcomes research designed to help patients and those who care for them make better-informed decisions. As the years have progressed—and as we’ll surely see in future publications and the study results summaries being posted on the PCORI website—we’ve been able to zero in more and more on the questions that patients and other healthcare stakeholders tell us are important to them. These include such issues as how to best manage chronic pain and opioid use, determine the most effective and patient-centered treatment options for multiple sclerosis, and support adolescents with sickle cell disease as they transition to adulthood.
I hope you’ll check back regularly as we continue to report to you on those and other studies that we believe have the potential to change clinical practice and improve patient care and outcomes.