Dear adults with PKU (16 years plus) and parents/caregivers of adults with PKU (16 years plus),    

We would like to invite you to complete a short (approximately 10 minutes) survey to ascertain if there is a need for new treatment options for PKU in Ireland. Responses to this survey will be used by the PKU Association of Ireland (PKUAI) for a submission to the National Centre for Pharmacoeconomics (NCPE) in support of a recent application by Biomarin to the NCPE, requesting reimbursement of Palynziq for citizens of the Republic of Ireland (16 years plus) with PKU.    

Palynziq is a new enzyme replacement therapy for adults with PKU (16 years plus) and is also known as Pegvaliase or PEG-PAL.

Hopefully this is the first step to reimbursement for Palynziq for citizens of the Republic of Ireland with PKU, aged 16 years or over, who have inadequate blood phenylalanine (Phe) control (blood Phe levels greater than 600 micromol/L) despite prior management with available treatment options.

Please make sure to respond by 06/12/2020 as there is a strict timeline for submitting our input, and this may well be critical to the NCPE’s Health Technology Assessment (HTA) of Palynziq.

Don’t forget to pass this survey on to all Irish adults (16 years plus) with PKU and/or their parents/caregivers.

Thank you in advance for your time.