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
Kylie Tingley, D. Coyle, I. Graham, L. Sikora, P. Chakraborty, K. Wilson, John J. Mitchell, S. Stockler-Ipsiroglu, B. Potter
Orphanet Journal of Rare Diseases