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NHS England: Allow My Daughter Access to Eculizumab, the Lifesaving Medicine You Allow Others

30/06/2016 15:56

In May 2013 my family metaphorically car crashed into a world we knew little about. We were suddenly treading water manically to keep our heads, and members of our family firmly above the surface. Cases had been packed for a family holiday and in 24hrs we should have been boarding a plane. But we were going nowhere.

Previously healthy, active and academically excelling our 10-year-old daughter was fighting to keep her kidneys working. The hospital team worked hard to save Abi's kidneys. At one point a 'wonder drug' with a £250k price tag was talked of, but her condition deteriorated rapidly within weeks and her kidneys failed leaving her dependant on dialysis.

Five weeks later she was just one of two patients in a million to walk away with an incurable rare condition called Dense Deposit Disease. A disease triggered by just two throat infections two months earlier.

So many hurdles lay ahead: dietary restrictions, liquid restrictions, hair loss, rebuilding of her immune system, muscle wastage, mobility issues, fear of needles, so many tablets, a year of constant vomiting dropping her weight to a dangerous level, repeat trips to theatre to support the transition from haemodialysis to peritoneal...the list goes on.

However, this isn't though a bid for sympathy.

It's a time we look back and recognise and admire the journey of this young renal patient whose strength, selflessness and determination finally got Abi to the point of being ready for a kidney transplant one year later.

It was then that the 'wonder drug' made a repeat appearance. Eculizumab, the only treatment available to protect her transplanted kidney from the disease attacking again. Eculizumab, now classified as an 'Ultra Orphan' drug, which holds an indefinite patent.

Two applications were submitted to NHS England, both rejected on the grounds of no clinical evidence. Abi was caught in a 'catch 22', too rare to have a policy in place to dictate a treatment plan but not rare enough to be classed as exceptional to access this lifesaving medicine. Frustratingly for her consultant, this drug was sat on the shelf of our hospital and administered to other children and patients but he had no means of reaching it for Abi.

And so we started our petition on Change.org, and 15 months of campaigning NHS England and fundraising to prove the drug works.

Abi currently has continued on dialysis and supported by 1,095 dialysis sessions. Over 13,000 hours of childhood and 780 days of education have been lost to three years of living with a life threatening disease.

We have worked around the clock to educate ourselves to make sense of the facts. Abi, bravely sharing her journey publicly, only driven by helping others and never for her own benefit. We weathered frustration to see the wrong policy produced, controlled outrage to be told that no evidence existed that the drug works and then the bittersweet news that Scottish patients were now successfully applying for this medicine with Abi's original case evidence

Miraculously, we now have a NHS England policy which illustrates the medicine IS clinically effective and they WILL commission Eculizumab for post-transplant patients with reoccurrence.

However, NHS England Still has to agree to fund this life saving medicine yet today sits on the shelves of hospital trusts throughout England.

Yesterday, today and tomorrow NHS England provide Eculizumab to children and adults of 2 other rare kidney diseases for exactly the same purpose that Abi and other patients like her need this treatment.

So we're asking the Kidney APPG to support Abi and to ask how the priceless strategy 'Taking Organ Transplantation to 2020' can really meet expectations of our Ministers for Health, our Secretary of State for Health and most importantly the 7,000 patients who wait for the priceless gift of life if patients such as Abi cannot access medicine which was used yesterday, today and tomorrow for other patients with identical disease characteristics to keep their transplants working.

A year ago, Abi wrote to David Cameron. 'This Morning' invited Abi on the program when she unexpectedly received a reply from the Prime Minister committing to help. We have no doubt David Cameron's powerful direction to NHS England was the catalyst of so much progress.

Labelled the 'Poster Girl of Rare Disease' by Phillip Schofield on This Morning, despite her young age she's already driven significant change and supported by a 205,000 strong petition.

Abi's picture was then displayed alongside David Cameron in a conference in Uppsala in Sweden to demonstrate the plight of patients with rare kidney diseases and the battle they face in accessing treatment. 82 scientists from across the world were so move they pledged to centralise intelligence regarding research, trials, treatment and cures to support clinicians in their applications for treatment for their patients.

This isn't a battle to illustrate political preference, nor a campaign to humiliate an institution with which we all entrust with our life. Our point is very simple, this medicine is proven to work and is purchased by NHS England today to treat other English patients so why not Abi?

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