Outcomes of Prophylactic Peritoneal Dialysis Catheter Insertion in Children Undergoing Cardiac Surgery: A Systematic Review and Meta-Analysis.

OBJECTIVES: The objective of this Prospective Register of Systematic Reviews (CRD42022384192) registered systematic review and meta-analysis was to determine whether prophylactic peritoneal dialysis (PD) catheter insertion at the time of pediatric cardiac surgery is associated with improved short-term outcomes. DATA SOURCES: Databases search of the MEDLINE, EMBASE, CINAHL, and Cochrane Library completed in April 2021 and updated October 2023. STUDY SELECTION: Two reviewers independently completed study selection, data extraction, and bias assessment. Inclusion criteria were randomized controlled trials (RCTs) and observational studies of children (≤ 18 yr) undergoing cardiac surgery with cardiopulmonary bypass. We evaluated use of prophylactic PD catheter versus not. DATA EXTRACTION: The primary outcome was in-hospital mortality, as well as secondary short-term outcomes. Pooled random-effect meta-analysis odds ratio with 95% CI are reported. DATA SYNTHESIS: Seventeen studies met inclusion criteria, including four RCTs. The non-PD catheter group received supportive care that included diuretics and late placement of PD catheters in the ICU. Most study populations included children younger than 1 year and weight less than 10 kg. Cardiac surgery was most commonly used for arterial switch operation. In-hospital mortality was reported in 13 studies; pooled analysis showed no association between prophylactic PD catheter placement and in-hospital mortality. There were mixed results for ICU length of stay and time to negative fluid balance, with some studies showing shortened duration associated with use of prophylactic PD catheter insertion and others showing no difference. Overall, the studies had high risk for bias, mainly due to small sample size and lack of generalizability. CONCLUSIONS: In this meta-analysis, we have failed to demonstrate an association between prophylactic PD catheter insertion in children and infants undergoing cardiac surgery and reduced in-hospital mortality. Other relevant short-term outcomes, including markers of fluid overload, require further study.

Logical Inconsistencies in the Health Years in Total and Equal Value of Life-Years Gained.

OBJECTIVES: This study aimed to assess whether recently proposed alternatives to the quality-adjusted life-year (QALY), intended to address concerns about discrimination, are suitable for informing resource allocation decisions. METHODS: We consider 2 alternatives to the QALY: the health years in total (HYT), recently proposed by Basu et al, and the equal value of life-years gained (evLYG), currently used by the Institute for Clinical and Economic Review. For completeness we also consider unweighted life-years (LYs). Using a hypothetical example comparing 3 mutually exclusive treatment options, we consider how calculations are performed under each approach and whether the resulting rankings are logically consistent. We also explore some further challenges that arise from the unique properties of the HYT approach. RESULTS: The HYT and evLYG approaches can result in logical inconsistencies that do not arise under the QALY or LY approaches. HYT can violate the independence of irrelevant alternatives axiom, whereas the evLYG can produce an unstable ranking of treatment options. HYT have additional issues, including an implausible assumption that the utilities associated with health-related quality of life and LYs are "separable," and a consideration of "counterfactual" health-related quality of life for patients who are dead. CONCLUSIONS: The HYT and evLYG approaches can result in logically inconsistent decisions. We recommend that decision makers avoid these approaches and that the logical consistency of any approaches proposed in future be thoroughly explored before considering their use in practice.

A Framework for the Fair Pricing of Medicines.

As high-cost medicines put increasing pressure on public health care budgets, the need to identify 'fair' prices for medicines has never been greater. This paper proposes a framework, built upon fundamental economic principles, that allows for the consideration of 'fair' prices for medicines. The framework incorporates key considerations from conventional supply-side and demand-side approaches for specifying a cost-effectiveness 'threshold', including the health opportunity cost borne by other patients ([Formula: see text]) and society's willingness to pay for marginal improvements in population health ([Formula: see text]). The costs incurred by manufacturers in developing and supplying new medicines are also considered, as are the incentives for manufacturers to strategically price up to any common price per unit of benefit (cost-effectiveness 'threshold') specified by the payer. The framework finds that, at any 'fair' price, a medicine's dynamically calculated incremental cost-effectiveness ratio (ICER) lies below [Formula: see text]. When pricing medicines collectively, the framework finds that a common price below [Formula: see text] is required to maximize population health (consumer surplus) or to maximize total welfare (consumer and producer surplus). This framework has important policy implications for payers who wish to improve population health outcomes from constrained health care budgets. In particular, existing approaches to 'value-based pricing' should be reconsidered to ensure that patients receive a 'fair' share of the resulting economic surplus.

We need to talk about values: a proposed framework for the articulation of normative reasoning in health technology assessment.

It is acknowledged that health technology assessment (HTA) is an inherently value-based activity that makes use of normative reasoning alongside empirical evidence. But the language used to conceptualise and articulate HTA's normative aspects is demonstrably unnuanced, imprecise, and inconsistently employed, undermining transparency and preventing proper scrutiny of the rationales on which decisions are based. This paper - developed through a cross-disciplinary collaboration of 24 researchers with expertise in healthcare priority-setting - seeks to address this problem by offering a clear definition of key terms and distinguishing between the types of normative commitment invoked during HTA, thus providing a novel conceptual framework for the articulation of reasoning. Through application to a hypothetical case, it is illustrated how this framework can operate as a practical tool through which HTA practitioners and policymakers can enhance the transparency and coherence of their decision-making, while enabling others to hold them more easily to account. The framework is offered as a starting point for further discussion amongst those with a desire to enhance the legitimacy and fairness of HTA by facilitating practical public reasoning, in which decisions are made on behalf of the public, in public view, through a chain of reasoning that withstands ethical scrutiny.

Housing for vulnerable populations-identifying system cost implications: an analytic literature review.

We explored how investments in housing for vulnerable populations (including those experiencing homelessness) are described as leading to cost containment for the health, justice, and social service systems; the nature of any costs and benefits; and variations by housing type and over time. A structured search of peer-reviewed academic research focused on the core concepts of economic benefit, public housing programs, and vulnerable populations. Findings from 42 articles reporting on cost containment specific to health, justice, and social service systems at the municipal, regional, and/or state/provincial level were synthesized. Most of the studies focused on supportive housing interventions, targeted adults (mainly men) experiencing chronic homelessness in the USA, and reported results over 1-5 years. Approximately half of the articles reported on the costs required to house vulnerable populations. About half reported on funding sources, which is critical information for leadership decisions in cost containment for supportive housing. Most of the studies assessing program cost or cost-effectiveness reported a reduction in service costs and/or greater cost-effectiveness. Studies mostly reported impacts on health services, with hospital/inpatient care and emergency service use typically decreasing across the intervention types. All the studies that assessed cost impacts on the justice system reported a decrease in expenditures. Housing vulnerable populations was also found to decrease shelter service use and engagement with the foster care/welfare systems. Housing interventions may offer cost-savings in the short- and medium-term, with a limited evidence base also demonstrating long term benefit.

Prediction of odds for emergency cesarean section: A secondary analysis of the CHILD term birth cohort study.

INTRODUCTION: Previously developed cesarean section (CS) and emergency CS prediction tools use antenatal and intrapartum risk factors. We aimed to develop a predictive model for the risk of emergency CS before the onset of labour utilizing antenatal obstetric and non-obstetric factors. METHODS: We completed a secondary analysis of data collected from the CHILD Cohort Study. The analysis was limited to term (≥37 weeks), singleton pregnant women with cephalic presentation. The sample was divided into a training and validation dataset. The emergency CS prediction model was developed in the training dataset and the performance accuracy was assessed by the area under the receiver operating characteristic curve(AUC) of the receiver operating characteristic analysis (ROC). Our final model was subsequently evaluated in the validation dataset. RESULTS: The participant sample consisted of 2,836 pregnant women. Mean age of participants was 32 years, mean BMI of 25.4 kg/m2 and 39% were nulliparous. 14% had emergency CS delivery. Each year of increasing maternal age increased the odds of emergency CS by 6% (adjusted Odds Ratio (aOR 1.06,1.02-1.08). Likewise, there was a 4% increase odds of emergency CS for each unit increase in BMI (aOR 1.04,1.02-1.06). In contrast, increase in maternal height has a negative association with emergency CS. The final emergency CS delivery predictive model included six variables (hypertensive disorders of pregnancy, antenatal depression, previous vaginal delivery, age, height, BMI). The AUC for our final prediction model was 0.74 (0.72-0.77) in the training set with a similar AUC in the validation dataset (0.77; 0.71-0.82). CONCLUSION: The developed and validated emergency CS delivery prediction model can be used in counselling prospective parents around their CS risk and healthcare resource planning. Further validation of the tool is suggested.

Evaluating the Individual Healthcare Costs and Burden of Disease Associated with RSV Across Age Groups.

BACKGROUND: Respiratory syncytial virus (RSV) is a major cause of acute respiratory infection (ARI), with high morbidity and mortality worldwide. RSV costing and burden estimates can highlight the potential benefits of future vaccination programs and are essential for economic evaluations. OBJECTIVE: We aimed to determine RSV healthcare costs across age groups and the overall disease burden of medically attended RSV in Canada. METHODS: We conducted a retrospective case-control study to estimate the attributable healthcare costs per RSV case in Alberta. We used two case definitions to capture diversity in case severity: laboratory-confirmed RSV and ARI attributable to RSV. Matching occurred on five criteria: (1) age, (2) urban/rural status, (3) sex, (4) prematurity and (5) Charlson Comorbidity Index score. We calculated the age-specific burden of medically attended RSV in Canada from 2010 to 2019 by multiplying the weekly age-specific incidence of medically attended ARI with the RSV positivity rate. RESULTS: Costs per laboratory-confirmed RSV case were (in Canadian dollars [CAD], year 2020 values) $CAD12,713 and 40,028 in the first 30 and 365 days following diagnosis, respectively, whereas a case of ARI potentially attributable to RSV cost $CAD316 and 915, in 30 and 365 days, respectively. Older (aged ≥ 65 years) and younger (aged < 90 days) age groups had the highest case costs. The average medically attended RSV incidence rate across nine seasons was 1743 cases per 100,000 people per year. CONCLUSIONS: RSV is a common and expensive infection at the extremes of life, and the development of immunization programs targeting older and younger ages may be important for the reduction of RSV burden and cost.

Trends in Author-Reported Cost-Effectiveness Thresholds in the United States from 1995 to 2018: Implications for Discount Rates.

BACKGROUND: Decisions based on cost-effectiveness analyses (CEAs) using equal discount rates for health and cost outcomes are consistent with using a constant cost-effectiveness threshold over time. We sought to analyze trends in author-reported cost per quality-adjusted life-year (QALY) thresholds from CEAs published for the US setting over 24 y to retrospectively assess whether the recommended equal discount rates for costs and health were consistent with trends in the CEA literature. METHODS: We used the Tufts CEA Registry to assess whether author-reported cost-effectiveness thresholds changed in CEAs published for the US setting between 1995 and 2018 and back-calculated the implied discount rate for health based on these trends for inflation-adjusted cost-effectiveness thresholds and an annual discount rate for costs of 3%. RESULTS: We found 1995 CEAs published for the US setting and found that average nominal and inflation-adjusted cost-effectiveness thresholds increased over that time period. The discount rate for health would need to equal 2.43% to 2.48% (depending on the subset of CEAs analyzed) to be consistent with the observed trends in inflation-adjusted author-reported cost-effectiveness thresholds. We also found that restricting our analysis to currency years between 1995 and 2014 would result in a back-calculated discount rate for health of 2.99% to 3.28%. CONCLUSIONS: We found that CEA researchers have implicitly assumed that inflation-adjusted cost-effectiveness thresholds in the United States have been increasing over time (1995-2018), which is inconsistent with the recommended and prevailing choice of equal discount rates for health and cost outcomes. Our results are sensitive to the cutoff year used in the analysis. HIGHLIGHTS: We show visually and through equations that the recommended and prevailing practice of using equal discount rates for cost and health outcomes in cost-effectiveness analyses (CEAs) logically implies a constant inflation-adjusted cost-effectiveness threshold over time.Using data from the Tufts CEA Registry, we found that author-reported cost-effectiveness thresholds used in CEAs published for the US setting with currency years between 1995 and 2018 increased over time (both with and without adjustment for inflation).Assuming an annual discount rate for costs equal to 3%, the discount rate for health would need to equal approximately 2.5% to preserve consistency across decisions taken at different dates given the observed trends in inflation-adjusted author-reported cost-effectiveness thresholds.This finding depends on the cutoff year used in the analysis (data from currency years 1995-2014 would support use of equal discount rates, whereas data after 2014 would suggest a sharper trend toward increasing cost-effectiveness thresholds).

Economic Evaluation of Sucrose Octasulfate Dressing for Treatment of Diabetic Foot Ulcers in Patients with Type 2 Diabetes.

OBJECTIVES: Patients with noninfected neuroischemic diabetic foot ulcers (DFUs) treated with sucrose octasulfate (SOS) dressing have been shown to have improved healing compared with patients wearing a similar type of dressing without SOS. In this study, we aimed to estimate the cost-effectiveness of SOS dressing compared with conventional dressings from a Canadian public payer's perspective. METHODS: We built a Markov model in a hypothetical cohort of 1,000 inpatients with type 2 diabetes with DFUs. The time horizon was 5 years, and the cycle length was 3 months. We incorporated effectiveness data from the Explorer trial and cohort studies, cost data (2020 Canadian dollars) from published Canadian studies and administrative databases, and utility parameters from the Alberta's Caring for Diabetes cohort. We used probabilistic analysis to calculate the incremental cost-effectiveness ratio of SOS dressing compared with conventional dressings. RESULTS: In the comparison with conventional dressings, use of SOS dressing resulted in an expected increase of 0.16 quality-adjusted life-year (QALY) and an expected $5,878 decrease in health-care costs over 5 years. Adding SOS dressing resulted in a cost savings of $37,061 for every QALY gained. The probability that adding SOS dressing is cost-saving and cost-effective compared with conventional dressings was 89% and 86%, respectively, at a $50,000/QALY willingness-to-pay threshold. CONCLUSIONS: SOS dressing accelerates ulcer healing and helps reduce the spending induced by persistent ulcer management and amputation. Therefore, SOS dressing is likely to be cost-effective and cost-saving, which is consistent with previous health technology assessments in other health-care systems.

Quality of reporting of economic evaluations in rehabilitation research: a systematic review.

BACKGROUND: The quality of reporting of health economic evaluations for rehabilitation services has been questioned, limiting the ability to provide accurate recommendations for health decisions. PURPOSE: To document current overall reporting quality of the published literature for economic evaluations of rehabilitation services using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS), and to identify factors that could influence the quality of reporting. DATA SOURCES: Electronic literature searches were performed using MEDLINE and the NHS Economic Evaluations Database via the Cochrane Library. STUDY SELECTION: Prospective rehabilitation economic evaluation articles from 2013 to 2020 were selected. DATA EXTRACTION: Data were extracted by one reviewer and independently verified by a second reviewer. DATA SYNTHESIS/RESULTS: Title and abstracts of 3,454 papers were reviewed. 204 papers were selected for a full text screening. From those, 129 potential papers were identified to be included in this study. LIMITATIONS: Only two databases were used in data collection, and papers were selected from 2013 to 2020 only. CONCLUSIONS: Inconsistent reporting in health economic evaluations of rehabilitation services has continued, despite the availability of the CHEERS checklist. The methods of the analyzed studies were frequently under-reported, thereby creating challenges in determining whether the results reported were valid.IMPLICATIONS FOR REHABILITATIONVariable quality of reporting has been identified in rehabilitation research assessing cost-effectiveness.To grow as an area of expertise, the field of rehabilitation must produce research demonstrating its cost-effectiveness.Both rehabilitation clinicians and funders would benefit from full and transparent information to identify optimal solutions for effective and efficient care.

Small differences in EQ-5D-5L health utility scores were interpreted differently between and within respondents.

OBJECTIVES: This study aims to determine how population-based health-utility score (HUS) differences reflect individuals' health preferences using responses from the Canadian EQ-5D-5L Valuation Study, including time trade-off (TTO) and discrete-choice experiment (DCE) tasks (n = 1073). STUDY DESIGN AND SETTING: Cardinal TTO responses were transformed into pairwise comparisons to yield ordinal TTO responses. We investigated how EQ-5D-5L HUS differences differ from participants' stated cardinal preferences, and determined the smallest HUS difference that may be expected to represent participants' ordinal preferences. RESULTS: HUS differences near zero have 30.6% (95% confidence interval: 29.1-31.9%) probability of representing a tie in individuals' TTO values. Differences in EQ-5D-5L HUS of -0.054 (-0.071 to -0.029) and 0.047 (0.026-0.076) maximized the sensitivity and specificity of discriminating transitions to worse/better health states. For small HUS differences of ±0.03 to ±0.07, the magnitude of respondents' average TTO difference on the cardinal scale was 0.17 and 0.35 whether ties were included or excluded, respectively. Absolute HUS differences between 0.042 and 0.062 had a 50% probability of representing respondents' ordinal preferences. CONCLUSION: A HUS needs to be large enough to reflect individuals' stated health preferences, which may lend support to the application of a minimally important difference for decision-making.

Empirical Estimates of the Marginal Cost of Health Produced by a Healthcare System: Methodological Considerations from Country-Level Estimates.

Many health technology assessment committees have an explicit or implicit reference value (often referred to as a 'threshold') below which new health technologies or interventions are considered value for money. The basis for these reference values is unclear but one argument is that it should be based on the health opportunity costs of funding decisions. Empirical estimates of the marginal cost per unit of health produced by a healthcare system have been proposed to capture the health opportunity costs of new funding decisions. Based on a systematic search, we identified eight studies that have sought to estimate a reference value through empirical estimation of the marginal cost per unit of health produced by a healthcare system for England, Spain, Australia, The Netherlands, Sweden, South Africa and China. We review these eight studies to provide an overview of the key methodological approaches taken to estimate the marginal cost per unit of health produced by the healthcare system with the aim to help inform future estimates for additional countries. The lead author for each of these papers was invited to contribute to the current paper to ensure all the key methodological issues encountered were appropriately captured. These included consideration of the key variables required and their measurement, accounting for endogeneity of spending to health outcomes, the inclusion of lagged spending, discounting and future costs, the use of analytical weights, level of disease aggregation, expected duration of health gains, and modelling approaches to estimating mortality and morbidity effects of health spending. Subsequent research estimates for additional countries should (1) carefully consider the specific context and data available, (2) clearly and transparently report the assumptions made and include stakeholder perspectives on their appropriateness and acceptability, and (3) assess the sensitivity of the preferred central estimate to these assumptions.

Modifying the quality-adjusted life year calculation to account for meaningful change in health-related quality of life: insights from a pragmatic clinical trial.

BACKGROUND: We propose a modified quality-adjusted life year (QALY) calculation that aims to be consistent with guidance for interpreting change in patient-reported outcomes. This calculation incorporates the minimally important difference (MID) in generic preference-based health-related quality of life (HRQL) change scores to reflect what might be considered meaningful HRQL improvement/deterioration. In doing so, we review common issues in QALY calculations such as adjustment for baseline scores and standardizing for between-group differences. METHODS: Using EQ-5D-5L outcome data from the Alberta TEAMCare-Primary Care Network trial in the management of depression for patients with type 2 diabetes (n = 98), this study compared results from different QALY calculation methods to investigate the impact of (i) adjusting for baseline HRQL score, (ii) standardizing between-group differences at baseline, and (iii) adjusting for 'meaningful' HRQL changes. The following QALY calculation methods are examined: area under curve (QALY-AUC), change from baseline (QALY-CFB), regression modelling (QALY-R), and incorporating an MID for HRQL changes from baseline (QALY-MID). RESULTS: The incremental QALY-AUC estimate favoured the Collaborative Care group (0.031) while the incremental QALY-CFB (-0.028) estimate favoured Enhanced Care. Adjusting for meaningful HRQL changes resulted in a crude incremental QALY-MID of -0.023; however, after adjusting for between-group differences at baseline, QALY-R and adjusted incremental QALY-MID estimates were -0.007 and -0.001, respectively. In addition, recursive regression analyses showed that very low baseline HRQL scores impact incremental QALY estimates. CONCLUSIONS: Uncertainty in incremental QALY estimates reflects uncertainty in the value of small within-individual change as well as the impact of small differences between groups at baseline. Applying a responder-definition approach yielded crude and adjusted QALY-MID estimates that were more in favour of Collaborative Care than QALY-CFB and QALY-R estimates, respectively, suggesting that ambiguous small changes in HRQL scores have the potential to influence QALY outcomes used in economic or non-economic applications.

Noninvasive management of soft tissue disorders of the shoulder: A clinical practice guideline from the Ontario Protocol for Traffic Injury Management (OPTIMa) collaboration.

OBJECTIVES: Objective of this study is to develop an evidence-based guideline for the noninvasive management of soft tissue disorders of the shoulder (shoulder pain), excluding major pathology. METHODS: This guideline is based on high-quality evidence from seven systematic reviews. Multidisciplinary experts considered the evidence of effectiveness, safety, cost-effectiveness, societal and ethical values, and patient experiences when formulating recommendations. Target audience is clinicians; target population is adults with shoulder pain. RESULTS: When managing patients with shoulder pain, clinicians should (a) rule out major structural or other pathologies as the cause of shoulder pain and reassure patients about the benign and self-limited nature of most soft tissue shoulder pain; (b) develop a care plan in partnership with the patient; (c) for shoulder pain of any duration, consider low-level laser therapy; multimodal care (heat/cold, joint mobilization, and range of motion exercise); cervicothoracic spine manipulation and mobilization for shoulder pain when associated pain or restricted movement of the cervicothoracic spine; or thoracic spine manipulation; (d) for shoulder pain >3-month duration, consider stretching and/or strengthening exercises; laser acupuncture; or general physician care (information, advice, and pharmacological pain management if necessary); (e) for shoulder pain with calcific tendinitis on imaging, consider shock-wave therapy; (f) for shoulder pain of any duration, do not offer ultrasound; taping; interferential current therapy; diacutaneous fibrolysis; soft tissue massage; or cervicothoracic spine manipulation and mobilization as an adjunct to exercise (i.e., range of motion, strengthening and stretching exercise) for pain between the neck and the elbow at rest or during movement of the arm; (g) for shoulder pain >3-month duration, do not offer shock-wave therapy; and (h) should reassess the patient's status at each visit for worsening of symptoms or new physical, mental, or psychological symptoms, or satisfactory recovery. CONCLUSIONS: Our evidence-based guideline provides recommendations for non-invasive management of shoulder pain. The impact of the guideline in clinical practice requires further evaluation. SIGNIFICANCE: Shoulder pain of any duration can be effectively treated with laser therapy, multimodal care (i.e., heat/cold, joint mobilization, range of motion exercise), or cervicothoracic manipulation and mobilization. Shoulder pain (>3 months) can be effectively treated with exercises, laser acupuncture, or general physician care (information, advice, and pharmacological pain management if necessary).

Impact and Cost-Effectiveness of Oncotype DX for Guiding Adjuvant Chemotherapy Decisions in Early Breast Cancer.

INTRODUCTION: Oncotype DX is approved in multiple countries but its cost-effectiveness is a matter of considerable health debate. Lebanon is high-middle income country according to the World Bank classification however it is facing a mounting financial and health care burden from cancer. Therefore, we conducted a costeffectiveness analysis of Oncotype DX based Lebanese on real-life data. METHODS: We updated a Canadian cost-effectiveness model of Oncotype DX by incorporating Lebanese data. The patient population was a real-life cohort of 82 women diagnosed with hormone receptor - positive and HER2 - negative early breast cancer. RESULTS: Overall, providing Oncotype DX to only intermediate Adjuvant! Online risk patients costs an additional $83 CAD (93,883 LBP) per additional QALY. From this point, extending provision to also cover high Adjuvant! Online risk patients costs an additional $736 CAD (831,578 LBP) per additional QALY. From this point, extending provision further to also cover low Adjuvant! Online risk patients (such that Oncotype DX is provided to all patients) costs an additional $14,562 CAD (16.46m LBP) per additional QALY. Given that most women in our population-based sample were classified as intermediate Adjuvant! Online risk patients, our study focused on this subset in the second analysis. Providing Oncotype DX to intermediate Adjuvant! Online risk patients has a relatively small additional cost compared to not providing Oncotype DX, and results in a relatively large QALY gain. The incremental cost per QALY is $2,022 CAD (2.29m LBP), implying that Oncotype DX is cost-effective for intermediate Adjuvant! Online risk patients if the willingness-to-pay for a QALY is greater than 2.29m LBP. CONCLUSION: As one of the few economic evaluations to date conducted using Lebanese data, this evaluation provides information to decision makers regarding the cost-effectiveness of providing Oncotype DX to Lebanese patients.

NICE's Discounting Review: Clear Thinking on Rational Revision Meets Obstacle of Industrial Interests.

The National Institute of Health and Care Excellence (NICE) recently published a review of discounting practice and theory as part of a consultation on its current methods guidelines. The review examines the case for revision or retention of current methods. The changes considered include eliminating favourable rates in certain special cases and the reduction of the base-case rate for costs and health effects from 3.5 to 1.5%. The review also notes the potential need to reduce the cost-effectiveness threshold to accommodate a discount rate reduction, explaining that an agreement between the UK government and the pharmaceutical industry proscribes changing NICE's threshold range until the end of 2023. We believe NICE should be commended for a useful overview of the existing literature and relevant issues. We firmly endorse NICE's view that favourable discount rates are not a good way to apply a preference for certain interventions. Similarly, we support the option of reducing the discount rate to 1.5%, which better accords with real government borrowing costs. We suggest further work to clarify the appropriate theoretical basis for the NICE's social discount rate and the sensitivity of the threshold to changes in discounting. The prospects of a necessary discount rate reduction appear to depend on whether a threshold reduction can be achieved within NICE's current range or if the range itself must be revised downwards. NICE has usefully informed the debate around discount rates. Ultimately, the path to a methodologically consistent and evidence-based revision of discounting depends on whether NICE needs to adjust the threshold too and if it is free to do so.

Modifying NICE's Approach to Equity Weighting.

The UK's National Institute for Health and Care Excellence (NICE) recently launched a consultation on the methods it uses to evaluate new health technologies, and has highlighted the issue of how 'modifiers', including equity weights, should be incorporated into its processes. The practice of applying equity weights to specific population subgroups, as a means for increasing the effective cost-effectiveness threshold for some new health technologies, is well established in health technology assessment. It is also the subject of extensive discussion in the academic literature. In this paper, we demonstrate that NICE's current approach to equity weighting has the effect of reducing both population health and equity-weighted population health, a fundamental problem that appears to place NICE in contravention of its principles and obligations. We consider two potential methods for modifying NICE's current approach to address this problem. We also consider the merits of NICE abandoning its current approach to equity weighting and adopting a standard 'net benefit' approach in its place. We find that adopting a standard 'net benefit' approach is the most desirable option, as it provides for the most transparency while avoiding specific issues that arise when attempting to modify NICE's current approach. Regardless of the approach NICE uses for equity weighting, we find that protecting the health of National Health Service patients requires that some new technologies be evaluated using an effective cost-effectiveness threshold lower than the 'supply-side' cost-effectiveness threshold. This poses a particular challenge for NICE, given its obligations under the 2019 'Voluntary Scheme' between the UK pharmaceutical industry, the National Health Service, and the UK Government. We conclude by making some recommendations as to how NICE can move forward with the use of 'modifiers' in its decision making.