Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU.
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Review and comparison of clinical evidence submitted to support European Medicines Agency market authorization of orphan-designated oncological treatmentsAccess to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 CountriesEffectiveness, safety and costs of orphan drugs: an evidence-based reviewCollaborative development of 2-hydroxypropyl-β-cyclodextrin for the treatment of Niemann-Pick type C1 diseaseIdentifying priority medicines policy issues for New Zealand: a general inductive study.Introduction to a supplement on innovative approaches to studying health outcomes in rare diseases.Orphan drugs expenditure in the Netherlands in the period 2006-2012.A comparison of interventional clinical trials in rare versus non-rare diseases: an analysis of ClinicalTrials.gov.Value-based reimbursement decisions for orphan drugs: a scoping review and decision framework.Can the EVIDEM Framework Tackle Issues Raised by Evaluating Treatments for Rare Diseases: Analysis of Issues and Policies, and Context-Specific Adaptation.The availability and affordability of orphan drugs for rare diseases in ChinaPotential impact of the implementation of multiple-criteria decision analysis (MCDA) on the Polish pricing and reimbursement process of orphan drugs.Systematic review of available evidence on 11 high-priced inpatient orphan drugs.Clinical evidence for orphan medicinal products-a cause for concern?Determining the value of medical technologies to treat ultra-rare disorders: a consensus statement.Reconciling uncertainty of costs and outcomes with the need for access to orphan medicinal products: a comparative study of managed entry agreements across seven European countries.Registering medicines for low-income countries: how suitable are the stringent review procedures of the World Health Organisation, the US Food and Drug Administration and the European Medicines Agency?Strategies for postmarketing surveillance of drugs for rare diseases.Are new models needed to optimize the utilization of new medicines to sustain healthcare systems?Points to consider: efficacy and safety evaluations in the clinical development of ultra-orphan drugs.Clinical relevance of information in the Summaries of Product Characteristics for dose adjustment in renal impairment.A systematic literature review of evidence-based clinical practice for rare diseases: what are the perceived and real barriers for improving the evidence and how can they be overcome?ERDO - a framework to select an appropriate randomization procedure for clinical trials.Incremental cost per quality-adjusted life year gained? The need for alternative methods to evaluate medical interventions for ultra-rare disorders.Addressing challenges for sustainable healthcare in Central and Eastern Europe.Revealed preferences towards the appraisal of orphan drugs in Poland - multi criteria decision analysis.Characteristics of clinical trials in rare vs. common diseases: A register-based Latvian study.Using a meta-narrative literature review and focus groups with key stakeholders to identify perceived challenges and solutions for generating robust evidence on the effectiveness of treatments for rare diseases.Rare disease registries: potential applications towards impact on development of new drug treatments
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Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU.
description
2012 nî lūn-bûn
@nan
2012年の論文
@ja
2012年学术文章
@wuu
2012年学术文章
@zh
2012年学术文章
@zh-cn
2012年学术文章
@zh-hans
2012年学术文章
@zh-my
2012年学术文章
@zh-sg
2012年學術文章
@yue
2012年學術文章
@zh-hant
name
Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU.
@en
Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU.
@nl
type
label
Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU.
@en
Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU.
@nl
prefLabel
Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU.
@en
Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU.
@nl
P2093
P2860
P1476
Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU.
@en
P2093
Roberta Joppi
Silvio Garattini
Vittorio Bertele'
P2860
P2888
P304
P356
10.1007/S00228-012-1423-2
P577
2012-10-23T00:00:00Z