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.

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.

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.

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.

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.

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.

Difficult decisions in times of constraint: criteria based resource allocation in the Vancouver Coastal Health Authority.

OBJECTIVES: The aim of the project was to develop a plan to address a forecasted deficit of approximately $4.65 million for fiscal year 2010/11 in the Vancouver Communities division of the Vancouver Coastal Health Authority. For disinvestment opportunities identified beyond the forecasted deficit, a commitment was made to consider options for resource re-allocation within the Vancouver Communities division. METHODS: A standard approach to program budgeting and marginal analysis (PBMA) was taken with a priority setting working committee and a broader advisory panel. An experienced, non-vested internal project manager worked closely with the two-member external research team throughout the process. Face to face evaluation interviews were held with 10 decision makers immediately following the process. RESULTS: The recommendations of the working committee included the implementation of 44 disinvestment initiatives with an annualized value of CAD $4.9 million, as well as consideration of possible investments if the realized savings match expectations. Overall, decision makers viewed the process favorably and the primary aim of addressing the deficit gap was met. DISCUSSION: A key challenge was the tight timeline which likely lead to less evidence informed decision making then one would hope for. Despite this, decision makers felt that better decisions were made then had the process not been in place. In the end, this project adds value in finding that PBMA can be used to cover a deficit and minimize opportunity cost through systematic application of criteria whilst ensuring process fairness through focusing on communication, transparency and decision maker engagement.

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.

Inhibition of the KCa3.1 channels by AMP-activated protein kinase in human airway epithelial cells.

The vectorial transport of ions and water across epithelial cells depends to a large extent on the coordination of the apical and basolateral ion fluxes with energy supply. In this work we provide the first evidence for a regulation by the 5'-AMP-activated protein kinase (AMPK) of the calcium-activated potassium channel KCa3.1 expressed at the basolateral membrane of a large variety of epithelial cells. Inside-out patch-clamp experiments performed on human embryonic kidney (HEK) cells stably transfected with KCa3.1 first revealed a decrease in KCa3.1 activity following the internal addition of AMP at a fixed ATP concentration. This effect was dose dependent with half inhibition at 140 muM AMP in 1 mM ATP. Evidence for an interaction between the COOH-terminal region of KCa3.1 and the gamma1-subunit of AMPK was next obtained by two-hybrid screening and pull-down experiments. Our two-hybrid analysis confirmed in addition that the amino acids extending from Asp(380) to Ala(400) in COOH-terminal were essential for the interaction AMPK-gamma1/KCa3.1. Inside-out experiments on cells coexpressing KCa3.1 with the dominant negative AMPK-gamma1-R299G mutant showed a reduced sensitivity of KCa3.1 to AMP, arguing for a functional link between KCa3.1 and the gamma1-subunit of AMPK. More importantly, coimmunoprecipitation experiments carried out on bronchial epithelial NuLi cells provided direct evidence for the formation of a KCa3.1/AMPK-gamma1 complex at endogenous AMPK and KCa3.1 expression levels. Finally, treating NuLi monolayers with the membrane permeant AMPK activator 5-aminoimidazole-4-carboxamide-1-beta-d-ribofuranoside (AICAR) caused a significant decrease of the KCa3.1-mediated short-circuit currents, an effect reversible by coincubation with the AMPK inhibitor Compound C. These observations argue for a regulation of KCa3.1 by AMPK in a functional epithelium through protein/protein interactions involving the gamma1-subunit of AMPK.

Evaluation of the impact of program budgeting and marginal analysis in Vancouver Island Health Authority.

OBJECTIVE: The objective of this research was to provide further insights into the ability of Program Budgeting and Marginal Analysis (PBMA) to help health care decision-makers in deciding where to allocate scarce resources so as to best meet their organizational objectives. METHODS: We report on a case study of PBMA implementation. The main source of information was two sets of semi-structured evaluation interviews conducted with senior decision-makers after each of the first two years of PBMA implementation in Vancouver Island Health Authority (VIHA), Canada. These interviews were analysed thematically, with initial coding based upon themes that had been identified in the previous stage of the research. RESULTS: Many of the initial problems with PBMA implementation resolved themselves over time as participants became more familiar with the process. However, some problems needed to be addressed explicitly through changes in procedures. Establishing procedures for handling 'must-dos' (i.e. spending priorities, that are externally mandated) did not replace the need to define explicitly the extent of the organization's discretionary spending authority. CONCLUSION: Faced with claims that typically outstrip available resources, health care decision-makers need a process to guide allocation decisions. PBMA has demonstrated at VIHA an ability to handle some of the key issues associated with this challenge. Our analysis has produced lessons that should facilitate future implementation but has also shown that resource allocation criteria selection and the extent of executive discretion are likely to be ongoing challenges.

How to control the costs of health care services--an inventory of strategic options.

This paper has two objectives: (1) to provide an inventory of popular strategies for cost reduction or cost containment in the health services research literature and (2) to propose a coherent framework to organize this inventory. The purpose of this framework is to inform decision-makers when grappling with the opposing forces they face in choosing a cost reduction strategy. The trade-off is clear: to access progressively more possible strategies, the decision-maker must be ready to expose the population and patients to more significant changes in services provided. On one hand, more choices are preferable because each strategy attacks the problem from a different angle and being restricted to fewer "angles" increases the likelihood that a specific "well" may have dried up. On the other hand, we know that change is often viewed, a priori, negatively in health care management, so there are pressures to limit the impact on services.

Decision maker views on priority setting in the Vancouver Island Health Authority.

BACKGROUND: Decisions regarding the allocation of available resources are a source of growing dissatisfaction for healthcare decision-makers. This dissatisfaction has led to increased interest in research on evidence-based resource allocation processes. An emerging area of interest has been the empirical analysis of the characteristics of existing and desired priority setting processes from the perspective of decision-makers. METHODS: We conducted in-depth, face-to-face interviews with 18 senior managers and medical directors with the Vancouver Island Health Authority, an integrated health care provider in British Columbia responsible for a population of approximately 730,000. Interviews were transcribed and content-analyzed, and major themes and sub-themes were identified and reported. RESULTS: Respondents identified nine key features of a desirable priority setting process: inclusion of baseline assessment, use of best evidence, clarity, consistency, clear and measurable criteria, dissemination of information, fair representation, alignment with the strategic direction and evaluation of results. Existing priority setting processes were found to be lacking on most of these desired features. In addition, respondents identified and explicated several factors that influence resource allocation, including political considerations and organizational culture and capacity. CONCLUSION: This study makes a contribution to a growing body of knowledge which provides the type of contextual evidence that is required if priority setting processes are to be used successfully by health care decision-makers.

Quality and cost in healthcare: a relationship worth examining.

Debate over healthcare often focuses on two key issues: quality and cost. However, because of the unique characteristics of healthcare, this relationship is not as simple as it might seem. A recent Supreme Court of Canada judgement directly addressing aspects of quality of care, combined with related government policy, provides an impetus for a review of research on quality as it affects costs of care. Our premise is that quality problems may not be the result of financing constraints but rather quality issues are a significant contributor to funding pressures. Theoretical and empirical evidence is reviewed and the implications for decision makers are discussed. A managerial focus on cost minimisation strategies makes implementing priority setting processes challenging because it calls for behaviours that typically counter professional self-interest. We suggest that a focus on quality would ultimately provide an effective strategy to contain costs, not to mention having a positive impact on patient well-being.

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.