There is growing awareness that patient engagement in health research is not only ethically important, but leads to evidence for developing the most effective interventions, policy and practice recommendations, and planning for ongoing research .
4.1 Example: United States
4.1.1 Mothers Donating Data: Going from Research to Policy to Practice
My son, Cal, and other newborn babies suffered from preventable brain damage in the United States as a result of the failure to test and treat neonatal jaundice (hyperbilirubinemia), known as kernicterus. After determining that a pre-discharge bilirubin test would have helped prevent our newborns from suffering, mothers of children with kernicterus formed a nonprofit organization (civil society), Parents of Infants and Children with Kernicterus (PICK) . The PICK Board of Directors, comprised of the mothers, had two specific goals: (1) co-design a safer healthcare system for newborns to include a universal, pre-discharge bilirubin test; and (2) co-design materials to empower parents with information. While the healthcare providers were sympathetic to these mothers, healthcare leaders stated clearly that changes to care or educational materials could not be made based solely on anecdotes; evidence-based research was necessary. PICK partnered with leading published researchers on neonatal jaundice, treating clinicians and patient safety experts to engage in developing the evidence necessary to revise clinical guidelines to include a universal newborn bilirubin test and revised parent education materials to empower parents to help prevent future harm to newborns from elevated bilirubin levels. Through the collaboration with the researchers, the PICK mothers helped to collect and donate clinical data of 125 newborns who were discharged as healthy from the place of birth but subsequently sustained kernicterus. The collection of data became known as the Pilot USA Registry of Kernicterus . PICK formed the Kernicterus Prevention Partnership Coalition that included various governmental agencies, academic institutions, and other stakeholders. These organizations were unified by a nonbinding memorandum of understanding. PICK and the researchers partnered with a leading public health agency to fund and analyze the data, the results of which indicated that kernicterus was an emerging public health issue and that implementation of a universal bilirubin (jaundice) test would help identify newborns at risk of hyperbilirubinemia and reduce the number of cases of kernicterus. PICK also partnered with a leading healthcare system with a large data set of clinical information on newborns. Analysis of their data also supported the implementation of a universal bilirubin test. A separate governmental health agency also partnered with PICK to fund the co-production, testing and the usability of parent education materials in different populations about the risks of newborn jaundice and included proactive steps they could take to identify and prevent harm to their newborns .
The outcome of PICK’s co-production in research contributed to the revision of clinical practice guidelines to include a universal pre-discharge bilirubin test  and the dissemination of a “Sentinel Event Alert” by a national hospital accreditor with recommendations on newborn jaundice management , established kernicterus as a “Never Event” per a national quality measures organization, developed a national parent education campaign  and materials and co-developed and co-delivered curricula for continuing medical education courses.
There were many factors that contributed to PICK’s achievements. One of the key factors was that a major national government agency invited stakeholders, including patients, to publicly comment at a National Summit on Medical Errors and Patient Safety. The organizers of this summit offered guidance to those unfamiliar with public comment how to best craft their testimony. At that summit, I, testified about the preventable harm that my son had suffered from undiagnosed and untreated neonatal jaundice and advocated for collaboration amongst all stakeholders to prevent future cases. Another success factor was the determination, persistence, and relentless call for action from the community of mothers with children with kernicterus who formed a respected, independent, nonprofit organization with by-laws and objectives to prevent harm to future newborns through a model of partnership. Further these mothers served as a “living repository” of clinical data for research regarding kernicterus unavailable through traditional data collection methods and were the conduit to collecting additional data from mothers across the world with children with kernicterus. This enabled the researchers to actively collaborate with the mothers as subject matter experts. As a result of these factors, deep, trusting, mutually beneficial relationships formed with patient safety experts, clinicians, and leadership in government agencies, research institutions, medical education, and healthcare systems who willingly partnered with the mothers, despite criticism from peers. The healthcare leaders voluntarily helped the mothers gain capacity to be effective advocates for changes in jaundice management protocols. They helped educate the mothers about the structure of the healthcare system, the responsibilities of the various decision-making bodies, the current science and evidence base for management of newborn jaundice and gaps in the literature. They provided guidance and tips on successful storytelling and public speaking skills, partnered as presenters at national conferences and in interviews with media and provided resources, infrastructure and credibility that facilitated the development of the necessary evidence for successful implementation of a systems-based approach to the prevention of kernicterus.
4.1.2 Civil Society: Driving Patient-Centered Research to Prevent Diagnostic Errors
Researchers estimate that up to 80,000 deaths per year in US hospitals can be attributed to some form of diagnostic error. Misdiagnosis affects 12 million Americans in ambulatory care settings annually. The National Academy of Medicine’s 2015 report, Improving Diagnosis in Health Care, highlights the urgent need for a research agenda on the diagnostic process and diagnostic errors and states that “patients are central to the solution” and there is a need to “establish partnerships with patients and families to improve diagnosis . The Society to Improve Diagnosis in Medicine (SIDM), where I serve as the Director of Patient Engagement, is a US-based nonprofit organization (civil society) dedicated to reducing diagnostic errors. We believed that if researchers joined forces with trained patients and family members with lived experience in diagnostic error to co-produce diagnostic safety research projects, the research questions and outcomes would be more relevant, effective, and patient centered. SIDM pursued funding from the Patient-Centered Outcomes Research Institute (PCORI) to (1) recruit patients and family members who had experienced diagnostic error and diagnostic safety researchers to co-develop a curriculum that provides patients and family members with the knowledge, skills, and tools to effectively partner in the design, execution, and dissemination of diagnostic research; and (2) collectively co-produce patient-centered research topics and questions to pursue to improve diagnosis .
SIDM collaborated with Project Patient Care, an independent nonprofit organization of patients, family members, and patient advocates and the Medstar Institute for Quality and Safety to help recruit the patient and family participants and to develop the curriculum. I led the project that included patients and family members from key disease-related organizations and representatives from Patient and Family Advisory Councils (PFACs) at major healthcare institutions—all who had experienced diagnostic error. Prominent diagnostic researchers from academic medical centers also participated in the project. Together with the project team, the patients and researchers co-produced an innovative, patient-centered curriculum. This curriculum was continuously evaluated and refined to ensure patient engagement in diagnostic research. Applying the knowledge and methods developed in the curriculum, patients and researchers co-produced a list of patient-centered diagnostic research topics and questions for future research. One of these resultant research questions focused on disparities in diagnosis due to visible factors of age, sex, and race/ethnicity. This project was awarded funding for a 2-year research project to be led by SIDM and a major academic institute.
The promising results of SIDM’s project are due to several factors. SIDM is an established nonprofit organization (i.e., a civil society organization) that has embedded patient and family engagement as a strategic priority in its mission and dedicated resources to employ a PFPS Champion as a full-time Director of Patient Engagement. Having SIDM develop and lead this project provided the credibility to secure funding from a large national research institute to support staff, the patients, family members, researchers, leadership, as well as an infrastructure designed to support sustainability. Patients and family members from national disease groups who have firsthand experiences with diagnostic error were invaluable in identifying research questions and topic suggestions that often went unrecognized or unconsidered. The project developed and delivered an innovative, patient-centered training curriculum that enabled patients and family members to effectively distill their personal stories of diagnostic error and participate as true partners in the development of research questions. Because of its success, the curriculum has been replicated in other training efforts in acute care settings and methods and tools from the curriculum have been shared nationally and internationally as an approach to engage patients, family members, and other stakeholders in diagnostic improvement efforts .