18 August 2023 Research
We must begin this week’s update by acknowledging this is an unprecedented moment of opportunity for the MND community. Every member of the MND community from patients and their carers to clinicians, researchers and regulators are having to rapidly adjust in a world of emerging treatments.
The MND Association as the largest MND patient organisation in the country, with more than 9,000 members, an active research programme and a support programme built around a network of care centres, is committed to embracing the opportunity this presents - ultimately to achieve the vision of a world free from MND. We are totally committed and focussed in working collaboratively to achieve this.
That having been said, we are the first to acknowledge that we have made some mistakes and are committed to remedy these.
We fully acknowledge the strength of feeling and the huge amount of work that a number of individual patients and groups of patients have put into driving forward the access to medicines for people with MND. This includes pushing for and getting meetings with key individuals and organisations. We were not clear enough saying this in our last update, for which we apologise.
NHS repurposing and early access
In last week’s update we informed you that repurposing of Proleukin (high-dose IL-2) for use by MND patients doesn’t fit within the criteria of the drug repurposing scheme. The NHS England Drug Repurposing Scheme is a process which identifies and develops opportunities to use existing medicines in new ways - to improve patient outcomes.
We interpreted our informal conversation with the NHS England Drug Repurposing Scheme as a final decision. However, since this update we have received a further communication from the team and the encouraging news is that we can make a formal application to the scheme on behalf of MND patients. The NHS Repurposing Scheme has also offered the opportunity for further discussions.
We are of course making the formal application and although it's difficult to involve everyone in that process, we will make sure we liaise with representatives from the United2EndMND patient group as the patient contribution is vital.
In addition to this, we are continuing to investigate the multiple paths for early access for low-dose IL-2. Another route we are looking at is compassionate use. This is where a pharmaceutical company, ahead of regulatory approval of their drug, can provide the unapproved treatment free to the NHS and it can then be applied for on a named-patient basis - in certain circumstances.
MIROCALS
People who took part in the original MIROCALS trial may be able to receive the drug free-of-charge from the manufacturer, in this case ILTOO Pharma, and we are working to speed up this process. We are in discussions with ILTOO Pharma (who already have some supplies of the drug, although a limited supply) and the lead trial investigators to take this forward.
We are very conscious that there has been some criticism of the MND Association both in terms of its role in the MIROCALS consortium and our perceived in-action in trying to get early access to IL-2. We’ve heard this via social media, in some conversations with patient groups and from some individuals.
We signed the agreement to be part of the MIROCALS consortium in 2015, and our role as a member was to provide some grant funding and ensure the trial concluded, which it has. However, we recognise the position we are in is very challenging. We are a consortium member but are primarily an organisation who is here to represent and fight for our MND community. We understand the frustration and criticism we are facing and are trying hard to gain legal clarity around this.
Moving forward
We will continue with our weekly updates and the FAQs, which we are updating regularly. We have also been contacting some people individually where they’ve raised issues with us. We are being transparent, but recognise we sometimes need to be much quicker in our actions.
We are clear as an Association we are absolutely working for the same goals and we will continue to work together with the MND community to get the best possible outcomes for people with MND and their families.
Tanya Curry, Chief Executive
Further Reading:
Previous statement on removing barriers to access from Chris James, Director of External Affairs