§ 3.27 p.m.
§ Earl Howe rose to ask Her Majesty's Government whether the risk-sharing scheme, designed to allow eligible patients with multiple sclerosis to have access to beta interferon and glatiramer acetate in controlled trials, is working satisfactorily.446
§ The noble Earl said: My Lords, I need not, I think, spend too much time setting out the background to my Question, but a few words of introduction may help.
§ In 1999 the Department of Health and the Welsh Assembly asked the National Institute for Clinical Excellence to appraise beta interferon and glatiramer acetate as treatments for multiple sclerosis. NICE concluded in July 2000 that routine use of these treatments could not be recommended, but following a reappraisal, it issued guidance in early 2002 that confirmed that the therapies were clinically effective for certain groups of patients but that sufficient evidence of long-term cost effectiveness was lacking. Shortly after this, the Department of Health announced the MS risk-sharing scheme. This is a scheme whose aims are to enable all eligible MS patients—that is to say, patients who meet the guidelines of the Association of British Neurologists—to receive treatment with disease modifying drugs and for the NHS to acquire these drugs in a manner which could be considered cost effective.
§ I am sure I was not alone in my utter dismay at the NICE determination in 2002. Nevertheless, the risk-sharing scheme seemed to me then, as it does now, a brave and welcome move by the Government to try to square the circle. The targets set under the scheme were for between 7,500 and 9,000 eligible patients to receive treatment with disease modifying therapies by November 2003, with 5,500 to 7,000 in the formal monitoring cohort. Clinical trials of their very nature last only two to three years, which means that the long-term benefits of MS treatments cannot be discerned with any accuracy from such trials. MS evolves over decades and it is therefore reasonable to evaluate the costs and benefits of the treatments over a period of 10 to 20 years. Any shorter period than that and we run the serious risk of failing to uncover possible long-term benefits. Also, the cost-effectiveness of the drugs will appear to be commensurately lower.
§ The risk-sharing scheme therefore represented a huge opportunity not just to demonstrate the long-term benefits of these therapies but to help real patients. The international comparisons are stark. In 2002, the proportion of MS patients in the UK receiving disease-modifying therapies was 6 per cent. In the EU as a whole, the figure was 28 per cent and in the United States it was 46 per cent. One of the looked-for benefits of the scheme was to redress that imbalance.
§ Unfortunately, the whole process got off to a very slow start. In many areas there was simply no infrastructure to get it off the ground—clinic space, administrative support and appropriately qualified clinicians and nurses were simply absent. There was a large backlog of patients to assess, and quite a number of PCTs did not have a precise idea of how many patients there actually were. Even one year into the scheme, many qualifying patients were still waiting for the drugs to which they were entitled.
§ The main reasons were stretched PCT budgets; systems that had not really been well prepared to cope with administering the scheme; and over-burdened 447 neurologists struggling to keep up with the workload. However, by late 2003 progress had been made. At the end of October, about 7,000 patients were being treated with a disease-modifying therapy—some 500 below the original target. Nevertheless, more than 3,000 people had been enrolled into the scheme over the 18 months from May 2002. Fifty-three new specialist nurse posts had been created, and there were 65 prescribing centres for multiple sclerosis around the UK, since increased to 67. All that is positive, but it really is not positive enough.
§ We are now more than two years into the scheme. After this length of time, we really would expect to see it working in the way that everyone had been hoping for, but I am sorry to say that it is not. The scheme was the subject of a formal ministerial direction. It is therefore mandatory. The detail of the scheme has the status of NICE guidance. Dedicated funding was made available to PCTs to implement it. Despite this, it is clear that many PCTs have relegated the scheme to a box marked "low priority".
§ Of the 67 prescribing centres, one—namely, Truro—is still not prescribing disease-modifying therapies at all. Of the rest, Aberdeen has not begun to recruit to the risk-sharing scheme, Leicester and Gloucester have stopped recruiting because of resource constraints, Swansea has not started, although it looks as though it may in the next couple of months, and North Staffordshire has a very long waiting list. Most serious of all is what appears to be a giant misunderstanding about the scheme targets. It is clear that some PCTs are under the mistaken impression that once the target for recruitment to the monitored cohort has been met—that involves 5,500 to 7,000 patients—the shutters can come down and no new patients need be given the therapies.
§ We understand from the Government that the patient number targets that were originally set are likely to be met later this year—about a year late. That target cannot be, and was never intended to be, the end of the story. This is a programme that places an obligation on PCTs to continue funding new access to treatment for 10 years. That obligation must be met for the sake of all MS patients.
§ One of the prime bottlenecks in rolling out the risk-sharing scheme is the chronic shortage of neurologists. In the UK, we have only one neurologist for every 177,000 people. In France, the ratio is four and a half times better—one in 38,000; in the Netherlands, it is nearly seven times better; and, in Denmark, it is eight times better. We are miles behind the bare minimum ratio recommended by the Association of British Neurologists of one in 80,000.
§ The key point here is that getting into the risk-sharing scheme in the first place depends on being seen and assessed by a neurologist. A lot of patients cannot get to see a neurologist at all. Someone in that position must resort to their GP, who almost certainly does not have any in-depth knowledge of MS and is unable to assess them for their eligibility to receive the therapies. The potential tragedy of the situation is this: should an MS patient suffer a significant delay in access to 448 treatment, he or she may be unable to benefit from it. The earlier we treat someone with MS, the more effective the treatment is. If someone suffers a progression of his disability, it may be irreversible, and the window of opportunity is lost. The risk-sharing scheme may have helped to iron out part of the postcode lottery of funding for drugs, but there is still a considerable regional variation in access to neurologists. In 2001, the Emergency Medicine Journal highlighted those wide regional differences.
§ In 2004, the percentage of MS patients in the UK receiving disease-modifying therapies is still only 8 per cent. In the EU as a whole, it is 35 per cent, and in the United States, it is 50 per cent. Let us, for the sake of simplicity, ignore the United States. What on earth can count as a satisfactory rationale for the gap between ourselves and the rest of the EU? There is no such rationale. The monster gap calls into question the estimates of the number of patients eligible for treatment under the ABN guidelines. It suggests that a great many more people might benefit from the therapies than actually receive them.
The European Parliament recently published a report on discriminatory treatment afforded to MS patients in the EU in consequence of a petition submitted by a UK citizen, Louise McVay. There are some pretty damning conclusions in that report, on which the Minister's comments would be welcome. On the UK, it says that the risk-sharing scheme,
represented a big step forward though it has not managed to resolve the therapy-related problems experienced by the majority of MS sufferers even today".
the disparities between the types of care and support available are much too great both within and between the member states of the Union. This must be remedied by raising the level of care provided across the board, bringing about equality of access as a clear objective. With free movement of persons recognised as a binding principle of the Union, better provisions must be put in place to enable the disabled person, including MS sufferers, with equal rights to treatment in all EU states".
The denial of healthcare to people with disabilities is described in the report as,
a fundamental breach of their human rights",
disastrously on an individual's ability to work, on his or her family life, freedom of movement and integration into society".
The designated therapies are recognised as making an "enormous difference" to sufferers and the enhancement of their lives. Securing best practice for equal access to therapies and treatments for people with MS should become a,
principal objective to be obtained by all health authorities in the European Union".
In each member state, there should be,
co-ordinated programmes designed in conjunction with the World Health Organisation".
What official comment does the Minister have on those conclusions?
§ We ought to be getting a regular progress report on the number of patients receiving MS therapies within the formal monitoring arrangements. I should be grateful if the Minister could tell us what the current 449 number is and how many patients have started receiving treatment since the inception of the scheme. How many patients are on the waiting lists for formal assessment?
§ It is not good enough for the Department of Health to adopt a laissez-faire stance on regional variations. Strategic health authorities need to remind PCTs of their statutory responsibilities. Above all, they should stress that the risk-sharing scheme is not a clinical trial about the efficacy of MS therapies; rather it is a study aimed at reducing the uncertainty around previous estimates of their cost-effectiveness over the long term. What action does the department plan to take to ensure that, when the monitored cohort has been recruited, disease-modifying therapies continue to be prescribed under the scheme? That is vitally important. PCTs need to be made aware that the time has passed for the "wait and see how it goes" attitude before treatment is started.
§ This is not a low-priority matter in any sense. Real patients are suffering as a result of systemic blockages, inadequate resourcing and, I think, basic misunderstandings. That situation cannot and must not go on.
§ 3.41 p.m.
§ Lord Clement-Jones
My Lords, the noble Earl, Lord Howe, has made a very powerful speech. He is to be congratulated, not only on giving us the background to some of the problems associated with the risk-sharing scheme, but on initiating the debate in the first place. I hope that the Minister will forgive me if I go over some of the same ground covered by the noble Earl.
Around 85,000 people in the UK have MS. Fifty people, usually between the ages of 20 and 40, are diagnosed with the disease every week. MS is the most common disabling neurological disorder which affects young people in the UK. It affects three women for every two men. The noble Earl, Lord Howe, has reminded us of the history of the issue, which he set out very clearly.
At the time of its publication, many of us expressed considerable criticism of the NICE cost-effectiveness appraisal, which was based on short-term data and short-term benefits. Many of us raised questions and continue to do so about the methodology used by NICE, which, in the case of glatiramer acetate and beta interferon, seems significantly in doubt. That is not the case in many of the other appraisals that NICE has carried out. The reason is that the benefits of disease-modifying therapies in MS stretch well beyond the short term. As the noble Earl described, it can take 20 years for the benefits to be taken fully into account. But the NICE methodology could not, or would not, take that into account. The noble Earl said that that was distressing; it was certainly distressing, and in some cases shocking.
Many of us appreciated very much the Government's rather creative approach when the risk-sharing scheme was set up. It was designed to allow 450 access to treatments, such as beta interferon and glatiramer acetate, despite the NICE appraisal not having accepted their cost-effectiveness.
As early as 2002, problems were identified with the scheme. I have been reminded that in autumn 2002 the MS Society and the MS Trust commissioned opinion leader research to gain an accurate picture of public health bodies' progress in implementing the MS risk-sharing scheme. They set out numerous issues and problems, not all of which, by any means, have been tackled in the mean time. That is the aspect on which many of us wish to probe the Government and call them to account. Having set up such a scheme, raised the hopes of so many MS suffers and, for the very best of motives, found a creative way round the lack of a positive NICE appraisal, it is incumbent on the Government to make jolly sure that, in the almost two years since the survey, they have tackled the problems.
In February last year an article published in the British Medical Journal put it in general terms. It stated:The scheme has several scientific and practical problems that we believe limit its ability to improve the care of patients in the long term".That seems a fairly sweeping statement. Administrative problems, from serious staff shortages to confusion over how to deal with the backlog of patients in some areas, have plagued the scheme from the start. In some areas the scheme was significantly delayed, as we heard from the noble Earl, with some clinicians concerned that MS patients were suffering as a result. Waiting lists have been long, and, as we know, some patients are still waiting to clear them in a number of areas.
One of the key points that the noble Earl made was on the shortage of neurologists. The current figures for neurologist numbers are quite dramatic—one in 177,000, with a proposed modest increase, in an extremely cautious approach by the British Association of Neurologists, to one in 80,000. But, compared to the continental figures of one in 25,000 and one in 35,000, we have a second-rate situation that needs to be tackled. When an MS sufferer cannot access a neurologist within a certain amount of time, as the noble Earl pointed out, he or she may miss out on the risk-sharing scheme if the disease progresses.
The shortage of personnel contributes to regional variances in prescribing and in medical access, although one of the objectives of the risk-sharing scheme was to eliminate the postcode lottery. Access to specialist care is lacking; patients are dependent on GPs, who, despite their best efforts, are not equipped to handle all aspects of such cases and certainly do not have the specialist knowledge of a neurologist. Clearly, the scheme has stretched PCT budgets and processes, demonstrating in another setting how ill-equipped PCTs are to deal with conditions affecting relatively small percentages of the population. We debated specialised commissioning not too long ago. Even the Health Minister in another place, Mr Hutton, has admitted that specialised commissioning is not going 451 as well as it should in the current circumstances, with consortia of PCTs. The same is very true of the situation with PCTs and multiple sclerosis.
Many PCTs are treating MS as a low priority, although, as the noble Earl has pointed out, there is a mandatory requirement to fund medicines under the risk-sharing scheme. Funding levels also vary between regions, pointing continually to the problems with addressing specialist care at PCT level. It is clear that the PCTs are not treating specialist conditions as a priority.
The noble Earl, Lord Howe, gave figures on the use of disease-modifying therapies. We have one of the lowest percentages in Europe. In other countries, 20 to 30 per cent of MS sufferers are on such treatments. The UK is well behind the curve on giving people access to those treatments. The importance of DMTs cannot be overstressed, because rather than purely managing MS, they tackle it, making a huge difference to patients' quality of life. The noble Earl graciously eliminated from his calculations the US, whose figures are staggering compared to those in this country. We are nowhere near the 10 per cent mark, and we have had two years' experience.
We have heard about the assessment centres. Yes, they have been set up, but we should ask why all of them are not prescribing DMTs. It seems quite extraordinary that Truro stands out as the lone exception that fails to prescribe.
Again, some PCTs seem to be under a misapprehension that once recruitment targets for the risk-sharing scheme are met they can stop recruiting new patients into the scheme, whereas of course they have an obligation to fund DMTs for 10 years.
I very much hope the Minister will address the single most important question to reassure MS sufferers and the patient organisations involved, that when recruitment to the monitored cohort has finished, which is likely I understand to be towards the end of the year, those PCTs which do prescribe will not cease doing so. That is a very important reassurance that the Minister can give in this debate.
Generally, the Department of Health and the NHS should be doing a much better job in getting the correct information to those at local level who are responsible for daily implementation of the risk-sharing schemes. Some PCTs still do not believe that they have an obligation to provide these treatments to all eligible patients and others are still under the impression that these treatments are not clinically effective.
It seems to me that the responsibility of getting the correct information to PCTs must be firmly laid at the door of the department and SHAs. It is very important that prescribing information and eligibility criteria needs to be communicated clearly to GPs and specialists responsible for prescribing, so that as many patients as possible can be offered access to these treatments.
To his great credit the noble Earl has been extremely consistent in raising these issues with the Government. I have in front of me the text of a Question he asked 452 exactly a year ago. The Government's response was that they did not hold precise details about how PCTs implement the scheme, and so on.
I hope that what the Minister has heard both from myself and the noble Earl will persuade her that the scheme is not going as well as it should. The noble Earl mentioned some significant progress that has been made, but of course that was all part of the essence of the scheme. Further progress needs to be made. I very much hope that the Minister can give us assurances that this very unsatisfactory situation will be improved.
I do not think that anyone denies that the Government were very imaginative in the way they set up the scheme, but they now really have a duty to make sure that it is properly implemented.
§ 3.52 p.m.
§ Baroness Andrews
My Lords, I am very grateful to the noble Earl for giving us the opportunity to put on record some information on progress and for me to respond to some of the questions raised. The noble Earl has indeed been very persistent on this issue and it is right that we should be able to respond at regular intervals because this is an imaginative scheme, a ground-breaking scheme and very much, as he said, a major opportunity for MS sufferers. We very much see it as our duty to make sure that they receive all the benefits that the scheme is intended to provide for them.
Multiple Sclerosis is a particularly cruel disease. It can target young people and young mothers. It is unpredictable. It has the worst possible combination of features. For many years we have been thrashing around trying to find the right therapies. The opportunity provided by the gift of the drugs that are now licensed for the lapsing/remitting form of the disease is incredibly important. I am again very grateful to the noble Earl for rehearsing the history of the risk-sharing scheme.
I hope that I can address some specific concerns and talk about some other ways in which we believe that progress is being made. Admittedly there is a great deal of progress still to be made, but I believe that over the past few years we have made significant progress.
Four drugs are licensed—the three beta-interferon products (Avonex, Rebif and Betaferon) and glatiramer acetate (Copaxone). Clinical trials have shown that all these products reduce the frequency of relapses and may reduce their severity. Disability progression also appears to be delayed by treatment, but the longer term effects are not certain.
Betaferon was the first product to receive a licence, back in 1995. At the time there were differing views about the evidence from clinical trials. The then cost of around £10,000 per year per patient was high. The dismay to which the noble Earl referred was in part the result of the NICE judgment on cost-effectiveness, but it was against that background of very high cost. Resource implications were left for local assessment. Inevitably the result is variable decision making and what we now refer to as "postcode prescribing".
453 I understand that the petition brought by Louise McVay, reflected in the European Parliament's report of December 2003, had its origins in that sort of problem. As the noble Earl said, the risk-sharing scheme and associated developments are intended to address many of those concerns. He asked me whether I would agree with the statement that best practice for MS sufferers should be a principal objective of every scheme in every country. Of course I would agree with that. Indeed, I do not want to limit my remarks simply to the scheme, I want to describe some of the context and how best practice is being made more accessible and more universal.
Obviously, the key conclusion arrived at by NICE was that on the balance of clinical evidence and cost-effectiveness the treatments could not be recommended for universal treatment of MS in the NHS. However, it went on to recommend that the health department, together with the manufacturers, should consider how to obtain the medicines for the NHS in a cost-effective manner. That scheme was set out in the key circular 2002/004 which was issued widely across the NHS.
All four products are included in the scheme. All have evidence of short-term clinical effectiveness based on randomised controlled trials, but there remain major uncertainties whether these benefits are maintained over the longer term course of the disease. The scheme addresses this uncertainty by setting target outcomes for each product based on the assumptions that short-term benefits are: first, maintained for as long as treatment continues; and, secondly, retained after treatment ceases. Models have been developed which estimate how the drugs might work in the longer term. We need that evidence because, as the noble Earl said, if the drugs delay progression of disability, not only would there be a cost saving, but that is precisely what we want to achieve for MS sufferers. The longer we can keep people mobile and out of wheelchairs, the better. That is what we aim to do. So we have agreed costs to the NHS that could be considered cost-effective if the assumptions are fulfilled. Some prices were reduced to gain entry to the scheme. If outcomes fall short of targets, the scheme provides for further cost reductions "by results". I put that on the record because that is the detail of the scheme.
In terms of the methodology, a cohort of patients will be observed for up to 10 years. It is long-term monitoring, not a controlled trial. We expect that approximately 5,000 patients will form the core of the monitoring group. Their disability will have been assessed at the time treatment starts and be subject to annual assessment thereafter. At pre-determined intervals the outcomes will be analysed and compared to that projected by the model and to data recording the natural progression of the disease in the absence of any drug intervention. The first analysis will be undertaken when at least two years data are available for all patients in the monitoring cohort. On current progress we estimate that results from the first analysis will become available in late 2006. Subsequent 454 analyses will be undertaken at two-yearly intervals. One reason we cannot produce regular annual reports is that we do not have any findings yet.
The scheme has been launched with the full support of the key stakeholders, including the Association of British Neurologists and the MS Society. Both noble Lords talked about delays in the setting-up of the scheme. We have made a significant achievement, but it was held back because there were many practical problems to be overcome—we would not dispute that. Patient recruitment occurred at a slower pace than originally anticipated. The PCTs were just being set up, as were the specialist commissioning arrangements. We had to overcome some of the variability in the postcode lottery, and it has taken some parts of the NHS longer than others to resolve some of the complex staffing and funding issues. I shall talk about that in a while.
Since then, we have had 7 per cent increases per year in funding for the health service. The PCTs are settling down and learning how to do the best job, and now the scheme is seen as part of the NHS architecture. It is an extensive operation and is co-ordinated in a very sophisticated fashion. As the noble Earl said, 67 centres are participating in the study. It is significant that originally we thought that there would be only 30. The fact that there are 67 reflects that local authorities have wanted to have a centre. The centre that is not prescribing—Truro—is likely to start doing so soon.
The centres generally provide dedicated services for MS patients, including assessment and follow-up. More than 8,000 patients are now prescribed treatment. Of those, nearly 4,000 have consented to be monitored as part of the study. The figures can be confusing. Some patients are eligible for treatment—they are able to walk independently and have suffered two relapses over two years; those are the ABN criteria—but are not suitable for the monitoring cohort because their initial diagnosis occurred before the scheme was set up. We lack sufficient details about the extent of disability at the time of diagnosis. An example is young women taking treatment breaks to start families.
Newly diagnosed patients are generally suitable. If current recruitment to the monitoring cohort continues at the present rate of 200 a month, we will certainly achieve the 5,000 target over the autumn. I cannot give the noble Lord figures on patients waiting for assessment, but we genuinely do not think that the numbers are likely to be great. It is a significant measure of success that, before the scheme was started, there were constant complaints to the Department of Health, led by the MS Society itself, about failure to treat. I am assured that those complaints have virtually dried up and that the MS Society is closely involved in the scheme. It obviously seems happy with how things are going.
We will publish regular reports on progress as appropriate, but one problem that is that delays have occurred in reaching agreement with the participating companies on a method to validate the information on patient numbers. We believe that there is an element of 455 under-reporting. It would be very helpful if companies would agree to an exchange of data, which would allow us to know how much of their product they are selling so that we have a better idea of how many people are being treated.
Noble Lords have raised important issues about what has been described as the PCTs seeing the scheme coming to an end when the monitoring cohort is complete. Let me give total assurance on that. Eligible patients not included in the monitoring cohort will, as now, continue to be initiated into treatment under the scheme. Decisions about eligibility and initiation of treatment will continue to be made by specialist clinicians. There will be no artificial capping of the number of patients eligible for treatment, whether they are in the monitoring scheme or outside it. When the monitoring cohort is complete, PCTs will carry on prescribing just as they do now.
I take the argument made by the noble Lords that some PCTs may have missed that point; I do not know that. The matter will be closely watched. Strategic health authorities were mentioned; they have now all appointed lead officers to monitor performance, and we shall monitor the SHAs. We will certainly watch out on the subject of information or for any confusion, as it is clearly very important.
I want to comment on the international differences. Noble Lords are probably right that a great deal more of the therapy is available in other countries. It is difficult to make international comparisons about treatment regimes, because clinical practice alters. We may offer a different mix of therapies from other countries, so I caution against an easy comparison.
It is also worth recording that there have been major developments in dedicated MS services as a direct result of the scheme. I particularly want to pick out the investment that we have made in specialist nurses. The Department of Health is contributing £2.8 million over three years, starting in 2003–04. That is part of a partnership between government, manufacturers and the voluntary service. We estimate that there are now between 150 and 200 specialist nurses in the specialist centres, which is about three per centre. Obviously, that will contribute enormously to the overall quality of care.
Moreover, the Long Term Conditions Care Group is working to increase the number of neurologists. Consultant neurologists have increased by 49 per cent since 1997. There are still gaps, but I assure noble Lords that the Speciality Workforce Advisory Group has been reviewing the number of consultants on an annual basis, along with the professional groups. It has come to the conclusion that there are sufficient higher specialist trainees for there to be enough qualifying for consultant posts to meet estimated future demands.
I do not want to underestimate the problems that shortages create, but we are moving to a better position. Central funding has also been distributed to support the implementation of 10 additional specialist registrar posts. Because physiotherapists are so important in the mix of therapy that we offer, we are 456 also pleased to say that we now have 26 per cent more physiotherapists than in 1997. Physiotherapy training places have gone up by more than 1,000 in the same period. We have addressed the problem. The NSF on long-term conditions, which will be produced next year, will give us another opportunity to address the strategy and review the care offered. So will the modernisation agency, which is also looking, with DoH funding, at how to create better pathways and processes.
I shall end where we began—with NICE. In November 2003, NICE issued guidelines for the NHS on the management of MS in primary and secondary care. They focus on the quality and configuration of services. Essentially, they look at better ways to deliver care. The noble Earl raised the issue of early intervention and waiting lists. Key priorities are that referral to a neurologist should take no longer than six weeks, with a further six weeks for investigations to be completed. We are not at that point yet. We have articulated it; I have said that we are doing what we can to generate greater numbers. But it will be given momentum by the publication of the national service framework next year. Continued progress is very much dependent on co-operation and effective working partnerships and I believe that the risk sharing scheme very much represents that.
In summary, we are encouraged by progress. We take the points that the noble Lords have made. We will keep a close eye on the issues. Treatment is now widely available in accordance with ABN guidelines, where it was not before. The scheme has been the catalyst for major improvements in services. We are seeing successful recruitment into the key specialities and, as I have said, people seem to be much happier that they can rely on receiving the service that they need and which a few years ago they certainly were not receiving. I hope that, with the reservations that have been expressed, noble Lords will feel comforted by my response.