The correlation between HTA recommendations and reimbursement status of orphan drugs in Europe [PDF]
, 2016 Background The aim of this study was to review and compare types of reimbursement recommendations for orphan drugs issued by eight European health technology assessment (HTA) agencies and the reimbursement status of these drugs in the corresponding ...
Kawalec, Paweł +2 more
core +2 more sources
Systematic review of available evidence on 11 high-priced inpatient orphan drugs [PDF]
, 2013 __Background__: Attention for Evidence Based Medicine (EBM) is growing, but evidence for orphan drugs is argued to be limited and inferior. This study systematically reviews the available evidence on clinical effectiveness, costeffectiveness and budget ...
Hakkaart-van Roijen, L. (Leona) +3 more
core +3 more sources
Saudi Pharmaceutical Journal, 2023 Background: Rare diseases are chronic, serious, and life-threatening conditions that have not received sufficient attention from drug developers due to their rarity.
Bander Balkhi +2 more
doaj +1 more source
Long way to go: Progress of orphan drug accessibility in China from 2017 to 2022
Frontiers in Pharmacology, 2023 Introduction: Over 400 million patients worldwide suffer from rare diseases. Access to orphan drugs is, therefore, crucial for this population. China has been actively working on improving orphan drug accessibility in the past decades, especially since ...
Jia Liu +7 more
doaj +1 more source
Hangsúlyeltolódások a hazai gyógyszerek finanszírozásában. A ritka betegségek kezelésére szolgáló árva gyógyszerek támogatása. Enzimpótló kezelések finanszírozása hazánkban [PDF]
, 2015 Focusing on the benefits of patients with rare disease the authors analysed the aspects of orphan medicines financed in the frame of the Hungarian social insurance system in 2012 in order to make the consumption more rational, transparent and predictable.
Boncz, Imre +3 more
core +1 more source
G protein-coupled receptor 35: an emerging target in inflammatory and cardiovascular disease [PDF]
, 2015 G protein-coupled receptor 35 (GPR35) is an orphan receptor, discovered in 1998, that has garnered interest as a potential therapeutic target through its association with a range of diseases.
Amanda E Mackenzie +4 more
core +3 more sources
Orphan drug: Development trends and strategies
Journal of Pharmacy and Bioallied Sciences, 2010 The growth of pharma industries has slowed in recent years because of various reasons such as patent expiries, generic competition, drying pipelines, and increasingly stringent regulatory guidelines. Many blockbuster drugs will loose their exclusivity in
Aarti Sharma +3 more
doaj +1 more source
Pricing and reimbursement of orphan drugs: the need for more transparency
Orphanet Journal of Rare Diseases, 2011 Pricing and reimbursement of orphan drugs are an issue of high priority for policy makers, legislators, health care professionals, industry leaders, academics and patients.
Simoens Steven
doaj +1 more source
Successful private–public funding of paediatric medicines research: lessons from the EU programme to fund research into off-patent medicines [PDF]
, 2014 The European Paediatric Regulation mandated the European Commission to fund research on off-patent medicines with demonstrated therapeutic interest for children.
Baiardi, P +17 more
core +1 more source
Rare diseases and orphan drugs
Annali dell'Istituto Superiore di Sanità, 2011 According to the Regulation (EC) N. 141/2000 of the European Parliament and of the Council, rare diseases are life-threatening or chronically debilitating conditions, affecting no more than 5 in 10 000 persons in the European Community.
Domenica Taruscio +2 more
doaj +1 more source