Together for rare diseases

Stakeholders celebrate ten years of success of the EU regulation on orphan medicines at the European Parliament.

The joint EBE–EuropaBio Task Force on Rare Diseases and Orphan Medicines organized a special reception recently at the European Parliament, hosted by Madame Françoise Grossetête, MEP, to celebrate ten years of success of the EU Regulation on Orphan Medicinal Products adopted in December 1999.

This reception, which was attended by the key stakeholders active in the field of rare diseases, was the occasion to reflect on the positive impact this EU legislation has had on patients and biopharmaceutical R&D in Europe. As Madame Grossetête said: ‘It has been almost ten years since a partnership has been established in Europe between health professionals, civil society organisations and industry in the field of rare diseases.

'The results are already quite fruitful and tangible. Europe’s Orphan Drug Regulation has increased the life expectancy and quality of life of many rare disease patients. This has had an economic effect as well as an effect on the patients’ health: they now contribute to society rather than be dependent on it.’

Ten years after the adoption of the European Regulation, 63 new treatments for rare, chronically debilitating and life-threatening diseases have received a positive EU opinion from the European Medicines Agency. In addition, more than 720 applications for orphan designations have been granted for treatments in development for more than 200 different rare diseases.

The treatments approved as a result of the Orphan Regulation make a substantial difference for patients, bringing hope to some 30 million Europeans and their families affected by one of the 7000 rare diseases identified so far.

At the event, Yann le Cam, Eurordis’s CEO, made an intervention on behalf of European rare disease patients and Isabel de la Mata, DG Sanco’s Principal Adviser, outlined the new action plan being driven by the European Commission in this field.

Despite this positive assessment, ‘much still remains to be done’, as emphasized by Madame Grossetête, who noted that ‘many patients still face delays in access to treatment, and many of these diseases remain impossible to treat’. She called on Member States to ‘put in place national rare disease plans and hold to their commitments made in the Council Recommendation of June 2009 relative to an action in the field of rare diseases in order to improve the diagnosis, treatment and care brought to patients afflicted with rare diseases’.

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