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Kalydeco (VX770)

Have just read the devastating news on the CF Trust Forum that NICE are not going to recommend the NHS provide Kalydeco in UK and want to know your thoughts and any suggestions on this to get it available? Obviously as much media coverage as possible I imagine. Whilst NICE relates to England and Wales I believe Scotland and N Ireland also more or less follow what NICE recommends. Although only a small percentage of CF sufferers have the G551 gene the availability of Kalydeco was going to be seen as a major step to a better life for all CF sufferers. To those who do have the gene they have been waiting so long for it to be approved here it is just like being stabbed in the back and given a death warrant. Here is for the first time a drug that would improve and lengthen lives so much and now they are once again being sentenced to a slow, sure death. To think NICE are categorically stating this before the drug is even approved. Only the extremely rich will benefit in UK as far as I can see. It really is heartbreaking. I know in the past this has happened with Cancer drugs etc but sometimes the new drug was only going to add a few weeks to a persons life but Kalydeco looked like it was going to be a new beginning for those who would benefit from it. I believe in UK, according to NICE you could apply to your local Primary Care Trust but we all know they will not have funds. This being turned down will also ask the question of why our CF adults and children should risk drug trials for no benefit to them in the end. This is so unfair!!!
Dear Questioner
Thank you for your important question.

I appreciate there is a lot of interest and indeed concern about the pending outcome from the European regulatory bodies with regards to approval for Kalydeco (VX-770) in Europe and the UK.

Kalydeco has been approved for use in patients with a G551D mutation by the FDA in North America, based on the results from phase 3 trials in patients aged 6 and over. These results showed that patients receiving the treatment on the study experienced rapid, significant and sustained improvements across a variety of disease measures, including lung function and the majority of adverse events associated with Kalydeco were mild to moderate in severity and non-serious.

As the NHS in the UK have a very different healthcare system to the US, a separate review process must be completed and a separate recommendation made.

There is a lot of speculation from CF clinicians, parents and patients as to the outcome of regulatory body reviews. There has been a recent progress update on the approval process.
On the 24th May the CF Trust welcomed today's news that the European Committee for Medicinal Products for Human Use (CHMP) recommended that Kalydeco should be approved within the EU for people with CF aged six and over and who have at least one copy of the G551D mutation.
A decision on marketing approval should be made by 27 July 2012. For patients and families, final decisions on the availability of the drug to NHS patients by prescription will be taken by NHS commissioners following negotiations with the manufacturer Vertex.
Currently, Kalydeco will not be assessed by NICE. NICE have responded to calls for the treatment to be appraised by stating that, following evaluation against the Department of Health's criteria, the decision taken was that Ivacaftor should not proceed to technology appraisal. This is because the population size is too small to add value by publishing guidance. Ivacaftor has Ultra Orphan Drug status, which means that it will only be effective for a small population size.
The CF Trust is working with all the regulatory bodies to try to ensure Kalydeco is made available as quickly as possible to people with CF in the UK with gating mutations who stand to benefit from this treatment.

We appreciate it is very frustrating that there is no guaranteed benefit to participants who take part in any clinical trial, including the trials of Kalydeco. For this reason all trials are designed so as to minimise any harm to participants and operate on a voluntary basis. Future trials of medication for CF including future trials of Kalydeco are essential for these treatments to progress. We recognise the enormous contribution from patients to date and hope that this will continue.

Accurso, F.J., Rowe, S.M., Clancy, J.P., Boyle, M.P., Dunitz, J.M., Durie, P.R., Sagel, S.D., Hornick, D.B., Konstan, M.W., Donaldson, S.H., Moss, R.B., Pilewski, J.M., Rubenstein, R.C., Uluer, A.Z., Aitken, M.L., Freedman, S.D., Rose, L.M., Mayer-Hamblett, N., Dong, Q., Zha, J., Stone, A.J., Olson, E.R., OrdoƱez, C.L., Campbell, P.W., Ashlock, M.A. & Ramsey, B.W. 2010, "Effect of VX-770 in Persons with Cystic Fibrosis and the G551D-CFTR Mutation", N Engl J Med, vol. 363, no. 21, pp. 1991Cystic Fibrosis Trust

Best wishes
Katherine ONeill
Stuart Elborn
The answer is edited by: Katherine O'Neill