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

Abstract Introduction For many rare diseases, strong analytic study designs for evaluating the efficacy and effectiveness of interventions are challenging to implement because of small, geographically dispersed patient populations and underlying clinical heterogeneity. The objective of this study wa...

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Bibliografiske detaljer

Principais autores:	Kylie Tingley, Doug Coyle, Ian D. Graham, Lindsey Sikora, Pranesh Chakraborty, Kumanan Wilson, John J. Mitchell, Sylvia Stockler-Ipsiroglu, Beth K. Potter, in collaboration with the Canadian Inherited Metabolic Diseases Research Network
Format:	Artigo
Sprog:	Inglês
Udgivet:	BMC 2018-06-01
Serier:	Orphanet Journal of Rare Diseases
Fag:	Rare diseases Evidence generation Comparative effectiveness Patient-oriented outcomes Evidence synthesis Research methods
Online adgang:	http://link.springer.com/article/10.1186/s13023-018-0851-1
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