A Fair Deal for People with PKU in Scotland

BioMarin's plan to submit a proposal to the Scottish Medicine Consortium (SMC) re Kuvan is likely to be submitted in February 2018. The groups that are likely to be included are described below.

There is no guarantee that this policy will be successful - the SMC evaluates the drug against strict criteria. The NSPKU has the opportunity to explain to the SMC how difficult PKU can be to live with. We will use data from the ongoing NSPKU survey to give the patient’s perspective of PKU to the SMC.

The proposal which will be submitted to the Scottish Medicines Consortium...

Kuvan will be reimbursed for responsive patients for the following groups: -

Children 4 and under with blood phenylalanine levels outside European guidelines more than 50% of tests in a three month period.

Children aged 5 to 18 and under with blood phenylalanine levels outside European guidelines more than 50% of tests in a 6 month period.

Children aged 18 and under who have a co-morbidity (ie other health condition) or other clinical difference which makes dietary adherence significantly more burdensome than the general patient cohort. Examples of such factors would be diabetes, autism, cancer and psychological disorders.

Adults with blood phenylalanine levels outside European guidelines with symptoms or co-morbidities (ie other health conditions).

Women in the period in which they are actively planning a pregnancy and during their pregnancy.

NSPKU’s position and the campaign for Kuvan in Scotland...

The NSPKU is disappointed that the proposal does not include all people who might benefit from Kuvan treatment. BioMarin have informed us that they have designed the submission to maximise the chances that it will be accepted under the SMC’s current rules. The NSPKU actively campaigns for all people with PKU to have access to proven therapies that might help them live with their condition.  

We wish to ensure that we fight as hard as we can for people with PKU to get a fair deal in Scotland. The NSPKU has written a campaign document for Scotland. Please send this to your MPs in Westminster and to your Scottish Members of Parliament. A suggested template letter is also available - but we find that politicians are more likely to help if they hear your own personal story of living with PKU. You can send this by email – there is no need to post it.

If you need help or support in contacting your MP or MSP or if you would like a copy of the template letter, please call 030 3040 1090 or email info@nspku.org

Please also let us know if you have been in touch with your MP or MSP - so that we can follow up with them.