Most Cited

Vaccination has made an important contribution to the decreased incidence of numerous infectious diseases and associated mortality. In 2013, it was estimated that 103 million cases of childhood diseases in the United States had been prevented by the...

Objective: To understand the different methodologies used to elicit willingness to pay for health and the value of a statistical life year through surveys. Methodology: A systematic review of the literature was undertaken to identify studies using su...

Introduction: A rapid literature review (RLR) is an alternative to systematic literature review (SLR) that can speed up the analysis of newly published data. The objective was to identify and summarize available information regarding differ...

Background: In 1989, the concept of human gene therapies has emerged with the first approved human gene therapy trial of Rosenberg et al. Gene therapies are considered as promising therapies applicable to a broad range of diseases. Objective: The obj...

The importance of understanding the impact of disease and treatment on children’s Health-Related Quality of Life (HRQoL) has given rise to an increasing use of child self-report and observer or proxy instruments. In this article, we review the status...

Background: The experience of Kymriah^® and Yescarta^® provides real-world examples of how health-care systems approach and manage the reimbursement of one-off, high-cost, cell, and gene therapies, and the decision uncertainty and affordability challen...

Background and objectives: External reference pricing (ERP) is a price regulation tool widely used by policy makers in the European Union (EU) Member States (MS) to contain drug cost, although in theory, it may contribute to modulate prices up and do...

Background: Advanced therapy medicinal products (ATMPs) are innovative therapies that encompass gene therapy, somatic cell therapy, and tissue-engineered products. These therapies are expected to bring important health benefits, but also to substanti...

Background & Objectives: Potential drivers and barriers of biosimilar uptake were mainly analysed through qualitative approaches. The study objective was to conduct a quantitative analysis and identify drivers of biosimilar uptake of all availabl...

Background: Spinal muscular atrophy type 1 (SMA1) is a devastating genetic disease for which gene-replacement therapy may bring substantial survival and quality of life benefits. Objective: This study investigated the cost-effectiveness of onasemnog...

Theoretical and empirical evidence has demon- strated that health has a major role to play as a driver for economic growth [...]

A heated discussion has recently broken out in Europe about the price of Zolgensma, ‘the most expensive drug ever’ [...]

Background: Medicinal products that have been developed and approved for one disease may be the object of additional clinical development in other disease areas or of additional pharmaceutical development for new and different formulations. The newly...

Background: Artificial intelligence (AI) enables remote patient monitoring (RPM) which reduces costs by triaging patients to optimize hospitalization and avoid complications. The FDA regulates AI in medical devices and aims to ensure patien...

Objective: Doctor-shopping has significant consequences for patients and payers and can indicate misuse of drugs, polypharmacy, less continuity of care, and increased medical expenses. This study reviewed the literature describing doctor-shoppers in...

Objective: This study aimed to provide an overview of biosimilar policies in 10 EU MSs. Methods: Ten EU MS pharmaceutical markets (Belgium, France, Germany, Greece, Hungary, Italy, Poland, Spain, Sweden, and the UK) were selected. A comprehensive lit...

Background: Repurposing has become a mainstream strategy in drug development, but it faces multiple challenges, amongst them the increasing and ever changing regulatory framework. This is the second study of a series of three-part publication project...

Objective: The aim of this research is to identify the pricing, reimbursement, and market access (P&R&MA) considerations most relevant to advanced therapy medicinal products (ATMPs) in the Big5EU, and to inform their manufacturers about the k...

Objective: Advanced therapy medicinal products (ATMPs) constitute a class of innovative products that encompasses gene therapy, somatic cell therapy, and tissue-engineered products (TEP). There is an increased investment of commercial and non-commerc...

Background: Recent cost-utility analysis (CUA) models for onasemnogene abeparvovec (Zolgensma^®, formerly AVXS-101) in spinal muscular atrophy type 1 (SMA1) differ on key assumptions and results. Objective: To compare the manufacturer’s proprietary C...

The use of vaccines to prevent diseases in children, adults, and the elderly results in fewer medical visits, diagnostic tests, treatments, and hospitalisations, which leads to substantial savings in healthcare costs each year in Europe and elsewhere...

Background and objectives: MCDA is a decision-making tool with increasing use in the healthcare sector, including HTA (Health Technology Assessment). By applying multiple criteria, including innovation, in a comprehensive, structured and explicit man...

Background and objectives: Governments in Asia Pacific (APAC) are increasingly using pharmaceutical pricing strategies to contain rising healthcare costs. The objective of this narrative review is to discuss formal pricing strategies for reimbursed p...

Background: Cell and gene therapies have the potential to provide therapeutic breakthroughs, but the high costs of researching, developing, manufacturing and delivering them translate into prices that may challenge healthcare budgets. Various measure...

Background: The umbrella term ATMPs (Advanced Therapy Medicinal Products) comprises cell therapies, gene therapeutics and tissue engineered products. After implementation of the Regulation 1394/2007, only a couple of products have obtained a centrali...

The health of a population is important from a public health and economic perspective as healthy individuals contribute to economic growth. Vaccination has the potential to contribute substantially to improving population health and thereby economic...

Background and objective: Drug repurposing has been considered a cost-effective and reduced-risk strategy for developing new drugs. Little is known and documented regarding the efficiency of repurposing strategies in drug development. The objective o...

Background & Objectives: Biological disease-modifying antirheumatic drugs (bDMARDs) for the treatment of rheumatoid arthritis (RA) are not always accessible to all patients in accordance with international guidelines, partly owing to their high d...

Introduction: The Indian healthcare system is evolving towards better healthcare implementation and coverage. However, even today, the health-care system faces several challenges, a few of which are yet to be addressed. The present review is aimed to...

Background: Some orphan drugs can cost hundreds of thousands of dollars annually per patient. As a result, payer sensitivity to the cost of orphan drugs is rising, particularly in light of increased numbers of new launches in recent years. In this ar...

The economic evaluation which supports Health Technology Assessment (HTA) should inform policy makers of the value to society conferred by a given allocation of resources. However, neither the theory nor practise of economic evaluation satisfactorily...

Introduction: Health technology assessment (HTA) agencies express a clear preference for randomized controlled trials when assessing the comparative efficacy of two or more treatments. However, an indirect treatment comparison (ITC) is often necessar...

Objective: Since 2004, the National Institute of Health and Clinical Excellence (NICE) has required manufacturers to conduct a probabilistic sensitivity analysis (PSA) in their technology appraisals. The objective of this review is to assess the cost...

Background and Objective: Orphan drugs have been a highlight of discussions due to their higher prices than non-orphan drugs. There is currently no European consensus on the method of value assessment for orphan drugs. This study assessed the relatio...

Introduction: Numerous real-world studies have compared non-vitamin K antagonist oral anticoagulants (NOACs) with vitamin K antagonists (VKAs) in patients with non-valvular atrial fibrillation (NVAF). A meta-analysis was performed to synthesize the a...

Quantitative patient preferences are a method to involve patients in regulatory benefit-risk assessment. Assuming preferences can be elicited, there might be multiple advantages to their use. Legal, methodological and procedural issues do however imp...

Background: Dementia has become a growing health-care problem in the rapidly ageing Japanese population. This study assesses the impact of dementia on quality of life, economic burden, and productivity loss. Objective: The objective of this study was...

Because of significant differences in institutional contexts, health technology assessment (HTA) systems that are in place in core pharmaceutical markets may not be suitable, fully or in part, for middle-income countries (MICs) and for other noncore...

Objectives: We aimed to conduct a modelling study to estimate and predict the economic burden of AD to support the healthcare management of AD in China. Methods: The economic burden of AD was estimated with an evaluation of the prevalence of AD patie...

Indication value-based pricing (IBP) has been proposed in the United States as a tool to capture the differential value of drugs across indications or patient groups and is in the early phases of implementation. In Europe, no major country has experi...

Background and Objective: China has managed to control the coronavirus disease (COVID-19) with confinement measurements and treatment strategies, while other countries are struggling to contain the spread. This study discusses the guidelines related...

Background & objectives: Despite the wide interest surrounding drug repurposing, no common terminology has been yet agreed for these products and their full potential value is not always recognised and rewarded, creating a disincentive for furthe...

Introduction: Prolonged Hospital Length of Stay (PLOS) is an indicator of deteriorated efficiency in Quality of Care. One goal of public health management is to reduce PLOS by identifying its most relevant predictors. The objective of this...

Objective: We describe and compare the availability and accessibility of administrative healthcare databases (AHDB) in several Asia-Pacific countries: Australia, Japan, South Korea, Taiwan, Singapore, China, Thailand, and Malaysia. Methods: The study...

Objective: The study compared the relative cost differences of similar orphan drugs among high and low GDP countries in Europe: Bulgaria, France, Germany, Greece, Hungary, Italy, Norway, Poland, Romania, Spain, Sweden, UK. Methods: Annual treatment c...

Background: Health technology assessment (HTA) has been reinforced in France, notably with the introduction of economic evaluation in the pricing process for the most innovative and expensive treatments. Similarly to the National Institute for Clinic...

Background: In most jurisdictions, policies have been adopted to encourage the development of treatments for rare or orphan diseases. While successful as assessed against their primary objective, these policies have prompted concerns among payers abo...

Background: Access to biologic medicines (including biosimilars) across Europe is largely governed by a process of tendering conducted by health authorities. Over-reliance on treatment costs in awarding tenders has the potential to hinder competition...

Objective: To provide recommendations for addressing previously identified key challenges in health economic evaluations of Gene Replacement Therapies (GRTs), including: 1) the assessment of clinical effectiveness; 2) the valuation of health outcomes...

Background: The use of pharmacokinetics is associated with cost savings in anti-tumor necrosis factor (anti-TNF) therapy, but the long-term cost savings in a large cohort of Crohn's disease (CD) patients are unknown. Aim: The goal of this study was t...