Optimising oncology drug expenditure in Ireland.

A combination of improvements in patient survival, increasing treatment duration, and the development of more expensive agents has led to a doubling of per-capita spending on cancer medicines in Ireland (2008-2018). Despite this, access to new drugs is poor in comparison to other EU countries. We examine methods to optimise oncology drug spending to facilitate access to newer anticancer agents. Key targets for spending optimisation (biosimilar use, clinical trials and expanded access programs, waste reduction, avoidance of futile treatment, and altered drug scheduling) were identified through an exploratory analysis. A structured literature search was performed, with a focus on articles relevant to the Irish Healthcare system, supplemented by reports from statutory bodies. At the present time, EMA-approved agents are available once approved by the NCPE. Optimising drug costs occurs through guideline-based practice and biosimilar integration, the latter provides €80 million in cost savings annually. Access to novel therapies can occur via over 50 clinical trials and 28 currently available expanded access programmes. Additional strategies include reversion to weight-based immunotherapy dosing, potentially saving €400,000 per year in our centre alone, vial sharing, and optimisation of treatment schedules. A variety of techniques are being employed by oncologists to optimise costs and increase access to innovation for patients. Use of biosimilars, drug wastage, and prescribing at end of life should be audited as key performance indicators, which may lead to reflective practice on treatment planning. Such measures could further optimise oncology drug expenditure nationally facilitating approval of new agents.

Cost-efficiency and budget-neutral expanded access modeling of the novel PD-1 inhibitor toripalimab versus pembrolizumab in recurrent or metastatic nasopharyngeal carcinoma.

AIMS: To estimate, in the setting of recurrent or metastatic nasopharyngeal carcinoma (R/M NPC) for an assumed 1,207 incident US cases in 2024, (1) the cost-efficiency of a toripalimab-gemcitabine-cisplatin regimen compared to a similar pembrolizumab regimen; and (2) the budget-neutral expanded access to additional toripalimab cycles and regimens afforded by the accrued savings. METHODS: Simulation modeling utilized two cost inputs (wholesale acquisition cost (WAC) at market entry and an ex ante toripalimab price point of 80% of pembrolizumab average sales price (ASP)) and drug administration costs over 1 and 2 years of treatment with treatment rates ranging from 45% to 90%. In the absence of trial data for pembrolizumab-gemcitabine-cisplatin in R/M NPC, it is assumed that such a regimen would be comparable to toripalimab-gemcitabine-cisplatin in efficacy and safety. RESULTS: In the models utilizing the WAC, toripalimab saves $2,223 per patient per cycle and $40,014 over 1 year of treatment ($77,805 over 2 years). Extrapolated to the 1,207-patient panel, estimated 1-year savings range from $21,733,702 (45% treatment rate) to $43,467,404 (90% rate). Reallocating these savings permits budget-neutral expanded access to an additional 2,359 (45% rate) to 4,717 (90% rate) toripalimab maintenance cycles or to an additional 126 (45% rate) to 252 (90%) full 1-year toripalimab regimens with all agents. Two-year savings range from $42,259,976 (45% rate) to $84,519,952 (90% rate). Reallocating these efficiencies provides expanded access, ranging from an additional 4,586 (45% rate) to 9,172 (90% rate) toripalimab cycles or to an additional 128-257 full 2-year toripalimab regimens. The ex ante ASP model showed similar results. CONCLUSION: This simulation demonstrates that treatment with toripalimab generates savings that enable budget-neutral funding for up to an additional 252 regimens with toripalimab-gemcitabine-cisplatin for one full year, the equivalent of approximately 21% of the 2024 incident cases of R/M NPC in the US.

An estimated 1,207 patients will be diagnosed with late-stage nasopharyngeal cancer in the US in 2024. Toripalimab is a novel PD-1 inhibitor drug approved by the US Food and Drug Administration on October 27, 2023 as first-line treatment for patients with recurrent or metastatic nasopharyngeal cancer when used in combination with gemcitabine and cisplatin. We conducted economic evaluations of the costs of this toripalimab regimen versus the costs of a similar regimen with the PD-1 inhibitor pembrolizumab. Our simulation models used two pricing scenarios: the wholesale acquisition cost (WAC) or "list price" at market entry and, as no average sales price (ASP) will be available for toripalimab for several quarters, an estimated toripalimab price point of 80% of the pembrolizumab ASP. We compared the savings in each scenario when between 45% and 90% of the 1,207 patients are treated with the toripalimab regimen. We then evaluated how these savings could be re-allocated, on a budget-neutral basis and without requiring extra cash outlays, to provide more patients with access to toripalimab treatment; specifically, how many toripalimab doses and how many full toripalimab regimens could be purchased to provide more patients with treatment. We found that, if 90% of new cases of recurrent or metastatic nasopharyngeal cancer were treated with toripalimab over 1 year, these savings are enough to purchase up to 4,717 additional doses on a budget-neutral basis, which could provide up to an additional 252 newly diagnosed patients with 1 year of treatment with toripalimab. In combination with gemcitabine and cisplatin, toripalimab can markedly improve access to care for patients with recurrent or metastatic nasopharyngeal cancer in a cost-responsible way.

Finding value in novel antibiotics: How can infectious diseases adopt incremental cost-effectiveness to improve new antibiotic utilization?

Research and development of innovative antimicrobials is paramount to addressing the antimicrobial resistance threat. Although antimicrobial discovery and development has increased, difficulties have emerged in the pharmaceutical industry after market approval. In this minireview, we summarize clinical trial data on recently approved antibiotics, calculate incremental cost-effectiveness ratio (ICER) values, and explore ways to adapt ICER calculations to the limitations of antimicrobial clinical trial design. We provide a systematic review and analysis of randomized, controlled studies of antibiotics approved from 2014 - 2022 and extracted the relevant clinical data. Adapted-ICER (aICER) calculations were conducted using the primary condition-specific outcome that was reported in each study (percent mortality or percent cure rate). The literature search identified 18 studies for the 8 total antibiotics which met inclusion criteria and contained data required for aICER calculation. aICER values ranged from -$17,374 to $4,966 per percent mortality and -$43,931 to $2,529 per percent cure rate. With regards to mortality, ceftolozane/tazobactam and imipenem/cilastatin/relebactam proved cost efficacious, with aICER values of $4,965 per percent mortality and $1,955 per percent mortality respectively. Finding value in novel antibiotic agents is imperative to further justifying their development, and aICER values are the most common method of determining value in healthcare. The current outcomes of clinical trials are difficult to translate to aICER, which most effectively use Quality-Adjusted Life Years (QALY) as the quality standard in other fields such as oncology. Future antimicrobial trials should consider introducing methods of assessing measures of health gain such as QALY to better translate the value of novel antimicrobials in healthcare.

[Translated article] Cost of Treating Cutaneous T-Cell Lymphoma in Spain: Analysis of MICADOS Study Data by Disease Stage.

BACKGROUND AND OBJECTIVE: The cost of treating cutaneous T-cell lymphoma (CTCL) in Spain is unknown. With the advent of new treatments, it is more important than ever to gain an accurate picture of the true costs involved. The MICADOS study had 2 primary objectives: 1)to evaluate the impact of CTCL on patient quality of life, and 2)to evaluate the costs associated with the disease. This article reports the results of the cost analysis. METHODS: We estimated the cost of treating CTCL over a period of 1year from the perspective of the Spanish National Health System. Twenty-three dermatologists and hematologists from 15 public hospitals analyzed data for adult patients with mycosis fungoides (MF) or Sézary syndrome (SS). RESULTS: A total of 141 patients (57.4% male) with a mean age of 63.6 years (95%CI: 61.4-65.7 years) were included. The mean direct annual cost of treating CTCL was €34,214 per patient. The corresponding costs by stage were €11,952.47 for stageI disease, €23,506.21 for stageII disease, €38,771.81 for stageIII disease, and €72,748.84 for stageIV disease. The total direct annual cost of treating MF/SS in public hospitals in Spain was estimated at €78,301,171; stageI disease accounted for 81% of all costs, stageII for 7%, and stagesIII andIV for 6% each. CONCLUSIONS: The MICADOS study offers an accurate picture of the direct cost of treating CTCL in patients with MF/SS in Spain and shows that costs vary significantly according to disease stage. Patient-borne and indirect costs should be analyzed in future studies.

Cost-effectiveness analysis of epidural morphine/ropivacaine treatment in patients with cancer pain.

BACKGROUND: Cancer pain is one of the main causes of human suffering, which can generate disabilities and compromise quality of life, giving rise to several psychosocial and economic consequences. AIMS: This quantitative study sought to perform a cost-effectiveness pharmacoeconomic analysis to assess the impact of implanting epidural morphine associated with ropivacaine treatment in gastrointestinal cancer patients with pain that is difficult clinical control, compared with conventional oral treatment. MATERIALS AND METHODS: The study population consisted of 24 patients with gastrointestinal neoplasia who underwent treatment for cancer pain that was difficult to clinically control. 12 patients each were recruited into the control and intervention groups, respectively. While patients in the control group were administered drug treatment orally, patients in the intervention group underwent a surgical procedure for subcutaneous implantation of a catheter that allowed epidural administration of morphine and ropivacaine. For pain assessment, the Visual Analogue Scale was applied. Data analysis had a descriptive character of costs, taking into account the costs for the year 2021. The study perspective was the Brazilian public healthcare provider, referred to as the Unified Health System (Sistema Único de-SUS in Portuguese). Costs were computed over the time horizon corresponding to the duration of treatment, from the first medical consultation (when the treatment was defined) to the end (end of treatment, disease progression, or death). Treatment duration was divided into three phases (first 60 days, support with palliative care, and end-of-life care). To assess the robustness of the economic analysis, sensitivity analyses were performed, considering the effectiveness of pain reduction on the Visual Analogue Scale, and a comparison of results using the median prices of pharmaceutical components used in the study. RESULTS: The mean age of patients was 59.3 years. The results from the cost-effectiveness analysis showed the epidural morphine/ropivacaine treatment to be more effective with regard to pain reduction on the pain scale, particularly for end-of-life care, when compared to the conventional oral treatment, however, at a significantly higher cost. DISCUSSION: From the accomplishment of this research, it was observed that the application of the pain assessment scale is a way to better interpret and understand the patient's pain, facilitating care planning and decision-making by health professionals, as well as monitoring the effectiveness of the proposed new treatment. CONCLUSION: To present a better cost-effectiveness ratio, a reduction in the cost of the new epidural technology or an increase in the value of the existing oral intervention would be required. However, the latter is not feasible and unlikely to occur. A value judgement to decide whether the incremental benefit associated with the use of the new intervention is worth the extra cost will have to be made by the healthcare provider. Interventions that can relieve cancer pain symptoms should be investigated continuously, in search of evidence to support clinical practice and promote better quality of life for patients.

Cost of Treating Cutaneous T-Cell Lymphoma in Spain: Analysis of MICADOS Study Data by Disease Stage. / Evaluación de los costes asociados a la enfermedad de pacientes con linfoma cutáneo de células T en España: análisis en función del estadio clínico (estudio MICADOS).

BACKGROUND AND OBJECTIVE: The cost of treating cutaneous T-cell lymphoma (CTCL) in Spain is unknown. With the advent of new treatments, it is more important than ever to gain an accurate picture of the true costs involved. The MICADOS study had 2 primary objectives: 1)to evaluate the impact of CTCL on patient quality of life, and 2)to evaluate the costs associated with the disease. This article reports the results of the cost analysis. METHODS: We estimated the cost of treating CTCL over a period of 1year from the perspective of the Spanish National Health System. Twenty-three dermatologists and hematologists from 15 public hospitals analyzed data for adult patients with mycosis fungoides (MF) or Sézary syndrome (SS). RESULTS: A total of 141 patients (57.4% male) with a mean age of 63.6 years (95%CI: 61.4-65.7 years) were included. The mean direct annual cost of treating CTCL was €34,214 per patient. The corresponding costs by stage were €11,952.47 for stageI disease, €23,506.21 for stageII disease, €38,771.81 for stageIII disease, and €72,748.84 for stageIV disease. The total direct annual cost of treating MF/SS in public hospitals in Spain was estimated at €78,301,171; stageI disease accounted for 81% of all costs, stageII for 7%, and stagesIII andIV for 6% each. CONCLUSIONS: The MICADOS study offers an accurate picture of the direct cost of treating CTCL in patients with MF/SS in Spain and shows that costs vary significantly according to disease stage. Patient-borne and indirect costs should be analyzed in future studies.

Cost-effectiveness of adding serplulimab to first-line chemotherapy for extensive-stage small-cell lung cancer in China.

OBJECTIVE: The aim of the current study was to evaluate the cost-effectiveness of serplulimab plus chemotherapy compared chemotherapy alone as first-line strategy for patients with ES-SCLC in China. METHODS: A decision-analytic model that based on the Chinese health-care system perspective was conducted to evaluate the economic benefits for the two competing first-line treatment. The clinical survival and safety data were obtained from the ASTRUM-005 trial, cost and utility values were gathered from the local charges and previously published study. Both cost and utility values were discounted at an annual rate of 5%. Sensitivity analyses and subgroup analyses were performed to examine the robustness of the model results. RESULTS: Serplulimab plus chemotherapy could bring additional 0.25 QALYs with the marginal cost of $37,569.32, resulting in an ICER of $147,908.74 per additional QALY gained. Sensitivity analyses confirmed that model results were robust. Subgroup analyses revealed that adding serplulimab to first-line chemotherapy were unlikely to be the cost-effective option for all subgroup patients. CONCLUSIONS: Serplulimab plus chemotherapy was unlikely to be the cost-effective first-line strategy compared with chemotherapy alone for patients with ES-SCLC in China. Reduced the price of serplulimab could increase its cost-effective.

The Rate of Infusion Represents an Important Aspect of Administering Anticancer Agents.

Background: Infusion rate is one of the essential elements that should be included in all intravenous orders. Patients may experience adverse consequences or risks associated with inappropriate infusion. Meanwhile, there is growing pressure on the chemotherapy unit to deliver treatment quickly, efficiently, and safely, and thus it is very necessary to improve the chemotherapy process and service to cancer patients. Clinicians should consider how to further standardize infusion therapy, and innovate new infusion strategies to increase efficacy, reduce toxicity, improve patient satisfaction and save health resource costs. Sporadic studies have evaluated the effects of infusion rates of anticancer agents on clinical outcomes, economic benefits, and administration efficiency. However, an update review has not been available. Methods: Relevant literature was identified by search of PubMed until September 2023. Results: Infusion rates may have significant effect on the efficacy of anticancer agents (e.g., methotrexate, fluorouracil, and arsenic trioxide). Slow infusion is safer for platinum compounds, doxorubicin and carmustine, whereas fast infusion is safer than slow infusion of gemcitabine. Optimal flow rates of paclitaxel and fluorouracil are based on the balance between multiple risks of toxicity. Optimal infusion rate may bring economic benefits. If efficacy and safety are not compromised, shortened infusion may result in higher patient satisfaction, improved institutional efficiency and more nursing time available for other activities (e.g., biosimilar products, endostar). Other concerns about infusion rate include clinical indications (eg, paclitaxel and rituximab, methotrexate), severity and type of hypersensitivity reactions (e.g., platinum compounds), formulation features (e.g., paclitaxel, doxorubicin), and genetic polymorphism (e.g., gemcitabine, methotrexate). Conclusion: The latest knowledge of infusion rate concerns will enhance the appropriateness and accuracy in intravenous administration. Interdisciplinary teams should collaborate and implement relevant risk management and healthcare policy. It is worthwhile to conduct comparative studies of intravenous therapy with different infusion speeds.

Challenges and opportunities in building a health economic framework for personalized medicine in oncology.

Personalized medicine has allowed for knowledge at an individual level for several diseases and this has led to improvements in prevention and treatment of various types of neoplasms. Despite the greater availability of tests, the costs of genomic testing and targeted therapies are still high for most patients, especially in low- and middle-income countries. Although value frameworks and health technology assessment are fundamental to allow decision-making by policymakers, there are several concerns in terms of personalized medicine pharmacoeconomics. A global effort may improve these tools in order to allow access to personalized medicine for an increasing number of patients with cancer.

Pharmacoeconomic Appraisal of Treating Head and Neck Cancer with Various Chemotherapy and Chemoirradiation Regimens Using Branded and Jan-aushadhi Drugs in India.

According to recent studies, head and neck cancers (HNC) make up a substantial proportion of all cancers in India and are proportionately high in the lower socioeconomic people practising tobacco and alcohol. Chemotherapy is a crucial component of treating HNC, and this study was carried out to determine the Cost Minimization Analysis (CMA) by comparing the price of the high and least expensive branded drugs marketed in India. This study was performed to understand the cost difference (CD), Cost ratio (CR) and percentage of cost variation (PCV) of both individual drugs and the standard anticancer regimens. The Current Index of Medical Stores was used to determine the costs of the most and least expensive branded medications and analysed. The results indicated that Paclitaxel-Cisplatin-5 Fluoruracil chemotherapy regimen exhibited the greatest variance in cost, with the Jan Aushadhi brand of medicine costing 8.1 times and 4.3 times less than the most expensive and cheapest branded drugs, respectively. The cost of the concurrent cisplatin regimen with Jan Aushadi Medicine was Rs 1764, Rs 3489.64 with the lowest branded drug, and Rs 8477 with the most expensive branded drug. Also when compared to Jan Aushadhi medication, the cost of Ciplatin was 4.8 times higher with the most expensive branded drug and 2 times higher with the least expensive branded drug. As far as the authors are aware of this is the first study that addresses the pharmacoeconomic appraisal of treating HNC with various chemotherapy and chemoirradiation regimens. This study will help oncologists, pharmacists, and healthcare workers comprehend the financial advantages of treating breast cancer with less expensive chemotherapeutic agents instead of brand-name medications.

[The proper use of carboxymaltose iron and its economic impact in the geriatric wards of a French university hospital]. / Bon usage du fer carboxymaltose et impact économique dans les services gériatriques d'un CHU.

The proper use and economic impact of carboxymaltose iron were evaluated for patients hospitalized in the geriatric wards of a French university hospital from November 2019 to April 2020. Martial supplementation was recommended for 75.7% of the 173 patients who received carboxymaltose iron: 43.4% had a real indication for carboxymaltose iron, while 14.4% could have received sucrose iron and 17.9% could have received per os iron. Compliance with the recommendations would have generated savings of 10,345.80 euros (32.1%).

The Switch from Ferric Gluconate to Ferric Carboxymaltose in Hemodialysis Patients Acts on Iron Metabolism, Erythropoietin, and Costs: A Retrospective Analysis.

Background and Objectives: Iron deficiency and anemia characterize patients on chronic hemodialysis (HD). Available intravenous iron agents, such as ferric gluconate (FG) and ferric carboxymaltose (FCM), vary in dosing regimens and safety profiles. The aim of the present study was to analyze the modification of the iron status, the correction of anemia, and the economic implications after the shift from FG to FCM therapy in chronic HD patients. We evaluated, during the study, the variations in iron metabolism, assessing ferritin and transferrin saturation, erythropoietin-stimulating agent (ESA) doses and the number of administrations, the effects on anemic status, and consequent costs. Materials and Methods: A retrospective study was performed with a follow-up period of 24 months, enrolling forty-two HD patients. The enrolment phase started in January 2015, when patients were treated with iv FG, and continued until December 2015, when FG was discontinued, and, after a wash-out period, the same patients were treated with FCM. Results: The iron switch reduced the administered dose of ESA by 1610.500 UI (31% of reduction; p < 0.001) during the entire study period and reduced the erythropoietin resistance index (ERI) (10.1 ± 0.4 vs. 14.8 ± 0.5; p < 0.0001). The FCM group had the highest percentage of patients who did not require ESA treatment during the study period. The FCM patients were characterized by higher levels of iron (p = 0.04), ferritin (p < 0.001), and TSAT levels (p < 0.001) compared to the FG patients. The annual cost during FG infusion was estimated at EUR 105,390.2, while one year of treatment with FCM had a total cost of EUR 84,180.7 (a difference of EUR 21,209.51 (20%), saving EUR 42.1 per patient/month (p < 0.0001). Conclusions: FCM was a more effective treatment option than FG, reducing ESA dose requirements, increasing Hb levels, and improving iron status. The reduced ESA doses and the decreased number of patients needing ESA were the main factors for reducing overall costs.

Pharmacoeconomics of novel pharmacotherapies in triple-negative breast cancer.

INTRODUCTION: Triple-negative breast cancer (TNBC) represents the most aggressive breast cancer subtype carrying unfavorable clinical outcomes. Although traditionally chemotherapy has represented the only systemic treatment option available, novel drugs have changed the treatment landscape, granting approval by regulatory agencies worldwide, while determining new economic struggles on health systems for their high prices. AREAS COVERED: In this review, we provided a comprehensive analysis of pharmacoeconomic studies of drugs recently approved in the early and advanced settings of TNBC. EXPERT OPINION: Novel systemic treatment options redefined the therapeutic algorithms for TNBC by establishing new paradigms, based on substantial clinical benefits. Pembrolizumab and olaparib in the curative setting portend high value, as shown with the use of value frameworks, resulting in cost-effective interventions. In the metastatic setting, new drugs have demonstrated mixed improvements in patient-centric end-points, resulting often in interventions unlikely to have good value for money. We believe that cost-effectiveness alone is not a metric to inform the opportunity to invest on new interventions, while the intrinsic value of medicines should be the driver of the decisions. We endorse a patient-centric priority setting that can ensure health system sustainability, to timely deliver innovative cancer care to all in need and positively impact on population health.

The improved health utility of once-weekly subcutaneous semaglutide 2.4 mg compared with placebo in the STEP 1-4 obesity trials.

AIM: To assess health utility values in the Semaglutide Treatment Effect in People with obesity (STEP) 1-4 trials. MATERIALS AND METHODS: The STEP 1-4 phase 3a, 68-week, double-blind randomized controlled trials assessed the efficacy and safety of semaglutide 2.4 mg versus placebo in individuals with a body mass index (BMI) of 30 kg/m2 or higher or a BMI of 27 kg/m2 or higher and at least one comorbidity (STEP 1, 3 and 4), or a BMI of 27 kg/m2 or higher and type 2 diabetes (STEP 2). Patients received lifestyle intervention plus intensive behavioural therapy in STEP 3. Health-related quality of life was assessed using the Short Form 36-item Health Survey version 2 (SF-36v2) at baseline and week 68. Scores were converted into Short Form Six-Dimension version 2 (SF-6Dv2) utility scores or mapped onto the European Quality of Life Five-Dimension Three-Level (EQ-5D-3L) utility index using UK health utility weights. RESULTS: At week 68, semaglutide 2.4 mg was associated with minor health utility score improvements from baseline (all trials), while scores for placebo typically decreased. SF-6Dv2 treatment differences by week 68 for semaglutide 2.4 mg versus placebo were significant in STEP 1 and 4 (P ≤ .001), but not STEP 2 or 3. EQ-5D-3L treatment differences by week 68 for semaglutide 2.4 mg versus placebo were significant in STEP 1, 2 and 4 (P < .001 for all), but not STEP 3. CONCLUSIONS: Semaglutide 2.4 mg was associated with improvement in health utility scores compared with placebo, reaching statistical significance in STEP 1, 2 and 4.

Challenges for Medicare and universal health care in Australia since 2000.

OBJECTIVES: To identify the financing and policy challenges for Medicare and universal health care in Australia, as well as opportunities for whole-of-system strengthening. STUDY DESIGN: Review of publications on Medicare, the Pharmaceutical Benefits Scheme, and the universal health care system in Australia published 1 January 2000 - 14 August 2021 that reported quantitative or qualitative research or data analyses, and of opinion articles, debates, commentaries, editorials, perspectives, and news reports on the Australian health care system published 1 January 2015 - 14 August 2021. Program-, intervention- or provider-specific articles, and publications regarding groups not fully covered by Medicare (eg, asylum seekers, prisoners) were excluded. DATA SOURCES: MEDLINE Complete, the Health Policy Reference Centre, and Global Health databases (all via EBSCO); the Analysis & Policy Observatory, the Australian Indigenous HealthInfoNet, the Australian Public Affairs Information Service, Google, Google Scholar, and the Organisation for Economic Co-operation and Development (OECD) websites. RESULTS: The problems covered by the 76 articles included in our review could be grouped under seven major themes: fragmentation of health care and lack of integrated health financing, access of Aboriginal and Torres Strait Islander people to health services and essential medications, reform proposals for the Pharmaceutical Benefits Scheme, the burden of out-of-pocket costs, inequity, public subsidies for private health insurance, and other challenges for the Australian universal health care system. CONCLUSIONS: A number of challenges threaten the sustainability and equity of the universal health care system in Australia. As the piecemeal reforms of the past twenty years have been inadequate for meeting these challenges, more effective, coordinated approaches are needed to improve and secure the universality of public health care in Australia.

Cost-minimisation analysis of intravenous versus subcutaneous trastuzumab regimen for breast cancer management in Hong Kong.

INTRODUCTION: In 2017, breast cancer was the most common cancer and third leading cause of cancer death among women in Hong Kong. Approximately 20% of patients were human epidermal growth factor receptor-2 (HER2)-positive. This study was conducted to investigate cost differences between intravenous and subcutaneous trastuzumab regimens in Hong Kong using medical resources utilisation data from other countries. METHODS: A cost-minimisation model was developed to compare the cost of total care, including direct medical cost and full-time equivalent (FTE) hours. The drug acquisition cost was obtained from the manufacturer, whereas the costs for hospitalisation and clinic visits were acquired from the Hong Kong Gazette. Time (in FTE hours) was determined by literature review. All costs were expressed in US dollars (US$1 = HK$7.8). Costs were not discounted because of the short time horizon. One-way deterministic sensitivity analysis was performed to identify the effects of changes in drug acquisition cost, changes in FTE hours (based on confidence intervals reported), and changes in body weight (±20%). RESULTS: Literature review indicated that 0.18 FTE hour of nursing time (7.9 hours) and 0.14 FTE hour of pharmacist time (6.2 hours) could be saved each week if the subcutaneous formulation was used. Using data in 2017, after 18 cycles of treatment with subcutaneous trastuzumab, the drug acquisition and healthcare professional time costs were reduced by US$9451.28 and US$566.16, respectively, yielding an annual savings of over US$8 million. CONCLUSION: The subcutaneous formulation of trastuzumab is a potential cost-saving therapy for HER2-positive breast cancer patients in Hong Kong. The drug acquisition cost was the parameter with the greatest effect on the total cost of treatment.

Economic evaluation of ferric carboxymaltose compared with placebo in iron-deficient patients with heart failure: a systematic review.

BACKGROUND: It has been shown that ferric carboxymaltose (FCM) improves symptoms and quality of life in iron-deficient patients with heart failure (HF). AIM: We aimed to systematically review studies conducted on the cost-effectiveness of FCM compared to placebo in iron-deficient patients with HF. METHOD: We searched PubMed, EMBASE, Scopus, and Web of Science to find the relevant studies. After removing duplicates, two authors independently evaluated the titles, abstracts, and full texts. We included studies that investigated the full economic evaluations of FCM in HF patients with iron deficiency (cost-effectiveness analysis, cost-utility analysis, and cost-benefit analysis) and used the CHEERS tool to evaluate the quality of the studies. RESULTS: Seven studies were included which evaluated the economic analysis of treatments with FCM in iron-deficient patients with HF. The CHEERS scores for most of the studies (n = 6) were 0.77 or higher (very good quality). The lowest incremental cost-effectiveness ratio (ICER) per quality-adjusted life years (QALY) of FCM ($1801.96) was from Italy, and the highest ICER per QALY of FCM ($25,981.28) South Korea. Results of the studies showed that FCM, compared to placebo, was cost-effective in iron-deficient patients with HF. CONCLUSION: FCM is a cost-effective treatment for iron-deficient patients with HF. Considering the fact that all the included studies in the present systematic review took place in high-income countries, we recommend further studies investigating the cost-effectiveness of FCM in low- and middle-income countries.

Cost and public reimbursement of cancer medicines in the UK and the Republic of Ireland.

INTRODUCTION/AIMS: There are disparities in the availability of systemic anticancer therapies (SACTs) globally. We set out to investigate the cost and reimbursement of SACTs in the United Kingdom (UK) and the Republic of Ireland (ROI) in conjunction with efficacy and licensing authority decisions in the United States (US) and the European Union (EU). METHODS: We sought data pertaining to licensing in the EU, reimbursement in ROI/UK and cost/efficacy of SACTs licensed by the Food and Drug Administration (FDA) between January 2015 and May 2021. Independent samples t tests, chi-square test and Pearson's correlation were used for statistical analysis. RESULTS: We identified that the majority of FDA-approved regimens are licensed by the European Medicines Agency (EMA) (n = 91, 67.9%). However, only a minority of these are currently reimbursed in the UK (n = 60, 45%) or the ROI (n = 28, 21%) as of the 1st of May 2021. In addition, only a minority of regimens have demonstrated a statistically significant OS benefit (n = 54, 40%). There was no association between cost of regimens and either the presence (t = 0.846, p = 0.40) or duration of OS benefit (t = - 0.84, p = 0.64). CONCLUSIONS: Our study highlights that many licensed systemic anticancer treatments are not currently reimbursed in ROI/UK. The high cost of these medicines is independent of the presence of an OS benefit. Collaboration between regulatory agencies, governments and industry partners is needed to ensure health expenditure is directed towards the most effective treatments.

Análise farmacoeconômica do uso de omeprazol por idosos em uma das unidades básicas de saúde do Brasil / Pharmacoeconomic Analysis of the Use of Omeprazole by Elderly Patients in One of the Primary Healthcare Centers in Brazil

O omeprazol é inibidor de bomba de prótons mais prescrito no Brasil e é indicado para o tratamento de doenças por refluxos gastroesofágicos, úlcera péptica, esofagite erosiva, erradicação de Helicobacter pylori, síndrome de Zollinger-Ellison, gastrinomas, gastrite e distúrbios hipersecretores e prevenção de úlcera péptica. O uso indiscriminado e desnecessário, principalmente por idosos, é uma importante questão de saúde pública a ser tratada. Apesar de ser conhecida a existência do uso indiscriminado do omeprazol, identifica-se a necessidade de se verificar como ocorre a prescrição desse medicamento no contexto do Sistema Único de Saúde (SUS) do Brasil. Além disso, acredita-se que estudos que analisam de modo famacoeconômico a prescrição do omeprazol podem contribuir para a revisão ou implementação de diretrizes e protocolos que envolvam o uso desse medicamento. O objetivo foi caracterizar e realizar análise farmacoeconômica do uso do omeprazol em Unidade de Atenção Primária a Saúde (UAPS) em Minas Gerais, Brasil. Trata-se de estudo descritivo com análise das prescrições de 41 pacientes idosos atendidos no período de abril/maio de 2018 e foram identificadas as seguintes variáveis: idade, sexo, dose, indicação, realização de endoscopia, demais medicamentos em uso e custo do tratamento. Na análise farmacoeconômica realizou-se o cálculo do custo médio da quantidade de omeprazol prescrita por paciente e considerou-se a realização ou não de endoscopia. Na população estudada, 29 (70,3%) do sexo feminino com mediana de idade: 69 anos. Apenas em 4 prontuários (9,8%) havia indicação para uso e em 18 (43,9%) pacientes, a utilização era feita há mais de dois anos e em 7 (17,1%) a mais de cinco anos. Apenas 3 (7,3%) realizaram endoscopia e foram utilizados 371 meses totalizando um valor financeiro de R$35.657,23. Os achados sugerem prescrições em longo prazo e sem registro da indicação em prontuário. O custo referente às prescrições poderia ser otimizado com a realização de endoscopia e suspensão do uso, quando constatada ausência de indicação.

Omeprazole is the most prescribed proton pump inhibitor in Brazil and is indicated for the treatment of diseases caused by gastroesophageal reflux, peptic ulcer, erosive esophagitis, eradication of Helicobacter pylori, Zollinger-Ellison syndrome, gastrinomas, gastritis, and hypersecretory disorders, as well as peptic ulcer prevention. The indiscriminate and unnecessary use, mainly by the elderly, is an important public health issue to be addressed. Despite the existence of indiscriminate use of omeprazole being known, there is a need to verify how this medication is prescribed in the context of the Unified Health System (UHS) in Brazil. In addition, it is believed that studies that aim to analyze the prescription of omeprazole in a pharmacoeconomic way can contribute to the review or implementation of guidelines and protocols involving the use of this drug. The objective of this study was to characterize and perform a pharmacoeconomic analysis of the use of omeprazole in a Primary Healthcare Centers (PHC) in Minas Gerais, Brazil. This is a descriptive study analyzing the prescriptions of 41 elderly patients treated in the period of April/May 2018 and the following variables were identified: age, sex, dose, indication, endoscopy, other medications in use, and cost of treatment. In the pharmacoeconomic analysis, the average cost of the amount of omeprazole prescribed per patient was calculated and whether or not endoscopy was performed was considered. In the studied population, 29 (70.3%) were female with a median age: 69 years. Only in 4 medical records (9.8%) was there indication for use, and 18 (43.9%) patients had been using omeprazole for more than two years and 7 (17.1%) for more than five years. Only 3 (7.3%) patients underwent endoscopy, and 371 months-worth of omeprazole were used, totaling a financial value of R$35,657.23. The findings suggest long-term prescriptions and no record of their indication on medical records. The cost related to prescriptions could be optimized by performing endoscopy and discontinuing its use when no indication is found.

Appraisal of Novel Oncological Therapies by the Scottish Medicines Consortium and the National Institute for Health and Care Excellence: A Comparative Study of Six Years of Data.

Background and aims Pharmacoeconomic assessment of novel oncological therapies is an increasingly important factor in determining patient access to therapies. Organisations such as the National Institute for Health and Care Excellence (NICE) in England and the Scottish Medicines Consortium (SMC) in Scotland assess medications for their cost-effectiveness through health technology assessments (HTA) and provide guidance on whether the public health service should fund a therapy. We assessed six years of data to determine if there were any differences in timescales and decisions between NICE and SMC for new oncological therapies. Methods and results Time (days) from marketing authorisation (MA) to publication of final HTA guidance was calculated for single technology appraisals published by NICE and SMC between January 1, 2017, and December 31, 2022, for oncological therapies. We assessed 161 HTAs by NICE and 148 HTAs by SMC published in the study period. The median time from MA to publication of HTA guidance was 291 days (IQR 222-406) for SMC and 257 days (IQR 167-448) for NICE (p=0.054). For solid organ cancer therapies, NICE was significantly faster in publishing guidance, with a median of 231.5 days (IQR 148-392.25), compared to SMC, which took 273 days (IQR 202-378) (p=0.039). Overall recommendation of technologies was similar between the SMC and NICE (90.5% and 89.4%, respectively), with discordance in a minority of cases (12.6%). Conclusions Recommendation rates for single HTAs are similar between NICE and SMC for oncological therapies with discordance in a minority of cases. The time from MA to publication of HTA guidance was similar overall, but NICE was faster in publishing HTA guidance for solid organ cancer indications. Differences in methodology and process between the two organisations, in particular the presence of the Cancer Drugs Fund in England, may explain this difference in publication times.