Cambridge University Hospitals NHS Foundation Trust Joins the Risdiplam EAMS for Adults

23 December 2020

Yesterday, in reply to letters we wrote jointly with MDUK, we were pleased to receive the following email from Roland Sinker, Chief Executive of Cambridge University Hospitals NHS Foundation Trust:

‘Thank you for your email and apologies for the delay in getting back to you. 

I am writing to provide an update from Cambridge University Hospitals NHS Foundation Trust on the use of Risdiplam for the treatment of adults with type two spinal muscular atrophy (SMA). 

As you are aware, the Trust has been undertaking an ongoing review of the use of this drug and this was considered further by the Trust’s Joint Drugs and Therapeutics Committee (JDTC) at a meeting held earlier this week.

While the Committee remained concerned about the lack of evidence as to the drug’s effectiveness and the appropriate dose in adults, it was mindful of the hope it brings to affected families and the Trust’s commitment to support patients in making informed decisions in relation to their health and medical care. 

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.

The health, safety and welfare of our patients is paramount and it has therefore been important for our clinicians to give careful consideration to all the available information in reaching this difficult decision.’

Adults, clinicians and patient groups worked hard to provide information to enable the Committee to reach this position. 

Cambridge joins more than 15 other Trusts prescribing risdiplam via the Early Access to Medicines Scheme. Jointly with MDUK 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.