2. Will the Cabinet Secretary provide an update on discussions the Welsh Government has had with Vertex Pharmaceuticals about the cystic fibrosis drug Orkambi? OAQ52768

Thank you for the question. Neither I, nor my officials, have had any formal discussions with Vertex about Orkambi. I have, though, reminded Vertex that a medicine must be appraised and recommended by the National Institute for Health and Care Excellence or the All Wales Medicines Strategy Group before it can be routinely provided by NHS Wales. It is Vertex's responsibility to comply with the appraisal process, which they are both aware of and fully understand. 

It is regrettable, Cabinet Secretary, that you haven't had direct conversations with them, although you do point out that you had some discussion about their responsibilities. In other parts of the United Kingdom, this drug is available for cystic fibrosis sufferers and, regrettably, it's not available here in Wales, as I understand it. Why has there been so little progress in making that drug available here in Wales? As I understand it, other health departments have come to arrangements with the pharmaceutical company to make it available. Will you commit to working with the pharmaceutical company to make this drug available here in Wales, because the benefits have been proven and are clear to cystic fibrosis sufferers? Sadly, cystic fibrosis is a condition that is lifelong— it's not something that comes to you as you progress through life—and, so, therefore, the better the treatment we can offer, the longer the life, and the quality of life is improved for the individual. So, I'm asking you: please will you commit, as the Minister and the department, to engage positively to make this drug available here in Wales? 

I must confess that I'm regularly frustrated at the manner in which the discussion around Orkambi has taken place. At the centre of all this, we should remember, are people, families, who want to have the best possible treatment available on our national health service. Every single company that manufactures a medicine available for the NHS understands the process they must go through, and that process is to make sure that medicines are safe and effective and that there is an appropriate value for that treatment. This treatment—Orkambi—is not readily available on the national health service in any other UK nation. That is simply not the case. It is potentially—. It is not available in other nations within the United Kingdom, nor is it available in Wales. It is available on individual patient funding requests, and it has been made available on a trial basis by the manufacturer on a limited basis for people in each of the different UK nations. So, it is simply wrong to suggest that NHS Wales has taken a different view. That is part of my frustration, because I accept that Members in every party have had correspondence from the company, and I believe I've had correspondence from Members in each and every party in this Chamber who have written to me about the issue. And I am frustrated about the lack of clarity in the process. Because I do not believe that Members are recounting matters—they have not been told, and accepted in good faith.

The benefits are not proven—that is one of our biggest problems in this area. Orkambi did not simply fail a NICE appraisal on cost grounds, but NICE, on the information available to it on clinical data provided by Vertex, put it onto the 'do not use' list. Now, that is highly unusual, and that is because of the state of the clinical data available at the time. I have said, more than once, directly in correspondence to Orkambi, and, indeed, when I got doorstepped above this place by the company and their representative, that they need to engage in the process that they understand. And that is part of my frustration. I do not believe that you are having the full story painted to you. In the Petitions Committee, when they considered this matter—[Interruption.] In the Petitions Committee, when this matter was raised, it was stated that, since the NICE appraisal, there are now 96 weeks of additional clinical data available. NICE and the All Wales Medicines Strategy Group cannot assess that information, because it has not been provided to them. Once that information is provided to NICE, then a decision will be made. But it is the company's responsibility, and nobody else's, to provide that data and information, because they have access to it and no appraisal body does. I'm more than happy to talk to Members outside here, but my frustration is that I do not believe that the full picture is being provided to you about the manner in which this drug is and is not available and responsibility for that.

I too have had discussions with Vertex, and I've made representations to you about a particular constituent of mine, who I know is very concerned that her daughter, who's only about seven years old, can have access to this drug. But, in my discussions with Vertex, I've been, like you, hugely frustrated—they seem to understand what the process is, but, for whatever reason, are not engaging in the way that they know that they have to. But my question to you is: if those proper processes were followed by Vertex, and Orkambi was to meet all the tests of both NICE and the All Wales Medicines Strategy Group, would it then be eligible under the new treatment fund?

Yes, and I think that's quite helpful, because, whilst I am frustrated that there hasn't been a meaningful engagement in the process, if they did, this is exactly what the new treatment fund is for. So, when new treatments become available that are NICE approved—so, they are proven to be effective and safe and cost-effective—there is often a challenge in getting those drugs in an equitable manner into our system. It is exactly what the new treatment fund is designed for, and I can say, yes, this would be eligible for support within the new treatment fund to make it available on a rapid and consistent basis. And, as we know, that means that, here in Wales, effective medication, with an evidence base behind it, is available more quickly here than in any other UK nation, and I am very proud of that.

I think if it's the case that people are not informed, then that's why people are raising it here, so that they can get information from you as Cabinet Secretary, and I think that that would be something that we should be mindful of here. I understand, from speaking with Vertex, that there was some frustration in dealing with all-Wales medical procurement, which resulted in significant delays in this process. Perhaps it was an isolated incident—and I hope it was—but it's something that should be looked into in relation to this particular case. Could I ask you whether there are any plans to amend processes to permit the assessment of a portfolio of medicines, showing the flexibility needed for these types of medicines for rare diseases? Now, we've been here before with Kalydeco, and had a change of mind from the Minister at the time. A portfolio of types of medicines for rare diseases would be something that would be welcomed for you to look at, at least, and I'm sure not only Vertex would welcome that, but us as directly elected representatives also.

I accept completely that Members in every party are acting in good faith in asking questions around this. I completely accept that. There has been engagement between our appraisal process on a number of occasions, which has not resulted in the additional clinical data being provided. The procurement question is interesting. But you get to that point once you have gone through the appraisal process. Because, if the manufacturer cannot demonstrate that the medicine on offer is effective and safe, then it doesn't really matter what the price is. If Vertex offered something for £1 a go—well, if you can't demonstrate that it's effective, then it isn't going to get through the gate. The challenge about procurement and a possible portfolio approach becomes interesting once the clinical data is available, and that would then potentially allow us to take a different and a broader view, in exactly the same way that a number of drug manufacturers already do; they call them 'patient access schemes'. So, yes, I am open and our system here in Wales—as indeed is every other UK nation—is open to a sensible conversation about cost once Vertex comply with the process that they, and every other drug manufacturer, understand and they make available that clinical data for a proper and independent appraisal.