Accelerated Access Review

David Mowat Excerpts

Tuesday 13th December 2016 Westminster Hall

[The Parliamentary Under-Secretary of State for Health (David Mowat)]

It is good to serve under your chairmanship today, Sir Alan. I congratulate the hon. Member for Dudley North (Ian Austin) on leading the charge in this debate. The right hon. Member for Leigh (Andy Burnham) rightly said in his very good speech that this is not really a political issue. Every Member in this Chamber has constituents who would benefit from these drugs. There are 10,500 people in the country with cystic fibrosis and it is massively important that we do everything we can to make progress on the issue. I also congratulate the Cystic Fibrosis Trust on its work and on its “Stopping the Clock” campaign. Debates such as this give prominence to these issues and to the need to make progress.

The debate is really about two drugs, a drugs company and an evaluation process. I shall speak about all of those and about where we are going with the accelerated access review. The two drugs are Kalydeco, which applies to something like 4% of cystic fibrosis sufferers, and Orkambi, which would apply to a further 40% of sufferers. Both are relatively small populations: for Kalydeco it is something like 400 people in England, and for Orkambi it is something like 2,700 or 3,000. Kalydeco has been routinely available on the NHS since 2013. As mentioned today, it was extended on 4 December to children aged two to five. It makes a big difference and we are pleased to have made that progress. Both Kalydeco and Orkambi are produced and owned by a Boston-based drugs company called Vertex, which I shall talk about later.

Orkambi could be used by around 3,000 patients. It has a price of something like £100,000 per annum—the implication being that the cost of its approval in England would be in the order of £300 million or £400 million a year. As several Members have said, it is obviously right that there is a process that weighs that cost of £300 million to £400 million a year against other NHS priorities and other drugs. That process is the NICE process. A number of comments have been made about the efficacy of that process, and it has been suggested that it may have deficiencies in respect of providing precision drugs to small numbers of users. I will try to address those concerns. I think everybody agrees that we need a consistent method of evaluating these matters, and there needs to be a way forward based on that.

When NICE evaluated Orkambi in July, it found that it had clinically significant and important benefits, which several Members have spoken about. There is no dispute about that, but the evaluation process—which is based on quality-adjusted life years, as has been said—also found that it was not cost-effective. I spent some time last night reading the NICE evaluation, and make the point to colleagues and other Members that it was not a near miss. It looks like there is a factor of 10 in NICE’s evaluation of its cost-effectiveness. I guess that is largely driven by the price of £100,000 per annum and what that would mean.

[Andy Burnham]

It is obviously reassuring to everybody that the Minister has taken such a close interest in the issue before coming to the debate. He says it was not a near miss. That may have been the case on the data that NICE had, but does he accept the point made by my hon. Friend the Member for Dudley North (Ian Austin) and other Opposition Members that those data were very limited indeed? The 96-week trial data that are now available would probably have produced a very different overall calculation.

[David Mowat]

To be honest, I am not qualified to have an opinion on that. The right hon. Gentleman rightly said that decisions of this sort should not be made by politicians and that there has to be a process around them. It is clear that if NICE is presented by Vertex with new clinical data, or indeed new price data—this is perhaps equally relevant, but we have not really discussed it—a review could be carried out quickly without any need for us to go through the whole process again. There is a precedent for that, and if those data exist and Vertex presents them, they would be looked at. I give my commitment, and certainly that of the Minister responsible for this policy area, that that would be the case and there is no impediment to that. I do not want to raise false hopes by saying that, and I do not think I have done so. The fact that it is not a near miss—it is possibly out by a factor of eight or 10—implies that there is quite a lot of work to do on pricing.

It is worth recapping what other countries have done. Orkambi is available in Germany, although it appears from the data available that its use there is quite mixed, with perhaps no more than one in five eligible people having access to it. In France, the other country in Europe that has authorised it, Vertex has booked no sales yet this year. The picture seems quite mixed in those countries. The countries that have not authorised Orkambi include Scotland.

[Jim Shannon]

In my speech I mentioned a young girl from my constituency, Rachel, who has been on the Orkambi trials and shown exemplary improvements in her health. That is an example we can all point to of where goodness has come out of the drug for those who have had the opportunity to have it, and that is true not only in my constituency but throughout the whole of the United Kingdom of Great Britain and Northern Ireland.

[David Mowat]

There is no dispute that the drug works, and there is no dispute at all that it is life-changing. The issue before us is the extent to which it justifies a price tag of £300 million to £400 million versus other NHS priorities. All I can say on that is that it is right that the decision is not made by politicians, for the reasons given earlier by the right hon. Member for Leigh.

I was discussing the countries that have so far not authorised Orkambi. Neither Scotland nor the Republic of Ireland accepted that it was cost-effective, and it is not used in Scandinavia or Canada either.

[Mark Durkan]

The Minister mentioned Scotland and the Republic of Ireland, where there are clearly challenges—we only have to look at the pictures in The Irish Times yesterday to see the graffiti about Orkambi in Dublin. Will he commit to working with colleagues from across these islands to use the underdeveloped and underused machinery of the British-Irish Council to literally get our act together when it comes to rare diseases? We should combine our purchasing power when negotiating with the drugs companies and ensure that there are much better networks for referral and treatment. We should improve that collaboration and literally get our act together on these islands.

[David Mowat]

I, too, saw the press. I think the Republic of Ireland drugs Minister has talked about writing around to that effect, and it would be a great idea were we to use our combined procurement muscle in that regard. He is certainly pressing at an open door.

I wish to spend a little time talking about Vertex. The company owns the drugs we are discussing and is worth $18 billion. As well as looking at the NICE review last night, I spent quite a lot of time looking at Vertex’s financial position. The company needs to sell these products; indeed, its continued functioning as a major pharmaceuticals company depends on that. Its share price has fallen by a third during the course of this year—I estimate that is a loss of value of something like $7 billion—because its sales are not adequate. There needs to be a meeting of minds here. I am sure that people from Vertex are listening to this debate, as will people from other places, too. We all want a solution whereby the drug becomes available at a cost-effective price, but the negotiation is not a one-way street; Vertex is part of it as well. Were the company to come forward with different pricing data, those would be looked at very quickly. At some point in the future—I know it will be a long time—the drug will be available generically, although I accept that that will not give hope to some of the people we have heard about today.

In the couple of minutes I have remaining, I wish to discuss the accelerated access review, which was a manifesto commitment we made at the election. We set up the review panel. The basic intent was to enable transformative drugs to come forward more quickly and for there to be, as Members have mentioned, a commercial unit in the NHS that is empowered to do deals and bring treatments forward more rapidly. In October the review team and panel published the final report, to which something like 600 stakeholders contributed. It is a valuable piece of work and we know its direction of travel: bringing drugs into the system more quickly, allowing the NHS to set priorities for the drugs its wants, and giving drugs companies some notice and knowledge that if they develop drugs, they will be used. That will mean that a lot of the commercial discussions can happen earlier and progress can be made more quickly.

The Government are reviewing the results of the accelerated access review. There is much in it, if not all of it, that will be accepted, although I am not in the position to accept it today—that is not my role here. We do, though, want to make progress, which should give some hope for the potential of another review of the matters we have been discussing. Nevertheless, I must say again to Government and Opposition Members that the NICE process and the people carrying it out—they are rigorous scientists and serious doctors—need to be treated and understood with respect. We can all agree that the current situation is heartbreaking for many people. The world has a drug that would change people’s lives, but the world has not rolled that drug out to them because of real and reasonable financial issues. I accept that that is a very difficult thing to explain to people and it is very difficult to accept.

Motion lapsed (Standing Order No. 10(6)).