In the media

A BBC news interview with Ruth Bell, a PNH patient, and her thoughts about the trial and the decision by the Department of Health here:

<a href=”http://news.bbc.co.uk/1/hi/health/7609962.stm”>http://news.bbc.co.uk/1/hi/health/7609962.stm</a>

Thank you

This week, we were informed that the Rt Hon Dawn Primarolo, Minister of State for Health approved funding for:

- a nationally commissioned service for PNH patients –at King’s College Hospital and Leeds Infirmary

- funding of Eculizumab for those patients who clinically need it.

The funding will begin from April 2009.

We would like to thank all our friends who supported the PNH patient group and helped any way they could. All the people out there who came by to read this blog, signed our petition, wrote to their MPs and the Department of Health, spread the word and let us know we weren’t alone. All the doctors and nurses who have taken care of us and kept us going. All the people whose research resulted in this drug. The NCGs and our government who made this difficult decision.

We get to keep our lives. It’s very hard to express into words what the decision and the enormous amount of support we received means to me. All I can do is thank you from the bottom of my heart.

My Life with PNH: backstory, present story, future story.

If you would like to help us, please go to the bottom of this post for details on how to write to Health Minister, Dawn Primarolo and details of our online petition.

I am 32 years old. I am a daughter and a sister; I am a friend and a colleague; I am a volunteer, a hobby pianist and a scifi fan; I am short; I am good with pets and small children; I am a council and income tax payer; I am a Londoner through and through.

I am also someone who has a condition called Paroxysmal Nocturnal Haemoglobinuria (PNH). PNH is a rare blood condition that affects one in about a million. I am one of eight people in London & the South East on an experimental drug called Soliris, which was developed to treat the most severe symptoms of this condition. I am one of thirteen people in London and the South East who has symptoms so severe that we need to be on this drug in order to live a “normal” life. Walking up a flight of stairs without having to rest, working full time to make a living, not feeling fatigued all the time, not having to have blood transfusions incessantly to live — these are all luxuries that I have been afforded after I received Soliris during an experimental medical trial.

My government are about to decide whether it is cost effective to give this drug to people like me. The decision is being taken by the Health Minister, the Right Hon. Dawn Primarolo shortly. We are very few — there are 13 of us in London and the South East and a further 20-odd patients in a trial site in Leeds — and we need your help. Yes, this is yet another tired human interest story that shouldn’t exist in the welfare state and like others, we need your interest because our own might not be enough.

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BACKSTORY

Paroxysmal Nocturnal Haemoglobinuria (PNH) is a rare blood condition that affects one in about a million. It’s a haematopoietic stem cell disorder and an acquired genetic disorder that causes haemolytic anaemia. Acquired as in I wasn’t born with it but I can’t catch it or give it to someone else; genetic in that it works on a gene level. My bone marrow grows mutant blood cells and this effects my red blood cells the most. My immune system attacks these mutant cells because they don’t recognise them as my own cells. As a result, I have a low blood count and an abnormally low red blood cell count; there isn’t enough oxygen in my system.

There is still very little known about the disease. They don’t know why one in a million people spontaneously develop the condition, they don’t know how to stop it, they don’t know what triggers the haemolytic episodes, they don’t know how to cure it and, until four years ago, there wasn’t even a uniform way to manage the symptoms. The median life expectancy after diagnosis is estimated to be 10 years.

Patients don’t manifest the same symptoms, patients do not follow a set path of getting worse/better and different patients react to the same meds in wildly different ways.

What they do know is the only way to remove PNH is through bone marrow transplant; a rarely taken alternative for patients. The survival rate for pnh bone marrow transplants has improved in the last ten years. It used to be 40% and now it’s about 60%. PNH sometimes reoccurs after bone marrow transplant.

As it is hard to generalise because everyone has such different experiences, I’ll tell you about myself. I was twenty three when I was diagnosed.  It was discovered by sheer dumb luck. Almost nine years ago, I moved in the spring and while changing GPs, I mentioned to the doctor that I hadn’t been able to shake off the cold I’d caught the previous winter and was tired. Ten minutes later, I was having a routine blood test and that afternoon, the doctor called me to tell me that I needed to go to A&E. I stayed there for the next ten days while the doctors tried to find out what was wrong with me. My haemaglobin was 4.1, the normal count is between 12-16 grammes per litre. Later, the doctors told me that they had never seen anyone walking vertically with that kind of HGB, let alone live a normal life.

I was ill for the next four years of my life.

My symptoms were on the severe end of the scale. I was perpetually anaemic, jaundiced and chronically fatigued; I couldn’t study full time, I couldn’t work full time; I had very regular washed red cell blood transfusions — at one point, I was being transfused with three units of blood every week and at the furthest apart, one transfusion every six weeks — I had extreme fatigue from minimal activity, I was perpetually jaundiced, I had cannula and needle marks and bruises all over my arms from constant blood tests and infusions, I had palpitations, I had kidney and liver problems and I was at high risk for blood clots.

I couldn’t walk up a flight of stairs without becoming short of breath and dizzy. Sometimes, I had trouble walking down the road and there were a lot of days when all I did was lie in bed. Carrying two grocery bags was a luxury that I couldn’t afford. I played in netball and hockey and lacrosse teams until I was 18, I had travelled extensively in my early twenties. I was about to graduate Graduate school, I was ready for my adult life, I had never been and wasn’t prepared to be a delicate flower.

They started a search for a bone marrow donor after the first six months because they felt my body couldn’t hold up very much longer. I don’t have a genetically matched donor, I am unlucky in that respect. I do live in a country that has never asked me to pay for eight years of intensive health care I have received from the very best of professionals, I am incredibly lucky in that respect.

Then, in mid 2003, my symptoms eased to the point where I was being transfused every 3-4 weeks and it wasn’t as hard as it had been before. I got on all right on a haemoglobin of 7-8 and only got transfusions when I dropped below 6 grams, I wasn’t ever going to back to my life before diagnosis but I was living a very slow and small life by then.

This changed in 2004 when I was invited by Dr. Dupe Elebute, then of St. George’s Hospital, to join a Phase III medical trial for a drug called Eculizumab. It was the first drug of its kind, an intravenous drug that we are required to receive every two weeks, to manage the most severe symptoms of PNH — haemolysis. The trial sites were in Australia and North America as well as in Europe. The UK was one of the leading research centres. Patients with extremely severe symptoms were asked to join the trial; to qualify, one had to have received more than four blood transfusions in one calendar year. Eight people joined the trial at  St. George’s Hospital, the trial site in London.

As I understand it, the drug doesn’t stop the production of mutant cells but it disables my immune system a bit and blocks complement so my immune system wouldn’t attack my mutant blood cells. (Attack of the Mutant Blood Cells!!! I should be a movie.) The risks are, well, they’re disabling part of my immune system. Someone on the trial fell seriously ill after catching a rare strain of meningitis. It’s the first drug of it’s kind; no one can anticipate what will happen to us if we are on the drug long term.

After three years of testing, the drug was proven a success. There are risks but the risks, such as increased risk for meningitis, are outweighed by the benefits. Eculizumab, which is now known by its retail name Soliris has stopped us from losing our own blood and it allowed us to be relatively healthy and active. Our quality of life was dramatically improved. I am 18 months transfusion free. I will never go back to the active life I had before, my blood count is still lower than normal but I have a full time job. I have energy. I can walk with a shopping bag full of groceries. I am no longer perpetually jaundiced, the doctors are no longer worried about iron build up or finding a bone marrow donor.

I have the infusion every two weeks, it takes about an hour and a half, start to finish. And it has changed my life. Or rather, it has changed it back to a facsimile of how it had been before I got sick.

I still go to hospital nearly every week, I still have to attend three separate clinics, I still have to take a big handful of meds, I still have perpetual bruises down my arms and on the back of my hands because of the cannulas and the blood drawing but the drug? It works.

For some, it doesn’t work at all; for others, it works perfectly and they are transfusion free. I’m one of the minority that the drug hasn’t completely removed the dependence on transfusions since we have been receiving it. I still need occasional transfusions but these are few and far between and I recover much quicker.

Our quality of life was dramatically improved. I am 18 months transfusion free. I will never go back to the active life I had before, my blood count is still lower than normal but I have a full time job and a social life. I have energy. I can walk with a shopping bag full of groceries. I am no longer perpetually jaundiced, the doctors are no longer worried about liver or heart transplants or finding a bone marrow donor.

I’ll never forget that first time I did that even without thinking. It wasn’t a grand, revelatory moment in my life, with swelling background music and sparkles. It was, simply, an everyday moment; I was simply free not to worry about running for the bus.

Whatever happens, I will always be grateful to Alexion for the last two years. I will be grateful to the NHS for supporting the trial and making sure I didn’t have to pay the costs the patients in the US did to participate in the trial.

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PRESENT STORY

If this was the end of the story, this would be the place for happy ever afters. But this isn’t a story, this is life and things are never quite as easy as we hope. In our case, as with many similar drug issues, we are having trouble with NHS funding.

Soliris was approved last year but the NHS has yet to fund the drug. The American pharmaceutical company, Alexion, which developed the drug, is charging the NHS a hefty £250,000 per annum, per patient for this first-of-its-kind drug. The seven other patients and I are currently receiving the drug due to a grace period by the pharmaceutical company. We have no idea when this grace period ends. Other countries that were trial sites, including Canada, Germany and Australia, are funding this drug for patients. Countries that were not trial sites, such as Greece and Turkey, have approved funding for people who have PNH and need the drug.

The United Kingdom is the only country that has licensed but not provided funding for Soliris to patients.

This drug was developed with the help of leading UK research. Applications for funding have been made to PCTs but because of the high cost of the drug, the trial site consultants who applied on behalf of patients have been, in some cases, refused or their applications delayed. Last autumn, an application for national  funding was made by King’s College Hospital, where our treatment has since been moved, and Leeds University. In December, the National Commissioning Group decided to approve funding for two specialist PNH centres in London and Leeds but deferred the decision about funding to a higher body, the National Commissioning Specialist Group.

However, a few weeks ago, the National Commissioning Specialist Groups reversed the decision taken by the NCG and turned down the application for two specialist PNH centres in London and Leeds as well as the funding for the drug. The NCSGs, in turn, have handed over the decision making to Health Minister, Dawn Primarolo. Our consultant has been told that Ms. Primarolo is set to make a very quick decision, perhaps in the next two or three weeks, while parliament is out of session. We can not even petition the Prime Minister online; his online petition services are on holiday while he is.

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FUTURE STORY

If national funding is not approved, it will go back to the PCTs and the postcode lottery — one that I’m doubtful, with the price tag being £250,000 per patient, per year, for at least the next six years, many will win. If we do not receive Soliris, we have been told that we will revert back to life before Soliris in two weeks. As well as the eight whose symptoms were so severe that we qualified for the trial, there are five others in London & the South East who now desperately need to be on the drug but aren’t because they have been refused funding from their PCTs.

Two patients in the north are being funded regionally, other patients have been refused. If the government does not provide funding centrally, it will be a case of the postcode lottery, something this government not only a month ago was promising us was something of the past. For original trial patients, we are in state of limbo. We have not been informed when the grace period from Alexion will end and we do not know what will happen after it ends. I do not think I can adequately describe the feeling of this constant state of suspense, of only knowing for sure that once it is taken away, I will go back to the way I was in two measly weeks.

Not everybody who has PNH needs the drug, just the patients who have the most severe symptoms. And when the ultra-orphan drug patent that allows the high cost runs out in six years, the price of the drug will hopefully come down. We need help from the NHS and our government for the next six years. There are only eight of us, we have a tiny little voice, we have no patient group, this condition is so rare that until the trial started, many of us had not met another patient with PNH before. We have no funding to set up a campaign and we personally do not have the money to privately pay for our own treatment. At this price, none of us do.

Asking for so much to benefit so few people, to benefit *me* — this seems somehow greedy and selfish when there is so much need. And yet, the government has promised us improvement to the NHS, has promised us the end of the postcode lottery for drugs, promised to care for us the best it knows how. And I am selfish; I don’t want to go back, I don’t want to go back to being chronically ill, I don’t want to stop working full time — I love my job, I love the novelty of working full time, I love I can do things and people don’t ask me why I look so awful.

I don’t want to stop living my life simply because the system decides my life isn’t cost effective enough. I don’t want to when there is a drug out there that will let me live my life. I don’t want to have to go back to the life that was because medical decisions are now being made by administrators and politicians instead of doctors.

We ask Health Minister, Dawn Primarolo to:

(a) accept the medical advice of the consultants who were researchers on the trial in their recommendation of Soliris as a treatment management drug for PNH;

(b) accept the results of the trial studies to date, all of which support the vast improvement of quality of life for PNH patients;

(c) expand the provision of Soliris to patients who were not on the original trial to ensure access to humane health care to PNH suffers in the UK.

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If you are reading still this, thank you. We need your help, I need your help, to make our voices and concerns heard. If you would like to help us, please write to the Health Minister, the Right Hon. Dawn Primarolo on our behalf here:

Dawn Primarolo MP
PO Box 1002
Bristol
BS99 1WH

or

Dawn Primarolo MP
House of Commons
London
SW1A 0AA

(email) primarolod@parliament.uk

(tel) 0117 909 0063; 0117 909 0064.

Please also come by and sign our online petition here:

http://www.gopetition.co.uk/online/20877.html