PharmacoEconomics Open最新文献

{"title":"Cost-Effectiveness Analysis of Sequential Treatment Strategies for Moderate to Severe Crohn's Disease in Spain: Where Should Biosimilar Ustekinumab Be Positioned?","authors":"Iago Rodríguez-Lago, José Ignacio Peinado-Fabregat, Marcelo Guigini, Mónica Cerezales, Francisco Ibáñez, Carlos Crespo, Manuel Barreiro-de Acosta","doi":"10.1007/s41669-026-00642-1","DOIUrl":"10.1007/s41669-026-00642-1","url":null,"abstract":"<p><strong>Background/objective: </strong>Crohn's disease is associated with a high economic burden for the Spanish National Healthcare System, driven by pharmacological expenses. Following the recent incorporation of ustekinumab biosimilars into the market, this study aimed to evaluate the cost effectiveness of different treatment sequences, including biosimilar ustekinumab (bsUST), for moderate-to-severe Crohn's disease from the NHS perspective in Spain.</p><p><strong>Methods: </strong>Treatment sequences were defined according to clinical practice. A Markov model with 2-week cycles and a lifetime horizon was developed, including health states such as active disease, response, remission, surgery, and death. Funnel states simulated induction and treatment changes after loss of response, and surgery was considered only after four pharmacological lines. Efficacy data for biosimilar adalimumab (bsADA), biosimilar intravenous infliximab (bsIFX), biosimilar ustekinumab (bsUST) risankizumab (RIS), upadacitinib (UPA), vedolizumab (VDZ), and surgery (Q) were obtained through published literature, including network meta-analyses and clinical trials. Unitary costs were sourced from Spanish databases and literature. Pharmacological costs were ex-factory prices applying Royal Decree-Law (RDL) 8/2010 discount. For bsADA and bsIFX, average biosimilar market prices were used. A 30% discount was assumed for bsUST versus reference ustekinumab. Sequence 1 (bsADA-bsUST-UPA-RIS-Q) was set as the reference.</p><p><strong>Results: </strong>Alternative strategies provided QALY gains ranging from 0.06 to 0.34 compared with the reference. However, due to varying incremental costs, Sequence 2 (bsUST, bsADA, UPA, RIS, Q) emerged as the only cost-effective strategy at a willingness-to-pay threshold of €27,000/QALY, with an incremental cost-effectiveness ratio (ICER) of €8672.6/QALY. Conversely, all other sequences exceeded the threshold, with ICERs starting at €42,594/QALY. Probabilistic sensitivity analysis confirmed the robustness of these findings, identifying Sequence 2 as the cost-effective option in 98.03% of simulations.</p><p><strong>Conclusions: </strong>Positioning bsUST as a first line of treatment appears to be cost effective and an efficient alternative from the NHS perspective in Spain. Additionally, none of the alternative sequences proved to be cost effective compared with the sequence starting with bsADA-bsUST. bsUST may significantly impact Crohn's disease management in Spain, improving patient access due to its lower drug acquisition costs.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"557-566"},"PeriodicalIF":2.4,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13194847/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147390595","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-Effectiveness of Follitropin Delta Compared with Follitropins Alfa and Beta in Controlled Ovarian Stimulation for Assisted Reproductive Technologies (ART) in France.","authors":"Samir Hamamah, Jeremy Carette, Henri Leleu, Marie Markert","doi":"10.1007/s41669-026-00641-2","DOIUrl":"10.1007/s41669-026-00641-2","url":null,"abstract":"<p><strong>Background: </strong>Follitropin delta, using a personalized dosing regimen, is an effective treatment option for women undergoing controlled ovarian stimulation (COS) for in vitro fertilization (IVF)/intracytoplasmic sperm injection (ICSI).</p><p><strong>Objective: </strong>The aim of this study was to develop a model to determine cost effectiveness of follitropin delta compared with follitropins alfa and beta for women undergoing IVF/ICSI in France.</p><p><strong>Methods: </strong>A decision-tree model was developed comparing the outcomes of treatment with follitropin delta versus other follitropins through ongoing pregnancy (OP) and live birth (LB) rates in fresh cycles. Pooled data from the pivotal clinical trials ESTHER (EU + rest of world; NCT01956110), GRAPE (Pan-Asia; NCT03296527), and STORK (Japan; NCT03228680) was used for the economic model. The analyses were stratified by age and ovarian reserve profile and reflected a single COS cycle. Costs were estimated from the healthcare perspective in France, and uncertainty was assessed through sensitivity analyses.</p><p><strong>Results: </strong>In women with an elevated anti-Müllerian hormone level (≥15 pmol/L), follitropin delta achieved a higher rate of LB (31.4% vs 25.8%, p = 0.01) and a numerically higher rate of OP (35.7% vs 31.6%) compared with follitropins alfa/beta. Additionally, treatment with follitropin delta was associated with numerically fewer miscarriages (4.3% vs 5.8%) and lower ovarian hyperstimulation syndrome (OHSS) incidence (8.2% vs 11.5%). Total treatment cycle cost with/without delivery cost was €5479/€4099 for follitropin delta, €5335/€4191 for follitropin alfa, and €5387/€4243 for follitropin beta. The incremental cost-effectiveness ratio was €2579/LB for follitropin delta versus follitropin alfa. Follitropin beta was shown to be less efficient, and more costly (i.e. dominated). Excluding the delivery cost, follitropin delta was more efficient and less costly (i.e. dominant) versus other follitropins. Probabilistic sensitivity analyses supported the deterministic results, showing > 76% probability of follitropin delta being dominant when assessing cost per additional OP. Similar results were observed in the overall population of women.</p><p><strong>Conclusions: </strong>Follitropin delta provides an effective alternative to follitropin alfa and beta with a potential cost-savings opportunity, excluding the delivery cost, due to higher OP and LB rates in the fresh cycle transfers.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"519-529"},"PeriodicalIF":2.4,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13194827/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147434658","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Cost-Analysis and Micro-Costing Study of the Man Van Project: Comparing Prostate-Specific Antigen Testing Via a Nurse-Led Mobile Service with Primary Care.","authors":"Masood Moghul, Deokhee Yi, Fionnuala Croft, Fiona Mutch, Elisabeth Westaway, Netty Kinsella, Patrick Kierkegaard, Declan Cahill, Nicholas David James","doi":"10.1007/s41669-025-00637-4","DOIUrl":"10.1007/s41669-025-00637-4","url":null,"abstract":"<p><strong>Background: </strong>The Man Van project is designed to address health inequalities and barriers to accessing healthcare related to the early diagnosis of prostate cancer with novel community-based targeting of high-risk groups using a mobile clinical unit. These inequalities are particularly relevant for men from ethnic minorities and lower socio-economic groups. We used a cost analysis with a micro-costing study to examine the costs of using the Man Van model for community-based prostate-specific antigen (PSA) testing compared with primary care-based PSA testing over the course of a 2-year period.</p><p><strong>Methods: </strong>The Man Van service has a nurse-led model with initial counselling carried out by band 8 (pilot) or 7 (phase 2) nurses assisted by a Band 4 care support worker. The Man Van operated in North and South-West London between 2021 and 2024. PSA counselling and blood testing were performed within the same appointment. PSA testing in primary care is performed by general practitioners with counselling followed by a phlebotomy appointment. In both primary care and the Man Van service, normal results are usually texted to patients with telephone calls for abnormal results. A micro-costing study and cost analysis was undertaken to compare costs of the primary care and Man Van models of PSA testing from the perspective of the National Health Service. Baseline staffing costs were approximated from data obtained from the Personal Social Services Research Unit based in the UK and include direct and indirect overheads.</p><p><strong>Results: </strong>A micro-costing analysis showed costs per participant seen of £81.11 in the pilot phase of the Man Van, reducing by 33% to £54.32 in phase 2. Costs per prostate cancer diagnosis were £5552.20 in the pilot, reducing to £3319.29 in phase 2. A cost analysis found a 25% saving of the Man Van model versus the primary care model with costs reducing from £68.44 to £51.11. A one-way sensitivity analysis varying individual cost inputs by ±20% showed that the total cost difference between the Man Van and primary care models ranged from a saving of approximately £2.92 to £31.72 per participant, depending on the parameter varied.</p><p><strong>Conclusions: </strong>Like all government funded healthcare services, a health-economic analysis is integral to supporting the case for the Man Van project into the future. The project has shown cost efficiency, saving a quarter of costs compared with the standard process of primary care-led PSA testing. Further work is needed to understand the economic impact, in particular of the inclusion of other health checks in the Man Van model.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"577-588"},"PeriodicalIF":2.4,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13194830/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146030564","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Incremental Disease Burden (Healthcare Costs and Resources) of Duchenne Muscular Dystrophy in the US: A Matched Cohort Analysis.","authors":"Mena Scavina, Nate Posner, Patricia Dorling, Anna Talaga, Anna Stachel, Jose Alvir","doi":"10.1007/s41669-025-00612-z","DOIUrl":"10.1007/s41669-025-00612-z","url":null,"abstract":"<p><strong>Background: </strong>Duchenne muscular dystrophy (DMD) is a rare genetic condition that causes progressive muscle weakness and reduced life expectancy. Detailed economic studies on the impact of DMD disease burden, costs, and healthcare resource utilization (HCRU) are limited.</p><p><strong>Objective: </strong>To compare healthcare costs, HCRU, and ambulatory status between a cohort of DMD patients and a matched cohort of patients without muscular dystrophies (non-MD).</p><p><strong>- methods: </strong>A retrospective study was conducted using Merative MarketScan^® Commercial and Medicaid databases. Patients with DMD were identified using a validated algorithm. The index date for the DMD cohort was the first date with an ICD-10 code: G71.01. The study included males without cancer, aged ≤ 40 years, and consistently enrolled for 3 months pre-index and 12 months post-index. Patients with DMD were matched 1:1 with non-MD patientsby age, region, race, insurance, and index date year. Patients with DMD were compared for healthcare costs and HCRU on the basis of ambulatory function versus non-ambulatory status (wheelchair purchase).</p><p><strong>Results: </strong>The DMD and non-MD cohorts included 782 matched pairs. Patients with DMD had significantly higher mean unadjusted total healthcare costs (US$103,826.05 ± 309,335.71) per patient per year compared with patients with non-MD (US$3225.30 ± 13,865.14; p < 0.0001) per patient per year. The total healthcare cost was primarily driven by pharmacy and outpatient costs. The DMD cohort showed significantly higher HCRU than the non-MD cohort across physician offices, outpatient, emergency room, speech therapy, physical therapy, endocrinology, cardiology, orthopedic, pulmonology, and physical medicine visits. DMD patients with non-ambulatory status were older, medically progressed, and incurred higher costs than patients with ambulatory function.</p><p><strong>Conclusions: </strong>DMD imposes a substantial cost and healthcare resource burden on patients and the healthcare system. The results suggest a high unmet need that requires the development of novel therapies to treat DMD.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"491-501"},"PeriodicalIF":2.4,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13194850/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146011451","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-Utility Analysis of Faricimab Versus Aflibercept in Treating Neovascular Age-Related Macular Degeneration (nAMD) in the United States.","authors":"Ahmead Baljoon, Vakaramoko Diaby, Sandra Suther, Matthew Dutton, Askal Ali, Islam Eljilany","doi":"10.1007/s41669-026-00643-0","DOIUrl":"10.1007/s41669-026-00643-0","url":null,"abstract":"<p><strong>Background/objective: </strong>Neovascular age-related macular degeneration (nAMD) imposes a substantial clinical and economic burden in the United States (US). Faricimab offers the potential for longer dosing intervals than aflibercept, but its economic value in the US setting has not been established. This study evaluated the cost utility of faricimab versus aflibercept for treating nAMD from the US payer perspective.</p><p><strong>Methods: </strong>A Markov model of five health states defined by best-corrected visual acuity (BCVA) simulated disease progression over 5 years. Clinical inputs were drawn from pooled 1-year data of the TENAYA and LUCERNE phase III trials (n = 1329), which compared faricimab with aflibercept for treating nAMD patients. Direct medical costs encompassed drug acquisition, intravitreal administration, monitoring, office visits, and management of endophthalmitis. Outcomes included total costs, quality-adjusted life years (QALYs), incremental cost-effectiveness ratio (ICER), and net monetary benefit (NMB) at a willingness-to-pay (WTP) threshold of US$100,000/QALY. Scenario, deterministic, and probabilistic sensitivity analyses (10,000 iterations) were conducted.</p><p><strong>Results: </strong>In the base case, faricimab yielded 2.80 QALYs for US$52,797, whereas aflibercept produced 2.72 QALYs at US$62,367. Faricimab was therefore dominant (- US$9570; + 0.08 QALY), with an incremental NMB of US$17,981. Faricimab required markedly fewer injections over 5 years (22.6 vs 34). Scenario analyses did not materially change the directionality of the base-case results, with faricimab being dominant or cost effective. The model was most sensitive to drug acquisition prices and BCVA-state utilities, yet faricimab remained below the WTP threshold across all plausible ranges. Probabilistic analysis showed a 97% probability of faricimab being cost effective at US$100,000/QALY.</p><p><strong>Conclusions: </strong>Faricimab is estimated to be a cost-effective treatment option compared with aflibercept for nAMD from a US payer perspective. This finding may support its inclusion in treatment guidelines and formulary decisions, pending confirmation in real-world practice.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"457-469"},"PeriodicalIF":2.4,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13194801/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147366125","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Transferable Exclusivity Extension Vouchers for Antimicrobials: Incentive Design, Implementation Challenges, and Policy Trade-Offs.","authors":"Mario García-Díaz, Jorge Mestre-Ferrándiz, Jaime Espín","doi":"10.1007/s41669-025-00628-5","DOIUrl":"10.1007/s41669-025-00628-5","url":null,"abstract":"<p><p>Antimicrobial resistance (AMR) is one of the greatest threats to public health, and forecasts for 2050 are even worse. In response, the European Commission (EC) is proposing the Transferable Exclusivity Extension Voucher (TEEV), which aims to encourage the development of innovative antimicrobials. It consists of extending data exclusivity for a medicine selected by the beneficiary company or another company to which the voucher can be sold (i.e. not necessarily the antimicrobial). One requirement for obtaining it is to declare any public contributions received for the antimicrobial R&D. Based on a review of the literature and interviews with five AMR experts, a theoretical and real view of how the TEEV works and its estimated costs is presented, in addition to the impact that the requirement for transparency in R&D costs would have. On the one hand, the results show that a (well-designed) TEEV, coordinated by the EC, offers significant public health benefits compared with the cost of inaction and provides predictability for investors, although, by its nature, it delays the entry of generics/biosimilars and does not provide an access scheme to new antimicrobials. Recent assessments estimate the total healthcare cost at €162 million per voucher and the average cost for each Member State at €6 million. On the other hand, transparency of R&D costs could enable policymakers to design policies focused on public health needs and better design the optimal mix of (push and pull) incentives for new antimicrobials, but they run the risk of threatening trade secrets, discouraging private investment and promoting the application of cost-plus pricing regulations. In conclusion, the TEEV could be a great incentive to combat AMR inaction, but it has not yet been implemented and needs to be complemented by other push and pull incentives. Furthermore, more information is needed on the impact of R&D costs transparency in the context of antimicrobials.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"393-406"},"PeriodicalIF":2.4,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13194829/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145959990","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comparative Cost-Effectiveness Analysis of Multiple First-Line Treatments for HER2-Negative Unresectable Advanced or Recurrent Gastric Cancer in Japan.","authors":"Kosuke Morimoto, Kensuke Moriwaki, Yoshitaka Nishikawa, Tomomi Maeda, Mayumi Toyama, Nobukazu Agatsuma, Satomi Kojima, Takahiro Utsumi, Yoshimitsu Takahashi, Kojiro Shimozuma, Takeo Nakayama","doi":"10.1007/s41669-025-00634-7","DOIUrl":"10.1007/s41669-025-00634-7","url":null,"abstract":"<p><strong>Background/objective: </strong>HER2-negative unresectable advanced or recurrent gastric cancer (GC) has a poor prognosis. This study evaluated the comparative cost-effectiveness of multiple first-line treatment options from the Japanese healthcare payer perspective.</p><p><strong>Methods: </strong>A partitioned survival model was developed to estimate the cost-effectiveness of each regimen. Hazard ratios were derived from a network meta-analysis to project long-term costs and quality-adjusted life years (QALYs) gained. Regimens included pembrolizumab plus chemotherapy (Pem + Chemo); zolbetuximab plus chemotherapy (Zolb + Chemo); nivolumab plus chemotherapy (NIVO + Chemo); 5-fluorouracil, oxaliplatin, and levofolinate (FOLFOX); capecitabine and oxaliplatin (CapeOx); S-1 and oxaliplatin (SOX), capecitabine and cisplatin (Cape + CDDP); and S-1 and cisplatin (S-1 + CDDP). Cost parameters were obtained from a Japanese medical claims database. Incremental cost-effectiveness ratios (ICERs) were calculated for each regimen. Sensitivity analyses were conducted to assess parameter uncertainty. Scenario analysis was conducted by incorporating tislelizumab, which is approved and recommended in countries outside Japan.</p><p><strong>Results: </strong>On the basis of the base-case analysis, CapeOx, SOX, Pem + Chemo (5-FU + CDDP, CapeOx), and Zolb + CapeOx were located on the efficiency frontier. The ICER of SOX versus CapeOx was USD 85,649/QALY. Pem + Chemo versus SOX had an ICER of USD 345,103/QALY. Zolb + CapeOx versus Pem + Chemo had an ICER of USD 1,637,571/QALY. Probabilistic sensitivity analysis showed SOX had the highest probability (40.33%) of being the most cost-effective.</p><p><strong>Conclusions: </strong>At a willingness-to-pay threshold of USD 100,000/QALY (Japan's HTA upper reference), SOX was identified as the most cost-effective first-line treatment for HER2-negative unresectable advanced or recurrent GC in Japan.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"471-489"},"PeriodicalIF":2.4,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13194900/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146158046","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Content Validity of Two Generic Preference-Based Measures in Retinitis Pigmentosa, Including X-Linked Retinitis Pigmentosa.","authors":"Louis S Matza, Timothy A Howell, Qiaoyi Zhang, Tom Denee","doi":"10.1007/s41669-025-00617-8","DOIUrl":"10.1007/s41669-025-00617-8","url":null,"abstract":"<p><strong>Introduction: </strong>Retinitis pigmentosa (RP) is a group of retinal diseases characterized by night blindness and impairment in visual field and acuity. The most severe form of RP is X-linked RP (XLRP). The relevance of common generic preference-based measures (GPBMs) in visual conditions like XLRP is often questioned. The purpose of this study was to assess content validity of the EQ-5D-5L and Health Utilities Index (HUI) in patients with XLRP or other types of RP, collectively abbreviated as (XL)RP.</p><p><strong>Methods: </strong>People with (XL)RP in the US participated in qualitative interviews conducted via telephone/videoconference. Interviews began with concept elicitation to identify the impact of (XL)RP on quality of life. Participants then completed the instruments and answered questions about their clarity, relevance, and comprehensiveness.</p><p><strong>Results: </strong>The sample included 23 participants with RP, including 12 with XLRP (73.9% male; mean age 50.2 y). Participants reported a variety of impacts of (XL)RP, some of which overlap with the EQ-5D-5L and HUI (e.g., emotional impact, usual activities). Participants stated that both instruments were clear and easy to understand (n = 21, 91.3% for EQ-5D-5L and n = 18, 78.3% for the HUI), but also identified important concepts that were not assessed by the questionnaires. Eighteen participants (78.2%) identified at least one important concept missing from the EQ-5D-5L and 20 participants (87.0%) identified at least one concept missing from the HUI. Missing concepts included visual symptoms, driving, impact of light/darkness, and ability to do things they used to enjoy.</p><p><strong>Conclusion: </strong>While the EQ-5D-5L and HUI include some content relevant to people with (XL)RP, both instruments appear to be missing concepts that are specifically relevant to this patient population, which could limit content validity and sensitivity to disease severity. Therefore, it may be useful to consider alternative methods for estimating utilities associated with (XL)RP.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"341-354"},"PeriodicalIF":2.4,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13000043/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146030530","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-Effectiveness of RefluxStop Versus Nissen Fundoplication and Proton Pump Inhibitors for Refractory Gastroesophageal Reflux Disease: A Spanish Healthcare Perspective.","authors":"Sam Harper, Khanh Ha Bui, Stuart Mealing, Daniel Sanchez, Pablo Priego Jiménez, Carlos Moreno Sanz","doi":"10.1007/s41669-025-00636-5","DOIUrl":"10.1007/s41669-025-00636-5","url":null,"abstract":"<p><strong>Background: </strong>Gastroesophageal reflux disease (GERD) affects 6.8 million individuals in Spain, incurring €56 million/year in healthcare costs. Standard-of-care treatment (SOC) in Spain includes medical management with proton pump inhibitors (PPIs) and laparoscopic Nissen fundoplication (LNF) in selected cases. The limitations of PPIs, including high rates of unresponsiveness, adverse events (AEs) associated with long-term use, and nonindicative misuse, increase the economic strain on European healthcare systems. The durability of LNF treatment is hindered by reoperation and postoperative complications. RefluxStop, a novel implantable device, restores the anti-reflux barrier without encircling the esophagus and confers long-term efficacy and safety in the treatment of GERD.</p><p><strong>Objective: </strong>This study assessed the cost-effectiveness of RefluxStop compared with medical (PPI-based) and surgical (LNF) SOC for refractory GERD in Spain.</p><p><strong>Methods: </strong>The cost-effectiveness of RefluxStop versus PPI-based medical management and LNF was assessed from the Spanish National Health System (SNS) perspective over a lifetime horizon (monthly cycles, 3.0% annual discount rate). A Markov model adapted from a published UK National Health Service (NHS) cost-effectiveness analysis of this device was used. Quality-adjusted life years (QALYs) and total costs were calculated for each intervention, while inter-arm differences were evaluated using incremental cost-effectiveness ratios (ICERs).</p><p><strong>Results: </strong>RefluxStop yielded ICERs of €557 and €2393 per QALY gained compared with medical management and LNF, respectively. At the cost-effectiveness threshold of €30,000 per QALY gained for Spain, the probability of RefluxStop being cost-effective was 100% compared with medical management and 93% compared with LNF. Over a lifetime horizon, the per-patient cost differences and QALY gains for RefluxStop were €1472 and 2.64 versus medical management and €2111 and 0.88 versus LNF, respectively. The model results remained robust to sensitivity analysis.</p><p><strong>Conclusions: </strong>RefluxStop was estimated to be cost-effective relative to SOC in Spanish healthcare settings for the treatment of adult patients with refractory GERD, consistent with recently published findings in the UK, Switzerland, Sweden, and Norway. It is acknowledged that the model has limitations, including its reliance on single-arm trial data and indirect comparisons using heterogeneous literature sources, which limit the precision and generalizability of its findings.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"249-263"},"PeriodicalIF":2.4,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13000083/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145966911","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost Analysis of Immunoglobulin Maintenance Treatment of Patients with Chronic Inflammatory Demyelinating Polyneuropathy in Spain.","authors":"Sofía de Pedro, María Presa, Josep Gamez, Francisco Javier Gómez Fernández, Luis Querol","doi":"10.1007/s41669-025-00633-8","DOIUrl":"10.1007/s41669-025-00633-8","url":null,"abstract":"<p><strong>Background/objective: </strong>Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare, immune-mediated disorder, which causes peripheral nervous system dysfunction. Guidelines recommend maintenance treatment with intravenous (IVIG) or subcutaneous (SCIG) immunoglobulins. Drawbacks for IVIG include the burden of hospital administration, which is less for SCIG, which allows self- or caregiver-assisted administration at home. However, conventional SCIG (c-SCIG) requires more frequent administration than IVIG. Recently, facilitated-SGIG (f-SCIG) was developed, spacing injections so that administration frequency is analogous to IVIG. The aim of this study was to estimate and compare the costs of IVIG, c-SCIG, and f-SCIG for CIDP maintenance treatment in Spain.</p><p><strong>Methods: </strong>A cost-analysis model based on a decision tree was developed to evaluate the annual cost per patient treated with each immunoglobulin from a societal perspective. Costs considered included direct health costs (pharmaceutical acquisition and administration), non-health costs (transport to hospital), and indirect costs related to labor productivity. All costs were valued in 2024 euros.</p><p><strong>Results: </strong>The annual cost for a patient with CIDP receiving maintenance treatment was €109,212.67 with IVIG, €98,161.97 with c-SCIG, and €98,191.37 with f-SCIG. Across all treatments, direct healthcare costs were the highest. The main driver of the difference between SCIG and IVIG cost was treatment administration (-€10,705.78). Sensitivity analyses were generally consistent with the base case results.</p><p><strong>Conclusions: </strong>Overall, the findings of this analysis suggest that SCIG may be a cost-saving alternative to IVIG for patients with CIDP requiring maintenance treatment in Spain, with a similar cost between c-SCIG and f-SCIG.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":" ","pages":"381-391"},"PeriodicalIF":2.4,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13000016/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145959938","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}