NICE Appraisals and Rare Diseases

I was pleased to see that on the 21st March 2019 a debate took place in the House of Commons on NICE Appraisals and Rare Diseases. Members from across the House raised concerns regarding the availability of treatments to those with rare diseases, including Batten Disease. I also understand that Batten Disease has been raised separately in the House and the Secretary of State of Health will be meeting with members to discuss the issue further.

You can read the full debate here, I have included the Minister’s response at the end of the debate below.

The Parliamentary Under-Secretary of State for Health and Social Care (Steve Brine)
Let me add my congratulations to the hon. Member for Blaydon (Liz Twist) on securing the debate. I was not familiar with the north-east massive, although I am now. I was familiar with the individual component parts of the north-east massive, especially my shadow, the hon. Member for Washington and Sunderland West (Mrs Hodgson), but I have not seen them as a collective before. I must say that they hunt well as a pack.

I know of the work of the hon. Member for Blaydon, who chairs the all-party parliamentary group on phenylketonuria. We have discussed these matters before, in Westminster Hall, and I do not doubt that we will discuss them again.

Other Members have spoken passionately today, on behalf of their constituents, about the importance of access to new medicines. As the hon. Member for Dudley North (Ian Austin) rightly said, Members are doing their job, and I am doing mine: as requested, I have listened very carefully to the debate. I thank my hon. Friend the Member for Reigate (Crispin Blunt), and the hon. Members for Wolverhampton South West (Eleanor Smith), for Bristol East (Kerry McCarthy), for West Ham (Lyn Brown), for Liverpool, West Derby (Stephen Twigg), for Heywood and Middleton (Liz McInnes), for Dudley North, for South Shields (Mrs Lewell-Buck)—part of #massive—for Strangford (Jim Shannon) and for Motherwell and Wishaw (Marion Fellows), who speaks for the Scottish National party.

The hon. Member for Liverpool, West Derby touched on the subject of cannabis, and I will happily write to him about that in more detail if he wants. As he knows, the all-party group report came out yesterday. I have not had a chance to look at it yet; I am aware of it, obviously. The Government changed the law and specialist doctors can now provide cannabis-based medical products where there is clinical benefit. To support doctors in this—because the politicians are ahead of the clinicians on this one—we asked NICE to develop new clinical guidelines and we asked Health Education England to provide additional training, while encouraging more national research to develop the evidence base in this. I have said before and I shall say again that I am very clear that we need to provide more support and encouragement to commissioners in this space because, as I said, politicians are ahead of the clinicians on this one.

Everyone has spoken incredibly passionately and there has been very little politics in the debate, which has been excellent, at the end of this week. It is very good to hear Parliament discussing something else, which of course it does all the time; it is just that that is never reported— I dare say this will not be, either.

The Government share the view of everyone in the House that it is in the interests of all NHS patients that we have the best system in place for making evidence-based decisions on whether new medicines are routinely available. Of course it is easier when one is on the Back Benches to just say the system is hopeless, but we have to work ​with the system we have, or change it. We inherited the NICE set-up. It was mentioned in the first Labour Queen’s Speech, in ’97. It was created in 1999 in a Delegated Legislation Committee by then Health Minister of State John Denham, who is a good man. He set it up. When he did so, he said it was set up to make independent, evidence-based recommendations for the NHS on whether drugs represent an effective use of NHS resources.

NICE is widely recognised as a world leader in the field of health technology assessments. Its methods and processes have been developed and continuously refined over the last 20 years through periodic review and engagement with stakeholders. Politicians are not in the middle of those decisions, and rightly so; that is how the system was set up by the aforementioned Mr Denham. Maybe the hon. Member for Heywood and Middleton was right to trust her instincts—

Lyn Brown
Will the Minister give way?

Steve Brine
No, because the hon. Lady has had her say and I am going to have mine in the short time I have. If I have some time—

Lyn Brown
It is on behalf of my constituents.

Steve Brine
Mr Deputy Speaker, I think the hon. Lady has spoken on behalf of her constituents a lot today, and very well. [Interruption.] I do not think, given the tone of this debate, we need to get unpleasant in here. [Interruption.] I will address the points in my speech and, if the hon. Lady does not like that, I am sorry, but I do not think anybody watching this, least of all the families affected, need to hear that tone.

As a result—

Lyn Brown
rose—

Mr Deputy Speaker (Sir Lindsay Hoyle)
Order. Whether the Minister wishes to give way is up to the Minister and we must let him finish his speech.

Steve Brine
Indeed, Mr Deputy Speaker; some people just cannot help themselves.

NICE operates two separate programmes for the assessment of new medicines. First, there is a technology appraisal programme through which NICE assesses the vast majority of new medicines. Secondly, as has been said, there is a highly specialised technologies, or HST, programme that is reserved for the evaluation of very high-cost drugs for the treatment of the very small number of patients in England treated in a handful of centres in the NHS. Decisions on whether a medicine should be routed to NICE’s mainstream technology appraisal, or the HST programme are taken through an established topic selection process that includes consideration against published criteria and engagement with a wide range of stakeholders. When NICE recommends a drug for use through either route, NHS organisations are legally required to provide funding so that it is made available to patients.

Today, we have heard concerns that NICE’s technology appraisal programme is not suited to the assessment of medicines for rare diseases, with some calls for individual drugs to be assessed through the HST programme instead. We have also heard calls for a third appraisal route for rare diseases not eligible for the HST programme. I have listened very carefully to all of them and will ​reflect on them all. There is some sense in a lot of what has been said. Indeed, over the last 20 years, NICE has made positive recommendations in 75% of its appraisals of orphan medicines. By comparison, NICE recommends around 80% of medicines for more common diseases.

I shall give the House two recent examples, because of course we only ever hear about the examples that are stuck or refused. NICE has been able to recommend orphan medicines for neuroblastoma, a cancer of the nerve cells that affects children—this has been widely welcomed—and for primary biliary cirrhosis, a progressive liver disease. Moreover, through its HST programme, NICE has to date been able to recommend a further eight medicines for NHS patients outside of the standard appraisal route. In each case, NICE’s recommendation is subject to a managed access agreement negotiated between the drug company and NHS England.

There will always be cases where NICE is unable to recommend a medicine because the price set by the company does not reflect the benefits that it brings. That is a fact. Hon. Members have of course spoken about the rare diseases of people in their constituencies—they are doing their job—but NICE is an independent body and it should be allowed to develop its guidance free from politicians. The hon. Member for Heywood and Middleton said that that was her initial instinct before she became a politician. That is the foundation of NICE’s reputation as a world leader in its field, and it is in the best interests of patients that it does that.

The hon. Member for Blaydon, in introducing the debate, raised concerns about Kuvan, the treatment for phenylketonuria. NICE has initiated an appraisal of Kuvan, and officials from NICE, NHS England and our Department have been reconsidering the appropriate assessment route in the light of the new available information that the hon. Lady mentioned. Riley is right: we have to make this fair. I am told that a final decision will be taken promptly—I urge that again from the Dispatch Box today—and with the minimum impact on the timescale for NICE’s assessment.

The hon. Members for Strangford, for Bristol East and for Dudley North have all spoken about the issue of Orkambi so many times and so well. It is incredibly frustrating and disappointing to Ministers, just as it is to them and everyone else, that Orkambi is not available to NHS patients in England at the moment. I understand and share that frustration. This is why my right hon. Friend the Secretary of State held a meeting with Vertex, NHS England and NICE a couple of weeks ago. I was at that meeting, at which the parties again discussed how best to reach a conclusion. I am pleased to say that they are meeting again today to continue the discussions and decide on the next steps. Decisions about the availability of drugs in Scotland are of course a devolved matter, and that is up to Scotland. I understand that no decision has been taken on routine funding for NHS patients in Scotland, but the hon. Member for Motherwell and Wishaw asked me to look again at the Scottish system. I will do that and I will ask the Minister responsible for this policy area to do so.​
The hon. Member for North Tyneside and others raised the issue of the drug Spinraza for the treatment of spinal muscular atrophy. I understand that NICE’s independent appraisal committee met earlier this month to consider its recommendation on Spinraza following new evidence being put forward by the company. NICE wrote to the company and patient groups last week to say that it was not yet able to provide an update on the outcome of the meeting, but that it would provide an update soon. Again, I encourage that to happen even sooner. I recognise that the protracted process in this instance is hugely frustrating for patients and their families and, whatever our differences across the Dispatch Box, of course I feel the deep hurt that the hon. Ladies who spoke on the subject have laid out. I hope they will appreciate that a final decision has not yet been made and that NICE must be allowed to complete its work free from political interference.

I do not have time to go into a huge amount of detail. I have been asked lots of questions during the debate, but I have little more time than the people who have spoken today. I thank Members for speaking so passionately and I hope that they will welcome the forthcoming review of NICE’s methods and processes over the course of this year for both its technology appraisal and its highly specialised technologies programme, which is at least partly what today’s motion calls for. It would not be appropriate to pre-empt the review by commenting in detail on what it should look at, but I will ensure that it is directed towards the motion before us today and to the transcript of today’s deliberations. I now want to give time to the hon. Member for Blaydon, who introduced the debate, to close it in the time that we have left.