Family's campaign over vital drug is raised in Parliament
Leanne and Peter and the twins in 2019
A FAMILY'S battle for access to treatment for phenylketonuria (PKU) has been raised in Parliament.
Leanne and Peter Barnett, from Kingswood, want the drug Kuvan to be made available on the NHS. They say it could help their daughters Olivia and Ruby, who are now three years old, to live more normal lives.
The family say they have been told the drug is not funded because it is too expensive - it could cost £50,000 a year for each girl. The condition means the children cannot process protein properly so have to stick to a strict diet.
Kingswood MP Chris Skidmore raised the family's case in a Westminster Hall debate on rare diseases and the implementation of the UK Rare Diseases Framework.
In the debate, which took place on March 24, Mr Skidmore outlined how, although licensed in 2008 to treat PKU, Kuvan has only been available to patients in the UK in very limited circumstances.
The MP, who is a member of the All-Party Parliamentary Group on PKU, has raised Leanne’s concerns with Ministers at the Department of Health and Social Care and has written to the Health Secretary Matt Hancock to support the campaign to increase access and availability of treatment such as Kuvan for patients and families in the UK.
Mr Skidmore told the debate: “For anyone who is a parent of young children, babies or toddlers, managing any diet is challenging, but managing a diet with low protein is extremely difficult. Everyday life becomes filled with anxiety, putting incredible strain upon the parents, who know that one mistake might cost the child their life or lead to brain damage”.
Chris continued: “This dietary treatment can also be incredibly costly for families, particularly if they are on a low income, which is a real barrier”.
The MP also raised recent concerns about a recent proposal recommending the use of Kuvan for children up to the age of 18 only. He said: “This is a lifelong condition and therefore we need lifelong treatment of Kuvan on the NHS."