Journal Of Medical Economics[JOURNAL]
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200 papers
Effectiveness of the etonogestrel implant: an economic model of immediate postpartum use on pregnancy outcomes.
Patel VP, Collins K, Heinemann K … +2 more , Ngantung W, Saeedian M
J Med Econ · 2025 Dec · PMID 41307487 · Publisher ↗
OBJECTIVE: Long-acting reversible contraception (LARC) is the most effective form of reversible contraception. Leading organizations agree that LARC should be offered immediately postpartum. The etonogestrel implant is t...
OBJECTIVE: Long-acting reversible contraception (LARC) is the most effective form of reversible contraception. Leading organizations agree that LARC should be offered immediately postpartum. The etonogestrel implant is the most effective LARC but carries higher upfront acquisition costs. This study evaluated whether the acquisition costs for the etonogestrel implant are offset via pregnancy prevention and pregnancy-related costs in the year after childbirth. METHODS: A Markov model with 1-year time horizon simulated pregnancy outcomes among 1,000 women 18-49 years initiating 1 of 8 hormonal contraceptives for postpartum contraception: progestin-only oral contraception (OC), combined OC, progestin-only injectable, etonogestrel/ethinyl estradiol vaginal ring, norelgestromin/ethinyl estradiol transdermal patch, 3- or ≥5-year levonorgestrel IUD, and the etonogestrel implant. Time to postpartum initiation for each method and IUD expulsion rates were incorporated. Contraceptive acquisition costs, failure rates, discontinuation rates, pregnancy outcomes and costs, and healthcare resource use were examined from Commercial and Medicaid payor perspectives. Sensitivity analyses were conducted. RESULTS: The etonogestrel implant followed by IUDs were associated with the fewest short-interval pregnancies (8 [etonogestrel implant], 49 [3-year IUD], and 47 [≥5-year IUD], respectively) and the lowest Commercial (Medicaid) per-woman costs ($1,650 ($1,535) [etonogestrel implant]; $2,364 ($1,979) [3-year IUD]; and $2,519 ($2,145) [≥5-year IUD], respectively). In comparison, short-acting reversible contraception (SARC) resulted in 59-167 pregnancies, and Commercial (Medicaid) per-woman costs ranged from $2,244 ($1,749) to $5,102 ($4,106). Acquisition costs and discontinuation rates were the key drivers impacting overall costs. CONCLUSIONS: The immediate postpartum provision of the etonogestrel implant produced the greatest cost savings due to fewer short-interval pregnancies and related expenditures. These results highlight the etonogestrel implant as a cost-effective option for clinicians seeking to reduce short-interval pregnancy and health system costs.
Chronic myeloid leukemia treatment intolerance imposes additional resource and economic burden on oncology practices in the United States.
Shafrin J, Jadhav K, Warren C … +7 more , Quddus S, Zawadzki N, Yang D, Damon A, Spurrier K, Batt K, Wei D
J Med Econ · 2025 Dec · PMID 41295925 · Publisher ↗
OBJECTIVE: Tyrosine kinase inhibitors (TKIs) have transformed the prognosis of chronic myeloid leukemia (CML) into a manageable chronic condition, but TKI intolerance remains a significant issue. This study aimed to quan...
OBJECTIVE: Tyrosine kinase inhibitors (TKIs) have transformed the prognosis of chronic myeloid leukemia (CML) into a manageable chronic condition, but TKI intolerance remains a significant issue. This study aimed to quantify the economic burden incurred by oncology practices to manage a CML treatment intolerance episode. METHODS: This mixed-methods study leveraged interviews with US oncology practice managers (e.g. physicians, nurse leaders, advanced practice providers) and cost estimation analysis to estimate average time and cost per CML patient treatment intolerance episode, defined as an occurrence of any adverse effect associated with CML treatment from the perspective of an oncology practice. Cost analysis primarily focused on labor costs based on available data from interview responses. Participants were recruited via convenience sampling. RESULTS: = 10 academic oncology practice managers were interviewed. From the perspective of an oncology practice, average practice time and cost per treatment intolerance episode requiring hospitalization were 34 h and $3,380, driven by payer prior authorization interactions (10 h, $838), additional patient visits (8 h, $858) and inpatient care coordination (9 h, $782). Reimbursable activities represented 31.2% of incurred costs. CONCLUSION: These findings highlight the significant uncompensated burden placed on oncology practices, raising concerns about how non-reimbursed tasks may affect patient care, staff retention, and the financial sustainability of these practices.
Quantifying treatment value under IRA: a case study of rifaximin for the treatment of overt hepatic encephalopathy using QALY and non-QALY measures.
Wang S, Passos Chaves L, Olujohungbe O … +3 more , Muthukrishnan S, Chaudhary A, Shafrin J
J Med Econ · 2025 Dec · PMID 41295924 · Publisher ↗
BACKGROUND: Traditional cost effectiveness analyses frequently use quality-adjusted life years (QALYs) to quantify health benefits. The Medicare Drug Price Negotiation program, however, cannot use QALYs, but may consider...
BACKGROUND: Traditional cost effectiveness analyses frequently use quality-adjusted life years (QALYs) to quantify health benefits. The Medicare Drug Price Negotiation program, however, cannot use QALYs, but may consider alternative, non-discriminatory metrics. OBJECTIVE: To examine the impact of using alternative quantitative health benefit metrics on the economic value of new medications. The framework was applied to assess the cost-effectiveness of rifaximin for preventing overt hepatic encephalopathy (OHE) recurrence in adults. METHODS: A cost-effectiveness analysis evaluated the economic value of rifaximin ± lactulose versus standard of care ± lactulose in preventing recurrent OHE in adults over a lifetime horizon from US payer and societal perspectives. Clinical outcomes included time in remission and overt health states, number of liver transplants, and life years (LYs). Health benefit was quantified using QALYs, health years in total (HYT), equal-value of life years gained (evLYG), and generalized risk-adjusted cost-effectiveness (GRACE). Treatment value was measured using incremental cost effectiveness ratio (ICER). A societal perspective scenario added productivity and caregiving impacts to the model. RESULTS: Rifaximin patients spent >3 times as long in remission (54.4 vs. 17.3 months), comparable time in the overt health state (1.44 vs. 1.44 months), and had twice as many liver transplants (20 vs. 9), driven by longer survival (8.80 vs. 4.17 LYs), resulting in incremental gains of 3.12 QALYs, 3.26 HYT, 2.78 evLYG, and 3.16 GRA-QALYs. Total costs were higher with rifaximin ($182,369 vs. $38,313, Δ = $144,056), mainly due to drug costs (Δ = $133,330). Including caregiving and productivity reduced the incremental cost to $136,866. From a payer perspective, rifaximin ICERs were $46,215/QALY, $44,198/HYT, $51,847/evLYG, and $45,609/GRA-QALY. After incorporating societal costs, ICERs improved to $43,908/QALY, $41,992/HYT, $49,259/evLYG, and $43,332/GRA-QALY. CONCLUSION: Rifaximin is a cost-effective treatment for preventing OHE recurrence in adults using QALY and non-QALY health benefit measures.
Sex disparities of vaccine-preventable cancer mortality in Latin America.
Parellada CI, Siddiqui AH, Oliver E … +5 more , Hughes R, Meiwald A, Orengo JC, Eiden A, Bencina G
J Med Econ · 2025 Dec · PMID 41295897 · Publisher ↗
OBJECTIVE: This analysis estimated productivity losses and assessed sex disparities due to vaccine-preventable cancer deaths from hepatitis B virus (HBV) and human papillomavirus (HPV) in Latin America. METHODS: The numb...
OBJECTIVE: This analysis estimated productivity losses and assessed sex disparities due to vaccine-preventable cancer deaths from hepatitis B virus (HBV) and human papillomavirus (HPV) in Latin America. METHODS: The number of deaths and years of life lost (YLL) for 19 Latin American countries were sourced from the Institute for Health Metrics and Evaluation Global Burden of Disease (2019) for cervical, oral cavity, laryngeal, oropharyngeal, and HBV-related hepatic cancers. Attributable fractions were applied to estimate HPV-related mortality. The value of YLL (VYLL) was estimated using country-specific GDP per capita and discounted at 3%. Sensitivity and scenario analyses were conducted. RESULTS: In 2019, 38,786 vaccine-preventable cancer deaths resulted in over 1.1 million YLL and $5.9 billion VYLL across Latin America. Cervical cancer accounted for 81.6% of deaths and $4.9 billion in VYLL, with females representing 87.0% of the total VYLL. For males, hepatic cancer was the highest VYLL at $295 million and oral cavity the lowest ($94 million). AYLL was higher in females (30 years) than males (27 years). CONCLUSIONS: HBV- and HPV-related cancers impose a significant economic and mortality burden in Latin America, with marked sex disparities. Cervical cancer remains the dominant contributor among females, while males are disproportionately affected by head and neck and hepatic cancers. These findings underscore the need for sex-specific public health strategies, including expanded vaccination, improved screening, and timely treatment access across the region.
The price of progress: balancing short-term gains and long-term value in aortic valve replacement.
Layton G, Robayo V, Rathakrishnan B … +2 more , Okoh A, Hirji S
J Med Econ · 2025 Dec · PMID 41273707 · Publisher ↗
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Real-world disease burden and health care resource utilization for patients with Barth syndrome.
Marjoram L, Huang Y, Koenig MK … +2 more , Cohen BH, Anderson E
J Med Econ · 2025 Dec · PMID 41268908 · Publisher ↗
BACKGROUND: Barth syndrome (BTHS) is an ultra-rare, X-linked genetic disorder for which there is limited economic data. Because compiling such data that target rare indications is difficult, we assessed real-world data t...
BACKGROUND: Barth syndrome (BTHS) is an ultra-rare, X-linked genetic disorder for which there is limited economic data. Because compiling such data that target rare indications is difficult, we assessed real-world data to increase understanding of the cost of BTHS based on disease burden and health care resource utilization (HCRU). METHODS: A search of the published medical literature identified individual case studies and registry data used to assess the burden of disease and potential costs associated with BTHS, including the potential ability of developing treatments to positively impact those costs. In addition, a claims database analysis was conducted to assess HCRU and associated costs in the United States for patients with BTHS. These real-world data were summarized and compared with registry data. RESULTS: The diagnostic journey for patients with BTHS is difficult, with the majority of affected individuals not receiving a diagnosis until after the development of cardiomyopathy or until a family member is diagnosed, even in those who are symptomatic. Of the living individuals known to have BTHS, a high proportion experience heart failure, with >15% requiring heart transplantation. Data extrapolated during the claims database analysis demonstrated that patients with diagnostic codes linked to BTHS are complex, with a high percentage of complications, necessitating a high level of HCRU and associated costs of care in the inpatient setting. Specifically, National Inpatient Sample hospital cost per claim was $32,702 and the Kids' Inpatient Database hospital cost per claim was $62,596. CONCLUSIONS: Health economic evaluations for rare diseases are scarce. With a noteworthy absence of pharmacoeconomic evidence, utilization of combined clinical case report data from the medical literature, along with registry and claims datasets, demonstrate that BTHS is a costly disease associated with high disease burden and excessive HCRU. Forthcoming treatments (e.g. elamipretide) have the potential to reduce the high disease burden/HCRU.
A budget impact model for biosimilar denosumab for skeletal-related events and fractures in the United States oncology population.
Flanigan J, Chaplin S, van Stiphout J … +5 more , Schauf M, Tindal M, Gundlach A, Li E, Lyman GH
J Med Econ · 2025 Dec · PMID 41258696 · Publisher ↗
BACKGROUND: The significant costs associated with cancer and related treatments are concerning for patients, providers, and payers. Denosumab is indicated to treat a variety of skeletal-related events (SREs) and fracture...
BACKGROUND: The significant costs associated with cancer and related treatments are concerning for patients, providers, and payers. Denosumab is indicated to treat a variety of skeletal-related events (SREs) and fractures in cancer patients but remains costly. Biosimilars offer an affordable treatment option, which can potentially reduce healthcare costs and expand access to biological therapy. OBJECTIVE: Evaluate the budget impact of incorporating biosimilar denosumab into a health plan formulary for the oncology indications of reference denosumab products and estimate costs associated with SREs and fractures after reinvesting savings from biosimilar conversion. METHODS: Two models were developed to estimate the budget impact of biosimilar denosumab from a United States payer perspective over a 5-year period. The first model focused on total cost savings associated with switching to biosimilar denosumab from reference denosumab. In the second model, savings estimated in the first model were reinvested for conversion from zoledronic acid to biosimilar denosumab. RESULTS: The introduction of biosimilar denosumab resulted in total cost savings of $38,677,606 or $0.59 per member per month at year 5 in the base case. Cost savings were also seen with high and low scenarios as well as when the average sales price varied from 5% to 85%. Approximately 100 additional patients could be treated with biosimilar denosumab and an additional three SREs and fractures could be prevented with each 10% increase in reinvestment. LIMITATIONS: As the price of biosimilar denosumab was based on prior experiences of biosimilar prices relative to reference prices, current prices or payment limits of biosimilar denosumab may not be reflected. CONCLUSIONS: The availability of denosumab biosimilars provides an opportunity for oncology practices to assess the cost-effectiveness of biosimilar denosumab and expand access to biologicals to more patients.
Hexaminolevulinate-enhanced photodynamic diagnosis in the management of non-muscle-invasive bladder cancer (NMIBC): the influence of differing European health care payment systems on the potential financial impact of adoption.
Belsey J, Lapon J
J Med Econ · 2025 Dec · PMID 41247459 · Publisher ↗
INTRODUCTION: The management of NMIBC is based on primary resection, followed by a surveillance programme stratified according to the risk status of the tumour. The completeness of the primary resection will determine th...
INTRODUCTION: The management of NMIBC is based on primary resection, followed by a surveillance programme stratified according to the risk status of the tumour. The completeness of the primary resection will determine the likelihood of early recurrence and requirement for re-resection. The use of photodynamic diagnosis (PDD) has been shown to extend the median time to first recurrence compared to white-light cystoscopy alone (WLC). However, PDD requires the use of a fluorescent imaging agent, which incurs an additional cost. This analysis explores the impact of the health care payment system on the budgetary impact of PDD adoption by a hospital. METHODS: A previously published budget impact model was adapted to allow for the exploration of costs across four different payment environments: Denmark, France, Italy and Finland. Using the same set of clinical assumptions around disease risk profiles, recurrence rates and usage of PDD, coupled with country-specific costs, the net budget impact per patient with NMIBC was estimated for each country over a 3-year time horizon. The analysis was carried out from the perspective of a hospital with a protocol-driven strategy for PDD adoption. RESULTS: In Denmark, with a differential tariff system between PDD and WLC, the additional cost of the technology was fully offset by the tariff, with a net surplus of €170 per patient. In France and Italy, with a flat-rate tariff, there was a net cost of €108 and €120 per patient respectively. In Finland, with a block contract system, the net cost per patient was €206. CONCLUSIONS: Despite a consistent clinical benefit associated with PDD, differences in healthcare payment systems across Europe impact on its net financial impact. The use of flexible budgetary impact models may allow tailored implementation plans to be developed, allowing local needs to be balanced with cost consequences., thereby allowing for targeted adoption.
Longitudinal analyses of healthcare resource utilization and costs among patients with obstructive hypertrophic cardiomyopathy.
Reza N, Butzner M, Batra K … +4 more , Amos Q, Buikema A, Shreay S, Owens A
J Med Econ · 2025 Dec · PMID 41241831 · Full text
AIMS: Obstructive hypertrophic cardiomyopathy (oHCM) is associated with substantial disease burden, healthcare resource utilization (HCRU), and healthcare costs. This study assessed HCRU and healthcare costs among patien...
AIMS: Obstructive hypertrophic cardiomyopathy (oHCM) is associated with substantial disease burden, healthcare resource utilization (HCRU), and healthcare costs. This study assessed HCRU and healthcare costs among patients with oHCM treated in real-world settings over 5 years of follow-up. METHODS: This retrospective cohort study used the Optum database (January 2013-December 2021) and included patients aged ≥18 years with ≥2 claims for oHCM and continuous health plan enrollment for ≥6 months pre-initial and post-initial oHCM claim (index date). Patients with ≥5 years of follow-up data were analyzed. All-cause and HCM-related HCRU (ambulatory visits [physician office visits and outpatient visits], emergency room [ER] visits, hospital admissions, length of stay [LOS], and pharmacy use) and healthcare costs were assessed in US dollars. RESULTS: In total, 5,129 patients with oHCM were identified: 5,056 (98.6%) had an all-cause ambulatory visit and 4,669 (91.0%) had an HCM-related visit. 4,079 (79.5%) had an all-cause ER visit, and 1,499 (29.2%) an HCM-related ER visit. 2,949 (57.5%) reported an all-cause admission, and 2,232 (43.5%) an HCM-related admission. The mean (SD) per-person count of all-cause ambulatory visits was 101.0 (90.7) and 15.3 (18.7) for HCM-related ambulatory visits. Mean (SD) all-cause admissions per patient were 1.8 (3.8), and 0.9 (1.6) were HCM related. Mean (SD) all-cause LOS was 18.0 (54.7) days, and HCM-related LOS was 10.0 (39.6) days. Mean (SD) all-cause healthcare cost per patient was $181,968 (241,608), of which $67,531 (114,705) was HCM related. Hospital admissions were the largest proportion of medical costs: all-cause admissions cost $71,001 (149,533) during follow-up, of which $44,781 (99,431) was HCM-related. LIMITATIONS: Analyses were unadjusted for covariates or confounding factors. CONCLUSION: Patients with oHCM experience substantial HCRU and costs over 5 years of follow-up. These results indicate a need for new treatment options which could decrease HCRU and improve patient outcomes.
The estimated long-term clinical effects of BRCA testing and olaparib treatment of early breast cancer in the US population: a population impact model.
Kasle A, Li Q, Tung A … +2 more , Xu X, Veenstra D
J Med Econ · 2025 Dec · PMID 41185933 · Publisher ↗
AIMS: Olaparib is approved for the treatment of germline BRCA mutant (gBRCAm) high-risk early breast cancer (eBC) following treatment with neoadjuvant or adjuvant chemotherapy. The potential long-term outcomes of widespr...
AIMS: Olaparib is approved for the treatment of germline BRCA mutant (gBRCAm) high-risk early breast cancer (eBC) following treatment with neoadjuvant or adjuvant chemotherapy. The potential long-term outcomes of widespread germline BRCA testing and treatment with olaparib in the US have not been quantified. METHODS: We developed a decision-analytic model comparing a scenario with BRCA testing and olaparib treatment to a scenario with no testing and no treatment in an olaparib-eligible population. Olaparib-eligible population estimates were derived from published literature; long-term treatment outcomes were based on a published cost-effectiveness analysis. Breast cancer recurrences and life-years were projected over a lifetime. Scenario analyses were conducted to test different high-risk and testing uptake assumptions. RESULTS: We estimated that 3,983 eBC patients in the US were eligible for olaparib in 2024. Compared with no testing and no treatment, testing and olaparib treatment resulted in 272 (22% reduction) and 68 (22% reduction) fewer metastatic breast cancer (mBC) and non-metastatic breast cancer (non-mBC) recurrences, respectively, over a lifetime. A 9% increase in life expectancy would be achieved from adopting testing and olaparib treatment. Over 10 years, we estimated 40,094 olaparib-eligible eBC patients. 2,496 mBC (22% reduction) and 618 non-mBC recurrences (22% reduction) would be prevented, and 78,672 life-years would be saved vs no testing. Scenario analyses with different high-risk definitions and testing assumptions demonstrated a maintained clinical benefit (range of 82 - 552 mBC and 20 - 137 non-mBC recurrences avoided over a lifetime). LIMITATIONS: Results were based on long-term outcomes modeled on the results of the OlympiA clinical trial; these uncertainties were evaluated using sensitivity analyses. CONCLUSION: BRCA testing and subsequent treatment with olaparib results in fewer recurrences in eBC and a longer life expectancy vs no testing and no treatment, suggesting there is substantial clinical value in widespread BRCA testing for this population.
Healthcare resource utilization and costs in patients with multiple myeloma who received 1 to 3 prior lines of therapy, including a proteasome inhibitor and an immunomodulatory drug, and were exposed to and discontinued lenalidomide in the United States.
Jagannath S, Kharat A, Chinaeke E … +4 more , Fu AZ, Perciavalle M, Huo S, Qureshi ZP
J Med Econ · 2025 Dec · PMID 41178750 · Publisher ↗
BACKGROUND: Patients with refractory multiple myeloma (MM) often progress through lines of therapy (LOTs) comprising multiple drug classes, which may impose severe economic burden. In this retrospective US claims databas...
BACKGROUND: Patients with refractory multiple myeloma (MM) often progress through lines of therapy (LOTs) comprising multiple drug classes, which may impose severe economic burden. In this retrospective US claims database study, we examined healthcare resource utilization (HCRU) and costs of patients with MM who received 1 to 3 prior LOTs. PATIENTS AND METHODS: Adults with MM from the IBM Truven MarketScan Claims Database (1 January 2011-22 April 2023) were required to be continuously enrolled in a medical benefit/pharmacy plan for ≥12 months before initial MM diagnosis date and to have received 1 to 3 LOTs (including receiving ≥1 proteasome inhibitor and immunomodulatory drug and receiving and discontinuing lenalidomide) after diagnosis date. Index dates (start of subsequent treatment after fulfilling inclusion criteria) occurred after 1 January 2018, to capture contemporary cost estimates. Primary outcomes included all-cause and MM-related healthcare costs and HCRU after index date. RESULTS: The primary analysis included 338 patients with MM without post-index stem cell transplant (SCT), with a mean age of 61.1 years (55.3% male). During an average follow-up of 11.5 months, total all-cause healthcare costs averaged US $41,614 per patient per month. MM-related healthcare costs ($39,699) contributed 95% to total all-cause costs. Most MM-related monthly costs were attributed to drug/infusion costs (71%; $28,144). Sensitivity analyses that included patients with post-index SCT ( = 520) yielded similar results. CONCLUSIONS: Patients with MM with 1 to 3 prior LOTs experienced high economic burden largely attributable to MM-related treatment, highlighting the need for more cost-effective therapies.
Economic evaluation of a bivalent respiratory syncytial virus prefusion F vaccine for older adults in Sweden: cost-effectiveness and budget impact.
Szilcz M, Naghipour P, Fridh AC … +2 more , Palmborg A, Toghanian S
J Med Econ · 2025 Dec · PMID 41137859 · Publisher ↗
AIMS: Respiratory syncytial virus (RSV) is a major cause of lower respiratory tract infections among older adults, leading to hospitalizations, higher healthcare costs, and mortality. In Sweden, the bivalent RSV prefusio...
AIMS: Respiratory syncytial virus (RSV) is a major cause of lower respiratory tract infections among older adults, leading to hospitalizations, higher healthcare costs, and mortality. In Sweden, the bivalent RSV prefusion F (RSVpreF) vaccine was approved in August 2023 for individuals aged 60 years and older, prompting a need for a health economic evaluation. We estimated the cost-effectiveness and budget impact of vaccinating older adults with RSVpreF versus no vaccination in Sweden. METHODS: We implemented a population-based, multicohort Markov model using a lifetime horizon. Costs included direct medical care, vaccination and its administration. Rates of medically attended RSV-related lower respiratory tract infection, case-fatality rates and vaccine effectiveness were estimated by age group, comorbidity profile, and care setting. Adults were characterized based on age (60-74, 75+ years) and comorbidity profile (high risk vs. low risk) and a 75% vaccine uptake was assumed in the base case. We calculated budget impact for a five-year time horizon. RESULTS: The cost-effectiveness model projected fewer RSV-related hospitalizations (-12,9%), outpatient visits (-7,5%), and deaths (-11,3%) with RSVpreF vaccination among high-risk adults aged 60-74 and all adults ≥75 years compared to no intervention, with most benefits within the first 4 years (46,3%, 26,6%, and 46,2% averted, respectively). These reductions lowered direct healthcare costs (-2,8%) and increased QALYs over a lifetime, making RSVpreF cost saving compared to no intervention. In the budget impact analysis, initial investment costs were offset by direct medical savings in year 3, yielding ≈1,76 billion Swedish kronor (approximately 187 million USD) in net savings over five years. CONCLUSION: Introducing RSVpreF vaccination for older adults in Sweden has the potential to reduce RSV-related hospitalizations, deaths, and healthcare costs. Findings support the implementation of RSV vaccination to older adults and at-risk individuals as a cost-effective and budget-saving strategy in the short term.
Nirmatrelvir/ritonavir treatment for COVID-19: an economic value systematic literature review.
Mugwagwa T, Marcano Belisario J, Hartley L … +1 more , Phan NTN
J Med Econ · 2025 Dec · PMID 41134222 · Publisher ↗
AIMS: Nirmatrelvir/ritonavir (NMV/r) is an antiviral drug used to treat mild-to-moderate coronavirus disease 2019 (COVID-19) in patients at high risk of progression to severe COVID-19. We conducted an economic systematic...
AIMS: Nirmatrelvir/ritonavir (NMV/r) is an antiviral drug used to treat mild-to-moderate coronavirus disease 2019 (COVID-19) in patients at high risk of progression to severe COVID-19. We conducted an economic systematic literature review to assess key parameters, structures, and outcomes of existing models and analyses of the economic value of NMV/r treatment. METHODS: A systematic search of Embase, MEDLINE, and MEDLINE In-Process, as well as bibliographies of systematic reviews and conference and health technology assessment websites, identified relevant articles published between 2022 and 2024. A quality assessment was conducted for each economic evaluation using the Drummond checklist. RESULTS: Of the 22 included economic evaluations, most were cost-utility analyses ( = 10) and cost-effectiveness analyses ( = 9). Most used a short-term decision tree with a long-term Markov model ( = 5), with time horizons between 28 days to a lifetime, and were from a healthcare system perspective ( = 7). Most conducted scenario analyses ( = 15) and/or sensitivity analyses ( = 18), and many based treatment effectiveness estimates on the Evaluation of Protease Inhibition for COVID-19 in High-Risk Patients (EPIC-HR) randomized controlled trial ( = 7). Overall, NMV/r showed economic value across different willingness-to-pay thresholds when compared with standard of care or best supportive care, and all economic evaluations were high or moderate quality. LIMITATIONS: Most included economic evaluations were from high-income countries and all were written in English and may not be generalizable to low-income countries or healthcare systems. CONCLUSION: While economic evaluations were heterogeneous in terms of modeling approach, population, treatments, and context, these findings indicate that NMV/r has economic value for patients with mild-to-moderate COVID-19 with a high risk of progression to severe disease. Significant changes to the burden of COVID-19 due to the evolution of COVID-19 variants and new vaccination strategies, may warrant updates models.
Prostate cancer progression: a scoping review, pharmacoeconomic assessment, and evaluation of quality of life.
Dinis de Sousa R, Zagalo DM, Gouveia M … +7 more , Gomes L, Santos Dias J, Mansinho A, Santos S, Mariano J, Canhão H, Rodrigues A
J Med Econ · 2025 Dec · PMID 41128722 · Publisher ↗
AIMS/BACKGROUND: Prostate cancer is the most common malignancy in men and a leading cause of cancer-related death. Progression from non-metastatic castration-resistant prostate cancer (nmCRPC) to metastatic CRPC (mCRPC)...
AIMS/BACKGROUND: Prostate cancer is the most common malignancy in men and a leading cause of cancer-related death. Progression from non-metastatic castration-resistant prostate cancer (nmCRPC) to metastatic CRPC (mCRPC) significantly worsens health-related quality of life (HRQoL), increases mortality, and raises healthcare costs. This study assessed the impact of avoiding or delaying progression to mCRPC on HRQoL, mortality, and economic outcomes, incorporating patients' lived experiences and unmet needs. METHODS: Three complementary studies were conducted. Study 1 was a scoping review of HRQoL and functional outcomes across disease stages, analyzing 56 studies (27 RCTs, 29 observational). Study 2 used a pharmacoeconomic survival-partition model of apalutamide, calibrated for the Portuguese healthcare system, to estimate utility gains, mortality impacts, and healthcare costs associated with delaying progression (excluding drug costs). Study 3 comprised two virtual focus groups ( = 5) exploring patient experiences, including symptom burden, psychological impact, daily life disruption, coping strategies, and care-related unmet needs. RESULTS: High-risk nmCRPC patients had higher HRQoL and better function than mCRPC patients. Symptomatic mCRPC had the lowest HRQoL (EQ-5D 0.63-0.90 vs 0.85-0.86; FACT-P 93-123 vs 109-121). Delaying progression yielded an estimated utility gain of 0.192, reduced annual mortality (0.1% vs 19.1%), and 4.4-fold lower healthcare costs. Focus groups confirmed greater physical symptoms, emotional distress, and social disruption in mCRPC, while nmCRPC experiences centered on monitoring and uncertainty. Patients identified gaps in supportive care, including psychosocial, sexual, and functional needs. CONCLUSIONS: Delaying progression from nmCRPC to mCRPC confers substantial HRQoL, survival, and economic benefits. Patient perspectives highlight gaps in supportive care and the value of early targeted interventions. LIMITATIONS: Small qualitative sample, reliance on baseline HRQoL without longitudinal adjustment, heterogeneity across studies, and exclusion of nmCRPC treatment costs may limit generalizability and precision.
Cost-utility analysis of an exclusive human milk diet for very low birthweight infants in Japan.
Igarashi A, Reyes SM, Mizuno K
J Med Econ · 2025 Dec · PMID 41117636 · Publisher ↗
OBJECTIVE: To assess the cost-utility of an exclusive human milk diet (EHMD) compared to conventional nutrition for very low birthweight (VLBW, <1500 g) infants from Japan's healthcare payer perspective. METHODS: We cond...
OBJECTIVE: To assess the cost-utility of an exclusive human milk diet (EHMD) compared to conventional nutrition for very low birthweight (VLBW, <1500 g) infants from Japan's healthcare payer perspective. METHODS: We conducted a cost-utility analysis using used a Markov state-transition model built with TreeAge. Effectiveness was assessed using Quality-Adjusted Life Years (QALYs). The primary outcome was the incremental cost-effectiveness ratio (ICER) expressed as cost per QALY gained. Using 2024 prices, EHMD cost was JPY 2,000,000 (USD 13,220), varying up to JPY 4,000,000 (USD 26,440) in sensitivity analyses. Deterministic and probabilistic sensitivity analyses were performed. RESULTS: Base case analysis showed an ICER of JPY 2,949,278 (USD 19,495) per QALY gained, well below the hypothetical willingness-to-pay threshold value of JPY5,000,000 to JPY7,500,000 (USD 33,050 to USD 49,575). Sensitivity analysis indicated EHMD remained cost-effective up to JPY 4,750,000 (USD 31,398) per course against a revised JPY 7,500,000 (USD 49,575) per QALY threshold. CONCLUSION: This cost-utility analysis demonstrated that an EHMD may be a cost-effective option for VLBW infants in Japan at 2024 costs, and provides evidence to inform decision makers considering its adoption.
Toward an ethical future for orphan drugs: balancing access, affordability, and innovation.
Alum EU, Ekpang Ii JE, Ekpang PO … +4 more , Ainebyoona C, Nwagu KE, Nwuruku OA, Muhammad K
J Med Econ · 2025 Dec · PMID 41117352 · Publisher ↗
BACKGROUND/OBJECTIVES: Orphan drugs (ODs) provide transformative treatments for rare diseases but raise ethical and policy challenges due to high costs and unequal access. This review explores these dilemmas (balancing a...
BACKGROUND/OBJECTIVES: Orphan drugs (ODs) provide transformative treatments for rare diseases but raise ethical and policy challenges due to high costs and unequal access. This review explores these dilemmas (balancing access, affordability, and innovation across income settings), and proposes practical, actionable strategies to translate ethical principles into implementable solutions. METHODS: This narrative review and policy analysis explores global OD frameworks, emphasizing regulatory definitions, incentives, health technology assessment (HTA) practices, pricing, reimbursement, and delivery-system needs. It integrates policy tools, payer models, and real-world cases, alongside an ethical evaluation grounded in justice, beneficence, and equity, with a focus on LMICs. The analysis is based on peer-reviewed English-language studies from Scopus, PubMed, and Web of Science. RESULTS: We identify persistent inequities despite innovation: <5% of ∼7,000 rare diseases have approved therapies; high prices limit uptake, especially in LMICs. Structural barriers span affordability, diagnostics capacity, and fragmented governance. The review outlines an actionable package: (i) context-sensitive value-based tiered pricing blended with outcomes-based agreements; (ii) regional pooled procurement to aggregate demand and bargaining power; (iii) transparency HTA benchmarks to guide negotiations; (iv) inclusivity enablers: registries, ORPHA/ICD coding, decentralized/hybrid trials; and (v) delivery models (in-country build-out, regional hubs, or cross-border referral) tied to readiness criteria. LIMITATIONS: As a narrative synthesis drawing from secondary sources across varied country contexts, the findings lack quantitative generalizability. Emerging proposals like solidarity funds remain supported by limited prospective data, and their feasibility depends on local legal, fiscal, and health-system capacities. CONCLUSION: An ethical, sustainable OD ecosystem is achievable by pairing innovation incentives with equity-first operations. Priority next steps are to (i) expand outcomes-linked access and pooled-procurement pilots for high-cost therapies, (ii) accelerate rare diseases data visibility through ORPHA-code adoption, and (iii) embed patient co-leadership across research and policy so that geography and income no longer determine access to life-saving treatments.
Cost-effectiveness of non-statin lipid-lowering therapies as an add-on to statins for achieving low-density lipoprotein cholesterol therapeutic targets in very high-risk patients in the Spanish setting.
Climente-Martí M, García-González X, Torres-Bondia FI … +2 more , Lozano J, Gómez-Navarro V
J Med Econ · 2025 Dec · PMID 41111438 · Publisher ↗
AIMS: Our study aimed to estimate the cost-effectiveness of non-statin lipid-lowering therapies (LLTs) in patients with very-high cardiovascular risk, based on their relative efficacy and current yearly treatment costs i...
AIMS: Our study aimed to estimate the cost-effectiveness of non-statin lipid-lowering therapies (LLTs) in patients with very-high cardiovascular risk, based on their relative efficacy and current yearly treatment costs in the Spanish setting. MATERIALS AND METHODS: We generated a cohort of patients in secondary prevention with low-density lipoprotein cholesterol (LDL-C) levels >100 mg/dL using a Monte Carlo simulation. Based on the relative LDL-C reductions described in the literature for each studied non-statin LLTs, we estimated the percentages of cohort patients that would achieve the 2019/2025 European Society of Cardiology (ESC) and European Atherosclerosis Society (EAS) dyslipidemia guidelines treatment targets (LDL-C levels <55 mg/dL and ≥50% reduction from baseline; defined as effectively treated patients). Derived, the annual costs per effectively treated patient were calculated. RESULTS: Evolocumab 140 mg every two weeks (Q2W), followed by alirocumab 150 mg Q2W were modeled to be the most efficacious non-statin regimens, with 80% and 70% of effectively treated patients, respectively. The results for inclisiran and the other studied doses of alirocumab were modeled to be more modest (20% to 33%). The mean annual cost per patient effectively treated with evolocumab 140 mg Q2W was 6,200.1€, compared with 7,126.5€ for alirocumab 150 mg Q2W, 15,159.1€ with alirocumab 75 mg Q2W and 19,254.9€ with alirocumab 300 mg every month. Inclisiran resulted in higher costs both during the first year (31,329.1€) and the subsequent time scenarios (26,107.6€ and 22,974.7€ during average first two and five years, respectively). LIMITATIONS: No head-to-head clinical trials comparing non-statin LLTs are available. We only considered the published direct pharmacological costs in the Spanish setting. CONCLUSIONS: In our simulation study, evolocumab 140 mg Q2W resulted in similar (versus alirocumab 150 mg Q2W) or better cost-effectiveness in achieving 2019/2025 ESC/EAS LDL-C targets for secondary prevention patients compared to other LLTs.
Development and validation of an agent-based cost-effectiveness model of varicella vaccination strategies in Australia.
Hodgkinson B, Scuffham P
J Med Econ · 2025 Dec · PMID 41111311 · Publisher ↗
AIMS: Varicella zoster virus (VZV) is highly infectious and initially presents as varicella (chickenpox, CP); following recovery VZV poses a lifetime risk of re-emergence as herpes zoster (shingles, HZ). At the time of t...
AIMS: Varicella zoster virus (VZV) is highly infectious and initially presents as varicella (chickenpox, CP); following recovery VZV poses a lifetime risk of re-emergence as herpes zoster (shingles, HZ). At the time of this study, the Australian National Immunisation Program funded a single dose of VZV vaccine as MMRV (measles, mumps, rubella, varicella) at 18 months of age, and a HZ vaccine for persons aged 70 to 79 years. However, numerous studies have concluded that a two-dose VZV regimen during childhood may be more effective and that the optimal timing for HZ vaccination is uncertain. The aim of this paper was to describe the development and validation of an agent-based model designed to evaluate the cost-effectiveness of various varicella vaccination strategies in Australia. METHODS: During calibration, model parameter values were similar to an earlier published model, with strong agreement between modelled and published CP seropositivity and HZ incidence. Modelling an unvaccinated population, the model displayed a steady state incidence of CP and HZ, with the associated hospitalisations, deaths, and costs in alignment with published values. In the vaccination scenario, the model predicted the marked reduction in CP incidence and characteristic initial increase in HZ reported in published studies. RESULTS: During calibration, model parameter values were similar to an earlier published model, with strong agreement between modelled and published CP seropositivity and HZ incidence. Modelling an unvaccinated population, the model displayed a steady state incidence of CP and HZ, with the associated hospitalisations, deaths, and costs in alignment with published values. In the vaccination scenario, the model predicted the marked reduction in CP incidence and characteristic intial increase in HZ reported in published studies. CONCLUSIONS: This agent-based model can be used to provide cost-effectiveness estimates for various VZV vaccination strategies in an Australian context.
The cost-effectiveness of subcutaneous semaglutide 2.4 mg in the management of people living with obesity and prediabetes in England.
Ramos M, Larsen S, Fusco F … +2 more , Lamotte M, Capehorn M
J Med Econ · 2025 Dec · PMID 41090945 · Publisher ↗
BACKGROUND: Prediabetes and type 2 diabetes (T2D) are more prevalent amongst people living with obesity (PwO). T2D and obesity increase the risk of cardiovascular disease. In the STEP clinical development program, once-w...
BACKGROUND: Prediabetes and type 2 diabetes (T2D) are more prevalent amongst people living with obesity (PwO). T2D and obesity increase the risk of cardiovascular disease. In the STEP clinical development program, once-weekly subcutaneous semaglutide (2.4 mg) reduced body weight by 10-15% and reversed prediabetes in about 80% of patients. The aim of this study was to assess the cost-effectiveness of semaglutide plus diet and exercise (D&E) compared with D&E alone in PwO and prediabetes in England based on the STEP-10 trial. METHODS: The published Core Obesity Model was populated with baseline characteristics and treatment efficacy from STEP-10. This study assessed the reversal of prediabetes at 52 weeks. Baseline age and body mass index (BMI) were 53 (SD = 11) years and 40.1 (6.9) kg/m. At 52 weeks, semaglutide plus D&E and D&E alone reversed prediabetes in 80% and 12% of patients, and decreased BMI by 13.9% and 2.7%, respectively. The perspective of the NHS was considered using annual discounting rates of 3.5% on costs and outcomes. Several subgroups were assessed: Different baseline BMI, number of comorbidities, proportions of prediabetes and different treatment durations. Individuals losing ≥ 5% of body weight after 20 weeks (responders) were analysed separately. RESULTS: The treatment duration in the base case analysis was 20 years. The use of semaglutide resulted in 1.105 QALY gained at an additional cost of 19,391 GBP. The resulting ICER was 17,547 GBP/QALY gained. Treatment with semaglutide extended life expectancy by 1.68 years. Ranging treatment duration between 1 year as per STEP 10 trial and lifelong resulted in an ICER of 7,152 to 17,695 GBP/QALY. Results did not differ significantly by subgroup. The probabilistic sensitivity analysis indicated a 90% chance of semaglutide being cost-effective at a willingness-to-pay threshold of 20,000 GBP/QALY. CONCLUSION: Semaglutide is a cost-effective treatment option for PwO and prediabetes in England based on the results of the STEP-10 trial.
Cost-consequence analysis of three cardiac ablation technologies in paroxysmal atrial fibrillation.
Neužil P, Ferrero de Loma-Osorio A, Martínez Brotons Á … +6 more , Bondanza Saavedra L, Mei DA, Moučka P, Uffenorde S, Vieira Ruiz J, Boriani G
J Med Econ · 2025 Dec · PMID 41090941 · Publisher ↗
BACKGROUND AND AIM: The rapid evolution of catheter ablation technologies has introduced variability in clinical outcomes, procedural efficiency, and costs. This study aimed to evaluate the economic costs and clinical ou...
BACKGROUND AND AIM: The rapid evolution of catheter ablation technologies has introduced variability in clinical outcomes, procedural efficiency, and costs. This study aimed to evaluate the economic costs and clinical outcomes associated with radiofrequency ablation (RFA), cryoablation (CRYO), and pulsed field ablation (PFA) for the treatment of paroxysmal atrial fibrillation (AF). METHODS: A cost-consequence analytical model was developed to assess the economic impact and clinical outcomes of three treatment alternatives for adult patients with paroxysmal AF, from the hospital's perspective, in the short (index hospitalization) and medium-term (1 year). Real-world data were collected across three European specialty centers (Czech Republic, Italy, and Spain). The collected data captured procedural durations (including pre-procedural, skin-to-skin, and post-procedural phases), resource consumption, and staff workload. Costs were retrieved from institutional economic databases and published cost repositories. Costs were expressed in Euro (2025). Medium-term outcomes (complications, reinterventions, hospitalizations, cardioversions) were sourced from literature. RESULTS: A total of = 270 patients were included in the analysis. PFA was associated with consistency and predictable procedure duration compared to the other treatment alternatives. This efficiency may support increased capacity within the healthcare systems. PFA demonstrated cost saving of 10% compared to CRYO and 22% compared to RFA procedures, primarily driven by procedure time. Additionally, PFA showed a cost per responder of €2,406, versus €2,873 for CRYO (+19%) and €3,436 for RFA (+43%), reflecting both lower procedural costs and superior clinical outcomes. CONCLUSION: These findings suggest that PFA technology may offer economic and operational advantages, including more efficient resource utilization, reduced procedural complexity and consumables use, compared to traditional ablation modalities. However, variations in hospital clinical practices may limit the generalizability of results.
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