The New Year rings in resolutions across Europe to forward actions in the field of rare diseases and orphan drugs
At the beginning of this New Year, a time of resolutions, there is every reason to feel optimistic that the commitment to initiatives for rare disease patients and their families will continue to expand in 2011. Despite the gloomy economic climate still gripping much of the world, more European Union Member States are expected to come forward this year with a distinct strategy for their rare disease patients, in accordance with the Council Recommendation on an action in the field of rare diseases (learn more).
So many reasons for hope ...
One particularly bright spot on the horizon is the newly-formed European Union Committee of Experts on Rare Diseases (EUCERD), which convened for the first time in December to discuss a variety of activities promoting the understanding and treatment of rare diseases. Led by a team of experienced professionals, with the dedicated support of government at both the EU and Member State levels and the valuable input of stakeholders from medicine, research, government, patient organisations, and the biopharmaceutical industry, it is sure that via EUCERD initiatives in the field of rare diseases will progress in 2011.
Other important activities brewing include the CAVOD project, which is exploring the creation of a mechanism to exchange knowledge of the Clinical Added Value of Orphan Drugs between the Member States and EU-level authorities, in a bid to harmonise and accelerate the process of getting drugs to market in the Member States. There is also a new call for rare disease research proposals from E-Rare 2 (supported by the European Commission under the Seventh Framework Programme ERA-Net scheme), and the ongoing success of the Orphan Drug Regulation (141/2000) that encourages the development of medicinal products for rare diseases. The European Medicines Agency anticipates that a dozen orphan products will be authorised for marketing in 2011.
Furthermore, the successful Rare Disease Day, held on the last day of February, serves to deepen awareness around the world for the many issues rare disease patients and their families face – including a lack of diagnostics, treatment, and services. This year’s theme calls attention to the inequalities that rare disease patients encounter (learn more).
Overseas, the commitment to furthering efforts for rare disease patients is equally strong. A particularly productive meeting took place between rare disease research stakeholders from the USA and the EU in late 2010. Not wishing to lose momentum, a second meeting is already being planned for Spring 2011 that will seek to create an international consortium on rare diseases to prioritise and organise research. It is possible that Japan will also join this endeavour. In an article appearing in Nature Drugs Discovery last month, the FDA declared its commitment to accelerating orphan drug development (learn more).
Such rich evidence leads to the inevitable conclusion that 2011 will see an expansion of efforts designed to make life less difficult for millions of rare disease patients across Europe and around the world, as well as their families and all others who care - both professionally and personally.
Happy New Year from OrphaNews Europe