Rare disease communication results online
The European Commission’s DG Health and Consumer Protection has posted online all the responses to the Consultation regarding European Action on Rare Diseases. Replies are organised into the following groups: Patients and their families, National patient organisations, International patient organisations, Member states (national authorities), International and European organisations, Commercial organisations, companies and their associations, Universities and other experts, Reference centres, Researchers, and Others. All the responses to the Consultation are considered valuable – and there were some notable contenders from the full spectrum of the rare disease stakeholders who weighed in on the subject - the European Medicines Agency’s Committee for Orphan Medicines Products (COMP), the Standing Committee of European Doctors, the EC Rare Disease Task Force, the European Cancer Patient Coalition, the Karolinska Institutet, the Slovenian PKU Association, the Association Prader Willi France, the Society for Inborn Errors of Metabolism in Adults in Internal Medicine, the Cyprus Institute of Neurology and Genetics, the Italian Medicines Agency, the Medical Genetics College at the Portuguese Medical Association, the Polish Association for Genetic Disorders in Children, BioMarin, Genzyme, Shire Human Genetic Therapies and EuroGentest are just some examples of the diversity of responses. Member state authorities responding to the Consultation include representatives from Denmark, France, Germany, Hungary, Malta, the Netherlands, Norway, Spain, Slovenia, Sweden and the UK.
The responses to the Consultation provide a rich resource yielding multiple perspectives. The individual replies from patients and parents of patients provide that crucial first-hand experience of what it truly is to live with a rare disease in Europe today – and what is needed to improve the quality of life for patients and their caregivers. The national and international patient groups possess in depth knowledge of a particular disease or group of diseases and are able to offer a unique perspective, including resources available and resources sorely missing. The health authorities hold particular information on the budgetry and legal obstacles that can prevent resources being created for patients, and the scientific and industry representatives are able to communicate what they need to go forward toward a better understanding of rare diseases and the development of therapeutic and diagnostic products for patients. OrphaNews Europe takes this moment to acknowledge all the various stakeholders who took the time to read and offer their thoughtful – and often thought provoking – reply to the Public Consultation for Rare Diseases: Europe’s Challenges.
The process of revising the rare disease communication in order to incorporate the various comments and suggestions made during the consultation period is already underway. The drafting group responsible for the task anticipates terminating the revision by the end of June. The modified document will then be presented and reviewed at the next bi-annual meeting of the Rare Disease Task Force, taking place in early July.
View the replies to the Consultation