Open wounds on arms of person with RDEB

In September, NICE (the National Institute for Health and Care Excellence) confirmed that Filsuvez®, a gel developed as a treatment to promote the healing of partial thickness wounds associated with dystrophic (DEB) and junctional (JEB) EB, was to be approved as the first EB drug treatment available for prescription in the UK.

To get to this point took a lot of effort from many different parties including of course the pharmaceutical company who developed the drug, Amryt Pharma, plus members of the DEBRA UK team, and crucially DEBRA UK members.

The member involvement, which included DEBRA UK members providing a patient perspective and sharing their unmet needs, was a key contributor to the approval of Filsuvez. The patient testimony provided helped NICE’s Highly Specialised Technology Evaluation Committee (HSTEC) assess the evidence for this topic and produce their guidance recommendations.

HSTEC members and the NICE team found the DEBRA UK members involvement to be very comprehensive and the input useful, particularly with regards to assessing the direct impact of EB on the patients’ quality of life, which helped put the clinical effectiveness into context. Hearing from a patient living with EB also allowed HSTEC to factor in the huge impact that even small quality of life gains could have on an EB patient, e.g., hearing the material impact on individuals of the potential for reducing dressing changes even by a small amount of time each day.

The DEBRA UK members patient testimony also helped validate other submissions including hearing directly about how the time spent on dressing changes was like a full-time job, and what it entailed simply to be able to join the meeting was deemed incredibly powerful.

By providing additional information about the experience of the condition that could not be captured in the data particularly around the extent of the pain, the impact of itching, and what a meaningful improvement would be, and by gaining detailed insight into the quality-of-life considerations of the condition, the committee members got a new appreciation of the severity of EB which they wouldn’t have got without the expert patient testimony provided.

It is clear from the feedback received from NICE that the patient testimony received during the approval process for Filsuvez was crucial to achieving a positive assessment and ultimately approval for this drug treatments to be made available to DEB and JEB patients aged 6 months+ in the UK.

On the behalf of everyone at DEBRA UK and the UK EB community we would again like to thank our members who participated in this process for the powerful patient testimony they shared, which has helped ensure that the UK will have the first of hopefully many approved drug treatments for EB.

There are many ways that you can get involved with DEBRA, from involvement in research projects, to sharing your lived experiences of EB and helping us improve the resources and support we offer to the wider EB community. Every action you take will have a positive impact on others living with EB.


Find out more about how you can get involved