It would be silly of me not to first mention the recent excitement around the Duchess of
Cambridge expecting another baby. Having attended the World Federation of Hemophilia
Congress in Orlando last summer, I know how fond Americans can be of our monarchy. It is
well-known that haemophilia was carried through the royal family. I have to say, without
committing ‘treason’ that Patrick, as a fellow haemophiliac, is every bit as much royalty to
So, I am honoured to be able to share my perspectives from the ‘across the pond’ for
My path into advocacy was cemented with The Haemophilia Society. They are the onlycharity for everyone affected by a bleeding disorder across the whole of the United
Kingdom (UK). With a team of dedicated staff and a network of super motivated volunteers,
they can offer vital help and support where needed to its over 5,500 members and the
In July, the news broke that a public inquiry - a legal investigation conducted by a judge
appointed by the Government - will be held into contaminated blood products. As in
America and many parts of the world, a large proportion of blood products supplied to
patients during the 70s and 80s in the UK were infected with HIV and/or hepatitis C.
This is bittersweet for The Haemophilia Society and activists, who have been campaigning
tirelessly for answers over decades-long. Thousands of people, the majority with
haemophilia and their families, were and still are devastated by this tragedy. Sadly, this
includes my own, with my mum losing both of her parents.
Consequently, I never got to meet my grandad and often wonder about what his life was
like with haemophilia at my age in 1953. Back then, there was no such thing as preventative
therapy and when he had a bleed, all he could really do was have bed rest and to see it
through. What would he think of my haemophilia treatment and care today?
As a moderate but with a severe phenotype, I have been able to self-treat prophylactically
since I was around 10-years- old. Just recently, through personalising my care plan with my
specialist clinical team, I have been able to switch to an extended half-life product, which
are now on license here. This has meant that I no longer have to infuse daily, whilst offering
me a certain level of protection.
However, availability of these and even standard factor concentrates that are safe and
sustainable vary across Europe. Organisations like the European Haemophilia Consortium
(EHC), based in Brussels – the hub of the European Union - exist to fight the health
inequality amongst approximately 90,000 people 1 diagnosed with a rare bleeding condition
that they represent.
A snapshot of the disparities in access to treatment and health services across Europe is
illustrated in the EHC’s documentary, Haemophilia Stories 2 . It is a really eye-opening and
emotional account, and validates why having a voice is not a choice to some, but a
necessity. I urge you to watch it.
In October, the EHC are hosting their annual conference in Vilnus, the capital of Lithuania in
Eastern Europe. This is an important forum for patients, healthcare professionals, the
scientific community and the pharmaceutical industry to share expertise and address unmet
needs. I understand that Lithuania itself, since first hosting the conference in 2009, now
provides prophylactic treatment to children and adults 3 . I have the privilege to be speaking
as part of a company symposium about the patient journey and overcoming the remaining
milestones in haemophilia care from a patient perspective.
Meetings like that of the EHC do also give a platform to showcase the latest flurry of
research and innovation in haemophilia, with the hope that new therapeutic options could
dramatically change the treatment landscape on a large scale. In fact, it was only last year at
the same conference in Norway that I first heard, ‘cure of haemophilia by gene transfer’
becoming a reality – something certainly my grandad would never have dreamt possible!
The quest to meet patients’ expectations for the future is somewhat evident from the
ongoing clinical trials that I know of in the UK, especially for my friends and peers with
inhibitors. Although I know personally that progress can come at a price, such poignant
losses must not be in vain; we have come this far, let us not stop now!
1 European Haemophilia Consortium. 2013. Mission and Objectives. [ONLINE] Available at:
https://www.ehc.eu/about-ehc/mission- and-objectives/#. [Accessed 11 September 2017]
2 Haemophilia documentary project. 2016. Haemophilia Stories. [ONLINE] Available at:
http://haemophiliastories.eu/. [Accessed 11 September 2017]
3 European Haemophilia Consortium. 2017. EHC Conference. [ONLINE] Available at:
http://www.ehcconference.org/2016-01- 09-15- 17-45/welcome- 5.html. [Accessed 11 September 2017]