Health: Rare Diseases

Lord Turnberg Excerpts

Tuesday 3rd March 2015 Lords Chamber

Asked by

[Lord Turnberg]

To ask Her Majesty’s Government what action they are taking to improve access to treatments for patients with rare diseases.

[Lord Turnberg (Lab)]

My Lords, many rare diseases can be severe and life threatening, and the problem for patients unlucky enough to suffer from them is that there are few really effective treatments for most. So among the small number for whom a new treatment seems to hold out some promise, there is intense interest and renewed hope. Of course, it is reasonable that new treatments should be evaluated for their effectiveness and that they should be prioritised before they can be commissioned. But therein lies the rub—because, although these diseases each affect a small number of patients, there are more than 8,000 individual rare and ultra-rare diseases in existence. Although there are no treatments for most, more than 100 drugs licensed by the European Medicines Agency are awaiting approval for funding by NHS England, and many of them are very expensive.

It is not surprising that companies that develop such drugs for very small numbers of patients find it difficult to recoup their investment without a high charge; and it is hardly surprising that NHS England, which foots the bill for specialised services, is pretty cautious about what it can afford to pay. This budget is already overspent by £900,000. So we can no longer avoid recognising the dilemma of how to pay for these expensive drugs. My first question to the noble Earl is: what effort are the Government making to face up to this problem? We clearly now need to have the open discussion and thorough review that the topic deserves. The consultation exercise that NHS England is currently conducting is interesting but limited in scope, and the whole issue of affordability of expensive drugs is one that should be opened with the public by the Department of Health itself.

Leaving aside that issue for the moment, nothing can provide an excuse for the enormous delays in decisions being taken by the bureaucratic systems that we have set up. They are not only complex but so completely opaque that clinicians and the pharmaceutical industry despair, and the poor patients are completely bewildered and perplexed. I have spent a considerable amount of time trying to understand the ways in which these treatments are assessed, and here I have relied heavily on advice from the Rare Disease UK alliance, whose members represent a large number of patient groups.

The problems begin when anyone who is proposing that a new treatment should be centrally funded is faced with no fewer than seven possible routes to take. The first three are through NICE—although only one of these, the HST or highly specialised technology route, is really capable of assessing these types of treatment. The problem here is that the HST can manage to deal with only three proposals a year. It does not have the capacity to cope with more. It has, in fact, so far approved only one treatment—eculizumab, for atypical haemolytic uraemic syndrome—since it took over responsibility for rare disease treatments more than a year ago. Yet we know that the EMA is licensing many more new treatments for rare diseases, and that many of these, at least 10 a year, have to be redirected to NHS England itself. However, the rationale behind which three treatments NICE will take on itself and which it will hand on is clouded in mystery. Even worse, it seems that a few drugs have to go through an assessment by both organisations. Can the Minister say how NICE decides which treatments to examine itself and which to hand on—and, more importantly, why on earth we need two separate systems of appraisal? Would it not save a lot of time and money if we had one well resourced and efficient system?

Then we come to the remarkable apparatus set up by NHS England, where we also have several possible entry routes that I will not bore noble Lords with. The main route is through the clinical reference groups, of which there are 75, each assessing different types of potential treatments. Their views are then transmitted to one of five programme of care boards—and that is just the beginning. Approval is then sought from the clinical effectiveness team and the finance group, before the matter is sent on to the clinical priorities advisory group, which in turn passes it on to the specialised commissioning oversight group and the directly commissioned services committee. I hope that noble Lords are keeping up. If it gets through that lot, it is sent out for public consultation and, if approved, has to wait until the beginning of the next financial year for funding.

You can imagine the frustration and angst that all this creates among patient groups who feel alienated by the whole prolonged and tortuous process, which is made worse by the fact that the way in which these committees go about their business seems quite opaque. No minutes seem to be available, decisions are hard to come by and patient involvement is tangential to say the least. The noble Earl, in a response to my Written Question on 1 December, tried to reassure me that patients were involved in a number of advisory committees, but I have to tell him that many patient groups feel quite ostracised by the systems that have been set up. There are no less than seven serial committees, and a cynic might think that this complicated system has been devised to avoid having to make any funding decision. You might well think that; I couldn’t possibly comment.

As if that is not enough, the whole apparatus has now ground to a halt after a legal challenge to CPAG on behalf of a child with Morquio’s syndrome. No new treatments have been examined since December and there is no end in sight until at least next June. There is now a backlog of some 80 treatments awaiting a decision. Can the Minister say when we might expect this matter to be resolved?

With such a complicated system, it is little wonder that there are inconsistencies in the decisions that have been taken. For example, why has funding been agreed for patients with tuberous sclerosis who have brain tumours but not for those with kidney tumours that are equally life threatening? How come treatments have been approved for a very small and specific subgroup of patients with cystic fibrosis but not for a small specific subgroup of patients with gastrointestinal stromal tumours? The numbers affected are similar and the treatments equally effective for each of these rare subgroups, but there is no consistency in the decisions taken. Will the noble Earl press NHS England to rethink this problem of inconsistency?

On another issue, can the noble Earl say why the commissioning through evaluation system, set up in 2013, has apparently not yet approved any drugs brought to it—that is, zero drugs—despite having an available budget of some £16.9 million? Why has this innovative scheme not been activated?

Then there is the question of the high costs that drug companies have in developing new treatments for these very small markets. There is a need here for negotiation between government and industry on price—and there is clearly room for negotiation where industry could be asked to justify its high charges when it has such small patient groups to test and can avoid the very high costs of large-scale phase 3 trials. In practice I understand that companies find that, despite the price access scheme, it is extremely difficult to fix up a meeting even to begin negotiations. Can the noble Earl explain why there seems to be this reluctance to negotiate?

There is also a particular problem when a clinical trial of a new innovative treatment is coming to an end and patients are seen to be benefiting from it. Industry funds the trial but expects to be able to hand on the costs of continuing the treatment after a successful trial has finished. This area of negotiation is particularly fraught since many patients who have benefited are desperate to continue the treatment yet find themselves in limbo because of a reluctance to reach a decision on who pays. Can the Minister clarify what engagement the Government are having with companies and patients who find themselves in this position?

Finally, there is a mind-blowing system of committees and advisory groups set up in NHS England. Can all the CRGs, PoCs, CPAGs, SCOGs, DCSCs and RDAGs be justified? I doubt it. Is it not time for NHS England to get a grip and radically prune this morass of committees? Will it take advantage of its current consultation exercise to think again, and will it kick-start some interim measure to get past the logjam while it is cogitating? I hope that the noble Earl will exert some pressure on it to provide a simple, clear and transparent system of appraisal in a timely way that takes full account of patients’ views. We clearly do not have that now and it is desperately needed.

I return, finally, to my plea for a thorough and open inquiry into the ways in which it might be possible to cover the costs of expensive drugs for rare diseases in an equitable way. That is something that the department itself can hardly avoid tackling. I look forward to the noble Earl’s response.

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[The Parliamentary Under-Secretary of State, Department of Health (Earl Howe) (Con)]

My Lords, I congratulate the noble Lord, Lord Turnberg, on securing the debate and raising this issue. I am acutely aware that access to treatments for patients with a rare disease is of great importance to him and many others. We have had some excellent contributions from noble Lords this evening.

The Government are committed to improving the life of all those affected by rare disease. The UK is a recognised leader in research, treatment and care for rare diseases. We are at the forefront of the genomics revolution, which has the potential to radically transform the way that we diagnose and treat people with rare conditions. The UK strategy for rare diseases, which my noble friend Lady Bottomley kindly mentioned, is a high-level framework that sets out our strategic vision. I am sure she will know that raising awareness of rare diseases is a key aim of the strategy. To that end, the department has worked with Health Education England to produce two videos: one aimed at parents and patients, the other aimed at healthcare professionals, particularly GPs. These were launched last week at the Rare Disease Day event, which the noble Lord, Lord Walton, referred to.

I was interested to hear about lymphangioleiomyomatosis, or LAM. Access to treatment for that condition, as for others, is based strictly on clinical need, as set out in NHS England’s published clinical policies, irrespective of tariff arrangements. The key in so many cases such as this is what is often referred to as the diagnostic odyssey—the delay that patients experience before getting a diagnosis.

I listened with huge interest to the noble Lord, Lord Rogan, talking about ITP. The UK Strategy for Rare Diseases sets out our strategic vision for improving the lives of all those affected by rare diseases, including ITP, the autoimmune disease. He will be impressed to know that my officials furnished me with an extensive note on ITP as he was speaking.

NHS England has been charged with taking forward many of the strategy’s commitments and in the Five Year Forward View it sets out a contextual backdrop for the strategic work during the next few years. Due to their rarity and their low patient populations, services for rare conditions in England are directly commissioned nationally by NHS England as specialised services. I am sure that the noble Lord, Lord Patel, will agree that commissioning these services nationally means that NHS England can commission each service to a single national standard with single national access criteria. It ensures that patients have the same access to specialised services regardless of where they live in England. NHS England has made significant progress in developing a set of nationally consistent service specifications and commissioning policies which ensure equity of access to high-quality services across the country.

Since April 2013, the National Institute for Health and Care Excellence, or NICE, has been responsible for the evaluation of selected high-cost, low-volume drugs under its highly specialised technologies programme, as has been mentioned. This plays an important role in ensuring that commissioning decisions are based on a robust and thorough assessment of the available evidence. NHS commissioners are legally required to fund treatments recommended by NICE in its highly specialised technologies guidance. Until NICE’s guidance is available, commissioners make their funding decisions based on the available evidence.

Topics are referred to that programme by Ministers, following a topic selection process that is overseen by NICE. At the core of the topic selection process is a set of prioritisation criteria that are used to determine whether a topic is suitable. These criteria are published on NICE’s website. The process for deciding which topics to refer to NICE is carried out with as much transparency as possible and includes consultation with stakeholders once a topic has passed the early stages of the process. However, it must be recognised that some degree of confidentiality is required, particularly early on in the process, as information on new drugs can be commercially sensitive before they are licensed. The noble Lord, Lord Turnberg, might be interested to know—as he was asking me about this—that NICE has recently taken steps to improve the level of transparency in the topic selection process, and has begun to publish more detailed information about the rationale for its topic selection decisions.

NHS England’s Clinical Priorities Advisory Group formulates recommendations on commissioning of new treatments for rare diseases in England. In order to ensure that the maximum number of patients benefit from innovative treatments coming on stream, hard choices need to be made about which of these to fund routinely. Patient groups asked NHS England to consult on changes to the principles and processes by which it makes these decisions. At its board meeting on 17 December, NHS England decided that a 90-day consultation would be carried out on the prioritisation framework and decision-making process that NHS England should use to make commissioning decisions on new treatments and interventions. The length of that consultation period reflects the importance of these decisions and the advice received from patient groups. The decisions on prioritisation will not be completed until the consultation has closed and the responses have had due consideration. The consultation was launched on 27 January, and it is open for responses until 27 April.

In carrying out its public consultation, NHS England will ensure that the principles and processes for making these decisions are well informed, evidence-led and in line with the expectations of patients and the public. I am sure that the noble Lord, Lord Turnberg, will agree that it is important that NHS England has a robust decision-making process in place and that that process must be followed to ensure that NHS England is treating all patients with rare diseases fairly.

[Lord Turnberg]

My Lords, will the Minister give way?

[Earl Howe]

I will give way, but I am sure that the noble Lord understands that we are time-limited.

[Lord Turnberg]

I just wanted to check that the Minister is aware that there are no fewer than seven serial committees that it has to go through. That does not seem very efficient.

[Earl Howe]

I note that point, my Lords, and I will reflect carefully on it. If NHS England has a comment to make, I shall be glad to write to the noble Lord about that process.

Clearly, once the prioritisation consultation finishes, and in the light of NICE assessments, NHS England will take stock of its position in relation to new treatments.

Our priority must always be to ensure that patients with rare conditions have access to new and effective treatments on terms that represent value to the NHS and the taxpayer. I am sure noble Lords will agree that it is very important that NHS England has a robust decision-making process in place and that, as I have described, it makes sure that all patients with rare diseases are treated equitably.

I also recognise that it is vital that we speed up the discovery, design and take-up of new, innovative 21st-century medicines and treatments in the NHS. We will continue to work with industry and our European partners to increase access to these innovative medicines for patients with rare conditions. In April last year, we launched the early access to medicines scheme, which aims to give patients with life-threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorisation, or licence, when there is a clear, unmet medical need. More generally, our Strategy for UK Life Sciences sets out an ambitious, long-term programme of action to improve the wider environment for health life-sciences companies and overcome the barriers that prevent discoveries being translated into commercial opportunities and new treatments with real benefits for patients.

The noble Lord, Lord Rogan, stressed the importance of research. Undertaking research into rare diseases requires effective partnerships between patients, their families, clinicians, researchers and industry. Of course, health research holds the promise for breakthroughs and improvements in the way that we diagnose and treat people with rare diseases. For example, the NIHR’s world-class Rare Diseases Translational Research Collaboration, launched in parallel with the UK rare diseases strategy, is just about to start 14 new projects. We also want to make it faster to start rare disease research in the NHS. That is why we asked the Health Research Authority to bring in a new single approvals process. This will speed up access for patients to new and effective treatments.

I will cover as many questions as I can in the time available. My noble friend Lady Thomas referred, very powerfully, to the value of hydrotherapy and the importance of appropriate equipment being commissioned by CCGs. As she will know, CCGs are autonomous statutory bodies. Decisions are made locally but I am happy to look into the matters she raised and will write to her on what I fully agree is a very important issue.

The noble Lord, Lord Patel, asked about the successor arrangements to AGNSS. Responsibility for evaluating the use of new and existing highly specialised medicines and treatments within the NHS in England transferred from the Advisory Group for National Specialised Services—AGNSS—to the National Institute for Health and Care Excellence in April 2013. So far, that is working satisfactorily. However, there is the other group—the Rare Diseases Advisory Group—which was referred to by the noble Lord, Lord Walton. Where does that fit it? In terms of rare diseases and highly specialised services, the RDAG makes recommendations to NHS England and the devolved Administrations on issues related to highly specialised services. Its further role is to have an overview across the four countries on the development and implementation of the UK strategy for rare diseases and highly specialised services. It makes recommendations to the Clinical Priorities Advisory Group about how highly specialised services should be commissioned.

The noble Lord mentioned the cancer drugs fund, which has helped more than 60,000 people with cancer to get life-extending drugs that would not otherwise have been available to them. NHS England is now responsible for the operational management of the fund. It is currently working to ensure the very latest, most clinically effective drugs can be made available to patients. We will carefully consider with NHS England what arrangements should be put in place for the fund in the longer term.

The noble Lord, Lord Turnberg, questioned the inconsistency in funding arrangements for different conditions. He mentioned tuberous sclerosis and kidney tumours. Over the next few weeks, the six programmes of care responsible for advising NHS England on specialised services will draw up the work programme for 2015-16, which will include developing a number of policies. Two policies—one for the provision of everolimus for tuberous sclerosis complex-related renal angiomyolipoma and the other for subependymal giant cell astrocytoma or SEGA—will be considered for inclusion in this process.

The noble Lord asked about commissioning through evaluation. That programme was established by NHS England in 2013 as an innovative mechanism to capture further evaluative data and I will write to him further about progress on that front. As regards negotiation on patient access schemes, companies that are members of the pharmaceutical price regulation scheme have the opportunity to propose a patient access scheme to improve the cost effectiveness of their drug, as part of a NICE appraisal. Departmental officials stand ready to meet with any company that wishes to discuss its options.

Time has run out. I conclude by saying that I am committed—indeed, the Government are committed—to ensuring that patients with rare conditions get the same quality, safety and efficacy in medicines and other treatments as those who have more common conditions.