Orkambi in the UK: Our work begins

Following the Trust taking part in a meeting with National Institute for Health and Care Excellence (NICE) to discuss the process for appraising the new cystic fibrosis drug Orkambi, Chief Executive Ed Owen, who attended to represent the Trust, looks a the road ahead for getting the combination therapy to people with CF.

The news two weeks ago that Vertex’s latest drug, Orkambi (a combination of ivacaftor or Kalydeco and lumacaftor), has demonstrated impact for the most common mutation of cystic fibrosis was greeted with enthusiasm and excitement.

TheBBC called it a “breakthrough drug” and it is the first therapy that targets the basic genetic defect of those with two copies of the F508d mutation affecting about half the cystic fibrosis population in the UK.

It is a significant development. But we should be under no illusions that this is anything but the beginning of what is most likely to be a tough fight to ensure that all those who might benefit from Orkambi in the UK get access to it.

First of all, it has to be licensed for use by the European Medicines Agency, which is the primary body responsible for granting drug authorisation across all EU member states, including the UK. This decision is expected in the autumn, two or three months after the equivalent decision in the USA by the Federal Drugs Administration.

Then, if the EMA does grant a licence to Orkambi, it will need to be assessed by each of the four devolved NHS systems in the UK (in England, Wales, Scotland and Northern Ireland) and each will need to decide whether and how to allocate funding to pay for it.

In Scotland, the drug will be assessed by the Scottish Medicines Consortium while in England it is likely to be subject to an appraisal by NICE, the National Institute of Health and Care Excellence.

Yesterday, in Manchester, I attended a meeting organised by NICE and including cystic fibrosis clinicians and health professionals along with NHS commissioners and others to discuss possible options of how it might undertake such an appraisal. If it does do so, NICE will look at the clinical effectiveness of the drug as compared to existing therapies available and, even if that is accepted, then take a view on whether the likely clinical impact justifies the likely cost to the NHS.

Of course, calculating the cost means knowing the price – and Vertex has yet to announce the price it will charge for Orkambi. Most experts assume it will not come cheap.

As my colleague, Nick Medhurst, said yesterday, it is encouraging that NICE is engaging early on this issue, in advance of the EMA’s decision. We do have concerns, however, that the methods it uses to carry out drug appraisals are not particularly well-suited to assessing potentially transformational drugs in relatively rare conditions like cystic fibrosis.

And an appraisal can often take six months or more with no guarantee of a positive recommendation at the end of it.

At the appropriate time, Vertex is likely to be asked to submit detailed evidence to NICE in order to demonstrate that Orkambi is both effective and affordable. The Trust will also submit evidence to communicate the voice and experience of people with cystic fibrosis themselves, and their carers.

This process is unlikely to begin much before the end of this year in the event of a positive decision by the EMA. Yet we are already listening and engaging with the wider cystic fibrosis community to ensure we are able to speak clearly and forcefully on behalf of everyone with the condition in this forthcoming process.

So, it will not be an easy ride. But we will always be committed to getting therapies that make a difference to people’s lives to those that need them as soon as possible. In order to do this we will need both Vertex and the NHS to do the right thing, and we will work hard to ensure they do.

As a community, we will need to show patience, resilience and determination throughout if we are to help get the right outcome for people with cystic fibrosis on this issue and other potential therapies further down the line targeting other mutations.

And, most of all, we will need unity. By sticking together and making our voice heard as one we can change the world of cystic fibrosis. We have done it before and we can do it again.