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
Author:
Funder
Canadian Institutes of Health Research
Publisher
Springer Science and Business Media LLC
Subject
Pharmacology (medical),Genetics(clinical),General Medicine
Link
http://link.springer.com/content/pdf/10.1186/s13023-018-0851-1.pdf
Reference89 articles.
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3. Wästfelt M, Fadeel B. Henter J-I. A journey of hope: lessons learned from studies on rare diseases and orphan drugs. J Intern Med. 2006;260:1–10.
4. Augustine EF, Adams HR, Mink JW. Clinical trials in rare disease: challenges and opportunities. J Child Neurol. 2013;28:1142–50.
5. Kaptchuk TJ. The double-blind, randomized, placebo-controlled trial: Gold standard or golden calf? J Clin Epidemiol. 2001;54:541–9.
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