pharmacoeconomic

BACKGROUND: In 2016, England launched the largest nationwide diabetes mellitus prevention programme, the NHS Diabetes Prevention Programme (NHS DPP). This paper seeks to evaluate the long-term cost-effectiveness of this programme. METHODS: A Markov cohort state transition model was developed with a 35-year time horizon and yearly cycles to compare referral to the NHS DPP to usual care for individuals with non-diabetic hyperglycaemia. The modelled cohort of individuals mirrored the age profile of referrals received by the programme by April 2020...

OBJECTIVES: The primary focus of this research is the proposition of a methodological framework for the clinical application of the long coronavirus disease (COVID) Symptom and Severity Score (LC-SSS). This tool is not just a self-reported assessment instrument developed and validated but serves as a standardized, quantifiable means to monitor the diverse and persistent symptoms frequently observed in individuals suffering from long COVID. METHODS: A three-stage process was used to develop, validate, and establish scoring standards for the LC-SSS...

OBJECTIVES: This study aims to provide subjective well-being (SWB) population norms in Hungary and explore the contribution of explanatory factors of SWB inequality among the Hungarian adult general population. METHODS: The data originated from a large representative internet-based cross-sectional survey in Hungary, which was conducted in 2020. We applied validated multi-item instruments for measuring SWB, namely SWLS and WHO-5. Multiple linear regressions were employed to examine the relationship between demographic-socioeconomic-health status and both well-being instruments...

OBJECTIVES: The results of a recent single-arm trial (ZUMA-5) of axicabtagene ciloleucel (axi-cel) for relapsed/refractory (r/r) FL demonstrated high rates of durable response and tolerable toxicity among treated patients. To quantify the value of axi-cel compared to standard of care (SOC) to manage r/r FL patients who have had at least two prior lines of systemic therapy (3L+), a cost-effectiveness model was developed from a US third-party payer perspective. METHODS: A three-state partitioned survival cost-effectiveness model was developed with a lifetime horizon...

OBJECTIVES: This study aims to systematically review evidence on the cost-effectiveness of chimeric antigen receptor (CAR)-T therapies for patients with cancer. METHODS: Electronic databases were searched in October 2022 and updated in September 2023. Systematic reviews, health technology assessments and economic evaluations that compared costs and effects of CAR-T therapy in cancer patients were included. Two reviewers independently screened studies, extracted data, synthesized results, and critically appraised studies using the Philips checklist...

OBJECTIVES: Health economic (HE) models are often considered as "black boxes" because they are not publicly available and lack transparency, which prevents independent scrutiny of HE models. Additionally, validation efforts and validation status of HE models are not systematically reported. Methods to validate HE models in absence of their full underlying code are therefore urgently needed to improve health policy making.This study aimed to develop and test a generic dashboard to systematically explore the workings of HE models and validate their model parameters and outcomes...

PURPOSE: This study aimed to assess the health-related quality of life (HRQoL) of the Chinese population by using the Chinese medicine quality of life-11 dimensions (CQ-11D) questionnaire and to identify factors associated with HRQoL. METHODS: The data was derived from a survey conducted by the Institute of Pharmacoeconomics Evaluation at Beijing University of Chinese Medicine on the quality of life of the Chinese population. The sex and age of respondents were considered through quota sampling...

OBJECTIVES: The Inflation Reduction Act (IRA), enacted in 2022, brings substantial reforms to the US healthcare system, particularly regarding Medicare. A key aspect includes the introduction of Medicare price negotiation. The objective of this commentary is to explore the implications of the IRA for US pharmaceutical companies, with a specific focus on the role of real-world evidence (RWE) in the context of Medicare reforms. METHODS: The commentary employs a qualitative analysis of the IRA's provisions related to healthcare and pharmaceutical regulation, focusing on how these reforms change the evidence requirements for pharmaceutical companies...

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...

BACKGROUND: Non-communicable diseases (NCDs) affect a large number of people globally and their burden has been growing. Healthcare for NCDs often involves high out-of-pocket expenditure and rising costs of providing services. Financing and providing care for NCDs have become a major challenge for health systems. Despite the high burden of NCDs in India, there is little information available on the costs involved in NCD care. METHODS: The study was aimed at finding out the average monthly cost of outpatient care per NCD patient...

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...

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)...

With the premise of drug safety and effectiveness, pharmacoeconomic evaluation can provide optimal solutions for diversified decision-making application scenarios from different research perspectives while maximizing the rational utilization of existing healthcare resources. Chinese patent medicine is an essential component of pharmaceutical utilization in China and a significant part of healthcare expenditure in China. However, the economic evaluation of post-marketing Chinese patent medicine is lacking. These evaluations often lack standardization, exhibit varying quality, and are unable to effectively support healthcare decisions, indicating a need for improvement in overall quality...

Based on literature and questionnaire research, related evidence and related data on Shexiang Tongxin Dropping Pills were collected in terms of safety, effectiveness, economy, innovation, suitability, and accessibility. In addition, multi-criteria decision analysis(MCDA) model was used to comprehensively evaluate the clinical value of Shexiang Tongxin Dropping Pills. Quality control was carried out strictly based on evidence-based medicine evaluation. Shexiang Tongxin Dropping Pills were recommended for stable fatigue angina of coronary heart disease with Qi deficiency and blood stasis by guidelines and experts...

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...

BACKGROUND AND OBJECTIVES: There are significant challenges when obtaining clinical and economic evidence for health technology assessments of rare diseases. Many of them have been highlighted in previous systematic reviews but they have not been summarised in a comprehensive manner. For all stakeholders working with rare diseases, it is important to be aware and understand these issues. The objective of this review is to identify the main challenges for the economic evaluation of orphan drugs in rare diseases...

OBJECTIVES: Faster regulatory approval processes often fail to achieve faster patient access. We seek an approach, using performance-based risk-sharing arrangements (PBRSA), to address uncertainty for payers regarding the relative effectiveness and value for money of products launched through accelerated approval schemes. One important reason for risk-sharing is to resolve differences of opinion between innovators and payers about a technology's underlying value. To date, there has been no formal attempt to set out the circumstances in which risk-sharing can address these differences...

OBJECTIVE: Calcitonin gene-related peptide monoclonal antibodies (CGRP mAbs) are novel high-cost treatments for the prevention of migraine. This study presents data on utilisation, expenditure and treatment patterns with CGRP mAbs available under a managed access protocol in Ireland, to a cohort of treatment refractory patients (failed three or more previous treatments) with chronic migraine. METHODS: Data was extracted from the Primary Care Reimbursement Service (PCRS) High Tech claims database and special drug request online system, and analysed using Microsoft Excel and SAS...

BACKGROUND: The current use of health economic decision models in HTA is mostly confined to single use cases, which may be inefficient and result in little consistency over different treatment comparisons, and consequently inconsistent health policy decisions, for the same disorder. Multi-use disease models (MUDMs) (other terms: generic models, whole disease models, disease models) may offer a solution. However, much is uncertain about their definition and application. The current research aimed to develop a blueprint for the application of MUDMs...

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