What you can do

Biomarin, the company licensing Kuvan in Europe, has offered a one month free trial of Kuvan for every PKU patient in the UK. However, this is on the condition that the patient’s Primary Care Trust (PCT) agrees to pick up the funding, after this free trial, should the drug prove to be effective. The PCTs will not commit to this. We have been advised by a legal expert with much experience of PCT practices that we need as many parents as possible to request Kuvan through the Individual Funding Request route (IFR) in order to stand a chance of achieving NHS funding. The steps are as follows:

1. Ask your consultant. You may find that your PCT has not even looked at Kuvan as no one has requested treatment. In this case, you will first need to request the drug from your consultant by asking for the onemonth free trial. Your PCT will be contacted regarding this request. It is likely that your PCT will refuse. Once Kuvan has been refused by your PCT, you can then go down the Individual Funding Request (IFR) route.2. The IFR form should be completed by your consultant and sent to your PCT.

3. If the IFR is then refused, you can appeal. This too may be rejected. At this point, we will be able to get legal aid (in particular all children are entitled to legal aid regardless of how much their parents earn). The government will then have to pay to meet our case against the PCTs and at this point a real incentive exists for the PCT in question to reconsider its position. We have been advised by a barrister specializing in this process that the greater the number of IFR requests coming from patients, the better our chances of success.

There are other things you can do:

4. Contact your MP. Some MP’s have already been contacted and asked to approach Biomarin, the company responsible for licensing Kuvan in Europe, in order to negotiate the cost of the drug. The more MPs that contact Biomarin the better, as this is all about applying pressure. You can also ask your MP to write a letter of support to your local PCT for your application for Kuvan.

5. Spread the word: We suggest also that you make patients who attend your treatment centre aware of this campaign. The PKU clinic at Great Ormond Street for example has already agreed for Phed up to hold an information and discussion meeting, to which they will invite patients, parents and carers.

The history of PKU shows us that change has to been made to happen. The discovery of PKU in 1934 was itself instigated by a mother’s persistence and absolutely crucial things which we take for granted in the current management of PKU, the universal heel-prick test, for example – did not happen automatically: they had to be fought for. Unfortunately, no one will just give us Kuvan, PEG-PAL or any other future treatment if we do not show the wider society that the PKU community is committed to obtaining the best treatment. Kuvan is the best place to start the campaign and the time to do this is now.

You can contact Phed up via email: info@phedup.co.uk