Cynulliad Cenedlaethol Cymru

In Wales, we take an evidence-based approach to deciding which treatments should be routinely available in the NHS. This is to help ensure the best care possible is available for the people of Wales. We rely on the expert advice of the National Institute for Health and Care Excellence (NICE) and our own appraisal body, the All-Wales Medicines Strategy Group (AWMSG). All medicines that have been recommended by NICE or AWMSG are routinely available in Wales where clinically appropriate.

In September 2023, NICE published guidance relating to Birch bark extract (Oleogel-S10) for treating skin wounds associated with dystrophic and junctional epidermolysis bullosa. Details can be found here: Overview | Birch bark extract for treating epidermolysis bullosa | Guidance | NICE

Our patient population in Wales affected by epidermolysis bullosa (approximately 10 patients) are currently seen in DEBRA centres in London and Birmingham, with clinical support delivered locally in Wales. If these specialist centres prescribe Birch Bark extract, these treatments will be supported. However, we are unaware of such treatments are currently being prescribed.

NICE is currently in the process of evaluating the clinical and cost effectiveness of Beremagene gerperpavec for treating skin wounds associated with dystrophic epidermolysis bullosa. However, the project is in its early stages and no date has been set for publication. The progress of this evaluation can be found here: NICE Evaluation - Beremagene gerperpavec.

There are limited opportunities for our researchers and patients to be involved in Rare Disease research in these new treatments, but the recent Rare Disease Research landscape report has enabled us to consider the data gathered in the report, to look for strengths and gaps with a view to being able to find ways to assist Welsh researchers access more support.

We will continue to monitor developments and offer support where required.