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Medicinski pregled, 2013 Introduction. Drugs used for treatment of rare diseases are known worldwide under the term of orphan drugs because pharmaceutical companies have not been interested in ?adopting? them, that is in investing in research, developing and producing these drugs.
Svetlana Golocorbin-Kon +4 more
+8 more sources
Nature, 1992 Diseases lacking in satisfactory therapies, named orphans (over 60% of the known), raise the problem of the availability of new drugs to be discovered and evaluated on toxicological and clinical bases. The estimated cost for the full development of a drug makes it not profitable (and therefore orphan drug) in the case of rare diseases and the typical ...
openaire +4 more sources
QJM, 2006 Hughes et al .1 recently discussed arguments for and against giving special funding status to orphan drugs in this journal. They concluded that there should be a uniform policy across Europe, that complete restriction was impractical, and that UK policy should aspire to the values of the EU directive.
McCabe, C. +3 more
openaire +3 more sources
Orphan drugs and the NHS: Should we value rarity [PDF]
, 2005 Cost effectiveness plays an important part in current decisions about the funding of health technologies. Drugs for rare disease (orphan drugs) are often expensive to produce and, by definition, will benefit only small numbers of patients.
Claxton, K., McCabe, C., Tsuchiya, A.
core +2 more sources
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
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
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