Question reference: S6W-05334
- Asked by: Monica Lennon, MSP for Central Scotland, Scottish Labour
- Date lodged: 22 December 2021
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Current status: Answered by Humza Yousaf on 10 January 2022
Question
To ask the Scottish Government what discussions it has had regarding the drug, sapropterin, being made available to patients in Scotland with phenylketonuria (PKU).
Answer
The Scottish Government notes the updated National Institute for Health and Care Excellence (NICE) guidance on sapropterin published September 2021 following a resubmission by its manufacturer Biomarin. The Scottish Medicines Consortium (SMC) previously considered sapropterin (Kuvan) in August 2018 and the medicine was not recommended for use in NHSScotland because the submitting company’s justification of the treatment’s cost in relation to its health benefits was not sufficient and in addition the company did not present a sufficiently robust clinical and economic analysis to gain acceptance by SMC. The submitting company (BioMarin) has not been in contact with SMC regarding a resubmission for this medicine.
The first generic version of sapropterin has received a Marketing Authorisation from the Medicines and Healthcare products Regulatory Agency (MHRA). To support ensuring best value for NHSScotland, NSS National Procurement will shortly be tendering for the future supply of sapropterin to NHS Scotland.
Given the launch of the first generic, we are currently considering how best to provide advice to Health Boards on whether sapropterin should be made available for routine use in NHSScotland, based on the latest available evidence.