In 1997 I was a young twenty-something, ‘climbing the corporate ladder’ of a pharmaceutical company and dreaming of becoming a Barrister. But after developing swollen fingers with tight, shiny skin and a variety of symptoms, I was diagnosed with the rare autoimmune disease, scleroderma and Raynaud's. I was told nothing could be done and given just 15 months to live.
The prognosis was devastating but not one to accept ‘it’s over’ for an answer, and with my dream job still in sight I started looking for answers. 12 months later, I discovered the Scleroderma Unit at the Royal Free Hospital in London and heard about Clinical Trials there for my condition.
Fast forward another 17 years, including 4 spent studying and passing exams to become a Barrister; many clinical trials later, my skin has been cured of Scleroderma. I’m now very keen to spread the word about research and have my own website, blog, Facebook Page and Twitter feed to help inform others with rare diseases.
In 2013 I became a patient advocate on the NHS England Clinical Reference Group for Specialised Rheumatology; In 2013, a patient expert adviser with the European Medicines Agency; and in 2018 NIHR, Patient Research Ambassador Initiative. Having blogged about the importance of an early diagnosis; the role of medical research; the importance of expert specialist centres; and my personal experiences of taking part in research trials, I was already very involved in promoting the interests of patients and research, so officially joining the PRA programme was a natural next step.
I am eternally grateful to the clinicians whose commitment and dedication to unlocking the scleroderma enigma, is nothing other than superhuman. I have seen the immense good that can come of research and our NHS and wanted to share my experience. I very much hope to utilise my professional skills and qualifications along with my patient experience, to help spread the word about research.
Living with a rare disease is daunting in every way imaginable. In my view, Research provides immense hope. Hope that the desperately needed understanding as to cause and cure will be discovered. With an improvement in medicines, being a soother in the meantime.
Over the course of the last few decades, I have taken part in several clinical research trials and been involved with many clinical trial design setups. In relation to the clinical trial design for scleroderma patients: ALL clinical trial assessment criteria now include digital ulcer involvement, further to my insistence!
As well as my blog and website, I give interviews for press articles about research and earlier this year I took part in a live Twitter chat with North Thames CRN for Rare Disease Day.
I also review documents for the European Medicines Agency. As well as, I have taken part in, (and still do), various Scientific Advice Working Party SAWP and Committee for Orphan Medicinal Products COMP meetings.
I’d certainly recommend getting involved in a Clinical Trial to any patient with a rare disease, in fact to anyone at all, if given the opportunity. I’m now 14 years immuno-suppressant / chemotherapy free. But even if you don’t benefit directly, it helps advance medicine and understanding and can be beneficial for other patients with a health problem in the future. Above all, it gives you an insight into the work being done to help treat diseases and that provides immense hope.