Identification of research priorities in CHD:Empowering patients and families through participation in the development of formal research agendas

Background: Conquering CHD, formerly known as the Pediatric Congenital Heart Association (PCHA), is the leading congenital heart disease (CHD) patient advocacy organisation in the United States of America, and places high priority on patient engagement in the research process. Participatory design is an approach to problem-solving that utilises the knowledge and opinions of groups of people to generate plans and new ideas. Utilising this mode of patient engagement, patients and families engaged with Conquering CHD assisted in developing a list of research priorities which was then distributed to the larger membership with instructions to rank the priorities in order of importance. Upon completion, these items were compared to the current scientific literature to assess correlation with current publications. This cross-sectional study and literature review aimed to assess the priorities of patients and families in CHD research and to determine the reflection of these areas in the current body of scientific literature. Methods: This cross-sectional study utilised a survey asking participants to rank the importance of research items within categories including Technology Advances, Genetic and Cellular Research, Broad Understanding of CHD, and Psychosocial Outcomes which was distributed through social media and email to 43,168 accounts across all platforms. Respondents were asked to place each item in a ranked order in each category, with the value 1 representing the most preferred for each participant. Anyone engaged with Conquering CHD was eligible to complete the study, including patients and families. Subsequently, a literature review of the largest medical databases including PubMed, Scopus, and ScienceDirect was undertaken to determine the number of articles published per each topic which was then assessed to determine if there is a correlation between patient-ranked priorities and the current body of literature. Results: The study generated a total response of 527 participants. Regarding Technology Advances, valve replacement was the preferred topic (mean rank 2.07, IQR 2). Stem cell research was the favoured topic in Genetic and Cellular Research (mean rank 2.53, IQR 2). Access to care was the priority in the Broadening Understanding of CHD (mean rank 1.24, IQR 1). Pertaining to Psychosocial Outcomes, psychological/emotional effects was the highest ranked topic (mean rank 1.46, IQR 1). The literature review returned a total of 135,672 articles in the areas of interest. For Valve Replacement, 8361 articles resulted reflecting a proportion of 0.097 of total articles. For Stem Cell Research, 9921 articles resulted reflecting a proportion of 0.115 of total articles. For Access to Care, 7845 articles resulted reflecting a proportion of 0.091 of total articles. For Psychological/Emotional Effects, 6422 articles resulted reflecting a proportion of 0.074 of total articles. A Spearman's correlation demonstrated no correlation between the preferred domain of CHD research and the number of articles published for that domain (rs = 0.02, p = 0.94). Conclusions: This process demonstrates the effectiveness of participatory design, using a patient and family network to determine the research items of concern to those affected by CHD. The cross-sectional survey was effective in assessing patient and family priorities but was limited by access to reliable internet and delivery only in English. Though the study had a large response rate, it was limited to patients already engaged with Conquering CHD. For these reasons, it may not completely reflect the opinions of the total population affected by CHD. However, this offers valuable insight into patient-determined priorities and reveals that the current scientific literature does not correlate with these items. These data serve to inform individual and institutional research agendas to better reflect the needs and desires of this population.</p

Development of an international standard set of clinical and patient-reported outcomes for children and adults with congenital heart disease: A report from the international consortium for health outcomes measurement congenital heart disease working group

Aims: Congenital heart disease (CHD) is the most common congenital malformation. Despite the worldwide burden to patient wellbeing and health system resource utilization, tracking of long-term outcomes is lacking, limiting the delivery and measurement of high-value care. To begin transitioning to value-based healthcare in CHD, the International Consortium for Health Outcomes Measurement aligned an international collaborative of CHD experts, patient representatives, and other stakeholders to construct a standard set of outcomes and risk-adjustment variables that are meaningful to patients.Methods and results: The primary aim was to identify a minimum standard set of outcomes to be used by health systems worldwide. The methodological process included four key steps: 1) develop a working group representative of all CHD stakeholders; 2) conduct extensive literature reviews to identify scope, outcomes of interest, tools used to measure outcomes, and case-mix adjustment variables; 3) create the outcome set using a series of multi-round Delphi processes; and 4) disseminate set worldwide. The WG established a 15-item outcome set, incorporating physical, mental, social, and overall health outcomes accompanied by tools for measurement and case-mix adjustment variables. Patients with any CHD diagnoses of all ages are included. Following an open review process, over 80% of patients and providers surveyed agreed with the set in its final form.Conclusion: This is the first international development of a stakeholder-informed standard set of outcomes for CHD. It can serve as a first step for a lifespan outcomes measurement approach to guide benchmarking and improvement among health systems

Development of an international standard set of clinical and patient-reported outcomes for children and adults with congenital heart disease: A report from the International Consortium for Health Outcomes Measurement Congenital Heart Disease Working Group

Aims: Congenital heart disease (CHD) is the most common congenital malformation. Despite the worldwide burden to patient wellbeing and health system resource utilization, tracking of long-term outcomes is lacking, limiting the delivery and measurement of high-value care. To begin transitioning to value-based healthcare in CHD, the International Consortium for Health Outcomes Measurement aligned an international collaborative of CHD experts, patient representatives, and other stakeholders to construct a standard set of outcomes and risk-adjustment variables that are meaningful to patients. Methods and results: The primary aim was to identify a minimum standard set of outcomes to be used by health systems worldwide. The methodological process included four key steps: (i) develop a working group representative of all CHD stakeholders; (ii) conduct extensive literature reviews to identify scope, outcomes of interest, tools used to measure outcomes, and case-mix adjustment variables; (iii) create the outcome set using a series of multi-round Delphi processes; and (iv) disseminate set worldwide. The Working Group established a 15-item outcome set, incorporating physical, mental, social, and overall health outcomes accompanied by tools for measurement and case-mix adjustment variables. Patients with any CHD diagnoses of all ages are included. Following an open review process, over 80% of patients and providers surveyed agreed with the set in its final form. Conclusion: This is the first international development of a stakeholder-informed standard set of outcomes for CHD. It can serve as a first step for a lifespan outcomes measurement approach to guide benchmarking and improvement among health systems