Journal of Market Access & Health Policy, Volume 5, Issue 1

Background: An elevated level of low-density lipoprotein cholesterol (LDL-C) constitutes one of the most important modifiable risk factors for cardiovascular disease (CVD). Individuals with heterozygous familial hypercholesterolaemia (HeFH) are parti...

Background: Adverse event (AE) reporting in clinical trials does not fully capture the patient-level perspective and comparing tolerability across treatments or among studies is difficult. Objective: This study was designed to develop a treatment tol...

Background: It is unclear if the burden associated with schizophrenia is affected by the type and severity of patient’s symptoms. Objective: This study aims to quantify healthcare resource use associated with different profiles of schizophrenia sympt...

In Poland, two proposed amendments to the reimbursement act are currently in preparation; these are likely to substantially change the pricing and reimbursement landscape for both drugs and medical devices. Proposed changes include: alignment of medi...

Background and Objective: Ibrutinib has recently been approved in Europe for Waldenström Macroglobulinemia (WM) in symptomatic patients who have received at least one prior therapy, or in first-line treatment for patients unsuitable for chemo-immunot...

Background: Policies to reduce methicillin-resistant Staphylococcus aureus (MRSA) infections, both healthcare-acquired (HA-MRSA) and livestock-associated (LA-MRSA) are implemented Europe-wide, but evaluations are difficult for countries yet to implem...

In 2014, balanced assessment systems (BAS) were proposed as a resource-conscious, ‘fit-for-purpose’ form of health technology assessment for middle-income countries which lack resources and competences necessary for resource-intensive health technolo...

Introduction: Health technology assessment (HTA) in Poland supports reimbursement decisions via the Polish HTA Agency (AOTMiT), whose guidelines were updated in 2016. Methods: We identified key changes introduced by the update and, before guideline 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 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...

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 and objective: Automated healthcare databases (AHDB) are an important data source for real life drug and healthcare use. In the filed of depression, lack of detailed clinical data requires the use of binary proxies with important limitatio...

​Since we were born, we all take preventative actions to avoid unpredictable adverse conditions. Some actions are done automatically. Others require a conscious choice , either for personal or social benefit. A distinction can therefore be drawn betw...

This paper describes how the economic assessment of vaccines is performed today. It discusses why it may be incomplete and explores potential approaches to adjust the analysis to be more comprehensive. Besides helping protect against serious disease,...

Background: In 2015, Ikervis® became the only EMA-approved cyclosporine A (CsA) eye-drop for the treatment of severe keratitis in adult patients with dry eye disease, which has not improved despite treatment with tear substitutes. Since the 1980s, Cs...

In two previous papers we argued on current vaccines economic assessment not fully comprehensive when using the incremental cost-utility analysis normally applied for treatments. Many differences exist between vaccines and drug treatments making vacc...

Background: Cost-containment initiatives are re-shaping the pharmaceutical business environment and affecting market access as well as pricing and reimbursement decisions. Effective price management procedures are too complex to accomplish manually....

Background: Despite the guidelines for Economic and Public Health Assessment Committee (CEESP) submission having been available for nearly six years, the dossiers submitted continue to deviate from them, potentially impacting product prices. Objectiv...

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...

Background: To date, no specific instruments exist to measure the quality of the patient-surgeon relationship despite its potential to influence clinical and economic outcomes in patients undergoing surgery for musculoskeletal disorders (MSDs). Objec...

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...

Global disparities in medical technologies, laws, economic inequities, and social–cultural differences drive medical tourism (MT), the practice of travelling to consume healthcare that is either too delayed, unavailable, unaffordable or legally prosc...

New health technology comes on the market at a rapid pace and—sometimes—at a huge cost. Providing access to new health technology is a serious challenge for many countries with mandatory health insurance. This article analyses access to new health te...

Background: Lennox-Gastaut syndrome (LGS) is a severe form of childhood-onset epilepsy associated with serious injuries due to frequent and severe seizures. Of the antiepileptic drugs (AEDs) approved for LGS, clobazam is a more recent market entrant,...

Background: Hematopoietic stem cell transplantation (HSCT) is a medically complicated therapy with a long recovery time. In Tunisia, the National Health Insurance Fund (CNAM) covers only the first year post-transplantation, after which the costs are...

Establishing a national health policy at a macro level involves the integration of a series of health initiatives across a spectrum of activities, including clinical care. Evaluation of the safety and efficacy of a new medical device ultimately evolv...

Background and Objective: This study assessed price differences by comparing annual treatment costs of similarly available orphan drugs in France, Germany, Italy, Norway, Spain, Sweden, and UK. Methods: Annual treatment costs per drug were calculated...

Background: Many established products (EPs – marketed for eight years or more) are widely used off-label despite little evidence on benefit–risk ratio. This exposes patients to risks related to safety and lack of efficacy, and healthcare providers to...

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...

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: 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...

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...

Background: Orphan drugs (ODs) are pharmaceuticals manufactured for rare conditions that affect less than 200,000 people in the US. ODs are therefore produced in small quantities to meet sparse demand. Since 2010, OD shortages have become frequent, b...

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...

Objective: To evaluate the cost-effectiveness of bedaquiline plus background drug regimens (BR) for multidrug-resistant tuberculosis (MDR-TB) and extensively drug-resistant tuberculosis (XDR-TB) in Italy. Methods: A Markov model was adapted to the It...