EURORDIS has published its response to the Commission’s Discussion Document, “Health in Europe: a Strategic Approach” (see Orphanews Europe - 7 Feb edition - Health in Europe : a Strategic Approach). In this 7-page contribution, the European patients' organisation sets out a comprehensive set of recommendations relating to what it identifies to be the main points of DG SANCO’s proposed 10-year Health Strategy recently undergoing public discussion.
Supporting what it believes to be the Community’s legitimate role in taking global European action where justified as more effective than at the national or regional level, EURORDIS argues that this action is particularly important, given the specific nature of rare disease (RD) needs. It points out that the rare disease sector is carrying out pioneering work at the cutting edge of future health policy, and cites progress achieved in areas such as improvements in access to health services, compensation of inequalities and disabilities, and a comprehensive approach to patient care. EURORDIS sets out its own “concrete objectives” in its fight against the negative impact of rare diseases on the lives of patients, stressing the importance of improving access to healthcare, information, exchange of good practices and quality of life whilst also promoting research at fundamental and clinical level to develop orphan drugs and RD therapies. It urges the European Community to integrate these objectives into its new strategic framework.
This paper reminds readers of the many achievements accomplished for rare diseases over the past decade, especially in terms of the development of treatment and public awareness, tracing the Commission’s progressive approach to this since 1999, with key legislation including Orphan Drugs Regulation, FP7 priority on RD and the Community Action Programme on Public Health, in addition to the development of more specific RD policies by certain Member States at national level. It points out that the timescale is different with regard to RDs since 5-year actions are often too short for RD projects compared to those from other health sectors, due to a general shortage of information and expertise as well as a scarcity of patients themselves. With this in mind, EURORDIS advocates a two-tier approach in 1) the promotion of: national policies within Member States and increased cooperation between them, and 2) a coordinated EU action to be guided by common guidelines, guaranteeing a coherent global approach. It adds its support to the drafting of a Commission Communication and Council RD Recommendations, which it qualifies as essential in achieving this common European coherence.
Key proposals in this document are the creation of an EU High Level Group on Rare Diseases designed to help stimulate action within agreed policy guidelines and designed to serve as a monitoring body, with an assessment based on specific indicators to be determined. Another proposal is the creation of a Community Agency for Rare Diseases (CARD), intended to manage the long-term implementation of RD policies at Community level. This would oversee the perenniality and coherence of relevant EU strategies for patient registries, biobanks, clinical trials, RD information, networks of centres of reference, etc. EURORDIS develops the importance of working with other relevant policy areas and identifies the key sectors in which RDs should be taken into account.
This Contribution document concludes with a section on Global Issues, which points out that patients share the same basic needs and preoccupations worldwide. Drawing on some of the ideas already put forward in a previous EURORDIS Position Paper on a WHO Report on Priority Medicines for Europe and the World, it describes how RDs may provide useful models to help understand other, more common diseases and adds that many orphan drugs are actually innovative biotechnological products which have acted as a starting block for a number of small biotech companies. Finally, the importance of coordinated international action through bodies such as ICORD (International Coordination for Orphan Drugs and Rare Diseases) is stressed, with the conclusion that the estimated 30 million European patients living with RDs have an indisputable need for the action that the EU Health Strategy Discussion Document aims to generate.
Read the full EURORDIS Response to the Discussion Document for a Health Strategy
Read “Rare Diseases: understanding this Public Health Priority”
