Registration opens for workshop on clinical trial design in neuromyelitis optica to facilitate global medicine development in a rare and serious disease

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10/07/2014

Registration opens for workshop on clinical trial design in neuromyelitis optica to facilitate global medicine development in a rare and serious disease

Registration is now open for a regulatory workshop on clinical trial design in neuromyelitis optica and spectrum disorders at the European Medicines Agency on 10 October 2014.

Expressions of interest should be sent using the registration form provided to jane.moseley@ema.europa.eu by 1 September 2014.

The aim of the workshop is to better understand the most appropriate choice of comparator to be used in the clinical development of new medicines for the prevention and treatment of neuromyelitis optica.

Neuromyelitis optica is a rare inflammatory disease of the optic nerve and the spinal cord that can lead to the reduction or loss of vision as well as weakness and paralysis of the arms and legs. It is a debilitating disease that can be life threatening for patients because of damage to the nervous system function.

The choice of comparator to be used in clinical trials has proven to be very difficult due to the severity and irreversibility of the relapses in untreated patients and the evidence base for standard of care.

Regulatory agencies across the world have taken different standpoints on the appropriate choice of comparator for the assessment of the safety and efficacy of new medicines intended for neuromyelitis optica attack prevention.

Given the significant unmet medical need of patients with this disease and the need for a global development due to the limited patient population, the EMA has decided to organise this workshop to kick off a wide debate to facilitate medicine development for the benefit of patients with this disease.

This workshop aims to bring together patient representatives, clinical neurologists and other health care professionals, methodologists, European and non-European regulators, medicines developers and ethicists.

The workshop will be broadcasted live on the EMA website.

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