|Summary||This workshop brings together patient representatives, healthcare professionals, regulators, Pharma and ethicists to discuss trial designs in NMO, a rare neurological disorder with a natural history worse than multiple sclerosis.
Generally, regulators request randomised placebo-controlled comparisons for evidence of efficacy, even when active treatments are already in widespread use.
In NMO, the choice of comparator is problematic. The severity/lack of reversibility of NMO relapses, indicate that placebo comparators are difficult but evidence for current treatments appears lacking. Different regulatory agencies have different views on comparators in NMO attack prevention.
For a rare debilitating disease with unmet medical need, a wider debate is being held to facilitate drug development for the benefit of NMO patients. Registration open until 1 September 2014. See 'Documents’ tab for agenda and registration form.|