Institutional Priority-Setting for Novel Drugs and Therapeutics: A Qualitative Systematic Review.

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.

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.

Standard of Care in Pediatrics: Integrating Family-Centred Care and Social Determinants of Health.

In pediatric healthcare, there is a recognized need to address social determinants of health (SDOH) to positively influence child health and development. In addition, family-centred care (FCC) recognizes the value of the family unit in affecting individual patient experiences of illness and care. However, pediatric healthcare that incorporates principles of FCC and SDOH may be interpreted as calling on clinicians to deviate from or add to practices that form an accepted standard of care. This paper explores the legal and ethical considerations of doing so and describes practical responses to these challenging situations.

The Potential Value of the U.N. Convention on the Rights of the Child in Pediatric Bioethics Settings.

This article provides support for the use of a particular international human rights law document, the U.N. Convention on the Rights of the Child (CRC), in contemporary pediatric bioethics practice without relying on the legally binding force of the document. It first demonstrates that the CRC's core commitments and values substantially overlap with the core commitments and values of mainstream bioethics and with the laws of many domestic jurisdictions where mainstream bioethics are currently practiced. It then explores some implications of this overlap. For instance, the substantial international human rights law scholarship on how to understand these commitments and values can be helpful in suggesting ways to operationalize them in domestic bioethics practice and can offer insightful, internationally generated ethical perspectives that may not have been considered. The article also argues that the CRC can help health-care organizations develop policies consistent with the best interests of children and that the CRC can serve as a common language of values for transnational health-care collaborations. However, as a final case discussion demonstrates, whatever the merits of the CRC, one may face practical difficulties in trying to use it.

Identifying prioritization criteria to supplement critical care triage protocols for the allocation of ventilators during a pandemic influenza.

The purpose of this study was to identify supplementary criteria to provide direction when the Ontario Health Plan for an Influenza Pandemic (OHPIP) critical care triage protocol is rendered insufficient by its inability to discriminate among patients assessed as urgent, and there are insufficient critical care resources available to treat those in that category. To accomplish this task, a Supplementary Criteria Task Force for Critical Care Triage was struck at the University of Toronto Joint Centre for Bioethics. The task force reviewed publically available protocols and policies on pandemic flu planning, identified 13 potential triage criteria and determined a set of eight key ethical, legal and practical considerations against which it assessed each criterion. An online questionnaire was distributed to clinical, policy and community stakeholders across Canada to obtain feedback on the 13 potential triage criteria toward selecting those that best met the eight considerations. The task force concluded that the balance of arguments favoured only two of the 13 criteria it had identified for consideration: first come, first served and random selection. The two criteria were chosen in part based on a need to balance the clearly utilitarian approach employed in the OHPIP with equity considerations. These criteria serve as a defensible "fail safe" mechanism for any triage protocol.

Adoption of an innovation to repair aortic aneurysms at a Canadian hospital: a qualitative case study and evaluation.

BACKGROUND: Priority setting in health care is a challenge because demand for services exceeds available resources. The increasing demand for less invasive surgical procedures by patients, health care institutions and industry, places added pressure on surgeons to acquire the appropriate skills to adopt innovative procedures. Such innovations are often initiated and introduced by surgeons in the hospital setting. Decision-making processes for the adoption of surgical innovations in hospitals have not been well studied and a standard process for their introduction does not exist. The purpose of this study is to describe and evaluate the decision-making process for the adoption of a new technology for repair of abdominal aortic aneurysms (endovascular aneurysm repair [EVAR]) in an academic health sciences centre to better understand how decisions are made for the introduction of surgical innovations at the hospital level. METHODS: A qualitative case study of the decision to adopt EVAR was conducted using a modified thematic analysis of documents and semi-structured interviews. Accountability for Reasonableness was used as a conceptual framework for fairness in priority setting processes in health care organizations. RESULTS: There were two key decisions regarding EVAR: the decision to adopt the new technology in the hospital and the decision to stop hospital funding. The decision to adopt EVAR was based on perceived improved patient outcomes, safety, and the surgeons' desire to innovate. This decision involved very few stakeholders. The decision to stop funding of EVAR involved all key players and was based on criteria apparent to all those involved, including cost, evidence and hospital priorities. Limited internal communications were made prior to adopting the technology. There was no formal means to appeal the decisions made. CONCLUSION: The analysis yielded recommendations for improving future decisions about the adoption of surgical innovations. ese empirical findings will be used with other case studies to help develop guidelines to help decision-makers adopt surgical innovations in Canadian hospitals.