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

In 2000, regulation on orphan medicinal products was adopted in the European Union with the aim of benefiting patients who suffer from serious, rare conditions for which there is currently no satisfactory treatment. Since then, more than 850 orphan drug designations have been granted by the European Commission based on a positive opinion from the Committee for Orphan Medicinal Products (COMP), and more than 60 orphan drugs have received marketing authorization in Europe. Here, stimulated by the tenth anniversary of the COMP, we reflect on the outcomes and experience gained in the past decade, and contemplate issues for the future, such as catalysing drug development for the large number of rare diseases that still lack effective treatments.

[1]  Timothy R. Coté,et al.  Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years , 2010, Nature Reviews Drug Discovery.

[2]  H. Leufkens,et al.  Rare essentials: drugs for rare diseases as essential medicines. , 2006, Bulletin of the World Health Organization.

[3]  H. Leufkens,et al.  Predictors of orphan drug approval in the European Union , 2008, European Journal of Clinical Pharmacology.

[4]  Alasdair Breckenridge,et al.  Balancing early market access to new drugs with the need for benefit/risk data: a mounting dilemma , 2008, Nature Reviews Drug Discovery.

[5]  J. Torrent-Farnell,et al.  Does orphan drug legislation really answer the needs of patients? , 2008, The Lancet.

[6]  B. Buckley.  Clinical trials of orphan medicines , 2008, The Lancet.

[7]  A. Breckenridge,et al.  Expediting Patients' Access to Medicines by Improving the Predictability of Drug Development and the Regulatory Approval Process , 2010, Clinical pharmacology and therapeutics.

[8]  S. Mariz,et al.  EU regulations , 2011, BMJ : British Medical Journal.

[9]  R. Kurzrock,et al.  Uncommon tumors and exceptional therapies: paradox or paradigm? , 2007, Molecular Cancer Therapeutics.

[10]  Communication from the European commission to the council, the European parliament, the economic and social committee and committee of the regions , 2002 .

[11]  C. Morel.  Faculty Opinions recommendation of Rare essentials: drugs for rare diseases as essential medicines. , 2007 .

[12]  H. Eichler,et al.  Factors associated with success of market authorisation applications for pharmaceutical drugs submitted to the European Medicines Agency , 2009, European Journal of Clinical Pharmacology.

[13]  Mark C. Fishman,et al.  Pharmaceuticals: A new grammar for drug discovery , 2005, Nature.

[14]  Robert Zorec,et al.  Challenges with advanced therapy medicinal products and how to meet them , 2010, Nature Reviews Drug Discovery.

[15]  Sabine Urban.  Europe’s Challenges , 1996 .