e once thought of disease and its treatments with a one-size-fits-all mindset. That’s changing, and not just because “personalized medicine” has become the buzz phrase of modern health care. A new era of research delivering truly patient-centered results is also providing individuals with the information they need to select the treatment that most closely meets their specific needs.
How we experience disease and how we choose to be treated for it differ from person to person. For a young mom with cancer, longevity may be the desired outcome, while being able to text with friends may be what a teenager with a degenerative neurological disease most wants. The ability to support a family or care for a child or frail parent during treatment may factor heavily on the options an individual might consider.
To wisely make such choices, we need useful, evidence-based information that lets us evaluate and select the high-value health care options that meet our needs. To use a car buying analogy, when choosing treatment for a condition, you don’t want to walk out with a sports car when what you need is an SUV.
One organization uniquely positioned to develop and disseminate such evidence is the Patient-Centered Outcomes Research Institute (PCORI). This independent, nonprofit, nongovernmental organization develops useful and actionable information that helps people answer the question, “What are the best options for people like me?”
Established in 2010 by Congress, PCORI helps people make informed health care decisions while also improving health care delivery and outcomes. It does this by producing and promoting high-quality, evidence-based information that emerges from research guided by patients, caregivers, and the broader health care community. Instead of focusing solely on investigator-initiated research ideas, PCORI focuses on research that patients, family caregivers, and other stakeholders have identified as meaningful, relevant, and useful to them.
The end result, patient-centered outcomes research, can help inform the practice of medicine, the delivery of care, the design of health insurance, and how we develop new treatments to truly meet the needs of patients and their families. PCORI’s emphasis on patient engagement has created a sea change that is rippling through the entire health care continuum.
As a member of PCORI’s inaugural Advisory Panel on Patient Engagement, I watched as the organization pioneered a new way of doing research, involving patients throughout the entire arc of research, from designing clinical trials to spreading the word about their results. Patient contributions prompt researchers and others to ask the right questions at the right time to identify and prioritize the outcomes that matter most to patients.
Here’s one example: The standard of care for children with a serious infection, such as a ruptured appendix, was intravenous antibiotics delivered at home. Imagine turning a child’s bedroom or your dining room into a hospital room so you could stick a needle in your child’s arm and slowly drip antibiotics into a vein while trying to keeping him or her still for hours. A recent PCORI study, which is already affecting the practice of medicine, demonstrated that an oral antibiotic is just as effective, if not more so. That’s useful, patient-centered information that any parent might want.
Science and technology are continually expanding treatment horizons. Every year brings new treatment options for many diseases. The accessibility of medical information, the shifting of health care costs to patients, and the desire for a personalized approach to medicine are rapidly driving consumerism in health care, much as we have seen happen in other areas.
Say you need a new refrigerator. Online retailers address your needs in the most efficient, user-friendly way possible. Information about options and features are at your fingertips. I may want a compact one for my tiny condo, while you need a family-sized fridge with water and ice dispenser. We can easily find comparative information online to make the choice that suits our needs.
Now say you need something more important than a refrigerator, like treatment for diabetes or multiple sclerosis or lupus. For these and many other chronic diseases, there are multiple treatment options that have different outcomes, side effects, quality-of-life issues, and convenience. Gathering information about all of these can be difficult. Finding cost information is even harder. The bottom line is that understandable or actionable health care information that would help get you the right treatment is often limited to nonexistent.
More than ever we need the kind of useful information being created by PCORI so people can make informed health care decisions for themselves and their families. Patients should not have to close their eyes, reach in to a hat, and blindly pick out a treatment.
Marc Boutin, J.D., is the chief executive officer of the National Health Council, an organization that provides a united voice for people with chronic diseases and disabilities and their family caregivers. Its membership includes patient advocacy and other health-focused nonprofit organizations as well as insurance, pharmaceutical, biotechnology, and medical device companies. The National Health Council has received the Eugene Washington Grant from PCORI for $246,300.