By Peter O’ Donnell.
This week (February 28), Europe will celebrate Rare Disease Day. This is an interesting example – one might almost say a rare example – of a successful attempt by a European interest group to capture public attention. It is now an annual awareness-raising event co-ordinated by the European rare diseases lobby group, Eurodis.
For this year’s celebration, on February 28, Eurordis has shown commendable imagination as well as energy in seeking to maximize the profile. It is, after all, a campaigning event by a campaigning organization. The main objective is to make sure that the general public and decision-makers take more notice of rare diseases and their impact on patients’ lives. So it targets not only the man or woman in the street, but also patients and patient representatives, politicians, public authorities, industry representatives, researchers, health professionals, and anyone who has a role in relation to rare diseases. For six months, Eurordis has been flagging up the event through media announcements, posters, social networks, mail shots, and even a video – and it has managed not only to make it look attractive, but to attract wide support. More than 70 countries across the world are taking part too, ranging – alphabetically and geographically – from Algeria and Argentina to Venezuela and Vietnam, and organizing everything from festivals and dinners to conferences and concerts.
This year’s slogan, “Join Together for Better Care,” encourages the rare disease community to continue finding ways to work together to provide the different kinds of care that people living with a rare disease need. It explicitly aims to promote solidarity and to overcome the sense of isolation that many victims of rare diseases suffer. The success of this approach is neatly illustrated by a comment on the campaign’s Facebook page: “E’ sempre una grande emozione….l’attesa, la sorpresa, la gioia di condividerlo! Grazie Eurordis!” (It’s always a huge emotion… the expectation, the surprise, the joy of sharing it! Thank you Eurordis!)
But the true success of the Rare Disease Day and all the associated campaigning will be judged against tougher criteria – in terms of how far it improves the current situation and future treatment of patients. Eurordis itself claims that “the political momentum resulting from the Day has also served for advocacy purposes. It has notably contributed to the advancement of national plans and policies for rare diseases in a number of countries.” What remains to be seen is how far this European organization can shape the political and regulatory environment governing rare disease research and treatment.
Its Berlin conference in May, co-organized with the Drug Information Association, will represent a rather more hard-core confrontation with the decision-makers that influence the fate of rare disease sufferers. It will be examining opportunities in collaborative clinical research into rare diseases, the challenges of disease-specific registries, improvements to gene testing, and how to sustain investor interest in rare disease research.
Another test is how Eurordis and its constituency will manage to benefit from the new expert group on rare diseases that the European Commission has created, and that held its first meeting in Luxembourg on 11-12 February. This replaces an earlier advisory forum, and one of the novelties is that EU officials have a much more prominent role in running the group. Above all, success for Eurordis and those it represents will depend crucially on the outcome over the next two years of the development and implementation of national plans for rare diseases. It will take more than a fancy website to make all that come right.