On September 30th, congressional authorization for the Patient–Centered Outcomes Research Institute (PCORI) expires. PCORI is a nonprofit research organization devoted to helping patients and doctors make informed decisions based on the evidence. Whereas other research institutes like the National Institutes of Health (NIH) help uncover cures, PCORI uses evidence that’s already out there to help doctors and patients make the decisions that are right for them. And it does so with unprecedented levels of patient involvement. PCORI-supported research is important to patients of all ages, including kids.
I am a pediatric rheumatologist and serve as Chair of Pediatrics and Chief Scientific Officer at Nemours Children’s Hospital. I am also Nemours executive lead for a PCORI-supported research network called PEDSnet. Nemours is one of the 8 founding members of PEDSnet — a community of patients, families, clinicians, scientists, and healthcare system leaders who work together to identify and implement new ways of providing the best care and ensuring the best outcomes for kids.
The power of PCORI-supported networks like PEDSnet is enabling researchers to share data and ask questions about chronic disease that can be answered only by studying millions of people. Imagine finding a way to prevent a million cases a year of new-onset asthma; in fact, earlier this year, Nemours and our PEDSnet partners published a study leveraging information from close to 20 million medical encounters with over half a million children to demonstrate that obesity is the only preventable risk factor for asthma. In addition, in children, many health conditions – muscular dystrophy, for example – are uncommon or rare, and “Big Data” are needed to answer important questions about care and cure of a rare disease. A shared data resource like the 6.2 million patient records available through PEDSnet enables us to conduct studies that would not be feasible for any one institution alone.
For rare and common diseases alike, knowing the risks and benefits of different treatments for a disease is of critical and sometimes life-saving importance. Did you know that even with all our technological advances in medical care, we don’t know if surgery is always needed if your child presents to an emergency room with appendicitis? Or if post-surgical antibiotics will improve the outcome? Or which pain medications may be given without risk of overdose or addiction?
While clinical trials are considered the gold standard for research, they are neither cost effective nor necessary in all cases. In fact, the rigor of this gold standard may result in more than 20 years from the discovery of a new life-saving medicine and the use of that medicine to save the life of a child. PEDSnet enables us to generate evidence based on existing data in an effective and timely fashion. Furthermore, since patients and families help to select and inform the studies funded by PCORI and PEDSnet, researchers are able to harness their voices to address questions about treatments, side effects and outcomes of disease that matter most to them. As a practicing physician, I know how much this matters.
I also know our legislators are paying attention – when I was recently in Washington, D.C., in meetings with key congressional advisors on both sides of the aisle, I saw enthusiastic interest in PEDSnet and PCORI, and recognition of how their funded research projects empower patients and families to impact their healthcare decisions.
Unless Congress acts, on September 30th, PCORI will no longer have authority to commit to new research grants. The work that PCORI and its funded networks like PEDSnet do is impactful and should be continued.
The opinions expressed in this blog post are the author's own and do not necessarily reflect the view of AcademyHealth.
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