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Journal of Market Access & Health Policy

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https://www.readbyqxmd.com/read/28473889/filling-the-gap-in-cns-drug-development-evaluation-of-the-role-of-drug-repurposing
#1
A Caban, K Pisarczyk, K Kopacz, A Kapuśniak, M Toumi, C Rémuzat, A Kornfeld
Background and objective: 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 of this article is to assess the extent and meaning of this process in the CNS area. Methods: In order to identify repurposed drugs that target the CNS, an extensive search was performed. For each identified case, its initial and target indication, development status and the type of repurposing strategy (repositioning, reformulation or both) was recorded...
2017: Journal of Market Access & Health Policy
https://www.readbyqxmd.com/read/28473888/do-payers-value-rarity-an-analysis-of-the-relationship-between-disease-rarity-and-orphan-drug-prices-in-europe
#2
Goran Medic, Daria Korchagina, Katherine Eve Young, Mondher Toumi, Maarten Jacobus Postma, Micheline Wille, Michiel Hemels
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 relationship between the prevalence of rare diseases and the annual treatment cost of orphan drugs in France, Germany, Italy, Norway, Spain, Sweden, and UK. Methods: Approved orphan drugs and prevalence data were extracted from the European Medicines Agency website. Annual treatment costs were calculated using ex-factory price...
2017: Journal of Market Access & Health Policy
https://www.readbyqxmd.com/read/28473887/a-comparative-study-of-orphan-drug-prices-in-europe
#3
Katherine Eve Young, Imen Soussi, Michiel Hemels, Mondher Toumi
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 using ex-factory prices from IHS POLI and country price databases. The treatment cost in the comparator country was compared to the UK and ratios were analysed. Subanalyses were done on disease areas and UK cost quartiles. Results: 120 orphan drugs were included...
2017: Journal of Market Access & Health Policy
https://www.readbyqxmd.com/read/28321285/role-of-preliminary-registry-data-in-development-of-a-clinical-trial-for-an-innovative-device-a-small-but-integral-piece-of-a-health-policy-initiative
#4
Donald R Ricci, Joost de Vries, Raphael Blanc
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 evolves to testing in humans. The pathway to a formal prospective clinical trial includes a stepwise appreciation of pre-clinical data and detailed analysis of data obtained from preliminary registries, where information about appropriate patient selection and use of the device is obtained...
2017: Journal of Market Access & Health Policy
https://www.readbyqxmd.com/read/28265350/cost-effectiveness-of-bedaquiline-in-mdr-and-xdr-tuberculosis-in-italy
#5
Luigi R Codecasa, Mondher Toumi, Anna D'Ausilio, Andrea Aiello, Francesco Damele, Roberta Termini, Alessia Uglietti, Robert Hettle, Giorgio Graziano, Saverio De Lorenzo
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 Italian setting to estimate the incremental cost-effectiveness ratio (ICER) of bedaquiline plus BR (BBR) versus BR in the treatment of MDR-TB and XDR-TB over 10 years, from both the National Health Service (NHS) and societal perspective. Cost-effectiveness was evaluated in terms of life-years gained (LYG)...
2017: Journal of Market Access & Health Policy
https://www.readbyqxmd.com/read/28265349/key-drivers-for-market-penetration-of-biosimilars-in-europe
#6
Cécile Rémuzat, Julie Dorey, Olivier Cristeau, Dan Ionescu, Guerric Radière, Mondher Toumi
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 available biosimilars in the European Union (EU). Methods: A three-step process was established to identify key drivers for the uptake of biosimilars in the top 10 EU member states (MS) pharmaceutical markets (Belgium, France, Germany, Greece, Hungary, Italy, Poland, Spain, Sweden, and the UK): (1) literature review to identify incentive policies in place to enhance biosimilars adoption; (2) assessment of biosimilar market dynamics based on database analysis; (3) regression model analysis on price using the following explicative variables: incentive policies; price difference between the biosimilar and the originator product; distribution channel; generic uptake and generic price cut; pharmaceutical expenditure per capita; and market competition...
2017: Journal of Market Access & Health Policy
https://www.readbyqxmd.com/read/28265348/gene-therapies-development-slow-progress-and-promising-prospect
#7
Eve Hanna, Cécile Rémuzat, Pascal Auquier, Mondher Toumi
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 objective of this study was to review the descriptive data on gene therapy clinical trials conducted worldwide between 1989 and 2015, and to discuss potential success rates of these trials over time and anticipated market launch in the upcoming years. Methods: A publicly available database, 'Gene Therapy Clinical Trials Worldwide', was used to extract descriptive data on gene therapy clinical trials: (1) number of trials per year between 1989 and 2015; (2) countries; (3) diseases targeted by gene therapies; (4) vectors used for gene delivery; (5) trials status; (6) phases of development...
2017: Journal of Market Access & Health Policy
https://www.readbyqxmd.com/read/28265347/value-added-medicines-what-value-repurposed-medicines-might-bring-to-society
#8
Mondher Toumi, Cécile Rémuzat
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 further development. The objectives of the present study were to assess from a wide perspective which value drug repurposing might bring to society, but also to identify key obstacles for adoption of these medicines and to discuss policy recommendations. Methods: A preliminary comprehensive search was conducted to assess how the concept of drug repurposing was described in the literature...
2017: Journal of Market Access & Health Policy
https://www.readbyqxmd.com/read/28003867/what-makes-a-compliant-phase-iii-and-pre-launch-patient-advocacy-strategy
#9
Nick Hicks, Keith Allan, Christoph Thalheim, Paul Woods
BACKGROUND: A key task for the pharmaceutical industry is to understand the compliance implications of engaging with a patient advocacy group (PAG). This presents challenges for the industry to negotiate the ethical and reputational issues that can arise when working with a PAG. OBJECTIVE: To gain the views of pharmaceutical industry executives on future compliance challenges when working with PAGs. STUDY DESIGN: We conducted two surveys among two sets of industry executives: one group focussed on market access roles and the other focussed on non-market access roles...
2016: Journal of Market Access & Health Policy
https://www.readbyqxmd.com/read/27867466/a-training-paradigm-to-enhance-performance-and-safe-use-of-an-innovative-neuroendovascular-device
#10
Donald R Ricci, Thomas R Marotta, Howard A Riina, Martina Wan, Joost De Vries
Training has been important to facilitate the safe use of new devices designed to repair vascular structures. This paper outlines the generic elements of a training program for vascular devices and uses as an example the actual training requirements for a novel device developed for the treatment of bifurcation intracranial aneurysms. Critical elements of the program include awareness of the clinical problem, technical features of device, case selection, and use of a simulator. Formal proctoring, evaluation of the training, and recording the clinical outcomes complement these elements...
2016: Journal of Market Access & Health Policy
https://www.readbyqxmd.com/read/27857828/determining-the-value-of-medical-technologies-to-treat-ultra-rare-disorders-a-consensus-statement
#11
Michael Schlander, Silvio Garattini, Peter Kolominsky-Rabas, Erik Nord, Ulf Persson, Maarten Postma, Jeff Richardson, Steven Simoens, Oriol de Solà-Morales, Keith Tolley, Mondher Toumi
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 about the economic burden that might be caused by an annual cost per patient in some cases exceeding 100,000 Euro. At the same time, many drugs for rare disorders do not meet conventional standards for cost-effectiveness or 'value for money'...
2016: Journal of Market Access & Health Policy
https://www.readbyqxmd.com/read/27857827/perceptions-and-factors-affecting-pharmaceutical-market-access-results-from-a-literature-review-and-survey-of-stakeholders-in-different-settings
#12
Semukaya Sendyona, Isaac Odeyemi, Khaled Maman
BACKGROUND: A change in the pharmaceutical environment has occurred from previously only needing to convince regulators of a product's safety and efficacy to obtain marketing authorisation to now needing to satisfy the value perceptions of other stakeholders, including payers, to attain market access for products. There is thus the need to understand the concept of market access that may be defined as 'the process that ensures the development and commercial availability of pharmaceutical products with appropriate value propositions, leading to their prescribing and to successful uptake decisions by payers and patients, with the ultimate goal of achieving profitability and best patient outcomes'...
2016: Journal of Market Access & Health Policy
https://www.readbyqxmd.com/read/27826381/the-quality-of-life-of-spanish-patients-with-huntington-s-disease-measured-with-h-qol-i-and-eq-5d
#13
Julie Dorey, Emilie Clay, Amine Khemiri, Anis Belhadj, Patricia Trigo Cubillo, Mondher Toumi
BACKGROUND AND OBJECTIVE: Huntington's disease (HD) is an inherited neurodegenerative disorder that heavily affects the patient's motor, cognitive, and psychological functions. Yet, very few studies have measured the impact of this disease on the patient's health-related quality of life (HRQoL) with specific and validated instruments. The aim of this study was to explore the impact of HD on the HRQoL of Spanish HD patients using the self-reported, Huntington Quality of Life Instrument (H-QoL-I) and the generic instrument EuroQoL five dimensions (EQ-5D-3L) and thereafter compare the results obtained with the two instruments...
2016: Journal of Market Access & Health Policy
https://www.readbyqxmd.com/read/27695606/a-price-comparison-of-recently-launched-proprietary-pharmaceuticals-in-the-uk-and-the-us
#14
Jesper Jørgensen, Panos Kefalas
OBJECTIVE: To explore the relationship between prices charged by manufacturers of proprietary pharmaceuticals in the US and in the UK in recent years (2013-2016), expressed as a multiplier, and to detail to what extent this relationship differs for high-cost therapies used in smaller patient populations, as compared to lower-cost drugs. METHODOLOGY: Therapies assessed by the Scottish Medicines Consortium (SMC) in the UK between 1 January 2013 and 1 June 2016 were identified; only in-patent therapies were included in the analysis (to avoid the impact of price erosion post patent expiry); results were grouped according to annual cost per patient (whether considered high-cost, i...
2016: Journal of Market Access & Health Policy
https://www.readbyqxmd.com/read/27570614/risk-of-discontinuation-of-advanced-therapy-medicinal-products-clinical-trials
#15
Eve Hanna, Cecile Rémuzat, Pascal Auquier, Mondher Toumi
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-commercial sponsors in this field and a growing number of ATMPs randomized clinical trials (RCT) and patients enrolled in such trials. RCT generate data to prove the efficacy of a new therapy, but the discontinuation of RCTs wastes scarce resources...
2016: Journal of Market Access & Health Policy
https://www.readbyqxmd.com/read/27489586/challenges-in-striving-to-simultaneously-achieve-multiple-resource-allocation-goals-the-pan-canadian-oncology-drug-review-pcodr-example
#16
Heather McDonald, Cathy Charles, Laurie Elit, Amiram Gafni
The pan-Canadian Oncology Drug Review (pCODR) makes recommendations to Canada's provinces and territories (except Quebec) to guide their cancer drug funding decisions. The objective of this paper is to explore, using an economic perspective and the pCODR as an example, the challenges associated with striving to simultaneously achieve the goals of maximizing health benefits with available resources and improving access to a more consistent standard of care across Canada. The first challenge concerns how to interpret the goals in order to determine how resources should be allocated to achieve each goal...
2016: Journal of Market Access & Health Policy
https://www.readbyqxmd.com/read/27489585/demonstrating-the-value-of-medicines-evolution-of-value-equation-and-stakeholder-perception-of-uncertainties
#17
Siva Narayanan
It is important to evaluate how the value of medicine is assessed, as it may have important implications for health technology and reimbursement assessments. The value equation could comprise 'incremental benefit/outcome' (relative results of care in terms of patient health, comparing the innovation to best available alternative(s)) in the numerator and 'cost' (relative costs involved in the full cycle of care (or a defined period) for the patient's medical condition, incorporating the relevant cost-offsets due to displacement of best available alternative(s)) in the denominator...
2016: Journal of Market Access & Health Policy
https://www.readbyqxmd.com/read/27429720/cost-effectiveness-analysis-of-the-direct-and-indirect-impact-of-intranasal-live-attenuated-influenza-vaccination-strategies-in-children-alternative-country-profiles
#18
Edward Gibson, Najida Begum, Federico Martinón-Torres, Marco Aurélio Safadi, Alfred Sackeyfio, Judith Hackett, Sankarasubramanian Rajaram
BACKGROUND: Influenza poses a significant burden on healthcare systems and society, with under-recognition in the paediatric population. Existing vaccination policies (largely) target the elderly and other risk groups where complications may arise. OBJECTIVE: The goal of this study was to evaluate the cost-effectiveness of annual paediatric vaccination (in 2-17-year-olds) with live attenuated influenza vaccination (LAIV), as well as the protective effect on the wider population in England and Wales (base)...
2016: Journal of Market Access & Health Policy
https://www.readbyqxmd.com/read/27429719/cost-effectiveness-of-ocriplasmin-for-the-treatment-of-vitreomacular-traction-and-macular-hole
#19
Craig Bennison, Stephanie Stephens, Benedicte Lescrauwaet, Ben Van Hout, Timothy L Jackson
BACKGROUND: If left untreated, vitreomacular traction (VMT) will infrequently improve through spontaneous resolution of vitreomacular adhesion (VMA), and patients remain at risk of further deterioration in vision. The mainstay of treatment for VMT is vitrectomy, an invasive procedure that carries the risk of rare but serious complications and further vision loss. As such, a 'watch and wait' approach is often adopted before this surgical intervention is performed. Ocriplasmin (microplasmin) is a potential alternative treatment for patients with symptomatic VMT that may remove the requirement for vitrectomy...
2016: Journal of Market Access & Health Policy
https://www.readbyqxmd.com/read/27386054/validation-of-disease-states-in-schizophrenia-comparison-of-cluster-analysis-between-us-and-european-populations
#20
Katia Thokagevistk, Aurélie Millier, Leslie Lenert, Shamil Sadikhov, Santiago Moreno, Mondher Toumi
BACKGROUND: There is controversy as to whether use of statistical clustering methods to identify common disease patterns in schizophrenia identifies patterns generalizable across countries. OBJECTIVE: The goal of this study was to compare disease states identified in a published study (Mohr/Lenert, 2004) considering US patients to disease states in a European cohort (EuroSC) considering English, French, and German patients. METHODS: Using methods paralleling those in Mohr/Lenert, we conducted a principal component analysis (PCA) on Positive and Negative Syndrome Scale items in the EuroSC data set (n=1,208), followed by k-means cluster analyses and a search for an optimal k...
2016: Journal of Market Access & Health Policy
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