Cost Of Therapy Research Articles

Oral pathologies in anticancer therapy: a literature review

Anticancer therapy is associated with a wide range of systemic symptoms, including oral pathologies, such as dry mouth, mucositis, masticatory spasm, osteoradionecrosis, dental caries, opportunistic infections, dysphagia, hypogeusia, dysgeusia, and hyposalivation. Dental professionals are the most qualified experts in the diagnosis and treatment of oral pathologies, as well as their prevention and control in cancer patients receiving radiation therapy or chemotherapy. The aim of the review was to assess domestic and international literature on primary oral complications associated with anticancer therapy. The review also aimed to highlight the importance of dental care in improving the quality of life of these patients, as well as to provide practical guidelines for managing these complications. The search was performed using electronic databases PubMed, eLIBRARY.RU, and Google Scholar. The search yielded 55 literature sources, which were included in the review. Anticancer therapy may result in significant oral pathologies, including mucositis, infections, hyposalivation, changes in taste, and pain. These symptoms can affect various functions of the oral cavity and oropharynx, causing dry mouth and difficulties chewing and swallowing, which impairs nutrition. Moreover, they can affect speech, oral hygiene, denture treatment, and appearance, all of which can be detrimental to oral health, as well as social and emotional wellbeing. These side effects may also reduce compliance with anticancer therapy, affecting treatment outcomes, overall health, and therapy costs. When examining cancer patients, a dental professional must consider the prescribed therapy, as well as potential long-term complications similar to those in patients receiving bisphosphonates. As a result, a cancer patient will continue to receive the necessary treatment, and any new problems will be identified early.

Cost-effectiveness of using granulocyte colony-stimulating factor in the treatment of uterine body leiomyosarcomas. A retrospective analysis

Background. The main approach in the treatment of patients with uterine leiomyosarcoma is surgery followed by chemotherapy, which is the basis of treatment. Febrile neutropenia (FN) is a life-threatening condition, so the use of granulocyte colony-stimulating factor (G-CSF) after each cycle of chemotherapy in this category of patients remains an integral part. Aim. To evaluate the pharmacoeconomic efficacy of the use of short and long-acting G-CSF forms in a round-the-clock inpatient facility (RIF). Materials and methods. A retrospective analysis of economic expenditures was performed at the Chelyabinsk Regional Clinical Center of Oncology and Nuclear Medicine in the RIF setting for the period from January 2018 to December 2023 for the treatment of 62 patients with high-grade uterine leiomyosarcomas of stage IB–IVA according to the classification of the International Federation of Gynecology and Obstetrics (2009). All patients underwent surgical treatment, complete hysterectomy with appendages, followed by antitumor drug therapy with a high risk of FN. In the economic cost analysis, the treatment regimens gemcitabine 900 mg/m2 on day 1 and day 8 + docetaxel 100 mg/m2 + empegfilgrastim (regimen code sh1207.1) and gemcitabine 900 mg/m2 on day 1 and day 8 + docetaxel 100 mg/m2 + filgrastim (regimen code sh1070) were compared. The costs of clinical blood tests and staying in RIF were also considered. Results. The stay in the RIF was 9 bed days with a long-acting G-CSF regimen and 15 days with a short-acting G-CSF regimen. When using a treatment regimen with filgrastim in 6 patients, the number of bed days increased to 18 due to persistent neutropenia. No FN events were reported. The calculations consisted of the cost of a bed day in the RIF (1,660 rubles) and purchasing of short- and long-acting G-CSF, without the cost of the main therapy. The costs of treating patients with short-acting G-CSF were comparable to those with long-acting G-CSF. Conclusion. The regimen with long-acting G-CSF is effective, easy to use, and cost-effective in RIF settings.

The socio-economic burden of migraine: yesterday and today

Migraine is the most frequent and costly type of headache. The analysis of the «cost of the disease» implies an assessment of direct and indirect costs expressed in monetary units. Drastic changes have recently occurred in approaches to the treatment of the disease, in connection with which there are large differences in the structure of the economic burden «yesterday and today». «Yesterday» knowledge about the pathophysiology of migraine was insufficient and various nonspecific agents were used to treat the disease, the antimigrenous effect of which was manifested as an additional one. The relatively low prices of these ineffective drugs for relieving attacks and preventing migraines were accompanied by high indirect costs due to temporary disability of patients, frequent time off from work, decreased labor productivity, family troubles, etc. Therefore, «yesterday»: the indirect costs associated with migraine therapy outweighed the direct costs. «Today» the cost of drugs for the prevention and pathogenetic agents for the relief of migraine attacks has become significantly higher and their costs are the main part of the costs of migraine therapy, However, the effectiveness of new pathogenetic agents for the treatment of migraines helps to reduce indirect costs and improve the quality of life. Awareness of the change in the ratio of direct and indirect costs, as well as the causes of this phenomenon, will increase patient compliance, correctly allocate costs and ultimately save personal and public financial resources.

Evaluation of the anticancer properties of the phytochemicals present in <em>Annona muricata</em>

Cancer is a complex disease characterized by the unrestrained proliferation and dissemination of abnormal cells. The issue continues to be a notable public health concern on a global scale, with millions of new cases and fatalities being reported annually. Despite significant advances in cancer treatment, it remains a major challenge due to the high cost and toxicity of current therapies. Phytochemicals naturally found in plants are crucial resources for developing novel drugs and potential cancer therapies. The clinical efficacy of these anti-cancer agents has been demonstrated against diverse neoplastic cell lines. Additional investigation in this field can result in improved cancer therapies. Among many other plants effective against cancer, this literature review explores the anti-cancer properties of Annona muricata, a plant commonly known as soursop, which is a tropical plant species native to Central and South America, the Caribbean, and West Africa known for its edible fruit which has some medicinal merits, but also some toxicological effects. Annona muricata has been traditionally used for medicinal purposes, including cancer treatment. Acetogenins, alkaloids, phenolic compounds, and other active compounds are only a few of the phytochemicals that this review cites as being present in A. muricata. The review also looks into A. muricata’s biological abilities to fight lung, liver, prostate, colon, pancreas, and breast cancers. The toxicity of A. muricata and possible adverse effects are also covered in the review. The review also looks at the usage of nanoparticles with A. muricata as a cancer treatment. Overall, this literature review provides valuable insights into the potential anti-cancer properties of A. muricata and its potential as a therapeutic agent in cancer treatment.

Optimizing CAR-T cell therapy for solid tumors: current challenges and potential strategies.

Chimeric antigen receptor (CAR)-T cell therapy demonstrates substantial efficacy in various hematological malignancies. However, its application in solid tumors is still limited. Clinical studies report suboptimal outcomes such as reduced cytotoxicity of CAR-T cells and tumor evasion, underscoring the need to address the challenges of sliding cytotoxicity in CAR-T cells. Despite improvements from fourth and next-generation CAR-T cells, new challenges include systemic toxicity from continuously secreted proteins, low productivity, and elevated costs. Recent research targets genetic modifications to boost killing potential, metabolic interventions to hinder tumor progression, and diverse combination strategies to enhance CAR-T cell therapy. Efforts to reduce the duration and cost of CAR-T cell therapy include developing allogenic and in-vivo approaches, promising significant future advancements. Concurrently, innovative technologies and platforms enhance the potential of CAR-T cell therapy to overcome limitations in treating solid tumors. This review explores strategies to optimize CAR-T cell therapies for solid tumors, focusing on enhancing cytotoxicity and overcoming application restrictions. We summarize recent advances in T cell subset selection, CAR-T structural modifications, infiltration enhancement, genetic and metabolic interventions, production optimization, and the integration of novel technologies, presenting therapeutic approaches that could improve CAR-T cell therapy's efficacy and applicability in solid tumors.

Economic analysis of implementing Systemic Coronary Risk Estimation (SCORE2) scale and long-term consequences

Objective: to assess the demand for pharmacotherapy in risk groups on adverse cardiovascular (CV) events identified during prophylactic medical examination of the adult population according to the SCORE2 (Systemic Coronary Risk Estimation) scale, which is being introduced into clinical practice.Material and methods. To analyze the economic consequences of including diagnostic examinations to assess the risk by SCORE and SCORE2 scales, a Markov model was developed, which described the course of lipid metabolism disorders in adult patients aged 40 to 69 years without concomitant diseases identified during prophylactic medical examination. The model cycle was 2 months (the average waiting time for response to therapy); the modeling horizon was 3 years; the calculated values were estimated for the period 2024–2026. The economic analysis of long-term consequences involved calculating the cost of pharmacotherapy and the costs associated with CV events at outpatient and inpatient stages of medical care. The costs of drug therapy were determined based on dosage regimens presented in instructions for medical use in Russia, and clinical guidelines.Results. According to the modeling results, in the period 2024–2026, the administration of pharmacotherapy to reduce the risks of CV events will enable to achieve 23,224 potentially saved lives through exclusively government funding or 23,605 through mixed sources of funding (government and citizens’ own funds). The cost of the analyzed pharmacotherapy upon the introduction of the SCORE2 scale will be from 50.18 billion rubles (with government funding) to 318.14 billion rubles. Concurrently, pharmacocorrection will provide a reduction in the costs of inpatient treatment and outpatient medical services to 4.1 billion rubles due to the achieving of intended low-density lipoprotein level, and 1.6 billion rubles due to the achieving of intended triglyceride levels.Conclusion. Using new SCORE2 scale may lead to changes in the practice of prescribing lipid-lowering prophylaxis of CV diseases, as well as to a sharp increase in the number of patients who may require earlier prescription of pharmacotherapy to prevent CV events.

Nutrition Modulation of Cardiotoxicity in Breast Cancer: A Scoping Review

Background/Objectives: Advancements in breast cancer therapeutics, such as anthracyclines, are improving cancer survival rates but can have side effects that limit their use. Cardiotoxicity, defined as damage to the heart caused by cancer therapeutics, is characterised by a significant reduction in left ventricular ejection fraction (LVEF) and symptoms of cardiac dysfunction. Multiple oral supplements exist with antioxidant and anti-inflammatory properties that have the potential to lower cardiotoxicity risk and ameliorate the complications associated with left ventricular dysfunction. In this review, we evaluate the current status of using nutritional interventions to modulate cardiotoxicity. Methods: We used specific keywords to search for articles that met our predetermined inclusion and exclusion criteria to review the evidence and provide insights for future research. Results: Seven studies were identified as eligible for this review: six focused on oral supplementation strategies in breast cancer patients undergoing chemotherapy, and one focused on nutritional counselling and adherence to the Mediterranean diet in breast cancer survivors’ post-treatment. There was a significantly attenuated reduction in LVEF in five studies that monitored cardiometabolic health, and there were significant improvements in blood serum levels of cardiac biomarkers across all studies. Conclusions: Current evidence suggests that appropriate nutritional interventions, alongside chemotherapy, can modulate the risk of cardiotoxic side effects. This highlights the potential of oral antioxidant supplementation and Mediterranean diet counselling to decrease tertiary cancer therapy costs associated with cardiovascular complications.

Significance of increased 18F-FDG uptake in determining pathological status in post-chemotherapy lymphoma cases involving head-neck region on PET-CT

BackgroundCurrently, all follow-up cases of lymphoma showing fluorodeoxyglucose (FDG) avid uptake on positron emission tomography/computed tomography (PET-CT) in head-neck region undergo biopsy for tissue confirmation of diagnosis. There are no consensus or practice guidelines in literature pertaining, whether to biopsy all such FDG avid lesions post-chemotherapy. Hence, this study was conducted to determine the significance of PET-CT (SUVmax 5.0 or above) in determining remission or pathological status in these patients. MethodsA retrospective cohort analysis conducted between July 2019 and May 2023 at our institute of 40 follow-up cases of lymphoma post-chemotherapy (Group A) showing FDG Uptake with SUVmax 5.0 or above on PET-CT in Waldeyer's Ring (WR) or neck, and 40 non- lymphoma cases (Group B) showing FDG Uptake with similar SUVmax in WR or neck. All cases in group A underwent tonsillectomy and results of histopathology were correlated with PET-CT findings. All cases in group B underwent thorough clinical correlation with respect to PET-CT findings. A Receiver Operating Characteristic (ROC) curve for SUVmax was plotted to statistically analyse this data. ResultsStatistical analysis of our observations by plotting a ROC curve for SUVmax revealed a sensitivity of 90% with 10% specificity and an insignificant p-value of 0.733. ConclusionWe propose that unnecessary biopsy in all cases with FDG avid uptake must not be encouraged unless accompanied by a suspicious holistic clinical picture of asymmetry, significant lymph node involvement with FDG avid uptake and counterpart finding on CT. A tissue confirmation in all such cases may result in unnecessary risk and increase the cost of therapy.

12404 Infertility Therapy Costs for Women Following Hysteroscopic Adhesiolysis (HA) Compared to Those With No and Other Previous Uterine Surgery

12404 Infertility Therapy Costs for Women Following Hysteroscopic Adhesiolysis (HA) Compared to Those With No and Other Previous Uterine Surgery

Health-related quality of life and associated factors among children with Transfusion-dependent β-thalassaemia: a cross-sectional study in Guangxi Province

BackgroundTransfusion-dependent β-thalassemia (TDT) is a severe inherited disorder. Without regular treatment, patients with TDT experience complications that can significantly shorten life expectancy and severely impact both their quality of life and that of their families. The condition has attracted significant attention in global health discussions. Due to the challenges of blood supply shortages, the high costs of iron chelation therapy, and hematopoietic stem cell transplantation (HSCT), TDT presents a serious health risk to patients and imposes a substantial burden on families and society. However, research on the health-related quality of life (HRQoL) of thalassemia patients in China remains limited. This study evaluated the factors affecting the HRQoL of these patients, with the goal of developing strategies to improve their quality of life.MethodsIn this cross-sectional study, children with TDT were recruited from five treatment centers in Guangxi, a province with a high prevalence of thalassemia in China. Structured questionnaires were employed to gather relevant data on sociodemographic variables, disease characteristics, treatments, and associated costs. The HRQoL was assessed using the Transfusion-Dependent Quality of Life (TranQoL) questionnaire, with a proxy version for patients aged 0–11 years and a child version for those aged 12–18 years.ResultsThe study included 418 participants, yielding an overall TranQoL score of 60.6 ± 16.3 among thalassemia patients. Multiple linear regression analysis revealed a negative correlation (P < 0.05) between overall TranQoL scores and several factors: increasing patient age, the presence of multiple thalassemia patients within a family, and undergoing HSCT. Conversely, adherence to regular treatment was positively correlated with higher TranQoL scores (P < 0.05).ConclusionThe study demonstrates that HRQoL among Chinese patients with TDT is at a low level. Age, treatment adherence, family support, and socioeconomic status were identified as key determinants influencing HRQoL. It is essential to further enhance and optimize health insurance policies and medical services to support comprehensive treatment strategies for these patients.

Malpractice litigation in multidisciplinary cancer care: Navigating medico-legal challenges and shared decision making in India

Healthcare professionals participating in multidisciplinary team (MDT) cancer meetings may not have a comprehensive understanding of their medicolegal responsibilities in current times. This article aims to delineate the principal medicolegal issues pertinent to multidisciplinary cancer care and offer recommendations for future implementation. Key concerns highlighted in this literature encompass patient consent and privacy during MDT meetings, professional liability, the formal expression of dissenting views and the duty of care. The analysis of existing literature prioritizes several recommendations for addressing these issues. Given the limited precedent available for formulating recommendations, this article identifies foundational evidence that can inform the practicing clinicians of these concerns in future MDT practices.Navigating decision-making processes in cancer care poses significant challenges for patients. This article seeks to elucidate the significance of shared decision making (SDM) within the Indian clinical context while pin pointing disparities compared to Western practices. Through a systematic search conducted in Medline and Google Scholar from 2000 to 2019, approximately 400 articles were screened, resulting in the selection of 43 relevant articles (5 from India and 38 from Western sources). The literature underscores a scarcity of information on shared decision making in India as compared to Western contexts, potentially leading to adverse physical, psychological and financial consequences reported by patients. According to one study, While Western data demonstrate extensive involvement of both patients and physicians in consensus-building for treatment decisions, such engagement in India is predominantly observed in tertiary care settings, academic institutions or cases with high therapy costs. Cultural beliefs and biases further influence patient engagement, while communication breakdowns correlate strongly with medicolegal malpractice litigations. Future research endeavours are warranted to explore strategies for integrating shared decision making into routine oncology practice in India. Physicians must actively involve patients or their immediate family members in decision-making processes to ensure a patient-centric approach, thereby mitigating the risks of un-informed decision-making or mistrust in the treating physician's expertise. Efforts such as physician and patient education, tool development, policy formulation, widespread implementation and periodic assessments hold promise for advancing the practice of shared decision making.

A cost-effectiveness analysis of stereotactic ablative radiotherapy versus conventionally fractionated radiotherapy in the management of stage 1 non-small-cell lung cancer: Results from the TROG 09.02 CHISEL study.

Stereotactic ablative radiotherapy (SABR) is a standard of care treatment for medically inoperable early-stage non-small-cell lung cancer (NSCLC). The CHISEL trial was a phase 3 randomised controlled trial that compared SABR to conventional radiation therapy (CRT). Using patient-level data, we compared the cost-effectiveness of SABR and CRT for early-stage NSCLC. Data on treatment exposure, outcomes (recurrence, survival) and quality of life (QoL; EORTC QLQ-C30) were sourced from the trial. Quality-adjusted life years (QALYs) were estimated for the trial period using Australian utility weights for the EORTC QLQ-C30-derived QLU-C10D. Costs related to simulation, planning, delivery, verification and post-treatment monitoring were estimated by applying Australian Medicare Benefits Schedule fees. The costs of post-progression therapy and grade ≥3 toxicity were estimated using trial data and relevant literature sources. Cost-effectiveness was investigated as the incremental cost per QALY gained for SABR compared to CRT. Complete QoL data were available for 21 patients: 14 in the SABR arm and 7 in the CRT arm. Mean QALYs discounted at 5% per annum were similar between arms: 12.68 months for SABR and 12.12 months for CRT. The mean costs of delivering SABR and CRT were $4763 and $6817, respectively. The costs of monitoring were similar in both arms, $4856 and $4853 for SABR and CRT. The mean costs of post-progression therapy were $24,572 for SABR and $42,801 for CRT. The mean costs of grade ≥3 toxicity were $809 in the SABR arm and $132 in the CRT arm. Therefore, the total mean cost for SABR over the period of interest was lower for SABR than CRT. Given lower mean costs and numerically higher QALYs for SABR compared with CRT, an incremental cost-effectiveness ratio was not calculated. Compared to CRT, SABR is a cost-effective treatment for early-stage NSCLC as the estimated upfront treatment cost and the cost of subsequent care are lower for SABR for comparable mean QALYs. Assessment of the lifetime QALYs and projections of cost estimation will provide a better indication of the long-term cost-effectiveness of SABR.

Redefining chimeric antigen receptor T-cell (CAR-T) regulation: China's responses to address secondary cancer risks of CAR-T therapy.

Since the United States Food and Drug Administration (FDA) approved the first chimeric antigen receptor T-cell (CAR-T) therapy in 2017, it has marked a major breakthrough in cancer treatment, leading to a surge in global research and applications in this field. In recent years, China has made rapid progress, quickly catching up through heavy investment in CAR-T construction, preparation processes, and treatment strategies. China's CAR-T therapy market is driven by substantial pharmaceutical investment targeting its vast population, yet high therapy costs remain uncovered by basic medical insurance. In November 2023, FDA issued a warning about the risk of secondary cancers in patients undergoing CAR-T therapy, sparking global concern. In fact, the China National Medical Products Administration (NMPA) preemptively implemented a series of measures to address the safety concerns of CAR-T therapy, emphasizing the risk of secondary cancers and advising lifelong monitoring as part of the approval process for CAR-T products. Nevertheless, additional regulatory measures are needed to address emerging risks, particularly the threat of secondary cancers. The authors believe that raising the standards for Investigational New Drug (IND) approval and establishing a dynamic reporting and feedback system based on real-world data will strengthen regulatory oversight and support the sustainable growth of the CAR-T industry in China.

Cost-Utility Analysis of PCSK9 Inhibitors and Quality of Life: A Two-Year Multicenter Non-Randomized Study.

The primary objective of this study was to conduct a cost-utility analysis of proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors in real-world, comparing their use with standard care for managing cardiovascular disease. A multicenter prospective study was conducted across 12 Spanish hospitals from May 2020 to April 2022, involving 158 patients with hypercholesterolemia or atherosclerotic cardiovascular disease. This study assessed health-related quality of life (QoL) using the EQ-5D-3L questionnaire. The cost-utility analysis evaluated the economic impact of PCSK9 inhibitors when used with standard care compared to standard care alone, calculating the incremental cost-effectiveness ratio (ICER). This study included 158 patients with an average age of 61 years, male (66.5%). For patients initiating PCSK9 inhibitors, the treatment cost was EUR 13,633.39, while standard therapy cost EUR 3638.25 over two years. QoL for PCSK9 inhibitors stood at 1.6489 over two years, compared to 1.4548 for standard therapy. The results revealed favorable cost-utility outcomes, with an ICER of EUR 51,427.72. Significant improvements were observed in the domains of mobility, self-care, daily activities, pain/discomfort, and anxiety/depression (p < 0.001). This study presents the first real-world cost-utility analysis of PCSK9 inhibitors, supporting their economic rationale and highlighting their benefits in clinical practice. Healthcare decision-makers can use these results to inform their decisions and reimbursement policies concerning PCSK9 inhibitors. Trial Registration clinicaltrials.gov Identifier: NCT04319081.

The high costs of anticancer therapies in the USA: challenges, opportunities and progress.

The USA spent $99 billion on orally administered and clinician-administered anticancer therapies (excluding supportive care) in 2023 and spending is projected to increase to $180 billion by 2028. This increased spending on anticancer therapies largely reflects the high launch prices of novel therapeutics and increases in the prices of existing products, even in the absence of new evidence of clinical benefit or changes in use. Consequently, high prices have impeded Americans' access to and affordability of necessary anticancer therapies and thus increased their risk of cost-related non-adherence, cancer recurrence and mortality. To address the rising prices and concerns regarding Americans' spending on anticancer therapies, state and federal governments have, over the past decade, enacted legislation that caps out-of-pocket spending, expands subsidies and requires drug price negotiations. In this Perspective, we summarize US policies aimed to lower the costs of anticancer therapies, discuss the implications of such reforms and propose additional solutions needed to reduce costs and increase value.

Review of open libraries for pharmacoeconomic analysis in R environment

Background. To reveal the full complexity of the relationship between medical intervention and disease outcome, new methods of analysis and modeling are actively being developed, and tools are becoming more complex, for the use of which it is important to understand their limitations and advantages.Objective: conducting a critical review of the main open-source packages in R environment for conducting pharmacoeconomic analysis.Material and methods. The selection of libraries used for pharmacoeconomic analysis in the R environment was carried out based on the keywords “health economic”, “DALY”, “QALY” in the CRAN repository. Only libraries that were valid on the date of the review were included in the study. The selected 10 R software libraries for pharmacoeconomic analysis were reviewed from the standpoint of the number of tools they support, the format of the data used, the possibilities of visualizing results and generating reports, the presence of vignettes and the possibilities of parallelizing calculations.Results. The selected libraries can be divided into three classes: packages for calculating various quality of life indices, libraries for calculating indicators and indices of economic effectiveness of medical interventions (DALY, QALY, ICER), libraries for performing sensitivity analysis of the effect of medical interventions based on decision tree algorithms and Markov models. The libraries “heemod”, “hesim”, “rdesign” allow building simple Markov and semi-Markov models, but preference should be given to “heemod” due to the presence of vignettes. To conduct an analysis using cohort Markov models, partitioned survival models, it is recommended to use the “hesim” library, if there are gaps in the results, it is recommended to use “missingHE”. The “rdesigin” library allows building decision trees indicating the risk of developing certain conditions and the cost of therapy. The “survHE” library for survival analysis, used specifically in health economics, allows you to carry out probabilistic sensitivity analysis based on survival models. To calculate the survival models themselves to identify predictors of a patient's transition from one health state to another, you will need to additionally install the “flexsurv” library. To visualize the results of pharmacoeconomic modeling, you should additionally install the “diagram” and “ggplot2” libraries. Conclusion. The conducted critical review of open source libraries in R environment can serve as a navigator for choosing a tool for performing pharmacoeconomic analysis.

Cost-effectiveness Analysis of Tumor Treating Fields Therapy Combined With Immune Checkpoint Inhibitor in Metastatic Non-small-cell Lung Cancer

BackgroundThe LUNAR clinical trial revealed that incorporating Tumor Treating Fields (TTFields) therapy alongside immune checkpoint inhibitor (ICI) significantly prolonged the overall survival of patients with metastatic, platinum-resistant non-small-cell lung cancer (NSCLC). However, the cost of TTFields therapy is high and may further increase the financial burden for patients. Our research aims to evaluate the cost-effectiveness of TTFields therapy addition with ICI for metastatic NSCLC. MethodsWe constructed a Markov model to evaluate the healthcare costs associated with TTFields therapy combined with ICI for the treatment of advanced NSCLC. In this model, the clinical data utilized came from the LUNAR trial, while drug costs and health state utility values were extracted from public databases and relevant scholarly publications. The major outcomes incorporated costs, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratio (ICER). ResultsCompared with ICI therapy alone, ICI combination with TTFields therapy resulted in 0.42 QALYs at the cost of $167,329, with an ICER of $398,402.38 per year. The calculated ICER surpassed the generally accepted US willingness-to-pay (WTP) threshold of 150,000 per QALY. One-way sensitivity analyses demonstrated that the utility of progression disease is the most influential factor, followed by the cost of TTFields therapy, the utility of progression-free survival, the cost of ICI, and the cost of adverse events in TTFields therapy combined with ICI. Only when the cost of TTFields therapy is reduced by approximately 80.48%, it would be cost-effective within the commonly accepted WTP threshold of $150,000/QALY. ConclusionsAccording to the US WTP, the combination of TTFields therapy with ICI does not currently represent a cost-effective strategy for metastatic NSCLC followed progression on platinum-resistant therapy. Considering its promising clinical outcomes for metastatic NSCLC, it is necessary to control the expenses of this therapeutic strategy in future applications.

Predicted Expenditure for Prescription Drugs for Multiple Sclerosis in the Italian Market Between 2023 and 2028: Results of the Oracle Project.

Multiple sclerosis (MS) is a chronic neurodegenerative disease that leads to impaired cognitive function and accumulation of disability, with significant socioeconomic burden. Serious unmet need in the context of managing MS has given rise to ongoing research efforts, leading to the launch of new drugs planned for the near future, and subsequent concerns about the sustainability of healthcare systems. This study assessed the changes in the Italian MS market and their impact on the expenditures of the Italian National Healthcare Service between 2023 and 2028. A horizon-scanning model was developed to estimate annual expenditure from 2023 to 2028. Annual expenditure for MS was calculated by combining the number of patients treated with each product (clinical inputs) and the yearly costs of therapy (economic inputs). Baseline inputs (2020-2022) were collected from IQVIA® real-world data, while input estimation for the 5-year forecast was integrated with analog analyses and the insights of clinicians and former payers. The number of equivalent patients treated in 2028 in Italy was estimated at around 67,000, with an increase of 10% versus 2022. In terms of treatment pattern evolution, first-line treatments are expected to reduce their shares from 47% in 2022 to 27% in 2028, and Bruton tyrosine kinase inhibitors are expected to reach 23% of patient shares. Overall, expenditure for MS is estimated to decrease from €721 million in 2022 to €551 million in 2028, mainly due to losses of exclusivity and renegotiation of drug prices. Despite the increase in the number of patients treated for MS and the launch of new molecules that will reach high market penetration, the model confirmed sustainability for the Italian National Healthcare Service.

A systematic review of cost-effectiveness analyses of gene therapy for hemophilia type A and B.

In 2022-2023, the US Food and Drug Administration approved 2 novel gene therapies, valoctocogene roxaparvovec and etranacogene dezaparavovec, for hemophilia A and B, respectively. These one-time-administered gene therapies have been marketed at prices that create financial challenges for payers and patients. Understanding the magnitude and uncertainties around the long-term value of these therapies and how they can potentially relate to managed care practices is of high interest to the payer and patient community. To conduct a systematic review of cost-effectiveness analysis (CEA) studies to assess (1) the long-term value of valoctocogene roxaparvovec and etranacogene dezaparavovec and (2) the relevance and validity of the underlying data and assumptions used in the CEA models and discuss how they relate to the challenges identified for CEAs of gene therapies. A systematic review of cost-effectiveness studies of novel hemophilia A and B gene therapy was conducted. PubMed and Embase were searched for published studies from inception to January 12, 2024. Original research articles published in English that conducted a CEA on gene therapy treatments for hemophilia A and B, with a comparison of incremental costs and health effects, were considered. Critical appraisal of the quality of reporting and the underlying modeling assumptions were conducted to assess the relevance and validity of the results. Two hundred thirty-eight studies were identified, of which 4 met the inclusion criteria. Three studies were conducted from a US health care perspective and 1 from a Dutch societal perspective. Despite the high upfront costs of the gene therapies, all included studies' (3 hemophilia A and 1 hemophilia B) modeled results showed that gene therapies had lower overall costs and better health outcomes compared with factor concentrate replacement therapies and emicizumab. The results were driven by the assumption that gene therapies will have a durable effect of at least 10 years and offset the high cost of the current standard of care. The modeled health improvements varied substantially across studies, showing that the long-term value is sensitive to varying clinical and economic assumptions. The novel hemophilia gene therapy treatments can potentially be a cost-effective use of treatment resources if the treatment effects are durable over time. To reduce the risk for payers while still facilitating patient access, outcomes-based agreements similar to what has recently been proposed by the Centers for Medicare & Medicaid Services for sickle-cell therapies are well supported.

Tucaresol: A Clinical Stage Oral Candidate Drug with Two Distinct Antiviral Mechanisms

Globally, approximately 39 million people are living with Human Immunodeficiency Virus, HIV, arising from approximately 86 million infections since this epidemic began in 1981. However, the number of HIV infections is unevenly distributed with two thirds of global infections confined to Sub-Saharan Africa. Due to viral drug resistance, the most effective treatment requires a triple drug combination thereby adding to the complexity and cost of therapy. As such, many people living with HIV or at risk of infection do not have access to prevention or treatment of this potentially fatal disease. There is no cure for HIV [1]. Tucaresol is an orally active clinical stage drug which functions as a host targeted antiviral agent by protection or reconstitution of CD4+ T helper immune cells. We report herein that Tucaresol also displays in-vitro activity against HIV in infected human peripheral blood mononuclear cells. Although this in-vitro antiviral activity is not potent, the excellent safety profile and bioavailability of Tucaresol, along with its low Molecular Weight, support attainment of relevant drug concentrations in man to achieve significant in-vivo activity. This is demonstrated by previously reported stabilization of viremia in a prior proof of concept phase 1b/2a HIV clinical trial [2]. It is possible that the significant in-vivo activity of Tucaresol arises from synergy between co-stimulation of CD4+ T helper cells and the direct activity against virally infected cells. A pan in-vitro viral screen of Tucaresol further revealed a weak, direct antiviral activity against human herpes virus 6B, human papillomavirus 11, measles virus and hepatitis B virus.