Explore the vast range of titles available.

Background and objectives Motor Neuron Diseases (MND) are rare diseases but have a high impact on affected individuals and society. This study aims to perform an economic evaluation of MND in Germany. Methods Primary patient-reported data were collected including individual impairment, the use of medical and non-medical resources, and self-rated Health-Related Quality of Life (HRQoL). Annual socio-economic costs per year as well as Quality-Adjusted Life Years (QALYs) were calculated. Results 404 patients with a diagnosis of Amyotrophic Lateral Sclerosis (ALS), Spinal Muscular Atrophy (SMA) or Hereditary Spastic Paraplegia (HSP) were enrolled. Total annual costs per patient were estimated at 83,060€ in ALS, 206,856€ in SMA and 27,074€ in HSP. The main cost drivers were informal care (all MND) and disease-modifying treatments (SMA). Self-reported HRQoL was best in patients with HSP (mean EuroQoL Five Dimension Five Level (EQ-5D-5L) index value 0.67) and lowest in SMA patients (mean EQ-5D-5L index value 0.39). QALYs for patients with ALS were estimated to be 1.89 QALYs, 23.08 for patients with HSP and 14.97 for patients with SMA, respectively. Cost-utilities were estimated as follows: 138,960€/QALY for ALS, 525,033€/QALY for SMA, and 49,573€/QALY for HSP. The main predictors of the high cost of illness and low HRQoL were disease progression and loss of individual autonomy. Conclusion As loss of individual autonomy was the main cost predictor, therapeutic and supportive measures to maintain this autonomy may contribute to reducing high personal burden and also long-term costs, e.g., care dependency and absenteeism from work.

Background Amyloid positron emission tomography (PET) is instrumental in achieving an accurate diagnosis and may help to limit health‐seeking behavior. Currently, amyloid‐PET is not routinely used in clinical practice due to lack of evidence on cost‐utility. We assessed the cost‐utility of early versus no amyloid‐PET in the diagnostic work‐up of memory clinic patients after 6 months. Methods We assessed cost‐utility of patients enrolled in AMYPAD‐DPMS (EudraCT Number: 2017‐002527‐21) from six European memory clinics and randomized in ARM1; early amyloid‐PET, ARM2; no amyloid‐PET or ARM3; (amyloid‐PET at request of the managing physician). ARM3 was not part of the cost‐utility analysis. The EuroQol classification system (EQ‐5D‐5L), visual analogue scale (VAS), and ICEpop Capability measure for older people (ICECAP‐O) were collected at baseline and 6 months. Costs were calculated from cost diaries at baseline, 3 and 6 months. The incremental cost‐effectiveness ratio (ICER) was calculated using EQ‐5D‐5L and a societal perspective. Results From April 2018, to October 2020, 844 participants were screened and 840 were randomized (290 ARM1; 270 ARM2 and 280 ARM3). N = 514 (250 ARM1; 264 ARM2) were included in the economic evaluation. Amyloid‐PET resulted in higher costs at 6 months (ARM1 vs. ARM2 ∆€1384, bootstrapped 95% CI [7, 2761]). No significant difference in EQ‐5D‐5L, VAS or ICECAP‐O was found. The incremental cost‐effectiveness ratio (ICER) was €461,333 per QALY. Conclusion Although patients receive an early etiological diagnosis, the cost‐utility after 6 months is not favorable for amyloid‐PET. The cost‐utility will need to be reassessed when considering amyloid‐PET to select patients for anti‐amyloid biologics.

Background: A number of different measures can be used within cost-utility analyses, we compared results according to both the EQ-5D and SF-6D. Methods: A randomized trial was conducted to compare 4 options for people with knee pain. Over the 2 year trial period, the change in cost to health-service was estimated, and both the EQ-5D and SF-6D were used to estimate the change in quality-adjusted life years (QALYs). Using a complete case analysis, the cost-utility (incremental cost-effectiveness ratio [ICER]) of each option, according to both the EQ-5D and SF-6D, was calculated and assessed in relation to the cost-effectiveness threshold of £20,000 per QALY. Results: Of the 389 participants, 247 had complete cost, EQ-5D and SF-6D data. According to the EQ-5D, option 1 had an estimated ICER of £ 10,815 (compared with option 4), option 2 was dominated by option 1, and option 3 was subject to extended dominance. Conversely, according to the SF-6D, option 3 had an ICER of £ 9999 (compared with option 4), option 2 had an ICER of £36,883 (compared with option 3), and option 1 was subject to extended dominance. Conclusion: The EQ-5D and SF-6D estimated that different options (1 and 3, respectively) were cost- effective at the £20,000 per QALY threshold, demonstrating that the choice of measure does matter.

BACKGROUND: Spinal cord stimulation (SCS) is an effective treatment for chronic refractory pain. The evidence of pain reduction, improvement of function, quality of life, and cost-effectiveness are strong in developed countries. Nevertheless, there is still a lack of economic studies of SCS in the context of low- and middle-income countries. OBJECTIVES: To evaluate the cost-effectiveness and cost-utility of additional SCS to conventional management (CMM) in patients with chronic refractory pain in Thailand. STUDY DESIGN: Prospective observational study. SETTING: The pain clinic at Siriraj Hospital, a tertiary care center in Thailand. METHODS: This study recruited patients undergoing SCS implants due to refractory pain to CMM from varieties of conditions and followed up to 36 months. The clinical outcomes, quality of life, and costs of treatment were collected before and after SCS implantation. A decision tree and Markov model were developed to estimate the total lifetime costs and health benefits of SCS using a societal perspective. The results were presented as an incremental cost-utility ratio (ICUR) in 2021 Thai Baht (THB) per quality-adjusted life year (QALY) gained and an incremental cost-effective ratio (ICER) in 2021 THB per numeric rating pain score (NRS) reduction. RESULTS: Twenty-nine patients who underwent SCS implantation reported pain intensity and increased utility at every point in time. Compared to pre-implantation, the QALY gained in both rechargeable and non-rechargeable SCS was 2.13 QALYs. The economic analysis showed the ICUR in the rechargeable and non-rechargeable SCS + CMM group was 660,106.96, and 620,120.59 THB/QALY gained, respectively, which was higher than Thais’ willingness-to-pay (WTP) threshold at 160,000 THB/QALY gained. Pain score reduction was 2.49/10 at the 3-year point, and the ICER was 496,932.08 and 337,341.77 THB/NRS reduction for rechargeable and non-rechargeable SCS, respectively. LIMITATIONS: As this is a single-center prospective cohort study, the results might be subject to selection bias and may not truly represent all patients from a developing country. The cost-effectiveness results should also be carefully interpreted for generalizability. CONCLUSION: Spinal cord stimulation is effective in pain control and improves the quality of life for patients with chronic refractory pain. However, the ICUR of SCS is above the WTP, leading to the interpretation that SCS is still not a cost-effective treatment in the current context in Thailand. KEY WORDS: Spinal cord stimulation, effectiveness, quality-adjusted life year, incremental cost-utility ratios, cost-utility analysis, cost-effectiveness analysis, willingness to pay, developing country

Background Actinic keratoses have a high prevalence in the older Australian population, with most patients presenting with field actinic damage. Despite this high prevalence, no field therapies are subsidised under the Pharmaceutical Benefits Scheme. Objectives To determine which therapy for field actinic damage is the most cost‐effective when comparing 5‐fluorouracil (5‐FU), imiquimod (IMQ), and methyl‐aminolevulinate photodynamic therapy (MAL‐PDT) at 12 months post‐treatment. Methods A decision tree was constructed using TreeAge Pro, representing the likely clinical trajectories of patients with field actinic damage treated with 5‐FU, IMQ, and MAL‐PDT. The cost‐effectiveness analysis was performed from the patient perspective, assuming an outpatient setting. Efficacy data was derived from a single‐blinded, multi‐centre prospective randomised control trial. Cost data were derived from Australian dermatology clinics and pharmacies. One‐way and probabilistic sensitivity analyses were conducted. Results 5‐FU was the most cost‐effective treatment. It was cheaper and more effective than all other treatments, with a cost‐effectiveness ratio of AU $201 per patient achieving ≥75% clearance in field actinic damage. The cost‐effectiveness ratios of IMQ, and MAL‐PDT were AU$ 940, and AU$8058 per patient achieving ≥75% clearance respectively. Both sensitivity analyses showed certainty in 5‐FU's dominance over the other treatments. Conclusions 5‐FU is the most cost‐effective treatment option for Australian patients presenting with actinic field damage on the head area. Key points/Highlights Why was the study undertaken? There are multiple treatment modalities available for field actinic damage, but none are subsidised under the Australian Pharmaceutical Benefits Scheme. What does this study add? We conducted a modelled cost‐effectiveness analysis which demonstrated 5% 5‐fluorouracil cream to be the cheapest and most effective treatment option for field actinic damage over a twelve‐month time horizon. What are the implications of this study for disease understanding and/or clinical care? Cost‐effectiveness analyses are often used by healthcare policymakers to guide the optimal distribution of limited healthcare resources and funding. Our finding that 5‐fluorouracil cream is the cheapest and most effective treatment option for field actinic damage may be used to support funding proposals for its subsidisation and optimise patient care.

Curative therapies (CTx) to achieve durable remission of HIV disease without the need for antiretroviral therapy (ART) are currently being explored. Our objective was to model the long-term health and cost outcomes of HIV in various countries, the impact of future CTx on those outcomes and the country-specific value-based prices (VBPs) of CTx. We developed a decision-analytic model to estimate the future health economic impacts of a hypothetical CTx for HIV in countries with pre-existing access to ART (CTx+ART), compared to ART alone. We modelled populations in seven low-and-middle-income countries and five high-income countries, accounting for localized ART and other HIV-related costs, and calibrating variables for HIV epidemiology and ART uptake to reproduce historical HIV outcomes before projecting future outcomes to year 2100. Health was quantified using disability-adjusted life-years (DALYs). Base case, pessimistic and optimistic scenarios were modelled for CTx+ART and ART alone. Based on long-term outcomes and each country's estimated health opportunity cost, we calculated the country-specific VBP of CTx. The introduction of a hypothetical CTx lowered HIV prevalence and prevented future infections over time, which increased life-years, reduced the number of individuals on ART, reduced AIDS-related deaths, and ultimately led to fewer DALYs versus ART-alone. Our base case estimates for the VBP of CTx ranged from $5400 (Kenya) up to $812,300 (United States). Within each country, the VBP was driven to be greater primarily by lower ART coverage, lower HIV incidence and prevalence, and higher CTx cure probability. The VBP estimates were found to be greater in countries where HIV prevalence was higher, ART coverage was lower and the health opportunity cost was greater. Our results quantify the VBP for future curative CTx that may apply in different countries and under different circumstances. With greater CTx cure probability, durability and scale up, CTx commands a higher VBP, while improvements in ART coverage may mitigate its value. Our framework can be utilized for estimating this cost given a wide range of scenarios related to the attributes of a given CTx as well as various parameters of the HIV epidemic within a given country.

To perform a comprehensive economic evaluation of border closure for an island nation in the face of severe pandemic scenarios. The costing tool developed by the New Zealand (NZ) Treasury (CBAx) was used for the analyses. Pandemic scenarios were as per previous work;1 epidemiological data were from past New Zealand influenza pandemics. The net present value of successful border closure was NZ$7.86 billion for Scenario A (half the mortality rate of the 1918 influenza pandemic) and $144 billion for preventing a more severe pandemic (10 times the mortality of scenario A). Cost–utility analyses found border closure was relatively cost‐effective, at $14,400 per QALY gained in Scenario A, and cost‐saving for Scenario B (taking the societal perspective). This work quantifies the economic benefits and costs from border closure for New Zealand under specific assumptions in a generic but severe pandemic threat (e.g. influenza, synthetic bioweapon). Preparing for such a pandemic response seems wise for an island nation, although successful border closure may only be feasible if planned well ahead. Policy makers responsible for generic pandemic planning should explore how border closure could be implemented, including practical and legal frameworks.

Aims The DELIVER study demonstrates a significant improvement in cardiovascular death or hospitalization for heart failure among heart failure with mildly reduced ejection fraction (HFmrEF) or heart failure with preserved ejection fraction (HFpEF).Cost‐utility of the adjunct use of dapagliflozin to standard therapy among patients with HFpEF or HFmrEF remains unclear. Methods and results A five‐state Markov mode was constructed to project health and clinical outcomes of the adjunct use of dapagliflozin to standard therapy among 65‐year‐old patients with HFpEF or HFmrEF. A cost‐utility analysis was performed based on the DELIVER study and national statistical database. The cost and utility was inflated to 2022 by the usual discount rate of 5%. The primary outcomes were total cost and quality‐adjusted life‐years (QALYs) per patients as well as the incremental cost‐effectiveness ratio. Sensitivity analyses were also applied. Over a 15 year lifetime horizon, the average cost per patient was$7245.77 and $ 5407.55 in the dapagliflozin group and the standard group, along with an incremental cost of$1838.22. The average QALYs per patient was 6.00 QALYs and 5.84 QALYs in the dapagliflozin group and the standard group, along with an incremental QALYs of 0.15 QALYs, resulting in the incremental cost‐effectiveness ratio of $ 11 865.33/QALY, which was below the willingness‐to‐pay (WTP) of$12 652.5/QALY. The univariate sensitivity analysis indicated the cardiovascular death in both group was the most sensitive variable. Probability sensitivity analysis revealed that when the WTP thresholds were $ 12 652.5/QALY and$37 957.5/QALY, the probabilities of being cost‐effective with dapagliflozin as an add‐on were 54.6% and 71.6%, respectively. Conclusions From a public healthcare system perspective, the adjunct use of dapagliflozin to standard therapy among patients with HFpEF or HFmrEF generated advantages in cost‐effectiveness in China at a WTP of $ 12 652.5/QALY, which promoted the rational use of dapagliflozin for heart failure.