Following my meeting with DEBRA UK President – Simon Weston CBE and CEO – Tony Byrne, I was pleased to open a short debate in the Senedd on repurposing drugs to treat rare diseases such as Epidermolysis Bullosa (EB).
EB is a rare genetic condition that causes the skin to blister at the slightest touch. At its most severe EB can affect the eyes and internal organs. Blinking can cause damage to the surface of the eye, leading to extreme pain, scarring and blindness.
Sadly, 95% of rare diseases do not have a licensed treatment, meaning 175,000 rare disease sufferers in Wales face the terrifying future of no medical treatment being available to them. However, this doesn’t need to be their reality. By investing more resources into studying how existing drugs can be repurposed, sufferers can have more hope and optimism.
In my mind repurposing drugs for rare diseases is a ‘win-win’ scenario. Not only would it improve the quality of life of sufferers, but it can also reduce the amount of care that families and social services are required to provide. Some diseases such as EB, can make prisoners of those who suffer and their caregivers. It can limit their socialisation, ability to form relationships, work and travel. Why wouldn’t we invest in an opportunity to improve their lives?
Investing in drug repurposing can also save the NHS money. Research by King’s College London has shown that certain drugs have reduced skin itch for EB sufferers by 60%, thus reducing bandaging time and the number of out-patient appointments needed. It also suggests a decreased impact on patient and family mental health.
The Welsh Government are missing a golden opportunity to improve the quality of life of those suffering from rare diseases. Charities such as DEBRA UK have done an enormous amount to raise awareness and strive for cures. Yet, it is simply not right that charities and individuals are left to raise funds for drug repurposing trials themselves. The Welsh Government must better understand the needs of rare disease patients in Wales and commit to providing more funding for drug repurposing.
ENDS
