A Geographically Weighted Cost-effectiveness Analysis of Newborn Cytomegalovirus Screening.

Background: Early identification of newborns with congenital cytomegalovirus (CMV) is necessary to provide antiviral therapy and other interventions that can improve outcomes. Prior research demonstrates that universal newborn CMV screening would be the most cost-effective approach to identifying newborns who are infected. CMV is not uniformly prevalent, and it is uncertain whether universal screening would remain cost-effective in lower-prevalence neighborhoods. Our aim was to identify geographic heterogeneity in the cost-effectiveness of universal newborn CMV screening by combining a geospatial analysis with a preexisting cost-effectiveness analysis. Methods: This study used the CMV testing results and zip code location data of 96 785 newborns in 7 metropolitan areas who had been tested for CMV as part of the CMV and Hearing Multicenter Screening study. A hierarchical bayesian generalized additive model was constructed to evaluate geographic variability in the odds of CMV. The zip code-level odds of CMV were then used to weight the results of a previously published model evaluating universal CMV screening vs symptom-targeted screening. Results: The odds of CMV were heterogeneous over large geographic scales, with the highest odds in the southeastern United States. Universal screening was more cost-effective and afforded more averted cases of severe hearing loss than targeted testing. Universal screening remained the most cost-effective option even in areas with the lowest CMV prevalence. Conclusions: Universal newborn CMV screening is cost-effective regardless of underlying CMV prevalence and is the preferred strategy to reduce morbidity from congenital CMV.

Application of Multi-Criteria Decision Analysis (MCDA) to Prioritize Real-World Evidence Studies for Health Technology Management: Outcomes and Lessons Learned by the Canadian Real-World Evidence for Value of Cancer Drugs (CanREValue) Collaboration.

Multi-criteria decision analysis (MCDA) is a value assessment tool designed to help support complex decision-making by incorporating multiple factors and perspectives in a transparent, structured approach. We developed an MCDA rating tool, consisting of seven criteria evaluating the importance and feasibility of conducting potential real-world evidence (RWE) studies aimed at addressing uncertainties stemming from initial cancer drug funding recommendations. In collaboration with the Canadian Agency for Drugs and Technologies in Health's Provincial Advisory Group, a validation exercise was conducted to further evaluate the application of the rating tool using RWE proposals varying in complexity. Through this exercise, we aimed to gain insight into consensus building and deliberation processes and to identify efficiencies in the application of the rating tool. An experienced facilitator led a multidisciplinary committee, consisting of 11 Canadian experts, through consensus building, deliberation, and prioritization. A total of nine RWE proposals were evaluated and prioritized as low (n = 4), medium (n = 3), or high (n = 2) priority. Through an iterative process, efficiencies and recommendations to improve the rating tool and associated procedures were identified. The refined MCDA rating tool can help decision-makers prioritize important and feasible RWE studies for research and can enable the use of RWE for the life-cycle evaluation of cancer drugs.

An Economic Analysis of the Association Among Secondary Health Conditions, Health Care Costs, and Quality of Life for Persons With Spinal Cord Injury.

Background: A previous analysis using the Canadian Spinal Cord Injury (SCI) Community Survey data identified that there were individuals with a high number of secondary health conditions, high health care utilization, poor health outcomes, and unmet health care needs. Objectives: The objectives of this study were to estimate the annual health care costs of persons with SCI who report secondary health conditions, and to determine the association between these secondary health conditions with health care utilization and self-reported life satisfaction and quality of life. Methods: The survey respondents were divided into four groups: traumatic SCI (tSCI; those who said they received needed care and those who said they did not) and nontraumatic SCI (ntSCI; those who said they received needed care and those who said they did not). The average annual health care costs per respondent were estimated for each group. Using regression analysis, we estimated the change in average annual health care costs that were associated with an additional secondary health condition for respondents in each group. Results: Participants who reported not receiving needed care had on average 23% more secondary health conditions than those receiving needed care. The increase in average annual health care costs associated with one additional secondary health conditions was between $428 ($37-$820) (ntSCI, receiving needed care) and $1240 ($739-$1741) (tSCI, not receiving needed care). Conclusion: This study provides insight into potential cost savings associated with a reduction of secondary health conditions as well as an estimate of the reduction in health care costs associated with moving from not receiving all needed care to receiving needed care.

Development of a Multi-Criteria Decision Analysis Rating Tool to Prioritize Real-World Evidence Questions for the Canadian Real-World Evidence for Value of Cancer Drugs (CanREValue) Collaboration.

The Canadian Real-world Evidence for Value of Cancer Drugs (CanREValue) collaboration developed an MCDA rating tool to assess and prioritize potential post-market real-world evidence (RWE) questions/uncertainties emerging from public drug funding decisions in Canada. In collaboration with a group of multidisciplinary stakeholders from across Canada, the rating tool was developed following a three-step process: (1) selection of criteria to assess the importance and feasibility of an RWE question; (2) development of rating scales, application of weights and calculating aggregate scores; and (3) validation testing. An initial MCDA rating tool was developed, composed of seven criteria, divided into two groups. Group A criteria assess the importance of an RWE question by examining the (1) drug's perceived clinical benefit, (2) magnitude of uncertainty identified, and (3) relevance of the uncertainty to decision-makers. Group B criteria assess the feasibility of conducting an RWE analysis including the (1) feasibility of identifying a comparator, (2) ability to identify cases, (3) availability of comprehensive data, and (4) availability of necessary expertise and methodology. Future directions include partnering with the Canadian Agency for Drugs and Technology in Health's Provincial Advisory Group for further tool refinement and to gain insight into incorporating the tool into drug funding deliberations.

Addressing prioritization in healthcare amidst a global pandemic.

Trade-offs abound in healthcare yet depending on where one stands relative to the stages of a pandemic, choice making may be more or less constrained. During the early stages of COVID-19 when there was much uncertainty, healthcare systems faced greater constraints and focused on the singular criterion of "flattening the curve." As COVID-19 progressed and the first wave diminished (relatively speaking depending on the jurisdiction), more opportunities presented for making explicit choices between COVID and non-COVID patients. Then, as the second wave surged, again decision makers were more constrained even as more information and greater understanding developed. Moving out of the pandemic to recovery, choice making becomes paramount as there are no set rules to lean back into historical patterns of resource allocation. In fact, the opportunity at hand, when using explicit tools for priority setting based on economic and ethical principles, is significant.

Practices of decision making in priority setting and resource allocation: a scoping review and narrative synthesis of existing frameworks.

BACKGROUND: Due to growing expenditures, health systems have been pushed to improve decision-making practices on resource allocation. This study aimed to identify which practices of priority setting and resource allocation (PSRA) have been used in healthcare systems of high-income countries. METHODS: A scoping literature review (2007-2019) was conducted to map empirical PSRA activities. A two-stage screening process was utilized to identify existing approaches and cluster similar frameworks. That was complemented with a gray literature and horizontal scanning. A narrative synthesis was carried out to make sense of the existing literature and current state of PSRA practices in healthcare. RESULTS: One thousand five hundred eighty five references were found in the peer-reviewed literature and 25 papers were selected for full-review. We identified three major types of decision-making framework in PSRA: 1) Program Budgeting and Marginal Analysis (PBMA); 2) Health Technology Assessment (HTA); and 3) Multiple-criteria value assessment. Our narrative synthesis indicates these formal frameworks of priority setting and resource allocation have been mostly implemented in episodic exercises with poor follow-up and evaluation. There seems to be growing interest for explicit robust rationales and ample stakeholder involvement, but that has not been the norm in the process of allocating resources within healthcare systems of high-income countries. CONCLUSIONS: No single dominate framework for PSRA appeared as the preferred approach across jurisdictions, but common elements exist both in terms of process and structure. Decision-makers worldwide can draw on our work in designing and implementing PSRA processes in their contexts.

Priority setting and resource allocation in the US health system: is there a place for hard caps?

PURPOSE: The United States devotes a larger share of its GDP to health care and spends more on health care per capita than any other country. The sheer size of the total spending on health care, at approximately $3.5 trillion in 2017, puts significant pressure on all payers and crowds out other forms of public and private spending. DESIGN/METHODOLOGY/APPROACH: In this brief commentary the authors suggest that, as part of the effort to deal with this pressure, the United States should look at borrowing a cost containment strategy from other countries: the use of hard caps on spending growth. The authors draw on our their experience of working with decision-makers over the last 20 years on the topic of priority setting to put forward some ideas on whether there is potential for application of trade-offs in the United States. FINDINGS: As hard caps force choices to be made, a necessary condition for successful implementation of this policy is the presence of an effective priority-setting framework to ensure that the right choices are made in operationalizing spending limitations. Work on this topic elsewhere can provide some insight into the use of a criteria-based framework for priority setting that purports transparency in decision-making to achieve value-based decisions. ORIGINALITY/VALUE: Other countries still have much work to do, but there is a substantial track record of using formal priority-setting approaches that could potentially inform practice in the United States. We suggest that there are key segments of the US healthcare system where the adoption of formal priority-setting frameworks to guide trade-off decisions is feasible. Piloting such activity in these contexts is the next natural step in this line of inquiry.

Value assessment of oncology drugs using a weighted criterion-based approach.

BACKGROUND: Globally, the rising cost of anticancer therapy has motivated efforts to quantify the overall value of new cancer treatments. Multicriteria decision analysis offers a novel approach to incorporate multiple criteria and perspectives into value assessment. METHODS: The authors recruited a diverse, multistakeholder group who identified and weighted key criteria to establish the drug assessment framework (DAF). Construct validity assessed the degree to which DAF scores were associated with past pan-Canadian Oncology Drug Review (pCODR) funding recommendations and European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS; version 1.1) scores. RESULTS: The final DAF included 10 criteria: overall survival, progression-free survival, response rate, quality of life, toxicity, unmet need, equity, feasibility, disease severity, and caregiver well-being. The first 5 clinical benefit criteria represent approximately 64% of the total weight. DAF scores ranged from 0 to 300, reflecting both the expected impact of the drug and the quality of supporting evidence. When the DAF was applied to the last 60 drugs (with reviewers blinded) reviewed by pCODR (2015-2018), those drugs with positive pCODR funding recommendations were found to have higher DAF scores compared with drugs not recommended (103 vs 63; Student t test P = .0007). DAF clinical benefit criteria mildly correlated with ESMO-MCBS scores (correlation coefficient, 0.33; 95% CI, 0.009-0.59). Sensitivity analyses that varied the criteria scores did not change the results. CONCLUSIONS: Using a structured and explicit approach, a criterion-based valuation framework was designed to provide a transparent and consistent method with which to value and prioritize cancer drugs to facilitate the delivery of affordable cancer care.

Assessing value in health care: using an interpretive classification system to understand existing practices based on a systematic review.

BACKGROUND: Implementing adequate strategies to assess the value of health services plays a central role in the effort to deal with the financial pressures faced by health care systems worldwide. This study aimed to understand which approaches to value assessment have been used in developed countries. METHODS: We conducted a rapid review and a gray literature search to identify value assessment frameworks. A two-stage screening process was utilized to identify existing approaches and cluster similar frameworks. In addition, we developed an interpretive classification system to make sense of existing approaches. RESULTS: One thousand one hundred seventy-six references were identified and 38 papers were selected for full-review. Among these 38 articles, 22 distinct approaches to assess value of health care interventions were identified and classified according to four points: 1) use of single or multiple considerations to base value estimates; 2) use of disease-specific or generic criteria; 3) reliance on process-based or outcomes-based consideration; and 4) type of input and evidence considered. CONCLUSIONS: The contextual nature of value assessment in health care becomes evident with the diversity of existing approaches. Despite the predominance of cases relying on the Incremental cost-effectiveness ratio as the measure of value, this approach has not been sufficient to meet the needs of decision-makers. The use of multiple criteria has become more and more important, as well as the consideration of patient-reported measures. Considerations of costs are not always explicit and consistent.

An initial health economic evaluation of pharmacogenomic testing in patients treated for childhood cancer with anthracyclines.

BACKGROUND: Anthracyclines are a class of highly effective chemotherapeutic drugs commonly used to treat cancer patients. Anthracyclines, however, are associated with the development of serious adverse reactions, including anthracycline-induced cardiotoxicity (ACT). It is not possible, within current practice, to accurately individualize treatment to minimize risk. PROCEDURE: Recently, genetic variants have been associated with the risk of ACT in children. Building on these findings and the related genetic test, a predictive model was developed which classifies pediatric patients by their risk of developing ACT. We assessed the value of this ACT-predictive risk classification in addressing ACT. RESULTS: With current care, the estimated average lifetime cost of ACT is $8,667 per anthracycline-treated patient and approximately 7% of patients are expected to die from ACT. The projected impact of the information from the new predictive model is a 17% reduction in the risk of mortality from ACT and savings of about 6%: lives saved and lower costs. CONCLUSION: The newly identified genetic variants associated with the risk of ACT provide information that allows a more reliable prediction of the risk of ACT for a given patient and can be obtained at a very moderate cost, which is expected to lead to meaningful progress in reducing harm and costs associated with ACT.

Cost-effectiveness of Universal and Targeted Newborn Screening for Congenital Cytomegalovirus Infection.

Importance: Congenital cytomegalovirus (cCMV) infection is a major cause of childhood deafness. Most cCMV infections are not diagnosed without newborn screening, resulting in missed opportunities for directed care. Objective: To estimate the cost-effectiveness of universal and targeted newborn cCMV screening programs compared with no cCMV screening. Design, Setting, and Participants: Models were constructed using rates and outcomes from prospective cohort studies of newborn cCMV screening in US postpartum care and early hearing programs. Costs of laboratory testing, treatment, and hearing loss were drawn from Medicaid data and published estimates. The benefits of cCMV screening were assumed to come from antiviral therapy for affected newborns to reduce hearing loss and from earlier identification of hearing loss with postnatal onset. Analyses were performed from July 2014 to March 2016. Interventions: Models compared universal or targeted cCMV screening of newborns with a failed hearing screen, with standard care for cCMV infection. Main Outcomes and Measures: The incremental costs of identifying 1 cCMV infection, identifying 1 case of cCMV-related hearing loss, and preventing 1 cochlear implant; the incremental reduction in cases of severe to profound hearing loss; and the differences in costs per infant screened by universal or targeted strategies under different assumptions about the effectiveness of antiviral treatment. Results: Among all infants born in the United States, identification of 1 case of cCMV infection by universal screening was estimated to cost $2000 to $10 000; by targeted screening, $566 to $2832. The cost of identifying 1 case of hearing loss due to cCMV was as little as $27 460 by universal screening or $975 by targeted screening. Assuming a modest benefit of antiviral treatment, screening programs were estimated to reduce severe to profound hearing loss by 4.2% to 13% and result in direct costs of $10.86 per newborn screened. However, savings of up to $37.97 per newborn screened were estimated when costs related to functionality were included. Conclusions and Relevance: Newborn screening for cCMV infection appears to be cost-effective under a wide range of assumptions. Universal screening offers larger net savings and the greatest opportunity to provide directed care. Targeted screening also appears to be cost-effective and requires testing for fewer newborns. These findings suggest that implementation of newborn cCMV screening programs is warranted.

Navigating stormy waters in times of fiscal uncertainty: Mitigating the challenges.

New approaches to resource allocation are providing healthcare managers with ways to meet budget pressures while maximizing benefit to patients and populations. But putting these approaches in place often involves significant organizational change to which some degree of resistance must be expected. The authors have seen seven common objections raised time and again. Here, we offer our best advice on how healthcare leaders can anticipate and respond proactively to these challenges.

Developing a multi-criteria approach for drug reimbursement decision making: an initial step forward.

BACKGROUND: Coverage decisions for a new drug revolve around the balance between perceived value and price. But what is the perceived value of a new drug? Traditionally, the assessment of such value has largely revolved around the estimation of cost-effectiveness. However, very few will argue that the cost-effectiveness ratio presents a fulsome picture of 'value'. Multi-criteria decision analysis (MCDA) has been advocated as an alternative to cost-effectiveness analysis and it has been argued that it better reflects real world decision-making. OBJECTIVE: The objective of this project was to address the issue of the lack of a satisfactory methodology to measure value for drugs by developing a framework to operationalize an MCDA approach incorporating societal values as they pertain to the value of drugs. METHODS: Two workshops were held, one in Toronto in conjunction with the CAPT annual conference, and one in Ottawa, as part of the annual CADTH Symposium. Notes were taken at both workshops and the data collected was analyzed using a grounded theory approach. The intent was to reflect, as accurately as possible, what was said at the workshops, without normative judgement. RESULTS: Results to date are a set of guiding principles and criteria. There are currently ten criteria: Comparative effectiveness, Adoption feasibility, Risks of adverse events, Patient autonomy, Societal benefit, Equity, Strength of evidence, Incidence/prevalence/severity of condition, Innovation, and Disease prevention/ health promotion. CONCLUSION: Much progress has been made and it is now time to share the results. Feedback will determine the final shape of the framework proposed.

Managing healthcare budgets in times of austerity: the role of program budgeting and marginal analysis.

Given limited resources, priority setting or choice making will remain a reality at all levels of publicly funded healthcare across countries for many years to come. The pressures may well be even more acute as the impact of the economic crisis of 2008 continues to play out but, even as economies begin to turn around, resources within healthcare will be limited, thus some form of rationing will be required. Over the last few decades, research on healthcare priority setting has focused on methods of implementation as well as on the development of approaches related to fairness and legitimacy and on more technical aspects of decision making including the use of multi-criteria decision analysis. Recently, research has led to better understanding of evaluating priority setting activity including defining 'success' and articulating key elements for high performance. This body of research, however, often goes untapped by those charged with making challenging decisions and as such, in line with prevailing public sector incentives, decisions are often reliant on historical allocation patterns and/or political negotiation. These archaic and ineffective approaches not only lead to poor decisions in terms of value for money but further do not reflect basic ethical conditions that can lead to fairness in the decision-making process. The purpose of this paper is to outline a comprehensive approach to priority setting and resource allocation that has been used in different contexts across countries. This will provide decision makers with a single point of access for a basic understanding of relevant tools when faced with having to make difficult decisions about what healthcare services to fund and what not to fund. The paper also addresses several key issues related to priority setting including how health technology assessments can be used, how performance can be improved at a practical level, and what ongoing resource management practice should look like. In terms of future research, one of the most important areas of priority setting that needs further attention is how best to engage public members.

Allocating limited resources in a time of fiscal constraints: a priority setting case study from Dalhousie University Faculty of Medicine.

Facing a projected $1.4M deficit on a $35M operating budget for fiscal year 2011/2012, members of the Dalhousie University Faculty of Medicine developed and implemented an explicit, transparent, criteria-based priority setting process for resource reallocation. A task group that included representatives from across the Faculty of Medicine used a program budgeting and marginal analysis (PBMA) framework, which provided an alternative to the typical public-sector approaches to addressing a budget deficit of across-the-board spending cuts and political negotiation. Key steps to the PBMA process included training staff members and department heads on priority setting and resource reallocation, establishing process guidelines to meet immediate and longer-term fiscal needs, developing a reporting structure and forming key working groups, creating assessment criteria to guide resource reallocation decisions, assessing disinvestment proposals from all departments, and providing proposal implementation recommendations to the dean. All departments were required to submit proposals for consideration. The task group approved 27 service reduction proposals and 28 efficiency gains proposals, totaling approximately $2.7M in savings across two years. During this process, the task group faced a number of challenges, including a tight timeline for development and implementation (January to April 2011), a culture that historically supported decentralized planning, at times competing interests (e.g., research versus teaching objectives), and reductions in overall health care and postsecondary education government funding. Overall, faculty and staff preferred the PBMA approach to previous practices. Other institutions should use this example to set priorities in times of fiscal constraints.

The challenge of obtaining information necessary for multi-criteria decision analysis implementation: the case of physiotherapy services in Canada.

BACKGROUND: As fiscal constraints dominate health policy discussions across Canada and globally, priority-setting exercises are becoming more common to guide the difficult choices that must be made. In this context, it becomes highly desirable to have accurate estimates of the value of specific health care interventions.Economic evaluation is a well-accepted method to estimate the value of health care interventions. However, economic evaluation has significant limitations, which have lead to an increase in the use of Multi-Criteria Decision Analysis (MCDA). One key concern with MCDA is the availability of the information necessary for implementation. In the Fall 2011, the Canadian Physiotherapy Association embarked on a project aimed at providing a valuation of physiotherapy services that is both evidence-based and relevant to resource allocation decisions. The framework selected for this project was MCDA. We report on how we addressed the challenge of obtaining some of the information necessary for MCDA implementation. METHODS: MCDA criteria were selected and areas of physiotherapy practices were identified. The building up of the necessary information base was a three step process. First, there was a literature review for each practice area, on each criterion. The next step was to conduct interviews with experts in each of the practice areas to critique the results of the literature review and to fill in gaps where there was no or insufficient literature. Finally, the results of the individual interviews were validated by a national committee to ensure consistency across all practice areas and that a national level perspective is applied. RESULTS: Despite a lack of research evidence on many of the considerations relevant to the estimation of the value of physiotherapy services (the criteria), sufficient information was obtained to facilitate MCDA implementation at the local level. CONCLUSIONS: The results of this research project serve two purposes: 1) a method to obtain information necessary to implement MCDA is described, and 2) the results in terms of information on the benefits provided by each of the twelve areas of physiotherapy practice can be used by decision-makers as a starting point in the implementation of MCDA at the local level.

Innovations in health service organization and delivery in northern rural and remote regions: a review of the literature.

OBJECTIVES: To identify and review innovations relevant to improving access, quality, efficiency and/or effectiveness in the organization and delivery of health care services in rural and remote areas. STUDY DESIGN: Literature review. METHODS: Key bibliographic databases that index health research were searched: MEDLINE, EMBASE and CINAHL. Other databases relevant to Arctic health were also accessed. Abstracts were assessed for relevancy and full articles were reviewed and categorized according to emergent themes. RESULTS: Many innovations in delivering services to rural and remote areas were identified, particularly in the public health realm. These innovations were grouped into 4 key themes: organizational structure of health services; utilization of telehealth and ehealth; medical transportation; and public health challenges. CONCLUSIONS: Despite the challenges facing rural and remote regions, there is a distinctly positive message from this broad literature review. Evidence-based initiatives exist across a range of areas - which include operational efficiency and integration, access to care, organizational structure, public health, continuing education and workforce composition - that have the potential to positively impact health care quality and health-related outcomes.