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NICE Appraisals: Rare Diseases Treatments

Volume 656: debated on Thursday 21 March 2019

I beg to move,

That this House believes that NICE appraisal processes do not properly address the medical treatment needs of people with rare diseases such as muscular dystrophy, phenylketonuria and cystic fibrosis; and calls on NICE to urgently review the appraisal process.

I thank my hon. Friend the Member for North Tyneside (Mary Glindon), who is chair of the all-party group on muscular dystrophy, and the hon. Member for Strangford (Jim Shannon), who is chair of the all-party group on cystic fibrosis, for their help in securing this debate.

My constituent Archie McGovern is 12 years old. He is bright and lively, and full of beans now, but it has not always been like that way because Archie has PKU— phenylketonuria. Putting it simply, PKU is a genetic condition that means Archie and others are unable to handle phenylalanine, which is found in protein—so no meat, no fish and no dairy products. There is a whole range of other things that we would not think had protein in them: the list is endless. On top of that, he has to take a protein substitute drink—if we can call it that, as it is very unpleasant—to keep the balance right.

At present PKU is not curable, and a hugely restrictive diet is the only way of controlling the condition throughout childhood and adult life. The condition is picked up by the pinprick test at birth, and for those identified as having PKU that is the start of a difficult lifetime of dietary control. For children that is especially difficult, but it is also very important because failure to control the condition can lead to serious neurological problems.

That is how it was for Archie until quite recently, but there is a treatment that can help to control PKU. It is called Kuvan, and although it was licensed 10 years ago and is widely available in many countries in Europe, and further afield, it is not available to patients in the UK. Not everyone with PKU responds to Kuvan, but it is believed that more than 20% of people will respond well and see a significant improvement in their life.

The hon. Lady makes an important point about Kuvan and PKU. As she will know, last year I and other Members—including the hon. Member for Dudley North (Ian Austin)—took part in the MPs’ PKU diet challenge, so I am well aware of the restricted diet that is required for those who suffer from that condition. The way that NICE evaluates Kuvan does not take into account the social costs associated with the alternative treatment for PKU. That highly restrictive diet impacts on families and wider society, and NICE should consider that point.

Like the hon. Gentleman, I took part in the PKU diet for a day challenge. It was indeed very restrictive, even though I knew it was only for one day.

Archie is one of those who responds well to Kuvan, and last year, his parents took the difficult decision to pay to buy him the drug. That took a great deal of soul searching on their behalf, and it cost them dear—£25,000 a year, even though Archie is currently on just half a dose. They know that for many people with PKU, or for parents with more than one child who has PKU, it is simply not possible to self-fund, and they are acutely conscious of the unfairness of that. The difference that Kuvan has made to Archie is real and significant: increased concentration and energy, so that he can make the best of his education; no recurring mouth ulcers, which were a real problem; and a chance substantially to increase the number of exchanges he can have, and eat a more normal diet. For Archie, Kuvan has made a real difference.

Archie’s case, and those of many others in other constituencies, prompted us to form the all-party group on phenylketonuria, and to consider how Kuvan could be made available to those who would benefit from it. Nearly 10 years after Kuvan was approved, that treatment does not seem to have been an appraised, and in England it is still not available on the NHS. Since we set up the group, there has been a move for Kuvan to be appraised by NICE, and discussions have been held with NHS England about a managed access agreement. We were disappointed to learn just before Christmas that no agreement had been reached on that managed access agreement, and that the NICE appraisal was to be via the single technology appraisal route, and not the highly specialised technologies programme. I understand that following a legal challenge, the Department of Health and Social Care is again considering the appropriate appraisal route, and the all-party group has made representations on that point.

When talking about the NICE appraisal system it is easy to get lost in technical details—QALYs, and everything else, that means nothing to people on the street—but what really concerns people is whether or not there is a fair chance that the drugs they need will be fairly assessed and made available on the NHS.

I thank my hon. Friend for securing this debate. One of my constituents suffers from Batten disease and is receiving cerliponase alfa on compassionate grounds. However, NICE will not now recommend that treatment, which in part is due to cost. Does my hon. Friend share my view that allowing patients on to clinical trials when there is no funding to deliver the treatment, places them and their families in an incredibly difficult and uncertain position regarding their future?

I certainly agree, and such things happen too often. We need a system that properly evaluates those drugs.

I have certainly found that NICE sometimes says that the pool of people is not big enough to evaluate, but the clue is in the title: these are rare diseases. NICE cannot carry on doing that, particularly in cases where it is clear that the drug has a really positive effect.

I agree with my right hon. Friend. Indeed, that is the burden of my speech.

As I was saying, I am not going to get into the fine detail of the process. It seemed to us in the all-party group that many conditions, as my hon. Friends have said, face the same problems. The all-party groups for muscular dystrophy and for cystic fibrosis are two that come to mind, but there are many other rare diseases, as we have heard, that do not have all-party groups but face exactly the same difficulties. Since this debate was announced, some other organisations have contacted me to ask me to make sure that we do not forget their concerns.

We found that there are a number of aspects of the NICE appraisal system that are problematic in assessing Kuvan and many other rare drugs. The existence of just two appraisal routes for treatments to be assessed by NICE results in the likelihood of two or more treatments being stuck in the middle by not meeting the restrictive criteria of the highly specialised technology route and therefore being assessed under a single technology appraisal route. Some of them are rare but not rare enough. As we have heard, the majority of treatments for rare diseases are likely to be assessed within the single technology appraisal, which is designed for non-rare treatments. This impacts on both the cost threshold and the approach to evidence, which are all designed for more common diseases.

On lifelong chronic conditions, NICE’s approach values the lifelong cost of treatments. It looks for near future benefits as well. That means it is difficult for chronic diseases such as PKU and treatments that produce lifetime and life-enhancing effects to get access to new treatments. NICE cost appraisals assume that patents do not expire. NICE will assume the existing price of the drug will stay the same. That is illogical as, particularly with older drugs such as Kuvan, the drug will soon go off-patent. This affects the benefits assessment. On non-health costs, NICE performs its calculations based on costs paid and saved by the NHS. That ignores the wider cost to society and individuals caused by diseases like PKU.

I do not seek to criticise NICE staff. They work within a system that we have given them, but it is clear from the many questions to Ministers, debates in this Chamber and in Westminster Hall, the creation of all-party groups, and correspondence with Ministers about individual cases that there is a very real issue here which must be addressed. That is why we are asking, in this motion, for NICE to review its processes to reflect the current issues we face.

Many drug companies have been in touch since this debate was granted to send me briefings. They have been keen to explain their side of the argument and to point out what they see as the problems in the NICE appraisal process for their drugs. There is some overlap with patient concerns, but I am here today to speak on behalf of the community of people with rare diseases, not on behalf of the drug companies. Let me be clear, the fact that this debate is about NICE appraisal processes does not excuse the pharmaceutical companies from their responsibilities. There is a balance to be struck between their need to recover the cost of the development of drugs and make a reasonable profit, and a huge responsibility on them to make their drugs affordable for our NHS.

In this debate, I have focused on PKU and Kuvan. With another drug treatment for PKU on the horizon, Pegvaliase, there is a real worry that even with a drug that may produce really life-changing results for a wider group of patients, those with PKU will again be left without the treatment they need, even when it exists. There are treatments for other rare diseases, too: Spinraza for spinal muscular atrophy and Orkambi for cystic fibrosis, which are not only life-improving but life-extending.

This is quite simple. There are drugs available that can drastically improve the lives of those affected by rare diseases. When I hear that NICE’s appraisal process is an obstacle to improving lives, I feel really angry. We are reducing the lives of children and adults to a cost-effectiveness analysis. We need to find a way forward to amend the appraisal system so that we do not let people fall through the cracks or fall behind. The drug companies must also do their bit to ensure that their drugs are affordable for the NHS, especially when early access via a managed access agreement is being discussed.

That is why today we are calling on NICE to review its appraisal processes and make the necessary changes to stop people falling through the cracks and make available these drugs, which can make such a difference to patients—to people such as my constituent Archie McGovern, whose mum Barbara set me on this path as a new MP.

I am delighted to support the motion standing in the names of the hon. Members for Blaydon (Liz Twist) and for North Tyneside (Mary Glindon). This is a life-changing issue for thousands of our fellow citizens who simply do not have time on their side. A number of my constituents, including the parents of my young constituents battling with cystic fibrosis, have brought to my attention the weaknesses in the current NICE appraisal model. I look forward to reinforcing the arguments of the hon. Member for Blaydon with some details that they have provided.

NICE’s appraisal model has led to a horrendous block on life-changing cystic fibrosis drugs being made available to those young people. Vertex’s three approved medicines and investigational triple regimen may be able to treat the underlying cause of cystic fibrosis for up to 90% of patients. There is currently no cure for cystic fibrosis, and half of people with the disease will die before they are 31. I recommend that my hon. Friend the Minister instructs NICE to review its current single technology appraisal, which is used to appraise inherited rare diseases, in order to come to a solution that can work best for all parties. The current model, which specifically affects Vertex drugs such as Orkambi, is fundamentally flawed. It directly affects the lives of not only my young constituents suffering from cystic fibrosis, but those with spinal muscular atrophy, Batten disease and PKU, to name but a few.

NICE’s single technology appraisal has been used for the past 20 years, and although it served as an important new way to assess the cost-effectiveness of new treatments, it has failed to keep pace with advances in science. No model should be biased towards favouring specific medicines, but there remains an unwillingness to accept that new precision medicines that treat the underlying cause of disease and have the potential to extend life are fundamentally different from the medicines that existed when NICE’s processes were first developed. The idea of working on an innovative new model for appraising rare diseases is also supported by the Genetic Alliance.

When performing a single technology appraisal, NICE applies the same methodology and cost-effectiveness criteria regardless of whether it is appraising a single-use medicine for an acute condition or a lifetime medicine for an inherited, progressive, incurable, life-limiting disease. The current evaluation process turns on the incremental cost-efficiency ratio, measured in quality-adjusted life years. With acute conditions resulting from shorter-term treatment, the ICER is moderated even if the drug is very costly. Conversely, with chronic and lifelong conditions, the drug must be taken every day for life, and the cost of lifelong treatment prevents downward moderation of the ICER. That means that, when evaluating medicines that extend life, those that treat conditions from which patients would die within a short period are favoured over those that would extend life far into the future.

That unfairness is doubly compounded by the fact that, when computing the number of quality-adjusted life years attributable to a treatment, NICE usually applies a “discount rate” of 3.5% per annum, based on the Treasury’s Green Book, on both the costs and health effects of the medicine by reference to how far into the future those life years will be added to the patient’s life. In essence, the longer a patient lives, the more expensive they are to the system and the higher the cost per quality-adjusted life year.

Let me give an example. If a treatment were projected to extend the life expectancy of a six-year-old cystic fibrosis patient from 47 to 57 years, the “present value” of those additional 10 years would be less than two once they were discounted. By comparison, an oncological orphan medicinal product may add five life years, starting immediately, to a patient’s life expectancy, so discounting would reduce those five years to 4.66 for the purpose of calculating quality-adjusted life years. That approach cruelly fails to account for the fact that every year of additional survival, regardless of whether it is gained in the short or the long term, will be valued equally in the mind of a cystic fibrosis patient and his or her family.

To add insult to injury, NICE currently does not take into account the fact that when medicines lose their market exclusivity after patent expiry, their cost to the NHS falls dramatically, typically by 80% to 90%. It is unrealistic to assume that a medicine would remain at its currently listed price over the entire model horizon, particularly when that can be upwards of 40 years. There is no reason why NICE could not model the effect of a post-patent expiry price reduction by reference to available evidence from the pharmaceutical market. That is yet another example of NICE’s discrimination against treatments for chronic and incurable conditions in favour of those for acute conditions.

Finally, while NICE recognises that medicines for very rare diseases—ultra-orphan medicines—need a higher threshold and more discretion in the way in which they are appraised, it does not allow cystic fibrosis medicines to be judged against that threshold. That is because, although cystic fibrosis is a rare disease globally, its prevalence in England is such that NICE insists that it is appraised via the conventional approach.

Vertex is not the only manufacturer of precision medicines for rare diseases to experience challenges with NICE. Both the Bioindustry Association and the Association of the British Pharmaceutical Industry have, on behalf of their members, repeatedly highlighted the need for NICE to be reformed, to

“take a broad view of the value of new treatments and innovations to the health service”,

and to incorporate a wider range of factors and flexibilities, beyond the standard cost per quality-adjusted life year gained. It is right for us to ensure that NICE processes are modern and up to date with the evolution of precision medicines.

My constituent Sharon Cranfield is a bit disappointed with a letter sent to the Minister by the Chairman of the Health Committee, outlining the conclusions of the Committee’s public hearing on 8 March. She says that the points that I had raised on her behalf

“appear not to have been considered and the findings of the Committee seem to lie with continuing to defend the NICE model that has been used for the last 20 years and an unwillingness to accept that they need to be re-evaluated to reflect the current and near term developments in precision medicines.”

I understand that the Committee may have advisers who were associated with the setting up of NICE. I think that Ministers, NICE, and everyone who is engaged in this should look forward to a model that will actually work for the people whom we represent.

NICE must re-evaluate the way in which it values rare disease medicines. I sincerely hope that following today’s debate, it will do more to achieve alignment on value, evidence and price for the sake of patients, and will address, once and for all, the limitations of the current NICE STA process for diseases such as cystic fibrosis. That would also benefit all the other patients who already suffer enough after being diagnosed with a rare disease.

It is an honour to follow the hon. Member for Reigate (Crispin Blunt).

I thank the Backbench Business Committee for agreeing to the debate. I also congratulate my hon. Friend the Member for Blaydon (Liz Twist), who is, as she said, my fellow member of the all-party parliamentary group for muscular dystrophy. She made a lucid and compelling case for the review of the NICE appraisal process.

I have had the honour of chairing the APPG for several years. It works closely with our secretariat, Muscular Dystrophy UK, and with patients and carers, on a number of issues that affect the lives of those with muscular dystrophy and other neuromuscular conditions. Perhaps one of the most important issues that we consider is the ability of patients to access treatments for their conditions.

For more than a year, access to the drug Spinraza, manufactured by the company Biogen, has been the focus of the APPG’s concerns about, and frustrations with, the NICE appraisal process. Spinraza is the first and only treatment for patients with spinal muscular atrophy, a rare inherited neuromuscular condition that leads to the gradual loss of the ability to walk, move, breathe and swallow. It currently affects about 2,000 adults and children in the UK. There are several types of SMA, with type 1 being the most severe, usually resulting in the death of infants before they reach their second birthday. However, clinical trials of Spinraza have had amazing results for many of the patients who have tried it. It has been so positive for children with type 1 that over two years ago Biogen opened its global expanded access programme to provide the drug free to type 1 patients.

Spinraza is currently available across 24 European countries and in the US, but for patients in this country access to the drug is being held up by lengthy delays to the NICE appraisal process.

Does my hon. Friend agree that the hon. Member for Reigate (Crispin Blunt) is right that there needs to be a new model, and more importantly that something must be done about the cost of drugs? We cannot carry on with the escalation of the cost of these drugs because, as the hon. Gentleman said, that affects many families in different ways.

That is true, and I think it will be highlighted again and again in this debate.

The delay for patients in this country is made all the more frustrating because the Scottish Medicines Consortium approved Spinraza for children with SMA type 1 last May, and it now has a new ultra-orphan pathway and has speedily reassessed Spinraza, and as a result children and adults with SMA types 2 and 3 will be able to access the drug from next month.

In England the Spinraza appraisal has already been going on for 14 months. In January last year NICE announced that the pathway for the drug would, sadly, be the single technology appraisal, used for common diseases, rather than the highly specialised technology appraisal, which has been spoken about and is used for rare conditions. On 14 August, all hopes were shattered when NICE announced that it did not recommend funding by the NHS as the clinical effectiveness of the drug was not proven and the price was too high.

NICE launched a consultation and held a committee meeting in October to review all responses. There was still no progress for patients. Then, following a previous announcement, on 1 November Biogen closed the expanded access programme for type 1 to all new infants, so although 80 children remain on the programme, any child born after that date with type 1 has no access to this life-saving drug. The process drags on, and NICE had its third committee meeting earlier this month, but as yet no information has been published.

Biogen maintains that the STA process is not appropriate for rare disease medicines, because the smaller patient populations in rare diseases make it inappropriate to expect treatments to achieve the same cost-effectiveness thresholds as medicines in disease areas with much larger patient populations. It has also pointed out that it is very difficult to measure the quality of life in a young paediatric population. However, that is a major determining factor in the STA process, so it is a stumbling block in approving Spinraza. The company still hopes that a managed access agreement can be reached with NICE and NHS England.

The truth is that NICE’s emphasis on cost-effectiveness stands in contrast to the focus on more flexibility and data gathering for future review, which has allowed Spinraza to be approved in Scotland and across Europe. A recent report by MAP BioPharma, “Access to orphan medicines”, highlighted that 75% of rare disease medicines recommended by NICE through an STA between 2013 and 2017 were due largely to rare cancer drugs that are covered by the cancer drugs fund, and none of the only six non-cancer orphan drugs reviewed by NICE through an STA has received a recommendation in line with full marketing authorisation.

The report makes five recommendations for the NICE STA methods review: making changes to the evidence requirements for orphan medicines; drawing from the HST methodology to consider introducing a sliding incremental cost-effectiveness ratio up to £100,000; considering adapting the evidence review group for orphan medicines; embedding formal opportunities for negotiation between companies and NHS England; and considering interim recommendations in line with the cancer drugs fund and the new Scottish ultra-orphan pathway. MAP BioPharma points out that those adaptations would help to level the playing field so that patients, clinicians and companies could be sure that all treatments for rare diseases would be considered under a fair appraisal and that access would not be held back as a result of treatments being referred for an inappropriate appraisal. I hope that those recommendations will be given due consideration by NICE, NHS England and the Department.

Meanwhile, for those awaiting a decision on Spinraza, the anxiety continues. They include families such as that of my seven-year-old constituent, Sam McKie, who has type 2 SMA. Sam loves playing wheelchair football and has played since he was three. He now plays for the Newcastle Magpies wheelchair championship team and is as good as many of the adult players. In fact, he is so good that, in November, the Newcastle United Foundation named him as its disability player of the year. Sam’s dad, Gary, wrote to me, and his words reflect the views of everyone affected by SMA. He said that

“children are facing an agonising and uncertain wait for approval whilst their condition deteriorates. Gaining early access to this drug could see Sam get stronger and gain new abilities. The SMA community would love to be able to access this drug to give our babies and children a chance, a chance they surely deserve. This drug is available now, and timely procedures are stopping our children from accessing it, this is wrong. Please help us.”

Will the Minister hear Gary’s words? Will he take action to ensure that delays do not happen in future? And will he work with Muscular Dystrophy UK and other charities towards making NICE take on board MAP’s recommendations, to help to create a new and fairer system, like that in Scotland, that will deliver for patients like Sam and, as Gary McKie says, give them the “chance they surely deserve”?

It is an honour to follow my hon. Friend the Member for North Tyneside (Mary Glindon), and I also thank my hon. Friend the Member for Blaydon (Liz Twist) for bringing the debate to the House.

I first heard of the drug Spinraza last year when Katie Prescott contacted me about her 10-year-old daughter, Heidi. She wrote:

“I have a daughter, Heidi, with a rare muscle wasting disease called Spinal Muscular Atrophy (SMA). SMA ranges from the very severe type 1 through to type 4. Heidi has type 3 and she is fast losing the ability to walk. She is 10 years old and it’s a devastating prospect for her. For the most severe cases of SMA, life expectancy is only 2 years without access to this drug.”

There is a treatment available for SMA, and it is called Spinraza. It is the one and only available treatment for SMA, and 22 other countries, including Scotland, have approved it. However, NICE is recommending that NHS England should not fund Spinraza for any types of SMA, meaning that thousands of people in the UK with SMA are left without a treatment. This is practically a death sentence for babies diagnosed with type 1, and it means that type 2 and type 3 children are destined for a life of deterioration. The issue is that NHS England and Biogen cannot agree on the costs. We need the Government, NHS England and Biogen to sit down and negotiate an agreement that has the SMA community at its heart.

In January 2019, I wrote to the Health Secretary about this. He replied:

“NICE is an independent body, and it would be inappropriate for me to comment on its guidance”.

When I wrote to NICE for clarification, it said:

“I fully accept that we, the company and NHS England have a responsibility to bring this matter to a conclusion quickly. We are working hard to do this through discussions with the company and I am hopeful of reaching a positive outcome.”

I also asked the Prime Minister this question:

“Why can this treatment not be accessible to my constituent Heidi and other children in England with this disease?”—[Official Report, 20 February 2019; Vol. 654, c. 1462.]

She answered that the next meeting between the parties would take place in March. Since then NICE, NHS England and Biogen have met, on 6 March in Manchester, and we now await their next judgment. That comes after 14 months of appraisals, with months between the stages of the negotiation process. What is it about this country that makes it so different from Scotland and the 22 other countries in Europe that have approved the drug? What have they done to be able to do that? How have they negotiated it?

When a child is born with a condition such as SMA, there is hope in every parent’s heart that a cure will be found—that a new drug will be developed to treat it. This is an example of that, but the drug has been denied to children suffering with SMA, and we must find a mechanism to resolve the problems. I understand that there are structural issues within NICE, which means that the treatments of conditions such as SMA end up being assessed in the same way as more common conditions, therefore making it extremely difficult for treatments to be assessed as being cost-effective. In the meantime, Heidi’s health continues to deteriorate. When I met her recently in my office, I was deeply impressed with her bravery and optimism, but her mother said to me:

“It is very frustrating that still no decision has been made. Heidi cannot walk unaided anymore and even then can only take a few steps. To know there is something that can help her, but she can’t access it, is very difficult and Heidi finds that hard to deal with too.”

Up and down the country, families like Heidi’s are fighting for treatment. Surely NHS England, NICE and the company have a duty of care and a moral obligation to give treatment to patients like Heidi when it is available. We must remember that there are human beings behind the statistics—caring families who are suffering because a medicine has been denied in this country.

I will be brief because I have spoken on this topic many times, specifically about cystic fibrosis, but before I get on to that I congratulate my hon. Friend the Member for Blaydon (Liz Twist) on her speech about PKU. I popped into the PKU drop-in event a few months ago purely by accident when I was doing one of those classic MP things where we are asked to go here, there and everywhere to be photographed holding up a sign or wearing a silly hat. I knew nothing about PKU, but I talked to an 11-year-old boy there about his diet. I am somebody who chooses to have a fairly restrictive diet in that I do not eat meat, dairy or fish, but I look for protein alternatives, and I was struck by the sheer difficulty that the boy has in functioning and by the fact that he is unable to enjoy the normal life of an 11-year-old. My heart went out to him, and I do not need to add any more, because my hon. Friend made such an excellent speech.

We have also had excellent speeches about the difficulties of obtaining Spinraza. I do not claim to be an expert, as some other Members are, but constituents have been in touch with me with similar problems. I would therefore support any efforts to get the drug approved, because that would make a huge difference to their lives.

As I said, I am here to talk about cystic fibrosis, and I have a personal interest in that my 14-year-old niece Maisie has CF. She must be the most mentioned niece in Parliament, because she gets name-checked quite a lot. Before I talk about Maisie, I want to mention the recent evidence to the Health and Social Care Committee from Oli Rayner, who lives with cystic fibrosis. He has just turned 40 and had a lung transport around 18 months ago, so he is at the healthier end of the spectrum, and he talked about being quite lucky until he reached his thirties, when his health started to deteriorate.

However, many cystic fibrosis patients are not as lucky. My constituent Lee Partridge lost both his daughters within eight months when they were both in their late teens, and it is common for people to start to deteriorate when they reach their teens. Luckily, my niece is very much at the healthier end of the spectrum, but that does not influence my judgment; I want to make the case for Orkambi to be available not just for her when it is needed, but for everybody else with the condition.

I have been trying to get my head around quality-adjusted life years, how the calculations are made, whether NHS England is doing the right thing and whether NICE is using the right sorts of calculations compared with other countries. NICE says the calculations factor in societal benefits, but it is not clear how they do that.

The hon. Member for Reigate (Crispin Blunt), who made an excellent speech, spoke about the cost of hospital treatment to society as a whole. If a person has to go into hospital several times a year, there is the cost of care, but there is also the cost of lost work time, the cost of care by family members and even the cost of transportation to hospital if they live in a rural area. There are so many factors. Although NICE says it takes those things into account, I cannot quite see them. Was it Hugh Dalton or Clement Attlee who was called a “desiccated calculating machine”? Anyway, the system seems to be based on desiccated calculations.

It may be incredibly naive of me, but why cannot the system just think of the impact on people’s lives and of the hell they are going through? It is incredibly difficult for teenagers to come to terms with discovering that they have a life-limiting condition, and there is also the issue of the availability of mental health support and counselling.

It is striking that everyone who gave evidence to the Health and Social Care Committee said that the drugs work. There is no question but that this would be of benefit to people. Kalydeco, the first drug, helped only 5% of patients, and it was funded in part because not many people would benefit and, therefore, the costs were lower. We now have Orkambi, and there will soon be a successor that will benefit about 40% of patients. For the future we are looking at triple therapies that will cover about 90% of the cystic fibrosis community. If 90% of people can benefit, of course the overall cost will go up, but the decision should not be made on the basis that we fund one thing because not many people will benefit.

I am concerned that Vertex and other companies would be deterred from exploring further therapies and treatments if they cannot get a commercial deal. The line that jumps out of the Health and Social Care Committee’s letter to Vertex, which it wrote shortly after the meeting, is:

“Vertex appears to have decided on the pricing of its therapies on the basis of the return it wants to make, rather than the value which they bring. NHS England is right to continue to take the wider patient population for whom it is responsible into account.”

The Minister has previously responded to such debates, so can we have an update on where the conversations have got with Vertex, including the Secretary of State’s meeting? There are so many cystic fibrosis patients waiting to hear the outcome. Last time I looked, the petition was up to about 80,000 signatures. Can we try to get some answers soon?

I am sure hon. Members will not be surprised to hear me talk again about baby Maryam, my seven-month-old constituent with spinal muscular atrophy type 1. Without treatment, Maryam will have only a few more months to live. She is waiting for NICE to announce a decision to allow access to Spinraza, the drug that she and other babies with the condition so desperately need.

I start by thanking my hon. Friend the Member for Blaydon (Liz Twist) for securing this important debate; the Daily Mirror, especially Pippa Crerar, for telling the stories of SMA families; the Newham Recorder, the campaigning newspaper, for its continued coverage and encouragement; and the amazing members of the local community in West Ham for their support. Councillor Mas Patel has been brilliant, as has Kevin White and many others who have taken the plight of this little baby into their hearts.

Most of all, I pay tribute to Shakil, Maryam’s father, and all Maryam’s family who have steadfastly campaigned despite knowing that, when we finally get access to Spinraza in England, it could so easily be too late for Maryam.

As we know, SMA1 is an awful disease. It is a progressive muscle-wasting condition, and for a baby it causes difficulties with breathing, swallowing and gaining weight. Babies with SMA1 may not even be able to cry aloud. Spinraza is an effective treatment for SMA. With this treatment, babies and children are living longer; they are even crawling and walking. I am told that by last August, when NICE first met to consider funding Spinraza on the NHS, none of the babies who had been treated with it in the UK had died as a direct result of their SMA. The drug was so effective that the trials stopped early, yet NICE refused to approve Spinraza at that August meeting, or at the second meeting in October or in November.

In November, the expanded access programme, which allowed treatment with Spinraza while negotiations were ongoing, was closed—Biogen, the drug company that developed Spinraza, closed it. So when Maryam was diagnosed in December last year, she had no access to this drug. Even if it could have been afforded privately and even if my community in West Ham had fundraised for it—I can tell Members that an awful lot of people have wanted to do just that—she could not have got it. We are not talking about huge numbers; we are talking about fewer than 100 babies a year. Treating this condition should not break our bank—it could not, surely.

NICE met again two weeks ago—we held a vigil while it was making its decision—but I am told it could be three or more weeks yet before that decision is known. This is callous. Making families, making Maryam and making the other babies wait this long is callous. The NICE approvals process for Spinraza has now taken 14 months. That is longer than many of the babies with SMA1 can be expected to live without the treatment. At the same time, agreements have been reached on Spinraza in the United States and in 25 European countries, including Scotland, Germany, Italy, Lithuania and Romania.

NICE has failed Maryam and all the other babies with SMA, but others are culpable, too. The closure of the access programme no doubt saved Biogen some money, but I am sure that it was closed to increase pressure on NICE as well. I cannot say that that is definitely true, but what I will say is that if it is true, the suffering of tiny babies, and the pain and suffering of their families, too, has been used in the service and pursuit of profits. I now believe that our whole system of for-profit medical research combined with public healthcare encourages this kind of brinksmanship, it encourages delaying tactics and it encourages the exploitation of sick children and their families.

Let us also consider the actions or inaction of the Government. I am sure hon. Members will recall how in the spring statement the Chancellor talked of a

“proud, successful, outward-looking nation, with no limit to our ambition and no boundaries to what we can achieve.”—[Official Report, 13 March 2019; Vol. 656, c. 352.]

He bragged about

“the single largest cash commitment ever made by a peacetime British Government”.—[Official Report, 13 March 2019; Vol. 656, c. 346.]

This was for the NHS. Surely we can afford to help these babies, too. Currently, we are the fifth richest country in the world, yet we are withholding vital medical treatment from babies. This is medicine that 25 other European countries, including some much poorer than we are, are willing to provide. What does that say about us? What does that say about our values? What kind of a country have we become?

I have asked the Prime Minister to act—twice. I have asked the Health Secretary to act. As far as I can see, nothing has happened. I hope that Conservative Members will forgive me when I say that I thought the Prime Minister’s response to me last week was cold; it was more about defending an outdated process than about understanding and empathising with the desperate plight of Maryam and her family. Let us remember that 25 European countries already have access to this drug, We do not, yet it seems to be nothing to do with the Prime Minister.

The hon. Lady is making a good and powerful speech. Two of my constituents, Mark and Panna Wilson, are in a situation similar to that of her constituents: their son Aadi is only three years old and is regressing every day because he, too, has SMA, which is a terrible condition. They told me that

“without access to new treatments, each and every day is a step, not towards an exciting future, but towards a painful and dark place.”

The hon. Lady is right, and I hope that we will find a new route to the approval of Spinraza in this country. The route it is currently going through is intended for much more generic medicines and it has not been put through the route for rare medicines. We need either to put it through the route for rare medicines or to find or invent a new mechanism so that we can get treatment for these people. As she points out, this is a rich country: we must be able to save these babies and toddlers.

The hon. Gentleman is absolutely right. This is a political issue—what we are discussing today is political. It is about which of our constituents get access to which medicines, and when; who our public healthcare service helps with our public money; and who we allow to die without help.

Members on both sides of the Chamber have recently urged the Government to use their Crown use licensing powers in the case of Orkambi. Those powers can get around medicine patents when pharmaceutical companies are simply refusing to deal honestly with the NHS, and they are an option. There are always options for the Government if they wish to use them, but they seem to be content with voicing frustration. They do not do anything—they will not even threaten to do anything that might help. If the Patents Act 2004 is not working, as Ministers have said, we need to do something to replace it. Maryam’s family and others want action now. They need Spinraza now; when will they get it?

It is a pleasure to follow my hon. Friend the Member for West Ham (Lyn Brown), who made her case with great power and passion. I congratulate my hon. Friends the Members for Blaydon (Liz Twist) and for North Tyneside (Mary Glindon) on securing this important and timely debate.

Until recently, I had the pleasure of chairing the all-party group on rare, genetic and undiagnosed conditions, which receives secretariat support from Genetic Alliance UK. The all-party group aims to raise awareness in Parliament about such conditions and to ensure that patients and families can access appropriate care and support. My hon. Friend the Member for Hornsey and Wood Green (Catherine West) has taken over as chair of the all-party group. Unfortunately, she had prior commitments today, but otherwise would have been here to take part in the debate.

My constituency in Liverpool is home to the fantastic Alder Hey Children’s Hospital, which works in close partnership with the University of Liverpool and the charitable sector to provide more research opportunities for children with rare diseases and their families. For example, in the rare neuromuscular diseases, Alder Hey has worked with patient-advocate groups and national charities to increase capacity and resources to offer access to clinical trials of new disease-modifying agents. Since 2016, Alder Hey has opened 19 new clinical trials for Duchenne muscular dystrophy alone, enrolling nearly 100 boys in these important trials. By providing excellent, world-class clinical and research expertise and working in partnership with the charitable sector, Alder Hey has been able to provide more opportunities for children with rare diseases to take part in research studies.

In my role as chair of the all-party group, it was a privilege to get to know the rare diseases community. During that time, the issue that was consistently raised with me by patients and their families, clinicians and pharmaceutical companies was the challenge they face attempting to navigate NHS England and NICE’s appraisal processes for rare disease treatment. As other Members have said, patient access to new orphan medicines in the UK lags behind many other European countries, including Germany, France and Spain. The Office of Health Economics found that the UK is slower at making access decisions and approves far fewer medicines for reimbursement than other European countries. According to the recent MAP BioPharma report, which used data from a four-year period, almost a quarter of licensed orphan medicines have not been appraised at all by NICE or NHS England. This can prevent rare disease patients from having any opportunity to access treatment.

The lack of capacity to appraise orphan medicines is just one of the systemic flaws—we have heard about others—with the current NICE appraisal system. The multiple pathways through which medicines can be appraised create further complications and delays. It is often unclear why one route is chosen over the other, so very similar orphan medicines can be subject to vastly different assessment criteria. We also know that patients are often stuck in limbo waiting for the results of private negotiations about price between companies and the NHS years after the market authorisation of their potential treatment.

It is against that backdrop that the all-party group asked Genetic Alliance UK to propose a method of making decisions about rare disease medicines that would be more effective, transparent and fair. The project is called “resetting the model” and it aims to develop a flexible new vision for getting access to rare disease medicines for the UK, and it will be delivered in the coming months. It is absolutely clear that the current NICE appraisal process is simply not fit for purpose and is acting to restrict many rare disease patients from having the opportunity to get access to potentially life-changing treatment.

Let me address a related issue. NICE has been consulting on guidelines for cannabis-based products for medicinal use. On Tuesday, I had the pleasure of meeting my constituent, Lauren Abernethy, who is the mother of Nathaniel. Nathaniel is 10 months old and has a type of epilepsy that is so rare that when he was diagnosed his doctors told Lauren and James, his dad, that Nathaniel is the only known case with this type of genetic mutation recorded anywhere in the world. For the next three months of his life, Nathaniel was in Alder Hey Hospital undergoing tests, and for most of that time he was in critical care.

Lauren was here on Tuesday as part of the “End Our Pain” lobby of Parliament. Owing to the extremely rare type of epilepsy that Nathaniel has, antiepileptic drugs do not work, so Nathaniel was granted the use of Epidolex, a medical cannabis product. He has reacted positively to the treatment, going from being in a state of continuous seizures to now having, on a good day, as few as three to five seizures. However, despite that progress, he continues to live in great pain. He jerks and twitches continuously, which is known as myoclonic jerks, and has up to 100 of these every day. Access to full extract cannabis oil might offer Nathaniel a better quality of life, which he surely deserves, but like so many other children who are suffering from intractable epilepsy, Nathaniel has so far been denied access to that treatment.

I welcomed the decision last November by the Home Secretary to reschedule certain cannabis-based products for medical use, but the reality for patients such as Nathaniel is very different. Only a tiny handful have succeeded in getting a prescription. Patients, some of whom are as young as Nathaniel, are being blocked from access to medicine, which, as the evidence shows, has at least a possibility of relieving their symptoms. I urge the Minister in his response today to talk about how a policy can be put in place, based on evidence, that enables patients with rare diseases who could benefit from medical cannabis getting access to it.

More broadly, may I echo the comments that have been made on both sides of the Chamber and ask the Minister today to set out what he will do to take this crucial set of questions forward? In particular, will he work with Genetic Alliance UK so that we can reset the model, working with NICE and NHS England, so that patients with rare diseases get the fair access to medicines and treatment that, surely, they all deserve.

It is a pleasure to contribute to this important debate. I thank my hon. Friends the Members for Blaydon (Liz Twist) and for North Tyneside (Mary Glindon) for obtaining this debate, and the Backbench Business Committee for granting it.

Before I was elected to this House I worked for more than 30 years as a clinical scientist in our NHS. During that time, I developed a healthy scepticism for politicians advising on medical treatments, as it is a field that is best left to clinicians. However, I did want to take part in this particular debate because I know that issues around the availability of life-saving drugs and treatments for rare diseases are important to many people. My constituents have made me particularly aware of the unavailability of Orkambi to cystic fibrosis sufferers and of Kuvan to those affected by phenylketonuria.

Today’s debate is also extremely valuable because it focuses on the way NICE operates and questions whether that operation is appropriate for those suffering from rare diseases. A rare disease is generally defined as one affecting fewer than five people in 10,000, but many people are affected by rare conditions. The UK strategy for rare diseases estimates that, in the UK alone, more than 3 million people will suffer from a rare disease at some point in their life.

Given, however, that relatively few people are affected by a particular rare disease, there are specific challenges in ensuring speedy diagnoses and access to appropriate services and treatments. NICE’s technology appraisal process involves looking at evidence from clinical trials and peer-reviewed research showing how well a medicine or treatment works, including its likely impact on mortality and quality of life; at the economic evidence of how much it costs the NHS; and at the views of clinicians, patients and other stakeholders.

As well as looking at the clinical effectiveness of a treatment, single technology appraisals and highly specialised technology evaluations also assess its cost effectiveness, which, as many speakers have said, is usually measured in terms of the cost per additional quality-adjusted life year, and this is assessed by looking at how many extra months or years of life of a reasonable quality a person might gain as a result of treatment.

Following changes introduced in April 2017, NICE set a maximum additional quality-adjusted life year threshold of £300,000 for highly specialised treatments. Under that threshold, they would automatically be approved for routine commissioning. This is 10 times higher than the standard NICE threshold of £30,000 for non-specialised treatments. Owing to the nature of lifelong genetic diseases, however, the required quality of life improvements are likely to be unobtainable. The charity Genetic Alliance UK highlights that no ultra-orphan treatments—drugs used to treat extremely rare diseases—currently used by the NHS would pass this test.

It is clear that an urgent rethink is needed on these policies, which are focused almost exclusively on price, to the detriment of patient outcomes. Genetic Alliance UK has argued that these policies will halt future access to innovative treatments for rare genetic conditions in England and that they contrast with the stated aim of the UK strategy for rare diseases to ensure appropriate procedures for evaluating the costs and benefits of treatment for patients.

The problem with the current NICE appraisal process is that certain treatments, particularly those designated as orphan medicines, are neither eligible under the narrow criteria of the highly specialised technology process nor appropriate for the single technology appraisal process. The reason could be that the treatment’s patient population is marginally higher than the maximum size considered through the highly specialised technology process, while the single technology appraisal process is inappropriate for most orphan drugs because of limited trial data.

The accelerated access review, which aims to speed up access to innovative drugs, devices and diagnostics for NHS patients, recommends that NICE undertake a review of its methods and processes to ensure that they are fit for purpose, which I think everyone in the Chamber would agree is long overdue. The review warns that

“it is important that no groups of products can ‘fall between the cracks’ and struggle to find a decision-making process”.

We have heard heartfelt speeches today from my hon. Friends that have illustrated that some products are falling through the cracks and that families and young children are suffering.

The views of the accelerated access review are in line with those of the NHS five year forward view, which includes a recognition of a broad measure of value that goes beyond price alone. I have combed through the latest NHS long-term plan, and I cannot find any reference to this important issue. When the Minister responds, I would be grateful if he pointed out to me where the issue is mentioned in the most recent iteration of the plan.

NHS England and NICE need to reconsider how they account for rarity in their assessment process to support the NHS in its mission to provide a comprehensive service that is available to all at the point of need, including to those with rare conditions.

I thank the hon. Members for Blaydon (Liz Twist) and for North Tyneside (Mary Glindon), and the Backbench Business Committee, for arranging this debate. I am here to speak up for people in Dudley with cystic fibrosis, phenylketonuria—or PKU—and spinal muscular atrophy.

I have been campaigning with others to make Orkambi and other new treatments available for people with cystic fibrosis because I was contacted by Carly Jeavons from Dudley, who took part in the clinical trial for Orkambi, and by Samantha Carrier, whose baby daughter Daisy was diagnosed shortly after birth. Samantha has given up her career and now devotes her life to campaigning for access for these life-changing drugs. While listening to this debate, I have been receiving emails, texts and other messages from people around the country—heroic parents of children with cystic fibrosis who work so hard and campaign tirelessly for the treatments that their children need. I know that the Minister has met some of these parents, and I hope he will have some new answers for them today, because they have worked so hard to raise these issues, which really do need to be sorted out.

Before the clinical trial, Carly Jeavons had to take 90 tablets and do two hours of physiotherapy a day. She had a lung function of around 44% and spent two weeks in hospital every three months. She had to choose between the financial hardship of leaving work or her health being made worse as she struggled on at work. She told me:

“Orkambi has changed my life. My health has remained stable. I only need one or two courses of IV’s per year instead of the four previously, hospital visits have been massively reduced and admissions are non-existent.”

Since having Orkambi, she has been able to go on holiday abroad for the first time with her family, and she has got married. She has also started a business and is employing people, so she is making a much bigger contribution to the economy. The Government need to look at the contribution that people who get these drugs can make to the economy, not just at the costs of providing the drugs. I believe that NHS England and NICE are with Vertex this afternoon for yet another meeting about whether these cystic fibrosis drugs can be provided. But this comes three years after NICE appraised Orkambi. I really hope that the Minister will explain how this situation can be resolved and how other situations like it can be avoided, so that patients can get can get access to these drugs.

Let me turn to the issue of treatments for people with PKU. Some of the people who have been leading this campaign are sitting in the Public Gallery, watching this debate. Again, I hope that the Minister will have some good news for them today. As we have heard, PKU is a rare metabolic disease that leaves people unable to metabolise protein properly, leading to a toxic build-up of material that can cause irreversible brain damage. The only existing treatment is a strict diet of extremely low protein, meaning that almost all normal foods are off limits. The diet is lifelong, and sufferers find it stressful and difficult to cope with. I had never heard about this condition until a woman in Dudley called Kirsty Thornton got in touch with me. Since then, I have met the campaigners and taken part in a PKU diet challenge. I have also joined the cross-party parliamentary campaign led by the hon. Member for Blaydon to ensure that people with PKU get access to the treatments and supplements they need.

It is heartbreaking for parents of young kids with PKU who do not understand why they cannot go to their friends’ birthday parties in case they eat the wrong foods that will make them tired, sick and ill for the rest of the day, or for longer. Students say how difficult it is when their friends are going on nights out, or they move to university and the people they share a flat with are ordering in pizzas and so on, or they cannot go out on a date because they do not know what they are going to be able to eat or not eat. This must be really tough for young people.

My hon. Friend will probably be aware that the Government are looking at a new food strategy, and one of the things under consideration is better food labelling. A lot of people think that those who make certain dietary choices do it almost to be trendy, or just because it is the fashionable thing to do, but there are people whose lives are at stake if they cannot get the information on food that they need. Food manufacturers and anyone else involved in the provision of food need to be alert to the fact that it is important that people know what they are going to be eating.

That is a really important point. The PKU campaigners have explained to me that on some occasions when manufacturers have changed the ingredients in food or drinks, that has had a terrible impact because people with PKU have not necessarily known about it, so they have continued to drink or eat things that they have consumed without any problems in the past, but because the ingredients have changed, it has caused them a big problem.

For many people with PKU, taking Kuvan considerably increases the amount of protein that they can safely eat. We are therefore urging the Government, the NHS and BioMarin, which manufactures these supplements, to agree a deal so that people with PKU can enjoy a normal healthy life. I spoke to the National Society for Phenylketonuria this morning, and it told me that the whole PKU community are demoralised. They say that they are working hard but feel that not much progress is being made. What is the Minister going to do today to give these people, some of whom are in the Public Gallery, and others who are sitting at home with their kids watching this debate, to give them hope of this situation being resolved?

My next point is about Spinraza treatment for people with spinal muscular atrophy, or SMA, which affects an estimated 1,300 people across the UK. It can cause irreversible loss of a child’s ability to crawl, walk, breathe and swallow. In the most severe cases, it can cause death. Spinraza is the first possible treatment for those who have SMA types 1, 2 and 3. It can slow its progression and prolong life. From April, this treatment is going to be routinely available in Scotland, and it is already available in 24 other countries in Europe, yet it is still stuck in the NICE process for England, Wales and Northern Ireland.

That is why, in the end, the purpose of this debate is to ask the Government to look carefully at the way that NICE works. New drugs are being developed, and technological changes are happening, so rapidly that the Minister needs to be able to tell us how the way drugs are assessed and licensed, and then approved, will work in future. How is he going to ensure that these ground-breaking drugs are made available to the people who need them, when they need them? In 2016, NICE was not able to recommend the use of Orkambi because of uncertainty around its long-term value, impact and cost-effectiveness. But this drug is available in the USA, across Europe, and, more recently, in Scotland, so when do the Government think patients in England are going to get it? This is really urgent. The system has not worked and patients are being let down.

On PKU, NICE decided to start an appraisal of Kuvan in 2018, but this has since been suspended. NICE is currently reconsidering which appraisal process to use to access Kuvan, and the NHS is considering whether to fund an interim policy for the use of the drug. But, again, this is not enough, and not quick enough. The NICE process sees PKU as rare, but not rare enough. As we have heard, the majority of PKU treatments are assessed by the STA process, which is designed for non-rare treatments. NICE’s approach evaluates the lifelong costs of treatment, meaning that the cost thresholds and the approach to evidence are all designed for more common diseases than PKU. SMA sufferers are waiting for NICE, NHS England and Biogen, which manufactures Spinraza, to come to a deal. I hope we will hear better news on all those things from the Government than we have in the past.

It is my job to listen to people in Dudley who are living with cystic fibrosis, PKU, SMA and other rare diseases, to come down to London and speak up for them in Parliament and to demand, as we are doing this afternoon, that the Government ensure that they get access to the treatment they need and deserve.

I would like to thank my hon. Friends and neighbours from the glorious north-east, the Members for Blaydon (Liz Twist) and for North Tyneside (Mary Glindon), for securing a debate on this important topic, and the Backbench Business Committee for granting it.

I want to focus my comments on phenylketonuria, which is classed as a rare disorder and has stark impacts for sufferers. If left untreated, PKU can result in brain and nerve damage, behavioural difficulties, vomiting, tremors and epilepsy, yet some of the impacts can be mitigated to a degree, if only the NHS would allow the drug Kuvan to be readily available.

PKU sufferers cannot metabolise protein and, as such, are kept on a strict diet and take a large number of supplements to ensure that they do not become deficient in anything that we would normally get from the foods they are unable to eat. Many sufferers also need to take an unbelievable number of tablets each day—tablets that are large and difficult to swallow. My constituent Harvey Parker has PKU. He is 15 years old and takes a staggering 65 of these tablets every single day. Although it is a distant memory for some of us, I am sure we all remember how difficult it is being a teenager, with that want to fit in and not stand out. Harvey told me that Kuvan would help him lead a more “normal” life.

Eating out and sharing food with others is an important part of socialising and something that most of us do without thinking, but many places do not cater for Harvey’s needs, and he ends up feeling “embarrassed”, “isolated”, “angry” and a “burden”. He said that when he has been out with his mates on a weekend, he tells his mam and dad that he has not eaten because he was not hungry, when really it was because he could not find anything that he could eat. Harvey told me:

“I don’t really talk to any of my friends about PKU as I get embarrassed and when I’m with my friends with no tablets to take, blood tests to endure or bland, unpalatable foods then I am just Harvey, one of the lads and not that boy with the rare invisible condition that no one has ever heard of.”

It is clear to see how access to Kuvan and the more relaxed diet that would follow could improve Harvey’s life. It has improved the lives of others who have taken it, so why is it being denied to so many sufferers?

Kuvan is used by more than 2,000 patients worldwide and is commonly used in 23 other European countries, but not England. Some sufferers have had access to the drug on the NHS after costly and lengthy court battles, but most are not that lucky. At an estimated annual cost of up to £50,000, the drug remains out of reach for so many sufferers. An adult with PKU’s protein-restricted diet costs the NHS £12,000 per year. Many sufferers get prescriptions for their food, which has a high price tag: a packet of pasta is £7.20, and a loaf of bread is £7.48. Our NHS was created on the principle that good-quality healthcare and medication should be accessible to all and free at the point of need, regardless of income, but time and again under this Government we are seeing the destruction of our NHS by stealth, with creeping privatisation and a rolling back of that principle.

Constant wrangling led to a situation where, four years ago, NHS guidance said that there was not enough evidence to prescribe Kuvan, and it then referred the issue to NICE to see whether Kuvan could be made readily available on the NHS. As we have heard, NICE has two methods of appraising medicine: the highly specialised technology appraisal for rare diseases, and the single technology appraisal route for common diseases. NICE decided to assess Kuvan using the same route as for common diseases, despite the fact that only one in 10,000 people has this illness—hardly “common”. Thanks to campaigning by the National Society for Phenylketonuria, NICE then suspended the appraisal and decided to reconsider, but the new process has yet to be started. However, as other Members have already noted, the HST route is not ideal for rare diseases, and a whole new different approach is needed.

The NHS also decided to prescribe Kuvan, then changed its mind due to costs. In short, it decided that it was too expensive. That is what happens when privatisation and outsourcing are prioritised over patients’ care and wellbeing. Harvey’s mam, Diane, said to me, “How can you put a price on my son’s health?” I sincerely hope the Minister will let Diane and Harvey know.

It is a pleasure to follow the hon. Member for South Shields (Mrs Lewell-Buck), with her passion for the subject matter and on behalf of her constituents, so well done to her. I thank the Backbench Business Committee for enabling this debate to take place. I was very pleased to go along to it with the hon. Members for Blaydon (Liz Twist) and for North Tyneside (Mary Glindon)—two formidable, diligent ladies who have put down their marker on this subject matter. I was very pleased to be the third in the trio involved, and well done to them for giving us the opportunity, on behalf of our constituents, to express our concerns today.

I am sure that, like me, most Members in the Chamber have been inundated with emails from people begging to be helped, and begging for the drug that they need to be made available. Every one of them has said that, and I do not believe that anyone can ignore it. It is not only in the emails, but in the interaction we have with our constituents when we hear their stories face to face.

I want to start with a good story and to say what happened—not, I have to say, through this Minister, but through the Home Office. I fought very hard to see medicinal cannabis legalised for my beautiful young constituent Sophia Gibson, and what a difference that has made in her life, as it has to the lives of others. Her parents had no option other than to uproot their family and move to Holland to get the treatment that Sophia needed. At the same time, they were respectfully knocking on doors and following the system through to a Home Office Minister, the Minister for Policing and the Fire Service, and getting clinical assistance in order to bring about a change. Eight months later, their daughter has been at school more than ever before in her whole lifetime. She has had substantially fewer admissions to hospital, and has attended school parties that were impossible—they were too much for her—without this medication. She is a very different young girl today because her parents, Darren and Danielle, fought the battle in the right way and in the right place. Today, we have all presented our cases for the right battle, in the right place, at the right time and with the right request.

Since that time, I have had many other parents coming to me and asking me to help secure the drug that their child needs. A mother in my constituency has an absolutely gorgeous two-year-old son—Lorcán they call him—whom Orkambi would help. Orkambi is a second precision medicine, which targets the root cause of the disease, and it would benefit about half the people with cystic fibrosis in the United Kingdom of Great Britain and Northern Ireland. Cystic fibrosis is a terrible illness that affects the lungs and digestive system of people with the condition, who have a median life expectancy of just 31 years old.

I say this very respectfully, and I hope it will be taken into account: Vertex Pharmaceuticals, NICE and NHS England must end the protracted negotiations for the drugs Orkambi and Symkevi. We must break the stalemate between the three parties and provide access to these drugs, which could so vastly improve the quality of life of my constituents and those of everybody here, as they deserve. They want and need that drug desperately.

I have often spoken about the need to allow people to access Orkambi on prescription, and I have often been beyond frustrated with the lack of movement between the drug company and NICE. There must be—indeed, there has to be—something that can be done to find a way forward, and I believe that it must be done in this House. Today, we look to our Minister and our Government to give us answers and, respectfully, the answers that we need. We must instruct the Department to negotiate a way forward to ensure that my young constituent and so many others like him can live a better life.

I was contacted by the Muscular Dystrophy Association regarding spinal muscular atrophy—other hon. Members have spoken about that condition on behalf of their constituents, and I will do the same. SMA is a rare inherited neuromuscular condition that affects lower motor neurons in the spinal cord. It leads to the gradual loss of someone’s ability to walk, crawl, move, breathe and swallow, and it requires complex medical support. About 100 children are born with the condition each year, and around 2,000 children and adults in the United Kingdom are living with SMA. Spinraza is the first and only treatment for patients with spinal muscular atrophy. It is meant to increase the body’s ability to produce a protein called survival motor neuron, which is essential for motor neuron health. The treatment is administered through an injection into the spinal canal. It is never an easy treatment, but if it provides an opportunity for better health, people should take it.

For children with SMA type 1, life expectancy is rarely longer than two years. Some children who have received Spinraza have seen their muscle strength improve, and have lived long enough to crawl and even walk. For those children, Spinraza has been a lifeline—perhaps I should say that Spinraza has been life itself, as that is the level we are talking about. Spinraza proved so effective in clinical trials for children with SMA type 1 —the most severe form of the condition—that the trial was stopped early so that all children affected by it could potentially access that treatment.

The Spinraza appraisal took almost 14 months, and currently 25 European countries—including Scotland in the United Kingdom of Great Britain and Northern Ireland—have already made it available, nearly two years after the European Medicines Agency granted it a licence. Although Translarna was eventually approved by NICE in July 2016, that was more than two years after the European Medicines Agency gave it a licence. Such lengthy delays to a process that could and should be significantly shorter have resulted in frustration and anxiety for many families who see a life-changing treatment within touching distance. They can almost reach out for it, but they can never get it, and that is where frustration creeps in. That group of people see a better life but are prevented from accessing it. There must be a better way of dealing with these issues, and we must find it or instruct the Department to find it.

My heart aches as a father and grandfather who would do anything for his children and grandchildren, and other right hon. and hon. Members would do the same for theirs. The block on life-changing drugs affects not only the child but the entire family. If a child or adult has a physical disability, their family also feel that and live with it every day. Many families in the UK do not have access to life-changing medication, and I wholeheartedly and sincerely urge the Minister and his Department to enter into talks, find a way forward, and do better so that people can live better. The Government have set aside £40 billion for extra health support. I welcome that, as do all hon. Members, and I gently suggest that some of that money should be set aside for life-saving drugs.

It is a pleasure to follow the hon. Member for Strangford (Jim Shannon), and I congratulate the hon. Members for Blaydon (Liz Twist) and for North Tyneside (Mary Glindon), and the Backbench Business Committee, on securing this debate.

This is not the first time that I have spoken in such a debate, and I declare an interest because my two-year-old granddaughter, Saoirse Fellows, has cystic fibrosis. We are very fortunate because she is pancreatic sufficient, but it is a real worry for our family. I should also say that I am waiting for granddaughter No. 4 as we speak, so if I am a bit lightheaded that is why. I have never listened to a debate in either Westminster Hall or the Chamber in which so many people have cited Scotland as a great example of how to do things. That is normally my job in these debates, so I am grateful to hon. Members who have already done part of that job for me.

An estimated 7,000 rare diseases affect about 3.5 million people in the UK, and around 80% of those are genetic. It has been reported that 95% of rare diseases have no approved treatments available. The default NICE referral route for the majority of orphan medicines is the single technology appraisal, but many will be close to meeting the selection criteria for NICE’s highly specialised technology programme, which has already been mentioned. Stakeholders are frequently sought to make the case for an HST referral, which is a more appropriate route for orphan medicines.

The Government have maintained the position that it is appropriate for orphan medicines to be considered under NICE’s standard STA and that orphan medicines have been successfully reviewed under the STA programme. However, many Members have said how that is not working for their constituents. The Scottish Medicines Consortium has adapted its processes to increase the input of clinicians and patient group experts in decisions for certain orphan medicines.

Mr Deputy Speaker, I am cutting my speech because I want to allow more time for both the Opposition spokesperson and the Minister. Scotland and the SMC has led from the front, and NICE has adopted many of its methods in the past. I encourage the Minister to look again at what Scotland is doing. We are a small country and a devolved nation, but we can do it, so I can see no reason why the England NHS should not be getting the same medicines as we do. We have a higher approval rate: 69% in Scotland to 55% in England. We have a new medicines fund specifically for orphan drugs, which is instrumental in helping to secure access to medicines for rare diseases. That does not exist in the other devolved nations. Both Scotland and Wales have taken a proactive approach to addressing concerns about the applicability of the standard processes for orphan treatments.

The new medicines fund has really helped to deliver better medicines more quickly to Scotland. The fund has covered the cost of orphaned drugs for individual patients where the condition affects fewer than one in 2,000 people. A total of £21 million was made available for the fund by the SNP Scottish Government. The Scottish Government have since made further improvements, reforming access to new medicines by creating the peer approved clinical system tier 2. The system allows clinicians, on behalf of their patients, to ask a PACS panel whether they can access a medicine that has not yet been recommended by the Scottish Medicines Consortium. That is exactly why Orkambi and Symkevi can be used in Scotland. I would suggest it is absolutely imperative that this is looked at in England. Spinraza, which has been mentioned in the debate, is already being used in Scotland. I ask the Minister this: why is it not possible for NHS England to use those drugs in England to the same extent that they are in my own country?

I do not want to bring too sour a note to the end of this debate—there is real consensus across the House, and Members have spoken sincerely and passionately on behalf of their constituents and others across the country—but I worry, and I think many other Members across the Chamber will be worried, that we are heading towards Brexit. No one knows where it is going or the impact it will have on the availability of medicines generally, and, more importantly for this debate, medicines that are not yet even available in England as they are in Scotland.

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.


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.

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—

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—

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—

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.

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?

I see the Minister nodding, so perhaps that can be recorded in Hansard. He also said that he has heard from us about these issues before, but I can tell him that he will hear from us again—

Motion lapsed (Standing Order No. 9(3)).