Kuvan for PKU patients
Deputy Healy-Rae raised the case of a Kerry family and the rejection of Kuvan funding for phenylketonuria, with Deputy MacSharry arguing Ireland's system fails rare-disease drugs. The Taoiseach said reimbursement decisions are made by the NCPE and HSE on objective clinical and economic grounds, not by Government.
My question relates to the programme for Government. As the Taoiseach knows, phenylketonuria is where sufferers carry a gene that inhibits them from breaking down the protein. The inability to break down the protein and the resulting build-up of it in the brain to toxic levels can lead to brain damage, seizures and behavioural problems. Kuvan is the only treatment available for PKU, but it was recently rejected as a candidate for funding by the National Centre for Pharmacoeconomics. In The Kerryman today, I read about the Quilter family from Lixnaw, County Kerry. The daughter, Maeve, who is only 11 years of age, is a PKU sufferer. This drug would help her and many more. What does the Taoiseach propose to do to help those who are suffering from this rare disorder, especially the young children?
Comment on this
The programme for Government refers to breakthrough treatments, the availability of new drugs and their cost. The previous Deputy mentioned that Kuvan is the only treatment for PKU, a disease that is prevalent in Ireland. Ireland has the highest incidence in the world at one in 4,500. Many countries throughout Europe have already approved this drug for supply. We have not done it here because it has failed the NCPE test on two occasions, in 2009 and 2017. This is largely because for orphan drugs and rare diseases our system is in effect, or would appear to be, designed to fail and such drugs have no chance.
Will the Government approve the provision of Kuvan for sufferers of PKU? Will the Government immediately reform the approach taken by the State to rare diseases? Will the Government amend the current assessment processes? They would appear to be designed to fail and do not take adequate cognisance of the benefits of these important treatments for patients.
Comment on this
The Government does not decide which medicines are reimbursed and which ones are not. There is a process in place to do that involving the National Centre for Pharmacoeconomics and the HSE. The decisions are based on objective criteria. To date, there have been two applications for reimbursement. It was determined by the medical, clinical and economic experts that this particular medicine was not sufficiently effective at the cost proposed.