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European Commission conference underlines value of orphan drug development

Industry welcomes the special conference on Rare Diseases Research: Building on Success, organised by DG Research to foster increased research, renewed Community funding and new partnerships to combat rare diseases.   

At the conference, the joint European Biopharmaceutical Enterprises (EBE) and EuropaBio Orphan Medicinal Products Task Force – representing companies active in researching and developing treatments for patients suffering from rare diseases – had the opportunity to present the industry’s current high level of activity in research on orphan drugs.  To further build on this, they highlighted the need for more partnerships between patients and industry, between science and the public sector, but also for improved access to the often life-saving orphan medicines resulting from such research and partnerships and the importance of funding clinical trials in this field.

Speaking at the conference, EBE President Dr Carlo Incerti, said “Collaboration between industry and academia in rare diseases research is essential to be able to convert an initial scientific idea into a real-life safe and effective medicine for patients.  This collaboration needs to further include other key stakeholders, such as patient organizations and regulators.  We welcome the Innovative Medicines Initiative and the opportunities that the EU Commission should make available through FP7 – in particular the proposal of an EU research grant programme for Clinical Trials for products designated as Orphan Medicines.”

Industry welcomes the remarks by Mr Poto?nik, the European Commissioner for Research, who told conference delegates that more needs to be done on rare disease research in Europe, as well as increased awareness on our past achievements and future challenges.  He said the European Commission and he personally, were committed to offering real progress in the area of rare diseases research as part of FP7 – the EU’s research programme.

Dr. Erik Tambuyzer, Chair of the Joint EBE and EuropaBio Task Force for Orphan Medicines, said that “Industry continues its commitment to – and investment in –  orphan medicines research and development.  We plan to maintain our efforts to ensure that new treatments for rare diseases are brought to the patients in Europe – a task partially facilitated by the favourable environment created by the Orphan Medicinal Products (OMP) Regulation in place since 2000.  As a result of the OMP Regulation, there are already more than 40 treatments either authorized by the Commission or having a positive CHMP Opinion, and over 470 designated products in development.  Yearly approvals of orphan drugs in the EU now compare in number to those in the US.  Cooperation between all stakeholders has achieved this,  and it is vital that this cooperation continue at all stages, including ensuring that European patients get equal and real access to authorized treatments in all Member States.  Industry also underlines the need to turn knowledge into innovation and that an enabling regulatory framework in Europe is essential to bring products to patients.

Link to Conference programme –
http://cordis.europa.eu/fp7/cooperation/health-events_en.html

For more information, contact

EuropaBio – Michael Leader, Director Healthcare Council - Tel: +32 2 735 0313 Mobile: +32 496 850 468
Email: >

European Biopharmaceutical Enterprises (EBE) - Emmanuel Chantelot, Executive Manager - Tel: + 32 2 626 2561
Email: >

About the Joint EBE/EuropaBio Task Force
The Task Force supports member companies in the research, development, registration and marketing of Orphan Medicinal Products in Europe and assists authorities in the successful implementation of the European OMP Regulation. 

About EuropaBio
EuropaBio is the European Association for Bioindustries, solely and uniquely bringing together bioscience companies from all fields of research and development, testing, manufacturing and distribution of biotechnology products.  It has 84 corporate members operating worldwide, 12 associate members, 5 BioRegions and 25 national biotechnology associations representing some 1800 small and medium sized enterprises involved in research. http://www.europabio.org

About EBE
EBE (European Biopharmaceutical Enterprises) is the European trade association that represents biopharmaceutical companies of all sizes operating in Europe. It has 64 member companies, which are engaged in research, development and marketing of new medicinal products using biotechnology. www.ebe-biopharma.org

Quick links to more information on Orphan Medicines

EuropaBio – Orphan Medicines Web pages –http://www.europabio.org/orphandrugsWP.htm
EBE – Orphan Medicines Web pages - http://www.ebe-biopharma.org/index.php?option=com_content&task=view&id=36&Itemid=135&lang=enCommunity Registry of Medicinal Products -    http://ec.europa.eu/enterprise/pharmaceuticals/register/index.htm

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  • Press Release: European Commission conference underlines value of orphan drug development, 13 September 2007
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