Journal of Market Access & Health Policy, Volume 8, Issue 1

Background: Many trials in actinic keratoses (AK) use complete clearance rate (100% reduction in number of lesions) as the primary endpoint. We explore limitations (predominantly baseline factors) associated with this outcome. Objective: This analysi...

Objective: The objective of this paper is to determine an upper price limit for an orphan drug by taken a broader perspective and, including also other monetary and non-monetary values for the society. Methods: This model is based on the expected fre...

Background: Spinal muscular atrophy (SMA) is a rare genetic neuromuscular disease. Objective: Characterize direct costs associated with SMA management. Data source: Truven Health Analytics MarketScan claims data (2012–2016). Patients: Eligible patien...

Background and objectives: Ukraine’s mental health system has been found to be inadequate and unresponsive to the needs of the population, in view of its emphasis on inpatient service delivery. This study sought to identify potential changes to the o...

Purpose: Gene therapy brings opportunities to discover cures for diseases for which there are no adequate treatments. As most gene therapies target rare diseases, several challenges are associated with their clinical development, such as limited popu...

Purpose A international registry analysis led by Mehra et al. to investigate the use of hydroxychloroquine (HCQ) and chloroquine (CQ) with or without a macrolide in 96,032 hospitalised COVID-19 patients were published on Lancet, which has raised cons...

Background: Organizational aspect is rarely considered in healthcare. However, it is gradually seen as one of the key aspects of the decision-making process as well as clinical and economic dimensions. Our primary objective was to identify criteria a...

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...

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: Non-medical switching refers to a change in a stable patient’s prescribed medication to a clinically distinct, non-generic, alternative for reasons other than poor clinical response, side-effects or non-adherence. Objective: To assess the...

Introduction: A non-medical switch is a change to a patient’s medication regimen for reasons other than lack of clinical response, side-effects or poor adherence. Specialist physicians treat complex patients who may be vulnerable to non-medical switc...

Background: Limited previous research and guidelines on the design of economic evaluation for biosimilars have led to unresolved methodological questions on how to assess biosimilars. Objectives: We want to raise awareness of and explore methodologic...

Introduction and objective: Vernal keratoconjunctivitis (VKC) is a rare allergic eye condition that occurs in children and is characterised by a combination of debilitating symptoms. Repeated use of topical corticosteroid rescue therapy is often nece...

Background: Current detection methodologies are often unable to identify the location and extent of recurrent prostate cancer (PCa) leading potentially to ‘futile’ local therapies in the presence of metastatic disease. The use of ¹⁸ F-fluciclovine PE...

The results of a clinical trial comparing hydroxychloroquine with or without azithromycin to the standard of care for the treatment of COVID-19 were recently published by Philippe Gautret et al. This study provides outstanding results for the combina...

Background: Ischaemic stroke (IS) is a major cause of death and disability and affects the quality of life of patients. Previous studies focused on urban populations. Objective: To evaluate the health-related quality of life (QoL) of patients with hi...

Background: Morbidity and mortality associated with non-valvular atrial fibrillation (NVAF) imposes a substantial economic burden on the UK healthcare system. Objectives: An existing Markov model was adapted to assess the real-world cost-effectivenes...

Background: In many countries, Budget Impact (BI) informs reimbursement decisions. Evidence has shown that decision-makers have restricted access based on high BI estimates but studies show that BI estimates are often inaccurate. Objective: To assess...

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...

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...

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...

Background: Cost-effectiveness analysis (CEA) is the economic analysis method most commonly applied today in the context of replacing one treatment with a new one in a developed healthcare system to improve efficiency. CEA is often requested by local...

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...