From the Press Release:

TREAT-NMD–EMEA workshop on clinical outcome measures in SMA signals collaborative approach to future clinical trials

TREAT-NMD, an EU-funded ‘network of excellence’ that aims to accelerate cutting-edge treatments for rare inherited neuromuscular diseases, announces a successful meeting on spinal muscular atrophy (SMA) outcome measures with the European Medicines Agency (EMEA).

A TREAT-NMD-led workshop hosted at the offices of the EMEA in London helped set the collaborative agenda for future trials in SMA. Participants included 50 representatives from the neuromuscular field, including healthcare professionals, scientists, patients and pharmaceutical industry representatives. EMEA representatives included the chairs of the Human Medicinal Products, Paediatric Medicines and Orphan Drugs Committees. There was active participation from all parties. Input from the International Care Committee (ICC) for SMA ensured that there was global representation at the meeting, the outcomes of which will also be shared with the US Food and Drug Administration (FDA).

In a new development for the neuromuscular field, the workshop focused not on discussing product-specific issues but on establishing broader common ground between the regulatory authorities and those interested in running clinical trials in SMA. In order for trials to move through the approval process without delays, consensus between trial planners and regulators on endpoints and novel methodologies is essential.

The SMA community is working extensively together and the meeting demonstrated this close link as all present spoke with a united voice on the most appropriate outcome measures for particular clinical situations. The community was complimented on its proactive approach to regulatory topics, its organisation and its international teamwork in addressing clinical trial questions for SMA.

To read the full press release, click here.