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

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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Bibliographic Details
Main Authors: 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: Article
Language:English
Published: BMC 2018-06-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Rare diseases
Evidence generation
Comparative effectiveness
Patient-oriented outcomes
Evidence synthesis
Research methods
Online Access:http://link.springer.com/article/10.1186/s13023-018-0851-1

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http://link.springer.com/article/10.1186/s13023-018-0851-1

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