Patients United was pleased to attend the March 13 All-Party Parliamentary Group (APPG) on MND meeting as virtual guests, along with representatives of the My Name5 Doddie Foundation, MND Scotland and Prof Ammar Al-Chalabi, King’s College, London. The topic was Progress in Research and Access to Treatments.
James Smith, who lives with MND, gave a moving account of his diagnosis, living with MND and the effects on his wife and young family. He also spoke about the need for treatments and, in particular, the urgent need for publication of the Mirocals trial results.
Nick Cole from the MND Association gave an update on progress in research and along with Alex Massey, also from the MND Association, pointed to an issue, announced on the day, with the potential approval of Tofersen for SOD1 MND by NICE in the UK. You can read the charities press release here. It is a subject of some concern and we will be monitoring closely. It is important to note, however, that eligible UK patients are currently already receiving the treatment courtesy of Biogen’s generous global EAP program. There should be no doubt that scientifically the treatment candidate is effective. The issue appears to be a technical appraisal concern which we all hope will be resolved over the coming months.
There was then a question and answer session.
Yvette Cooper MP asked what could be done to speed up publication of the Mirocals results. APPG chair, Andrew Lewer MP, said the group would look into this.
Our Patients United representatives, Nicola McFarlane and Dave Setters, were then able to make the following “asks” of MPs.
1) To acquaint themselves with the Mirocals situation as set out in our statement and website.
2) Encourage local NHS trusts to fund the drug on confirmation of a positive trial result once prescribed by the patient’s neurologist via the “named patient” NHS Specials pathway.
3) Ask Mirocals consortium members to provide transparency on its choice of commercial partner, in particular on a) any conflicts of interest arising AND b) the ability of the partner to deliver its new drug at sufficient scale and more quickly than the already available trial drug, Proleukin.
This last point is crucial for the immediate future and beyond because…
- If the commercial arrangements can be clarified this is more likely to result in less confusion and FASTER ACCESS to the trial drug.
- For future trials LESSONS NEED TO BE LEARNED, especially those using repurposed drugs and those publicly funded by UK and EU taxpayers and charity donors
Andrew Lewer thanked us for our “useful” insights and said that the group would look into what could be done.
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