The Patient-Centered Outcomes Research Institute funding will compare costs, and benefits and harms
By Sue Rochman
A research team led by Romain S. Neugebauer, PhD, an investigator at the Kaiser Permanente Northern California Division of Research, and Patrick O’Connor, MD, an investigator at HealthPartners Institute, received a $5.6 million funding award from the Patient-Centered Outcomes Research Institute (PCORI) to study the benefits — and potential harms — on heart health of the second-line medications used to treat type 2 diabetes.
The multi-center study will compare newer classes of drugs with older, lower-cost medications, and take a close look at whether the benefits and harms differ between patients who are at low risk or high risk of developing heart problems. The study will also look at whether benefits and harms differ based on the patient’s age, sex, race or ethnicity, heart and kidney function, or other health problems. The findings will help patients with type 2 diabetes and their doctors make more personalized medication treatment decisions.
“For more than 10 years, our team has developed and applied a combination of causal, statistical, and machine learning methods for informing critical treatment decisions faced by type 2 diabetes patients and their providers,” said Neugebauer. “We are very excited to continue this research and leverage our experience analyzing large electronic health record data to generate solid evidence that patients and providers can use to help reduce their risks of cardiovascular and other diabetes-associated complications.”
Neugebauer’s research focuses on the development, application, and dissemination of statistical methodologies to evaluate treatment effects in randomized experiments and observational studies. His particular interest in causal methods to evaluate the effects of treatment regimens with complex longitudinal data contributed to his team’s selection for this competitive PCORI award.
The study will involve a total of 6 large care delivery systems with 12 million patients ages 18 to 85, including more than 600,000 patients with type 2 diabetes. In addition to Kaiser Permanente Northern California and Health Partners Institute, the 6 study sites include Kaiser Permanente Southern California, Kaiser Permanente Hawaii, Geisinger, and Henry Ford Health System. The researchers will analyze patient data from 2014 to 2023 to compare the effects of 4 classes of diabetes medications on patients’ risk of diabetes-associated health complications, such as stroke, heart attack, heart surgery, heart or kidney failure, amputation, and weight gain.
“Kaiser Permanente and PCORI share the same goals,” said Yi-Fen “Irene” Chen, MD, associate executive director of The Permanente Medical Group. “We both focus on expanding the evidence base needed for patients and providers to make personalized, well-informed decisions about their health care. It’s exciting that we have the opportunity to partner on this important study to improve the medical care of people with type 2 diabetes.”
Julie Schmittdiel, PhD, a research scientist at the Division of Research will lead the study’s stakeholder engagement activities to ensure the research effort is patient centered, relevant, and useful. Evidence from this study will be based on the analysis of large electronic health record data using state-of-the-art statistical methodologies pioneered by co-investigator Mark van der Laan, PhD, a professor at the University of California, Berkeley.
The research team will be made up of 12 to 18 patients, caregivers, clinicians, and health plan stakeholders. The 4 classes of diabetes drugs being studied are sulfonylureas, DPP4 inhibitors, SGLT2 inhibitors, and GLP-1 receptor agonists.
The study, slated to begin on July 1, 2021, and end on May 30, 2024, is one of 4 complementary studies PCORI will fund under an initiative to help clarify the second-line treatment options for people with type 2 diabetes. PCORI announced the award on March 16.
“This study was selected for PCORI funding not only for its scientific merit and commitment to engaging patients and other stakeholders, but also for its potential to fill an important gap in our health knowledge,” said PCORI Executive Director Nakela L. Cook, MD, MPH. “These results aim to help clinicians and people with diabetes, particularly those at lower risk of cardiovascular disease, navigate the perplexing choice among many second-line medications. We look forward to following the study’s progress and working with the Kaiser Permanente Northern California Division of Research to share the results.”
This funding award has been approved pending completion of a business and programmatic review by PCORI staff and issuance of a formal award contract.
About the Kaiser Permanente Division of Research
The Kaiser Permanente Division of Research conducts, publishes and disseminates epidemiologic and health services research to improve the health and medical care of Kaiser Permanente members and society at large. It seeks to understand the determinants of illness and well-being, and to improve the quality and cost-effectiveness of health care. Currently, DOR’s 600-plus staff is working on more than 450 epidemiological and health services research projects. For more information, visit divisionofresearch.kaiserpermanente.org or follow us @KPDOR.
About the Patient-Centered Outcomes Research Institute
PCORI is an independent, nonprofit organization initially authorized by Congress in 2010. Its mission is to fund research that will provide patients, their caregivers and clinicians, and other healthcare decision makers with the evidence-based information needed to make better-informed healthcare choices. For more information about PCORI’s funding, visit pcori.org.