Child with rare disease takes NICE to court over access to life-changing therapy

NICE’s decision-making process will come under legal scrutiny in court this week, after a child and her family claimed the NHS cost-effectiveness body is unlawfully limiting access to a life-changing rare disease drug.

The crux of the case brought by 11 year-old Cait Cotter and her family is that NICE has opted to assess BioMarin’s phenylketonuria therapy Kuvan (sapropterin) using its standard assessment process.

But as this is a rare disease affecting only one in 12,000 babies, the Cotter family says NICE should be using its Highly Specialised Technology (HST) programme that gives much more flexibility on cost.

Lawyers acting on behalf of the family say there is no chance of Kuvan getting funded on the NHS using the standard assessment and will begin a challenge on this technicality in the High Court judicial review, which is set to begin tomorrow.

Cait’s condition is currently managed by a combination of a tough dietary regime, artificial protein and amino acid supplements and blood tests.

According to Cait’s family, her life would be “transformed” by access to Kuvan, which has been licensed in Europe to treat the disease since 2008.

BioMarin has already pulled out of the assessment process, saying that there is no way that funding criteria under a standard assessment could be met.

Under the HST programme, NICE allows up to £100,000 per Quality Adjusted Life Year for rare disease drugs and can move the threshold even higher for drugs that are shown to extend life for several decades.

Decision unlawful, says lawyer

Cait will be represented by Peter Todd of Hodge Jones & Allen solicitors during the one-day hearing. Todd said: “The claimant considers Kuvan meets all the statutory criteria to be assessed as a ‘Highly Specialised Technology’. However NICE decided it should instead be assessed as a ‘standard technology’.

“This was a crucial decision for the claimant, as the chances of Kuvan being recommended by NICE for NHS funding depended entirely on it being assessed as a High Specialised Technology rather than a standard technology.”

“The claim asks the High Court to quash NICE’s decision, on the grounds NICE did not apply the statutory criteria for assessment correctly, and was therefore unlawful.”

Restricting access

According to the charity Genetic Alliance UK, the case demonstrates a wider problem: that NICE is restricting access to rare disease drugs by using standard assessments instead of the HST programme.

CEO Jayne Spink said: “NICE created the Highly Specialised Technology programme with rules designed to be fair to diseases affecting small patient numbers.

“Unfortunately, NICE restricts access to the programme even for extremely rare conditions. The decision-making process is opaque. Since the programme was established in 2013, guidance has been published on only eleven rare disease medicines.

“This is a fraction of the number of drugs licensed for rare conditions in that period. This litigation highlights the need for wholesale reform of the system for appraising rare disease treatments. It is not currently fit for purpose.”

pharmaphorum has approached BioMarin for comment, but no-one was immediately available.

NICE will not comment until after the outcome of the judicial review, which is likely to be announced within the next week.