PCORI’s promise to patients

When patients and their doctors have multiple care options for a health problem, solid evidence is the compass that guides them toward the best decision. Yet despite amazing advances in medical research, patients and their doctors far too often must make critical choices without good evidence, basing their decisions on a combination of anecdotes and educated guesswork.

This is why the Patient-Centered Outcomes Research Institute (PCORI) was created: to fund more studies that help patients, families, and clinicians make better-informed health decisions by comparing which clinical options will be most effective given their individual needs and preferences.

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We’ve been clear in articulating PCORI’s purpose, but there seems to be some lingering confusion. These cases of mistaken identity offer an opportunity to remind everyone who we are and what we do.

PCORI is an independent, non-profit research institute authorized by Congress. It’s governed by a 21-member board representing the entire healthcare community, including patients, caregivers, clinicians, researchers, employers, insurers and industry. Our approach to research is unusual -- we believe the best way to provide patients and clinicians with the most useful information is to ask them, and other stakeholders, what kinds of evidence they need, then engage them as partners throughout the research process.

This helps ensure we address meaningful, real-world questions and focus on outcomes that matter to patients. It’s an approach I welcome as a practicing psychiatrist who daily faces the challenge of determining which treatments will result in the outcomes my patients seek.  For example, I frequently need more specific information about treatments most likely to relieve a patient’s depression symptoms without causing over-sedation and other side effects he or she might not be willing to tolerate.

PCORI’s approach differs from traditional research because it does not stop at what works best for the “average” patient. We ask whether treatment effectiveness varies depending on patient characteristics and preferences.  It’s a model that can advance personalized medicine by giving patients and those who care for them more personalized information about their options.

PCORI was established barely four years ago -- an incredibly short time for a major research institute. Yet we have begun to see our work yield results.

With PCORI funding, researchers at the prestigious Children’s Hospital of Philadelphia found that when treating a type of serious bone infection in otherwise healthy children, physicians should reconsider using intravenous antibiotics after hospital discharge if an equally effective oral alternative exists. As prolonged IV use is associated with more side effects, this is very useful information -- published Dec. 15 in JAMA Pediatrics -- for pediatricians and parents to use at their discretion.

And that’s an important aspect of PCORI’s work -- the studies we fund are not mandates for practice. We don’t dictate which care options are available for doctors to use. PCORI was created to provide patients and their doctors with information to better inform, not restrict, their choices.

We look forward to releasing the results of many more funded studies in coming years. We’re also working to support a large national health data network, called PCORnet, which can allow our country to produce greater volumes of patient-centered research, faster and at lower cost than ever before.

At the same time, we’re sharing with the entire healthcare community our growing experience with engaging patients and other stakeholders in guiding the research we fund. As we show impact, and develop methods and best practices to make this approach easier and more effective, we anticipate more researchers will follow our lead.

The astonishing advances of modern medicine have been built on the gradual emergence of more sophisticated research that helps us better understand and deliver quality patient care. PCORI’s ambitious goal, and our promise to patients, is to push us further down that path and contribute to a new era of personalized medicine and better-informed patients and clinicians.

Norquist is a professor in Emory University Department of Psychiatry and Human Behavior; chief of Psychiatry Service, Grady Health System; and chair of PCORI Board of Governors.