Page last checked: 19th December 2022

This 26th January 2022 answer is on the NICE website here.

1. Can people who have previously had any treatment for spinal muscular atrophy under a clinical trial (whether in the UK or abroad) have access to risdiplam, regardless of their clinical situation?

Patients / families should discuss their individual situation with their treating clinician. This discussion will need to cover what the terms and conditions were for the clinical trial, including what information about access to future treatments was given to the patient / family when they / their child were, enrolled for the trial.

Finally though, the primary consideration and decision whether the person should have access to risdiplam will be due to clinical reasons. These cases should be discussed with the NHS England SMA Clinical Panel.

2. Can patients who are on risdiplam trials – abroad or in the UK – now cease to be on these trials and receive the treatment on the NHS?

Patients / families should discuss their individual situation with their treating clinician, including how much longer the clinical trial is expected to last.

It’s important for patients to continue on ongoing clinical trials where feasible as trials are able to rigorously collect high quality data that can be used to make decisions about whether or not drugs are clinically effective and can be routinely funded by the NHS. Any patient transferring from an ongoing clinical trial will need to meet the eligibility criteria in the NICE guidance.