Rare diseases: what's next?

Empowerment of patients: lessons from the rare diseases community

Ségolène Aymé, Anna Kole, Stephen C. Groft · The Lancet · 2008 · 232 citations

Social Sciences, Health Policy, Health Equity +10

Does orphan drug legislation really answer the needs of patients?

Marlene E. Haffner, Josep Torrent-Farnell, Paul D Maher · The Lancet · 2008 · 79 citations

Pharmacy, Pharmaceutical Policy, Regulatory Pharmacy +12

Clinical trials of orphan medicines

Brendan M. Buckley · The Lancet · 2008 · 77 citations

Medicine, Pharmacology, Clinical Pharmacology +10

Orphan drug development is progressing too slowly

Roberta Joppi, Vittorio Bertelè, Silvio Garattini · British Journal of Clinical Pharmacology · 2006 · 70 citations · Full text

Pharmacy, Pharmaceutical Policy, Pharmacology +17

Arsenic patent keeps drug for rare cancer out of reach of many

David Cyranoski · Nature Medicine · 2007 · 41 citations · Full text

Medicine, Oncology, Pharmacology +13