Parents’ legal battle to get girls life-saving care
THE parents of two sisters battling a rare genetic disease have launched a legal battle to overturn a decision not to fund life-saving treatment. Nicole Rich, seven, and her younger sister Jessica, three, suffer from a rare neurodegenerative condition known as Batten disease, which robs children of the ability to walk, talk, see and even swallow. Youngsters with the condition who do not receive treatment are unlikely to live beyond the age of 12.
Nicole and Jessica currently receive access to a specialist treatment known as cerliponase alfa for their condition – one on a sibling trial and one as part of a compassionate use programme provided by BioMarin, the company that developed the drug.
The treatment halts the progress of the condition and could help people with Batten disease live for 60 years.
However, advisory body the National Institute for Health and Care Excellence has recommended that the drug will not be available on the NHS as they cannot be certain it would provide value for money.
NICE has yet to publish its final guidelines, but the decision has prompted Nicole and Jessica’s devoted parents – Gail and Matthew, of Throckley, Newcastle – to mount a legal challenge.
They have now applied to the High Court for a judicial review of the decision.
Lawyer Kirsty Stuart said: “This is a tragic case where, at present, cost is deemed to be more important than children’s lives.”
Gail, 41, said: “All we want is to be able to give them the best chance.”
Matthew, 31, a production operative, added: “What makes this even harder to try and come to terms with is the fact that 19 other countries fund the treatment.”