Last week (Wednesday 21st June) Joel James, MS for South Central Wales, spoke to the Welsh Senedd (Parliament) in a debate focused on drug repurposing to treat rare diseases including epidermolysis bullosa (EB).

The 8 minutes 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, which can make getting a diagnosis and access to specialist healthcare difficult, and limited or as in the case of EB, no licenced drug treatments being available.

The debate was effective in raising much needed awareness of EB and it again highlighted the ‘win -win’ opportunity that exists to use drugs that have already passed clinical trials and have been brought to market to treat other conditions, so that they can treat people with rare diseases too.

Joel closed the debate by stating that charities and individuals shouldn’t have to find effective treatments on their own, and that 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.

We are very grateful to Joel James for his ongoing support and for raising this important issue in the Welsh Senedd. We also welcome the response from Eluned Morgan, the Minister for Health and Social Services in Wales, and trust we can count on her support to navigate the medicines repurposing programme, which she referred to in the debate.

Read the transcript of the full debate.

 

Commenting on the debate, DEBRA President, Simon Weston CBE said:

The drug repurposing debate, which took place within the Senedd last week, 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 within the Senedd. 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.