Battling The System At Every Turn
The number of people affected by this campaign is not small.
It is estimated that over 3.5 million people will be affected by uncommon diseases.
Of these many will have significant unmet clinical need. And yet the process of drug development is slow, cumbersome and extraordinarily expensive, hampered by what we believe are regulations that are well overdue for review.
Our recent parliamentary launch of the Halpin Protocol proved an excellent opportunity to bring together the key people who can really make a difference to the drug approval process.
I am hugely grateful to those who attended, particularly Alan Thomas (who suffers from Ataxia) who had a 10-hour round trip just to be with us!
The protocol is a blueprint which I hope will result in changes to the way that drugs for people with serious and life-threatening conditions are developed and prescribed.
I am realistic enough to know that any change to the current regulatory environment in this area will likely be too late to have any significant impact on my condition.
But this isn't about me; there are 6,000 recognised rare diseases and only 250 currently have treatments. Many patients and families find themselves having to battle the system at every turn just to try and secure the drugs they so desperately need.
The Government has already taken some important steps, as seen in their Life Sciences Strategy, but there is still more that could be done in the short and medium term.
In the long term our campaign is looking to amend the law on strict liability, as introduced following the 1987 Consumer Protection Act. A law that we believe significantly holds back pharmaceutical companies, and therefore patients, in the pursuit of new medicines.
That is why I have set up this campaign. Too often I have heard other patient groups calling for faster access to medicines, and I now realise in many respects we are singing from the same hymn sheet.
That is why I am seeking to bring these voices together and pursue tangible outcomes. For many of us time is of the essence.
The original article appeared on the Sky News website