Myotonic Dystrophy Foundation

On June 12-14, 2009, MDF board members, Lisa Vittek and Don MacKenzie, attended a workshop entitled Patient Registries and Trial Readiness in Myotonic Dystrophy, jointly sponsored by TREAT-NMD, in Naarden, The Netherlands.  The twenty-six participants represented eight countries and covered a broad cross-section of the myotonic dystrophy field: scientists, clinicians, patient representatives and industry. The workshop built on the foundations established in two previous ENMC workshops on myotonic dystrophy and the clinical DM working group, and took advantage of the tools developed within the TREAT-NMD network for patient registries and outcome measures.

In recent years the need to collect patient data in a harmonized manner across multiple countries has become increasingly evident. This is particularly true in the rare disease field, where locating patients suitable for a particular trial or therapy poses even more challenges. The first day of the workshop was therefore devoted to establishing consensus on a core dataset for a TREAT-NMD international patient registry for myotonic dystrophy. Presentations on the existing patient data collections held at various institutions across the world were combined with overviews from industry and patient organizations on their expectations from this initiative. Industry participants stressed the value of an international approach which gives them rapid access to the patients who might be eligible for their trials and allows them to make feasibility assessments with substantially less effort than before. Pivotal trials will always need to be multinational in order to gain marketing approval worldwide, and this again speaks to the need for data to be pooled in a single international resource. Patient representatives were keen that registries should provide two-way communication, that patients should have a say in the way registries are developed and that they should offer ongoing feedback and act as a conduit for reliable information to patients and families. ...to see the complete article, visit TREAT-NMD's website.