The role of patients in health research is changing dramatically. Once considered simply study “subjects,” today patients play a meaningful and critical role in research. Such involvement ensures study results are more useful to them as well as others involved in making important health care decisions.
Patients bring issues that matter to them—such as quality of life and ability to function—to the forefront of conversations about how to evaluate the effectiveness of health care options. They are working alongside scientists as partners in designing and conducting research and are key to the successful dissemination and implementation of the results.
Considering patients’ perspectives makes it more likely that researchers will ask the right questions, conduct the studies best designed to answer them, and produce results more relevant to everyday care and more likely to be adopted in practice. That’s critical, because health care is increasingly judged on the value it delivers to patients, as well as clinicians and the nation as a whole.
Changing The Culture
This approach makes a lot of sense now. But it wasn’t always an accepted model.
Prior to the last few decades, clinical research did not routinely address some of the most relevant outcomes for patients. For example, a review of early clinical trials of epidural analgesia during labor found most did not determine whether it provided effective pain relief for women during childbirth but instead focused on changes in hormone levels or effects on the newborn. Similarly, research on psychiatric medications often did not concentrate on side effects, which were of most importance to people with psychiatric disorders.
But while putting patients’ concerns front and center in research is fairly new, the work of the Patient-Centered Outcomes Research Institute (PCORI) ensures this focus is increasing and will continue to be a critical component of health care research in the future.
PCORI was established by Congress in 2010 as a private, independent group to fund research on what works, for whom, under which circumstances, focusing on questions most relevant to patients and others across the health care community. We do that by involving patients, caregivers, clinicians, payers, industry, and other health care stakeholders throughout the research process.
In five years, we’ve seen great progress. We’re helping to create a research culture that prioritizes patients’ input by funding studies that incorporate their perspectives in a meaningful way. When engaged, patients, especially those with chronic or life-threatening conditions, have become enthusiastic research partners.
We’re also building the capacity for research by supporting the development of novel patient-led data networks and connecting them with health systems-based researchers. These networks are part of the National Patient-Centered Clinical Research Network, or PCORnet, a growing national resource harnessing data representing more than 70 million patients to date and already conducting several important demonstration studies.
A Growing Trend
PCORI is not alone in changing the conversation about health research. Congress, federal health agencies, health systems, and industry are taking similar approaches.
For example, the Food and Drug Administration (FDA) has announced its first patient engagement advisory committee, which will help define the benefits and risks of certain new devices and use patient perspectives in the regulatory process. The FDA’s new commissioner, Robert Califf, MD, who helped get PCORnet off the ground, has emphasized patient engagement as key to the FDA’s mission going forward.
The National Institutes of Health’s (NIH) Precision Medicine Initiative is building a cohort of 1 million Americans to explore the relationships among our genes, the environment, health, and disease. Patients are central to this effort; a number of those in leadership positions are bringing to the initiative insights from their work with PCORnet.
In Congress, the 21st Century Cures Act aims to put patients at the center of efforts to rapidly develop critical new therapies. Similar movements are gaining momentum in the pharmaceutical industry.
So although we’re not alone in this effort, we believe PCORI’s leadership is one of the key reasons this trend is accelerating. And we’re confident that as the results of this approach become clear, its value as a path to making health research more relevant, useful, and inclusive will be recognized more broadly.
That’s why we at PCORI are pleased to have supported the development of the April theme issue of Health Affairs featuring new research and provocative commentary on how patients, their families, and others use evidence to make better-informed health care decisions. Each article is compelling, but a few, including several by PCORI awardees, are particularly worth noting.
Steven Woolf and colleagues, for example, talk about the potential for full engagement of patients and the public to transform practice and policy. Danielle C. Lavallee and her colleagues examine opportunities for patient-reported outcomes to enhance health care delivery and outcomes. Ming Tai-Seale and her colleagues introduce a novel approach that improves communication between patients and clinicians and improves engagement in shared decision making. John M. Westfall and his colleagues share a process by which community members have translated more than 25 evidence-based medical guidelines into locally relevant messages. Mildred A. Solomon and her colleagues, meanwhile, identify challenges that will arise as patients and the public engage with evidence.
In addition to the articles, several of the April issue authors have provided short video interviews about the significance of their work. These include: Daniel Dohan; Ming Tai-Seale; Steven Woolf; and two of his patient partners, William Jacques and Chanel Bea.
Patients are writing the next chapter in health research. We look forward to helping them obtain the tools they need to make health care decisions that yield the outcomes they want and need. This is a critical move forward and one that is long overdue.