Effectiveness, safety and costs of orphan drugs: an evidence-based review

Shining a light in the black box of orphan drug pricing

(2014) Eline Picavet et al.   Orphanet Journal of Rare Diseases

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

(2013) Tim A Kanters et al.   Orphanet Journal of Rare Diseases

Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU

(2012) Roberta Joppi et al.   EUROPEAN JOURNAL OF CLINICAL PHARMACOLOGY

Oral Ibuprofen versus Intravenous Ibuprofen or Intravenous Indomethacin for the Treatment of Patent Ductus Arteriosus in Preterm Infants: A Systematic Review and Meta-Analysis

(2012) Roland Neumann et al.   Neonatology

Pricing and reimbursement of orphan drugs: the need for more transparency

(2011) Steven Simoens   Orphanet Journal of Rare Diseases

Critical Assessment of Belgian Reimbursement Dossiers of Orphan Drugs

(2011) Alain Denis et al.   PHARMACOECONOMICS

GRADE: an emerging consensus on rating quality of evidence and strength of recommendations

(2008) Gordon H Guyatt et al.   BMJ-British Medical Journal