This petition was submitted during the 2019-2024 parliament
Petition Fund Kuvan (sapropterin) on the NHS for people with PKU
The drug Kuvan allows people with the rare condition PKU to manage their condition. The treatment has been licensed for 11 years but has never been commissioned by the NHS.
The government should make funding available for this treatment for all PKU patients that could benefit.
More details
PKU is a rare condition, diagnosed at birth. People with PKU cannot process protein through their body properly and without careful management an amino acid (phenylalanine) causes brain damage. The only treatment funded by the NHS is a restricted diet containing virtually no natural protein. The PKU diet is stressful and difficult to cope with. Outcomes are variable.
Kuvan (sapropterin) is a life changing treatment available in almost every country in Europe. It needs to be available here.
This petition is closed This petition ran for 6 months
Government responded
This response was given on 7 April 2020
We understand how difficult it is living with phenylketonuria. NICE is developing guidance on the use of Kuvan and the Government urges BioMarin to engage with the NHS to agree a fair price.
Read the response in full
This Government is committed to ensuring that patients with rare diseases can secure a diagnosis and access appropriate and effective treatment, and we understand how difficult it is for patients living with phenylketonuria (PKU) and their families.
The Government wants NHS patients to benefit from effective treatments at a price that is fair to the NHS and the taxpayer. It is important that we have a system in place for making evidence-based decisions on whether new medicines should be routinely funded by the NHS, based on their clinical and cost effectiveness.
The National Institute for Health and Care Excellence (NICE) is the independent body that makes recommendations on whether new drugs work well enough to justify the cost to the NHS. NHS commissioners are legally required to fund medicines recommended in NICE technology appraisal guidance. Many thousands of patients have benefitted from access to effective treatments as a result of NICE’s guidance.
In July 2018, the then Secretary of State asked NICE to develop guidance on whether Kuvan represents a clinically and cost-effective use of NHS resources and should, therefore, be made routinely available to patients. These are very difficult decisions to make and it is right that they are made independently based on an assessment of the evidence.
NICE’s appraisal was unfortunately suspended as the manufacturer of Kuvan, BioMarin, withdrew from the process. However, I am pleased that BioMarin has now agreed to re-engage in the appraisal and NICE will now aim to publish guidance on Kuvan as soon as possible. The Government urges BioMarin to engage constructively in negotiations with NHS England and Improvement and NICE to agree a fair price for Kuvan.
Up to date information on the progress of the appraisal is available on NICE’s website at https://www.nice.org.uk/guidance/indevelopment/gid-ta10378.
Department of Health and Social Care