Motion made, and Question proposed, That the sitting be now adjourned.—[Siobhain McDonagh.]
I wish to declare an interest—I am a trustee of the Pulmonary Hypertension Association UK—which is listed in the Register of Members’ Interests. I am grateful for the opportunity to bring to the House’s attention the grave concerns of expert clinicians, patients and their advocates about the preliminary recommendations in the National Institute for Health and Clinical Excellence appraisal consultation document, “Pulmonary arterial hypertension (adults)”. The NICE appraisal committee is meeting today to consider the submissions to its consultation, which ended on 20 March, and I hope that it will take account of our deliberations.
Pulmonary arterial hypertension is a condition that causes high blood pressure in the pulmonary arteries, which carry blood from the heart to the lungs. The condition causes those arteries to thicken and narrow, restricting blood flow in the lungs. As a result, the right side of the heart must pump much harder. The symptoms are breathlessness, chest pain, angina, fatigue and fainting spells. So far, it is not curable, and if not treated, it leads to premature death. Treatment aims to improve patients’ quality of life and extend life, enabling lung or heart and lung transplants to be made in suitable cases. Pulmonary arterial hypertension affects children and adults. It is a rare condition: NICE estimates its prevalence to be between 15 and 50 cases per million, which means that, at the top end of the range, 2,165 adults in England and Wales may have the condition. Approximately 1,500 patients are currently being treated.
The vast majority of PAH patients are treated in specialist centres, of which there are six in England and one in Scotland. The centres were designated by the Department of Health in 2001, are recognised internationally as centres of excellence and are world leaders in the development of PAH treatments. As a result of the expertise developed in the specialist centres, PAH patients’ quality of life and life expectancy have improved significantly. Many patients who had to give up work have been able to resume their careers. Women with PAH have been supported successfully through pregnancy, which might have proved fatal a decade ago. Other patients have lived long enough to have transplants. Those life-transforming effects have been achieved through the skill and dedication of expert doctors and nurses working in specialist treatment centres and developing drug therapies that are effective in tackling this life-threatening condition.
Last year, the Department of Health asked NICE to appraise the main drug therapies for adults in specialist centres: epoprostenol, iloprost, bosentan, sitaxentan and sildenafil. The NICE appraisal committee published its preliminary recommendations and asked for comments by 20 March 2008. NICE recommends sildenafil for the treatment of PAH in adults. Where sildenafil is not effective or causes side effects, bosentan and sitaxentan may be used. Epoprostenol and iloprost, known as prostacyclins, are specifically not recommended for the treatment of PAH. Patients currently using prostacyclins may continue to use them, but the recommendation prevents new or existing patients from moving to those proven and successful treatments or using the drugs in combination with other authorised drugs proven to be effective. The recommendations do not apply to children.
Patients and clinicians have reacted to the preliminary recommendations with shock and disbelief. According to NICE, its recommendations that the use of prostacyclins be stopped are based purely on grounds of cost-effectiveness. The NICE appraisal committee states in its report:
“The Committee reviewed the clinical effectiveness of each of the technologies under consideration. It agreed that studies had demonstrated the efficacy of intravenous epoprostenol, inhaled iloprost and oral bosentan, sitaxentan and sildenafil in improving exercise and functional capacity and symptoms of PAH relative to supportive care alone.”
NICE does not question the clinical effectiveness of the drugs; the problem is the cost of prostacyclins. They are expensive drugs. Even though the price has fallen somewhat, annual drug costs for some patients may exceed £100,000. In NICE’s opinion, because quality-adjusted life year costs exceed its recommended £20,000 to £30,000 threshold, the treatment is not cost-effective.
I congratulate my hon. Friend on securing this debate. Like many people, I have been contacted by constituents, including Anna Baker, with pulmonary arterial hypertension, even though the number of PAH patients in my constituency is small. They make the point that for a relatively rare disease—although PAH is probably under-diagnosed due to its non-specific symptoms—it is not appropriate to use the £30,000 threshold. If the number of diagnoses were higher, the unit cost of treatment would no doubt fall to a level that NICE might find acceptable. Does my hon. Friend agree?
My hon. Friend will recall that at a meeting held in the House a few years ago, the manufacturer of the main beneficial drug justified its price not according to the costs of production but according to the drug company’s problems—I believe that some of its other major drugs had failed and, in order to stay solvent, it had to charge an extraordinarily high price for the drug. Is it not true that, in many cases in which NICE must attempt to set a limit, the problem is not with the drugs’ efficacy but with their excessive cost, which is intended to provide solvency to drug companies? Would it not be better if the patents were handed out to other companies so the drugs could be sold for a price closer to the cost of production?
That is an interesting way of looking at it, but we must remember that drugs cost many millions of pounds to develop. I shall touch on ultra-orphan drugs, which include the drugs that we are discussing. NICE and the Department of Health should consider such drugs differently, and perhaps even support their production, so that people with rare conditions such as PAH are not prevented by the cost of the drugs and the scarcity of patients from getting a life-saving treatment.
I welcome you to the Chair, Mrs. Dean. I congratulate the hon. Member for Carmarthen, West and South Pembrokeshire (Nick Ainger) on securing this debate. In relation to drugs and the treatment of PAH, does he know which of the drugs considered by NICE are used regularly in the United States and other countries?
I am well aware, as I am sure NICE is, that the drugs are used universally for the treatment of PAH. I am also aware that the cost of prostacyclins in the United States eight years ago was between one eighth and one tenth of the price in this country. There was a huge difference in price. Epoprostenol—its brand name is Flolan—which is manufactured in GlaxoSmithKline’s plant in Dartford in Kent, was exported to the United States, where it was one eighth to one tenth of the price charged in this country. The situation has improved, but, as my hon. Friend the Member for Newport, West (Paul Flynn) said, the unit price of such drugs is an important matter, to which I shall return later.
On 10 March, the Pulmonary Hypertension Association UK organised a hearing in the House of Commons that took evidence from patients, clinicians, patients’ advocates and a health economist. I am sure that hon. Members who attended that hearing will wish to catch your eye later, Mrs. Dean. The patients and clinicians who gave evidence explained that, for patients with advanced PAH, prostacyclins are the only effective treatment and that without them they would die—there is no alternative.
Ms Sam Kahn, who is a PAH patient, described the history of her diagnosis and treatment, illustrating how she started with relatively inexpensive treatments, but how, as her condition worsened, those early treatments became ineffective. She now uses inhaled iloprost, which NICE recommends should not be used. However, thanks to that treatment, she has managed to keep working and has a good quality of life. Professor Andrew Peacock, of the Western infirmary in Glasgow, which is one of the designated specialist treatment centres, told us:
“We are concerned that if you take away the best drug, the patient still has to be treated—they will be in hospital very sick, having other sorts of therapies but not the one that they need. The cost will still be large, it may be even larger.”
Ian Armstrong—he is here today, listening to our debate—is chairman of the Pulmonary Hypertension Association UK, and a specialist nurse at the Royal Hallamshire hospital in Sheffield, which is also a specialist treatment centre. He told us:
“If we are not careful, the NICE proposals will allow any clinician anywhere in the country to say, ‘All we can do is give you a cheaper medication, Sildenafil, and if that does not work then there is nothing else we can do’”.
He also quoted a survey of PAH patients in which 83 per cent. of respondents said that their symptoms and quality of life had improved since being treated with prostacyclin.
Lisa Needham, an NHS manager at the Royal Hallamshire hospital, said:
“If we are not going to keep that specialist expertise, why have we invested for 12 years or 14 years in developing the world renowned care that we…in the UK”
have delivered in our specialist treatment centres? Finally, Adam Lloyd, a health economist, told the hearing:
“NICE has recommended withdrawing the prostacyclins, which have been the treatment of choice for almost 15 years and there is no recognition that this is not a case of preventing a new therapy or of preventing a new drug from coming into use, we are actually withdrawing something that has been the standard of care.”
I am not here to attack NICE, which does a very difficult and important job, but the recommendation is wrong. I am sure that all Members agree that NICE should appraise the cost-effectiveness of treatments—no one would dispute that it should do so—but when there is no alternative to a proven, clinically-effective drug used to treat a very rare condition, different criteria should apply. Such drugs are known as ultra-orphan drugs. Indeed, the principle that such drugs should be appraised differently was accepted by NICE, but it has not been applied in the appraisal that we are discussing.
Paragraph 4.9 of a NICE paper dated 16 March 2006 and entitled, “Appraising Orphan Drugs”—it is still available on the NICE website—states:
“If the Institute is to appraise ultra-orphan drugs, and be prepared to accept substantially higher ICERs [incremental cost effectiveness ratios] than those hitherto considered to be cost effective, then separate decision rules (ie the range of ICERs considered ‘cost effective’) will need to be developed and adopted for these products. The Institute proposes that these ultra-orphan drug decision rules are based in the ICERs of those ultra-orphan drugs currently on the UK market.”
“The further work would determine a rationale for a new ultra orphan-drug threshold. However, it appears that at current prices indicative ICERs for ultra-orphan products are in the range of £200,000 to £300,000”—
not £20,000 to £30,000—
“per QUALY [quality adjusted life year] (ie a ten-fold increase on the decision rules currently applied in conventional appraisals).”
I have three concerns about the appraisal committee. First, inexplicably, it made no mention of the ultra-orphan status of PAH prostacyclins. Under NICE’s own criteria, prostacyclins are orphan drugs, and its March 2006 draft report would permit their continued use. The use of the £20,000 to £30,000 threshold for common diseases such as diabetes is understandable, as millions of patients are involved, as my hon. Friend the Member for North-West Leicestershire (David Taylor) said. However, it is not right to do so when only 1,500 patients receive treatment for PAH, fewer than 400 of whom use prostacyclins either as a monotherapy or in combination with other drugs.
My second concern about the report relates to the way in which the appraisal covers the clinical use of the two drugs. Specialist centres assess what is the right therapy for the individual patient. In the majority of cases, particularly where there has been an early diagnosis of the condition, other therapies are used. Prostacyclins are not the first drug therapy to be used, unless the patient’s PAH is well advanced, and cheaper therapies are used until they are no longer effective. However, the appraisal evaluates the prostacyclins as first-line treatments and compares them with the cost-effectiveness of other therapies. However, that is wrong. The treatment of PAH changes as the condition changes, and according to the patient’s reaction to the treatment. The appraisal does not take that into account.
My third major criticism of the appraisal is that prostacyclins have been used in the treatment of PAH for 15 years. It is the main weapon used by clinicians to tackle the disease in its later stages. Clinicians are genuinely shocked that NICE is removing an effective treatment from their armoury, and they are not aware of any other decision that removes drugs that NICE accepts as being clinically effective and for which there is no alternative.
It is estimated that the cost of using all available treatments, including prostacyclins, is £20 million to £25 million a year. All that clinicians and patients are asking is that the best available treatments be used. They want to be able to use all the tools in their toolbox, as my hon. Friend the Member for Stockton, North (Frank Cook) put it at the hearing on the 10 March. Prostacyclin’s effectiveness was shown in a withdrawal trial. Clinicians do not like withdrawing treatment, but in that trial, all patients who had their prostacyclin withdrawn showed a significant deterioration in their condition before the treatment was restored.
I recognise that NICE is independent—it should remain so—but I ask the Minister to raise with it a number of issues: first, why were those drugs not treated as ultra-orphan drugs in the appraisal? Secondly, will she ask it to reconsider how prostacyclins are used in the progress of the disease? Thirdly, will she ask it to explain what effective alternative there is to this treatment? I also urge her to raise with NICE the fact that every expert clinician in our treatment centres is appalled at the prospect of ending the use of prostacyclins. They are the acknowledged experts, and only use prostacyclins when that is in the patient’s best interests. They are well aware of the costs involved and do not prescribe them unless they are effective and there is no alternative. They are saying, quite simply, that if the preliminary recommendation is confirmed, not only will patients’ quality of life suffer, but their lives will shorten substantially.
One worry that I have is about the knock-on effect in the care sector. A friend of mine does not get all the care that she deserves, but if we downgrade the condition, people will need additional care. I worry about whether the care agencies will recognise that fact or further downgrade the condition.
I do not know whether my hon. Friend’s friend uses prostacyclin, but if prostacyclins are not available to clinicians to prescribe, there is no question but that the quality of life and the condition of PAH patients will decline dramatically. Their care, whether it is delivered at home or in hospital, will increase substantially, so there will be an enormous increase in the resources needed to care for them. It is a serious issue, and it is a cost that the NHS and social services have to bear. I do not think that the appraisal committee has taken that factor into account.
That is the dramatic difference that the treatment can make to people’s lives. Not only does it improve the quality of their life, but it keeps them alive. That is what has shocked those expert clinicians, who are world leaders in the treatment of the condition: NICE, without proposing an alternative, has suggested that the drugs be withdrawn.
I wonder whether the hon. Gentleman is aware of the Raynaud’s and Scleroderma Association, which was founded in my constituency and based there. Only a very small group of people suffers from those conditions—scleroderma in particular—and the drugs to which he refers are the only ones that can give any relief at all. The alternative is death. Will he ask the Minister to explain when she makes her winding-up speech why, on that one and only occasion, NICE has denied patients a life-saving drug when it has not done so for any other condition?
Indeed. I think that I have already made that point to the Minister. I have great sympathy with her in this case, however, because so far, it has nothing to do with the Department of Health; it is purely a preliminary recommendation by the NICE appraisal committee.
The Minister must be aware that if the decision is confirmed, it may well—returning to the point that I made to my hon. Friend the Member for Newport, West—discourage the pharmaceutical industry from investing in research to find treatments for rare conditions such as PAH. If that is the climate in which orphan drugs are treated and NICE does not take account of the far higher costs of treating rare conditions such as PAH, what incentive is there for the industry? My hon. Friend the Minister should consider asking her officials to open negotiations with the two drug companies, GlaxoSmithKline and Bayer Schering Pharma, to see whether the price of the drugs can be reduced.
I became involved with the Pulmonary Hypertension Association because of a constituency case. One of my constituents, the father of a seven-year-old patient, wrote to me saying that he was very worried that the funding for epoprostenol, the drug that was keeping his son alive, would stop. That was in 1999, and he said in his letter that the drug cost £120,000 a year. I wrote to my friend, Jon Owen Jones, who was the Wales Office Minister with responsibility for health, and I put a PS at the bottom, saying, “I’m sure he’s got it wrong. It must be only £12,000.” Jon Owen Jones wrote back to me, saying, “No, the figure is correct, and with the likely increase in dosage, next year the price for the drug will be £165,000.”
I took up the case and discovered, as I said to my hon. Friend the Member for Newport, West, that the price of that drug in north America was between one eighth and one tenth of its price in the United Kingdom. I made an arrangement with the pharmacist at Withybush hospital in my constituency, and we started to import drugs from north America at one eighth of the price that was quoted in this country. There is room for serious negotiation. I know that the price has come down; I do not dispute that. I know also that, because they are ultra-orphan drugs, they are extremely expensive. However, the price that is quoted in the NICE appraisal must be examined to check that it is the actual price, because the appraisal committee’s report refers to the fact that prices are negotiated locally. I cannot see why we should not have national negotiations for an ultra-orphan drug. We do not leave it up to individual pharmacists or hospitals to negotiate a separate deal, so surely, with only 400 patients, we should be able to negotiate a far lower price with those two companies, and perhaps compensate them by putting a penny on the price of aspirin or on something else.
I congratulate my hon. Friend on securing the debate and on his sterling work over the years on this important issue. He makes the eminently sensible suggestion that we should negotiate a national price, but would he consider negotiating an international price—perhaps throughout the European Union—and using EU buying capacity to reduce the cost of the drugs?
I do not care whether negotiations are national, international, universal or whatever. The important thing is that they are ultra-orphan drugs that are used on a very small number of patients. That is the only difference. The things work. There is no question about the clinical efficacy of the drugs; it is purely about their unit costs. If that is the issue, we need to address it nationally, and ideally, internationally.
I am fascinated by my hon. Friend’s enterprise as a drug importer, and I should like to know how successful it was. My hon. Friend the Member for Vale of Clwyd (Chris Ruane) points out that there is an international market in drugs, which are accessible through the internet and other sources internationally. Is it not possible to get around the extraordinary difference in the price that GlaxoSmithKline charges in this country and in America? Is it possible to do so by investing in the enterprise to which my hon. Friend the Member for Carmarthen, West and South Pembrokeshire (Nick Ainger) referred? If so, I shall buy shares in it first thing tomorrow.
Circumstances have changed since 1999-2000. Nevertheless, there is no question but that at the time the arrangement saved the national health service about £120,000 on that one patient, compared with the price that it would have paid the following year. The situation illustrates that throughout the world, different prices are charged for the same product. The irony was that we imported from the United States something that had been produced 250 miles away in Dartford, Kent.
That was nonsense. It was obscene that a system could operate whereby in the United States, which is not a developing country by any stretch of the imagination, the cost was one eighth to one tenth of the price in this country, because of the arrangements that were made there. I also learned that, for individuals who are on Medicaid in the US, once their Medicaid ended, the deal was that the drug supplier would give the hospital the prostacyclins free of charge, which was a very generous arrangement. Perhaps that arrangement is indicative of the different deals that can be done with the drug companies.
For 15 years, prostacyclins have significantly improved quality of life and extended the lives of PAH patients. If the drugs are withdrawn, patients will die unnecessarily. To withdraw prostacyclins on the basis of a flawed appraisal and prevent their future use is medically unsupportable, and I trust that NICE will take on board all the submissions that are made today and reverse its preliminary recommendations.
May I congratulate the hon. Member for Carmarthen, West and South Pembrokeshire (Nick Ainger) on securing this debate and on the very balanced and reasonable way in which he has advanced his case? I attended the meeting to which he referred in his speech and I am a sponsoring signatory of the early-day motion that he has tabled.
This is a very important debate. The Minister, with her nursing and medical experience, will take what is said today very seriously and I hope that she will ensure that NICE is made well aware of the strong feelings on this subject in the House of Commons.
As the hon. Member for Carmarthen, West and South Pembrokeshire said, there is understandably great concern among the health community and the pulmonary arterial hypertension community in this country about future treatment in the United Kingdom for this rare—he was right to describe it as such—but deadly disease. Patient organisations are not only disappointed but extremely concerned for their members about NICE’s preliminary recommendations on the treatment of PAH.
To take up another point made by the hon. Gentleman, it would appear that there has been a complete lack of understanding of the implications for patients, particularly those with connective tissue disease-related PAH, such as Raynaud’s and scleroderma, to which my hon. Friend the Member for Congleton (Ann Winterton) drew attention in her intervention.
PAH is the greatest single cause of death in people with connective tissue diseases, of which scleroderma is perhaps the most deadly. It causes a most distressing form of death. Until approximately 10 years ago, the only standard treatment was parental prostanoids. As the hon. Member for Carmarthen, West and South Pembrokeshire said, intravenous prostacyclin still remains the accepted therapy for very advanced cases of PAH in all treatment centres, not only in the UK but in the United States of America, Canada and many European countries.
Hope, in the form of oral therapies, has made a tremendous difference to a patient’s quality of life. Therefore, endothelin receptor antagonists, or ERA, should be the first line of treatment for use in cases of connective tissue diseases associated with PAH, for which it is considered to be the best treatment option. When patients deteriorate despite oral therapy, they should have the choice of using intravenous prostanoids or possibly combinations of oral therapies, as evidence of their success increases.
The hon. Member for Carmarthen, West and South Pembrokeshire has already stated this, but let us be clear that we are only considering a very small number of patients—approximately 1,500—of which a third have connective tissue disease. Without the option of prostacyclin, patients will have no option other than palliative care when their condition deteriorates. They will then be condemned to six to 10 months of what one can only describe as a miserable existence: being breathless; suffering progressive right heart failure; and experiencing recurrent prolonged hospital admission, with all the costs that the hon. Gentleman outlined in his speech.
That would not be the case if the treatments shown to be effective in other countries were standard in the United Kingdom in future. NICE has accepted that the therapies are effective; again, I take up a point made by the hon. Member for Carmarthen, West and South Pembrokeshire. The problems arise from the assertion that there is no difference between scleroderma and idiopathic PAH in terms of individual drug efficacy or modelling of cost-efficacy and that the appropriate cost comparator should be palliation. The standard therapy is intravenous prostanoids, which was proven more than 10 years ago to be effective, and which all guidelines accept is the gold standard therapy for the condition—I highlight that to the Minister.
As the hon. Gentleman also said, the model developed by NICE to determine cost-efficacy is not fit for purpose; I stress that to the Minister. The NICE appraisal acknowledged that the model failed to predict the actual outcome in the treated population. In fact, the national survey shows a 6 per cent. mortality rate every 12 weeks for patients with class 3 scleroderma-associated pulmonary hypertension on treatment. Surely it is totally irrational to choose to ignore real data and to determine the treatment of the condition on the basis of a model that is acknowledged to fail to accord with reality.
The PAH centres in the UK are the envy of the world, as was stated at the well-attended and well-informed meeting organised recently by the hon. Gentleman. However, should the NICE recommendation be implemented, very sick people will continue to die unnecessarily. Surely the Minister must accept that message and take it to NICE.
How can NICE even consider taking away the only hope from this small group of patients, who, through no fault of their own, have developed a highly lethal condition? It is tantamount to condemning them to a death sentence. That is a very harsh thing to say, and I am sure that that outcome is not what NICE wants, but if its recommendation is acted on that is precisely what it will be doing; it will be condemning a small number of people to a death sentence.
With that small patient population, it is estimated that appropriate access to all available treatments would cost the NHS between £20 million and £25 million per year; again, I quote the figures given by the hon. Gentleman. Surely that is a very small sum. Moreover, patient outcomes are vital. The importance of getting people back to work, with their subsequent financial contribution to the economy as a whole and the reduced costs for the NHS, together with the patient’s improved quality of life, are factors that do not appear to have been taken into account at all. That is an oversight, and a very sad one.
NICE must be urged to review its provisional guidance to ensure that patients continue to receive life-saving treatments and to maintain the UK service record as the envy of the world in this area of health care; that status was highlighted at the meeting to which the hon. Gentleman referred in his speech. I trust that NICE will reconsider this unbelievable and extremely serious recommendation and make patients, not just money, its main priority in its considerations on this critical matter.
This is the first time that I have had the opportunity to speak under your authority, Mrs. Dean, and I promise to do my utmost not to attract your displeasure.
I, too, offer the customary congratulation to my hon. Friend the Member for Carmarthen, West and South Pembrokeshire (Nick Ainger) on securing the debate, and I couple that congratulation with a commendation for his determination, because it is not easy to acquire these opportunities. Sometimes, one has to try and try again, and he has been an exemplar in that regard. I commend him heartily for that.
My hon. Friend said that he did not want to attack NICE, but I cannot promise to maintain the same degree of passivity. I have tangled with NICE before, when it issued its diktat that drug-eluting stents should not be used any more. As I have three of them in my left anterior descending artery, I felt a bit peeved about that decision. They might need replacing in the future, but the fact that they are drug-eluting has made their replacement less likely. I also thought that everyone, including me, should be able to have the same degree of comfort. Having taken issue with NICE, I looked at its systems of econometric assessment and found them to be somewhat lacking. The issue that we are discussing today is a similar example of accountants having gone mad. We have heard from my hon. Friend the Member for North-West Leicestershire (David Taylor), who is a well qualified accountant, but who is different from all the other accountants whom I have met in that he has a degree of compassion. Most accountants do not, so I think that we should put him in charge of NICE or in a similar position.
There is a mega weakness in NICE’s attitude to assessment. I am sure that it approaches its medical analyses in a professional way, but perhaps it gets so bogged down with them that it forgets other aspects. It is our job, in this place, to fight for individuals in our constituencies and throughout the nation, and it is with that intention that my hon. Friend the Member for Carmarthen, West and South Pembrokeshire secured the debate. I am pleased to join him in seeking to push the issue forward.
Thomas Woolley is 13 and a half years old, and he lives in my constituency on the Wynyard estate. His parents wrote to me and asked me to attend a meeting, which was convened by my hon. Friend. I had to struggle to get there; we always have lots of petitioners and people who are lobbying on this, that and the other, and it is not always possible to find out how important a matter is until one gets there. Had I not received a letter from Mr. and Mrs. Woolley, I probably would not have arrived at the meeting, but when I got there, I was stunned by the presentation, which was remarkably controlled. It had a great impact on me that people who live with such a problem could relate their difficulties in such a measured manner and with such politeness. That is why I have committed to give every kind of support that I can summon, meagre though it be.
I got another letter from Mr. and Mrs. Woolley, but it was not addressed to me. It was addressed to the hon. Member for Romford (Andrew Rosindell), who kindly passed it on. With your leave, Mrs. Dean, I shall read it out. It says:
“Dear Andrew, we came to ask for your help some time ago with regards to the Pulmonary Hypertension Association. At the time, we were living at 25 Albert Road, Romford, and our son Thomas had Pulmonary Hypertension. Since we last saw you we have moved home and on October 26th 2007, Thomas had a double lung transplant at Great Ormond Street Hospital. He has made an excellent recovery and is living a full and active life now. Once again we are asking for your support. If preliminary recommendations by the National Institute of Health and Clinical Excellence (NICE) are implemented, then drugs like Epoprostenol will not be available. As you know, this drug allowed Thomas to live a good quality of life for four years up to his transplant”—
I reiterate that point: the drug allowed him to live a good quality of life up to his transplant—
“and it would be a dreadful thing if other patients were not given this opportunity because of the expense of the drug. We hope this letter finds you well”.
If NICE had taken its decision five years ago, Thomas Woolley would be dead now. Is that the kind of health service that we display such pride about? Is it the kind of decision that NICE ought to be capable of? Are we going to remove tools from the toolbox that consultants normally have available, and say, “You can’t do that. There’s a life there, but you can’t save it”? How can we justify that?
The chief of the NHS gave evidence to the Select Committee on Health on 22 November 2007, which is not long ago—just three or four months. He said:
“I am trying to manage the resources of the NHS against a vote that Parliament gave me, and that is what we are trying to do in the most effective and efficient way. What I can say is that…decisions…can now be taken wholly on clinical grounds, not on the basis of whether we have the resource.”
If you will permit me, Mrs. Dean, I shall repeat that:
“What I can say is that…decisions…can now be taken wholly on clinical grounds, not on the basis of whether we have the resource.”
Our independent colleague, the hon. Member for Wyre Forest (Dr. Taylor), who is a professor of medicine, immediately piped up with the reply:
“Because there is enough money to do it on clinical grounds.”
Mr. Nicholson’s response was:
“The NHS, by its good management and organisation, has created a healthy financial position for most of the country.”
That is a pretty firm statement, in my catalogue—a healthy financial position for most of the country.
So, what are we about? We have heard some interesting suggestions about taking up drug trafficking, which has apparently been done very successfully. I wish that I could have bought a few shares in that. My hon. Friend the Member for Newport, West (Paul Flynn) made the even more effective suggestion that we should take more effective measures in that direction. We must certainly question NICE’s assessment. The response that I eventually got from the Minister—not this Minister but a different one at the time, although it was not so long ago—after a fairly long delay was that the evidence that NICE had examined when it came up with the drug-eluting stent decision and restriction was being re-examined. I hope that NICE has learned from that lesson and in future will check every kind of evidence that is placed before it with a view to arriving at decisions that are sustainable afterwards. The drug-eluting stent decision was not sustainable, and the recommendations on epoprostenol and similar drugs are equally unsustainable. I hope that NICE thinks again about its decision and that the Minister insistently recommends that it does so.
I did not intend to speak, but some balancing comments should be made about the National Institute for Health and Clinical Excellence. The debate seems to involve another attack on NICE for making a decision that none of us would like to make. We have set up NICE to shift away from politicians the impossible choices that have to be made under a limited health budget. It always is limited, but we do not want to believe that. It is unfair to put all the blame on NICE, or to suggest that it does not put patients first. I am sure that it does, but it must put itself in a position where it can challenge the pharmaceutical companies.
My hon. Friend the Member for Carmarthen, West and South Pembrokeshire (Nick Ainger) mentioned the usual story from pharmaceutical companies that they have to charge large amounts for some drugs because few clients use them, it is a small market, and they have to pay for the cost of research. That argument might be convincing if they, particularly GlaxoSmithKline, did not spend a great deal more on marketing drugs than they do on researching them. They spend a large amount on research for drugs for stomach pains, headaches and those ailments that involve huge demand but very little on research into drugs for ailments that affect a small number of people.
There can be no justification for blaming NICE if it asks the pharmaceutical company, “How on earth can you justify this life-saving drug”—which can offer so much, as we heard today in moving stories—“which is made in Britain, costing 10 times as much outside the factory gate as it costs on the other side of the world?” That cannot make sense, except for commercial reasons.
I repeat what I said in my intervention, because I remember the meeting held by my hon. Friend, who has been persistent and campaigned on this matter for years. I was shocked when we cross-questioned the lady who was running the drug company and she told us the cost. Twenty years ago, many of us would have thought that it was impossible for anyone to have a course of drugs that cost more than £10,000. One would not have believed that possible, but now we hear that the drug companies charge £165,000. What if they were to charge £1 million or £2 million per patient? Would we tell NICE to pay, regardless of the cost?
If children or someone in our constituency were about to be deprived of drugs, we would all say as constituency MPs what my hon. Friend the Member for Stockton, North (Frank Cook) said: “Pay it. It is only £20 million. It is nothing in the context of the health service budget,” but we must take some responsibility for the policy that NICE has to implement on our behalf—a thankless task—and acknowledge that NICE is not the problem. We beg NICE to pay for the drugs—I do not think that it has any choice—but, ultimately, the answer is not to ask NICE to pay whatever is demanded by greedy pharmaceutical companies that need to get their balance sheets right.
I repeat that I support the campaign of my hon. Friend the Member for Carmarthen, West and South Pembrokeshire, which is of long standing. I acknowledge what the hon. Lady says, but we have to take on what has been happening since NICE was formed. Pharmaceutical companies set up cynical campaigns to boost drugs and exaggerate the effects of drugs—not this one, but others.
I investigated a drug on behalf of a constituent who was suffering from a particular form of cancer. The drug was recommended as the cure for it. I said to her, “Go along and see your physician.” I was approached not by the drug company but by a group of its lobbyists who put forward a case for the drug that was wholly dishonest and optimistic. Having made inquiries, I learned that the cost was £16,000 for each patient, that the drug increased life expectancy for cancer sufferers by 12 days, and that it had adverse side effects, including death, in 10 per cent. of cases. The balanced judgment was that the drug, in the state that it was in then, was a poor gamble and certainly not worth investing £16,000.
We are being subjected to marketing and lobbying campaigns by pharmaceutical companies that are likely to destabilise NICE decisions altogether. We cannot constantly say to NICE, “Whatever the drug companies ask, you must pay.” I support this specific case entirely, but I cannot go along with many of the things that have been suggested today about a blanket condemnation of the decisions that NICE has to take. Thank goodness we as politicians do not have to take them.
I congratulate the hon. Member for Carmarthen, West and South Pembrokeshire (Nick Ainger) on leading this debate and on his persistence in the matter.
I cannot rival the hon. Gentleman’s knowledge, but I do know that pulmonary arterial hypertension, whether in the secondary or primary form, is dangerous, debilitating and difficult to treat. It is dangerous because it is often fatal. Life expectancy is not good: the death rate is high. It is debilitating because it destroys ordinary life. We heard some touching stories of heroic endurance. One wonders how people put up with the situation and do so with such apparent cheerfulness at times. The condition is also difficult to treat, because there is no cure, some of the treatment is invasive and good treatment is hard to access. In my constituency, people have to travel as far as Newcastle to get specialist treatment.
It has been suggested that NICE has added to the woes of poor, suffering patients by recommending no further use of prostacyclins, intravenous epoprostenol or inhaled iloprost, which are regarded as gold standard treatments. It is suggested that that adversely affects 25 per cent. of sufferers, particularly those who are most severely or terminally ill.
As has been said, all arguments about NICE tend to follow a familiar pattern. Incidentally, I must put on record the recent welcome change in NICE’s decision about Lucentis. That shows that NICE does occasionally listen to arguments and have second thoughts. However, what usually happens is that a cheaper alternative is held up by NICE and set against an expensive proprietary drug. The reasons that the drug is expensive have been explored in some depth already.
On one side, we have NICE and the primary care trusts, and, on the other side, we have patients, patient groups and the drug companies. NICE will normally claim that cheap alternatives are available—in this case, sildenafil—and that they are as efficacious as the more expensive alternatives. The drug companies and patient groups will argue, with some plausibility, that the more expensive alternatives have special benefits or—this is important—special benefits to particular patients, if not the whole patient group.
No one per se is against NICE, which is a necessary evil. We all must acknowledge that health care is, if not rationed, at least finite, but people will argue with decisions if NICE has not followed its own guidelines and procedures sufficiently rigorously, or if guidelines have been inappropriately applied. That, I understand, is the argument proposed by the hon. Gentleman in favour of ultra-orphan drugs, notwithstanding the cost. In layman’s terms, he suggests that the principles applicable to common complaints should be applied to this cruel and rare disease and its therapies, which is against NICE’s guidelines. On the face of it, he appears to be correct, and his case was extraordinarily well made.
However, I sound a general caution against assuming two things: first, NICE’s infallibility; and, secondly, the total objectivity of its procedures. NICE assessments are undeniably rigorous, and its inspection of evidence is thorough. I have attended sessions at which it inspected drugs, and there was a huge amount of documentation; they were long sessions, during which patients groups were consulted. It seems to be an impressive procedure, but at the end of the day I would say that it is a judgment call. That call is based on the assessment of this strange unit called the QALY—the quality adjusted life year.
The QALY reminds me of the great utilitarian 19th century philosopher Jeremy Bentham, who invented the hedon—a unit of happiness and pleasure. He suggested that all legislative and moral dilemmas could be solved by counting hedons, using what he called a hedonic calculus. One could work out units of happiness in order to discover whether proposed laws would increase or decrease the overall level of happiness. As a method, it has some advantages over parliamentary democracy. However, Bentham’s method led to interminable debates about how to count hedons.
Today, we have the same debate—how to assess the QALY. It is not simple arithmetic; indeed, it is not arithmetic at all. However, NICE and the QALY is the best that we have, provided always that NICE guidance is mediated and offset by patient experience, which is not always the case. Patient experience needs to be judged over time, and it needs to take account of proper clinical caution and casework experience. That is especially important for diseases that are rare and not understood, such as the one that we are debating today. We do not understand pulmonary arterial hypertension as well as we might. We know the symptoms, but we do not know its cause. Caution should be applied to removing therapies that are not obviously redundant or otiose in the light of patient experience and that of the clinicians who care for them.
When we do not understand the cause of a disease, we often do not know why a drug works for some individuals, but not all—or not with a control group. That is why the voice of the patient group must be heard. Thanks to the hon. Gentleman, it will be heard even more loudly. In this case, cautious wisdom genuinely has a place.
May I say what a pleasure it is, Mrs. Dean, to serve under your chairmanship?
I congratulate the hon. Member for Carmarthen, West and South Pembrokeshire (Nick Ainger), as have many other hon. Members, on securing this debate. I was touched by the passion that he displayed on the subject and the enormous amount of work that he has done on it. The debate is not about a new drug, or another NICE problem—something that we often face—but about an existing and established treatment that has been available for about 15 years. As many hon. Members said, the treatment is considered to be the gold standard.
Pulmonary arterial hypertension is one of five sub-types of pulmonary hypertension. It is rare; as the hon. Member for Carmarthen, West and South Pembrokeshire said, it is the result of an increase in pressure in the pulmonary artery. We have not focused much on the symptoms or how it might feel to be a patient with the disease, but it is characterised by fatigue, shortness of breath, chest pains, fainting, swollen ankles and legs and a swollen stomach. Patients often tell of extreme breathlessness and exhaustion.
It is important to remember that rare diseases are often difficult to diagnose. Of particular note with this disease is the fact that people might have been attending their GPs for some time before a diagnosis is made. Before treatment starts, they may have suffered a year or more with symptoms not being affected by the treatments offered. They will see a diminution in their quality life; and, as with any complex and incurable disease, the poor survival rate will be associated with depression. The disease has a profound effect on the family, which is another subject that has not been touched on today.
The debate is about whether NICE is operating within its guidelines. The hon. Member for Upper Bann (David Simpson) spoke of the cost of drugs in the US—an interesting point—and highlighted the much higher price that we pay for prostacylins, and I hope that the Minister will address that point.
My hon. Friend the Member for Congleton (Ann Winterton) mentioned another important group of patients—those who suffer similar disorders such as Raynaud’s and scleroderma. She said that NICE appears to be ignoring its own rules.
My hon. Friend the Member for Macclesfield (Sir Nicholas Winterton) spoke with his usual clarity about the subject. I was, as always, flabbergasted by his expert medical knowledge on such matters, especially as I used to be a nurse. He raised concerns about the preliminary recommendation and emphasised that we are considering a very small number of patients for whom there is no alternative. If NICE’s recommendation goes through, there will be no alternative.
My hon. Friend said that there is no argument about the efficacy of the drugs—NICE does not disagree—but he raised concerns about the modelling that is used by NICE. Although most hon. Members would support much of the work done by NICE, the debate has highlighted and drawn out concerns about the treatment of those with pulmonary hypertension. I hope that the Minister will take that point.
The hon. Member for Stockton, North (Frank Cook) spoke about his tangle with the NHS over stents. I am pleased to hear that his stents appear to be serving him well, enabling him to contribute with clarity and some passion to today’s debate. He reminded us—especially those on the Front Benches, who sometimes forget—that we are here to fight for individuals. He mentioned the case of one of his constituents, and we all appreciate it when Members quote from letters, as it brings home to us the fact that we are fighting for individuals. He said that epoprostenol and iloprost are part of a toolbox, which is a point that no other Member mentioned today. Clinicians have a range of drugs at their disposal, and if we remove some of those from their toolbox they will be less able to deal with symptoms.
The hon. Member for Newport, West (Paul Flynn) raised an extremely important issue, about which I feel strongly. It was good to hear someone speaking up for NICE, which does a good and difficult job. But he also raised the important question of the huge amount of money that drug companies put into marketing and selling their drugs. All Members have at some time been caught up with drug companies pushing particular treatments. Much more work needs to be done with drug companies, which often fund patient groups and work behind the scenes in ways that are not clear. I appreciate that they put a huge amount of money into research and development, but it is notable how much they put into marketing. To some extent, that is one of the challenges facing the NHS. Part of the money that, in fairness, the Government have put into the NHS has gone into drugs budgets.
The hon. Member for Southport (Dr. Pugh) said that, ultimately, however much evidence NICE has, it must make a judgment, which must be offset against patient experience and clinical expertise. Although that patient experience should not overrule its judgment, it is important, especially in relation to rare conditions for which robust evidence of what works is difficult to find. Conservative Members respect NICE’s independence, and it is important that calls on health service budgets are made by experts and not by politicians, who should not interfere. The criteria for assessing not only drugs, but treatments in the NHS, should be their clinical effectiveness and cost-effectiveness. It is also important that NICE conducts its work quickly, openly and transparently, and has the public’s confidence.
The current use of QALYs, which has come up again today, has often been criticised as inflexible. The Conservative party is considering whether larger societal costs should be taken into account. I go back to where I started, and to the impact that such diseases can have on families. The hon. Member for Carmarthen, West and South Pembrokeshire said that if alternative drugs are not available, withdrawal of a drug may incur considerable extra costs in hospital care, the patient losing their job and family members having to give up work to care for the person who is unwell.
Pulmonary arterial hypertension is not the only illness or disease in relation to which effective treatment could save society considerable costs in the long term. Investment up front will sometimes produce a saving further down the line. Therefore, removing some of NICE’s inflexibility is important. It is a shame that we do not always have the opportunity to consider the emotional and humane cost of treating diseases. Sadly, in this day and age, we are fixed on budgets. They are important, but it is also important to put on record that we recognise the profound emotional cost for families who are battling this illness.
We would like NICE’s role to be extended. Although it is not the subject of the debate, it is important to mention that NICE has a wider role in producing evidence and setting guidelines on standards of care that encourage best practice and achieve outcome objectives.
On NICE’s relationship with the drug companies, we want a better dialogue with the pharmaceutical industry, and the hon. Member for Carmarthen, West and South Pembrokeshire referred to that. NICE needs to be aware of what is being developed, what treatment regimes are available and what regimes are being trialled. It should work with drug companies to try to reduce prices. We must understand—I hope that the Minister will address this—why there are huge variations in the cost of drugs. That would help not only us in developing our ideas of where NICE should go, but clinicians and patient groups better to understand why its decisions are made.
We cannot turn the clock back on NICE’s preliminary report on PAH, but it will have caused considerable concern to those who are reliant on the drugs. Life is difficult enough for people who have a rare disease; it is difficult enough if they have an incurable disease; it is difficult enough if the symptoms interfere with daily activities, so that perhaps making a cup of tea is almost impossible. But this preliminary report is a huge added burden for them, given the concern about whether treatments will continue to be available, and, for clinicians, whether they will be available for new patients.
I urge the Minister to keep an open mind about NICE. This has been a non-partisan debate, and I always welcome such debates, but I would like to hear what she is doing and what steps she will take to ensure that NICE remains flexible and is encouraged to work more closely with drugs companies. I would also like to hear her response to the more general concerns about how NICE operates, and to understand why it seems to have ignored the ultra-orphan status of the group of drugs involved. I hope that she will address those matters.
It is brilliant to be here under your chairmanship, Mrs. Dean.
I congratulate my hon. Friend the Member for Carmarthen, West and South Pembrokeshire (Nick Ainger) on securing this debate. I know, as do all hon. Members, that for many years he has been a strong and persistent advocate of behalf of sufferers of pulmonary hypertension. He has actively engaged many of my predecessors, and rightly so, in helping to advance the case for improvements in treatment and to promote awareness of the disease throughout the United Kingdom, together with the Pulmonary Hypertension Association UK. I thank him, too, for sharing the transcript of the recent House of Commons stakeholder investigation, to which many patients, carers, professional staff and MPs contributed. Many of those MPs are present for this debate.
I agree that this has been an emotional and educative debate. Hon. Members who have taken part are senior and experienced, with a track record of standing up not only for their constituents, but on health issues. The hon. Member for Macclesfield (Sir Nicholas Winterton) has apologised for having to leave early to attend another meeting. Serious attention must be paid to what he, the hon. Member for Congleton (Ann Winterton) and all hon. Members who have made a contribution said. One could not help but be moved by the contribution of my hon. Friend the Member for Stockton, North (Frank Cook) about the little boy. I, too, have constituents with the disease. One little girl who had a transplant, sadly did not make it, but had often attended fund-raising events.
Hon. Members have described the effects of pulmonary hypertension, which is a rare and potentially fatal condition that affects the heart and lungs. It requires expert care, expensive drug treatment and sometimes specialised surgery if life is to be prolonged. There has been considerable progress in treatment over the years, and I hope that hon. Members will allow me to explain current services: what we have achieved, how services are organised, how the commissioning of specialised services for the condition has developed and changed over the past few years, and the role of guidance on clinical care. I shall also address the issues concerning NICE.
There are six centres for the treatment of adults with pulmonary hypertension in England, in Newcastle, Sheffield, Papworth, Hammersmith and the Royal Free and Royal Brompton hospitals. Several of those centres run outreach clinics at other hospitals to ensure that patients do not have to travel too far. Those centres offer a high-quality service and are run by dedicated, highly professional clinicians. It is to their credit that more patients are being treated and that outcomes are improving.
The six centres have been nationally designated by the National Specialist Commissioning Advisory Group, which became the National Commissioning Group in 2001, to ensure that highly specialised services are restricted to a small number of expert centres allowing the development and long-term maintenance of expertise in treating rare conditions. Commissioning responsibility has remained with local primary care trusts and specialised commissioning groups. The number of patients at the six centres has doubled over the past five years for two reasons. First, local hospitals are more aware of the condition and refer pulmonary hypertension patients to the centres at an earlier stage in the disease. The diagnosis is improving—I am aware that much more work must be done on that—and we should congratulate professionals and clinicians on the progress that has been made. The Pulmonary Hypertension Association can take great credit for that.
Secondly, patients who are treated at PH centres are surviving longer. I understand the concerns of my hon. Friend the Member for Carmarthen, West and South Pembrokeshire about delays in recognising the signs of pulmonary hypertension and obtaining an initial diagnosis. However, as I have said, I encourage pulmonary hypertension clinicians and the association to continue their efforts in raising awareness across professional boundaries, perhaps through the royal colleges. If my hon. Friend believes that I, as Minister, and the Department can do more to raise awareness, it is our duty to do so.
I am grateful to the Minister for her kind words, which were totally justified, about the effectiveness of the specialist treatment centres. She referred to my persistence in meeting her predecessors. The main issue that I raised with them was the fact that designated specialist treatment centres are still dependent on commissioners to provide the funding for the patients who are treated there. The argument that I put to her predecessors is that Great Ormond Street hospital, which treats child patients with PH, is now centrally—directly—funded. Will she meet members of the Pulmonary Hypertension Association and me to reconsider the possibility of funding those excellent treatment centres centrally?
Of course I will meet my hon. Friend and the people he would like to bring. The more I can learn about this subject, the better I can help.
On the care and treatment of children with pulmonary hypertension, as my hon. Friend has just said, the national network is organised on a hub-and-spoke principle. The hub is Great Ormond Street hospital for children and the spokes for follow-up care are seven centres of paediatric cardiology at Leeds General infirmary, Bristol Royal hospital for children, Freeman hospital in Newcastle, Birmingham children’s hospital, the Royal hospital for sick children in Yorkhill, Glasgow and the Royal Belfast hospital for sick children. I believe that Southampton has just joined that arrangement.
Since April 2007, pulmonary hypertension services for children have been nationally commissioned, and performance has been managed by the National Commissioning Group on behalf on the NHS. The centre at Great Ormond Street has enjoyed considerable success. Published studies show that treatment through the children’s pulmonary hypertension centre at Great Ormond Street has significantly improved survival rates for children with that serious condition. Studies have also reported improvements in psychosocial well-being and return to school. I pay tribute to that excellent service and the leadership of its outgoing lead clinician, Professor Haworth. Nearly all patients are investigated, diagnosed, receive treatment and have their care package organised by a multi-disciplinary team on site. All the services essential to the care of pulmonary hypertensive children are consultant-led and delivered at Great Ormond Street.
The centre at Great Ormond Street expects to treat 80 new cases in 2007-08, compared with the 20 new cases that were treated in 2001-02. Its caseload in 2007-08 is forecast to be 418 patients, compared with 60 patients in 2001-02. The Great Ormond Street service reports survival as 96 per cent., 91 per cent., and 83 per cent. at one, two and three years respectively for children. Those results compare favourably with other studies. I do not have the time to discuss the Carter review because I want to spend time talking about NICE. However, hon. Members, particularly my hon. Friend the Member for Carmarthen, West and South Pembrokeshire, will be aware of Sir David Carter’s report and the way in which it has changed commissioning.
On the consensus of clinical guidance, with the encouragement of my hon. Friend and many of my predecessor Ministers for health, lead clinicians agreed to work together to produce a consensus statement for the management of pulmonary hypertension in clinical practice. That has taken longer than originally anticipated, and I am sure that that reflects the complex and difficult nature of weighing evidence and capturing and describing best practice. I was pleased to note that the consensus statement was launched three weeks ago at the House of Commons, and I welcome its publication as a significant step forward in raising standards of clinical practice and ensuring that the best available evidence is taken into account. All the clinicians involved are to be congratulated on their efforts.
Many hon. Members rightly raised the issue of NICE, and I thank my hon. Friends for their praise of NICE and for acknowledging the complexities of the organisation. Today, the main focus has been on NICE’s appraisal of the cost and clinical effectiveness of drugs for the treatment of pulmonary arterial hypertension. The initial conclusions were recently issued with an appraisal consultation document, and stakeholders had until 20 March to submit comments. I learned from my hon. Friend that today is yet another deciding day. The Pulmonary Hypertension Association and clinical colleagues have made submissions to NICE, which should be fully considered by NICE in the next stage of formulating final guidance. Some people welcome NICE guidance and some do not. As has been well highlighted today, we recognise that the institute’s decisions have serious implications for patients and their carers. Such decisions are difficult, but they are made on the basis of the clinical evidence that has been submitted to NICE. Today’s decision is critical.
We ensured that NICE was independent, and it was right to do so. Health Ministers have always maintained that independence is important, and I would welcome Opposition Members’ agreement that that should be the case. At the end of the day, I am in an awkward position, because if that independence is to be retained, with the best will in the world, I can only continue to urge hon. Members to do all that they can, and I will continue to do all that I can.
My hon. Friend may not remember that one of our first conversations in the House was about a patient of mine who required the drug, and he told me about the purchase of drugs from the United States. I have an interest to declare because the headquarters of GlaxoSmithKline is in my constituency—I apologise for declaring that at the end, rather than the beginning, of my speech. I tried to undertake negotiations with GlaxoSmithKline, and with the help of my hon. Friend I was successful in looking at how such things could be negotiated. I do not come to this debate as a Minister who is simply reading a speech; I come to the debate as a former nurse and someone who is well aware of what quality of life is really about, as the hon. Member for Guildford (Anne Milton) and others have described so well.
This year, we are celebrating 60 years of the health service. In the Darzi review, we are looking at long-term and rare conditions, which have been included in the patient pathway. I give hon. Members my word that I will raise the issue of long-term conditions, particularly rare conditions, with Lord Darzi as part of his review. I hope that NICE brings us good news today.