The course will take the participants through the main concepts and practical steps that must be undertaken in the establishment and management of a rare disease registry to ensure its usefulness, soundness and sustainability. The course will provide basic notions on the methodology of observational studies with a view to the specificity of rare disease registries, on the selection of data elements with a focus on the interoperability of rare disease registries, on quality assurance and on the technical and legal tools that must be adopted to protect patients’ data confidentiality. The course will consist of frontal presentations followed by small group exercises.
24 April 2013 – application is open
30 July 2013 – deadline for application submission
1 August 2013 – confirmation of admittance
16-20 September 2013 – course
The course is open to the following profiles:
– professionals involved in the health care of rare disease patients;
– professionals working in public health institutions in the surveillance of rare diseases;
– persons working or volunteering for a patients’ association;
– professionals working in the pharmaceutical industry and especially in the development or surveillance of orphan medicinal products.
Priority will be given to participants:
1) already involved in the conduction and management of a rare disease registry or patient data collection;
2) with a concrete intention to establish a new registry for a rare disease.