ABSTRACT Malaria is a significant challenge in sub-Saharan Africa, especially in Cameroon, where children under 5 years of age are disproportionately affected. The WHO recommends seasonal malaria chemoprevention (SMC) for children aged 3-59 months in areas of high seasonal transmission. Conducted in northern Cameroon from December 2021 to June 2022, this cross-sectional study aimed to assess SMC acceptability among parents of children under 5. Structured questionnaires collected data from 58 households on sociodemographic, malaria and SMC knowledge, insecticide-treated net (ITN) utilization, child malaria positivity, and SMC acceptability. Logistic regression identified predictors of SMC acceptability. Respondents from 58 households, mostly with secondary education, showed high awareness of malaria and SMC and good preventive knowledge. ITN utilization varied. A total of 68.97% of the children tested for malaria were negative. Overall, 67.24% accepted SMC, with education and SMC knowledge as significant predictors. This study highlights the acceptability of SMC among parents of children under 5 in northern Cameroon. Achieving full SMC coverage and adherence remains challenging despite widespread awareness. Considering factors such as education and SMC knowledge, targeted interventions are crucial for reducing the malaria burden in this vulnerable population. Keywords: Malaria, Seasonal Malaria chemoprevention, Cameroon, Children under 5. Acceptability.

Vaccination has resulted in substantial public health benefits for human populations worldwide since it was first introduced more than a century ago. This article presents an overview of the history of vaccine development, its implementation, and price setting, the latter mainly from a developed world perspective. It considers potential issues and challenges. Over time, vaccine development and production has evolved to a market-driven approach, conducted largely by private commercial entities. The complex processes of identifying potential vaccine targets and developing and producing vaccines at scale have now become more efficient. However, vaccine pricing is an emerging concern. The elements that maximize the overall health benefit of vaccination include high volume, high coverage, and rapid initial implementation to achieve the high coverage with the vaccine as quickly as possible. It therefore requires substantial initial investment. Consequently, the price set for the vaccine should be reasonable to avoid limiting the coverage given the available budget. Suboptimal coverage leads to suboptimal benefit if herd protection is not fully achieved. This may disappoint health authorities and may result in program discontinuation. Conventional cost-effectiveness analysis is therefore not ideally suited to vaccine price setting, as it is based on the concept of ‘more for more’, i.e., higher health gain achieved at a higher reimbursement cost that does not account for limited budgets. Constrained optimization (CO) combines value assessment with constrained budget allocation into one analysis method and may therefore be the better option for vaccine pricing.

Purpose To evaluate the cost-effectiveness of regional citrate anticoagulation (RCA) or systemic heparin anticoagulation (SHA) during continuous renal replacement therapy (CRRT) in critically ill patients. Methods A decision-analytic model was developed to calculate an incremental cost-effectiveness ratio between RCA and SHA from a US healthcare payer perspective. Key differentiator was the risk of bleeding derived from the RCA and SHA groups in the RICH trial. Base case didn't consider the potential impact of bleeding on length of stay (LOS) or mortality. Three scenarios were considered: 1-impact of bleeding on LOS; 2-impact of bleeding on mortality; and 3-impact of bleeding on both LOS and mortality. Results Base case: RCA was associated with an incremental cost of +$577 compared with SHA. Scenario 1: RCA resulted in cost savings of -$311. Scenario 2: RCA incurred incremental costs of +$614 for +0.076 incremental QALYs (+$8131/QALY). Scenario 3: RCA appeared to be a dominant strategy over SHA. Sensitivity analyses showed the results were robust to parameter uncertainties. Conclusion RCA is an economically attractive alternative to SHA, especially when bleeding leads to longer hospital stays. Observational research is warranted to document the impact of bleeding on LOS to confirm the economic value of RCA over SHA.

Anti-PD-1 agents, inhibitors of programmed cell death protein 1 (PD-1), significantly improve clinical outcomes and overall survival for individuals with several metastatic and early-stage cancers. This study evaluates the health impact of using anti-PD-1 agents for early-stage disease (ESD) treatment of melanoma (stage IIB–C and III), renal cell carcinoma (RCC), and triple-negative breast cancer (TNBC) in Belgium (2023–2032). Belgian individuals eligible for ESD treatment (target population) entered a Markov-based health outcomes model in a recurrence/event/disease-free state. The model compared anti-PD-1 agents only for metastatic disease treatment (reference scenario) versus anti-PD-1 agents for ESD treatment (ESD scenario) from 2023 to 2032. Clinical outcomes of the model included recurrence/event/disease-free life-years (LYs), total LYs, quality-adjusted LYs (QALYs), recurrences/events, active treatments for metastatic disease, and total deaths. The cumulative health impact of ESD anti-PD-1 treatment in Belgium was calculated as the difference in health outcomes between the ESD and reference scenarios for the time horizon. Of the 14,306 eligible individuals, 11,065 were predicted to initiate treatment with anti-PD-1 agents for ESD. Anti-PD-1 therapies for ESD increased recurrence/event/disease-free LYs (+13.4

INTRODUCTION:Malaria remains a major public health threat in sub-Saharan Africa. In Cameroon, where malaria is endemic, pregnant women are especially vulnerable due to reduced immunity and placental sequestration of infected erythrocytes. Intermittent preventive treatment in pregnancy (IPTp) with sulfadoxine-pyrimethamine (SP) is recommended by the World Health Organization (WHO) to prevent malaria-related complications. However, national coverage remains below the 80% target. This study aimed to assess IPTp uptake and its associated factors among pregnant women in five diverse regions of Cameroon. METHODS:A cross-sectional study was conducted between 2020 and 2022 in five health areas, using two-stage cluster sampling. A total of 259 pregnant women aged 15-49 were interviewed using a structured questionnaire. Data collected included sociodemographic characteristics, antenatal care (ANC) attendance, knowledge of malaria and IPTp, and prevention practices. Malaria infection was assessed using rapid diagnostic tests (RDTs). Logistic regression analysis was used to identify factors associated with the uptake of at least three doses of IPTp. RESULTS:Among participants, 62.55% (CI95%: 56.31% to 68.40%) had received at least three doses of IPTp. Age was a significant predictor: women aged between 25 and 30 years had higher odds of optimal uptake (adjusted odds ratio (aOR): 4.87; 95% CI: 2.15-11.51), and those over 30 had even greater odds (aOR: 7.93; 95% CI: 3.08-21.71) compared to those aged between 21 and 25 years. Marital status also influenced uptake: married women (aOR: 9.52; 95% CI: 4.29-23.27) and cohabiting women (aOR: 2.85; 95% CI: 1.50-5.56) were more likely to receive three doses than single women. Parity was associated with higher adherence, with primiparous (aOR: 8.25; 95% CI: 3.02-26.69) and multiparous women (aOR: 7.26; 95% CI: 2.17-27.74) more likely to complete IPTp than nulliparous women. ANC attendance was a key determinant: women with more than two visits were significantly more likely to complete IPTp (aOR: 2.41; 95% CI: 1.17-4.96). Good malaria knowledge (aOR: 1.99; 95% CI: 1.12-3.58) and good IPTp knowledge (aOR: 2.19; 95% CI: 1.10-4.38) were also positively associated. Testing positive for malaria was associated with lower IPTp adherence (aOR: 0.53; 95% CI: 0.31-0.92). CONCLUSION:Despite improvements compared to national statistics, IPTp uptake remains below WHO recommendations. Key factors influencing adherence include age, marital status, parity, ANC frequency, and knowledge levels. Higher IPTp uptake was associated with lower malaria prevalence, which suggests but does not confirm a protective effect given the cross-sectional design. Public health strategies should focus on promoting early and regular ANC attendance, improving education on malaria prevention, and encouraging male involvement to boost IPTp coverage and improve maternal outcomes.

BACKGROUND:Malaria remains a significant public health challenge in Cameroon, particularly affecting children under 5 years of age. Despite these efforts, its prevalence persists, highlighting the need for comprehensive epidemiological studies to guide interventions. METHODS:A cross-sectional study was conducted in five randomly selected health areas across five regions of Cameroon. Data on sociodemographic profiles, insecticide-treated net utilization, and malaria incidence among children under 5 years of age were collected using structured questionnaires and rapid diagnostic tests. Statistical analysis was performed to identify factors associated with malaria positivity. RESULTS:The study included 1,200 households with children under 5 years of age, representing various sociodemographic profiles across regions. Among the respondents, 85% demonstrated a high awareness of malaria. While 92% reported ownership of insecticide-treated nets, only 67% reported consistent utilization. Alarmingly, 42% of children under 5 years of age tested positive for malaria. Factors associated with malaria positivity included the gender of the household head, marital status, insecticide-treated net availability, physical condition of insecticide-treated nets, and recent malaria episodes. CONCLUSION:While the study provided valuable insights, limitations such as its cross-sectional design and potential biases necessitate caution in interpreting the results. To address these issues, rigorous data collection methods and statistical analysis were employed, emphasizing the importance of targeted interventions and ongoing surveillance to combat malaria effectively.

New vaccination programs measure economic success through cost-effectiveness analysis (CEA) based on an outcome evaluated over a certain time frame. The reimbursement price of the newly approved vaccine is then often reliant on a simulated ideal effect projection because of limited long-term data availability. This optimal cost-effectiveness result is later rarely adjusted to the observed effect measurements, barring instances of market competition-induced price erosion through the tender process. However, comprehensive and systematic monitoring of the vaccine effect (VE) for the evaluation of the real long-term economic success of vaccination is critical. It informs expectations about vaccine performance with success timelines for the investment. Here, an example is provided by a 15-year assessment of the rotavirus vaccination program in Belgium (RotaBIS study spanning 2005 to 2019 across 11 hospitals). The vaccination program started in late 2006 and yielded sub-optimal outcomes. Long-term VE surveillance data provided insights into the infection dynamics, disease progression, and vaccine performance. The presented analysis introduces novel conceptual frameworks and methodologies about the long-term economic success of vaccination programs. The CEA evaluates the initial target vaccination population, considering vaccine effectiveness compared with a historical unvaccinated group. Cost-impact analysis (CIA) covers a longer period and considers the whole vaccinated and unvaccinated population in which the vaccine has direct and indirect effects. The economic success index ratio of CIA over CEA outcomes evaluates long-term vaccination performance. Good performance is close to the optimal result, with an index value ≤1, combined with a low CEA. This measurement is a valuable aid for new vaccine introductions. It supports the establishment of robust monitoring protocols over time.

Background: Malaria remains a significant public health concern globally, particularly in the WHO African Region, where Cameroon is among the countries bearing a high burden of the disease. In Cameroon, malaria is highly endemic, with millions of cases and thousands of deaths recorded annually. Insecticide-treated mosquito nets (ITNs) are a crucial preventive measure against malaria, yet their ownership, utilization, and physical condition in Cameroon require evaluation. Methods: A cross-sectional study was conducted in five regions of Cameroon, and data were collected through semiopen questionnaires from November 2020 to June 2022. The study assessed sociodemographic characteristics, ITN ownership and usage, and the physical integrity of ITNs. The proportionate hole index (pHI) was calculated to evaluate the ITN conditions. Malaria incidence was determined using rapid diagnostic tests (RDTs), and logistic regression analysis was performed to identify factors associated with ITN utilization. Results: Among the 1719 participants, the sex distribution was balanced, with the majority aged 31 to 40 years. Awareness of malaria was high, with 100% familiarity with the disease. However, only 28.8% mentioned the use of ITN for prevention. The ITN possession rate was 66.55%, with 82% acquired through government-led campaigns. Only 65.91% of the ITN owners slept under one the previous night. Reasons for nonusage included heat (71.02%) and suffocation (24.90%). Physical integrity assessment revealed that only 34.97% of the ITNs were in good condition, emphasizing the need for proper maintenance. The malaria incidencewas 25.54%, with a significant association between ITN ownership and lower malaria positivity. Factors influencing ITN usage included region, sex, number of ITNs, pHI, and recent malaria experience. Conclusion: This study underscores the importance of addressing barriers to consistent ITN usage and maintaining physical integrity. Health education programs should emphasize ITN effectiveness and proper care, particularly targeting regions with lower utilization rates. Additionally, interventions should consider sex, household characteristics, and recent malaria episodes when promoting ITN usage. By addressing these factors, Cameroon can enhance overall ITN utilization and contribute to reducing the burden of malaria on vulnerable populations.

BackgroundThe theory of planned behavior (TPB) postulates that behavioral performance is guided by the intention to perform that behavior, influenced by attitudes, subjective norms, and perceived behavioral control. This framework can be applied to studying interprofessional collaboration among healthcare professionals to enhance patient safety and public health within nursing homes.ObjectivesThis study aimed to explore the roles of physicians, pharmacists, and nurses in the interprofessional collaboration process while identifying facilitators and barriers to effective collaboration among healthcare professionals.MethodsA qualitative interpretative phenomenological analysis (IPA) was carried out. Individual semi-structured interviews were conducted with 19 healthcare professionals. Qualitative data were then integrated and analyzed through the lens of the TPB.FindingsThe IPA revealed the ten following themes, considered as both facilitators and barriers to interprofessional collaboration among healthcare professionals in the nursing home setting: communication, roles and responsibilities, willingness and recognition of collaboration's importance, mutual knowledge, trust, confidence, support from decision-makers, protocols, and technology were considered as facilitators while distance was considered as a barrier.ConclusionEnhancing pharmacist-physician collaboration and refining pharmacist-nurse collaboration were essential goals. Intention for collaboration was influenced by attitudes (such as communication and mutual understanding), subjective norms (including support from decision-makers), and perceived behavioral control (such as confidence and adherence to protocols and technology). Addressing these factors could improve collaboration, enhancing residents' quality of life and professionals' sense of achievement.

BACKGROUND:As the societal value of vaccines is increasingly recognized, there is a need to examine methodological approaches that could be used to integrate these various benefits in the economic evaluation of a vaccine. RESEARCH DESIGN AND METHODS:A literature review and two expert panel meetings explored methodologies to value herd immunity, health spillover effects (beyond herd immunity), impact on antimicrobial resistance, productivity, and equity implications of vaccines. RESULTS:The consideration of broader benefits of vaccines in economic evaluation is complicated and necessitates technical expertise. Whereas methodologies to account for herd immunity and work productivity are relatively well established, approaches to investigate equity implications are developing and less frequently applied. Modeling the potential impact on antimicrobial resistance not only depends on the multi-faceted causal relationship between vaccination and resistance but also on data availability. CONCLUSIONS:Different methods are available to value the broad impact of vaccines, and it is important that analysts are aware of their strengths and limitations and justify their choice of method. In the future, we expect that an increasing number of economic evaluations will consider the broader benefits of vaccines as part of their base-case analysis or in sensitivity analyses.

OBJECTIVES: Acute kidney injury (AKI) and fluid overload (FO) are among the top reasons to initiate intermittent hemodialysis (IHD) or continuous renal replacement therapy (CRRT). Prior research suggests CRRT provides more precise volume control, but whether CRRT is cost-effective remains unclear. We assessed the cost-effectiveness of CRRT for volume control compared with IHD from a U.S. healthcare payer perspective. DESIGN: Decision analytical model comparing health outcomes and healthcare costs of CRRT versus IHD initiation for AKI patients with FO. The model had an inpatient phase (over 90-d) followed by post-discharge phase (over lifetime). The 90-day phase had three health states: FO, fluid control, and death. After 90 days, surviving patients entered the lifetime phase with four health states: dialysis independent (DI), dialysis dependent (DD), renal transplantation, and death. Model parameters were informed by current literature. Sensitivity analyses were performed to evaluate results robustness to parametric uncertainty. SETTING: ICU. PATIENTS OR SUBJECTS: AKI patients with FO. INTERVENTIONS: IHD or CRRT. MEASUREMENTS AND MAIN RESULTS: The 90-day horizon revealed better outcomes for patients initiated on CRRT (survival: CRRT 59.2% vs IHD 57.5% and DD rate among survivors: CRRT 5.5% vs IHD 6.9%). Healthcare cost was 2.7% (+$2,836) higher for CRRT. Over lifetime, initial CRRT was associated with +0.313 life years (LYs) and +0.187 quality-adjusted life years (QALYs) compared with initial IHD. Even though important savings were observed for initial CRRT with a lower rate of DD among survivors (–$13,437), it did not fully offset the incremental cost of CRRT (+$1,956) and DI survival (+$12,830). The incremental cost-per-QALY gained with CRRT over IRRT was +$10,429/QALY. Results were robust to sensitivity analyses. CONCLUSIONS: Our analysis provides an economic rationale for CRRT as the initial modality of choice in AKI patients with FO who require renal replacement therapy. Our finding needs to be confirmed in future research.

Background All European countries have national immunization programs (NIPs) to protect gainst infectious diseases. We aimed to estimate the individual lifetime cost of vaccination in 23 European countries, assuming full compliance with NIP schedules. Research design and methods We used publicly available data to estimate the individual lifetime cost of vaccination with the vaccines that are currently recommended and funded in each country for healthy individuals and for individuals with underlying medical conditions. We included a scenario analysis for healthy individuals in which all currently recommended vaccines were universally funded, and compared the annual costs per person of vaccination to the annual per-capita costs of all-cause hospitalization and anti-infective medications. Results The individual lifetime cost of vaccination was €592–3,504 for healthy individuals (median: €1,663; 13–20 diseases), €744–9,081 for individuals with underlying conditions (median: €2,992; 13–21 diseases), and €1,225–4,832 (median: €2,565; 21–22 diseases) in the scenario analysis, with median values for vaccine acquisition of €1,203, €1,731, and €1,788, respectively. Conclusions Our estimates show that the maximum potential cost of vaccination requires a relatively low level of investment assuming full compliance. These data could be useful for policymakers in future financial planning and evaluation of NIPs.

Objective We evaluated the public health impact and return on investment of Belgium’s pediatric immunization program (PIP) from both healthcare-sector and societal perspectives. Methods We developed a decision analytic model for 6 vaccines routinely administered in Belgium for children aged 0–10 years: DTaP-IPV-HepB-Hib, DTaP-IPV, MMR, PCV, rotavirus, and meningococcal type C. We used separate decision trees to model each of the 11 vaccine-preventable pathogens: diphtheria, tetanus, pertussis, poliomyelitis, Haemophilus influenzae type b, measles, mumps, rubella, Streptococcus pneumoniae , rotavirus, and meningococcal type C; hepatitis B was excluded because of surveillance limitations. The 2018 birth cohort was followed over its lifetime. The model projected and compared health outcomes and costs with and without immunization (based on vaccine-era and pre–vaccine era disease incidence estimates, respectively), assuming that observed reductions in disease incidence were fully attributable to vaccination. For the societal perspective, the model included productivity loss costs associated with immunization and disease in addition to direct medical costs. The model estimated discounted cases averted, disease-related deaths averted, life-years gained, quality-adjusted life-years gained, costs (2020 euros), and an overall benefit–cost ratio. Scenario analyses considered alternate assumptions for key model inputs. Results Across all 11 pathogens, we estimated that the PIP prevented 226,000 cases of infections and 200 deaths, as well as the loss of 7,000 life-years and 8,000 quality-adjusted life-years over the lifetime of a birth cohort of 118,000 children. The PIP was associated with discounted vaccination costs of €91 million from the healthcare-sector perspective and €122 million from the societal perspective. However, vaccination costs were more than fully offset by disease-related costs averted, with the latter amounting to a discounted €126 million and €390 million from the healthcare-sector and societal perspectives, respectively. As a result, pediatric immunization was associated with overall discounted savings of €35 million and €268 million from the healthcare-sector and societal perspectives, respectively; every €1 invested in childhood immunization resulted in approximately €1.4 in disease-related cost savings to the health system and €3.2 in cost savings from a societal perspective for Belgium’s PIP. Estimates of the value of the PIP were most sensitive to changes in input assumptions for disease incidence, productivity losses due to disease-related mortality, and direct medical disease costs. Conclusion Belgium’s PIP, which previously had not been systematically assessed, provides large-scale prevention of disease-related morbidity and premature mortality, and is associated with net savings to health system and society. Continued investment in the PIP is warranted to sustain its positive public health and financial impact.

At least since the Age of Enlightenment, good health has been a tenet for society. Healthy societies could learn better, work harder, improve their wealth, and live longer. Today societies focus on life expectancy, as we value long and healthy lives. As illustrated by the provision of COVID-19 vaccines first for the elderly, societies value life-saving actions. Paradoxically, health economic assessments conventionally devalue long-lasting health through the practice of discounting health benefits along with costs. However, health, with its intrinsic and instrumental characteristics, is not synonymous with money cash, a tradeable asset that devalues with time. If improving healthy life expectancy is a societal ambition, it seems counter-intuitive to value future health less as a result of an artificial mathematical construct when evaluating economically new medical interventions. In this paper, we investigate the application of discounting health in healthcare and consider paradoxical findings, especially in relation to disease prevention with vaccination. We argue that there is no economically sustainable argument to discount health gains, except for the benefit of the payer with a goal of spending less on life-saving products. If that is the objective for discounting health, there are other means to achieve the same goal in a more transparent and simpler way. From the long-term perspective of healthcare development, not discounting health gains would encourage research that values long-term effects. This in turn has the potential to benefit the investor, the payer, and the patient/consumer, improving the situation from multiple perspectives.

Sickle cell disease (SCD) is an inherited blood disorder in which sickle hemoglobin (HbS) polymerizes, leading to red blood cell sickling and chronic hemolytic anemia, vaso-occlusive crises, and end-organ damage associated with early mortality. Despite standard of care, patients with SCD still experience complications and early mortality, highlighting remaining unmet treatment needs. Voxelotor is a first-in-class HbS polymerization inhibitor approved by the US Food and Drug Administration as a treatment for SCD and by the European Medicines Agency for hemolytic anemia due to SCD. In clinical studies, voxelotor has been shown to increase hemoglobin (Hb) and decrease hemolytic markers in patients with SCD. The objective of this study was to estimate the impact of voxelotor on the burden of SCD in France using a modeling approach, accounting for its anticipated adoption and diffusion over the next 5 years. We designed a sequential multi-cohort model to project and compare the cumulative incidence of SCD complications over a 20-year time horizon in a world with and without voxelotor. A distribution of patients was simulated across various levels of Hb response based on the phase 3 HOPE trial results, and relative risk reduction was adjusted using published meta-analysis results that projected risk reduction due to a 1 g/dL increase in Hb. In 6100 modeled patients with SCD treated with voxelotor, the model projected the number of deaths to decrease by 39.4%, with an increase of 1.8% in life-years gained. The model also projected life expectancy to increase by 15.8%, and incident cases of stroke, pulmonary hypertension, and chronic kidney disease to decrease by 19.8%, 24.5%, and 25.1%, respectively. The model suggests that improving Hb using a treatment such as voxelotor may have a positive public health impact by reducing the burden of SCD for patients and the healthcare system.

Achieving glycated hemoglobin (HbA1c) <7% remains one of the main treatment Objectives: for type 2 diabetes (T2D). While metformin monotherapy is the preferred first-line option, patients frequently receive add-on sulfonylurea (SU) or dipeptidyl-peptidase-4 inhibitor (DPP4i). We balanced the cost-consequences of gliclazide-modified released (MR) versus sitagliptin (among the most used SU and DPP4i respectively), as add-on second-line treatment. A cost-comparison model hypothetically sized at 10,000 T2D patients with HbA1c ≥7% while on metformin compared gliclazide-MR or sitagliptin as add-on second-line. HbA1c reductions were informed by a real-world study comparing 993 patients newly treated with gliclazide-MR with 933 matched patients newly treated with sitagliptin, and whose records were extracted from the UK Clinical Practice Research Datalink (CPRD). HbA1c-dependent risks of myocardial infraction (MI) were taken from a large prospective cohort study of the UK Biobank in which 7,316 MI events were documented from 471,399 individuals over a mean follow-up of 8.9 years. Daily drug costs were computed using defined daily doses and unit prices (€). Model parameters were varied deterministically. Within a year of treatment, 3,780 patients treated with gliclazide-MR reached the HbA1c <7% target as compared to 2,800 patients treated with sitagliptin (+ 980 patients), avoiding 2 additional MI event (16 MI avoided with gliclazide-MR versus 14 MI avoided with sitagliptin). Gliclazide-MR acquisition cost totaled €803,000 as compared to €11,753,000 for sitagliptin acquisition (- €4,781,500). This translated into drug cost savings per additional patient reaching the HbA1c <7% target of €10,950,000 per patient for gliclazide-MR versus sitagliptin. The results were robust to the sensitivity analysis. HbA1c <7% target can be achieved earlier at a much lower cost with second line gliclazide-MR as compared to sitagliptin in T2D patients with HbA1c ≥7% while on metformin treatment. Cost-saving could even be greater if the longer-term avoidance of MI is to be accounted in.

Recent evidence suggests that single-pill combination (SPC) could decrease blood pressure to a greater extent than free-equivalent combination (FEC), notably by virtue of better adherence. We examined to what extent this greater blood pressure reduction is cost-effective within the Italian healthcare setting. A Markov model was designed to project over a lifetime horizon the number of Major Cardiovascular Events (MaCEs), Life Years (LYs), Quality-adjusted Life Years (QALYs) and the direct medical costs. The incidence of MaCEs was projected using the fitted linear relationship between Systolic BP (SBP) reduction and risk of MaCEs from the meta-regression reported by the Blood Pressure Lowering Treatment Trialists' Collaboration. SBP reductions achieved by each treatment strategy were informed by a random effect meta-analysis. Italian healthcare costs were informed from literature. For treatment acquisition costs, we used the price of Triplixam®, a triple single-pill combination of perindopril, indapamide and amlodipine. We assumed price parity of FEC in the base case analysis. Considering a cohort of 1,000 patients projected over their lifetime, the SPC avoided 33 MaCEs (- 7.0%) and 2 CV death as compared to its FEC. This led to +0.084 LY and +0.062 QALY gained for SPC, respectively. These health benefits were associated with cost savings. SPC saved €655 of direct medical cost per patient over lifetime because of the lowered incidence of MaCEs. Hence, cost-effectiveness analysis revealed dominance of SPC versus FEC. The incremental net monetary benefit of SPC over FEC was +€1,896 (for a WTP of €20,000/QALY). The results were robust to sensitivity analysis. SPC is cost-effective versus its FEC in the management of hypertension and prevention of MaCEs and CV deaths.

BackgroundIn childhood autism spectrum disorder (ASD), the Childhood Autism Rating Scale–2nd edition (CARS2) is a condition-specific instrument to be filled out by clinicians, resulting in a score for diagnosis and severity. Our aim is to estimate a preference-based scoring of CARS2 to better understand the value of changes in the CARS2 score. Using caregivers and clinicians as proxies for autistic children, we assessed the feasibility of establishing preferences for CARS2-based attributes.MethodsThe 15 CARS2 items were assessed regarding their relevance as attributes and the appropriateness of their wording. Best-worst scaling (BWS) and discrete choice experiment (DCE) choice task designs were developed, as well as a mapping task between CARS2 and the EQ-5D-Y. Individual qualitative interviews with caregivers, clinicians, and autistic adults were conducted (N=10). A committee of experts advised on the study, including caregivers, clinicians, and specialists in CARS2, health technology assessment, and preference research methodology. ResultsThirteen of the 15 CARS2 items were deemed appropriate attributes for the preference study. Caregivers and autistic adults identified with the attributes and perceived them as comprehensive. The attribute definitions and level descriptions were shortened and refined to ensure comprehension for non-experts. The choice tasks were challenging for participants; however, 9/10 respondents could make a choice. Interviewees favored a two-profile DCE over a three-profile BWS design. In a subsequent internal pilot (N=13), participants favored a stacked layout, in which attributes with overlapping levels were clustered. The CARS2/EQ-5D-Y mapping task was feasible using step-by-step instructions. ConclusionA preference study in childhood ASD based on the CARS2 instrument is feasible with caregivers and clinicians as proxies for children’s preferences.