On Monday a meeting was convened by Siobhain McDonagh MP at which she was joined by three other All Party Parliamentary Group on Brain Tumours (APPGBT) members, Daisy Cooper, Holly Mumby Croft and John McDonnell.
Also there were Florent Texier (Managing Director UK & Ireland) and Kelly Gomes (Medical Lead – Oncology) from Servier Laboratories the manufacturers of Vorasidenib.
They were asked to give the current picture regarding future access to Vorasidenib for UK low grade glioma patients. Brain Tumour Research as providers of the APPGBT’s secretariat were also in the room for this meeting.
The meeting began with Servier explaining that the trial results for Vorasidenib had enabled the trial to be concluded 18 months earlier than planned and that their “priority now was to give access to this drug” and that they were working tirelessly to do so.
Whilst acknowledging that the production of the regulatory dossiers needed to gain market access is, indeed, a huge piece of work the attending Parliamentarians were keen to hear of Servier’s plans to get Vorasidenib available for those that need it now.
Servier confirmed that they will be sending off worldwide licencing applications including in the UK.
They will submit their application to the European Medicines Agency (EMA) early 2024 and have been granted an accelerated assessment by the EMA.
Once and if, a positive opinion from the CHMP (CHMP is the EMA’s committee responsible for human medicines) is granted in the course of 2024, Servier are then able to send off the application to the MHRA. The application to the MHRA will come under the International Recognition Procedure (IRP).
The IRP is a new procedure that allows medicinal products approved in other jurisdictions (such as Europe or the US) that meet certain criteria to undergo a fast-tracked MHRA review to obtain and/or update a marketing authorisation.
Servier estimate that in the best case scenario, Vorasidenib could be licenced in the UK by the end of 2024 although a further six month wait (June 2025) is a possibility depending on the level of assessment needed.
Early Access early access to medicines scheme (EAMS):
The early access to medicines scheme (EAMS) aims to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorisation when there is a clear unmet medical need.
Alternatives:
Rather than an EAMS scheme via the MHRA the alternative route for early access could be a Named Patient Programme (NPP) that provides patients and clinicians access to medicines that are not available to them in their own country where there is a high unmet clinical need.
Servier currently see this as their preferred route and although they cannot comment about the eligibility for this scheme at the moment they explained that it will be similar to the eligibility criteria that was used for the Vorasidenib clinical trial.
They are looking to open this by the end of the first quarter in 2024.
Other areas discussed on Monday
- Supply issues: When the clinical trial ended, Servier provided Vorasidenib to applicable patients on the trial who had been on the placebo arm. This took up a lot of the existing supply of the drug. Servier cannot open early access schemes until they have supply to meet expected demand. For the last six months they have been upscaling production and have made marked progress in this area.
- Servier are a multi-national company and authorisation for the progression of licensing documentation and similar regulatory procedures can be complicated and time consuming. Assurances were given that personnel are working around the clock to progress UK regulatory and market access.
- NICE – MPs heard that a NICE appraisal will be requested by NICE as part of their horizon scanning but this could happen whilst the licence application is still ongoing meaning that in an ideal world the MHRA and NICE are working in tandem and this would speed up patient access.
- NHS funding for long term survivorship in relation with access to Vorasidenib
Siobhain McDonagh said at the end of the meeting that it was appreciated that Servier had come to Westminster and that there were positives to take away. The case remained that new therapeutics delivered hope and that for them to be unavailable to UK patients, be that due to regulatory bodies, issues with the UK market or whatever, was unacceptable and that members of the APPGBT saw it as their duty to challenge the barriers and move things forward so funding for drugs that bring hope such as Vorasidenib could be available in the UK as soon as possible.