NICE Appraisals: Rare Diseases Treatments
(Limited Text - Ministerial Extracts only)
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Mrs Sharon Hodgson (Washington and Sunderland West) (Lab)
It is a real pleasure to take part in this very important debate. I start by thanking my hon. Friends the Members for Blaydon (Liz Twist) and for North Tyneside (Mary Glindon). We are part of the north-east massive, so I am very pleased to be joining them in this debate today. I thank them for their very passionate and heartfelt contributions. I should also mention, as part of the north-east massive—it goes without saying—my hon. Friend the Member for South Shields (Mrs Lewell-Buck). I thank all other hon. Members who spoke for their excellent contributions. They know who they are and I do not need to name them. They were all fantastic.
Throughout the debate we have heard of the heartbreak experienced by patients and their families when they are unable to access life-saving drugs on the NHS. We have heard of their determination to continue fighting to access those drugs, whether by writing to their MP or even by protesting in Parliament square. I was happy to join my hon. Friend the Member for Bristol East (Kerry McCarthy) there just two weeks ago for the Cystic Fibrosis Trust rally, where people were calling, “Orkambi now!”—they were so loud that we could hear them over the crowds chanting, “No Brexit!” or whatever the shout was at the time.
We must hear patients’ voices in this debate, as it is they and their families who are affected the most by the appraisal process, which is not fit for purpose. The Minister has heard about the real-life experiences of patients throughout this debate, and I am sure that he will continue to listen to them afterwards. I know that he is also in regular communication with patients. In my role as shadow Public Health Minister, I regularly meet patient groups and campaigners, so I know just how important access to these life-saving drugs is to them.
As a constituency MP, I recently met young Riley and his mum Michelle. Riley has phenylketonuria—PKU—and needs Kuvan. He is now 11 and at secondary school. He just wants to blend in with his mates and to be able to go on those first excursions out to the Metrocentre, and perhaps to get something unhealthy to eat from a takeaway, but obviously he cannot do any of that. I asked him about his life and how he felt not having access to Kuvan. He said that it was not fair and that it made him mad. Well, I agree with Riley.
It can take years to get the right diagnosis for a rare disease, so once patients get the diagnosis they are excited and feel that there has been a breakthrough, because they think that they will finally get the treatment they need and deserve. Instead, as we have heard today, they are back at the beginning of the fight, because the life-saving drugs that do exist are not available to them on the NHS. It is one hurdle after another for patients with rare diseases. That is why the Opposition strongly believe that patients should have fast access to the most effective new drugs and treatments. I am therefore pleased to support the motion.
As we have heard, a rare disease is generally considered to be one that affects fewer than five people in 10,000. According to the 2013 UK strategy for rare diseases, it is estimated that in the UK more than 3 million people will suffer from a rare disease at some point in their life. All those patients must have access to the drugs and treatments that they need. However, they are being failed by the NICE appraisal process, which is just not fit for purpose when it comes to assessing the suitability of drugs and treatments for rare diseases.
Patients with rare diseases are squeezed in the middle of two appraisal routes: the highly specialised technology evaluation programme and the single technology appraisal route. The HST evaluation programme is selected for most non-cancer rare disease medicines and is designed for evaluating medicines of that nature, with small patient populations. However, the HST evaluation programme currently lacks the capacity or capability to effectively appraise all new licensed orphan medicines. Since the HST evaluation programme was established in 2013, it has published guidance on eight medicines, which is much fewer than the 45 orphan medicines for non-cancer indications that have been licensed in the same period.
The STA route is designed to appraise treatments for more common conditions and those with existing treatments. This route is poorly suited to considering rare disease medicines, which tend to have small patient populations, a limited evidence base and benefits beyond direct health benefits—something the appraisal process just does not take into account. Some rare diseases are not rare enough for the STA route, and only a handful of medicines are being approved by the HST route. Yes, it is complicated, but it is clear that neither route is working for patients with rare diseases, so patients are missing out on crucial medicines.
Kuvan was licensed in 2008 to treat PKU patients, but it is still not available to patients in England. Orkambi was appraised by NICE in 2016 through STA, but was recommended for use. Three years later, as we have heard, people with cystic fibrosis still have no access to it. That has caused physical and psychological harm to patients and their families. Every day without the drugs that they need makes their condition worse. We must have an appraisal process that captures rare diseases effectively.
Medicines to treat rare diseases are often found to be cost-ineffective, which is why they are not approved for routine commissioning. However, establishing value for money is not straightforward, especially when population groups are small. It does not sit comfortably with me—or, I am sure, with any of us—that cost-effectiveness is prioritised above clinical need, or, as we have heard, the lives of children. Manufacturers want to make a reasonable return on their investment, although some of the figures are huge, but I do not think that that should be a priority. Manufacturers must not hold NICE or NHS England to ransom for their own financial gain.
The Parliamentary Under-Secretary of State for Health and Social Care (Steve Brine)
Or the Government.
Mrs Hodgson
Exactly.
Behind profit forecasts are thousands of people and families who need access to life-saving drugs, and they simply cannot wait any longer. We must not put businesses before patients. Because of the NICE appraisal process, patients and their families are being left in an awful limbo. The processes can be long-winded, confusing and difficult to navigate. Some medicines can undergo multiple assessments while others are not assessed at all, and that creates an unpredictable and unattractive system. As a result, patients are left in the dark about when, or if, they will have access to innovative treatments for their conditions.
When a drug is being appraised, patients live in hope that this time it will be approved for use by NICE—as in Maryam’s case, which was described so powerfully by my hon. Friend the Member for West Ham (Lyn Brown)—but they are almost always left to feel disappointed and helpless. Patients and their families must be involved in the processes, and the processes must be transparent.
The wait for access to drugs is excruciating, especially when the drug is available in nearby countries, or even—as we have heard—in Scotland. Spinraza is available to patients in Scotland, but not to those in England. My hon. Friend the Member for West Ham spoke passionately on behalf of her constituents and their seven-month-old baby Maryam. This sounds blunt, but she is dying, because she has been denied access to medication that could extend and enhance her life.
The pain and anguish that the parents of a critically ill child must feel when they are told that there is medicine available that will help but it is not available for their child are unimaginable. Knowing that if your child lived a few hundred miles away, in Scotland or perhaps somewhere in Europe, the drug would be available is heartbreaking and infuriating. Patients in England should not be left behind. We should be working to find ways to get these medicines to the patients who need them, on the NHS.
I hope that the Minister will consider the motion seriously, for the sake of patients with rare diseases and their families. They cannot be left behind any longer: they must have access to these life-saving drugs now.
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—
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—
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—
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.
Liz Twist
I hope that hon. Members will forgive me if I do not list them all, but I want to respond to the Minister. First of all, Minister, it is not easy for us on the Back Benches to criticise and lobby. It is really difficult for us, because these are our constituents. They are real people who have real conditions. We have tried to be constructive in the debate and said that we have identified issues around the NICE appraisal system that we think need to be addressed urgently. I notice that he mentioned the review and said that our concerns would be drawn to its attention, but will he ensure that there is dialogue and input before the review takes place to ensure that it is not just technical, but addresses the concerns that we have raised?