References

• 2000Randomized, Controlled Trials, Observational Studies, and the Hierarchy of Research Designs3,561 cited
• 2008Why rare diseases are an important medical and social issue621 cited
• 2006A journey of hope: lessons learned from studies on rare diseases and orphan drugs185 cited
• 2010Rare diseases, orphan drugs and their regulation: questions and misconceptions182 cited
• 2009The US Orphan Drug Act: Rare disease research stimulator or commercial opportunity?166 cited
• 2002Two decades of orphan product development138 cited
• 2010Societal views on orphan drugs: cross sectional survey of Norwegians aged 40 to 67103 cited
• 2006Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products88 cited
• 2009Translation of rare disease research into orphan drug development: disease matters75 cited
• 2006Orphan drug development is progressing too slowly70 cited
• 2009Orphan drug development is not taking off63 cited
• 2012Access to orphan drugs in Europe: current and future issues60 cited

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