NICE makes recommendations on new medicines by reviewing clinical and cost effectiveness evidence. When a medicine shows promising potential but there is significant uncertainty in the clinical evidence, it may be recommended for time limited NHS funded access in England as part of a Managed Access Agreement. This is a way for patients to receive promising new treatments, while further evidence is collected to assess the long-term benefits of a new medicine. Based on the evidence available when NICE made its decision about recommending risdiplam (Evrysdi) the long-term benefits for patients were still very uncertain.

The Risdiplam Managed Access Agreement was finalised in December 2021. You can read this here.

You can find out more about the announcement that was made about it by NHS England, here.

This MAA has been agreed between:

• NICE
• NHS England and NHS Improvement
• Roche (the drug company that makes Evrysdi™)
• SMA REACH UK – on behalf of the clinical network

The MAA has been designed to allow enough time for additional evidence to be generated for NICE. At the end of the MAA period, NICE will review the new evidence and review its guidance to indicate whether the medicine should be recommended to use in the NHS – this may result in a difference to what the NHS will pay for the drug for example. While most topics recommended for managed access go on to be recommended for routine use on the NHS, there is no guarantee that it will be recommended when it is reviewed by NICE.

It’s anticipated that the Roche will resubmit the new evidence available to NICE in March 2024. The submission will include the data that’s been collected from patients throughout the MAA (see what data is being collected and why) Patient groups and clinicians can also contribute to this process and make submissions.

The NICE appraisal committee will then evaluate the evidence submissions. They will consider both the clinical and cost effectiveness of the drug and make a final decision on whether risdiplam should continue to be funded on the NHS after the MAA has expired.