24 August 2023 Research
We are at an unprecedented time in MND research with more positivity around trials than ever before and a number of potential medicines now moving along the pipeline.
As an Association, we have been working in the research and drugs development area for decades, and so have built up a significant amount of inhouse expertise in that area.
We are now in the position, as the leading charity working for people with MND, of being able to look at how to ensure people with MND have timely and equitable access to those treatments proven, through clinical trials, to work. On that score, we are already engaging with the National Institute for Health and Care Excellence (NICE) around the most appropriate route for the evaluation of Tofersen.
This is a relatively new space for us, as we’ve said previously, and one we haven’t operated in before due to the lack of emergence of promising treatments. So it has been – and continues to be - a positive learning curve. As a team, we’ve been helped on that curve by experts in different disciplines, and from different organisations, and by people from our community who have generously given their time.
A key part of that learning has been to really understand all of the processes medicines have to go through once clinical trials show a drug is safe and effective, before they reach people with MND. That includes both the regulatory processes and early access programmes. These processes can be lengthy, complicated and involve multiple agencies. Often this is for good reason – it’s important to ensure medicines available for prescribing by clinicians are safe, effective and available to all they would benefit.
By gaining that solid understanding of all the stages involved, we now have real clarity on where we can add value, influence decisions and help to speed up processes.
Just as importantly, we have also learned – through trial and error in some cases - where we need to take a step back because there isn’t the option to influence or affect decisions.
In the same way, there are organisations with more expertise than us in some of these areas. Where that’s the case we will share their information and details with you so you can be as informed as possible.
This puts us in the strong position of now being able to focus all our efforts on those specific areas where we know we can make a real difference to people living with MND. That includes, importantly, working with the MND community to ensure your voices are heard by decision-makers. On that note, we are in the final stages of gathering information from a group of people with MND to add to our application to NHS England’s Drug Repurposing Scheme – that will be submitted next week. We have already asked how long it may be before we receive a response and been told once they have had sight of the submission they will let us know.
There is strength in numbers, we know that from our successful campaigning history with other charities and alliances. For that reason, we are actively seeking out organisations and alliances which already work in the access to medicines space, and charities which have been involved in navigating the systems. We want to learn more from them, collaborate with them and, if there are opportunities, to campaign with them.
MIROCALS
As I promised we would, we are continuing to liaise with the MIROCALS trial team about the results of the Interleukin-2 trial which we, as an MND community, are waiting for. I spoke to the team this week to, once again, urging them to release the data as soon as possible and to remind them of the strength of feeling in the community about this study. They are aware of the need to publish the data as soon as they can while reiterating their focus on ensuring they have done a thorough job so future processes and decisions are based on absolutely accurate data and information.
I am also continuing to seek legal clarity on our role in the Consortium and, when I have that, I will share what I am able to.
Moving forward
In the meantime, the work we are doing in this area is continuing with a number of our team dedicated to it. We will continue to update you over the coming weeks, as promised and we remain committed to our ultimate goal of supporting our MND community to have access to proven treatments.
Tanya Curry, Chief Executive