This year the Managed Access Agreement for Spinraza and Risdiplam expires. The MAA was put in place five years ago so the pharmaceutical companies could gather more evidence to resubmit to NICE for approval on the NHS with Biogen extending their MAA by a year.
Over the last five years TreatSMA has been on the MAA board for both drugs along with other charities, professionals and clinicians which looks at appeals for either a drug where a individual falls outside the criteria or where a individual wants to swap treatment.
Along side SMAUK and MDUK TreatSMA submitted their nominations for patient experts on the NICE panel representing the community.
For past appraisals TreatSMA has got at least one seat.
- For the drug Spinraza our trustee Gennadily got chosen as a patient expert and given a seat on the panel.
- For Risdplam both our trustees Lucy and Andi both got chosen as patient experts and got two seats.
- For Zolgensma Gennadily got chosen again as a patient expert.
With this previous experience in mind TreatSMA, along with SMAUK and MDUK, have nominated Gennadily, Andi and Lucy from TreatSMA and Portia from SMAUK.
Now only two seats are available but given in previous appraisals each drug was allowed two or even three patient experts (and this appraisal is for two drugs) the charities will be pushing for four seats.
Next week SMAUK is hosting a webinar with TreatSMA and MDUK and will be addressing all potential questions asked by the community.
Pls let us know if you have any in the comments or email to treatsma@treatsma.uk
