Daniel E. Wang - Department of Paediatrics, University of Toronto, Toronto, ON, Canada
Maram Hassanein - Department of Bioethics, The Hospital for Sick Children, Toronto, ON, Canada
Yasmeen Razvi - Temerty Faculty of Medicine, University of Toronto, Toronto, ON, Canada
Randi Zlotnik Shaul - Department of Paediatrics, University of Toronto, Toronto, ON, Canada
Avram Denburg - Department of Paediatrics, University of Toronto, Toronto, ON, Canada

Background  There is a lack of guidance on approaches to formulary management and funding for high-cost drugs and therapeutics by individual healthcare institutions. The objective of this review was to assess institutional approaches to resource allocation for such therapeutics, with a particular focus on paediatric and rare disease populations.  Methods  A search of Embase and MEDLINE was conducted for studies relevant to decision-making for off-formulary, high-cost drugs and therapeutics. Abstracts were evaluated for inclusion based on the Simple Multiple-Attribute Rating Techniques (SMART) criteria. A framework of ۳۰ topics across ۴ categories was used to guide data extraction and was based on findings from the initial abstract review and previous health technology assessment (HTA) publications. Reflexive thematic analysis was conducted using QSR NVivo ۱۲ software. Results  A total of ۱۶۸ studies were included for analysis. Only ۴ (۲%) focused on paediatrics, while ۲۱ (۱۲%) centred on adults and the remainder (۸۵%) did not specify. Thirty-two (۱۹%) studies discussed the importance of high-cost therapeutics and ۳۴ (۲۳%) focused on rare/orphan drugs. Five themes were identified as being relevant to institutional decision-making for high-cost therapeutics: institutional strategy, substantive criteria, procedural considerations, guiding principles and frameworks, and operational activities. Each of these themes encompassed several sub-themes and was complemented by a sixth category specific to paediatrics and rare diseases. Conclusion  The rising cost of novel drugs and therapeutics underscores the need for robust, evidence-based, and ethically defensible decision-making processes for health technology funding, particularly at the hospital level. Our study highlights practices and themes to aid decision-makers in thinking critically about institutional, substantive, procedural, and operational considerations in support of legitimate decisions about institutional funding of high-cost drugs and therapeutics, as well as opportunities and challenges that exist for paediatric and rare disease populations.

Health Technology Assessment, Paediatrics, Rare/Orphan Diseases, Institutional Policy, High-Cost Drugs, Resource Allocation