Orphan Drugs
ORPHAN DRUGS: NEED OF AN HOUR
... unprofitable drugs that were taken off the market, but later were reintroduced as a result of pressure from physicians, patients and other health ...these drugs is an orphan, either based on the ...
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Reimbursement of orphan drugs in Belgium: what (else) matters?
... Multi-criteria decision analysis (MCDA) has been pro- posed as a framework to evaluate new (orphan) drugs for reimbursement based on a combination of several criteria. In an MCDA, the relevant criteria and ...
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Effectiveness, safety and costs of orphan drugs: an evidence-based review
... of orphan drugs are reportedly due to the large amounts of funds invested in R&D by the drug ...branded drugs are compared with their generic versions makes it doubtful if the quoted R&D ...
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Orphan drugs program. Discussion paper. May 2015
... the Orphan drugs program was established it was generally recognised that support was required to bring these products to market due to low demand and the lack of financial incentive to develop or market a ...
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Rare diseases and orphan drugs in eastern european countries
... Results: The awareness and public health initiatives on rare diseases in Eastern European countries are generally low. EEC, which are also EU member states provide comparatively better healthcare services to people with ...
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Orphan drugs and the NHS: Should we value rarity
... for drugs with multiple indications ( M Hafner, Royal College of Phy- sicians conference on managing rare diseases, London, 5 October ...for orphan drugs ...
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Orphan drugs expenditure in the Netherlands in the period 2006–2012
... outpatient drugs we examined the Drug Information Project (GIP) database hosted by the Health Care Insur- ance Board (CVZ) ...Some orphan drugs were also prescribed for non-orphan ...these ...
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The availability and affordability of orphan drugs for rare diseases in China
... each orphan drug from the surveyed results of 24 public ...each orphan drug based on the package insert information, literature review and expert medical ...some orphan drugs, which refers to ...
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Determinants of orphan drugs prices in France: a regression analysis
... for orphan drugs [5, 34, ...describe orphan drug prices based on prede- fined disease and drug characteristics using a robust methodology remains very ...studied orphan drug prices in five ...
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Trends in individual reimbursement of orphan drugs in Latvia in 2008–2011
... Abstract. Orphan drugs (ODs) are medicinal products intended for diagnosis, prevention or treatment of life-threatening or very serious diseases affecting less than 5 in 10 000 people in the European Union ...
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Points to consider: efficacy and safety evaluations in the clinical development of ultra-orphan drugs
... Patient registries are actively implemented in the EU and the U.S., and mainly patients’ organizations are building these registries, such as the Orphanet [29] in the EU. The patient organizations in Japan are not ag- ...
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Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients’ health needs?
... marketed orphan drugs address only a fraction of the large number of rare diseases and conditions that affect millions of patients’ worldwide showing that the unmet needs of the rare disease patient ...
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<p>Orphan Drugs In Development For The Treatment Of Friedreich’s Ataxia: Focus On Omaveloxolone</p>
... an orphan disease, a label ascribed to conditions that affect less than 200,000 people in the United ...effective drugs, and often exorbitant costs for the few treatments that do ...“ orphan ...
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Multi-criteria decision analysis (MCDA): testing a proposed MCDA framework for orphan drugs
... This study sought to test the MCDA framework pro- posed by Hughes-Wilson et al. [8] to establish if such a simple framework, both in the suggested criteria and the simplistic scoring system, could be used to inform in ...
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Revealed preferences towards the appraisal of orphan drugs in Poland - multi criteria decision analysis
... In addition to MCDA studies, there are some publica- tions that provide insight into the value attributes used by manufacturers in price setting for OMPs as well. There- fore, some comparisons can be made between the ...
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Systematic review of available evidence on 11 high-priced inpatient orphan drugs
... on drugs has grown over the years, also for policy decisions concerning drugs for rare diseases ...(i.e. orphan drugs). Compared to the evi- dence base for common drugs, however, the ...
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The correlation between HTA recommendations and reimbursement status of orphan drugs in Europe
... of drugs and let allocate public cover- age on pharmacotherapy which should be reimbursed in the lack of sufficient financial resources; we speculate that in coming years in all EU member states such as- sessment ...
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The quality of economic evaluations of ultra-orphan drugs in Europe – a systematic review
... no drugs have been withdrawn from being reimbursed because of their unfavourable ICUR in the ...non-orphan drugs [33]. The value of ultra- orphan drugs is assessed in a ‘Highly ...
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The influence of the European paediatric regulation on marketing authorisation of orphan drugs for children
... Drugs for rare diseases are classified as orphan drugs (ODs). Developing ODs is very challenging. This is mainly due to the various factors that limit clinical stud- ies such as the small number of ...
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Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries
... to orphan drugs, there is a paucity of available treatments for rare ...of orphan drugs [3, 4, 8] and to address licens- ing regulations and pricing and reimbursement of these drugs [4, ...
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