Cambridge University Hospitals NHS Trust recommends use of risdiplam

We were delighted to learn today that the Joint Drugs and Therapeutics Committee (JDTC) at Cambridge University Hospitals NHS Trust has now recommended the use of risdiplam for treating spinal muscular atrophy (SMA) in adults (with type 1 and type 2 SMA) through the Early Access to Medicines Scheme (EAMS).

In early December, Muscular Dystrophy UK and SMA UK reported our disappointment that, despite the appeals we and others had made, the JDTC initially did not recommend the use of risdiplam, leaving eligible patients in Cambridge blocked from access to the treatment.

We have been working through various routes to urge the Trust to change its position so that people living in this area of the country who meet the EAMS eligibility criteria can get access to the risdiplam EAMS.

The Trust’s Chief Executive, Roland Sinker, responded:

The Committee was fully supportive of efforts to gain further information about the role of Risdiplam and, with this in mind, it agreed to providing patients with the option to access Risdiplam ahead of NICE approval on the understanding that the benefits and risks will be closely monitored and the outcomes will be collected through the national SMA REACH programme database.

The drug will be provided under the Early Access to Medicines Scheme (EAMS), which aims to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a UK marketing authorisation when there is a clear unmet medical need.

The first patients are expected to begin receiving the treatment in the New Year and, in turn, will contribute to the collection of clinically meaningful and scientifically valid outcome data.

This latest development is great news for eligible people living with SMA and for the local neuromuscular service team, who have been keen to support the implementation of the EAMS once approved by the committee.

Cambridge University Hospitals NHS Trust joins more than 15 other Trusts prescribing risdiplam via the Early Access to Medicines Scheme. Jointly with SMA UK and the community, we will continue to support the further roll out of the EAMS across the UK, working with local neuromuscular teams and Trusts wherever there are challenges.