Page last checked: 17th December 2024

Access to Risdiplam and Nusinersen

The NHS England Clinical Panel’s role is to ensure that decision making by Centres across the country about disease-modifying treatments is consistent and equitable. This includes access to one of these drugs as a second treatment following Zolgensma treatment.  This is only possible if the treating clinician has made a referral to the panel. This must say that, in their opinion, Zolgensma treatment has not been successful. The clinical panel must agree. All other eligibility criteria for the second treatment would also apply.

The definition of unsuccessful Zolgensma treatment >

The Nusinersen and Risdiplam Managed Access Agreements:

The Panel is there to:

• Provide advice to clinicians at treatment centres on interpretation of the MAA criteria, including: starting and stopping criteria, and diagnosis
• Advise NHS England on any patients who appear to meet stopping criteria but who remain on treatment
• Give advice to clinicians at treatment centres on patients who have received Zolgensma but in whom there is disease progression

In the specific case of nusinersen, it is also there to:

• give advice to clinicians at treatment centres on the feasibility of safe intrathecal administration of the drug, particularly in light of spinal surgery and taking into account spinal instrumentation.

In the specific case of risdiplam, it is also there to:

• give advice to clinicians at treatment centres on patients where eligibility for the MAA is unclear but who otherwise are benefiting or might potentially benefit from treatment
• advise clinicians on switching treatments where appropriate. See NICE website – questions and answers on the managed access agreement for risdiplam (January 2022) 

Membership of the Panel:

• Representatives from NHS England (who chair and run the Panel)
• Pharmaceutical adviser
• Two neuromuscular physicians who are expert in the treatment of children with SMA
• Two neuromuscular physicians who are expert in the treatment of adults with SMA
• Two physiotherapists who are expert in the treatment of patients with SMA

The Panel usually meets fortnightly on a virtual basis to allow rapid responses to queries.

Queries to the Clinical Panel are submitted via clinicians at treatment centres on a standard template (available from NHS England) to ensure that all relevant information is presented to the Clinical Panel. The chair of the Clinical Panel responds to the clinician in writing. The clinician may share this response with the patient or family if they wish. The clinical panel is also happy to consider any cases submitted by clinicians in Wales or Northern Ireland.