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  • Quality-adjusted Life Year Gain
  • Quality-adjusted Life Year Gain
  • Incremental Quality-adjusted Life Years
  • Incremental Quality-adjusted Life Years
  • Adjusted Life Years
  • Adjusted Life Years
  • Year Of Life
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  • New
  • Research Article
  • 10.1212/wnl.0000000000214866
Cost-Effectiveness of Thrombectomy With or Without Alteplase in Large Vessel Occlusion Stroke: A Meta-Analysis Considering Time-to-Treatment.
  • May 12, 2026
  • Neurology
  • Chi Phuong Nguyen + 24 more

In stroke patients directly admitted to thrombectomy-capable centers, the value of intravenous thrombolysis (IVT) with alteplase before thrombectomy is time dependent. While early IVT may improve outcomes, delayed IVT administration lowers the likelihood of benefit. To date, no previous cost-effectiveness study has considered onset-to-treatment time. This study evaluated the cost-effectiveness of intravenous (IV) alteplase plus thrombectomy vs thrombectomy alone in patients admitted directly to thrombectomy-capable centers across 16 countries, stratified by onset-to-IVT time. A decision tree integrated with a Markov model estimated costs, quality-adjusted life years (QALYs), and incremental net monetary benefit (INMB) over 15 years. A willingness-to-pay threshold of one gross domestic product per capita was applied for each country. Effectiveness data were derived from individual patient data from 6 trials including patients with anterior circulation large-vessel occlusion eligible for both IVT and thrombectomy who presented directly to thrombectomy-capable centers. Costs were obtained from a literature review. Onset-to-IVT time was categorized as <140, 140-169, 170-199, and ≥200 minutes. One-way sensitivity and probabilistic sensitivity analyses were performed to check robustness of results. Ninety-day functional outcome distributions from 2,268 patients (median age 71 years; 44% female) were used to model cost-effectiveness in a hypothetical cohort of 10,000 patients. Without accounting for onset-to-IVT time, IV alteplase plus thrombectomy seemed cost-effective in 13 countries (INMB: $85-$3,618; 50-65% probability of cost-effectiveness) and not cost-effective in the United States, China, and Vietnam, with modest health gains (0.06-0.08 QALYs per patient). Time-stratified analyses revealed that IVT plus thrombectomy was cost-effective in 16 countries when onset-to-IVT time was <140 minutes (INMB: $615-$30,645; 82%-98% probability) and at 140-169 minutes (INMB: $86-$16,918; 51%-77% probability). However, IV alteplase plus thrombectomy was no longer cost-effective in 8 countries at 170-199 minutes. Universally, the INMB was negative for onset-to-IVT times exceeding 200 minutes. Cost-effectiveness of IV alteplase plus thrombectomy varies per country and onset-to-IVT time. IV alteplase plus thrombectomy is cost-effective when IVT can be administered within 170 minutes from symptom onset. Cost-effectiveness of IV alteplase plus thrombectomy diminishes progressively with longer onset-to-IVT times and becomes detrimental after 200 minutes.

  • New
  • Research Article
  • 10.1245/s10434-025-18802-8
Clinical Benefit and Cost-Effectiveness of FOLFOX Versus LCCRT in Neoadjuvant Treatment for Patients with Locally Advanced Rectal Cancer: An Economic Analysis of the FOWARC and PROSPECT Trial.
  • May 1, 2026
  • Annals of surgical oncology
  • Tiantian Zhang + 12 more

FOLFOX has emerged as a new treatment paradigm for locally advanced rectal cancer (LARC). However, whether FOLFOX, which replaces radiotherapy with chemotherapy, offers comprehensive clinical and economic advantages over conventional long-course chemoradiotherapy (LCCRT), especially in high-risk patients, remains unclear. We conducted both within-trial and Markov model-based lifetime analyses to evaluate the short- and long-term economic and health outcomes of FOLFOX compared with LCCRT. Data were derived from the 3 and 10year follow-up of the FOWARC trial (NCT01211210) in China and published data from the PROSPECT trial (NCT01515787) in the USA. Main outcomes included health utility, patient-reported outcomes of adverse events, costs, and quality-adjusted life years (QALYs). Costs were evaluated from the healthcare perspective in 2023 US dollars. Subgroup, scenario, and sensitivity analyses were performed. Within-trial analysis showed that the FOLFOX group had an increasing utility trend, while LCCRT decreased. FOWARC found that LCCRT patients experienced more severe depression and social life impact compared with PROSPECT. Over 45.2 months, FOLFOX cost $3503 less and gained 0.18 more QALYs than LCCRT. Lifetime analysis estimated that FOLFOX gained 0.81 and 0.56 more QALYs, with cost savings of $12,018 and $87,643 compared with LCCRT in China and the USA, respectively. FOLFOX remained cost-effective in high-risk patients requiring supplementary radiotherapy, with LCCRT becoming preferable only when the proportion of these patients reached 64%. Selective radiotherapy avoidance with FOLFOX may be recommended for neoadjuvant treatment of LARC, even in high-risk patients. This substitution could save significant healthcare resources while improving QALYs globally. Trial Registration ClinicalTrials.gov NCT01211210, NCT01515787.

  • New
  • Research Article
  • 10.1111/bju.70218
Cost-effectiveness of percutaneous nephrostomy vs JJ stent in obstructive urolithiasis: economic evaluation alongside the STent Or NEphrostomy (STONE) randomised controlled trial.
  • May 1, 2026
  • BJU international
  • Hugo W Schuil + 27 more

To evaluate whether percutaneous nephrostomy (PCN) is cost-effective compared to JJ ureteric stent in the acute treatment of obstructive urolithiasis. A randomised controlled non-inferiority trial with 204 patients with obstructive urolithiasis requiring decompression. Participants were randomly assigned to either PCN or JJ stent, with follow-up until the stone was surgically removed or passed spontaneously. Effectiveness outcomes included time to recovery and quality-adjusted life years (QALYs). Costs were assessed from a societal perspective, incorporating healthcare, patient-related, and lost productivity costs. Weighted mean procedure costs were €685 for PCN and €792 for JJ. Mean total societal costs were €8468 for PCN and €7122 for JJ. Largest cost differences were in productivity loss and home care. Time to recovery and QALYs were similar between groups. The probability that JJ was cost-effective compared to PCN was 0.74 for quicker recovery time and 0.74 for QALYs at a willingness-to-pay threshold of €0. Some cost and quality-of-life data were self-reported, and the analysis reflects the Dutch healthcare system, which may limit generalisability. The JJ ureteric stent was found to be the likely cost-effective option compared to PCN, despite higher procedural costs. Procedural costs can be lowered by performing procedures outside of an operating theatre. Most societal costs were made during the waiting period for definitive stone removal; future research should investigate whether total cost can be lowered by reducing waiting times.

  • New
  • Research Article
  • 10.1111/dom.70593
Cost-effectiveness analysis of Dexcom ONE+ real-time continuous glucose monitoring versus self-monitoring of blood glucose in people with type 2 diabetes in Aotearoa New Zealand.
  • May 1, 2026
  • Diabetes, obesity & metabolism
  • Ryan G Paul + 4 more

Real-time continuous glucose monitors (rt-CGM) have been found effective and economical for the treatment of diabetes in many countries. The objective of this study was to provide a cost-effectiveness analysis of rt-CGM versus self-monitoring of blood glucose (SMBG) from the perspective of a healthcare payer in New Zealand. The cost-effectiveness of rt-CGM in patients with type 2 diabetes (T2D) who require intensive insulin therapy was analysed using the IQVIA Core Diabetes Model (CDM), providing outputs including life expectancy, quality-adjusted life years (QALYs), direct costs, incremental cost-effectiveness ratios (ICERs), and incidence rates of complications. A lifetime (50 years) time horizon was used with an annual discount rate of 3.5%. In the base case, rt-CGM was associated with a gain of 0.488 QALYs and incremental costs of NZD 5633 compared with SMBG, resulting in an ICER of NZD 11 533 per QALY. This corresponded to a gain of 87 QALYs per NZD 1 million invested. Scenario analyses suggested that CGM is potentially cost saving at earlier ages of rt-CGM initiation and among high-risk populations, such as Māori and Pacific Peoples, yielding higher gains in QALYs at lower total direct costs. Reductions were predicted in the risks of ophthalmic, renal, peripheral, and cardiovascular complications. This analysis provides insight into the cost-effectiveness of rt-CGM versus SMBG from the perspective of payers in New Zealand, demonstrating reductions in the risks of complications in addition to reductions in their associated costs.

  • New
  • Research Article
  • 10.1016/j.amepre.2025.108255
Increasing Colorectal Cancer Screening: A Systematic Economic Review of Patient Navigation Services.
  • May 1, 2026
  • American journal of preventive medicine
  • Jeffrey A Reynolds + 7 more

Increasing Colorectal Cancer Screening: A Systematic Economic Review of Patient Navigation Services.

  • New
  • Research Article
  • Cite Count Icon 1
  • 10.1016/j.ijrobp.2025.05.089
SABR Versus Radiofrequency Ablation and Cryoablation for Primary Renal Cell Carcinoma: An Economic Evaluation of the FASTRACK II Trial.
  • May 1, 2026
  • International journal of radiation oncology, biology, physics
  • Constanza Vargas + 16 more

SABR Versus Radiofrequency Ablation and Cryoablation for Primary Renal Cell Carcinoma: An Economic Evaluation of the FASTRACK II Trial.

  • New
  • Research Article
  • 10.1093/trstmh/trag050
Rapid diagnostic test-based algorithms for Chagas disease diagnosis: a cost-utility and budget impact analysis in Colombia.
  • Apr 24, 2026
  • Transactions of the Royal Society of Tropical Medicine and Hygiene
  • Mario J Olivera

Chagas disease remains underdiagnosed in endemic countries due to reliance on centralized laboratory-based algorithms. Rapid diagnostic tests (RDTs) may simplify diagnostic processes and expand access to decentralized testing, but uncertainty remains regarding their performance across heterogeneous settings and long-term economic value. This study evaluated the cost-utility and budget impact of RDT-based diagnostic strategies in Colombia. A cost-utility analysis was conducted from the Colombian health system perspective using a hybrid decision tree-Markov model. Three diagnostic strategies were compared: serial RDT with ELISA resolution, hybrid RDT-ELISA and the conventional ELISA algorithm with indirect immunofluorescence assay resolution. Lifetime costs and quality-adjusted life years (QALYs) were estimated. The model incorporated loss to follow-up between steps. Scenario analyses explored correlated test performance and earlier diagnosis among women of reproductive age. A 5-year budget impact analysis was conducted. Lifetime costs were US$319.27, US$266.03 and US$252.74, with corresponding QALYs of 12.30, 12.34 and 12.35 for the conventional, hybrid and serial RDT strategies, respectively. Both RDT-based strategies were economically dominant. RDT-based algorithms may improve access to diagnosis and reduce program costs. Improving completion of the diagnostic process may be a key driver of effectiveness and economic value.

  • New
  • Research Article
  • 10.1080/1750743x.2026.2660567
First-line treatments for metastatic non-small-cell lung cancer with sugemalimab plus chemotherapy: a China-based cost-effectiveness analysis.
  • Apr 24, 2026
  • Immunotherapy
  • Zixuan Zhang + 8 more

To assess the cost-effectiveness of sugemalimab plus chemotherapy as first-line treatment for metastatic Non-small cell lung cancer(NSCLC) in China. A three-state Markov model with 21-day cycles over 10 years was developed to compare sugemalimab plus chemotherapy versus placebo plus chemotherapy. Key model inputs were derived from the 4-year survival data of the GEMSTONE-302 trial and publicly available sources. Outcomes were measured in quality-adjusted life years(QALYs) and incremental cost-effectiveness ratios(ICERs). The willingness-to-pay(WTP) threshold was set at three times the per capita GDP of China in 2024(40,062.34$/QALY). Sensitivity analyses were performed to assess model robustness. Base-case ICERs were 65,766.82$/QALY for squamous and 70,117.72$/QALY for non-squamous NSCLC, both above the WTP threshold. Subgroup analyses indicated that patients with PD-L1 expression ≥50% had the most favorable ICER(60,516.14$/QALY), while those with PD-L1 < 1% had the least favorable outcome(80,599.12$/QALY). Sensitivity analyses identified drug price and utility values as the most influential parameters affecting ICERs. Sugemalimab plus platinum-based chemotherapy offers long-term clinical benefits for metastatic NSCLC but is not cost-effective at current prices in China. Improving cost-effectiveness may require targeting patients with high PD-L1 expression, adjusting reimbursement policies, and reducing drug prices.

  • New
  • Research Article
  • 10.1111/apt.70690
A Care Pathway for the Treatment of IBD Reduces Healthcare Costs and Is Cost-Effective: Results of the Multicentre IBD Value Study.
  • Apr 23, 2026
  • Alimentary pharmacology & therapeutics
  • Elyke H Visser + 12 more

An advanced therapy care pathway (ACP) for the treatment of patients with IBD can reduce practice variation and improve outcomes. This study aimed to estimate the effect of the ACP on costs and quality of life, and to assess the cost-effectiveness of the ACP. A cost-utility and cost-effectiveness analysis was conducted from a societal perspective. The ACP was implemented in six hospitals, and two hospitals in the same region served as controls. Costs and quality of life were assessed during a baseline period (December 2020-December 2021) and an intervention period after implementing the ACP (March 2022-March 2023). Quality-adjusted life years (QALYs) were derived from the EQ-5D-5L; disease control from the IBD-Control questionnaire. A difference-in-differences (DiD) analysis was conducted, and the net monetary benefit and incremental cost-effectiveness ratio (ICER) were calculated. In total, 1,173 patients were included (intervention n = 841, control n = 332). Baseline costs per patient were €23,259.96 in the intervention hospitals and €22,837.93 in the control hospitals. During the intervention period, costs decreased to €20,959.76 in intervention hospitals. This reduction was not observed in control hospitals (€22,191.21). The DiD-analysis showed cost savings of -€1933.69; QALYs (0.001) and disease control (0.15) did not change. The ICER indicated cost savings without compromising quality of life or disease control. The implementation of an ACP for the treatment of patients with IBD reduces costs, maintains quality of life and disease control, and is cost-effective. These results emphasise that the implementation of care pathways in current practices should be considered. NL-OMON21751. Website: Value-based healthcare for Inflammatory Bowel Disease: Improving (cost-) effectiveness.

  • New
  • Research Article
  • 10.1097/tp.0000000000005688
Clinical Impact and Cost-effectiveness of Everolimus Combination Therapy in Liver Transplant Recipients: Focus on the Risk of Cancer, Infection, and Renal Function.
  • Apr 23, 2026
  • Transplantation
  • Suk-Chan Jang + 4 more

Post-liver transplant complications-cancer, infections, and renal dysfunction-impose substantial clinical and economic impact on recipients. This study evaluated clinical impacts and cost-effectiveness of adding everolimus (EVR) to a calcineurin inhibitor (CNI)-based regimen in liver transplant recipients, aiming to identify how co-occurring major complications affect cost-effectiveness. We conducted a cohort analysis and a cost-utility analysis, dividing recipients into CNI with and without EVR groups. We calculated adjusted hazard ratios (HRs) for the risk of cancer and posttransplant infection with EVR combination therapy using a Cox regression model. A Markov model that captured the co-occurrence of liver disease, infection, and renal dysfunction was constructed to estimate costs and quality-adjusted life years (QALYs) and compare the incremental cost-effectiveness ratio (ICER) of adding EVR to CNI. A hypothetical cohort of 10 000 liver transplant recipients aged 55 y was simulated during 30 y. The addition of EVR significantly reduced the risk of cancer (HR 0.435; 95% confidence interval 0.279-0.678) while increasing the infection risk in subsequent years (HR 1.468; 95% confidence interval 1.053-2.045). Cost-utility analysis demonstrated the CNI with EVR strategy was cost-effective, with an ICER of US$5298/QALY, well below the $25 000/QALY threshold. In analyses quantifying how co-occurring events shift value, the ICER was $9307/QALY when limited to liver disease, decreased to $4363/QALY with renal dysfunction included, and increased to $10 464/QALY when infection was added. Overall, CNI with EVR regimen in liver transplant remained cost-effective across co-occurring outcomes and perspectives, supporting risk-benefit balancing across cancer, posttransplant infection, and renal function.

  • New
  • Research Article
  • 10.1038/s44325-026-00121-w
Cost-effectiveness of statins, berberine, and combination for primary cardiovascular disease prevention in Scotland.
  • Apr 23, 2026
  • NPJ cardiovascular health
  • Yuanqing Xia + 2 more

We aim to estimate and compare the cost-effectiveness of statins, berberine, and their combined use for primary cardiovascular disease (CVD) prevention. The Scottish CVD Policy Model was used to predict long-term health and cost outcomes in Scottish adults aged 40 years or older without pre-existing CVD. Intervention and cost inputs were sourced from published literature and health service cost data. The primary outcome measure was the lifetime incremental cost-effectiveness ratio (ICER), evaluated as cost per quality-adjusted life year (QALY) gained. Five strategies were analyzed for individuals with ASSIGN risk scores ≥20% and ≥10%: no intervention, atorvastatin 20 mg/day, berberine 1000 mg/day, simvastatin 20 mg plus berberine 1500 mg/day, and simvastatin 20 mg plus berberine 900 mg/day. All intervention strategies were cost-effective, compared to no intervention, at the threshold of ICER of £20,000 per QALY. Compared to statins, berberine was less cost-effective, but the combined interventions remained cost-effective. Notably, when using drug costs from China (reflecting lower berberine prices), berberine and the combined interventions were preferable to statins alone. Statins, berberine, and combined interventions are all cost-effective options for primary CVD prevention. Berberine could be considered a valuable alternative or complementary therapy, particularly if its price decreases below that of statins.

  • New
  • Research Article
  • 10.1227/neu.0000000000004053
Risk-Weighted Impact: Reframing Risk Analyses for Medical Decisions.
  • Apr 23, 2026
  • Neurosurgery
  • Zane Schnurman + 1 more

Developing precise metrics for clinical use is vital to translating outcomes data to practice. Risk probabilities, such as the complication rate of surgery or the lifetime risk of an aneurysm rupture, are widely used for counseling patients, but their comparison may be misleading if risks are spread over different time horizons. This study evaluates a new risk-assessment approach called risk-weighted impact (RWI) that applies event probabilities to estimate the average number of years of life impacted by event occurrence. Decision-making policies based on RWI and cumulative lifetime event risk were applied to determine management in a simplified model of incidental cerebral aneurysms through Monte Carlo simulation (1000 iterations of 10 000 synthetic patients). In addition, a web-based application was created to simplify risk-assessment calculations and comparisons. When treatment of incidental cerebral aneurysms was simulated using both risk assessment methods, there was disagreement in 25.2% (95% CI: 24.4%-26.1%) of cases, with the RWI policy preferring observation, while event-risk policy preferred intervention. In these patients, the number of poor outcomes was nearly the same, 110 (95% CI: 91-129) in RWI policy and 111 (95% CI: 90-132) in event-risk policy, but the RWI policy resulted in 874.8 fewer quality-adjusted life year lost (95% CI: 299.5-1466.6) due to adverse events occurring an average of 11.3 years later (95% CI: 8.2-14.1 years). Only using cumulative lifetime event risks may understate the impact of an up-front treatment given that a larger proportion of risk is assumed at an earlier age, when more years of life are in jeopardy. RWI offers an alternative approach to thinking about risk, using the same inputs (event probabilities and life expectancy) to compare estimated patient impact. RWI is more aligned with clinical objectives and is a valuable metric for risk assessment and decision making.

  • New
  • Research Article
  • 10.1097/js9.0000000000005080
Computer-Assisted Detection (CADe) of colonic polyps during colonoscopy reduces cancer-related healthcare costs – a single Singaporean institution cost effectiveness analysis
  • Apr 21, 2026
  • International Journal of Surgery
  • Gabrielle E Koh + 8 more

Background and aims: Colonoscopy remains as an invaluable diagnostic and therapeutic tool used for the screening and evaluation of colonic pathologies. Periodic endoscopic screening allowing for early detection and removal of pre-malignant lesions, resulting in significant overall decreased incidence and mortality from colorectal cancer. Despite its added costs, artificial intelligence (AI) has been increasingly utilized in the field of endoscopy, boasting benefits of improving adenoma detection rates while reducing endoscopists’ mental fatigue intra-procedurally. As such, further evaluation of the financial impact of such AI-usage in screening colonoscopies was performed to assess if routine use of AI-guidance in screening colonoscopies was justifiable. Methods: This study conducted a cost effectiveness analysis of the GI Genius™ CADx Intelligent Endoscopy Module (US-DG-2000309, 2021 Medtronic) system in our local Singaporean tertiary public healthcare institution. A decision tree model was used to calculate Incremental Cost Effectiveness Ratio (ICER), comparing cancer-related costs for patients who underwent conventional colonoscopy versus colonoscopy with AI guidance. Results: With a calculated ICER value of 0.72, our study suggests that AI-assisted colonoscopy was indeed cost effective. Quality-Adjusted Life Years (QALY) values derived for each subgroup of patients further demonstrated overall improved quality of life reported by patients who underwent AI-assisted colonoscopy, as compared to conventional colonoscopy. Conclusion: Our study supports the incorporation of AI guidance into routine colonoscopic evaluation in view of increased cost effectiveness and improved overall quality of life achieved. However, further studies and analysis must be undertaken before reliably determining the cost-effectiveness of AI on regional or international scales.

  • New
  • Research Article
  • 10.1080/14737167.2026.2650163
Cost-effectiveness analysis of point-of-care A1C tests in the primary care setting of Iran
  • Apr 21, 2026
  • Expert Review of Pharmacoeconomics & Outcomes Research
  • Abdoreza Mousavi + 2 more

ABSTRACT Background Given the critical role of timely monitoring in diabetes management, effective and accessible testing strategies are increasingly vital. This study evaluated the cost-effectiveness of point-of-care A1c (POC-A1c) testing compared with traditional laboratory-based A1c testing in primary care settings in Iran. Research design and methods A lifetime (40 year) Markov model was developed to estimate expected costs (valued in 2023 PPP-adjusted US dollars), quality-adjusted life years (QALYs), and incremental cost-effectiveness ratios (ICERs) for the two strategies. Deterministic and probabilistic sensitivity analyses were performed to assess uncertainty in key model parameters. Results POC-A1C was associated with an incremental cost of US$1,647.76 and a 0.15 QALY gain versus traditional laboratory testing, yielding an ICER of US$10,717.27 per QALY. Consequently, it was cost-effective at a willingness-to-pay threshold equal to Iran’s 2023 per capita GDP. Also, based on probabilistic sensitivity analysis, the probability of POC-A1C being cost-effective was 58%. Conclusions These findings suggest that integrating POC-A1c testing into primary care may represent a valuable strategy for strengthening diabetes management in Iran. While associated with higher direct costs, its potential to improve glycemic control and long-term health outcomes supports consideration by policymakers, particularly in the context of expanding access to timely diabetes monitoring.

  • New
  • Research Article
  • 10.1038/s41397-026-00411-7
Cost-effectiveness of genotype-guided acenocoumarol therapy in atrial fibrillation: a pharmacogenomic simulation study in the chilean population.
  • Apr 20, 2026
  • The pharmacogenomics journal
  • Maximiliano Mena + 5 more

Cardiovascular diseases are the leading cause of death in Chile and worldwide, representing a major public health challenge that demands urgent preventive and therapeutic strategies. In atrial fibrillation, anticoagulation is essential, and in Chile acenocoumarol rather than warfarin, used in most countries, is the standard agent. Its dosing shows substantial interindividual variability due to CYP2C9 and VKORC1 polymorphisms. We developed a cohort-based Markov model to compare standard care, genotype-guided dosing, and genotype-guided dosing adjusted for population-level adherence in 123 Chilean patients with atrial fibrillation and 123 matched simulated individuals. Outcomes were measured as quality-adjusted life years (QALYs) and direct medical costs, with cost-effectiveness assessed at a willingness-to-pay (WTP) threshold of US$17,093, estimated using the international approach of approximating the country's GDP per capita rather than a Chilean policy-based value. Genotype-guided dosing achieved the highest effectiveness (2938.34 QALYs) with an incremental cost-effectiveness ratio of US$436.86/QALY versus standard care, remaining cost-effective in sensitivity analyses up to test prices far exceeding the current US$190. The adherence-adjusted strategy was weakly dominated. These results strongly support implementing pharmacogenetic testing for acenocoumarol dosing to optimize anticoagulation safety, efficacy, and cost-effectiveness in Chile.

  • New
  • Research Article
  • 10.1186/s13561-026-00771-7
Consideration of the limitations of cost-effectiveness analysis for severe-rare disease drugs and potential solutions: a structured literature review.
  • Apr 18, 2026
  • Health economics review
  • Jia Pan + 7 more

Rare diseases affect more than 400 million people globally with only a small number having currently approved treatments. Drugs for rare diseases (medications developed to treat conditions affecting small patient populations), which may include innovative drugs (medications with novel mechanisms/new active ingredients) can face access challenges as they are often found not to be cost-effective under conventional health technology assessment methods. In this review we provide a comprehensive and up-to-date understanding of challenges associated with the economic evaluation of innovative drugs for severe-rare diseases, and any proposed solutions and associated barriers to solution adoption. This review included searches of electronic databases and select health technology assessment agency websites to identify relevant publications, guidelines or recommendations from 1st January 2014 to 29th July 2024. A pre-authorised protocol, including detailed eligibility criteria, was used to ensure robust methodology in the capture of materials. Relevant data extracted from included publications were synthesised thematically to support identification of the most prominent concepts within current literature. Of the 1,803 records identified, 31 publications and eight health technology assessment guidance were included. Main challenges were related to the utilitarian principle underlying cost-effectiveness analysis, the limited scope of the quality-adjusted life year and the value elements assessed. Associated solutions included the incorporation of a societal willingness-to-pay or inclusion of equity in healthcare decision making, reduced weighting of the economic evaluation in resource allocation, consideration of additional value elements, and introducing a quality-adjusted life year weighting or alternative measures of health outcomes. While some payers have implemented modifications to economic evaluation guidelines in an attempt to address the challenges, for example including the consideration of additional value elements, the solutions employed are not consistent across payers and often do not address the root issue. Economic evaluation as part of health technology assessment therefore remains challenging for manufacturers of drugs for rare diseases. Conventional methods of economic evaluation contribute to the access challenge for innovative drugs for rare diseases. Collaboration between payers, manufacturers, and healthcare decision makers to foster greater understanding of how to evolve and adopt these novel solutions will be key to ensuring timely patient access.

  • New
  • Research Article
  • 10.1016/j.vaccine.2026.128591
Estimating the cost-effectiveness of respiratory syncytial virus (RSV) vaccination program for older Australian adults.
  • Apr 18, 2026
  • Vaccine
  • Kishor K Paul + 7 more

Estimating the cost-effectiveness of respiratory syncytial virus (RSV) vaccination program for older Australian adults.

  • New
  • Research Article
  • 10.1136/bmjopen-2025-110008
Cost-effectiveness of osteoporotic fracture risk assessment in people with intellectual disabilities: a UK NHS modelling study.
  • Apr 17, 2026
  • BMJ open
  • May Ee Png + 7 more

We compared the cost-effectiveness of alternative fracture risk assessment strategies for people with intellectual disabilities (ID) aged ≥40 years from a UK National Health Services perspective over a lifetime horizon. Cost-effectiveness analysis using a lifetime decision-analytical model. UK primary care, with data from literature and national databases. People with ID. Three strategies were assessed: (S1) Risk assessment using the UK QFracture score; (S2) use of IDFracture (a fracture risk prediction tool specifically developed for adults with ID); and (S3) conducting a one-time dual-energy X-ray absorptiometry (DXA) scan in all. S1 and S2 were followed by DXA scan for those at risk. At-risk individuals received treatment according to UK practice (bisphosphonates plus vitamin D and calcium for osteoporosis, and vitamin D and calcium alone for osteopenia). Direct healthcare costs and quality-adjusted life years (QALYs), and incremental cost-effectiveness ratio (ICER). In the base case, S2 (ICER: -£2568/QALY) was dominant (ie, less costly and more effective) and S3 (ICER: £1678/QALY) was cost-effective relative to S1 for major osteoporotic fracture (MOF). For hip fracture, S2 (ICER: £32 116/QALY) and S3 (ICER: £49 536/QALY) were not cost-effective relative to S1 under the National Institute for Health and Care Excellence-recommended cost-effectiveness thresholds. Findings from the sensitivity analyses were predominantly consistent with the base-case results. Subgroup analyses showed that age-specific and gender-specific strategies could be used. For people with ID aged ≥40 years, a proactive approach to risk assessment for MOF is not only clinically beneficial, but also cost-effective.

  • New
  • Research Article
  • 10.3390/curroncol33040220
Cost-Effectiveness of Radiotherapy and Its Impact on Patient Quality of Life: A Real-World Cost Utility Analysis in Greece.
  • Apr 16, 2026
  • Current oncology (Toronto, Ont.)
  • Elissavet Vardaki + 3 more

The aim of this study was to estimate the economic burden of radiotherapy (RT) from the perspectives of payers, the healthcare system, patients, and society, and to assess associated quality-of-life (QoL) outcomes. The analysis examined direct medical and non-medical costs, as well as QoL, before, during, and up to six months after RT. Given the inclusion of multiple cancer types, the study reflects a heterogeneous real-world population. An exploratory comparison across RT techniques was also conducted to provide contextual economic insight. This analysis included data from 301 cancer patients undergoing RT using various techniques, including two-dimensional radiotherapy (2D), 3D conformal radiotherapy (3D-CRT), volumetric-modulated arc therapy (VMAT), and intensity-modulated radiotherapy (IMRT), at the University General Hospital of Heraklion, Crete, Greece. Clinical and cost data were collected retrospectively, while QoL data were collected prospectively using validated instruments at baseline, end of treatment, and six months post-treatment. Quality-adjusted life years (QALYs) were estimated. The primary analysis compared RT with a hypothetical "no RT" comparator derived from published evidence, while comparisons across RT techniques were conducted as exploratory analyses. Costs and QALYs were evaluated over a 6-month time horizon; therefore, discounting was not applied. Incremental cost-effectiveness ratios (ICERs) were calculated, and probabilistic sensitivity analysis was performed to account for parameter uncertainty. The cost per QALY gained with RT compared with the hypothetical "no RT" comparator varied substantially across techniques and cancer types. In the primary analysis, 2D radiotherapy yielded the lowest ICER (€13,043.27/QALY), while VMAT demonstrated an ICER of €29,945.12/QALY. In contrast, IMRT was associated with a substantially higher ICER (€135,529.51/QALY), suggesting limited cost-effectiveness under commonly accepted willingness-to-pay thresholds, whereas 3D-CRT was found to be dominant. Subgroup analyses revealed marked heterogeneity, with ICERs ranging from €3234.45 to €30,232.50 per QALY gained across cancer types. In certain subgroups, RT was either cost-saving or dominant, particularly in breast cancer (cost-saving with similar QALYs) and in skin cancer and sarcoma (dominant strategies). Sensitivity analyses highlighted considerable uncertainty, especially for 2D radiotherapy, primarily driven by small sample sizes and variability in QALY estimates. This study provides short-term, real-world evidence on the cost-effectiveness and quality-of-life outcomes of radiotherapy in a Greek healthcare setting. While simpler techniques such as 2D radiotherapy may appear economically favorable, their limited effectiveness and substantial uncertainty may reduce their overall value. In contrast, advanced techniques-particularly VMAT-demonstrate a more consistent balance between cost and clinical outcomes, supporting their role within value-based, patient-centered oncology care. However, the findings should be interpreted with caution due to population heterogeneity, small subgroup sizes, the short (6-month) time horizon, and the use of a hypothetical comparator. Further research with longer follow-up and disease-specific analyses is warranted.

  • New
  • Research Article
  • 10.1007/s41669-026-00650-1
Updated Health Economics Simulation Model of the Lifetime Progression of Multiple Sclerosis in an Australian Setting.
  • Apr 16, 2026
  • PharmacoEconomics - open
  • Andrew J Palmer + 6 more

Multiple sclerosis (MS) is a progressive disease leading to increasing disability. Our previous simulation model estimated lifetime impacts of MS on quality of life and associated economic costs but was limited by small cohort input data and simplistic structure. The aim was to improve our previous model by incorporating more granular disease states, improving the accuracy of input data using a large MS cohort, and including contemporary cost and health state utility (HSU) inputs, matching the more detailed health states. A Markov model was developed simulating MS progression from no disability to mild, moderate, and severe disability and death under contemporary treatment patterns. State-specific probabilities of progression, death, relapse, costs, HSUs, and relapse-related disutilities were considered. Life expectancy (LE), quality-adjusted life years (QALYs), and discounted lifetime total costs (2026 Australian dollars) were calculated. Probabilistic sensitivity analysis was performed. A typical MS cohort of 35-year-old females had a projected mean LE of 44.0 years, 27.3 and 11.3 undiscounted/discounted QALYs, respectively, and lifetime costs of $1,449,597. Compared with the Australian general population, those with MS had 6.8 years lower LE, 15.0 lower undiscounted QALYs, and $1.24 million higher costs. Our updated model showed higher LE and costs but lower QALYs compared with our previous model, due to differences in probability utilities and cost inputs. Our improved model, based on a larger cohort and updated inputs, provides a more nuanced assessment of the impact of MS on LE, QALYs, and costs, with improved face validity. It will be used for the cost-effectiveness analyses of current and future MS interventions.

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