NICE’s Final Guidance for Risdiplam confirms the Managed Access Agreement will go ahead

16 December 2021

NICE’s Final Guidance for Risdiplam confirms the Managed Access Agreement will go ahead

We are pleased to see this Guidance and all related papers published today on NICE’s website. 

You can see the Final Appraisal Document (FAD)
the Managed Access Agreement (MAA) and Appendix A (about the use of disease modifying medicines for SMA after onasemnogene abeparvovec) on the NICE website here and information for the public here.

Last week’s live webinar with an expert panel:

  • Prof. Muntoni (Consultant Paediatric Neurologist, GOSH);
  • Dr. Chiara Marini Bettolo (Consultant Paediatric and Adult Neurologist, Newcastle);
  • and Fiona Marley (Head of Highly Specialised Commissioning, NHS England & Improvement)

is now available to watch below. The webinar covered the planned roll-out of the programme in England and answers many questions the Community has been asking:


 

For more information about Risdiplam, please see this page.