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. It’s role for the nusinersen and risdiplam Managed Access Agreements is described further below.

Additionally, access to one of these two NHS-funded disease modifying second treatments is only possible if the treating clinician has made a referral to the panel to say that, in their opinion, Zolgensma treatment has not been successful, and the clinical panel agrees. All other eligibility criteria for the second treatment would also apply. Read more here.

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 who are on more than 16 hours of ventilation per day but who otherwise are benefiting/might potentially benefit from treatment and who should be included on the MAA.
• Give advice to clinicians at treatment centres on patients who received treatment under the Early Access to Medicines Scheme and who do not meet the eligibility criteria in the NICE guidance. This advice may conclude:
  • The patient is deriving benefit and should be included on the MAA (and be funded by the NHS)
  • The patient is deriving benefit but should not be included on the MAA (and be funded by Roche)
  • The patient is no longer deriving benefit and should not continue on treatment

Membership of the Panel comprises:

• 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 / family if they so wish. The clinical panel is also happy to consider any cases submitted by clinicians in Wales or Northern Ireland.