Accelerated Access Review

Julian Sturdy Excerpts

Tuesday 13th December 2016 Westminster Hall

[Ian Austin]

My right hon. Friend is completely right; he raises a point I will make shortly. It is good that he is here to support people with cystic fibrosis in his constituency, and to bring his knowledge and experience of the national health service to bear in the debate.

The Cystic Fibrosis Trust’s proposal would provide foundations for a managed access scheme for the drug. That was in line with the interim findings of the accelerated access review, which recommended the merits of such an approach and referred to the UK cystic fibrosis registry as an exemplar. I will say more about the accelerated access review in a few moments.

As expected, seven months later NICE referred to a lack of long-term data in rejecting Orkambi for use in the NHS. That was despite Orkambi’s being proven to halve hospitalisations and NICE’s recognising it as a

“valuable new therapy for managing cystic fibrosis”

with significant clinical benefits, as well as

“wider benefits to society for people with cystic fibrosis and carers of people with cystic fibrosis.”

[Julian Sturdy (York Outer) (Con)]

I congratulate the hon. Gentleman on securing this important debate. He correctly points out that this is not just about the way in which Orkambi improves quality of life, which I know is extremely important, but about cutting hospital admissions. That has to be taken into account when we look at the wider cost implications of the drug. What we need is time for the drug to be given the chance to prove its worth.

[Ian Austin]

The hon. Gentleman is completely right to say that Orkambi could reduce hospital admissions, and could shorten the amount of time people spend in hospital when they have been admitted.

In its statement, NICE referred directly to the trust’s proposal as a potential solution to the shortage of long-term data. With the NICE process exhausted and seven months wasted, we hoped that the way would be clear for direct negotiations between the drug manufacturer Vertex and NHS England, which would allow for a speedy resolution to the situation. However, Department of Health officials then demanded that the drug be put through a rapid review process, which, at 16 weeks, is anything but.That process is based on exactly the same criteria that had just seen Orkambi denied to those who need it. Vertex has declined to enter the process, because of the certainty that it will come to nothing.

New data published in October at the North American cystic fibrosis conference, which my right hon. Friend the Member for Leigh (Andy Burnham) mentioned, are based on 96 weeks of trials and show that Orkambi slows the decline in lung health by up to 42%. That is comparable with the 47% slow in decline caused by the transformational treatment Kalydeco, which is widely available in the UK for a less common mutation of cystic fibrosis. Those data were unavailable to NICE but clearly illustrate that drugs such as Orkambi need the chance to prove their worth in the long term. That also underlines the fact that we now have a situation where people with cystic fibrosis face discrimination by genotype, because they are being denied the same level of treatment that people with a different genetic mutation of cystic fibrosis receive.

Twelve months after licensing, negotiations are at a standstill. I understand that Vertex is keen to offer a substantial discount, but for commercial reasons would need to do so confidentially. It would like to take up the trust’s offer of monitoring the effectiveness of Orkambi for a trial period. That could build on the American data and allow NHS England to conduct final negotiations based on an accurate reflection of the drug’s effectiveness.