Statistical challenges in the evaluation of treatments for small patient populations.
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Systematic reviews in paediatric multiple sclerosis and Creutzfeldt-Jakob disease exemplify shortcomings in methods used to evaluate therapies in rare conditionsImportant role of translational science in rare disease innovation, discovery, and drug development.Challenges in developing a validated biomarker for angiogenesis inhibitors: the motesanib experienceMolecular profiling and targeted therapy for advanced thoracic malignancies: a biomarker-derived, multiarm, multihistology phase II basket trialUse of mechanical airway clearance devices in the home by people with neuromuscular disorders: effects on health service use and lifestyle benefits.Hartung-Knapp-Sidik-Jonkman approach and its modification for random-effects meta-analysis with few studiesUsing phase II data for the analysis of phase III studies: An application in rare diseases.Pulmonary arterial hypertension: challenges in translational research and a vision for change.Personalized neuroprosthetics.Secondary Prevention of Childhood Arterial Ischemic Stroke.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?Incremental cost per quality-adjusted life year gained? The need for alternative methods to evaluate medical interventions for ultra-rare disorders.Assessment of skin involvement in systemic sclerosis.A motor learning approach to training wheelchair propulsion biomechanics for new manual wheelchair users: A pilot study.Curtailed two-stage matched pairs design in double-arm Phase II clinical trials.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.
P2860
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P2860
Statistical challenges in the evaluation of treatments for small patient populations.
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article científic
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article scientifique
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articol științific
@ro
articolo scientifico
@it
artigo científico
@gl
artigo científico
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artigo científico
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artikel ilmiah
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artikull shkencor
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artículo científico
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name
Statistical challenges in the evaluation of treatments for small patient populations.
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type
label
Statistical challenges in the evaluation of treatments for small patient populations.
@en
prefLabel
Statistical challenges in the evaluation of treatments for small patient populations.
@en
P2860
P1476
Statistical challenges in the evaluation of treatments for small patient populations.
@en
P2093
Edward L Korn
Lisa M McShane
P2860
P304
P356
10.1126/SCITRANSLMED.3004018
P407
P577
2013-03-01T00:00:00Z