1 August 2023 Research
Since last week's update on this vital work, our Chief Executive Tanya Curry has, as promised, formally written to the lead physician for the MIROCALS study to understand more from them about the publication of the data. This step is required before there can be any move towards people with MND accessing the drug.
Our message was that the release of the data must happen urgently – we also asked for a timescale to be able to share with the MND community.
In line with all clinical trials, the results are owned and will be published by the trial Sponsor – in this case the University Hospital of Nîmes, and the Consortium as a whole. That involves 11 organisations spread across five European countries which must act within legally binding commitments.
We have subsequently contacted the University Hospital of Nîmes to share our concerns about the delay in publication of the data and urged them to put in place the processes needed to publish the full set of results as soon as it is available.
During our conversations, the lead physician assured us they are working hard to finish the Clinical Study Report (CSR) – this is the key document required by the pharmaceutical company involved (ILTOO) and by regulatory authorities (including NICE). They are working with a skilled medical writer with specific knowledge of the regulatory requirements.
At the same time, a scientific paper is also being drafted for publication in a medical research journal. Although the information provided in the scientific paper does not need to be as detailed as the CSR, the results must be identical. There is no way of cutting corners with the analysis.
The investigators emphasised how important it is to get the information, figures and analysis absolutely correct at this point, or risk rejection by the regulatory authorities due to error. This is time consuming but absolutely vital at this stage.
We want to emphasise that our focus is on early access - but we still need the final data to be able to push for that.
The MND Association has reiterated its previous offer to fund the recruitment of more staff to help with this process and this offer to provide further and immediate support remains on the table.
The MIROCALS research team is very aware of the frustrations of the community around the long wait for the full results of this trial and is working at speed to produce the necessary documents. They anticipate the CSR and scientific paper being completed by early Autumn.
The MND Association doesn't hold the legal authority, intellectual property or the clinical data for the MIROCALS trial. However, we will continue to press those who do, including the trial research team, Consortium members and the Sponsor, for the results to be published at the earliest opportunity. And, as promised, we will share regular updates as this work progresses.
In the meantime, our Taskforce is continuing to work with a range of experts to ensure that, once positive results from any of the many clinical trials currently underway are received, we can support their progress through the regulatory system and directly to people with MND.
Dr Brian Dickie, Director of Research Development
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