Concepedia

Outrageous prices of orphan drugs: a call for collaboration

Lucio Luzzatto, Hanna I. Hyry, Arrigo Schieppati, Enrico Costa, Steven Simoens, Franz Schaefer, Jonathan C. P. Roos, Giampaolo Merlini, Helena Kääriäinen, Silvio Garattini,

The Lancet · 2018 · 160 citations · 9 references

Concepts

PharmacyPharmaceutical PolicyDrug LawPharmaceutical RegulationAccess To MedicinesEssential MedicinesOrphan Drug DesignationPharmaceutical EconomicsOrphan Drug ActOrphan Drug DevelopmentOrphan Drug PolicyOrphan Drug Pricing PolicyOrphan Drug Regulations

References

9

Hydroxyurea for the Treatment of Sickle Cell Anemia

New England Journal of Medicine · 2008 · 353 citations

Medicine, Hematology, Pharmacotherapy +9

The $2.6 Billion Pill — Methodologic and Policy Considerations

Jerry Avorn · New England Journal of Medicine · 2015 · 324 citations

Pharmacy, Pharmaceutical Policy, Pharmacoeconomics +17

Hydroxyurea in the treatment of sickle-cell anemia.

Howard Lw, LeAnne Kennedy · PubMed · 1997 · 251 citations

Medicine, Hematology, Pharmacology +16

Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments?

Wills Hughes-Wilson, Ana Luiza do Rosário Palma, Ad Schuurman et al. · Orphanet Journal of Rare Diseases · 2012 · 169 citations · Full text

Pharmacy, Health Policy, Pharmaceutical Policy +17

Progress in cystic fibrosis and the CF Therapeutics Development Network

Steven M. Rowe, Drucy Borowitz, Jane L. Burns et al. · Thorax · 2012 · 71 citations · Full text

Medicine, Pharmacology, Pharmacotherapy +15