WASHINGTON – Patients, clinicians and families facing uncertainty about their choices for managing Type 2 diabetes and rare diseases will gain new evidence to inform their decision making from comparative clinical effectiveness research (CER) studies that the Patient-Centered Outcomes Research Institute (PCORI) Board of Governors today approved for a total of $42.7 million in new funding.
The PCORI Board approved another $5.8 million for four projects that aim to accelerate the adoption of useful findings from completed PCORI-funded research in clinical care. The Board also approved PCORI’s CER funding announcements on three topics: maternal health, urinary incontinence and mental health among people with intellectual and developmental disabilities.
Second-line medications to treat Type 2 diabetes
The Board approved $18.4 million to fund four studies assessing which second-line medications would be the most beneficial next step for people with Type 2 diabetes who are no longer able to control their blood sugar using the standard first-line drug alone. These studies aim to generate evidence to help patients and their clinicians navigate what can be a perplexing choice by comparing the newer second-line therapies—SGLT2 inhibitors and GLP-1 receptor agonists—and older, lower-cost options such as sulfonylureas and DPP-4 inhibitors.
The four studies will assess the second-line drugs with a particular focus on cardiovascular outcomes among individuals who neither have cardiovascular disease nor risk factors for heart problems. Previous research has shown that the newer medications can provide protective effects for patients with or at high risk for heart disease, but it is not clear if those benefits would extend to the many patients at lower risk. The observational studies will draw upon health data resources, including electronic health records, insurance claims data and large research networks, to compare outcomes experienced by tens of thousands of patients taking these medications.
Research on rare diseases using PCORnet®
The PCORI Board approved $19 million to fund four CER studies that will use PCORnet®, the National Patient-Centered Clinical Research Network, to provide new evidence about care of four rare diseases. With health records for 66 million patients available for observational studies, the PCORI-funded PCORnet provides vast scale to power research on conditions affecting even small numbers of people.
One study aims to produce information to help families of children with a rare form of epilepsy decide between additional anti-seizure medication or surgery based on how often patients using each option seek emergency room care and how the options affect the children’s developmental and functional difficulties. Another study, PRESERVE, will assess how effective different strategies to monitor and treat high blood pressure are at preserving kidney function in children who have chronic kidney disease.
The CER-NET study aims to determine the optimal sequencing of treatments for people with neuroendocrine tumors, a rare form of cancer. A fourth study seeks to understand whether people with complex congenital heart disease developed in childhood have better outcomes if they continue to receive recommended care from a heart disease specialist when they transition to adulthood rather than primary care alone.
Separately, the Board approved $1 million to fund the development of automated ways to more efficiently and accurately identify people with rare diseases or complex health problems via electronic health records, enabling treatments to start sooner and helping researchers offer individuals opportunities to participate in studies.
Facilitating results’ uptake in care delivery
Cutting the lag time and smoothing the way for the adoption of impactful findings in clinical care is the focus of PCORI’s dissemination and implementation efforts. The Board approved $5.8 million to support four new projects to promote the adoption of interventions shown through PCORI-funded research to improve patient outcomes.
A project aimed at reducing rates of unplanned pregnancy among adolescents will integrate Health-E You/Salud iTu, a mobile app found to improve the uptake of effective contraceptive methods, into school-based health centers in 10 states and Washington, D.C. Another project will support a self-care coaching program called GUIDED-HF becoming part of the standard procedures for discharging patients with advanced heart failure from emergency departments.
A third project will promote in 50 to 60 clinics across the nation the use of a program shown to help people with advanced kidney disease requiring dialysis to engage in discussions about advanced care planning. Another will promote the adoption of decision aids that help women with early-stage breast cancer decide between mastectomy or breast-conserving surgery at more than 30 health care sites.
New research funding opportunities
Following Board approval, PCORI will issue new funding announcements this year seeking proposals for CER on three critical topics:
- Figuring out which strategies most effectively increase early detection of and timely care for postpartum complications among new mothers, particularly Black, Hispanic/Latina and Native American women and those with low incomes or who live in rural areas. PCORI seeks to fund four to six studies, offering $5 million to $15 million in total costs per study.
- Understanding the comparative effectiveness of nonsurgical treatments for women experiencing urinary incontinence, a condition affecting 30 percent of older women. PCORI aims to fund five to eight studies, offering $3 million to $5 million in total costs per study.
- Assessing the comparative effectiveness of interventions that address mental health conditions in individuals with intellectual and developmental disabilities. PCORI proposes to fund as many as a dozen studies, offering $2 million to $3 million in total costs per study.
Details of all studies and projects approved for funding by the Board are on PCORI’s website. All funding awards were approved pending a business and programmatic review by PCORI staff and issuance of formal award contracts. With these latest awards, PCORI has invested $2.9 billion to fund patient-centered CER and to support other projects designed to enhance CER methods and the infrastructure necessary to conduct CER rigorously and efficiently.
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 with the evidence-based information needed to make better-informed health care decisions. PCORI is committed to continually seeking input from a broad range of stakeholders to guide its work.
Christine Stencel, (202) 570-9275, [email protected]