Final Report Summary - EUROWILSON (Wilson Disease: Creating a European Clinical Database and designing multicentre randomised controlled clinical trials) One of the first key tasks of the consortium was to design and develop the database, which was web-based using secure CPS (Carte Professionnel du Santé) cards and card readers to enter data. Diagnostic criteria for WD were established by an expert group, and algorithms created within the database to automatically validate the diagnosis in newly entered cases. Cases which did not meet the diagnostic criteria were reviewed by a validation committee, whose chairman then initiated an e-mail exchange with the data-entering clinician, usually to suggest further investigation. It was also a requirement that the patients be phenotypically characterised. This was easier for hepatic manifestations, which can be recorded in a tick-box (e.g. presence or absence of jaundice), than for neurological abnormalities. Controversy existed as to which physical signs are most important and how they should be assessed and recorded. To assist this, a neurological rating scale was devised and a DVD prepared to show each physical sign and examples of varying degrees of severity. The database was established to record details of patients with WD presenting in Europe since 1 January 2005. However, the collection of retrospective data is also possible. EUROWILSON was innovative in developing an EU database which also responded to clinicians, centres or national body requirements such as the French national rare disease plan or GeneMove in Germany. For example, French speakers use a translated version of the EUROWILSON database with additional pages and nominative data which communicates with the EU anonymous clinical database, thereby avoiding the possible duplication of databases. In the future, the Committee for Medicinal Products for Human Use (CHMP)/European Medicines Agency (EMEA) statement that registers should also be used to supply important information on the natural course of disease and may help in the assessment of orphan drug safety and efficacy, will be taken into account. The initial purpose of the database, to assess the feasibility of RCTs, was achieved, but in doing so, the project established a rich data resource on epidemiology, quality, and current choice of treatments. Follow-up of this uniquely well-documented cohort will provide information about outcomes from different treatment regimes. Longer term follow-up is necessary to study late neurological deterioration, neurological outcome after transplantation, and other current long-term concerns. Furthermore, the EUROWILSON database was linked to the French database within the national plan for rare disease (these national plans and European recommendations were being discussed in several countries at the time). Because these plans do not have the same duration as the EU projects, and because registries will supply important information on the history of the disease and for the development of orphan drugs, the database should continue. Related documents Final Report - EUROWILSON (Wilson Disease: Creating a European Clinical Database and designing multicentre randomised controlled clinical trials)
