Individual membership will be possible for those for whom no support network exists. Harry Marsh from Contact a Family set the scene for the day and Carol Youngs spoke about the key issues of concern to families in the UK including the fact that many raise money for research in hope of a cure sometime in the future. This must not be at the expense of care needed today and every day for those families for whom a cure is not a reality during their lifetime. The first one aims at developing means of information and support for affected families, as well as better tools to locate new emerging pathologies. The Regulation creates an economical environment favourable to the development of therapies against rare diseases.
Community plans to promote research are still uncertain.
These two proposals represent considerable progress and carry many hopes for affected families. This new Programme has been adopted with many amendments by the Parliament in December, but we still do not know how research actions on rare diseases will be integrated within a global Community policy on these diseases, nor how much money will be allocated to this research. Without a substantial commitment from this new Framework Programme, research on rare pathologies will severely suffer from money shortage. Development of medicinal products against rare diseases may, in the proper economical environment, be done by the pharmaceutical industry. However, basic research on these pathologies relies almost completely on public funding of research centers and universities.