Remit: To appraise the clinical and cost effectiveness of sapropterin within its marketing authorisation for treating phenylketonuria. Following a challenge to the decision to review sapropterin for treating phenylketonuria through the technology appraisal programme, NICE made the exceptional decision to pause the appraisal in January 2019. The appraisal was rescheduled as in development from April 2019 and was recommenced at the 'clarification stage' in August 2019.
Following recommencement of the appraisal and a company update, this appraisal topic has been suspended because BioMarin has withdrawn from the technology appraisal process. NICE will continue to monitor any development and provide further information, as available.
 
Status Suspended
Process STA 2018
ID number 1475

Project Team

Project lead Louise Jafferally

Email enquiries

Evidence Review Group / Assessment Group Liverpool Reviews and Implementation Group, University of Liverpool

Timeline

Key events during the development of the guidance:

Date Update
31 October 2019 Suspended, Following recommencement of the appraisal and a company update, this appraisal topic has been suspended because BioMarin has withdrawn from the technology appraisal process. NICE will continue to monitor any development and provide further information, as available.
19 August 2019 In development. This appraisal topic recommenced at ‘clarification stage’.
30 April 2019 In development. This topic will be rescheduled into the technology appraisal STA programme and will recommence at the ‘clarification stage'.
31 January 2019 Suspended. Following a challenge to the decision to review sapropterin for treating phenylketonuria through the technology appraisal programme, NICE has made the exceptional decision to pause the appraisal at this point and ask the topic selection decision making panel to reconsider whether this topic is suitable for the NICE highly specialised technologies or technology appraisal programme. After this a formal referral will be required from the Minister to determine the process under which sapropterin will be assessed. An update will be provided once a formal referral is received.
16 October 2018 Invitation to participate
09 October 2018 In progress, Topic referred
13 August 2018 - 10 September 2018 Consultation on suggested remit, draft scope and provisional matrix of consultees and commentators

For further information on how we develop guidance, please see our page about NICE technology appraisal guidance