ABSTRACT
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 30 topics across 4 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 12 software. RESULTS: A total of 168 studies were included for analysis. Only 4 (2%) focused on paediatrics, while 21 (12%) centred on adults and the remainder (85%) did not specify. Thirty-two (19%) studies discussed the importance of high-cost therapeutics and 34 (23%) 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.
Subject(s)
Health Facilities , Rare Diseases , Adult , Humans , Child , Rare Diseases/drug therapy , Hospitals , Biomedical Technology , Drug CostsABSTRACT
Many medical products that are widely available and commonly used are of animal origin, which can be problematic for those who identify as followers of a particular religion, or have moral commitments or dietary preferences that prohibit or restrict the ingestion of animal products. Given that people are becoming more conscious of the products they ingest, however, we suspect this might be an issue in the foreseeable future, particularly in a multicultural and diverse community like Toronto, Canada. Failure to provide services sensitive to these beliefs and preferences may result in a refusal of medical treatment. In this paper, we aim to identify and explore issues relating to the refusal of animal-derived medical products in paediatric settings by exploring three clinical cases.
