• Judicial review launched against NICE for denying access to rare disease drug Kuvan, which was licensed 12 years ago
  • Case challenges criteria for entry into NICE’s rare disease appraisal process
  • Jeremy Hunt has urged Matt Hancock to use “same magic” to secure access to Kuvan that he did with cystic fibrosis drug

Former health secretary Jeremy Hunt has urged his successor to find a way to ensure patients can access a high cost drug which is subject to a legal challenge against the National Institute for Health and Care Excellence.

A judicial review has been brought against NICE over its decision to refuse access to sapropterin.

The drug, also known by brand name Kuvan, reduces the severity of the condition phenylketonuria, a rare metabolic disease which can cause brain damage unless strictly controlled.

The review, brought last week by the family of 11-year-old Cait Cotter, challenges the criteria for entry into NICE’s rare disease appraisal process.

Peter Todd, of law firm Hodge Jones and Allen, who is representing the Cotters, said a successful challenge could have “wide application for all rare diseases”. The charity Genetic Alliance UK said the case showed NICE’s appraisal system for rare diseases is “not currently fit for purpose… [and] need[s] wholesale reform”.

An outcome is expected within the month.

The drug was licensed in 2008 but has never been made available on the NHS. It is available in at least 24 European countries, plus Russia and the US.

There are estimated to be around 6,200 people with phenylketonuria in the UK, of which Kuvan is estimated to work for around 20 per cent. The drug costs between £14,000 and £45,000 per patient, per year, which suggests the potential annual costs could range from around £17m to £56m.

Mr Hunt has urged health and social care secretary Matt Hancock to find a resolution, telling Parliament last week: “One of the most difficult challenges for the health secretaries of all parties is meeting people who are denied access to a medicine that is not available on the NHS and he did that with the Orkambi families just before the election and did a brilliant job in securing access to that medicine which will transform the lives of many, many families. I hope he will now use the same magic to get access to Kuvan…”

He also revealed Mr Hancock is to meet two of his constituents who live with the condition.

Assessment process

The claimant’s legal team has argued NICE uses stricter entry criteria for a drug to be reviewed under the rare disease appraisal process than laid out by law. They add that Kuvan meets the statutory criteria to be assessed as a highly specialised technology, as opposed to a standard technology, which would open the door to funding.

The standard process expects a drug to demonstrate a much higher ratio of clinical effectiveness to drug price. This is deemed by some, such as the Genetic Alliance, to disadvantage drugs for rare diseases as they are often expensive with a lower evidence base due to the low pool of patients they benefit. For those medicines such as Kuvan, that do not cure a patient but reduce the severity of an illness over their lifetime, there is also negligible evidence available of its long-term benefits.

One industry source said: “The criteria for entry into the highly specialised technology programme… are rather vague. This is certainly something industry would want both clarified and ideally broadened out so more treatments could qualify.”

They added: “If the judgment goes against NICE then I wouldn’t be surprised if it is used as a reference point for some of industry’s wider concerns feeding into [the review of NICE’s methods reporting this year].”

In July 2019, NICE announced a review of its drug appraisal methods which would include updating the criteria used for which drugs are evaluated through NICE’s HST programme. However, NICE said at the time “it is not intended that revised wording will increase or decrease the number of HST topics”.

Cait’s father, John Cotter said: “When my baby was diagnosed with a very serious and very rare disease, I trusted the NHS to provide the best care. I would never have dreamed that a decade later she would still not have access to a medication available in every country in Europe apart from Poland.”

The Cotters have been granted legal aid for the legal challenge by the Legal Aid Agency. NICE declined to comment.