Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, whether a single technology appraisal process or a highly specialised technology appraisal process is used for (a) phenylketonuria and (b) other rare diseases.

Medicines and other treatments being considered for assessment by the National Institute for Health and Care Excellence (NICE) are considered through an established topic selection process to ensure that they are suitable for referral to NICE and that they are routed to the most appropriate process within NICE. The topic selection process includes consideration of topic against published criteria and consultation with stakeholders. Most new medicines are referred for assessment through NICE’s technology appraisal programme though NICE also operates a separate highly specialised technology evaluation programme for a small number of very high cost drugs for the treatment of very rare diseases. Following consideration through the topic selection process, NICE is currently developing guidance on two drugs for the treatment of phenylketonuria through its technology appraisal programme.