Update on the NHS appraisal processes for Kuvan across the UK


Appraisal by NICE for England and Wales

In October 2018 NICE started an appraisal of Kuvan, which aimed to consider whether it should be used for adults and children with PKU in England and Wales.

NICE has two different methods for appraising medicines, the Highly Specialised Technology Appraisal (HST – for rare diseases) and Technology Appraisal route (TA - the standard route designed for common diseases). NSPKU were concerned that NICE decided to appraise Kuvan in the standard TA route which does not have the methodology or costs thresholds which are appropriate for rare disease treatments. In simple terms, the use of the inappropriate TA route decreases the chance that there would be a positive outcome for this appraisal.

In December 2018, NSPKU members Natasha and John Cotter instructed the solicitor Peter Todd of Hodge Jones and Allen to challenge NICE’s decision to use the standard TA route for Kuvan. NSPKU is pleased to announce that NICE have now decided to reconsider their decision without the need for litigation. We hope NICE will accept, on a closer examination of the criteria, that Kuvan should be appraised within the HST system which is designed for rare disease treatments. NSPKU wishes to place on record the gratitude of the Cotter family for the skilled and considerate approach of Peter Todd and his colleagues at Hodge Jones and Allen.

NSPKU are now working with NICE to progress the appraisal again. We are committed to ensure that the process is fair, uses an appropriate method and moves forward as quickly as possible.

Interim policy by NHS England (England only)

In December 2018, NHS England published a draft interim policy for Kuvan but did not fund the treatment for costs reasons. It was accepted that the treatment was safe and effective and that there was sufficient evidence to support its use.

NHS England has now confirmed that the policy will be re-entered into the May budget prioritisation round, with a decision likely to be published in June 2019. NSPKU is contributing to the process.

Appraisal by the Scottish Medicines Consortium

At the recent meeting of the APPG for PKU in Parliament, BioMarin was asked to clarify its plans for another appraisal for Kuvan in Scotland.

BioMarin confirmed that it would be communicating with the Scottish clinicians and intended to publish further evidence later this year. They committed to making a further submission as soon as possible thereafter. NSPKU will continue pressing BioMarin to make another submission for Scotland. 

28th January 2019