Leadership Perspective

Advancing Health Care, Enhancing Lives

Published: Feb. 18, 2025

A photo of PCORI Executive Director Nakela L. Cook, MD, MPH, for the Leadership Perspective

As the new year gathers steam, we celebrate both our achievements and the momentum we continue to build toward future opportunities. Reflecting on PCORI’s journey, I see more than a decade of collective progress — one where the successes continue to guide our path to impact in health care’s ever-evolving landscape. We are doing this by generating and disseminating relevant, high-quality evidence to help patients and other stakeholders make better-informed decisions about their health, while also building a more efficient, effective, patient-centered healthcare system.

With your support, PCORI reached some remarkable milestones in 2024. We awarded nearly $650 million to fund over 160 patient-centered comparative clinical effectiveness research (CER) studies and related projects, bringing the total awarded funding to more than $5 billion. We published the 500th final research report and exceeded 600 peer-reviewed publications of CER results. Most importantly, implementation projects have brought evidence from PCORI-funded research to hundreds of thousands of patients.

These are more than numbers — they represent real improvements for people's lives. Thanks to PCORI-funded CER, patients and those who care for them are better equipped to make informed decisions about managing conditions like asthma, preventing blood clots after surgery and choosing between antibiotics and surgery for appendicitis.

Looking Forward: Navigating Health Care's Future

The future of health care holds both challenges and opportunities. While artificial intelligence (AI) promises to transform both research and care, it also raises ethical dilemmas about privacy, transparency and patient-centeredness in the application of AI in care. The prevalence of chronic diseases continues to rise, and the aging U.S. population faces growing challenges, especially with common conditions like diabetes and cardiovascular disease. Maternal health in the United States compels sustained attention, with mortality rates continuing to surpass those of other developed nations.

These issues go beyond clinical care, touching the very foundations of people’s lives, including the non-medical factors that shape health outcomes at least as much as medical factors.

Our Response

PCORI remains focused on our work as the leading funder of patient-centered CER. By collaborating with patients, organizations and a vast range of stakeholders across the country, our research funding addresses the evolving needs of patients, caregivers and the broader healthcare community. PCORI’s application guidance sets the standard for meaningful engagement in research and supports applicants in developing engagement plans for research.

Our focus on patient-centered burdens and economic outcomes captures data, as appropriate, on the full range of patient-centered and clinical outcomes relevant to patients and other stakeholders — from lost wages to childcare expenses — in the research PCORI funds. This approach offers a more comprehensive view of the challenges patients face and contributes information to support patients’, caregivers’ and others’ healthcare decisions in meaningful ways.

The Health Systems Implementation Initiative connects research to clinical practice, leveraging insights from health systems to enhance care delivery and meet community needs. Additionally, PCORI’s updated Research Project Agenda — with added Topic Themes in cancer research, metabolic and endocrine health and sensory impairment and disability — continues to evolve over time to promote PCORI funding across the range of health issues and conditions experienced by the U.S. population.  

A Vision for the Future

We envision a future where research and clinical practice are seamlessly integrated, creating a continuously learning healthcare system. In this future, patients facing complex healthcare choices not only participate in studies but contribute to an active partnership between researchers, patients, caregivers, clinicians and other partners throughout the entire research process.

Digital tools will capture and analyze patient outcomes in near real time, so that evidence remains relevant and participation in research is open to everyone. By combining clinical and economic data, we can generate information to support more effective, patient-centered decisions by patients, providers and policymakers.

With 15 years of foundation to build on, PCORI is poised to lead this transformative shift toward better health outcomes for all. This progress is fueled by the collaborative spirit of researchers, patients and the broader healthcare community across this country — working together so that no one is left behind.