Priority-setting for hospital funding of high-cost innovative drugs and therapeutics: A qualitative institutional case study.

OBJECTIVES: Rising costs of innovative drugs and therapeutics (D&Ts) have led to resource allocation challenges for healthcare institutions. There is limited evidence to guide priority-setting for institutional funding of high-cost D&Ts. This study sought to identify and elaborate on the substantive principles and procedures that should inform institutional funding decisions for high-cost off-formulary D&Ts through a case study of a quaternary care paediatric hospital. METHODS: Semi-structured, qualitative interviews, both virtual and in-person, were conducted with institutional stakeholders (i.e. staff clinicians, senior leadership, and pharmacists) (n = 23) and two focus groups at The Hospital for Sick Children in Toronto, Canada. Participants involved in, and impacted by, high-cost off-formulary drug funding decisions were recruited through stratified, purposive sampling. Participants were approached for study involvement between July 27, 2020 and June 7, 2022. Data was analysed through reflexive thematic analysis. RESULTS: Institutional resource allocation for high-cost D&Ts was identified as ethically challenging but critical to sustainable access to novel therapies. Important substantive principles included: 1) clinical evidence of safety and efficacy, 2) economic considerations (direct costs, opportunity costs, value for money), 3) ethical principles (social justice, professional/organizational responsibility), and 4) disease-specific considerations. Multidisciplinary deliberation was identified as an essential procedural component of decision-making. Participants identified tension between innovation and the need for evidence-based decision-making; clinician and institutional responsibilities; and value for money and social justice. Participants emphasized the role of health system-level funding allocation in alleviating the financial and moral burden of decision-making by institutions. CONCLUSIONS: This study identifies values and processes to aid in the development and implementation of institutional resource allocation frameworks for high-cost innovative D&Ts.

Penicillin allergy de-labeling: Adaptation of risk stratification tool for patients and families.

Penicillin allergy is reported in 10% of the population; however, over 90% of patients are deemed non-allergic upon allergist assessment. The goal of this quality improvement project is to validate a patient-driven assessment tool to safely identify patients at low risk of penicillin allergy and de-label them. Pediatric patients and pregnant women referred to the institution's allergy clinics for penicillin allergy assessment were invited to use the patient tool to complete a self-assessment, resulting in the assignment of a risk category. The risk stratification determined using the patient tool was compared against the allergist's assessment. The patient tool demonstrated agreement with the allergist assessment in 57/84 (67.9%, 95% CI [56.7%,77.4%]) assessments, intra-class correlation (ICC) = 0.618, p < 0.001. In 22/84 (26.2%) assessments, the patient tool determined a higher risk category, primarily due to differences in patients' perceived timing and description of symptoms. Only 5/84 (6.0%) patients were placed in a lower risk category by the patient tool compared to the allergist assessment. The patient tool demonstrates good validity in determining penicillin allergy risk, offering potential as a method of empowering patients to advocate in their care. Iterative changes to the patient tool will be applied to increase agreement.

Gluten-Free Diet Knowledge and Adherence in Adolescents with Celiac Disease: A Cross-Sectional Study.

Objectives: This study examined the relationship between knowledge of, and adherence to, the gluten-free diet (GFD) in a local population of adolescents with celiac disease (CD). The secondary objectives were to identify information sources used to learn about the GFD and to compare adolescents' and parents' knowledge of the GFD. Methods: Adolescents (12-17 years) with CD and their parents from pediatric gastroenterology clinics in Calgary, Alberta, completed an online survey containing a knowledge assessment (Gluten-Free Diet Quiz [GFD-Q]), an adherence scale, questions about GFD information sources, and demographic/clinical information. GFD-Q scores were deemed "sufficient knowledge" with correct identification of 3/3 gluten-containing foods, ≥4/7 gluten-free foods, and ≥ 4/7 foods that may contain gluten; otherwise, scores were termed "insufficient knowledge". Results: Of the 40 adolescent-parent pairs, 15 of 40 adolescents (37%) had sufficient knowledge, and 25 of 40 adolescents (63%) had insufficient knowledge. Within the insufficient knowledge group, 14 of 25 (56%) did not correctly identify enough allowed gluten-free foods. Parents scored higher on the GFD-Q (67% had sufficient knowledge). Adolescents reported overall adherence to the GFD (88%), with adherence being similar between the sufficient and insufficient knowledge groups (80% versus 92%). The most helpful information sources included physicians, another person with CD, parent(s), and Google; apps were infrequently used. Conclusion: Adolescents report good adherence; however, they struggle with GFD knowledge, particularly in identifying gluten-free foods. Further research is required to explore GFD educational tools, including mobile apps and dietician-led teaching sessions.