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The Royal Society of Edinburgh Joint event supported by The Edinburgh Drug Absorption Foundation and the Ewan

& Christine Browns Charitable Trust Availability of Drugs for the Elderly
Report by Jennifer Trueland (commissioned by the RSE) 30 September 2008 The meeting was held against a background of rising drug costs and tightening NHS resources. Its aim was to encourage public debate about how we decide what drugs should be available and whether this has a particular impact on the elderly. The speakers included those who make decisions of this nature at a national and local level and also one of the UKs foremost health economists. Speakers Professor David Webb As a former chairman of the Scottish Medicines Consortium (SMC), Professor Webb ran through the processes by which the organisation assesses newly licensed drugs. He spoke of the benefits of SMC, which include its fast decision-making and the fact that it was created and is owned by the NHS. Dr Ken Paterson The current chairman of the SMC spoke about health economic models and the complexity behind issues such as cost-effectiveness. He said that while the current systems are far from perfect, they are the best that are available at the moment. Tom Divers The Chief Executive of Scotlands largest health board, NHS Greater Glasgow and Clyde, spoke about life at the sharp end of deciding how to use resources. He described how his board goes about prioritisation and spoke about the challenges of the current financial climate. Professor Alan Maynard Professor Maynard said rationing was inevitable and that healthcare decisions are not made on the basis of efficiency alone; there is an ethical dimension too. He called for governments to negotiate with the pharmaceutical industry to drive down prices, so that they are more closely aligned with the benefits to patients. Q&A The evening stimulated a wide variety of questions ranging from whether patients should be told the price of prescription medicines to the vexed issue of top-up fees or co-payments. Other areas considered included patient education; public involvement; the role of the media; and, the respective merits of the SMC and the National Institute for Clinical Excellence (NICE). Introduction and Speakers Chair: Dr David Lawson CBE, Honorary Professor of Medicine and Therapeutics, University of Glasgow As founding chairman of the Scottish Medicines Consortium and a former chairman of the UK Medicines Commission, Professor Lawson, was well-placed to chair the evening. He began with a short explanation of the current position. Briefly, the UK, as with the rest of the world, is struggling to pay its drug bill. This is particularly the case when expensive new treatments, such as biological therapies, come on stream. While products will receive a licence if they are safe, of good quality and efficacious, issues such as demand, need, cost and affordability are not taken into account in the licensing process. Every country is struggling to deliver, with finite budgets set against near

infinite demand, in populations resistant to raising taxes. The aim has been to make new medicines available and avoid postcode lotteries, he confirmed, but the question is how to achieve this. There are some who believe its better to spend the money buying drugs, than in inventing a new bureaucracy, he said provocatively. Professor David Webb, Christison Professor of Therapeutics and Clinical Pharmacology, Queens Medical Research Institute, University of Edinburgh. Scottish Medicines Consortium; principles and process, elderly-relevant medicines accepted and rejected. Professor Webb, who was chairman of the Scottish Medicines Consortium (SMC) until March 2008, gave an overview of the circumstances which led to the setting up of SMC as well as an explanation of how it works. He cited an Audit Scotland report from 1997 (The Bitterest Pill) which focused on the year-on-year rise in drugs costs within the NHS. At that time, said Professor Webb, Scotlands four large and 11 smaller health boards were each doing their own thing, with varying levels of expertise. This meant duplication of effort and, crucially, led to so-called postcode prescribing because different boards came to different decisions. The SMC was set up to look at all newly licensed medicines around 100 products per year and advise health boards and area drugs and therapeutics committees whether they should be available on the NHS in Scotland. The SMC makes decisions quickly (within 14 week), based on a submission from the licence holder, which is a novel method. Essentially the SMC makes a decision on whether the new product will provide value for money for the NHS, based on cost and efficacy. The assessment process is two-tier and involves a scientific committee of about 20 people, whose findings go to the main committee, which has 30 members, including lay members and representatives from the Association of British Pharmaceutical Industries (ABPI) (representing industry). The main committee revisits the scientific evidence and takes a broader societal perspective, he said. There is also an opportunity for patient groups to submit evidence. There are three potential outcomes: the drug is accepted; accepted with restrictions; or, not recommended. Re-submissions are encouraged and there is an appeals process. Acceptance does not necessarily mean the drug will go into health board formularies (the list of drugs which can be prescribed in each NHS board area). If the Area Drugs and Therapeutics Committee does not feel it will be of use for example, if it is the fourth or fifth me too drug and they already have others in use then it might not go in the local formulary. Around a third of submissions are rejected. In general, the cheaper the drug is, in terms of cost per QALY (quality adjusted life year a term used to judge how many years of good quality life a product will buy for a patient), the more likely it is to be accepted. If cost per QALY is under 20,000 there is an 80 per cent chance that it will be accepted whereas the SMC rejects around 50 per cent of those where the cost per QALY is between 20,000 and 30,000. If the cost per QALY is 40,000, then the chance of rejection is high, he said. These criteria are similar to those used by NICE, but are still well above what health economists tend to say we can afford, which would be more like 10,000 per QALY. (To add context, he said that beta interferon for multiple sclerosis costs 174,000 per QALY). The strengths of the SMC are that it has a bottom-up approach; is owned and was created by the NHS; is flexible; engages with industry; has a single consultation stage; and, makes decisions rapidly. It is also fully supported by the Scottish Government. He pointed out that of the 63 drugs looked at by both NICE and the SMC, the same decision was reached in 83 per cent of cases. Scottish patients get the advantage of getting drugs substantially earlier. The SMC had offered to work more closely with NICE but had been rebuffed. He accepted that the SMC takes an NHS perspective and doesnt look, for example, at the carer cost implications of rejecting or accepting a drug. 2

He also said there is a need to make sure that there is room for genuinely innovative drugs and cited the view of the Office of Fair Trading (OFT), that it might be better to price drugs based on the value one gets from them. He also pointed out that drugs are priced with the US market in mind, because thats where half the global sales are. In conclusion, he said that the SMC avoids duplication, is not influenced by affordability and makes early decisions. It is open, transparent, consistent and NHS-led. Dr Ken Paterson, Diabetes Centre, Glasgow Royal Infirmary and Chairman of the SMC Clinical Effectiveness: Sources and quality of data available to the Scottish Medicines Consortium Dr Paterson discussed the complexity and limitations of the health economics modelling which underlies the decision-making over whether a new treatment should be available in the NHS in Scotland. He looked in particular at the impact which economic modelling has on drugs for elderly people. Starting with the concept of cost-effectiveness he pointed out that it is not as simple as it seems. Cost doesnt refer only to buying the drug, but also to how much it costs to give it, monitor it and treat any side-effects. What matters is the overall cost, he said. Effectiveness is also tricky, especially when trying to place clinical trial data in a real world context. There can be problems with extrapolating short-term data to long-term; questions over looking at surrogate end points (such as blood pressure) rather than actual outcomes; and, issues over whether the trial compares the product to an old drug or even to no drug at all (placebo). Patients in clinical trials are unrepresentative of the real world patient in that they tend to be younger, fitter and have less wrong with them. In the real world patients are much older and often have multiple morbidities and are on a multitude of treatments. The typical trial patient will also get more monitoring than real life allows, which can also confound matters. Trial data cant simply be used uncritically, he said. It can inform decision-making but cannot be regarded as the last word. Health economic modelling involves taking the data into the real world and using the information to predict what would happen in routine practice. Clinician input is vital in this process, so it is important that they are engaged and involved. When looking at costs, its also important to note that cost savings predicted are putative rarely, if ever, will a new drug result in getting money back. When all the factors are not known, judgment calls come into play. He described the attempts to bring the question of health gain into a single measure, the QALY (qualify life adjusted year). Again this is a vexed issue. How is survival benefit measured when it might be based on younger, fitter patients than we have in Scotland and when the outcomes are being extrapolated from surrogate end points? The model tries to produce an outcome measurement, but it may be part of a range of estimates. Theres also the question of quality of life: is it from the perspective of the patient or of the NHS and how does one assess it? There are several methods, including asking patients to say how they feel (visual analogue scale); asking them how they feel about a time trade-off (eg 10 years of life feeling awful compared to two feeling good); and, standard gamble what risk of death would they take to have an injection which might make them better, or could kill them? These are decided on a scale where 0 equals death and 1 equals perfect health. There are other issues including the age of a patient (biological and actual) and special circumstances someone with six months left to live might not want to give up any of it so might inflate the gain in quality of life, while some older people might feel theyve had their three score years and ten so might be happier to take a chance and refuse treatment, which deflates it. All QALYs are allegedly valued equally by society, he said, but this ignores the questions such as, how we feel about whether children should take precedence or whether priority should be given to something which cures. How much should we pay for a QALY, he asked, saying that infinity is not an option. Do we bankrupt the NHS or bankrupt UK plc? Economists want to lower the threshold. This could involve restricting treatment or lowering prices. 3

With the QALY, all health gain is valued equally, whether it goes from 0.4 0.6 or 0.7 0.9 on the scale its the magnitude which matters. That means that older people may have an inherent advantage because more have diseases such as hypertension, osteoporosis and heart disease, which tend to inflate the QALY gain and makes treatment more cost-effective than in younger patients. Other factors such as co-morbidities, which may alter the efficacy or toxicity of a drug and biological age also need to be taken into account. He said there is a theoretical issue over what would happen if a drug took 20 years to become cost-effective. And niche drugs, which would only be used for a few people, also present challenges. He outlined a paradigm shift, where there is lower pricing based on how well a drug performs. Show us the benefits and well pay for it, he said. The current system isnt perfect, but is the best weve got. Otherwise decisions might be made on the basis of who shouts loudest. Tom Divers, Chief Executive, NHS Greater Glasgow and Clyde Affordability and Prioritisation Mr Divers began by saying that Scotlands NHS chief executives were very supportive of SMC. He proposed to describe some of the blunter methods used in making decisions at a regional and local level about which drugs are made available, once they have been accepted by the national body. He asked the audience to bear in mind that his NHS board the biggest in Scotland was trying to deal with the challenges of paying for new drugs against an increase in budget this year of just 3.15 per cent. He described the work of the West of Scotland regional advisory group, which involves NHS Ayrshire and Arran, Dumfries and Galloway, Lanarkshire and Forth Valley, as well as Greater Glasgow and Clyde. Other groups include the Area Drug and Therapeutics Committee, which makes the decision about which drugs make it to the local formulary, and the Prescribing Management Group, whose job includes horizon scanning, to see whats coming up and the financial and other implications. Each of these groups plays into each other. The approach is to have explicit criteria and an annual prioritisation round. This is based on business cases drawn up by each specialist service for example, cardiologists will be asked to rank new cardiology developments. A sub-group of the regional group short-lists these priorities, based on a process which now includes face-to-face meetings with clinicians. This has been a really valuable development, said Mr Divers, as it allows both sides to see what is behind decisionmaking. Health services in Scotland are facing real challenges. In recent years additional drug costs have generally been within boards uplift. This has nothing to do with the change in government, but up to this year, minimum uplift has been six per cent, so thus far we have been able to accommodate additional drug costs. That might become more challenging with smaller uplifts. He illustrated the difficulty by showing the increase in hospital prescribing costs double figure percentage rises, year-on-year making it one of the biggest pressures (along with fuel costs and surgical instruments and supplies). The Prescribing Management Group (PMG) uses horizon scanning and trend analysis, he said. Its a big group, mainly clinicians, and also considers the high costs of exceptional cases or expensive individual therapies. He stressed that discussions are on a board-wide basis and that all acute hospitals came under the one operating division, unlike the days of trusts and before when different hospitals were trying to carve each other up. He gave a couple of hard examples of coping with new developments. When SMC approved a new drug for wet AMD (age-related macular degeneration), a major cause of blindness, it underestimated the likely impact, in terms of cost and in terms of the facilities and staff needed to administer it. The treatment requires repeated injections of an antibody directly into the eyeball by an ophthalmic surgeon in a sterile room and the presence of an anaesthetist. The PMG called for a business case to look at how it could be introduced and how much it would cost. Meanwhile, the board came under pressure to introduce the drug and was accused of denying it to local patients. 4

We took the heat and said we were doing it as quickly as we could, said Mr Divers. When it was introduced, it cost 700,000 less than initially predicted (on a cost of 2.7 million). The other example was drug-eluting stents (a coronary scaffold placed into diseased arteries which slowly releases a drug to prevent clots). A minister had promised they would be made available so health boards had to make it happen. The regional cardiac group wanted to fund the more effective CRT (cardiac resynchronisation therapy), which appeared unaffordable. After a negotiation, it was agreed that if DES use was brought down to the level recommended by NICE guidelines, then the saving could part fund CRT. His final thought was that great rigor had to be applied to deciding what drugs the NHS should fund, particularly in an era of tight financial uplifts. A review of currently used treatments could also help create some headroom, he said. Professor Alan Maynard, Department of Health Science, University of York The ethics of treatment denial on the basis of cost Professor Maynard made the argument that rationing is inevitable and ubiquitous if not explicit in health services; the question is how it is done. The problem, he said, is both scarce resources and the fact that in this country we dont tend to be good at dealing with death. There are a number of ways to ration, including willingness to pay (which disadvantages the poor) and the toss a coin method where decisions are taken, for example, on the basis of religion or race or age and by looking at need, demand and supply. Economists, he said, want to improve the length and quality of life, but it is difficult to decide how best to achieve that. He pointed out that much of what is done in the health service has no basis in evidence and many studies are published which are imperfect. Even so, the health service at least has some evidencebase, especially when compared with other areas such as education, social work and police. At least medicine is making an attempt to be scientific, he said. He spoke about economic models including the QALY, but made it plain that health decisions are not always taken on a utilitarian basis; they tend to have an ethical dimension too. He gave the example of low birth weight babies; if considered on a purely utilitarian basis of getting the most benefit from resources, then treating these babies would not be seen as a good idea. The efficient route would be to let them die. Society is prepared to be inefficient, he said, adding that there was a challenge in squaring off both ethics and efficiency. When measuring cost, it is important to look at what is the value of what is given up if one person gets a treatment, another is therefore denied it. There are a number of potential solutions, including finding more resources. Rationing can be lessened by improving the efficiency of health care by, for example, reducing clinical practice variations. It has been recognised for quite some time now that considerable differences occur in the treatments carried out by doctors on apparently similar patients in terms of the frequency of physician visits, the number of diagnostic tests and length of hospital and intensive care (ICU) stays. Unfortunately the same studies show that a higher intensity of care and higher levels of spending are not associated with better quality of life or longer survival times even in the most renowned teaching hospitals. He said the UK had particularly high levels of variation and that these persisted, despite calls for action over many years, and asked whether incentives or penalties would help achieve change. Another way to reduce rationing would be to drive down the price of drugs. Its time for government to be a price maker, not a price taker, he said. He referred to the OFTs value-based pricing call (where manufacturers would be paid according to the benefit brought by their produce) but asked who would do it. We could avoid the top-up debate if industry was prepared to negotiate prices, he said. Factors other than cost or economics could affect whether patients are denied treatment. For example, its quite efficient to treat an 85-year-old cardiac patient but there might possibly be a view that its better to use resources inefficiently for young people instead. So should we swallow the health economists efficiency model or should we put social value weights in? Were pushing 5

down the efficiency route weve converted the medical profession to efficiency but there are other values, there are other things, he said. He outlined some rationing rules, including treating equals equally with due dignity (particularly when close to death); meeting peoples needs for healthcare as efficiently as possible; and, minimising health inequalities something which he said may be inefficient but ethical. He said that the way forward was for the public and politicians to decide, but reminded the audience of the reaction of former health minister Edwina Currie when asked to consider rationing: Bugger off, I want to be re-elected. Question and Answer Telling people what medicines cost The speakers were joined by Professor Henry Dargie, Department of Cardiology, Western Infirmary, Glasgow. Transparency about cost of medicines The lively question session was opened by a man who described himself as an elderly patient with Parkinsons. He suggested that people might act more responsibly and comply with treatment if there was more transparency about how much medicines cost. Professor Webb said that it might be helpful if patients understood what resources they were consuming or not consuming. Dr Paterson agreed there was a lack of transparency in how products were priced, but added that people should know the value of prescriptions as it might make them more likely to take the treatment or think twice about whether they needed to get the prescription made up. Professor Maynard was not convinced, however, saying that systems elsewhere in the world which had tried this had not noticed differences in behaviour. Professor Dargie said there could be problems if patients thought that a new drug costing 500 would necessarily be better than an existing 25 product, when there wasnt necessarily any extra benefit from the more expensive drug. He said that there was huge variation in patients and that while knowing the cost might have an effect on some, it wouldnt on others. Mr Divers called on clinicians to be more honest about the benefits of drugs, particularly nonformulary products, and asked them to get alongside managers over what was possibly within the resource envelope rather than handing the issue to a panel which decides on exceptional cases. Professor Angus Mackay, a psychiatrist, said there would be a problem in psychiatry in particular because patients with depressive illness may not think themselves worthy of expensive drugs. Professor Dargie said it was difficult for clinicians to get to know patients because consultation times were limited. He said it was important to explain what the patient would gain from a treatment, and the downsides. In some cases the patient might say they dont want to bother with that [a drug], particularly if its preventative. QALYs, top-up fees and orphan drugs A former physician from the Royal Infirmary of Edinburgh expressed alarm at how vague a QALY is. He also felt that those with rare diseases would get a bad bargain if denied new treatments on the basis of cost, particularly as those looking for further developments might gain knowledge which would benefit other conditions. He also asked about top-up or co-payments. All panellists agreed that there are clear limitations with the current approach, including the QALY, but said they are the best thing available at the moment. Professor Webb said that while economics helped form decisions, essentially they were clinical judgments, while Professor Maynard said that it would help if the drug industry could be persuaded to do better real world trials. On orphan drugs, Mr Divers said there is a debate within SMC about whether they should be subject to the same criteria as all other drugs and that there is a risk of leaving it up to local decision-making by each health board. 6

On top-up fees, Mr Divers said the current system (where patients cannot have NHS treatment if they opt to pay for something such as an expensive drug privately) came about because some hospital consultants allowed their private patients to jump the NHS queue and move in and out of private treatment. Professor Webb said that top-up payments are against the ethos of the NHS, but said he is troubled that people have to sell or remortgage their home to pay for treatment particularly if the effectiveness is marginal. Deciding on other areas of service provision A representative from the ABPI (pharmaceutical industry) questioned affordability, saying that if drugs are subjected to a maximum cost per QALY, what about the other 90 per cent of health intervention. Professor Maynard said that the cut-off was arbitrary, but that rationing has been submerged because so much money has been pumped into the NHS. He said politicians would have to look at the issue. Mr Divers said that some procedures, such as surgical practice, are looked at for costeffectiveness. Folk dont get a 50,000 operation nodded through, he said. Self-monitoring Asked if encouraging people to self-monitor would improve compliance, the panel was not convinced. While it might be good for some patients, possibly those who were engaged with selfmonitoring would have adhered to treatment in any case. Patient education and the media A pharmacist from Edinburgh asked about how to educate patients, when the media publicised the latest must-have drug. Professor Webb said it is important to educate the public that drugs could harm as well as benefit. Professor Maynard added that it is important to get information out to GPs so that they can deal with patients who demanded the latest drug, by, for example, being able to say the results had been based on a small trial. Professor Dargie said the media is part of the problem because what is written is at best inaccurate and is often misleading. It has to shock to be read, he said. Considering social care costs A representative of Alzheimer Scotland said that NICE had tried to take into account the costs of social care when making decisions about drugs for Alzheimers. She asked if there is a possibility that NHS-centric processes risk ignoring the impact on social care. Professor Webb said that if the NHS identified social care costs as part of the SMCs brief, they would look at them on a caseby-case basis. We dont look at carer costs, he said, adding that politicians would have to make that decision. Tom Divers said that the SMC got insights from patient interest group submissions, but that some groups didnt submit; some submissions were superb; and, others were patchy. Me Too Drugs Asked by Professor Harmer, a pharmacologist, about me too drugs, Professor Webb said they arent necessarily a bad thing. Having more than one drug in a class means that if patients react against one, they can try another. Also, extra benefits might be found from the third of fourth version he gave the example of the contraceptive pill, which has improved in safety as it has evolved. But he said it is important to be wary where pharmaceutical companies have a drug nearing the end of its patent and alter it slightly to preserve profits. SMC v NICE A member of the audience put the question of which was better, the SMC or NICE. The chairman replied that the two organisations were different creatures. The SMC makes rapid assessments and uses information provided early by the pharmaceutical industry and tries to form rather than change prescribing habits. NICE takes longer, but can make decisions based on newer information. Wed like them to use the SMCs data. Why reinvent the wheel? he said. Professor Webb said that NICE runs a public health programme, looks at health technology and at whole disease areas none of which is part of the SMC remit. These are hard nuts to crack, but it 7

is important work, he said. The SMC is very good at making fast judgements, but NICE does things that are just as important and which complement what the SMC does. Dr Paterson said that primary care trusts in England are increasingly looking to the SMC for advice while they wait for NICE to make decisions. Professor Maynard added it was very sad that more PCTs didnt do so. We have 150 PCTs doing their own thing. I think they should use SMC guidance and be more consistent. Patient involvement It was left to a woman active in patient involvement to put the last question, which was really more of a statement. She said she was heartened and encouraged by the debate and the thought and consideration behind it. Professor Lawson finished by calling on members of the public not necessarily representatives of patient groups to get involved in the debate. I think public involvement has greatly evolved were getting better at it, he said, but conceded that there is room for improvement.

Opinions expressed here do not necessarily represent the views of the RSE, nor of its Fellows The Royal Society of Edinburgh, Scotlands National Academy, is Scottish Charity No. SC000470