Dear Mrs May, Please help me live a longer and hap­pier life. Alex, aged 11


Manchester Evening News - - FRONT PAGE - By CHAR­LOTTE DOB­SON char­lotte.dob­son@trin­i­tymir­ @dob­sonMEN

A LIT­TLE girl with cys­tic fi­bro­sis has writ­ten to the prime min­is­ter ask­ing for a life-length­en­ing drug to be pre­scribed on the NHS.

Alex Darkin, 11, was di­ag­nosed with the de­bil­i­tat­ing con­di­tion at birth and knows it will shorten her life. The young­ster, from Prest­wich, has now writ­ten to Theresa May urg­ing her to sup­port a campaign to al­low peo­ple with cys­tic fi­bro­sis ac­cess to new medicines. One of the drugs is called Orkambi, a medicine that nearly half of peo­ple with cys­tic fi­bro­sis could take, in­clud­ing chil­dren aged six and over.

In her let­ter, Alex wrote: “My name is Alex, I’m 11 years old. I have a con­di­tion called cys­tic fi­bro­sis. It’s very an­noy­ing and bor­ing. Cys­tic fi­bro­sis means I’ve got to go into hos­pi­tal a lot and have a lot of med­i­ca­tion. There’s a drug called Orkambi. If I had it I wouldn’t have to go to hos­pi­tal as of­ten and I would live a longer and hap­pier life. It makes me sad when I have to go to hos­pi­tal be­cause I hate nee­dles and I miss my fam­ily and friends.”

Alex’s brother Har­ri­son, eight, has also writ­ten a let­ter about her ill­ness. “I miss Alex and my mummy when they have to go to hos­pi­tal, it makes me feel sad,” he wrote.

The sib­lings’ let­ters have now been handed in at 10 Down­ing Street. Orkambi can­not cure the con­di­tion, but it has been found to slow de­cline in lung func­tion – the most com­mon cause of death for peo­ple with cys­tic fi­bro­sis.

It has also been shown to sig­nif­i­cantly re­duce chest in­fec­tions re­quir­ing hos­pi­tal treat­ment.

Orkambi was recog­nised by the Na­tional In­sti­tute of Health and Care Ex­cel­lence (NICE) as an im­por­tant treat­ment in 2016.

How­ever it is not avail­able to the ma­jor­ity of cys­tic fi­bro­sis pa­tients be­cause the drug is not con­sid­ered cost-ef­fec­tive by NICE.

David Rams­den, chief ex­ec­u­tive of the Cys­tic Fi­bro­sis Trust, said: “Cys­tic fi­bro­sis is a ter­ri­ble con­di­tion. Sig­nif­i­cant ad­vances in med­i­cal science mean new pre­ci­sion medicines are avail­able or in de­vel­op­ment that have the po­ten­tial to trans­form the lives of peo­ple with cys­tic fi­bro­sis and we urge the prime min­is­ter to en­sure that they are avail­able for the thou­sands of chil­dren and adults who have been wait­ing far too long.”

A NICE spokesman added: “NICE is­sued guid­ance in July 2016 which did not rec­om­mend Orkambi to treat cys­tic fi­bro­sis. The de­ci­sion was based on the ev­i­dence and price pro­posal pro­vided by Ver­tex at the time of the ap­praisal. A con­sid­er­a­tion of a review of the guid­ance can be brought for­ward where there is new ev­i­dence that is likely to im­pact on the cur­rent recommendations. We re­main open to a fur­ther ap­proach from the com­pany, for ex­am­ple, with new data or a re­vised price.”

Alex Darkin with her let­ter to Theresa May, left

Alex Darkin and her let­ter she sent to Theresa May, in­set

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