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.
Thelma Walker (Colne Valley) (Lab)
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 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 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.
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.
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.”
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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 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.
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.
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”?
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.