Unraveling elements of value-based pricing from a pharmaceutical industry's perspective: a scoping review.

Health authorities use value-based pricing models to determine the value of innovative drugs and to establish a price. Pharmaceutical companies prefer value-based pricing over cost-based pricing. It is ambiguous whether value-based pricing has the same meaning to these stakeholders. We aimed to identify the elements that attribute to value-based pricing of innovative drugs from a pharmaceutical industry's perspective and as possible starting point for (value-based) contracting of drugs. We performed a scoping review of publications available in scientific databases with terms such as 'value-based pricing', 'pharmacoeconomics', 'drug cost', 'innovative drug' and 'drug therapy'. We included 31 publications, covering value elements of innovative drugs from a pharmaceutical industry's perspective. Overall, all found elements of value-based pricing were congruent with the elements of value-based pricing from a health authority's perspective. However, the emphasis placed on the elements differed. The most frequently mentioned elements in our review were economic considerations and cost aspects. Least mentioned were elements regarding cost-effectiveness, disease characteristics and patient characteristics. Although all elements in the drug value framework were present which indicate congruity, there seems controversy on the importance of cost-effectiveness as an element of value. Consequently, establishing a coherent and to all stakeholders' acceptable framework to value and price innovative drugs seems complicated. Mutual understanding can be found in the value elements societal considerations and healthcare process benefits. Our results supported the importance of economic and cost aspects regarding determination of prices of innovative drugs. Further research is required to quantify the weights of all relevant elements in the drug value framework, observe their possible interlinkages, and to weigh them over time.

Adverse event costs of systemic therapies for metastatic colorectal cancer previously treated with fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapy and biologics in the US.

Aim: The objective of this study was to compare adverse event (AE) management costs for fruquintinib, regorafenib, trifluridine/tipiracil (T/T) and trifluridine/tipiracil+bevacizumab (T/T+bev) for patients with metastatic colorectal cancer (mCRC) previously treated with at least two prior lines of therapy from the US commercial and Medicare payer perspectives. Materials & methods: A cost-consequence model was developed to calculate the per-patient and per-patient-per-month (PPPM) AE costs using rates of grade 3/4 AEs with incidence ≥5% in clinical trials, event-specific management costs and duration treatment. Anchored comparisons of AE costs were calculated using a difference-in-differences approach with best supportive care (BSC) as a common reference. AE rates and treatment duration were obtained from clinical trials: FRESCO and FRESCO-2 (fruquintinib), RECOURSE (T/T), CORRECT (regorafenib) and SUNLIGHT (T/T, T/T+bev). AE management costs for the commercial and Medicare perspectives were obtained from publicly available sources. Results: From the commercial perspective, the AE costs (presented as per-patient, PPPM) were: $4015, $1091 for fruquintinib (FRESCO); $4253, $1390 for fruquintinib (FRESCO-2); $17,110, $11,104 for T/T (RECOURSE); $9851, $4691 for T/T (SUNLIGHT); $8199, $4823 for regorafenib; and $11,620, $2324 for T/T+bev. These results were consistent in anchored comparisons: the difference-in-difference for fruquintinib based on FRESCO was -$1929 versus regorafenib and -$11,427 versus T/T; for fruquintinib based on FRESCO-2 was -$2257 versus regorafenib and -$11,756 versus T/T. Across all analyses, results were consistent from the Medicare perspective. Conclusion: Fruquintinib was associated with lower AE management costs compared with regorafenib, T/T and T/T+bev for patients with previously treated mCRC. This evidence has direct implications for treatment, formulary and pathways decision-making in this patient population.

Tumor-Homing Peptides as Crucial Component of Magnetic-Based Delivery Systems: Recent Developments and Pharmacoeconomical Perspective.

According to data from the World Health Organization (WHO), cancer is considered to be one of the leading causes of death worldwide, and new therapeutic approaches, especially improved novel cancer treatment regimens, are in high demand. Considering that many chemotherapeutic drugs tend to have poor pharmacokinetic profiles, including rapid clearance and limited on-site accumulation, a combined approach with tumor-homing peptide (THP)-functionalized magnetic nanoparticles could lead to remarkable improvements. This is confirmed by an increasing number of papers in this field, showing that the on-target peptide functionalization of magnetic nanoparticles improves their penetration properties and ensures tumor-specific binding, which results in an increased clinical response. This review aims to highlight the potential applications of THPs in combination with magnetic carriers across various fields, including a pharmacoeconomic perspective.

Cost-effectiveness analysis of bevacizumab for cerebral radiation necrosis treatment based on real-world utility value in China.

BACKGROUND: Bevacizumab shows superior efficacy in cerebral radiation necrosis (CRN) therapy, but its economic burden remains heavy due to the high drug price. This study aims to evaluate the cost-effectiveness of bevacizumab for CRN treatment from the Chinese payers' perspective. METHODS: A decision tree model was developed to compare the costs and health outcomes of bevacizumab and corticosteroids for CRN therapy. Efficacy and safety data were derived from the NCT01621880 trial, which compared the effectiveness and safety of bevacizumab monotherapy with corticosteroids for CRN in nasopharyngeal cancer patients, and demonstrated that bevacizumab invoked a significantly higher response than corticosteroids (65.5% vs. 31.5%, P < 0.001) with no significant differences in adverse events between two groups. The utility value of the "non-recurrence" status was derived from real-world data. Costs and other utility values were collected from an authoritative Chinese network database and published literature. The primary outcomes were total costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (ICER). The uncertainty of the model was evaluated via one-way and probabilistic sensitivity analyses. RESULTS: Bevacizumab treatment added 0.12 (0.48 vs. 0.36) QALYs compared to corticosteroid therapy, along with incremental costs of $ 2010 ($ 4260 vs. $ 2160). The resultant ICER was $ 16,866/QALY, which was lower than the willingness-to-pay threshold of $ 38,223/QALY in China. The price of bevacizumab, body weight, and the utility value of recurrence status were the key influential parameters for ICER. Probabilistic sensitivity analysis revealed that the probability of bevacizumab being cost-effectiveness was 84.9%. CONCLUSION: Compared with corticosteroids, bevacizumab is an economical option for CRN treatment in China.

Economic cost-benefit analysis of person-centred medicines reviews by general practice pharmacists.

BACKGROUND: Medicines reviews by general practice pharmacists improve patient outcomes, but little is known about the associated economic outcomes, particularly in patients at higher risk of medicines-related harm. AIM: To conduct an economic cost-benefit analysis of pharmacists providing person-centred medicines reviews to patients with hyperpolypharmacy (prescribed ≥ 10 regular medicines) and/or at high risk of medicines-related harm across multiple general practice settings. METHOD: Service delivery costs were calculated based on the pharmacist's salary, recorded timings, and a general practitioner fee. Direct cost savings were calculated from the cost change of patients' medicines post review, projected over 1 year. Indirect savings were calculated using two models, a population-based model for avoidance of hospital admissions due to adverse drug reactions and an intervention-based model applying a probability of adverse drug reaction avoidance. Sensitivity analyses were performed using varying workday scenarios. RESULTS: Based on 1471 patients (88.4% with hyperpolypharmacy), the cost of service delivery was €153 per review. Using the population-based model, net cost savings ranging from €198 to €288 per patient review and from €73,317 to €177,696 per annum per pharmacist were calculated. Using the intervention-based model, net cost savings of €651-€741 per review, with corresponding annual savings of €240,870-€457,197 per annum per pharmacist, were calculated. Savings ratios ranged from 181 to 584% across all models and inputs. CONCLUSION: Person-centred medicines reviews by general practice pharmacists for patients at high risk of medicines-related harm result in substantial cost savings. Wider investment in general practice pharmacists will be beneficial to minimise both patient harm and healthcare system expenditure.

Informing evidence-based medicine for opioid use disorder using pharmacoeconomic studies.

INTRODUCTION: The health and economic consequences of inadequately treated opioid use disorder (OUD) are substantial. Healthcare systems in the United States (US) and other countries are facing a growing healthcare crisis due to opioids. Although effective medications for OUD exist, relying solely on clinical information is insufficient for addressing the opioid crisis. AREAS COVERED: In this review, the role of pharmacoeconomic studies in informing evidence-based medication treatment for OUD is discussed, with a particular emphasis on the US healthcare system, where the economic burden is significantly higher than the global average. The scope/objective of pharmacoeconomics as a distinct scientific research program is briefly defined, followed by a discussion of existing evidence informed by data from systematic reviews, in addition to a convenience sample of recently published pharmacoeconomic studies and protocols. The review also explores the need for methodological advancements in the field. EXPERT OPINION: Despite the potential of pharmacoeconomic research in shaping evidence-based medicine for OUD, significant challenges limiting its real-world application remain. How to address these challenges are explored, including how to combine cost-effectiveness and budget impact analyses to address the needs of the healthcare system as a whole and specific stakeholders interested in adopting new OUD treatment strategies.

Impact of the genomic signature of 70-genes for breast cancer in the public system and in supplementary health care in a country of medium socioeconomic development.

INTRODUCTION: The financial impact of breast cancer has been discussed due to its high incidence and the increased costs of systemic therapy and is even more relevant in countries with low and medium socioeconomic development. OBJECTIVE: To evaluate the financial viability of using the MammaPrint™ (MP) genetic signature in a public and private system in a country with a medium socioeconomic development index. MATERIAL AND METHOD: A pharmacoeconomic trial with a cost-benefit analysis evaluating the reduction in costs of chemotherapy, support drugs, and materials used during chemotherapy infusion in high-risk hormone receptor-positive (HR+) breast cancer patients submitted to analysis using the MammaPrint™ genetic signature. RESULTS: The value of using MammaPrint™ in the Unified Health System (SUS) would bring an additional cost of US$ 1,334.56 per patient in the over-50 age group. In private medicine, the use of MammaPrint™ in the same population would result in cost savings ranging from US$ 2,422.53 to US$ 9,989.95 per patient. CONCLUSION: The use of MP in RH + breast cancer patients with high clinical risk and low genomic risk in Brazil leads to significant savings in resources when applied to supplementary healthcare. In the SUS, reducing the costs of MP for large-scale use could make its application viable. These values need to be re-evaluated in each institution, using the methodology applied in the trial, adjusting according to costs, to obtain a result that reflects its reality.

Factors influencing medical expenditures in patients with unresolved facial palsy and pharmacoeconomic analysis of upper eyelid lid loading with gold and platinum weights compared to tarsorrhaphy.

There are no standards in diagnostic and therapeutic approaches to eye care in incomplete eyelid closure due to unresolved facial palsy (FP). Loading of the upper eyelid (UELL) with gold weights (GWs) or platinum chains (PCs) is a highly effective procedure for the correction of lagophthalmos. Despite this, the procedure is used infrequently in our country because of the relatively high price of the implant and the lack of reimbursement. The aim of this research was to assess the factors influencing medical expenditures in this group of patients and to analyze utility costs for the UELL procedure with the use of GW and PC compared to tarsorrhaphy.Material and methods The costs of 88 surgical procedures (40 GWs, 11 PCs and 37 tarsorrhaphies) and medical expenditures before and after surgery were calculated based on reporting of materials, staff salaries and the SF-36 questionnaire. Distribution quartiles of the cost per QALY measure (dependent variable) was assessed via an ordered logistic regression model with eight explanatory variables.Results The calculated total cost of the surgery was US$209 for tarsorrhaphy, US$758 for UELL with a GW and US$1,676 for UELL with a PC. Bootstrapped costs per QALY values (CUI) in 88% of cases were below the US$100,000 cutoff. Etiology and duration of facial palsy and presence of Bell's phenomenon were factors that significantly influenced the CUI. Patient gender and age, history of previous eyelid surgery, and presence of corneal sensation were found to be not significant (p > 0.1). Calculated ICER for GW was US$1,241.74/1QALY and ICER for PC was US$13,181.05/1QALY compared to tarsorrhaphy.Conclusions Eye protection in patients with FP should be a crucial element of health policy. Findings suggest UELL procedure with a GW or a PC to be a cost-effective procedure with GW being the most cost-effective.

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.

Willingness to Pay (WTP) for Newer Treatment Options for Diabetes: A Study Among Patients at a Tertiary Care Centre.

Introduction Type 2 diabetes mellitus (T2DM) poses a substantial burden globally and particularly in India, affecting health, finances, and overall quality of life. The management of this condition relies on lifestyle modifications and advanced pharmacological interventions, with emerging drugs showing promise in areas such as administration, side effects, efficacy, and cardiovascular benefits. However, their market penetration is hindered by high costs. Understanding the target population's expectations and willingness to pay (WTP) for these drugs is crucial. WTP, a key concept in behavioral science, reflects the maximum price consumers are willing to pay for a product, aiding in healthcare cost-effectiveness evaluations. Despite its relevance, only one WTP study has been conducted in the Indian context for diabetes. This study explores WTP for two novel drugs: oral semaglutide and icodec (weekly insulin). Material and methods This observational study, conducted in a diabetes specialty clinic and telemedicine facility in All India Institute of Medical Sciences, Bhopal, India, involved adults (18-80 years) diagnosed with T2DM. Data collection adhered to ethical guidelines, and participants provided written informed consent. Face-to-face interviews were employed to gather socio-economic, demographic, and medical details. Participants estimated their WTP for oral semaglutide and weekly insulin, considering reference ranges for existing antidiabetic treatments. Statistical analyses, including t-tests and analysis of variance, explored sociodemographic and clinical factors influencing WTP. Results Of 105 approached patients, 87 (74.3%) participated. The majority were males (55.2%) with an average age of 57.2 years. The average WTP for oral semaglutide was INR 9.35±5.66 per pill, significantly lower than its market price (INR 315). For weekly insulin (icodec), the WTP was INR 157.25±112.60 per dose. Subgroup analyses revealed no significant correlations based on sociodemographic or clinical parameters. Conclusion This study demonstrated the feasibility of WTP assessments in an Indian outpatient setting, revealing a substantial cost disparity between patients' WTP for oral semaglutide and its market price. The findings underscore the importance of considering WTP in introducing new diabetes medications in India, offering valuable insights for healthcare decision-makers and developers.

Cost-Utility Analysis Comparing Pegcetacoplan to Anti-C5 Monoclonal Antibodies in the Treatment of Paroxysmal Nocturnal Hemoglobinuria.

Background: Paroxysmal nocturnal hemoglobinuria is a rare, acquired disease characterized by hemolytic episodes and associated with significant clinical burden. The introduction of C5 inhibitory monoclonal antibodies (C5i) represented a major breakthrough in PNH treatment, effectively reducing intravascular hemolysis (IVH) but showing limited impact on extravascular hemolysis (EVH). In 2021, the C3 inhibitor pegcetacoplan was approved by EMA and recently reimbursed in Italy, which also has the advantages in the reduction of both IVH and EVH, increasing hemoglobin values and simultaneously improving the quality of life and fatigue of patients. A cost-utility analysis was developed to compare pegcetacoplan to C5i (eculizumab and ravulizumab) in the PNH population who remain anemic after treatment with C5i for at least 3 months. Materials and Methods: The analysis employed a Markov model with a 5-year time horizon whereby patients can transition among 3 PNH health states, adopting the perspective of the Italian NHS. Efficacy data were sourced from the PEGASUS study, with drug prices reflecting ex-factory costs. Additionally, costs associated with resource utilization, adverse events, and complications were estimated based on outpatient and hospital care rates, excluding indirect expenses. Utility and disutility values related to transfusions were also considered, with pegcetacoplan allowing for dose escalation. Results: The cumulative cost of treatment per individual patient at 5 years was estimated to be €1,483,454 for pegcetacoplan, €1,585,763 for eculizumab, and €1,574,826 for ravulizumab. Pegcetacoplan demonstrated a superior increase in quality-adjusted life years (QALYs) compared to both eculizumab (0.51 increase) and ravulizumab (0.27 increase). Furthermore, pegcetacoplan showed a reduction in complication management costs (€22,891 less compared to eculizumab and €22,611 less compared to ravulizumab) and lower transfusion-related expenses (€14,147 less than both C5i treatments). Conclusion: Pegcetacoplan emerged as the dominant strategy in this analysis, being more effective, less expensive and improves quality of life in the analyzed population affected by PNH.

Short versus long-acting erythropoiesis-stimulating agents for anemia management in Egyptian hemodialysis patients.

BACKGROUND: Chronic kidney disease (CKD) often results in renal anemia, impacting the well-being of patients and causing various negative consequences. Erythropoiesis-stimulating agents (ESAs) offer promising solutions for managing anemia in CKD. This study aimed to evaluate and compare the effectiveness, safety profile, and cost-effectiveness of short-acting (Eprex®) and long-acting (Aranesp®) ESAs. METHOD: This comparative prospective cohort cost-effectiveness study was carried out over 6 months among adult Egyptian hemodialysis patients of either gender. Participants were categorized into two groups based on the type of ESA administered: the Eprex group, receiving epoetin alfa, and the Aranesp group, receiving darbepoetin alfa. These two treatment groups' efficacy, safety, and cost were analyzed and compared. RESULTS: Of 127 hemodialysis patients, 60 (47.2%) received Eprex, while 67 (52.8%) were treated with Aranesp. Target hemoglobin (Hb) was achieved by 50.6% of patients in the Eprex group versus 63.4% in the Aranesp group, with a significant difference (P < 0.001). Both treatment groups exhibited a similar safety profile, while Aranesp® was considered the cost-saving protocol. CONCLUSION: In hemodialysis Egyptian patients, Aranesp with extended dosing intervals proved to be more effective in achieving target Hb with comparable adverse effect profiles, a substantial cost-saving strategy, and offered time-saving advantages for medical staff workload compared to Eprex. TRIAL REGISTRATION: The Clinicaltrial.gov registration ID is NCT05699109 (26/01/2023).

[Economic evaluation of Annao Pills combined with antihypertensive drugs in treatment of primary hypertension: a study based on decision tree model].

The purpose of this study was to evaluate the economics of Annao Pills combined with antihypertensive drugs in the treatment of primary hypertension in the Chinese medical setting. TreeAge pro 2018 was used for cost-effect analysis and sensitivity analysis of the two treatment regimens. The intervention time of the simulation model was 2 weeks. The cost parameters were derived from Yaozhi.com, and the effect parameters were based on Meta-analysis of randomized controlled trial(RCT) involving Annao Pills. The experimental group was treated with Annao Pills combined with anti-hypertensive drugs(nifedipine controlled-release tablets + losartan potassium tablets), and the control group was treated with anti-hypertensive drugs(nifedipine controlled-release tablets + losartan potassium tablets). The basic analysis showed that the incremental cost-effect ratio(ICER) of the two groups was 2 678.67 yuan, which was less than 7.26% of the per capita disposable income in 2022. That is, compared with anti-hypertensive drugs alone, Annao Pills combined with antihypertensive drugs cost 2 678.67 yuan more for each additional patient with primary hypertension. The results of sensitivity analysis verified the robustness of the basic analysis results. The probability sensitivity results showed that when the patient's personal willingness to pay the price was higher than 2 650 yuan, the probability of the regimen in the experimental group was higher, which was consistent with the results of the basic analysis. In conclusion, when the price was higher than 2 650 yuan, Annao Pills combined with anti-hypertensive drugs was more economical than anti-hypertensive drugs alone in terms of improving the response rate of the patients with primary hypertension.

Asciminib for third-line treatment of chronic myeloid leukemia: Cost-effectiveness analysis based on treatment-free remission approach.

INTRODUCTION: The first targeted therapy in oncology, imatinib, revolutionized chronic myeloid leukemia (CML) treatment and spurred research in targeted therapies for various cancers. CML results from a chromosomal translocation, forming the BCR-ABL1 fusion gene. Asciminib has been recently approved for third-line refractory or intolerant patients. Treatment-free remission (TFR) is attainable with sustained deep molecular response (DMR) and this approach could be incorporated into pharmacoeconomic models. AIMS: To establish a cost-effectiveness model comparing asciminib to approved third-generation tyrosine kinase inhibitors (TKIs) (bosutinib and ponatinib) with a focus on achieving TFR. Additionally, the budgetary impact of incorporating asciminib as a therapeutic alternative is assessed. METHODS: This model is based on a Markov chain with 7 states. The condition for achieving TFR is to remain for 5 years in DMR state. Efficacy of the model was measured in QALYs, and the costs included in the base case analysis are based in Spain. A probabilistic (PSA) and deterministic analysis (DSA) were carried out to assess the variability of the model. There were achieved 2 independent models comparing asciminib vs bosutinib and asciminib vs ponatinib. RESULTS: Asciminib, when compared with ponatinib, is a cost-saving alternative, as efficacy is similar between alternatives, and asciminib have a lower cost of 30,275€. Asciminib showed 4.33 more QALYs and a higher cost (203,591€) than bosutinib, resulting in an ICER of €47,010.49 per QALY. PSA shows that the parameters with higher influence in the variability of the model were the probability of transitioning to BP and probabilities of achieving MMR and DMR. A one-way analysis reports that the drug cost has a higher influence on both models, and the discount rate significantly affects the asciminib vs bosutinib model. CONCLUSION: Asciminib broadens therapeutic choices for patient's refractory or intolerant to 2 prior lines of treatment in a cost-effectiveness manner. The costs of drugs significantly impact the overall cost of the disease, emphasizing the importance of the selected discount rates for each drug. Given the relatively low incidence of CML, the introduction of asciminib has a limited budgetary impact, warranting individualized decisions based on patient`s clinical characteristics.

Pharmacogenetic guided versus standard warfarin dosing for routine clinical care with its pharmacoeconomic impact: a randomized controlled clinical trial.

BACKGROUND: Empirical use of pharmacogenetic test(PGT) is advocated for many drugs, and resource-rich setting hospitals are using the same commonly. The clinical translation of pharmacogenetic tests in terms of cost and clinical utility is yet to be examined in hospitals of low middle income countries (LMICs). AIM: The present study assessed the clinical utility of PGT by comparing the pharmacogenetically(PGT) guided- versus standard of care(SOC)- warfarin therapy, including the health economics of the two warfarin therapies. METHODS: An open-label, randomized, controlled clinical trial recruited warfarin-receiving patients in pharmacogenetically(PGT) guided- versus standard of care(SOC)- study arms. Pharmacogenetic analysis of CYP2C9*2(rs1799853), CYP2C9*3(rs1057910) and VKORC1(rs9923231) was performed for patients recruited to the PGT-guided arm. PT(Prothrombin Time)-INR(international normalized ratio) testing and dose titrations were allowed as per routine clinical practice. The primary endpoint was the percent time spent in the therapeutic INR range(TTR) during the 90-day observation period. Secondary endpoints were time to reach therapeutic INR(TRT), the proportion of adverse events, and economic comparison between two modes of therapy in a Markov model built for the commonest warfarin indication- atrial fibrillation. RESULTS: The study enrolled 168 patients, 84 in each arm. Per-protocol analysis showed a significantly high median time spent in therapeutic INR in the genotype-guided arm(42.85%; CI 21.4-66.75) as compared to the SOC arm(8.8%; CI 0-27.2)(p < 0.00001). The TRT was less in the PG-guided warfarin dosing group than the standard-of-care dosing warfarin group (17.85 vs. 33.92 days) (p = 0.002). Bleeding and thromboembolic events were similar in the two study groups. Lifetime expenditure was ₹1,26,830 in the PGT arm compared to ₹1,17,907 in the SOC arm. The QALY gain did not differ in the two groups(3.9 vs. 3.65). Compared to SOC, the incremental cost-utility ratio was ₹35,962 per QALY gain with PGT test opting. In deterministic and probabilistic sensitivity analysis, the base case results were found to be insensitive to the variation in model parameters. In the cost-effectiveness-acceptability curve analysis, a 90% probability of cost-effectiveness was reached at a willingness-to-pay(WTP) of ₹ 71,630 well below one time GDP threshold of WTP used. CONCLUSION: Clinical efficacy and the cost-effectiveness of the warfarin pharmacogenetic test suggest its routine use as a point of care investigation for patient care in LMICs.

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.

The association between body mass index and health-related quality of life in the 2017 and 2018 health survey of England data: A cross-sectional observational analysis.

AIM: To provide an updated estimate of the association between body mass index (BMI) and health-related quality of life (HRQoL) among the general population in England and to identify population subgroups with the highest potential utility gains from obesity interventions. MATERIALS AND METHODS: The sample included 12 158 adults with valid HRQoL and BMI data from the 2017 and 2018 Health Survey for England. Robust standard error linear regression, controlling for demographic and socioeconomic characteristics, lifestyle behaviours and obesity-related comorbidities, was used for the baseline analysis. Robustness checks assessed the impact of (a) estimator selection; (b) model specifications; (c) statistical outliers at high BMI; (d) potential BMI measurement error; and (e) data pooling. RESULTS: The study found a significant association between HRQoL and BMI, which exhibited an inverted U-shaped relationship. The mean HRQoL peaked at 25.7 kg/m2 in men and 22.6 kg/m2 in women and was reduced in the underweight, overweight and obesity BMI ranges. Sensitivity analyses reported similar coefficients, suggesting a robust model specification. CONCLUSIONS: Reduced HRQoL beyond optimal BMI underlines the importance of maintaining a normal BMI range for overall health. The rising prevalence of class III obesity is a major public health concern given its disproportionate impact on health, health care utilization and costs. Obesity management is key to preventing the reduction in HRQoL associated with obesity-related comorbidities, and this analysis supports the development of targeted policies and population health initiatives for people with class III obesity.

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.