Systematic review of available evidence on 11 high-priced inpatient orphan drugs

Preferred Reporting Items for Systematic Reviews and Meta-Analyses: The PRISMA Statement

(2013) David Moher   ANNALS OF INTERNAL MEDICINE

Raising Orphans: How Clinical Development Programs of Drugs for Rare and Common Diseases Are Different

(2012) M Orfali et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Perspectives for improving the evaluation and access of therapies for rare lung diseases in Europe

(2012) Maurizio Luisetti et al.   RESPIRATORY MEDICINE

Determinants for successful marketing authorisation of orphan medicinal products in the EU

(2011) Michelle Putzeist et al.   DRUG DISCOVERY TODAY

Characteristics of Clinical Trials to Support Approval of Orphan vs Nonorphan Drugs for Cancer

(2011) Aaron S. Kesselheim   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Estimating the budget impact of orphan medicines in Europe: 2010 - 2020

(2011) Carina Schey et al.   Orphanet Journal of Rare Diseases

Access to orphan drugs despite poor quality of clinical evidence

(2010) Alain G. Dupont et al.   BRITISH JOURNAL OF CLINICAL PHARMACOLOGY

Pivotal studies of orphan drugs approved for neurological diseases

(2009) Jun Mitsumoto et al.   ANNALS OF NEUROLOGY

Clinical trials of orphan medicines

(2008) Brendan M Buckley   LANCET

Long-term Efficacy and Safety of Laronidase in the Treatment of Mucopolysaccharidosis I

(2008) L. A. Clarke et al.   PEDIATRICS