Comparative Pharmacoeconomic Effectiveness of Interleukin-17 Inhibitors for the Treatment of Ankylosing Spondylitis.

The objective of this study was to compare the clinical efficacy and cost-effectiveness of IL-17 inhibitors (SEC, IXE, NTK) in the treatment of adult patients with ankylosing spondylitis (AS) in the healthcare system of the Russian Federation. Materials and methods. The study is a sub-analysis of a previously published systematic review and network meta-analysis of the comparative efficacy of biologics in adult patients with AS in the Russian Federation. NNT values were calculated for BASDAI 50 and ASAS 20/40 after 16 weeks of therapy for all studied drugs. CpR was estimated for each biologic after 16 weeks and one year of therapy. Additionally, we carried out an assessment of the financial burden of the most cost-effective strategies for the treatment of AS. The use of NTK is characterized by an average of no more than three patients needed to treat to achieve one ASAS 20/40 or BASDAI 50 response, while on IXE and SEC no more than 4-5 patients need to be treated, depending on the estimated effectiveness criterion. According to CpR estimate, NTK is the most cost-effective IL-17 inhibitor for the treatment of AS, both after 16 weeks and after one year of therapy. The obtained results make it possible to compare the effectiveness of IL-17 inhibitors from a clinical and economic points of view and can be used both in decision making on treatment strategies for individual patients and at the population level when deciding on the reimbursement of drugs.

Emergent challenges and opportunities in drug discovery and commercialization.

We review medical economics literature presented at the 2023 annual AEA-ASSA convention, the largest gathering of economists worldwide. Pharmacoeconomic papers addressed a wide range of issues, including gender and racial gaps in clinical trials, hospital credit financing, drug rebates, covid-19 vaccine equality, and the opioid epidemic. Yet, they had some common identifiable themes. We examine them in the context of the "twin towers" of biopharmaceutical innovation: discovery and commercialization. Implementation outcomes and relative success of innovative solutions - whether in terms of products and services, structural design and arrangements, or policies - depend on how adequately they respond to questions and challenges that arise in drug discovery and commercialization, and who gains from them. That innovation's beneficiaries might not equally gain from its intended advantages is another unifying theme in the reviewed literature. Against this backdrop, biopharmaceutical innovation can breed new challenges and opportunities. And health policy can perform a critical, leveling function that reduces cost, increases access, and ensures quality of biopharmaceutical solutions.

Pharmacoeconomic issues in stem cell mobilization.

BACKGROUND: recently, stem cell mobilization has made dramatic progress, that ended up in an increasing number of aphereses at target for autologous peripheral stem cell transplantation (ASCT). The aim of this research is investigating the cost-effectiveness of stem cell mobilization. METHODS: a narrative review of the literature was carried out, searching for primary contributions written in English and published during 2000-2023 on cost-effectiveness analysis (CEA) of stem cell mobilization in patients entitled to ASCT. The PubMed database was searched with the following sets of keywords: cost-effectiveness AND apheresis AND myeloma (PubMed_1); cost-effectiveness AND stem cell mobilization (PubMed_2). Articles included in the analysis were assessed via two different checklists. RESULTS: sixty-six entries were retrieved. Five out of 66 (PubMed_1: 4 out 17; PubMed_2: 1 out of 49), 4 CEAs and 1 cost-utility analysis (CUA) fit the research goal. Four out of 5 contributions proved to be in line with most of the items included in the two assessment grids. However, the most relevant missing features in some of the included contributions were: study perspective, healthcare resources valuation, and sensitivity analyses. DISCUSSION: most of the articles included in this research show that chemotherapy-free stem cell mobilization is cost-effective according to different standpoints. Future health economic research on this topic should establish local threshold values for incremental apheresis at target and explore the heterogeneity of CEA (and CUA) to determine oncohaematological diseases and patient categories for which chemotherapy-free stem cell mobilization is cost-effective in different healthcare systems, given local budget constraints.

Interventional pharmacoeconomics for immune checkpoint inhibitors through alternative dosing strategies.

Immune checkpoint inhibitors (ICIs) are approved for the treatment of a variety of cancer types. The doses of these drugs, though approved by the Food and Drug Administration (FDA), have never been optimised, likely leading to significantly higher doses than required for optimal efficacy. Dose optimisation would hypothetically decrease the risk, severity, and duration of immune-related adverse events, as well as provide an opportunity to reduce costs through interventional pharmacoeconomic strategies such as off-label dose reductions or less frequent dosing. We summarise existing evidence for ICI dose optimisation to advocate for the role of interventional pharmacoeconomics.

Evaluating the Efficacy and Pharmacoeconomics of Semaglutide and Tirzepatide in the Setting of Obesity.

BACKGROUND: Obesity is a major and growing public health concern. The associated cost for obesity and its related comorbidities is approximately 30% of US health care expenditures annually. As additional pharmacotherapeutic options join the market to combat obesity, it is important to understand the financial impact it may have on overall health care costs. This article explores the efficacy and pharmacoeconomics of incretin mimetics, semaglutide and tirzepatide, in the setting of obesity. AREA OF UNCERTAINTY: The cost of incretin mimetics (semaglutide and tirzepatide) and its overall impact on obesity management within the health care arena is being explored. The cost comparison of these medications is to be determined; however, it may represent an added cost to the total US health care expenditures. DATA SOURCES: A PubMed and Google Scholar search was conducted using various search terms (eg, semaglutide, tirzepatide, pharmacoeconomics, and obesity). THERAPEUTIC ADVANCES: Based on the data reviewed, both semaglutide and tirzepatide are effective medication options for obesity management. Obesity-related management expenditures exceed $173 billion for the US health care system annually. The cost needed to treat for 1% of weight loss with semaglutide and tirzepatide was reported as $1845 and $985, respectively. More than 40% of adults (60 years or older) experience obesity. If 1%, 5%, or 10% of this population is treated with semaglutide, the annual Medicare costs will translate to excess of $2.6 billion, $13.3 billion, and $26.8 billion, respectively. Tirzepatide is not yet approved in the United States for obesity and its financial impact remains to be seen. CONCLUSIONS: Obesity is associated with burdensome health complications and costs. Semaglutide and tirzepatide are effective drug options for the management of obesity. The cost of these medications will no doubt present a challenge to the total health care expenditures, although the cost-benefit ratio may ultimately be favorable.

Pharmacoeconomic and antimicrobial stewardship analysis in waste management: Beyond switching drug administration route.

BACKGROUND: The aim of this study was to evaluate the impact of switch therapy of antimicrobials on cost reduction (pharmacoeconomic analysis) and hospital waste generation by switching from intravenous to oral therapy. This is a cross-sectional, observational, and retrospective study. METHODS: Data from 2019, 2020, and 2021, provided by the clinical pharmacy service of a teaching hospital in the interior of Rio Grande do Sul, were analyzed. The variables analyzed were intravenous and oral antimicrobials, frequency, duration of use, and total treatment time according to the institutional protocols. An estimate of the amount of waste not generated from the change of administration route was calculated by weighing the kits using a precision balance in grams. RESULTS: During the analyzed period, 275 switch therapy of antimicrobials were performed, resulting in US$ 55,256.00 of savings. The main antimicrobial classes that underwent changes were cephalosporins (25.1%), penicillins (22.55%), and quinolones (17.45%). Changing from intravenous to oral therapy avoided the generation of 170,631 g of waste, including needles, syringes, infusion bags, equipment, reconstituted solution bottles, and medication. CONCLUSIONS: The change from intravenous to the oral route of antimicrobials is safe for the patient, economically effective, and significantly reduces waste generation.

[Pharmacoeconomic evaluation of the tixagevimab and cilgavimab combination using for pre-exposure prophylaxis of COVID-19].

AIM: To evaluate pharmacoeconomic feasibility using of the tixagevimab and cilgavimab combination for pre-exposure prophylaxis of COVID-19 in immunocompromised patients. MATERIALS AND METHODS: Cost-effectiveness of tixagevimab and cilgavimab in persons ≥12 years old who weigh ≥40 kg and have either a history of allergy that prevents their vaccination against COVID-19 or moderate or immunocompromised was assessed based on PROVENT phase III study results. The quantity of life years or quality-adjusted life years gained was calculated. Direct medical cost associated with prophylaxis of COVID-19, treatment of infected patients and those experiencing long COVID post infection were assessed. Results were compared with wiliness-to-pay threshold, measured as tripled gross domestic product per capita and equal to 2.69 mln RUB in 2022. RESULTS: Pre-exposure prophylaxis of COVID-19 results in additional 0.0287 life years or 0.0247 quality-adjusted life years. The cost of additional life year gained is equal to 1.12 mln RUB, the cost of additional quality-adjusted life years is 1.30 mln RUB. Both costs of additional life year and cost of quality-adjusted life years appeared to be significantly less compared to wiliness-to-pay threshold. CONCLUSION: Pre-exposure prophylaxis of COVID-19 with combination of tixagevimab and cilgavimab is economically feasible and may be recommended for wide use in Russian healthcare system.

An Industry Survey on Unmet Needs in South Korea's New Drug Listing System.

INTRODUCTION: Since introducing the positive listing system in 2007, the South Korean government has undergone multiple changes in its drug listing system. As there is a lack of studies that evaluate the system from an industry perspective, this paper examined South Korea's new drug listing system from the suppliers' perspective. METHODS: We surveyed members of the three main pharmaceutical industry associations online. The survey (a 5-point Likert scale) covered their satisfactory levels, demands, and updates on the current new drug listing system, especially pharmacoeconomic evaluation, pharmacoeconomic evaluation exemption, and risk-sharing agreement. RESULTS: A total of 56 respondents participated in the survey. The self-reported satisfaction level for value recognition of new drugs was 1.6 (± 0.7) points (5 points = very satisfied). The most highly demanded reforms for PE, RSA, and PEE were incremental cost-effectiveness ratio threshold (92.9%), reimbursement scope expansion (91.1%), and eligible disease (83.9%). Lastly, they also claimed that the indication-based pricing system must be introduced (83.9%). CONCLUSIONS: Pricing and reimbursement policies need to improve in such a way that would enable better access to new drugs while still facilitating their development. Given the nature of the current system, some innovative rare disease treatments and anticancer drugs remain unreimbursed, resulting in low satisfaction levels across the pharmaceutical industry. Hence, pathways to speed up the reimbursement assessment process and expand the range of reimbursable diseases are required. Pharmaceutical companies are also important stakeholders, like in the case of clinicians and patients, and their opinions should also be considered in the process of pricing and reimbursement policy reforms.

RATIONAL PHARMACOTHERAPY ON THE BASIS OF PHARMACOECONOMIC JUSTIFICATION AND MARKETING RESEARCH ON THE APPLICATION OF IMMUNOMODULATORY PHYTOPREPARATIONS.

OBJECTIVE: The aim: Pharmacoeconomic substantiation and marketing research of immunoprotective phytopreparations in Ukraine to substantiate rational pharmacotherapy of the effectiveness of immunomodulatory drugs of plant origin and pharmaceutical care of patients to strengthen individual immunity. PATIENTS AND METHODS: Materials and methods: Research materials - data from the State Register of Medicinal Products of Ukraine; information content of the Public Health Center of the Ministry of Health of Ukraine; data of the State Register of Wholesale Prices for medicines declared in Ukraine under the international non-proprietary or common name as of 01.01.2023. Research methods: theoretical analysis of scientific sources, systematic, retrospective, descriptive and frequency analysis of information resources of databases; pharmacoeconomic analysis, marketing analysis of positioning in the pharmaceutical market of Ukraine to substantiate rational pharmacotherapy and the effectiveness of immunomodulatory drugs of plant origin to strengthen individual immunity. RESULTS: Results: Theoretical analysis and pharmacoeconomic substantiation of rational pharmacotherapy of efficiency of application of drugs of immunomodulatory action of plant origin and pharmaceutical care for strengthening of individual immunity of patients is carried out. The algorithm of pharmacoeconomic analysis of the use of immunomodulatory phytopreparations to ensure rational pharmacotherapy and pharmaceutical care of outpatients is substantiated. To substantiate the availability of effective immunomodulatory phytopreparations for patients, marketing research on the use of immunomodulatory phytopreparations in Ukraine has been conducted. CONCLUSION: Conclusions: The theoretical analysis shows that the use of immunomodulatory drugs of plant origin is appropriate in rational pharmacotherapy to strengthen the individual immunity of patients, which is especially relevant in an exacerbation of the epidemic situation caused by the spread of infectious diseases of viral origin. An algorithm of pharmacoeconomic substantiation has been developed, which provides an opportunity to confirm the therapeutic efficacy and pharmacoeconomic feasibility of immunomodulatory phytopreparations for rational pharmacotherapy and pharmaceutical care of patients. The results of marketing research provide an opportunity to determine the availability (positioning and price range) for patients of effective immunomodulatory phytopreparations in Ukraine and outline the prospects for pharmaceutical development and registration on the pharmaceutical market of Ukraine of new effective immunomodulatory drugs of plant origin.

Pharmacokinetic approach in therapeutic monitoring of antineoplastic drugs and the impact on pharmacoeconomics: A systematic review.

OBJECTIVES: Therapeutic drug monitoring aims to quantify the concentration of a drug in a biological matrix. In oncology, the therapeutic arsenal is vast and therapeutic drug monitoring optimizes treatment and reduces costs. This review will analyze the financial impact of therapeutic monitoring of anticancer drugs in healthcare institutions. METHODS: Keywords were selected using Decs (MeSH). Through the Pubmed, Scopus, and Virtual Health Library (VHL) databases, 74 articles were found, of which 4 meet the inclusion criteria. Methodological quality and risk of bias were assessed according to the Research Triangle Institute Item Bank (RTI-Item Bank) scale. KEY FINDINGS: Therapeutic drug monitoring is an important tool for dose reduction or dose increase due to toxicity and lack of response, respectively. The main barriers are associated costs and lack of cost-benefit data. An alternative is to use population pharmacokinetic models, measured plasma concentration(s) and relevant patient characteristics, estimated individual pharmacokinetic parameters, and predicted drug concentrations at any point in the dosing range. CONCLUSIONS: Therapeutic drug monitoring is understood as a technology that adds costs to payers. Future studies should generate clinical evidence of population pharmacokinetics from therapeutic drug monitoring studies.

What condition leads to an unreasonable pharmaceutical price? Impact research on the effect of medical service provision on pharmaceutical price regulation based on fuss-set QCA.

Given that the pharmaceutical market has experienced severe market failures, it is necessary that we regulate pharmaceutical prices for many countries. Toward ensuring that pharmaceutical price regulation is efficient, this study investigated the antecedents that lead to an unreasonable pharmaceutical price. Based on 33 case-study countries, this study utilized QCA to analyze the conditional configuration of unreasonable pharmaceutical prices from the perspective of medical service provision. The results showed that the causes of unreasonable pharmaceutical prices are configured by medical service provision, especially cost compensation systems and payment mechanism. This study's conclusions contribute to the research on pharmaceutical price regulation and the institution of medical service provision.

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.

Effect of the copayment reduction system on accessibility to orphan drugs in South Korea.

OBJECTIVE: This study aims to analyze the effect of the copayment reduction system on accessibility to orphan drugs (ODs) in South Korea. METHODS: Data on approval and reimbursement for drugs designated as ODs for the last 10 years (2012-2021) in South Korea were extracted. Among them, with 136 approved products as of 31 December 2022, the reimbursement rates and lead time to reimbursement between drugs for rare diseases (DRDs) and nondrugs for rare diseases (non-DRDs) were analyzed. The pricing and reimbursement (P&R) pathways between drugs for only rare diseases (DORDs) and drugs for rare and cancerous diseases (DRCDs) were compared. RESULTS: The reimbursement rates for DRDs and non-DRDs were 54.8% and 33.3%, respectively, and the lead time to reimbursement for DRDs and non-DRDs were 16.1 months and 31.2 months, respectively. The P&R pathways for DORDs and DRCDs were pharmacoeconomic evaluation waivers (21.7% and 52.6%), weighted average price (52.2% and 13.2%), and risk-sharing agreement (30.4% and 81.6%). CONCLUSION: The copayment reduction system may act as a driver and also barrier for the reimbursement of ODs. To expand treatment accessibility to ODs, it is necessary to consistently grants benefits in all processes from OD designation to market access.

Boletín de salud y economía. Edición N° 1 - Año 2 / Health and economy bulletin. Edition N° 2 - Year 1

La proporciona los resultados de sus estudios, trabajos de investigación, evaluaciones y similares en la que se presenten temas que vinculen la Salud y Economía, tales como: a) Producción, b) Financiamiento, c) Evaluaciones económicas, d) Inversiones, e) Costos, f) Presupuestos, g) Aseguramiento en Salud, h) Procesos, i) Tecnología en Salud, j) Atención Primaria y Hospitalaria, k) Farmacoeconomía, l) Capital Humano e Intelectual, entre otros, correspondiente a Enero 2023.

Pharmacoeconomic analysis (CER) of Dulaglutide and Liraglutide in the treatment of patients with type 2 diabetes.

Aim: To evaluate the treatment effect Fand pharmacoeconomic value of Dugaglutide in women with type 2 diabetes. Methods: Women (n=96) with type 2 diabetes recruited from June 2019 to December 2021 were randomized into two equal groups. The control group was treated with Liraglutide, and the observation group was treated with Dulaglutide, both for 24 weeks. The blood glucose levels, biochemical index, insulin resistance index (HOMA-IR), cost-effect ratio (CER), and drug safety were determined and compared between the two groups. Results: Blood glucose levels, the biochemical index, and HOMA-IR were lower in both groups after the treatment (P < 0.05), and there was no statistical difference in the blood glucose levels, biochemical index and HOMA-IR between the two groups (P > 0.05). The CER levels did not differ statistically between the two groups (P > 0.05). Both the cost and the incidence of drug side effects during solution injection were lower in the observation group than in the control group after 24 weeks of treatment (P < 0.05). Conclusion: Both Dulaglutide and Liraglutide can reduce blood glucose levels, improve biochemical index, and HOMA-IR levels in women with type 2 diabetes. Dulaglutide is more cost-effective and safe. Clinical trial registration: https://www.chictr.org.cn/index.aspx, identifier ChiCTR1900026514.

Multinational Analysis of Estimated Health Care Costs Related to Extended-Interval Fixed Dosing of Checkpoint Inhibitors.

Importance: New dosing options for immune checkpoint inhibitors have recently been approved by the US Food and Drug Administration (FDA), including fixed dosing with extended intervals. Although the dose intensity appears the same, there is expected to be some waste with extended-interval dosing, as some drug remains in the bloodstream once a decision to stop treatment is made. The economic impact of extended-interval fixed dosing is unknown compared with standard-interval fixed dosing. Objective: To analyze the potential health care costs of using extended-interval fixed dosing instead of standard-interval fixed dosing. Design, Setting, and Participants: This economic evaluation used a pharmacoeconomic model to simulate 2 cohorts of patients with platinum-resistant metastatic urothelial cancer receiving pembrolizumab as second-line therapy at different dosing intervals using 2020 pricing data. Data were analyzed from 2020 to 2022. Exposures: The simulated patients received FDA-approved regimens of either 200 mg every 3 weeks or 400 mg every 6 weeks. Main Outcomes and Measures: The progression-free survival curve from the KEYNOTE-045 trial was used to estimate treatment duration. Drug, imaging, and administration costs were included in analyses. Sensitivity analyses were performed to assess how different imaging frequencies would affect the model results. The potential overall costs of using the 2 different dosing strategies were assessed. The base case was set in the US, while sensitivity analyses were set in several other countries. Results: In the base case analysis, dosing every 6 weeks instead of every 3 weeks resulted in an estimated 8.9% increase in pembrolizumab costs for the health care payer. Accounting for a decrease in infusion costs would result in an estimated net additional cost of $7483 per patient in the US (7.9% cost increase). In the US, this would amount to an increase of approximately $28 million annually for health care payers. Similar percentages in cost estimate increases were found for health care payers around the world, such as in Israel, where the net additional cost would be $5491 per patient. Conclusions and Relevance: This economic evaluation assessed and quantified the potential increased costs related to extended-interval fixed dosing of pembrolizumab. The model method could be applied to other diseases and other drugs for which there has been a movement toward extended-interval dosing. Results may differ in other diseases owing to differing disease courses and patient profiles.