16th July 2018

Last week, we attended a Patient Group Consultative Forum meeting at the MHRA (Medicines and Healthcare Products Regulatory Agency). The forum is designed to help the MHRA engage patients and patient organisation in the regulatory process. The two topics discussed which we believe are of most interest to members of the Duchenne community are the Early Access to Medicines Scheme (EAMS) and Brexit. 

The Medical Assessor for the MHRA gave an overview of EAMS, a scheme which is designed to allow doctors to prescribe drugs which have not yet been approved for marketing to patients with seriously debilitating conditions. Treatments for Duchenne muscular dystrophy may be the perfect candidate for this scheme due to the life-threatening nature of the disease, and the fact there are currently no approved treatments for all patients.

Raxone, a treatment designed to help respiratory function in DMD patients, is on the EAMS scheme. This means that, although Raxone is not an approved drug, some patients are able to access the medicine. MHRA’s review of Raxone included a patient group meeting, which had representatives from the patient community and patient organisations.

Although the MHRA provide a sort of ‘rubber stamp of approval’ on unlicensed drugs through the EAMS scheme, this doesn’t mean that doctors will always prescribe them. One point raised at this meeting was that some health care professionals may not be aware when a treatment has been granted EAMs approval. The MHRA urged patients and patient groups to be proactive in informing healthcare professionals and other members of the community about the scheme, and any treatments which may be on it, so that patients can potentially get access to the medicine through EAMS.

The Head of EU, Brexit and International Policy for the MHRA then gave an insight into how Brexit may affect the work of the MHRA. This meeting happened to fall on the same day the government released its White Paper on Brexit. The White Paper outlined what the government is seeking to achieve in negotiations with the European Union during Brexit. The white paper includes information on the need for goods to flow across borders easily, for items such as time-critical medicines. Three topics regarding the impact of Brexit on the MHRA were covered in this meeting:

  1. Future economic partnerships: The UK is clear in wanting a associate membership of the EMA (European Medicines Agency). It is important to note the EMA is only one part of the EU medicine regulation system.
  2. Implementation period: (after we leave EU, 29th March 2019 through to end of 2020). From the patient and manufacturers point of view very little should change in this period. However, the UK will not be able to lead assessments/vote in committee in this period. This will impact the influence of the UK but should not have minimal impact on human health
  3. No deal readiness: The MHRA would like to clearly state that if we get to a no deal scenario, they will not put barriers in place to stop medicines reaching patients. Although, the MHRA cannot control what the EU will do. The MHRA will ensure public safety is the number one concern if we reach a no-deal scenario.

 

If you have any questions about this, or anything else, please get in touch: [email protected]