Individual Funding Requests for Kuvan (BH4) Treatment

The NHS does not routinely commission Kuvan treatment for phenylketonuria (PKU) except for pregnant women. The National Society for Phenylketonuria (NSPKU) is seeking to change this policy through our Call for Action campaign.

The NSPKU is aware that some children and adults can have difficult circumstances that can make living with PKU particularly challenging. We therefore wish to help support our members access Kuvan on an individual basis if at all possible.

Individual Funding Requests can sometimes be used as a means of prescribing a treatment that is not routinely offered by the NHS. An individual funding request for Kuvan can be made by your metabolic specialist, if they think your particular circumstances mean that this is the appropriate treatment for you and that there are no alternatives available on the NHS.

The High Court has ruled that Kuvan is a clinically effective treatment. However, to succeed in making an individual funding request, you will also need to show that your situation is "clinically exceptional". There is concern that consultants are not currently paying sufficient regard to the availability of funding through this route.

The clinically exceptional rule does not apply in Wales following a recent rule change. In Wales, the patient needs to show that the patient would gain significant clinical benefit. A panel then rules on whether the cost is reasonable in the circumstances of that patient.

The rules in Scotland are currently under review – and the system may become better for patients with very rare conditions. Therefore if you live in Scotland we recommend that you get in contact with NSPKU (info@nspku.org) before you proceed.  

 Individual Funding Requests have different names around the UK: -

  • England - Individual Funding Request (IFR)
  • Scotland - Individual Patient Treatment Request (IPTR)
  • Wales - Individual Patient Funding Request (IPFR)
  • Northern Ireland - Individual Funding Request (IFR)

If your metabolic team makes a request on your behalf, they complete an official form which is then considered by a panel. You will have the opportunity to see the form. In Scotland, you also have the ability to make a personal statement. You can obtain legal advice on the contents of the form if you wish, and some solicitors will do this for free. However, legal advice is not usually necessary at this stage.

 

NSPKU template letter to your metabolic specialist about Kuvan

The NSPKU has prepared standard form letters if you would like your metabolic team to consider an individual funding request for Kuvan treatment. Please contact the NSPKU to request the correct letter – for children or adults – and the one which is appropriate for the country in which you live.

The letters contain a list of the particular circumstances that you could ask your specialist to consider. Think about which issues that might be relevant and delete the ones which do not apply to you. If there are any other issues you would like considered, you could add these to the list.

The final paragraph asks for your metabolic team to schedule a meeting or call to discuss your request. If you feel you need some support to get your points across with your doctor, a support worker from the NSPKU can participate in a discussion by telephone call. As we are a small charity, we are not able to attend meetings at your clinic.

The decision whether or not to pursue an individual funding request for Kuvan is for your metabolic specialist. However, if your doctor does not proceed with a request, he or she should provide a clear explanation of why they believe you are not eligible, or why the treatment would not be appropriate for you.

 

Testing for responsiveness to BH4 (Kuvan)

The letter suggests analysis of PKU gene mutations. In the UK, we do not perform routine mutation analysis in patients with PKU, but this information may be helpful in predicting Kuvan responsiveness in patients with either two null mutations (complete absence of enzyme activity – so an unlikely Kuvan responder) or with two BH4 (Kuvan) responsive mutations (a likely Kuvan responder).

The NSPKU is gathering data about patient requests for Kuvan. If you have requested Kuvan from your metabolic team, please let us know. Your situation will be kept confidential.

If you need any guidance or support about making an individual funding request, please email or call the NSPKU helpline.

Email: - info@nspku.org

Telephone: - 030 3040 1090

Address: - NSPKU, PO Box 3143, Purley CR8 9DD