Rare Disease Day


Today is the international rare disease day (www.rarediseaseday.org) where rare disease national alliances from 25 European countries unite under the slogan “Rare but strong together”.

The fifth edition of this unique awareness day could not find a better theme for this year. Indeed, a wide range of initiatives, covering areas such as research and development, to access to patients living with rare diseases, are flourishing and it is only by working all together – patient advocacy groups, European and national policy makers, healthcare professionals, academia and industry – that we will make their promises a reality. A few examples of this coordination across the board include the International Rare Diseases Research Consortium (1) (IRDiRC) coordinating research efforts globally in the field of rare diseases field; the multi-stakeholder European Union Committee of Experts on Rare Diseases (2), which is in charge of supporting the European Commission with the preparation and implementation of Community activities in the field of rare diseases; and the Mechanism of Coordinated Access for Orphan Medicinal Products in the context of the Process on Corporate Responsibility in the field of Pharmaceuticals (3). These initiatives offer opportunities to jointly discuss, define and implement key actions to improve the development, approval and ultimately timely access for patients to orphan medicinal products (OMPs).

While progress are being made at international and European level, a number of EU Member States will need to develop and/or implement the 2009 European Council Recommendations on action in the field of rare diseases (4). This proposal recommends that by 2013, all EU Member States should establish national strategies to ensure that patients have access to high quality care, including diagnostics, treatments, rehabilitation for those living with the disease and, if possible, effective orphan drugs. EuropaBio believes that, building on best practices such as in Belgium or France (5), a comprehensive strategy can be put in place, supported by both European and international initiatives.

The healthcare biotech industry – the main provider of innovative rare disease treatments – remains committed to raise awareness about the specific challenges related to orphan medicinal products. As such, the Joint EuropaBio-EBE Task Force on Rare Diseases and OMPs published a brochure in June 2011 comparing the research, development and approval processes of a usual medicine with the specific features of OMPs (6). This document affirmed the continued commitment of the healthcare biotech sector to explore with all stakeholders the most appropriate and efficient ways to overcome hurdles in the field.


 (1) http://ec.europa.eu/research/health/medical-research/rare-diseases/irdirc_en.html
 (2) http://www.eucerd.eu/
 (3)    http://ec.europa.eu/enterprise/sectors/healthcare/competitiveness/process_on_corporate_responsibility/platform_access/index_en.htm
 (4) http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:C:2009:151:0007:0010:EN:PDF
 (5) http://ec.europa.eu/health/rare_diseases/national_plans/detailed/index_en.htm
 (6) http://www.europabio.org/development-process-orphan-medicines

Latest News