Clinical trial design workshop in Freiburg teaches the fundamentals of trial planning in rare NMDs

The TREAT-NMD Clinical Trial Coordination Centre has just hosted its second workshop on “clinical trials in neuromuscular disorders and other rare diseases”. Held in Freiburg, Germany from 24-26 June 2010, the workshop welcomed participants from 5 different countries as far afield as Hong Kong, who used this great opportunity to work in a small group and learn in detail about all aspects of clinical trials in neuromuscular disorders and other rare diseases.

The workshop consisted of presentations and practical working sessions in small groups. Speakers included neuromuscular experts from academic institutions, pharmaceutical industry representatives (Santhera and Novartis) and patient representatives (Initiative 'Forschung und Therapie für SMA). Prof. Elbers from the German regulatory authorities BfArM and member of the EMA committee for orphan medicinal products (COMP) presented the agency’s view of drug development in rare diseases.
 
During the course participants learned how to successfully develop and manage a clinical trial according the guidelines of ICH-GCP. Topics covered included regulatory, organisational and statistical aspects as well as adverse event reporting, outcome measures in neuromuscular disorders, and data management. During hands-on sessions participants worked on their own concrete study ideas and discussed their ideas with the group.

Feedback from participants was very positive and the overall evaluation revealed very positive marks for content (1.3), comprehension (1.1), and learning (1.1) on a scale from 1 to 6. (1=very good; 6= unsatisfactory). They felt that the workshop was well-prepared and that it helped to increase their level of awareness and fostered contacts all over the world.

For further information about trial design training opportunities in Freiburg, please contact Kathrin Gramsch.