11 JUN 2019

Speech on cystic fibrosis

John Howell (Henley) (Con)

It is a pleasure to serve under your chairmanship, Mr Bailey. My first introduction to cystic fibrosis came before I became an MP. I wrote some newsletters and did some public relations work for the Cystic Fibrosis Holiday Fund, the main objective of which was to provide holidays and ancillary facilities to under-18s who suffer from cystic fibrosis. On the basis of medical advice that was given in 2000, we now cannot take those children away together, so the fund spends most of its time generating respite break grants and providing the Family Revitalise programme. Those initiatives are both important, but do not compare with making available Orkambi or any of the other drugs that have been mentioned.

Two families in my constituency have children with cystic fibrosis. I have spent time with both families, and have seen that largely the children are happy, normal children who enjoy all the things that other children enjoy. Hanging over them, however, is the threat of a double-lung transplant just to stay alive.

Orkambi changes lives, and we need to look at ways that we can make it available. A number of structural difficulties were identified during the conversations that I have had on the matter. The first is one of commercial incentive and risk. To compound that point, one can look at the relative strength-in-numbers of those who suffer from diabetes or from cystic fibrosis: diabetes accounts for 4 million people, while cystic fibrosis accounts for only 70,000. A major hurdle is therefore already built in for those with cystic fibrosis to overcome. We should not forget that.

The issue of the time taken, which has already been raised, goes back to criticisms of the NICE process. The criticisms that I would make fall into three types: first, NICE adopts the same evaluation process for a drug that might treat tens of millions of people as it does for a drug that treats a few hundred thousand or, indeed, a few thousand. We need to bring home to NICE that that is not a right way to proceed.​

Secondly, the same evaluation process is also used whether the drug is taken for a brief period or a long one—in other words, whether it is a short use cancer-related drug or, as in the case of Orkambi, it must keep being taken over many long periods. That factor needs to be built into any evaluation of the drug as well.

The third criticism that I would make of the NICE process is that it is too focused on short-term benefits, and not on long-term benefits, which we know that Orkambi can produce. As has been mentioned, the data released by Vertex show that after 96 weeks of treatment, the rate of lung function decline reduced by 42%. That is a major long-term thing to hang on to. Furthermore, the net value of Orkambi is hard to calculate and therefore to capture accurately. A number of direct costs need to be taken into account, such as the cost of hospitalisation, and there is evidence that Orkambi starts to reduce the number of other medicines that need to be taken.

We have heard that Orkambi is available in many other countries in Europe, although I hear that the Spanish Government are having difficulties with Vertex, in the same way as we are, over the availability of the drug. The agreement that was reached with Vertex to make Orkambi available was a disappointing affair. We need to put on the pressure to ensure that that happens and that generic drugs are brought forward to be used instead. The example often cited is Ireland—both families in my constituency mentioned the situation there—and it is interesting to note that success story of the use of Orkambi. It has been very successful there, and we should all take that to heart in making progress to ensure that young people suffering from cystic fibrosis have access to this drug.

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