Last week (Wednesday, 21st June) Joel James, MS for South Wales Central, opened a short debate in the Senedd, focused on drug repurposing to treat rare diseases including epidermolysis bullosa (EB).
The 8-minute debate raised awareness of some of the challenges that people living with rare diseases such as EB face, including a lack of awareness and understanding of the condition amongst healthcare professionals. This can make getting a diagnosis and access to specialist healthcare almost impossible. In the case of EB, there are currently no licenced drug treatments available.
The debate was effective in raising much-needed awareness of EB and highlighted the ‘win-win’ opportunity of using drugs that have passed clinical trials and are already in the pharmaceutical market, to treat the rare disease.
Joel closed the debate by stating that charities and individuals should not have to find effective treatments on their own. There is a duty of care to help ensure that patients and their families across Wales have the same consistency of care and that they can access drugs, no matter what their condition, and that they are fully involved in understanding their condition.
Following the debate, Joel said:
Very few people are aware of EB and understand the impact it has on people’s lives. Also known as butterfly skin condition, EB can be extremely debilitating and severely impact the self-confidence of those living with the condition. I can’t imagine what it must be like to live with such a condition and know there is no cure available.
The purpose of the debate was to highlight that effective treatments are needed for those with rare diseases and we are missing a great opportunity by looking further into drug repurposing.
Commenting on the debate, DEBRA President, Simon Weston CBE said:
The Senedd drug repurposing debate is another positive step towards securing approved drug treatments for EB, and we are very grateful to our colleague, Joel James, for raising this issue. We do need help from the Health Minister in both Wales and the other devolved nations though to be able to progress this.
We have had dialogue with the medicines repurposing programme (MRP), but they have not been able to provide clear guidance as to the clinical trial requirements for a rare disease such as EB, and thus we can only conclude that the programme is not designed for rare conditions otherwise there would be a clear data requirement in place. We understand that we need more data, and we need to conduct clinical trials for evidence generation but to be able to plan these trials correctly we need clear guidance from the MRP otherwise we risk conducting a trial and at the end still not being able to enter the programme because the data does not meet the minimum threshold requirements.
People living with EB cannot afford to wait, they need effective treatments as soon as possible, and thus we would very much welcome the continued support of the Health Ministers in Wales, England, and Scotland to help us successfully enter the medicines repurposing programme and ultimately secure approved drug treatments that could help stop the pain of EB.
To watch Joel's contribution to the short debate in full, please click here.