AQW 4436/22-27

Ms Paula Bradshaw
Alliance Party
South Belfast

Tabled Date: 11/10/2022
Answered On Date: 25/10/2022
Priority Written: No

Question:
To ask the Minister of Health for an update on the availability of drugs against Phenylketonuria in people of all ages, given the relatively high prevalence in Northern Ireland.

Answer:

The treatment for (Phenylketonuria) PKU is a lifelong, very strict low protein diet with amino acid supplements. Many people with PKU only tolerate 4g or 5g of protein per day which is equivalent to the protein found in one slice of bread. Specialist low protein and protein substitute dietary products are required; specified products are supplied by GP prescription through Advisory Committee on Borderline Substance arrangements and can be found at the following link to the Northern Ireland Drug Tariff:

https://hscbusiness.hscni.net/pdf/DT_PART_10a-2210.pdf

Sapropterin (Kuvan®) is the only drug licensed in the UK for treatment of PKU, but is subject to managed entry in Northern Ireland. Managed Entry Decisions | NI Formulary (hscni.net). Sapropterin (Kuvan) for treating hyperphenylalaninaemia in phenylketonuria was recommended for use by NICE through TA 729 on 22 September 2021 and has been available as commissioned therapy since that date

In late September 2021 the HSCB met with clinical and management team in the Belfast Trust to discuss the arrangements for implementation. Trust colleagues highlighted some of the challenges with the delivery of this therapy associated primarily with a complex patient assessment pathway involving intensive monitoring arrangements and testing, and all of which needs to be completed to ensure that treatment is appropriate.

Funding to allow recruitment of significant additional staffing infrastructure investment to enable full implementation of the service, has now been made available to the Trust. The Trust is now actively undertaking the assessment and testing of patients and will provide treatment where appropriate to patients up to the age of 22, in line with the NICE Recommendations.