Room for improved application of Orphan Medicines rules in Europe, says biotech industry - New White Paper sets out key recommendations

Brussels, Friday, 4^th March 2005: As patient representatives meet today with the European Medicines Agency (EMEA) in London to debate orphan medicine rules, the healthcare biotechnology industry publishes its White Paper setting out ideas for an optimal, well functioning orphan medicines system in Europe. 

“The Industry has thought about what framework is needed to optimise the work on and access to therapies for patients with rare diseases and is proposing a comprehensive paper with suggestions for such a framework to be worked on by all involved stakeholders,” says Dr. Erik Tambuyzer, Chair of the Healthcare Council at EuropaBio – the EU association for bioindustries (1).   

This year will witness two reports on the EU’s orphan medicines rules - the five year review from the EMEA’s Committee on Orphan Medicinal Products (COMP) will be published this June and an EU Commission report is expected end 2005. The biotech Industry believes that the application of the EU’s orphan medicine rules and spirit is still in its infancy across the EU.  

“There is a comprehensive set of principles contained in the Regulation,” states Dr Andrea Rappagliosi, Orphan Medicines Chair at EuropaBio and one of the official industry representatives on the COMP’s Working Group for Interested Parties.  “Each of these is an integral part of the successful functioning of the EU’s Orphan system and each deserves to be applied fully.” 

Industry applauds the fact that there is an orphan medicines Regulation, which has been shown to work. Indeed, almost no EU orphan medicines for patients with rare diseases had been developed in the EU prior to 2000 when the EU regulation 141/2000 entered into force.  Since then 254 products have been designated as orphan medicines and of those 20 have been granted marketing authorisation in the EU.    “It is too soon to judge full results on a handful of products but the outlook is promising and we should all support the full application of this Regulation,” says Dr. Erik Tambuyzer.  “There is a lot to do on the understanding of the Regulation and its implications in the EU Member States including work on patient access as well as awareness and education about rare diseases among health professionals and clinicians.”  

Since 70-80% of rare diseases have a genetic origin, biotechnology will play a major role in developing treatments for these unmet medical needs.   Screening may become an important factor leading to timely diagnosis and therefore the EU biotech industry calls for implementation of the STRATA group recommendations as regards Genetic Testing (2). The Biotechnology Industry is also calling for the elimination of regional inequalities and disparities in patient diagnosis, access and reimbursement, which were highlighted in a recent EU study (3).  More information and education about disease prevalence and diagnosis are needed and greater efforts made to explain the Regulation in the EU Member States.   Industry also wants to see more coordination of research and the link made to the Regulation and research milestones.   

The much older US Orphan Drugs Act has helped give birth to a new emerging class of healthcare biotech companies developing products for small groups of patients.  In the US, about half of the biotech startups in the 80’s were based on the Orphan Drugs Act. “This is now also happening in Europe with emerging high growth biotech companies contributing to the “Lisbon knowledge based economy” and producing medicines to meet unmet medical needs. Industry is calling on EU Member States to improve competitiveness with the US.” says Dr. Johan Vanhemelrijck, EuropaBio Secretary General. 

The White Paper sets out a series of recommendations to optimise the Orphan Medicines Regulation 

1. By fully implementing the rules including efforts to be made by the EU Commission to get the Member States to provide the needed incentives foreseen in the Regulation.

2. More awareness about rare diseases to address the current issue of access and other aspects.

3. Support for an EU wide network for diagnostic testing of rare diseases to avoid late diagnosis.

4. Support a system for compassionate use to provide medicines to patients with the highest medical needs before approval or reimbursement.

5. Ensure the Regulation is applied in a clear, harmonised way throughout the EU and not in a piece meal fashion, disparities in the way the rules are applied must be eliminated.

6. Having predictable rules on market exclusivity that are correctly implemented throughout the EU and once and for all eliminate the confusion around the potential reduction of the 10-year exclusivity, which is today the most important incentive for companies to produce medicines for small populations of rare disease patients.

7. By encouraging a greater understanding of the proper degree of appropriate clinical and cost effectiveness data that can be demanded by Member State authorities given the rarity of the diseases being targeted.

8. Adapt the EU‘s Clinical Trials Directive to the requirements for clinical trials in the field of rare diseases.

9. Greater coordination and streamlining of EU rare disease research and therapies between EU Commission departments as well as the EMEA and the EU regulation and with the FDA.

Link to White Paper: “Towards an optimal Orphan Medicinal Products (OMP) Framework in Europe.”
http://www.europabio.org/articles/Orphan%20Medicines%20White%20Paper_FINAL.doc

- ENDS-  

Notes to Editors

(1)   EuropaBio, the European Association for Bioindustries, has some 50 corporate members operating worldwide and 25 national biotechnology associations representing some 1500 small and medium sized enterprises involved in research and development, testing, manufacturing and distribution of biotechnology products.  www.europabio.org 

(2)   The STRATA Group recommendations on genetic testing
http://europa.eu.int/comm/research/conferences/2004/genetic/index_en.htm 

(3)   The study on the price of Orphan Medicinal Products was commissioned by DG Enterprise and is available at: http://pharmacos.eudra.org/F2/orphanmp/index.htm 

For further information, please contact:

Adeline Farrelly, EuropaBio
Tel: +32 2 735 0313  / Mobile: + 32 475 93 17 24 email: a.farrelly@europabio.org