Development of a Value Assessment Framework for Pediatric Health Technologies Using Multicriteria Decision Analysis: Expanding the Value Lens for Funding Decision Making.

OBJECTIVES: A health technology assessment (HTA) does not systematically account for the circumstances and needs of children and youth. To supplement HTA processes, we aimed to develop a child-tailored value assessment framework using a multicriteria decision analysis approach. METHODS: We constructed a multicriteria-decision-analysis-based model in multiple phases to create the Comprehensive Assessment of Technologies for Child Health (CATCH) framework. Using a modified Delphi process with stakeholders having broad disciplinary and geographic variation (N = 23), we refined previously generated criteria and developed rank-based weights. We established a criterion-pertinent scoring rubric for assessing incremental benefits of new drugs. Three clinicians independently assessed comprehension by pilotscoring 9 drugs. We then validated CATCH for 2 childhood cancer therapies through structured deliberation with an expert panel (N = 10), obtaining individual scores, consensus scores, and verbal feedback. Analyses included descriptive statistics, thematic analysis, exploratory disagreement indices, and sensitivity analysis. RESULTS: The modified Delphi process yielded 10 criteria, based on absolute importance/relevance and agreed importance (median disagreement indices = 0.34): Effectiveness, Child-specific Health-related Quality of Life, Disease Severity, Unmet Need, Therapeutic Safety, Equity, Family Impacts, Life-course Development, Rarity, and Fair Share of Life. Pilot scoring resulted in adjusted criteria definitions and more precise score-scaling guidelines. Validation panelists endorsed the framework's key modifiers of value. Modes of their individual prescores aligned closely with deliberative consensus scores. CONCLUSIONS: We iteratively developed a value assessment framework that captures dimensions of child-specific health and nonhealth gains. CATCH could improve the richness and relevance of HTA decision making for children in Canada and comparable health systems.

Fake It 'Til You Make It.

In this narrative medicine essay, a medical student recounts the high price to their mental health for embracing the advice of their childhood therapist to "fake it 'til you make it.".

Transgender health objectives of training for adult Endocrinology and Metabolism programs: Outcomes of a modified-Delphi study.

BACKGROUND: Transgender people encounter significant barriers when seeking timely, high-quality healthcare, resulting in unmet medical needs with increased rates of diabetes, asthma, chronic obstructive pulmonary disease, and HIV. The paucity of postgraduate medical education to invest in standardization of transgender health training sustains these barriers, leaving physicians feeling unprepared and averse to provide transgender health care. Closing this education gap and improving transgender healthcare necessitates the development of consensus-built transgender health objectives of training (THOOT), particularly in Adult Endocrinology and Metabolism Residency programs. METHODS: We conducted a two-round modified-Delphi process involving a nationally representative panel of experts, including Adult Endocrinology and Metabolism program directors, physician content experts, residents, and transgender community members, to identify THOOT for inclusion in Canadian Endocrinology and Metabolism Residency programs. Participants used a 5-point Likert scale to assess THOOT importance for curricular inclusion, with opportunities for written feedback. Data was collected through Qualtrics and analyzed after each round. FINDINGS: In the first Delphi round, panelists reviewed and rated 81 literature extracted THOOT, achieving consensus on all objectives. Following panelists' feedback, 5 THOOT were added, 9 removed, 34 consolidated into 12 objectives, and 47 were rephrased or retained. In the second Delphi round, panelists assessed 55 THOOT. Consensus was established for 8 THOOT. Program directors' post-Delphi feedback further consolidated objectives to arrive at 4 THOOT for curriculum inclusion. CONCLUSIONS: To our knowledge, this is the first time a consensus-based approach has been used to establish THOOT for any subspecialty postgraduate medicine program across Canada or the United States. Our results lay the foundation towards health equity and social justice in transgender health medical education, offering a blueprint for future innovations.

The discovery of the DNA methylation episignature for Duchenne muscular dystrophy.

Duchenne Muscular Dystrophy (DMD) is an X-linked recessive neuromuscular disorder characterized by progressive muscle weakness due to loss of function mutations in the dystrophin gene. Variation in clinical presentation, the rate of disease progression, and treatment responsiveness have been observed amongst DMD patients, suggesting that factors beyond the loss of dystrophin may contribute to DMD pathophysiology. Epigenetic mechanisms are becoming recognized as important factors implicated in the etiology and progression of various diseases. A growing number of genetic syndromes have been associated with unique genomic DNA methylation patterns (called "episignatures") that can be used for diagnostic testing and as disease biomarkers. To further investigate DMD pathophysiology, we assessed the genome-wide DNA methylation profiles of peripheral blood from 36 patients with DMD using the combination of Illumina Infinium Methylation EPIC bead chip array and EpiSign technology. We identified a unique episignature for DMD that whose specificity was confirmed in relation other neurodevelopmental disorders with known episignatures. By modeling the DMD episignature, we developed a new DMD episignature biomarker and provided novel insights into the molecular pathogenesis of this disorder, which have the potential to advance more effective, personalized approaches to DMD care.

Translating Precision Health for Pediatrics: A Scoping Review.

Precision health aims to personalize treatment and prevention strategies based on individual genetic differences. While it has significantly improved healthcare for specific patient groups, broader translation faces challenges with evidence development, evidence appraisal, and implementation. These challenges are compounded in child health as existing methods fail to incorporate the physiology and socio-biology unique to childhood. This scoping review synthesizes the existing literature on evidence development, appraisal, prioritization, and implementation of precision child health. PubMed, Scopus, Web of Science, and Embase were searched. The included articles were related to pediatrics, precision health, and the translational pathway. Articles were excluded if they were too narrow in scope. In total, 74 articles identified challenges and solutions for putting pediatric precision health interventions into practice. The literature reinforced the unique attributes of children and their implications for study design and identified major themes for the value assessment of precision health interventions for children, including clinical benefit, cost-effectiveness, stakeholder values and preferences, and ethics and equity. Tackling these identified challenges will require developing international data networks and guidelines, re-thinking methods for value assessment, and broadening stakeholder support for the effective implementation of precision health within healthcare organizations. This research was funded by the SickKids Precision Child Health Catalyst Grant.