Who is NICE and what are they doing?


NICE is the “National Institute for Health and Care Excellence”.  It reviews medicines for the NHS and decides whether they are value for money.  NICE is currently reviewing the medicine Kuvan (also called sapropterin) for PKU and deciding whether it should be used by the NHS.

Kuvan is a medicine for people with PKU. Kuvan does not work for all people with PKU.  It is believed that Kuvan would work for about 30% of people with PKU in the UK.  You can read more about Kuvan here – https://nspku.org/download/kuvan-facts/

 I live in England/Wales/Northern Ireland/Scotland – what effect does this have on me?

 NICE guidance is binding on the NHS in England and Wales and should be implemented within 3 months.

In Northern Ireland, NICE guidance is usually followed but this does not happen automatically.

Scotland has its own organisation for reviewing medicines called the SMC (Scottish Medicines Consortium).  NICE decisions have no direct affect in Scotland because they follow SMC guidance instead. NSPKU wants the SMC to look at Kuvan again to update their guidance.

 What have NICE said? 

On Thursday 25th February NICE published drafted guidance about Kuvan for PKU.  This is draft guidance – this is NOT the final word.

Their draft recommendations are:

Kuvan is recommended in children up to the age of 18, because they say the treatment is cost effective for children.

Kuvan is not recommended in adults 18 and over, because they say they treatment is not cost effective for adults.

Kuvan is recommended at a lower dose of 10mg/kg.

What is the next stage of NICE’s decision making process?

NICE are asking people to give their opinions on this draft guidance through a consultation process.  This includes people with PKU and their families.

The consultation period runs until 18 March and we encourage people to have their say.  The link to the consultation is here https://www.nice.org.uk/guidance/indevelopment/gid-ta10378/consultation/html-content-2

After this, NICE will meet to consider all the comments made and will issue final guidance in April.

What is NSPKU’s views on the draft guidance?

 NSPKU is concerned that the draft NICE decision is not correct and needs changing:

  1. We welcome the recommendation that children can use Kuvan but NSPKU believes that patients of all ages with PKU who respond to treatment with Kuvan should be able to use this medicine.
  2. NICE has not considered the problem about young people stopping treatment with Kuvan on their 18th birthday.  Managing your phenylalanine levels with dietary treatment alone is very difficult, especially for teenagers. 18 year olds accustomed to a very relaxed diet through using KUVAN will not have the coping skills to switch to a strict diet. NICE has ignored this issue completely.
  3. NICE’s analysis underestimates the benefits of KUVAN treatment for adults with PKU. They say there is no risk of permanent damage to the brain after the age of 18, but this is contradicted by other statements  made by NICE which recognise that permanent harm can occur after the age of 18.  NSPKU also recognise that many adults with PKU have very serious symptoms caused by high phenylalanine levels in adulthood.  These problems in adults are undervalued by NICE.
  4. NICE has not considered the harm of Maternal PKU syndrome and the worry this can cause to women with PKU. When deciding that Kuvan is not cost effective in adults, NICE has ignored the benefits to women of using Kuvan to help women with PKU have safe and happy pregnancies.  NICE has recognised that controlling phe levels is important in early pregnancy, ideally before conception, and has recognised that this would reduce the risks of Maternal PKU syndrome.  However the harms from high levels in early pregnancy have not been included in their cost analysis.  NSPKU believes that NICE has failed to take account of the issues experienced by women with PKU and that this is a major failing in their draft guidance.
  5. NICE has ignored that many adults with PKU who have problems associated with high phenylalanine levels – like brain fog, depression or poor memory – simply cannot cope with dietary treatment. The NHS should not leave people without a realistic option for treatment.  NSPKU also believes that NICE has undervalued the work and impact that managing PKU can have on adults with PKU and their families.
  6. People with learning disabilities are at a higher risk of being unable to control their phenylalanine levels with dietary treatment. The need for help with dietary treatment might also restrict the independence of people with learning difficulties. NICE recognised that people with learning difficulties might struggle with dietary treatment but there is no evidence that this has been included in the cost analysis.
  7. NICE has recommended using KUVAN at a dose of 10mg/kg. NSPKU agrees that clinicians in the UK will prescribe more efficiently than in the US and that an average dose of 10mg/kg is appropriate for the cost analysis.  However NSPKU believes that clinicians should be able to prescribe within the marketing authorisation, which ranges from 5mg/kg to 20mg/kg.

What happens next?

NSPKU encourages as many people as possible to have their say on the draft guidance by participating in the consultation process.  If you are unsure about how to do this or what to say, please join our online meeting on Saturday (see further below).

NICE will meet again on 7 April 2021 and will publish final recommendations about using Kuvan shortly after this.

If Kuvan is recommended we would expect it to become available in England and Wales within 3 months.  Northern Ireland may take slightly longer but its decisions are usually followed.  NSPKU wants Kuvan to be available throughout the UK as soon as possible and we will encourage our members in Scotland to work with us to achieve that.

What about cheaper generic (i.e. “unbranded”) versions of Kuvan? 

NICE has assessed the cost effectiveness of BioMarin’s branded product Kuvan based on BioMarin’s pricing offer.  We know that cheaper unbranded versions of the medicine will become available soon.

NHS England have already said this:

“BioMarin’s exclusivity of Sapropterin is due to end in Europe shortly, meaning that generic versions of Sapropterin are now being manufactured. Should companies decide to make these available in England, NHS England can confirm that it would also commission any generic version(s) of Sapropterin from the next financial year onwards on the condition that these manufacturers price their products below the maximum level NICE advises as representing a clinically and cost-effective use of NHS resources.”

 NSPKU therefore believes that NHS England could commission generic (unbranded) versions of sapropterin/Kuvan for people with PKU of all ages, provided that it is offered at a price that NICE regards as cost effective.

What about those people who don’t respond to Kuvan?  Will they be left behind?

 NSPKU is committed to fighting for all treatments for PKU that are safe and effective.  We think getting Kuvan used by the NHS will be an important step which will help “pave the way” for other innovative treatments.

There are many different non-diet treatments being researched which we expect to become available in the future.  The injectable enzyme treatment Palynziq (also called pegvalaise) will also be considered by NICE in the future once further research is available.

NSPKU also strongly supports the need for patients to have access to the full range of options for dietary treatment, without good support from metabolic clinics.

I’m worried about finding out if I or my child would be responsive to Kuvan.  How will this work?

 Kuvan does not work for all people with PKU.  Arrangements for response testing are not in place yet and Kuvan is not available for anyone yet.

However we anticipate that response testing will be carried out by the patient using Kuvan for a short period of time.  Clinicians will review the patient’s phe levels before and after they are given Kuvan.  Patients who experience a drop in phe levels of 30% are usually deemed responsive.  Some patients may have particular genetic mutations which are known to mean the patient will not respond to Kuvan.

Further details about how the arrangements will work will be made available in the future if NICE confirms its decision to recommend using Kuvan.

NSPKU virtual meetings to discuss the NICE decision and the next steps…

We know that people may have lots of questions and we are therefore running two online meetings on Saturday to discuss the situation.

These sessions will include – Professor Anita MacDonald, Consultant Dietitian of Birmingham Children’s Hospital

Suzanne Ford, NSPKU’s dietitian adviser, who also works with adults with PKU at Southmead Hospital, Bristol

Kate Learoyd, NSPKU’s campaign manager and Eric Lange, NSPKU’s Chairman.

We hope you can join us  and ask any questions you have.  The sign up arrangements are here:


Should I speak to my metabolic team about Kuvan yet?

No, not yet.  Kuvan will not be available for anyone until after a final decision is made by NICE in April.