France’s national rare disease plan brings home its first report card
France’s National Plan for Rare Diseases – the first of its kind in the world – has undergone intense scrutiny since its four-year term concluded at the end of 2008. The overarching goal of the evaluation is to provide data that will inform the elaboration of the second version of the rare disease plan, due out in 2010. An Evaluation Committee (Codev) consisting of health, economic and sociology experts, under the authority of the French Council for Public Health, thus measured the initial objectives of the plan against the corresponding actions undertaken during the past four years. An official report of the evaluation was rendered to the French Minister of Health on 7 May. The document provides an analysis of the accomplishments, advances, and shortcomings of each of the ten axes of the plan. A series of propositions and recommendations is also furnished for the elaboration of the future plan.
Throughout the evaluation, the committee underscores the satisfaction of the different stakeholders vis-a-vis the overall results accomplished. The objectives judged most pertinent – the access to information (particularly Orphanet and MRIS, the French rare disease informational service helpline), medical care (centres of reference), research funding, orphan product accessibility, and partnerships with European institutions – have benefited from corresponding actions that have satisfactorily fulfilled these goals. The need to sustain these actions was reiterated in the evaluation. However, certain objectives languished – specifically those concerning epidemiology, professional training for rare diseases, and screening and diagnostic programmes - which were considered insufficiently developed. The strategies to meet these goals need to be reformulated taking stock of the difficulties encountered and defining tactics to overcome obstacles.
The tenth axis of the plan, “Develop national and European partnerships in the field of rare diseases” received an overall favourable evaluation with certain propositions presented to enhance and encourage European collaboration. Furthermore, the evaluating committee proposed the development of measures to bring non-European industrialised and developing countries into the fold. Indeed, throughout the evaluation of the plan, the necessity for European- and international-level coordination and resource-sharing is emphasised.
The evaluation, along with an additional report created by the Ministry of Health containing auto-evaluations from the plan’s pilot committee, and the testimonies of rare disease health professional, industry and patient organisation stakeholders who contributed actions to the first plan, will be discussed at the final meeting of the Follow-up committee for the first French National Plan, scheduled to take place on 28 May.
Consult the evaluation (in French language)