Nick Ainger (Carmarthen, West and South Pembrokeshire) (Lab): I wish to declare an interestI am a trustee of the Pulmonary Hypertension Association UKwhich is listed in the Register of Members Interests. I am grateful for the opportunity to bring to the Houses 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 NICEs opinion, because quality-adjusted life year costs exceed its recommended £20,000 to £30,000 threshold, the treatment is not cost-effective.
David Taylor (North-West Leicestershire) (Lab/Co-op): 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 diseasealthough PAH is probably under-diagnosed due to its non-specific symptomsit 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?
Paul Flynn (Newport, West) (Lab): 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 companys problemsI 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.
David Simpson (Upper Bann) (DUP): 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?
Nick Ainger: 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. Epoprostenolits brand name is Flolanwhich is manufactured in GlaxoSmithKlines 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 diethere 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 treatedthey 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 Armstronghe is here today, listening to our debateis 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.
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
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 treatmentsno one would dispute that it should do sobut 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.
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
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 NICEs 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 patients 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 patients 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. Prostacyclins 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 independentit should remain sobut 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 patients 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.
Mr. David Drew (Stroud) (Lab/Co-op): 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.
Nick Ainger: I do not know whether my hon. Friends 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.
David Taylor: Does my hon. Friend acknowledge the statistic that, correctly diagnosed and treated, two thirds of PAH patients survive for more than five years, and that without that treatment, more than half will die in less than two years?
Nick Ainger: That is the dramatic difference that the treatment can make to peoples 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.
Ann Winterton (Congleton) (Con): I wonder whether the hon. Gentleman is aware of the Raynauds and Scleroderma Association, which was founded in my constituency and based there. Only a very small group of people suffers from those conditionsscleroderma in particularand 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?
Nick Ainger: 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 wellreturning to the point that I made to my hon. Friend the Member for Newport, Westdiscourage 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, Im sure hes 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.
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