NHS England has launched a consultation on a draft policy to fund Kuvan (sapropterin) for people with PKU in England.  You can find it here (click the link):


NHS England considers different proposals for funding specialist treatments twice a year.  This is continuing despite the other pressures on the NHS at the moment.  This process decides how to spend money from a budget set aside for rare conditions and specialist services.

What is Kuvan/sapropterin?

Kuvan is a treatment for PKU which works for some people with PKU.  People taking Kuvan take pills in the morning and this allows them to eat more protein whilst controlling their phenylalanine levels.  You can read more about the treatment (and watch a video) here:


What are the views of NSPKU?

We believe the UK needs to catch up with the rest of Europe in the provision of treatments for PKU.  We have been involved in the development of this policy for Kuvan and support its adoption.  Kuvan does not work for everyone but it can be a life-changing treatment for people who respond to the treatment.

What about people outside England?

People living outside England are not included within this policy proposal.  However NSPKU believes that the adoption of Kuvan in any UK nation will help increase the chances of its adoption elsewhere.  People outside England are able to complete the online consultation.

Should I fill in the online consultation?

Yes!  It is important that the opinions and experience of people living with PKU are taken into account.  PKU is not well understood by those who do not live with it.

If you have experience of living with PKU you can write this in your response to the consultation.

The issues NSPKU will raise in the charity’s response include:

  • That the evidence review should have considered Maternal PKU issues and the use of Kuvan in pregnancy/pre-conception.  This is an important issue.
  • The evidence review should have considered evidence of the relationship between phenylalanine levels and issues such as mental health or cognitive functioning.
  • The policy should make it clearer that some people (e.g. with cognitive functioning deficits) can’t consistently cope with PKU dietary treatment unless they have help. These people do not have a treatment for their PKU at the moment.
  • The impact assessment accepts that there are significant costs savings from patients using less dietary products if they are taking Kuvan. However, as these savings are not recouped from local Clinical Commissioning Groups this is not treated as a saving by NHS England.
  • The budget impact assumes that the costs of the drug will remain the same for 5 years. This is not realistic as cheaper “generic” alternatives will be available soon.
  • The budget impact does not reflect the many wider savings to taxpayers if people with PKU are controlling their phenylalanine levels more easily; e.g savings in social security benefits, health, social care and education.
  • There is a high risk of “health inequalities” amongst people with PKU. People or families with vulnerabilities (e.g. mental health problems or poverty) may be less able to cope with the dietary treatment. Kuvan could give more people a fairer chance of better outcomes.

What is the decision making process for the Kuvan policy?

NHS England will have to review all the consultation responses.  A decision about whether the policy will be funded or not is likely to be made at the end of May.  You can read more about how this process works here.  https://www.england.nhs.uk/commissioning/cpag/