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Page last checked: 29th November 2022
Last updated: 15th February 2022


The MAOC is responsible for monitoring the implementation of the MAA and recommending actions to support its operation.

This may include:

  • Monitoring the progress of data collection and analyses as described in the MAA and data specification
  • Reviewing enrolment to and continuation in the MAA, including the number of patients who have discontinued treatment
  • Agreeing communications (including for patients, clinicians, service providers)
  • Considering proposed amendments to the MAA (which would be subject to renegotiation by NHS England and the Company)
  • Receiving updates from the NHS England Clinical Panel on patient referrals and prioritisation relevant to the data collection agreement

Membership is:

  • NICE Managed Access Associate Director (Chair)
  • NICE Technical Advisor or Analyst
  • NICE Data Collection Manager (Co-chair)
  • A representative from NHS England (who will also provide updates on behalf of the clinical panel)
  • Two paediatric clinical experts in the treatment of children with spinal muscular atrophy
  • One clinical expert in the treatment of adults with spinal muscular atrophy
  • One physiotherapist involved in the treatment of spinal muscular atrophy
  • Spinal Muscular Atrophy UK representative (patient organisation)
  • TreatSMA representative (patient organisation)
  • Muscular Dystrophy UK representative (patient organisation)
  • SMA-REACH Academic Lead
  • SMA-REACH (Global) Trial ManagerTwo standing representatives from Biogen (company) and 1 substitute representative.