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.

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.

Policy Rogue or Policy Entrepreneur? The Forms and Impacts of "Joined-Up Governance" for Child Health.

Joined-up governance (JUG) approaches have gained attention as mechanisms for tackling wicked policy problems, particularly in intersectoral areas such as child health, where multiple ministries that deliver health and social services must collaborate if they are to be effective. Growing attention to the need to invest in early childhood to improve health and developmental trajectories, including through developmental screening, illustrate the challenges of JUG for child health. Using a comparative case study design comprised of the qualitative analysis of documents and key informant interviews, this work sought to explain how and why visible differences in policy choices have been made across two Canadian jurisdictions (Ontario and Manitoba). Specifically, we sought to understand two dimensions of governance (structure and process) alongside an illustrative example-the case of developmental screening, including how insiders viewed the impacts of governance arrangements in this instance. The two jurisdictions shared a commitment to evidence-based policy making and a similar vision of JUG for child health. Despite this, we found divergence in both governance arrangements and outcomes for developmental screening. In Manitoba, collaboration was prioritized, interests were aligned in a structured decision-making process, evidence and evaluation capacity were inherent to agenda setting, and implementation was considered up front. In Ontario, interests were not aligned and instead decision making operated in an opaque and siloed manner, with little consideration of implementation issues. In these contexts, Ontario pursued developmental screening, whereas Manitoba did not. While both jurisdictions aimed at JUG, only Manitoba developed a coordinated JUG system, whereas Ontario operated as a non-system. As a result, Manitoba's governance system had the capacity to stop 'rogue' action, prioritizing investments in accordance with authorized evidence. In contrast, in the absence of a formal system in Ontario, policy 'entrepreneurs' were able to seize a window of opportunity to invest in child health.

Half a Century of Wilson & Jungner: Reflections on the Governance of Population Screening.

Background: In their landmark report on the "Principles and Practice of Screening for Disease" (1968), Wilson and Jungner noted that the practice of screening is just as important for securing beneficial outcomes and avoiding harms as the formulation of principles. Many jurisdictions have since established various kinds of "screening governance organizations" to provide oversight of screening practice. Yet to date there has been relatively little reflection on the nature and organization of screening governance itself, or on how different governance arrangements affect the way screening is implemented and perceived and the balance of benefits and harms it delivers. Methods: An international expert policy workshop convened by Sturdy, Miller and Hogarth. Results: While effective governance is essential to promote beneficial screening practices and avoid attendant harms, screening governance organizations face enduring challenges. These challenges are social and ethical as much as technical. Evidence-based adjudication of the benefits and harms of population screening must take account of factors that inform the production and interpretation of evidence, including the divergent professional, financial and personal commitments of stakeholders. Similarly, when planning and overseeing organized screening programs, screening governance organizations must persuade or compel multiple stakeholders to work together to a common end. Screening governance organizations in different jurisdictions vary widely in how they are constituted, how they relate to other interested organizations and actors, and what powers and authority they wield. Yet we know little about how these differences affect the way screening is implemented, and with what consequences. Conclusions: Systematic research into how screening governance is organized in different jurisdictions would facilitate policy learning to address enduring challenges. Even without such research, informal exchange and sharing of experiences between screening governance organizations can deliver invaluable insights into the social as well as the technical aspects of governance.

What's Involved with Wanting to Be Involved? Comparing Expectations for Public Engagement in Health Policy across Research and Care Contexts.

OBJECTIVES: We explored public preferences for involvement in health policy decisions, across the contexts of medical research and healthcare. APPROACH: We e-surveyed a sample of Canadians, categorizing respondents by preferences for decision control: (1) more authority; (2) more input; (3) status quo. Two generalized ordered logistic regressions assessed influences on preferences. RESULTS: The participation rate was 94%; 1,102 completed responses met quality criteria. The dominant preference was for more input (average = 52.0%), followed by status quo (average = 24.9%) and more authority (average = 21.1%), though preferences for more control were higher in healthcare (57.2%) than medical research (46.8%). Preferences for greater control were associated with constructs related to reduced trust in healthcare systems. CONCLUSION: The public expects health policy to account for public views, but not base decisions primarily on these views. More involvement was expected in healthcare than medical research policy. As opportunities for public involvement in health research grow, we anticipate increased desired involvement.

Using Newborn Screening Bloodspots for Research: Public Preferences for Policy Options.

OBJECTIVES: Retaining residual newborn screening (NBS) bloodspots for medical research remains contentious. To inform this debate, we sought to understand public preferences for, and reasons for preferring, alternative policy options. METHODS: We assessed preferences among 4 policy options for research use of residual bloodspots through a bilingual national Internet survey of a representative sample of Canadians. Fifty percent of respondents were randomly assigned to select reasons supporting these preferences. Understanding of and attitudes toward screening and research concepts, and demographics were assessed. RESULTS: Of 1102 respondents (94% participation rate; 47% completion rate), the overall preference among policy options was ask permission (67%); this option was also the most acceptable choice (80%). Assume permission was acceptable to 46%, no permission required was acceptable to 29%, and no research allowed was acceptable to 26%. The acceptability of the ask permission option was reduced among participants assigned to the reasoning exercise (84% vs 76%; P = .004). Compared with assume/no permission required, ordered logistic regression showed a significant reduction in preference for the ask permission option with greater understanding of concepts (odds ratio, 0.87; P < .001), greater confidence in science (odds ratio, 0.16; P < .001), and a perceived responsibility to contribute to research (odds ratio, 0.39; P < .001). CONCLUSIONS: Surveyed Canadians prefer that explicit permission is sought for storage and research use of NBS bloodspots. This preference was diminished when reasons supporting and opposing routine storage, and other policy options, were presented. Findings warrant consideration as NBS communities strategize to respond to shifting legislative contexts.

Governing population screening in an age of expansion: The case of newborn screening.

Newborn bloodspot screening is one of the most enduring and successful population screening initiatives. Yet technological innovation to permit simultaneous measurement of multiple biomarkers - and potentially, entire genomes - has spurred expansion and debate. Through a cross-jurisdictional comparison, we describe the varied roles and reach of screening-related governance structures in the United States, the United Kingdom, New Zealand and Canada, and highlight the distinct values and resources brought to bear by the genetics, public health and maternal-child health communities in adjudicating the benefits and burdens of expanded newborn screening. We call for the expansion of formal governance structures that are balanced in resources and perspective and mandated to ensure that the organization and delivery of newborn screening achieves optimal quality.

Public Perceptions of the Benefits and Risks of Newborn Screening.

BACKGROUND: Growing technological capacity and parent and professional advocacy highlight the need to understand public expectations of newborn population screening. METHODS: We administered a bilingual (French, English) Internet survey to a demographically proportional sample of Canadians in 2013 to assess preferences for the types of diseases to be screened for in newborns by using a discrete choice experiment. Attributes were: clinical benefits of improved health, earlier time to diagnosis, reproductive risk information, false-positive (FP) results, and overdiagnosed infants. Survey data were analyzed with a mixed logit model to assess preferences and trade-offs among attributes, interaction between attributes, and preference heterogeneity. RESULTS: On average, respondents were favorable toward screening. Clinical benefits were the most important outcome; reproductive risk information and early diagnosis were also valued, although 8% disvalued early diagnosis, and reproductive risk information was least important. All respondents preferred to avoid FP results and overdiagnosis but were willing to accept these to achieve moderate clinical benefit, accepting higher rates of harms to achieve significant benefit. Several 2-way interactions between attributes were statistically significant: respondents were willing to accept a higher FP rate for significant clinical benefit but preferred a lower rate for moderate benefit; similarly, respondents valued early diagnosis more when associated with significant rather than moderate clinical benefit. CONCLUSIONS: Members of the public prioritized clinical benefits for affected infants and preferred to minimize harms. These findings suggest support for newborn screening policies prioritizing clinical benefits over solely informational benefits, coupled with concerted efforts to avoid or minimize harms.

Public views on participating in newborn screening using genome sequencing.

Growing discussion on the use of whole-genome or exome sequencing (WG/ES) in newborn screening (NBS) has raised concerns regarding the generation of incidental information on millions of infants annually. It is unknown whether integrating WG/ES would alter public expectations regarding participation in universal NBS. We assessed public willingness to participate in NBS using WG/ES compared with current NBS. Our secondary objective was to assess the public's beliefs regarding a parental responsibility to participate in WG/ES-based NBS compared with current NBS. We examined self-reported attitudes regarding willingness to participate in NBS using a cross-sectional national survey of Canadian residents recruited through an internet panel, reflective of the Canadian population by age, gender and region. Our results showed that fewer respondents would be willing to participate in NBS using WG/ES compared with NBS using current technologies (80 vs 94%, P<0.001), or perceived a parental responsibility to participate in WG/ES-based NBS vs current NBS (30 vs 48%, P<0.001). Our findings suggest that integrating WG/ES into NBS might reduce participation, and challenge the moral authority that NBS programmes rely upon to ensure population benefits. These findings point to the need for caution in the untargeted use of WG/ES in public health contexts.

Technology-enabled academic detailing: computer-mediated education between pharmacists and physicians for evidence-based prescribing.

BACKGROUND: Academic detailing (AD) is the practice of specially trained pharmacists with detailed medication knowledge meeting with physicians to share best practices of prescribing. AD has demonstrated efficacy in positively influencing physicians' prescribing behavior. Nevertheless, a key challenge has been that physicians in rural and remote locations, or physicians who are time challenged, have limited ability to participate in face-to-face meetings with academic detailers, as these specially trained academic detailers are primarily urban-based and limited in numbers. OBJECTIVE: To determine the feasibility of using information technologies to facilitate communication between academic detailers and physicians (known as Technology-Enabled Academic Detailing or TEAD) through a comparison to traditional face-to-face academic detailing (AD). Specifically, TEAD is compared to AD in terms of the ability to aid physicians in acquiring evidence-informed prescribing information on diabetes-related medications, measured in terms of time efficiency, satisfaction of both physicians and pharmacists, and quality of knowledge exchange. METHODS: General Practitioner Physicians (n=105) and pharmacists (n=12) were recruited from across British Columbia. Pharmacists were trained to be academic detailers on diabetes medication usage. Physicians were assigned to one of four intervention groups to receive four academic detailing sessions from trained pharmacists. Intervention groups included: (1) AD only, (2) TEAD only, (3) TEAD crossed over to AD at midpoint, and (4) AD crossed over to TEAD at midpoint. Evaluation included physician-completed surveys before and after each session, pharmacist logs after each detailing session, interviews and focus groups with physicians and pharmacists at study completion, as well as a technical support log to record all phone calls and emails from physicians and pharmacists regarding any technical challenges during the TEAD sessions, or usage of the web portal. RESULTS: Because recruitment was very low for the cross over groups, we analyzed the results in two groups instead: AD only and TEAD only. 354 sessions were conducted (AD=161, TEAD=193). Of these, complete data were available for 300 sessions, which were included in analysis (AD=133, TEAD=167). On average, TEAD sessions were 49min long, and AD sessions 81min long. Overall, physicians enjoyed both modalities of academic detailing (AD and TEAD) because they received information that both reinforced their existing diabetes knowledge and also provided new prescribing insights and approaches. CONCLUSION: The results suggest that TEAD is an acceptable alternative to AD for providing physicians advice about prescribing. TEAD is more time efficient, facilitates effective knowledge exchange and interprofessional collaboration, and can reach those physicians virtually where face-to-face AD is not possible or practical. LIMITATIONS: Due to logistics, physicians were allocated, rather than randomized, to receive AD and/or TEAD.

Electronic communities of practice: guidelines from a project.

The timely incorporation of health research into the routine practice of individual health practitioners and interprofessional teams is a widely recognized and ongoing challenge. Health professional engagement and learning is an important cog in the wheel of knowledge translation; passive dissemination of evidence through journals and clinical practice guidelines is inadequate when used alone as an intervention to change the practices of the health professionals.An evolving body of research suggests that communities of practice can be effective in facilitating the uptake of best practices by individual health professionals and teams. Modern information technologies can extend the boundaries and reach of these communities, forming electronic communities of practice (eCoP) that can be used to promote intra- and interprofessional continuing professional development (CPD) and team-based, patient-centered care. However, examples of eCoPs and examination of their characteristics are lacking in the literature. In this paper, we discuss guidelines for developing eCoP. These guidelines will be helpful for others considering the use of the eCoP model in interprofessional learning and practice.