Radio Leeds invited Katy Brown to ask Ed Balls MP a question last week on Vimizim. Katy’s follow up letter to Mr Balls really highlights the issues that are currently being faced by those affected by Morquio.
Dear Mr Balls,
I felt the need to contact you post our brief conversation about the funding of Vimizim for Morquio sufferers on Radio Leeds this morning.
Firstly, my name is Katy, not Sam as you referred to me throughout. My son is Sam, he is 6, and it is his life at stake.
This morning you treated me as a journalist, a lobbyist, a fool. I am none of those things. I am a mum. You showed neither me nor Sam any empathy. I have a son with a serious, rare, and life threatening genetic disease. Every day in our lives is a battle. I will probably outlive my son. He will lose his mobility. He will probably die of pneumonia. He will need help all his life.
This morning it was not part of my job to ask you a question. It was part of a fight that I should not have to be fighting. Myself and my husband have worked and fought over the past four months to gain awareness of Sam’s situation and the deep injustice that sits behind the limbo we find ourselves in. In a flash this morning in full political bravado you discredited me. You belittled our battle. If you could have said to me that the issues behind this are far too complex for someone like me to understand, that is what you would have said. That certainly was the undertone.
I completely understand the economic and social factors sitting behind your comments. And I understand the economics of the funding of drugs for rare diseases better than you. Today you got your facts wrong in a severely damaging way. You assumed the drug was experimental, you assumed it was unproven, when in fact it is both licensed and extensively trialed. Do you understand that drugs for very rare diseases like Sam’s where there are only 100 or so sufferers are only expensive because there are so few people across whom fixed costs of R&D can be recovered? Is it Sam’s responsibility to solve that tricky commercial conundrum?
Do you think it is right that a country that has hosted a major global clinical trial and been paid handsomely for the kudos should be one of the last in the developed world to agree to fund the drug that it has encouraged its patients to trial? Sam spends a day a week in hospital and he has done for 3 years. We have committed our lives to this …. And most importantly it’s impact on Sam’s life and his physical development has been amazing. Do you think I would put him through all of this if I didn’t think it was working?
A blanket approach through NICE does not work for very rare diseases. The cost will always mean the answer is no. So should the conversation be at diagnosis …. Oh I’m really sorry, seems like you have one of these ultra-rare diseases. Unfortunately because treatment is expensive, your life is sadly less important than other people’s and we simply can’t afford to treat you! I’m not sure that sits well with a socialist ethos, or the principle of universal health care.
We throw money to treat people who make lifestyle choices. Sam has no choice whatsoever yet his treatment is questioned. Where is the justice in that.
Today I talked about process incompetence. In a nutshell. Prior to 2012 – world renowned process for the appraisal of drugs for rare diseases through AGNSS. 2012 – introduction of the Health and Social Care Act, abolishment of AGNSS, establishment of NHS England. No defined process. NHS England spend 18 months and millions of taxpayers money designing new process. Includes a scorecard that has never been consulted upon. After legal challenge in November 2014 by a Morquio sufferer, this is proven to be discriminatory to ultra-rare diseases and is scrapped by Jeremy Hunt. Only because he had no choice. December 15th decision date for Vimizim is dropped and families left in limbo, which is where we still are. David Cameron promises a decision in April but consultation doesn’t finish till the 27th April and purdah makes this impossible. Jeremy Hunt says June. NHS England imply September. And now the provision of Sam’s drug which he is currently receiving compassionately by the drug company will stop in May because they have no confidence in a solution and have tried to engage with NHS England and have been ignored. They have even put a substantial 5 year deal on the table which has had no response. This is entirely unacceptable .
I am sick and tired of being fobbed off. I am sick and tired of no-one taking any accountability for the situation.
Unlike your presumption this morning I am not an overly emotional mother just wanting a solution at all costs. All I want is the truth. An answer. A robust process. No more excuses, half-truths, and smoke and mirrors. This morning you severely damaged my ability to achieve this.
There is no wonder that the general public have so little faith in our politicians and political system.
I will not stop fighting for Sam. He deserves better than this.