Following a request by SMA Europe and together with Treat-NMD, the European Medicines Agency (EMA) hosted a one-day workshop on SMA. This workshop took place on 11th November 2016, at EMA headquarters in London, to allow SMA stakeholders (patient representatives, doctors, industry representatives, researchers and regulators), to have an open forum discussion on the current challenges that therapy development for SMA faced.
The programme for the day was divided into three sessions that covered a number of topics including:
Very importantly, the patient perspective on clinical trials and drug development was also presented. The importance of patients and carers’ input and involvement in the development of clinically meaningful outcome measures and how these are used in regulatory discussions and decisions was underlined throughout the day.
This initiative was very important as EMA plays a crucial role in the innovation and availability of medicines within the European Union and beyond. The Agency aims to improve the science and process of medicines regulation, in order to foster the development of medicines for unmet medical needs, such as SMA and to optimise their surveillance and management on the market. To do this, the agency fosters scientific excellence by ensuring it is aware of the latest scientific knowledge and methodologies by targeting engagement with stakeholders in a range of areas. We were therefore delighted that the agency recognised the high need for SMA and agreed to host a workshop.