Smaller and faster patient-centered outcomes research: More than 12 years ago

     I had the pleasure of working with Sue Sheridan at the October PCORI (Patient Centered Outcomes Research Institute) workshop , which brought together 150 members of the public to advise on a new process for funding research into patient-centered outcomes.  Sue was pulled into this work more than 12 years ago by the severe and permanent injury of her son at childbirth.  In hindsight, the reason is clear:  Hospital staff had not performed a simple bilirubin blood test that would have identified the risk he'd develop a rare, devastating condition related to jaundice ["kernicterus"] and cerebral palsy.

Sue and other mothers with similarly heartbreaking stories formed an advocacy organization, Parents of Infants and Children with Kernicterus [PICK].  PICK forcefully and successfully pushed for the American Academy of Pediatrics to issue a new standard of care, requiring all newborns to have an inexpensive blood test that can warn of incipient jaundice.  When hospital doctors and nurses prevent jaundice, which can be done inexpensively by using a special light (phototherapy), they forestall the rare but devastating related complications of kernicterus and cerebral palsy.   

Yet now, more than 12 years later, it's likely that Labor and Delivery doctors and nurses perform this blood test on only a fraction of newborn babies.  Hospital staff adopt best practices like these slowly; we often decry the 17-year gap from the identification of an improvement in medical practice until its widespread use.  And it's hard to believe, but we don't know how many babies go without this inexpensive, life-saving treatment.  

PCORI should immediately fund a study of hospital claims  to ascertain how many newborns lacked this formally approved life-saving inexpensive blood test. 

Even more importantly, this lag between knowledge and practice shows that our process for updating clinical practices is fundamentally broken.  This should have been an easy one:  heart-breaking permanent harm to babies in a relatively small number of treatment sites; inexpensive diagnosis; inexpensive treatment; enraged and organized mothers.

PCORI should fund a small project, for the analysis is very easy.  Indeed, it is particularly important that the study be small, for that will limit the tendency of academic researchers to inflate its scope to consider complexities that attract researchers.  Small is beautiful.  The projects will be finished soon, allowing their more rapid use. Funding small projects will allow innovation in multiple areas, planting many seeds in the knowledge that a fraction will sprout.  It hedges the risk that a few large studies may not yield persuasive and actionable recommendations.  Funding small studies will have political benefits as well, in empowering small organizations of activists that will support PCORI's mission.  

My son was born prematurely, and an alert nurse in the neonatal intensive care unit (NICU) noticed the yellowing jaundice of his skin just as it started.  They put my tiny boy under bright lights, which prevented any build-up of bilirubin, and prevented kernicterus.  There, but for the grace of God, went I.  Our luck held.  I hope PCORI's funding decisions will give outcomes like my son's to many more parents.