The Regulatory Workshop on Clinical Trials Designs in Neuromyelitis Optica (NMO) and Spectrum Disorders brought together patient representatives, healthcare professionals, regulators, pharma and ethicists to discuss trial designs in neuromyelitis optica (NMO), a rare neurological disorder. For evidence of efficacy, 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.
Please see the EMA page of this website to watch the videos which refer to the placebo and non-placebo clinical trials.