Family hit by spiralling cost of new life-saving drugs; MUCH CHEAPER TABLETS MAY BE FORCED OFF THE SHELVES.
A MOTHER has spoken of her anger after the supply of her sons' life-saving medicine became jeopardised by a legal loophole.
Kerry Webster fears her sons James and Adam will no longer be able to take a cheap but effective medicine after US drugs company, BioMarin, used European Union laws to produce a new drug that costs up to 70 times more.
BioMarin has produced a medicine similar to the one the two boys - who suffer from Congenital Myasthenic Syndrome - have taken for up to 10 years.
Under the EU law, which was intended to increase research and investment into treatments for rare diseases, the old unlicensed medicine 3,4DAP can no longer be produced.
The Webster family are now living from month to month, worried supplies of 3,4DAP, which costs up to pounds 1,000 person a year, will dry up.
The BioMarin medicine FirDapse - currently licensed for use in patients with Lambert-Eaton Myasthenic Syndrome - costs between pounds 40,000 and pounds 70,000 per patient a year, according to the Muscular Dystrophy Campaign.
Mrs Webster, who lives in Cardiff, said: "Both James and Adam are reliant on this medication, without it there would be life-threatening consequences.
"James wouldn't be in mainstream school without this drug - it's crucial for him, his life experiences and his life.
"This is something they will need for the rest of their lives."
The 41-year-old added: "At the moment we have managed to get an alternative supply of 3,4DAP every month, but we don't know how long that supply will remain open.
"It worries me that even if the Assembly Government said it would spend all that money on the drug [FirDapse] I'm not convinced by it.
"And I don't want the boys to have to go back to hospital for another clinical trial when there's a perfectly good, cheap drug available."
A group of 21 consultants last week wrote an open letter to Prime Minister David Cameron and English Health Secretary Andrew Lansley, about the unintended consequences of the EU legislation.
It said: "As the rules are currently enacted, many drug companies merely address their efforts to licensing drugs that are already available rather than developing new treatments. "Once a company has obtained a licence, the legislation then gives the company sole rights to supply the drug.
"This in turn allows the company to set an exorbitant price for this supply and effectively to bar previous suppliers of the unlicensed preparation from further production.
"We believe this behaviour is not in the best interests of patients or the NHS but is undoubtedly significantly advantageous to drug companies." Dave Anderson MP, chair of the all-party Parliamentary group for muscle disease, has said he will raise the issue with health ministers.
And Dr Marita Pohlschmidt, director of research at the Muscular Dystrophy Campaign, said: "We support the aims of EU legislation on drugs for rare disease because it encourages much-needed investment from biotech companies into new and improved treatments.
"However, it is vital the legislation also ensures that the costs of these treatments are not so high that they are denied to patients on the grounds that they are unaffordable to the NHS."
Dr Adrian Quartel, medical director of BioMarin, said: "There are no licensed treatments for CMS although a number of different medications are used. BioMarin will try and do what we can to help doctors access FirDapse amifampridine for their patients, should the prescribing doctor consider this appropriate treatment.
"We really do care for the patients."
The unlicensed tablets which may now be withdrawn Kerry Webster and sons James, 14, and Adam, 10, of Cyncoed, Cardiff, who both have a rare muscle disease PICTURE: Peter Bolter [umlaut]
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|Publication:||Western Mail (Cardiff, Wales)|
|Date:||Nov 23, 2010|
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