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A journey of hope: lessons learned from studies on rare diseases and orphan drugs

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References

2005Drugs for exceptionally rare diseases: do they deserve special status for funding?220 cited

Cited by

2008Incentives for orphan drug research and development in the United States138 cited
2018Challenges in Research and Health Technology Assessment of Rare Disease Technologies: Report of the ISPOR Rare Disease Special Interest Group131 cited
2017Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients’ health needs?113 cited
2022Children with a rare congenital genetic disorder: a systematic review of parent experiences88 cited
2010Access to orphan drugs despite poor quality of clinical evidence82 cited
2010A discrete choice experiment investigating preferences for funding drugs used to treat orphan diseases: an exploratory study76 cited
2009Translation of rare disease research into orphan drug development: disease matters75 cited
2015The quality of economic evaluations of ultra-orphan drugs in Europe – a systematic review75 cited
2012Strategic Corporate Social Responsibility and Orphan Drug Development: Insights from the US and the EU Biopharmaceutical Industry66 cited
2021Hope, Burnout, and Life Satisfaction: An Intriguing Trinity Among Investigators of the SWOG Cancer Research Network15 cited

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