AQW 1660/22-27
Ms Paula Bradshaw
Alliance Party
South Belfast
Tabled Date: 16/06/2022
Answered On Date: 28/06/2022
Priority Written: No
Question:
To ask the Minister of Health, pursuant to AQW 818/22-27, why no eligible patients have yet had access to Kuvan, even on a trial basis.
Answer:
Sapropterin (Kuvan) for treating hyperphenylalaninaemia in phenylketonuria was recommended for use by NICE through TA729 on 22 September 2021 and has been available as commissioned therapy since that date. The Health and Social Care Board (HSCB) met with clinical and management team in the Belfast Trust on 27 September 2021 to discuss the arrangements for implementation. At this time Trust colleagues set out some of the challenges with the delivery of this therapy associated primarily with a complex patient assessment pathway involving intensive monitoring arrangements and testing – all of which needs to be completed to ensure the treatment is appropriate. It was understood that as a result of this, implementation would take considerably longer and would require significant investment in staffing infrastructure to fully deliver the service. Opportunities to reduce the timeline for progress were explored including additional resourcing or developing links with other tertiary GB centres to provide support were considered. The Trust however advised that the timeline for implementation was broadly similar to other GB services and had no concerns that Northern Ireland was in any way different from other units taking forward the sapropterin pathway. The HSCB explicitly confirmed at that time that as a NICE TA recommended therapy, implementation would be fully supported by the commissioner. There is therefore no impediment to progress in relation to resourcing from a commissioning perspective. The Trust was required to submit a business case to inform the level of resourcing required for the staffing components, which are in addition to the drug costs. This was received by the Department’s Strategic Performance and Performance Group (SPPG) on 6 June 2022 and an allocation for the full costs as requested was issued on 23 June 2022. The SPPG (previously HSCB) has received five individual requests for the funding of sapropterin (Kuvan) for the treatment of hyperphenylalaninaemia in phenylketonuria between October 2020 and May 2022. All five requests were approved for funding via the Individual Funding Request (IFR) process or Cost per Case (CPC) process. However it should be noted that the SPPG does not hold information on whether the individuals subsequently received treatment. The treatment continues to be made available on a cost-per-case basis should a requesting clinician make application to the SPPG demonstrating that the relevant criteria have been met.
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