Regulatory workshop on clinical trials designs in neuromyelitis optica and spectrum disorders

• Date:
  Friday, 10 October 2014, All day
• Location:
  European Medicines Agency, Amsterdam, the Netherlands

This workshop brings 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. See 'Documents' tab for agenda and 'Multimedia' tab for video of the workshop.

Documents

Report of the regulatory workshop on clinical trials designs in neuromyelitis optica spectrum disorders

Reference Number: EMA/CHMP/SAWP/712652/2014

Agenda - Regulatory workshop on clinical trials designs in neuromyelitis optica and spectrum disorders

Reference Number: EMA/149595/2014

Registration form - Regulatory workshop on clinical trials designs in neuromyelitis optica and spectrum disorders

Speaker biographies - Regulatory workshop on clinical trials designs in neuromyelitis optica and spectrum disorders

Presentation - Setting the scene: Neuromyelitis optica (Jackie Palace)

Presentation - Natural history of neuromyelitis optica (Friedemann Paul)

Presentation - Diagnostic criteria for neuromyelitis optica 2014 (Brian G. Weinshenker)

Presentation - Clinical view on current standard of care in neuromyelitis optica (R. Marignier)

Presentation - Neuromyelitis optica: Is there a standard of care? (Eliezer Katz)

Presentation - Non-Placebo and Add on Clinical Trials (Warren W Wasiewski)

Presentation - Neuromyelitis optica: Is there treatment equipoise? (Bruce Cree)

Presentation - Methods to manage risk in a placebo trial (Susan VanMeter, Sarah Kavanagh)

Presentation - Neuromyelitis optica testimonial - Speech (Irene Wilson)

Presentation - Neuromyelitis optica testimonial (Irene Wilson)

Presentation - The ethics of placebo controlled studies - Speech (Simon Woods)

Presentation - Clinical trials designs in neuromyelitis optica and spectrum disorders: United States Perspective (Wiley A. Chambers)

Presentation - The ethical considerations of placebo study design in neuromyelitis optica (M. Rosa)

Presentation - Clinical trials designs in neuromyelitis optica and spectrum disorders: Europen Union clinical view (M. Isabel Leite)

Presentation - Endpoints in a treatment trial in neuromyelitis optica: Clinician’s view (Anu Jacob)

Presentation - Clinical trials designs in neuromyelitis optica and spectrum disorders: Endpoints industry (Ayesh Perera)

Presentation - Development of drugs for paediatric neuromyelitis optica (Cheryl Hemingway)

Presentation - Development of drugs in paediatric neuromyelitis optica and neuromyelitis optica spectrum disorders: Industry perspective (Josephine Glover)

Multimedia