PCORI awards $208 million to fund 17 new comparative clinical effectiveness research studies

The Patient-Centered Outcomes Research Institute (PCORI) today announced funding awards totaling $208 million to support 17 new comparative clinical effectiveness research (CER) studies, including four trials of particularly large scale and scope tackling complex questions. All the approved studies aim to fill evidence gaps and enhance health care decision making with a focus on a variety of high-burden concerns.

In addition, PCORI awarded $2 million for a project intended to accelerate the adoption of strategies shown in a PCORI-funded study to improve mental health care for people who are receiving dialysis.

These awards present significant opportunities to address urgent health challenges and empower patients and their families with actionable information about their health care choices. Facing a complex health care system and many care options, patients, caregivers, clinicians and other health decision makers need reliable information to help them understand which care options will best meet individual patient needs and circumstances. PCORI-supported evidence will improve health care and outcomes for people across the nation."

Nakela L. Cook, M.D., MPH, PCORI Executive Director

Ambitious trials take on questions about heart disease, palliative care, older adults and rare disease

Four large studies were awarded through a PCORI funding opportunity that supports ambitious CER trials taking on particularly complex or challenging health care dilemmas. Using a two-phase approach with an initial pilot stage, these large randomized controlled trials aim to maximize study feasibility. The studies will compare:

• Treatment of coronary artery disease with either open-heart surgery or less invasive stent placement with a focus on women and individuals from traditionally underserved groups.

• Delivery of palliative care led by specialists versus trained general care practitioners to seriously ill hospitalized patients.

• Various medications as second-line treatment for the 25 percent of children with a severe form of juvenile idiopathic arthritis who do not get better taking a first-line biologic drug.

• Impacts of annual wellness visits alone versus adding an integrated care model involving interprofessional teams and at-home visits among older adults with complex health care needs.

Additional CER focuses on telehealth, cancer care and women's health, among other topics

PCORI funds patient-centered CER on high-priority health needs identified and shaped through input the organization gathers from patients and other stakeholders as well as on research questions proposed by clinical scientists and their engaged partners. Among the other studies approved in this funding cycle are:

• Three studies comparing telehealth strategies to improve management of multiple chronic conditions in primary care settings focused respectively on concurrent chronic obstructive pulmonary disease and sleep apnea, obesity and asthma in children, and care for medically fragile children.

• A trial on breast cancer drugs called CDK4/6 inhibitors that will compare starting with a lower dose to starting with a full dose among older patients with metastatic breast cancer.

• Two studies looking at the diabetes drug metformin, one comparing it to insulin injections for treating gestational diabetes and another comparing risks of heart disease among women prescribed either metformin or contraceptive pills to treat polycystic ovary syndrome.

• Two studies comparing approaches to improving older adults' functional outcomes, one focused on individuals with co-occurring pain and early cognitive decline and the other on cancer survivors and their caregivers.

Promoting the uptake of useful evidence into practice

In addition to funding CER to generate new evidence, PCORI supports projects to facilitate the uptake of useful evidence in a variety of care settings where it can improve outcomes. A newly approved implementation project aims to make two treatments shown in a PCORI-funded study to be effective for managing depression among patients undergoing dialysis-;a medication and a cognitive behavioral therapy program delivered via telehealth-;available in dozens of dialysis units across the United States.

"PCORI's stewardship of patient-centered comparative clinical effectiveness research extends to ensuring useful findings can have a salutary impact in everyday clinical care, which is why we fund projects that encourage uptake of results," said Harv Feldman, M.D., MSCE., PCORI's deputy executive director for patient-centered research programs. "As a result of PCORI's latest implementation funding awards, clinicians and patients confronting decisions about mental health while undergoing dialysis may experience better care and outcomes."

Details of all the newly funded studies and projects are available on PCORI's website. All funding awards were approved pending a business and programmatic review by PCORI staff and the issuance of formal award contracts. With these latest awards, PCORI has invested more than $4 billion to fund patient-centered CER and other research-related projects.