A Letter From The British Government
When we left the UK for treatment in California four years ago Mathilda was, we were told, the youngest (diagnosed) Narcoleptic in the world. We understood she'd been hit hard with the symptoms, we were after all caring for her 24/7 and had gone from a fully functioning family (rooted in Bristol) to virtually house bound in a matter of months. Her quality of life was extremely poor and the impact of her condition on all of us was profound.
Despite repeated hospital admissions, numerous outpatient appointments where we appealed to Mathilda's doctors to provide her with Xyrem, letters to our MP, and conversations with specialist in England, Scotland, and France we hit a wall. The final straw came when Mathilda was assessed by a sleep specialist in Oxford, who contradicted our request for effective treatment and wrote in her notes that there was "absolutely no need to rush into Xyrem." And further that we as parents "should pay close attention to their daughters poor behavior and weight gain." It was at this point that we realized we were on our own.
Long before making the decision to move to the US for treatment, the Professor and I exhausted every possible avenue open to us with the exception of having Mathilda's face spread across British newspapers. Those offers we declined since the reporters I spoke to were interested in the H1N1 issue. None of the journalists would agree to putting our plea for Xyrem in print. It wasn't that the vaccine issue was irrelevant--far from it--but that being denied effective treatment, was to my mind, the most pressing concern.
Over those months it became clear that we could sit it out, wait for treatment in the hope that at some point down the line a bottle of Xyrem would turn up. But when would that happen? When she was 6? 10? When her childhood was over?
Maybe the post-code lottery would have swung in her favor. Maybe not.
In the meantime, Mathilda went on wrestling with hypnogogic hallucinations at night, and fighting off cataplexy and crippling sleepiness in the day. I went on sinking under the weight of it all.
A year later, with Xyrem still out of reach, the Professor was offered a job in SoCal, one that would effectively secure treatment and specialist care for Mathilda at Stanford, the world's leading center for Narcolepsy research. Mali Einen the clinical resource co-ordinator there told me that if we could get Mathilda to Los Angeles Prof. Mignot could be her' "local" doctor. It was a life line.
But upping sticks was a still a huge decision not only because of the risks (we didn't know how or if she was respond to Xyrem) but because it came at the personal expense of leaving our home and families, our lives. We also have two other children who were perfectly healthy and were perfectly happy. There was no way of quantifying or foreseeing the impact our move would have on any of us.
The stakes were very high, we knew that. But as parents we were compelled to provide Mathilda with the chance to lead a near normal life. And I'm happy to say she has responded well to Xyrem, once she was weaned off Modafinil and Gabapentin. It has not been straightforward but she is now well enough to go to school, and have friends, write little stories and whistle in her room.
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Since moving here to Los Angeles in July 2011, we have not returned to the UK as a family. While we have been away my parents (who haven't seen their grandchildren in several years) have petitioned the government to provide Xyrem for all sufferers of Narcolepsy in Great Britain. Last week they received this letter from George Freeman via their MP, Christopher Chope, reproduced here in full:
I re-read it a few times, trying to track with what George Freeman MP for Norfolk and Parliamentary under-Secretary of State for Health, was saying. My dad, as baffled as I was at the inadequacy of the letter, asked me to draft a response. After several angry and unpublishable attempts, this is what I wrote on behalf of my father:
Dear Mr Freeman,
Thank you for your letter concerning treatment for sufferers of Narcolepsy, specifically my granddaughter Mathilda Crisp who four years ago moved to the US to secure the drug in question, Xyrem. If I may, I would like to respond by drawing your attention to several pertinent facts.
The Department of health does not fully understand how distressing Narcolepsy is since it has failed to to recognize the extent to which the symptoms are crippling by denying treatment to suffers in the UK. Treatment in the form of Xyrem manages the plethora of disabling symptoms including excessive daytime sleepiness, cataplexy, and hallucinations. Having effective treatment can be the difference between being disabled and leading what is widely referred to as a near normal life. In other words, it can be life-changing.
Secondly, Xyrem is not especially new. It has in fact been reliably administered for the treatment of Narcolepsy in North America since 2002 when it was approved by the FDA and received expanded indiction for Narcolepsy and Cataplexy by the EU in 2002. According to the American Association of Sleep Medicine, Xyrem is the standard treatment for cataplexy, daytime sleepiness, and disrupted night time sleep and remains the single most effective drug of choice for 80% of Narcoleptics. Whilst Xyrem does not work for everyone, people should be given the opportunity to try it since the range of symptoms specific to Narcolepsy can be severely debilitating. As minister for Life Sciences with responsibility for the Clinical Institute of Care and Excellence (NICE), this begs the question, Why hasn't NICE issued guidelines on the administration and distribution of Xyrem?
You rightly point out that in the absence of NICE protocol, funding decisions should be made by the local commissioning group who are expected to have in place clear and transparent arrangements for local decision making for considering exceptional funding requests. But why is Xyrem considered exceptional by the UK when in other countries (including those with socialized health care) it qualifies as a standard treatment?
In Mathilda's case, an application for Xyrem was made first to the Pediatric neurologist in charge of her care in 2010, then to the ethical committee at the Bristol Children's hospital. As far as we know, it never went further up the chain of command. Perhaps this was in part due to the many layers of bureaucracy you refer to in your letter. After over a year of waiting and being refused treatment, my daughter and son-in-law decided to move their family to the US to ensure the health of my grand-daughter. Within 48 hours of landing, Mathilda was started on Xyrem and is now thriving. However, they should not have had to make that decision.
It is also worth pointing out that in July of this year, The Guardian reported that a young woman in England took her own life because she couldn't live with the symptoms of Narcolepsy. Her Psychosis was brought on by Modafinil (a stimulant) widely used in the treatment of Narcolepsy to maintain wakefulness. Unsurprisingly she was not on Xyrem. If she had been trialed with Xyrem, her depression may have remitted and her symptoms improved--she could still be here today. In her suicide note left to her parents and sister, she said: " you can't imagine my life. I have no quality of life."
During a debate in Parliament on the 25th February 2014, Julian Sturdy, MP presented the case of of a 6 year old untreated narcoleptic boy, and argued that he be rightfully given Xyrem. By way of response, Jeremy Hunt, MP and Secretary of Sate for Health stated that 'Xyrem is not recommended for use in children and adolescents.' This is simply not true. Not only is it recommended by the manufacturer, it is also recommended and prescribed widely by clinicians.
ln your letter you refer to the Accelerated Access Review, an expert advisory group that will ensure rapid access for NHS patients to innovative medicines. This Review, you say, will ensure the UK is a global leader in moving medicines from the lab to the patient as quickly and safely as possible. This is a most welcome development. I would ask that in implementing this Review you consider the thousands of untreated adult and child suffers of Narcolepsy, and weigh the cost of Xyrem against their crippling symptoms, putting their quality of their lives first.
(maternal grandfather to Mathilda Crisp)
No doubt this letter will be read by (at most) one or two bureaucrats in a government office somewhere in London. Most likely it won't make much difference in the grand scheme of things. But at some point, someone has to say something about the inadequacy of the government response to this issue. And as a parent of a Narcoleptic, I have to trust that all those incapacitated by this debilitating condition will get the help they need--hopefully, long before they're too desperate to keep fighting.
With grateful thanks to all those who have signed petitions to the British government on behalf of PWN, to Peter Todd who has offered his legal advice, and to my parents--who will land at LAX in a few weeks and be able to hold their grandchildren.