Action to Address Lack of Access to the Risdiplam Early Access to Medicines Scheme for Adults in the East of England

04 December 2020

SMA UK and Muscular Dystrophy UK were extremely disappointed to learn this week that, despite the appeals they and others have made, the Joint Drugs and Therapeutics Committee (JDTC) at Cambridge University Hospitals’ Trust has decided not to recommend the use of risdiplam for treating Spinal Muscular Atrophy (SMA) in adults (who have SMA Types 1 and 2) through the Early Access to Medicines Scheme (EAMS).

The local neuromuscular service team would have been keen to support the implementation of the EAMS if the Committee had approved it. We are working through various routes to urge the Trust to change its position and, in the meantime, to address how people living in this area of the country who meet the EAMS eligibility criteria can get access to the risdiplam EAMS.