European regulation on orphan medicinal products: 10 years of experience and future perspectives

Challenges with advanced therapy medicinal products and how to meet them

(2010)   NATURE REVIEWS DRUG DISCOVERY

Expediting Patients’ Access to Medicines by Improving the Predictability of Drug Development and the Regulatory Approval Process

(2009) L Liberti et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Factors associated with success of market authorisation applications for pharmaceutical drugs submitted to the European Medicines Agency

(2009) Jan Regnstrom et al.   EUROPEAN JOURNAL OF CLINICAL PHARMACOLOGY

Predictors of orphan drug approval in the European Union

(2008) Harald E. Heemstra et al.   EUROPEAN JOURNAL OF CLINICAL PHARMACOLOGY

Does orphan drug legislation really answer the needs of patients?

(2008) Marlene E Haffner et al.   LANCET

Clinical trials of orphan medicines

(2008) Brendan M Buckley   LANCET

Balancing early market access to new drugs with the need for benefit/risk data: a mounting dilemma

(2008) Hans-Georg Eichler et al.   NATURE REVIEWS DRUG DISCOVERY