EU Rare Disease Policies: An Overview

More than half of Member State legislation stem from EU policies and strategies, including directives, regulations and recommendations. Since EURORDIS represents rare disease patients throughout Europe (an estimated 30 million citizens), it is at the European Union (EU) level that EURORDIS can legitimately act to influence EU policy makers. 

congress - 10.2 kb

Therefore, EURORDIS focuses many advocacy activities at the EU level and also performs advocacy actions at Member State level in close cooperation with our members.

EURORDIS  interacts with the institutions governing the European Union, especially the European Commission and the European Parliament. As the European Council represents national interests, advocacy efforts directed towards governments are most effective at the national level and can be performed in cooperation between EURORDIS and its members.

The Berlaymont - 12.2 kbIn the European Commission, the most relevant Directorates General (DGs) for rare disease advocacy are:

  • DG Health and Consumers (DG SANCO) provides a funding instrument and is involved in health policy, safety and information, including health determinants, health systems and medical care;
  • DG Research and Innovation develops and manages the Framework Programmes of Research and Development - including rare disease-related research;
  • DG Enterprise and Industry is implicated in EU pharmaceutical policy, including orphan medicines, paediatric medicines, advanced therapies and medical devices.

EURORDIS is also exploring opportunities with other Directorates General, such as DG Social Affairs (including disability issues), DG Connect, DG Regional Policy and DG Education and Culture.

Meeting  - 9.6 kbThe advocacy activities of EURORDIS are varied.
They include initiatives such as:

  • Identifying key priorities for the rare disease community;
  • Influencing EU decision-makers on key priorities;
  • Writing position papers on key issues for the European rare disease community and disseminating them to regulatory authorities and decision-makers at EU level;
  • Monitoring relevant EU policies and keeping members informed;
  • Hosting, participating in, or attending meetings and forums of interest to the rare disease field;
  • Establishing and maintaining contacts with key decision-makers in EU institutions;
  • Establishing and maintaining working relationships with other umbrella organisations in the health sector;
  • Organising conferences and events at EU level on issues impacting the rare disease community;
  • Improving EURORDIS' visibility in the European health arena;
  • Expanding and diversifying financial resources for EURORDIS and its members by identifying new funding opportunities.

 

Some key EU level rare disease and orphan medicine policies in which EURORDIS was instrumental include:

  • EU Regulation on Orphan Medicinal Products in 1999
  • EU Regulation on Medicinal Products for Paediatric Use in 2006
  • EU Regulation on Advanced Therapy Medicinal Products in 2007
  • EU Commission Communication Rare Diseases: Europe's Challenges in 2008
  • EU Council Recommendation on a European action in the field of rare diseases in 2009
  • EU Directive on Patients’ Right to Cross-Border Healthcare in 2011
  • Promoting and maintaining rare diseases as a priority within:
        EU Public Health Programme
        EU Research Framework Programme
  • Promoting National Plans and Strategies on Rare Diseases in all 28 EU Member States and other European countries

 

Current EU policies and advocacy activities in which EURORDIS is closely involved include:

  • Revision of the EU Clinical Trials Directive
  • Revision of the EU Data Protection legislation
  • Revision of the Transparency Directive
  • Improving access to orphan medicinal products through:moting and maintaining rare diseases as a priority within:
        Clinical Added Value of Orphan Medicinal Products (CAVOMP)
       Mechanism of Coordinated Access to Orphan Medicinal Products (MoCA)
       Differential Pricing and Adaptive Licensing

 

Author: EURORDIS
Photo credits: congress © Clarita Natoli; The Berlaymont © Gemma Lougheed; Hands together © despair.com

Page created: 30/10/2009
Page last updated: 07/11/2014
 
 
La voz de los pacientes de enfermedades raras en EuropaEURORDIS La voz internacional de las personas que tienen enfermedades rarasRare Disease International Reuniendo a pacientes, familiares y expertos para compartir experiencias en un foro moderado en distintos idiomas. RareConnect El Programa Rare Barometer es una iniciativa de EURORDIS que realiza encuestas para transformar las experiencias de los pacientes con enfermedades raras en cifras que puedan compartirse con los responsables políticos.Rare Barometer Una campaña internacional de sensibilización que se celebra todos los años el último día de febrero, El Día de las Enfermedades Raras es una iniciativa de EURORDISRare Disease Day Únete al mayor grupo de partes interesadas en toda Europa, en la Conferencia Europea sobre Enfermedades Raras y Medicamentos Huérfanos que se celebra cada dos años. ECRD es una iniciativa de EURORDISEuropean Conference on Rare Diseases