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Baroness Thomas of Winchester
To ask Her Majesty’s Government what steps they are taking to ensure early access to innovative medicine for life-threatening conditions.
Baroness Thomas of Winchester (LD)
My Lords, I am grateful to have attracted such a stellar cast for the important matters that we will be talking about in this short debate this evening. I hope that it might build on the interesting debate on 13 March on regenerative medicine, in which my noble friend Lord Willis of Knaresborough said:
“The King’s Fund estimates that by 2070, 20% of the UK’s GDP will be spent managing long-term conditions”.—[Official Report, 13/3/14; col. 1944.]
Such a situation would be unsustainable and unaffordable, and we must ensure that new treatments and technologies are found so that the quality of life of those with long-term conditions is as good as possible.
The very next day after that debate last month, the Government announced the early access to medicines scheme, which offers a way by which unlicensed medicines can be made available to patients before approval of a licence to benefit public health. The scheme has been warmly welcomed by many of us who are involved with rare-disease patient groups and their families. Indeed, with the support of the Muscular Dystrophy Campaign, the All-Party Parliamentary Group for Muscular Dystrophy, in its report last year, highlighted the need for such a scheme. Ground-breaking research for potential treatments for rare diseases is set to grow and this scheme should ensure that patients will, in future, be able to get the treatments that they need at a much earlier stage of the process.
Professor Dame Kay Davies of the department of physiology, anatomy and genetics at Oxford University is leading the development of a potential treatment for Duchenne muscular dystrophy, which is currently in early clinical trials. She said:
“The introduction of a ‘fast-track’ system offers an exciting opportunity to intervene in a safe way and ensure effective medicines reach the people who need them as early as possible. It is good news for families affected by Duchenne muscular dystrophy, who are deeply anxious about the speed at which future potential therapies will reach their children. Several potential treatments are in clinical trials and further laboratory research projects are underway—the prospect of accelerating the progress of approaches that show particular promise is a very welcome one”.
This view is supported by many families of boys with Duchenne, who say that every second counts and time is not a luxury that they have.
Professor Dame Sally Davies, the Chief Medical Officer, said that the scheme would allow drug-makers to demonstrate the value of unlicensed medicines, improving their chances of eventual approval by regulators and NICE. Obviously, allowing patients early access to medicines is not without some risks, which is why the Government were right to be clear that sufficient data must be available to demonstrate safety before a drug can be considered under the early access scheme.
The Minister will know that I was bound to mention the dismay of many of us in this field at the fact that AGNSS—the Advisory Group for National Specialised Services—was being disbanded, along with NHS Specialised Services. AGNSS was responsible for the appraisal of very rare drugs, while NHS Specialised Services had responsibility for the commissioning of services for very rare diseases, as well as a ring-fenced fund to subsidise treatments for the drugs to treat these diseases. AGNSS’s duties have transferred to NICE—the National Institute for Health and Care Excellence—while NHS England has taken over the NHS Specialised Services role. However, little clarity was given on what the new appraisal and commissioning processes will look like and there was widespread concern that NICE’s “cost per quality-adjusted life year” approach to the appraising of new drugs will effectively exclude, on the grounds of high costs, treatments for small patient populations.
NICE has now established its highly specialised technology programme, responsible for the appraisal of orphan drugs. Unfortunately, early indications of its outlook on high-cost treatments do not appear promising, although it is still at an interim stage. A current example is the approval that NICE is considering for Soliris, a treatment for atypical haemolytic uraemic syndrome, a rare blood disorder that may be inherited. Before Soliris, there was no treatment available to prevent death or organ damage and up to 25% of patients would die following their first attack. Soliris was recommended for approval by AGNSS, based on its effectiveness in halting the progress of the disease and its low cost per quality-adjusted life year. It was assumed that, as a result of this recommendation, the drug would be available to patients from October 2012. However, in January 2013 the Government announced that Soliris would be subjected to a second assessment, under the new system for specialised services within NICE, and Alexion Pharmaceuticals, which developed the drug, has been asked to explain the high cost of Soliris.
NICE has also asked for advice from NHS England on what considerations relating to the management of its specialised commissioning budget it considers should be taken into account in formulating a recommendation. I hope that the fears that many people voiced when AGNSS was disbanded that treatments for rare disorders might be denied on grounds of high costs are not going to be realised. After all, patients with rare and life-threatening conditions deserve access to treatments just as much as those with more prevalent conditions.
The next matter that I wish to raise is the clinical trials process, which must be speeded up. One way of achieving this could be to authorise a process of study approval whereby various stages of clinical trials can be conducted in parallel with one another. This would avoid a lengthy sequential process. For example, in the case of exon-skipping technologies for Duchenne, each drug or molecular patch will treat only certain specific mutations causing the condition and, under current procedures, future molecular patches would have to go through the same lengthy requirements. Will my noble friend the Minister urge the Medicines and Healthcare products Regulatory Agency and NHS England to consider such a study approval process?
Another important consideration is the clinical trial infrastructure, including additional specialist centres to enable more patients to participate in clinical trials. During the APPG inquiry, we were concerned to hear about the cutting of administrative support by some hospital trusts. It is a false economy to cut back on this infrastructure, such as patient registries. At present, many patient registries are charity-funded, with little or no long-term funding security. With limited back-office support, some centres are finding that there is no one available to input patient data. Consequently, such cuts can seriously damage the ability of centres to carry out large-scale clinical trials. Will my noble friend say what steps the Government are taking to ensure that centres and clinics across the UK have the resources required to manage patient registries and for clinical trials to be carried out?
Finally, in last month’s debate my noble friend said that consideration of the Health Research Authority’s business case to bring together and streamline NHS approvals and local ethics approvals should be completed shortly. Will he update us on that? Will he also endorse the need to streamline NHS approvals so that unnecessary regulation and delays are avoided? We must always look forward with great optimism in the search for treatments for intractable conditions. The early access to medicines scheme is a very welcome initiative, which must not be allowed to fail because of bureaucratic obstacles in its path.
Baroness Jolly (LD)
I remind noble Lords that we are tight for time this evening. Six minutes is the limit and, as soon as the clock reaches six, your time is up.
Lord Kakkar (CB)
My Lords, I congratulate the noble Baroness, Lady Thomas of Winchester, on securing this important debate. In so doing, I declare my own interests in healthcare, in particular that I am chairman of UCLPartners and the UK business ambassador for healthcare and life sciences. I, too, congratulate Her Majesty’s Government on their early access scheme, recently announced, which is important for patients and healthcare professionals and in securing the ecosystem around the delivery of life sciences in our country. That is vital not only for the delivery of innovative healthcare, advancing clinical outcomes and improving the ability of our patients to benefit from advances in medical research, but because the life sciences sector in our country plays such an important role in the economy more broadly.
As we have heard from the noble Baroness, Lady Thomas, the scheme is fundamentally driven to ensure that innovative medicines that have undergone a degree of thorough scrutiny by way of clinical evaluation could be made available to patients with life-threatening and debilitating conditions before they would normally have received their full licensing, which is very important. I ask the noble Earl how Her Majesty’s Government plan to ensure equity of access to the scheme, bearing in mind that these innovative products will not, at the stage when they will become available as part of the scheme, have had approval for cost-effectiveness through the processes of the National Institute for Health and Care Excellence. Therefore, it is important that careful attention is paid to ensuring that, if those innovative medicines are to be provided as part of a specialist commissioning infrastructure within NHS England, it is done in such a way that patients throughout the NHS in England can avail themselves of those important innovations, because they will be some of the most vulnerable patients whom we see with life-threatening conditions or chronic disabling and debilitating conditions.
In that regard, I wonder whether the noble Earl can give a view on the potential role that academic health science networks, such as UCLPartners, might play in promoting access to innovative therapies as part of the early access scheme. As part of their original designation and licence obligations, the academic health science networks had to give undertakings with regard to the diffusion at scale of innovation across the populations for which they have responsibility. Do Her Majesty’s Government see a role for the academic health science networks in the early access scheme with regard to those diffusion of innovation obligations? If so, does the noble Earl believe that the arrangements for funding the academic health science networks, bearing in mind that the scheme will run for a number of years, are sufficiently secure?
Beyond the important contributions that Her Majesty’s Government have driven over the past four years in innovation in healthcare, through the development of the academic health science centres and networks and through the promotion of the National Institute for Health Research and its translational medicine research elements, which are vital to the early access scheme by encouraging industry and other biotechnology partners to work with the institute to undertake the research evaluation that will bring those innovative medicines more quickly to bear in the management of patients in the most desperate of circumstances, there is also the important question of the legal framework within which innovation can take place. That relates to innovation not across the system but to an individual clinician innovating for an individual patient and, therefore, the culture of innovation in our healthcare system.
The noble Lord, Lord Saatchi, recently promoted the Medical Innovation Bill, which is intended to address the important question of whether a legal impediment to innovation resulting from an accumulation of case law has changed the way in which clinical practice is discharged. I know that on 22 November 2013 the Secretary of State recognised, in a Written Statement, that there may well indeed be unintended consequences of the way in which case law is now interpreted that will stifle innovation with regard to the individual clinician providing care for the individual patient. As a result, Her Majesty’s Government are currently undertaking a broad consultation on the question of whether a Bill to help to clarify those ambiguities in law is necessary. Can the noble Earl provide some further insights into how the consultation is proceeding and where Her Majesty’s Government’s thinking is with regard not only to dealing—as they have done effectively—with the systems issues about innovation at scale and pace for large populations, but to the ability of individual clinicians to practise innovative care in a responsible and structured framework for the individual patient in front of them, often making use not only of personalised medicine and the revolutions in genomic medicine that will drive individual decision-making for patients, but also of this impressive early access scheme?
Lord Colwyn (Con)
My Lords, in the late 1970s and early 1980s, I regularly annoyed House of Lords Health Ministers from both sides of the House with what I described as innovative treatments for common diseases which had not completed the necessary trials and approvals, but which were harmless and cheap. Those treatments tended to involve homoeopathy, acupuncture, herbal medicine and traditional Chinese medicine. At the time, I was president of the Natural Medicines Society, and I declare my interest now as president of the All-Party Parliamentary Group for Integrated Health Care.
Recently, there have been many instances of the need to try new drugs which have not completed their normal phase trials and testing but might be able to be used on patients who have few remaining treatment options. My noble friend will forgive me if I resurrect some of those ideas, which were rejected and never used to treat patients in the UK. The Innovative Medicines Initiative will pave the way for new vaccines, medicines and treatments to tackle major health challenges, but many of the challenges have been there for many years. Artherosclerosis is by far the largest health problem affecting the western world. Attitudes to its prevention and to the ability to reverse its effects have ranged from the extremes of those who feel that it is completely preventable and reversible to those who regard it as an inevitable ageing process for which there is no remedy.
Lower limb peripheral arterial disease can affect about 9% of the population, and the incidence increases with age. About 20% of people aged over 60 have some degree of peripheral arterial disease. Incidence is higher in people who smoke, people with diabetes and people with coronary artery disease. Peripheral arterial disease occurs when the vascular system becomes obstructed due to atherosclerosis. The obstruction leads to gradual tissue death in the lower legs because of the lack of blood, which carries vital nutrients and oxygen. Critical limb ischaemia is characterised by severely diminished circulation, ulceration, tissue loss and gangrene. Amputation is a major risk for those patients, particularly those who have diabetes.
I invite my noble friend to revisit the techniques of oxidative therapy, first reported in the Lancet in 1920. There are many theories about the different functions of hydrogen peroxide in the body, and a great deal of scientific material supports almost every one. At one time in my dental career and as president of the Arterial Health Foundation, I tried to persuade the Government to examine the claims of the practitioners of EDTA chelation therapy. I have some personal experience of that technique. Over a period of six months at a clinic in Eindhoven, I followed the treatment plans of several patients who were unable to walk more than a few paces and had all been recommended for amputation. After two months, they were walking and then were able to run. Seeing those patients improve is something that I shall never forget.
Chelation therapy removes heavy metals from the arteries and is able to improve the blood flow to all areas and so preserve health and normal function by re-establishing peripheral circulation—supplying oxygen and essential nutrients. Ethylene diamine tetra acetic acid is introduced into the blood by intravenous drip and binds itself to heavy metals, such as lead, mercury, cadmium and other minerals, including calcium, and is excreted normally via the kidneys.
For many years, physicians in the US and Europe have used the chelating agent EDTA as an anti-atherosclerosis drug. A large anecdotal history has grown up supporting its value. In the 1950s and 1960s, a number of uncontrolled trials reported favourably on its value in cardiovascular disease, ischaemic heart disease and peripheral vascular disease. However, none of the trials was properly controlled by the double-blind procedure and thus the use of EDTA has not gained acceptance among the majority of physicians.
A patient might need 20 infusion visits of three hours. The EDTA solution travels through every blood vessel in the body, treating every vessel from the aorta to the smallest capillary. Chelation therapy was first developed in the US, and has been approved by the FDA as a way to remove toxic metals such as lead and mercury from the bloodstream, but no such approval has been granted for its use in unclogging the arteries of heart patients. Its use in that area remains controversial.
Intravenous EDTA chelation therapy, properly administered, is a safe, economical and effective treatment for the symptoms of atherosclerosis caused by free radical pathology. There is insufficient time to examine the technique in more detail, but I believe that it is a treatment that should be carefully considered before resorting to amputation.
About 20 years ago, I had a course of treatment myself, partially to eliminate small deposits of mercury, which tended to build up in dental practitioners, and partially to demonstrate to the Secretary of State for Health at the time—my noble friend Lady Bottomley—that the treatment was harmless. The heavy metals were removed and I survived the treatment. If I was told that I needed to have a leg removed, I think that I would seriously consider chelation as a first line of defence.
Baroness Masham of Ilton (CB)
My Lords, I thank the Baroness, Lady Thomas of Winchester, for obtaining this debate and I congratulate her on all she does for people with muscular dystrophy. I also congratulate the All-Party Group on Muscular Dystrophy on its report on access to high-cost drugs for rare diseases. I know first-hand how desperate it can be when someone one knows and loves needs a drug that may not be registered but may help them in a deteriorating situation. To quote from the report:
“To ensure that patients with rare diseases are not denied vital and cost-effective treatments we need to see the NHS develop a new model with regulators and the industry”.
There are many people across the country who have rare conditions and need specialist treatment. There are many parliamentary groups dealing with particular diseases, illnesses and disabilities. The message I get from them all is that the delivery of services is patchy across the country. The message that I get from the Government is that services should be dealt with locally. However, the National Health Service should at least have national minimum standards and the guidelines set out by NICE should be adhered to.
I agree with the statement in the report that the Government should establish a ring-fenced fund for rare disease drugs to ensure that patients affected by rare diseases are not denied treatment. Is there not such a fund in Scotland? In England, the cancer drugs fund has been of great help to people with rare cancers. There was also a recommendation about ensuring that NHS England provides specialist centres equipped with an appropriate range of health professionals to deliver treatments. If patients do not get the correct diagnosis, treatment and advice, their condition nearly always gets worse and there is so much suffering and anguish.
I am always concerned when I hear it said that people with long-term conditions should be treated at home. They need the very best diagnosis and treatment, and to be taught how to manage their conditions by experts before going home. All interested parties should work in communication and co-operation to help the patients. Some of these conditions mean a shortened life, therefore patients should have the best quality possible for that life—and that means expert treatment and advice, and ongoing support when needed.
I stress how important specialist nurses are to these people who have rare conditions or complicated diseases. They are a vital support to consultants and patients, as well as teaching patients’ families and general nurses how best to look after those with special needs. They are the link between hospital and the community, so not to have them is false economy. I met a splendid group of specialist nurses last week, who told me that morale among nurses is not good. This is bad for patients. One of the reasons, they told me, was the shocking situation which happened at Mid Staffordshire NHS Foundation Trust and other tragic events. Yet another recent incident was highlighted over the weekend, but I can only praise the specialist nurses I have met, who are dedicated to helping their patients achieve the best possible life, often in very challenging situations. I hope that the Minister will agree that specialist nurses are essential.
There are so many rare conditions which need research that can give hope for the future. Approaches which undervalue new treatments can impede innovation, distort therapeutic decision-making and undermine global welfare. We need progress, not limitations.
Baroness Grey-Thompson (CB)
My Lords, I thank the noble Baroness, Lady Thomas of Winchester, for tabling this debate. This is not a subject which I have previously spoken about in your Lordships’ Chamber, but a chance conversation in the corridor encouraged me to listen, and then to speak.
The subject of what medication should be given to patients with life-threatening conditions to ensure good-quality life is very interesting to me as, linking it to other work that I am doing on disability rights and quality of life, it has helped me to further examine the relationship between the public’s view of being sick or ill and being disabled. All too often the impression is given that if you are disabled, you must probably also be ill and therefore have a high cost attached to you. It is important to understand that the two are not synonymous.
I am interested in this subject because I have a number of friends who have been involved in various drugs trials and, while I do not require any medication, in my time as a wheelchair user I have certainly been offered experimental surgery to “fix” what were seen as my problems. This is of course not related to the issue of medication. However, one of the procedures that I was offered was a leg-lengthening operation when I was already paralysed; it would have made no difference to whether I could walk or not, or my ability to use callipers. Then years later, it was revealed on a television programme as an innovative procedure.
Concerning “life-threatening conditions”, I, like many others, would like to see further clarification on what that definition involves. I would also like to see what other options could be explored, such as developing physical literacy, physical health and healthy lifestyles. I know that this happens in many cases but I would like to be assured that this is the norm because, while medication is part of the answer, it is not the only answer. Many of my friends experience quite a lot of difficulties with urinary tract infections, but I have not had one of those since I was 13. I think that is because of my training and being active, and all the other things that are part of a healthy lifestyle.
I was interested to read the documentation provided in the Library, in which the Health Secretary, Jeremy Hunt, said:
“Making Britain the best place in the world for science, research and development is a central part of our long term economic plan”.
He added:
“Most people are only too happy to altruistically volunteer for medical research if it helps save lives”.
Encouraging investment in the UK is important in the light of some pharmaceutical companies choosing to reconsider where they are based and what research they are involved in. It is a useful aspiration to have, but we must of course be conscious of the balance between companies funding research for ultimate profit and giving people positive options.
In researching for this debate, I found many cases where hundreds of millions of pounds were spent on trials that may or may not be successful. My husband is a research chemist, so I have a little understanding of the cost of developing and testing new products, although in his case it is high-temperature chain oils. I also think that many of us who are not medics or involved in that profession often do not understand the cost of some of these medications. I was in the USA recently with my family when my daughter developed an ear infection. A trip to see a nurse practitioner was $100; it was another $100 to see a doctor and a very small bottle of branded medication was $400.
I will also briefly refer back to a Question asked by the noble Lord, Lord Clement-Jones, which is listed in Hansard as HL 991. The noble Earl responded by saying that:
“We are exploring ways in which patients can continue to benefit from innovative cancer drugs at a cost that represents value to the NHS, in the context of developing the new pricing arrangements for branded medicines”.—[Official Report, 25/6/13; col. WA 111.]
This cannot be simply about businesses investing in the UK. Somewhere, we have to balance the budget.
Within this, I also believe that doctors have tremendous power to influence patients. I have been hugely fortunate and have had some amazing doctors in my life. In fact, without the NHS I would not be here. However, it is important to have appropriate guidelines for explaining to patients which drugs they are being offered, and for them to know whether they are new drugs or medication that has been previously been developed but is now being suggested for new indications. In terms of research, it is absolutely imperative that we have the appropriate number of people using medication to report back the data so as to be able properly to monitor it. There is no doubt that this work is important for many people who have life-threatening conditions. I look forward to future debates in this area.
Lord Walton of Detchant (CB)
My Lords, in thanking the noble Baroness, Lady Thomas, for initiating this debate, I declare an interest as the honorary life president of the Muscular Dystrophy Campaign. I hold similar appointments with many other medical charities.
This is a very important issue. I have given support over the past few years to the Rare Diseases Research Consortium and Genetic Alliance UK, chaired by Alastair Kent. Many of the 300-plus rare diseases that have been carefully characterised and identified by those organisations affect the neurological, neuromuscular and other, similar systems. Some are fatal but virtually all cause increasing disability of various kinds. Advances in molecular genetics over the past few years have been immensely exciting. In many of these diseases—in fact, the majority are genetically determined—the causal gene has been identified and, often, located.
The missing gene product has also been identified, as in, for example, dystrophin, normally a constituent of the muscle fibre membrane missing in Duchenne muscular dystrophy. Similarly, in Pompe disease, a condition causing severe muscle paralysis and affecting the heart, acid maltase has been identified as the missing substance. However, in many other neurological disorders, including the various cerebellar ataxias, the condition has been found to be due to multiple amino acid triplet repeats that actually, instead of being absent and therefore not causing absent metabolic activity, damage the human cells.
Treatments have begun to emerge in diseases such as cystic fibrosis, and acid maltase can be effectively treated by gene therapy in Pompe disease. As the noble Baroness, Lady Thomas, said, haemolytic uraemic syndrome, which affects only a few dozen people in the UK and is inherited, is effectively treated with a drug called eculizamab, which is highly effective. It is a life-saving treatment; without it, the disease moves to a fatal conclusion.
Many more such drugs are being developed, and in Duchenne muscular dystrophy drugs have been identified and used for the technique called exon skipping, which appears to work only in specific mutations and by no means in all. But Dame Kay Davies and her colleagues in Oxford are working on a mechanism of utrophin uptake regulation, persuading utrophin to move along the muscle fibre membrane to replace the missing dystrophin. These drugs are being trialled in excellent treatment trials in London, Oxford and Newcastle, funded by the Medical Research Council, the Muscular Dystrophy Campaign and other organisations.
So what is the problem? The problem is that more and more such drugs are coming on stream but, because the number of patients affected by these rare diseases is comparatively small, the drugs are not likely to be commercially successful. The industry has been extremely helpful in making them available for trials, but this cannot go on indefinitely. The drugs that are likely to treat rare diseases affecting 100-plus people are called ultra-orphan drugs, whereas those affecting 1,000-plus people are called orphan drugs, and it is clear that they are going to present an increasing problem over the coming years.
The problem is that the cost-benefit analysis that has usually been employed by NICE in assessing the value of these drugs is not likely to be helpful or even appropriate in assessing their value in the case of rare diseases. However, we await the outcome of NICE’s consultation on what it calls value-based medicines, and we hope that this will be positive in relation to these drugs. The government initiative of creating a rare diseases advisory group answerable to NHS England has been helpful but, most particularly, the early access scheme, to which other speakers referred, is most welcome as being a very exciting development for the future.
As time goes by, though, in my opinion those initiatives are not in themselves going to be enough. As my noble friend Lady Masham said, we miss the advisory group on national specialist services, AGNSS for short, which had ring-fenced funding. At the end of the day, it is more than likely that ring-fenced funding will be needed for the management and treatment of these conditions with orphan and ultra-orphan drugs. I hope that the Government will agree that in their new structure a neuromuscular clinical reference group should be established for this purpose.
Human suffering is not something that can be measured in numerical terms. The needs of these patients and their families are paramount. Somehow or other, this problem is one with which this and future Governments are going to have to come to terms.
Lord Hunt of Kings Heath (Lab)
My Lords, I declare my interests as chair of an NHS foundation trust, president of GS1 and a consultant and trainer with Cumberlege Connections.
I, too, congratulate the noble Baroness, Lady Thomas, on securing this debate on this vital subject. I welcome the Government’s announcement about the early access to medicines scheme. However, like the noble Lord, Lord Kakkar, I would like some assurance about the scheme being operated equitably. Will the Minister assure me that it will not apply just to patients being treated by specialist centres where research or clinical trials have been undertaken? I would also like to pick up the point made implicitly by the noble Baroness, Lady Grey-Thompson, which is that patients must be able to make an informed choice about whether to consent to the treatment being offered.
I also believe that this question raises much wider issues about the NHS approach to innovative medicines. It is a critical issue for NHS patients but is also critical in terms of the health of UK pharma and the contribution that it makes to our economy. I have long been troubled by the UK approach to innovative medicine. We have a hugely strong science base and a strong pharma R&D presence that cannot be taken for granted, yet traditionally we have been very slow on the general uptake of proven new medicines and treatments. Within the NHS, it is remarkable that drug costs are treated as a disturbing cost pressure to be held down, if they can be, whereas increased spending on clinical staff and medical equipment is seen as a good thing, per se.
The noble Earl and I have been debating these issues for many years. We debated the introduction of NICE. He will recall that NICE was introduced as a way of speeding up the introduction of new, innovative, proven treatments, but we know that we have some way to go. We also know that there are still some concerns about the conduct of medical trials in this country, which has a direct bearing on our approach to innovative new medicines and treatments. I refer the noble Earl to an interview given by the director of the Wellcome Trust, in which he expresses real concern about the continuing delay in medical trial approvals. He recommends generic protocols preapproved by ethic committees and institutions at a national level. I know the noble Earl has a particular responsibility in this area, and I would be very interested if he were able to comment on it.
We also know that, as the noble Lord, Lord Walton, suggested, there are gaps in the NICE methodology. The cancer drugs fund is an example of one government response to gaps in the methodology. This early-access scheme is another. The noble Baroness, Lady Thomas, and the noble Lord, Lord Walton, discussed orphan drugs. That is another area where, left to itself, the market will not enable them to be brought to market at an affordable cost. As the noble Lord, Lord Walton, said, NICE is working on a value-based approach. It is clearly early days, but if the noble Earl were able to comment on the extent to which he thinks that would enable NICE to produce new methodologies that would cover these areas, that would be very welcome. The alternative is that government is simply going to have to fund and top-slice various little funds to meet gaps in the NICE approach. At some point, that will call into question the whole NICE approach. I think we have recognised for some years that, while overall the NICE approach is lauded internationally, there are gaps that we have to find a way of filling.
My final question for the noble Earl is about clinical commissioning groups. One of the issues about innovative medicines is the decisions now being taken by these new organisations. The noble Earl will be aware of research in the past few months that shows that in relation to technology appraisals, there can be up to a twentyfold variance in uptake in different parts of the country. He needs no reminding that there is a legal requirement on clinical commissioning groups to fund NICE technology appraisals. I am concerned that clinical commissioning groups are ignoring their statutory responsibilities. This is the other end of the whole approach to innovation. If we are unable to guarantee to patients that these innovative treatments are going to be introduced in the NHS, we are letting patients down. I hope the noble Earl will be able to give us some reassurance on this.
The Parliamentary Under-Secretary of State, Department of Health (Earl Howe) (Con)
My Lords, I congratulate my noble friend Lady Thomas on securing this debate and I am grateful to her for providing the opportunity to update your Lordships on, in particular, the early access to medicines scheme. It is just one way in which the Government are supporting improving patients’ access to new medicines.
I begin by making it clear that our priority is to ensure that patients, including those with rare and life-threatening or life-limiting conditions, have access to new and effective treatments on terms that represent value to the NHS and the taxpayer. I agree with the noble Baroness, Lady Masham, that it is essential that people get the medicines and treatment that they need. That is why we have set up the cancer drugs fund and why we have NICE to give evidence-based advice to clinicians and the NHS.
On 5 December 2011, the Prime Minister announced a new strategy for UK life sciences. One of its commitments was that,
“early in 2012 the MHRA will bring forward for consultation proposals for an ‘Early Access Scheme’”.
I am pleased to be able to say that, following public consultation co-ordinated by the Medicines and Healthcare products Regulatory Agency—known as the MHRA—and engagement with patient groups and industry, we announced the early access to medicines scheme on 14 March. The purpose of the early access to medicines scheme is to support access in the UK to promising new, unlicensed or off-label medicines in areas of unmet medical need. Under the scheme, the Secretary of State for Health, acting through the MHRA, will provide a scientific opinion on such medicines to treat, diagnose or prevent life-threatening or seriously debilitating conditions that do not have adequate treatment options. This could include patients with advanced cancer or children with Duchenne muscular dystrophy.
MHRA is responsible for managing the scientific aspects of the scheme, which will follow a two-step process. Step one involves giving a new medicine a promising innovative medicines designation, known as a PIM designation, which will provide an early indication that a product may be a possible candidate for entry into the early access to medicines scheme, based on the available clinical data. Companies that wish to move to step two must hold a PIM designation and provide further relevant data on their product’s quality, safety and efficacy. At step two, the MHRA will produce a scientific opinion describing the benefits and risks of the medicine, based on information submitted by the applicant after sufficient data have been gathered from the patients who will benefit from the medicine.
The scientific opinion will be made available on the MHRA’s website to assist clinicians and patients in making treatment decisions and to support informed consent by patients by informing them of the risks and benefits of the product. The scheme will be launched and ready to receive applications from Monday of next week, 7 April 2014. I understand that full details, together with guidance, will be published on the MHRA website at this time.
The noble Lords, Lord Kakkar and Lord Hunt, asked about equal access for patients under the scheme. EAMS medicines will be provided for free by the company concerned. The scientific opinion will be available on the MHRA website, as I have mentioned, to allow doctors and patients to make treatment decisions. That will provide an equitable platform for patient access.
The noble Lord, Lord Kakkar, also asked about academic science input into the scheme. The scheme is open to academics, industry and charities, provided that the criteria are met. Step one, the PIM designation, would also be open to academic research units such as at UCL. As the noble Lord mentioned, academic health science networks could well have a role in promoting the scheme. All AHSNs are now up and running and their funding is assured for the immediate future.
It is important to recognise that the early access to medicines scheme is a UK-only scheme that relates to unlicensed medicines en route to market. It is always better for a patient to receive a licensed medicine where possible and for companies to have the legal certainties that come with a marketing authorisation. For this reason, the MHRA continues to engage at both European and global level to explore how the medicines licensing process can become more efficient. We welcome the European Medicines Agency’s launch of its adaptive licensing pilot on 19 March 2014, as the MHRA has had a leading role in the preparation of the pilot and accompanying guidance.
Adaptive licensing is a prospectively planned, adaptive approach to bringing drugs to the market. It seeks to maximise the positive impact of new drugs on public health by balancing timely access for patients with the need to provide adequate evolving information on benefits and harms. Adaptive licensing uses the regulatory processes and flexibilities within the existing EU legal framework, such as conditional marketing authorisation. The pilot will explore the strengths and weaknesses of all options for development, assessment, licensing, reimbursement, monitoring and utilisation pathways in a confidential manner and without commitment from regulator or company. I plan to meet relevant government and industry partners later this month to ensure that the UK can capitalise on the pilot. I hope that UK-based companies will be at the forefront of those presenting products to the EMA.
The noble Lord, Lord Kakkar, asked about the draft Medical Innovation Bill, which aims to encourage responsible innovation in certain circumstances and to discourage irresponsible innovation. I echo the noble Lord’s thanks to my noble friend Lord Saatchi for putting these concepts before your Lordships’ House previously. We are currently consulting on this draft Bill. The consultation paper was published on 27 February and the consultation runs until 25 April. For that reason, I am afraid that I am not yet in a position to make a definitive pronouncement on the Bill’s provisions.
The noble Lord, Lord Kakkar, asked about equality of access to specialised services. NHS England, as he knows, is now responsible for commissioning prescribed specialised services across England, so patients should know what services they can expect. The Government remain totally committed to making the UK a world-leading place for life sciences investment. The noble Lord was right to say that trialling drugs is an expensive business, but good progress is being made through the life sciences strategy to build a better life science ecosystem to attract and develop talent, to reward innovation and to overcome barriers to innovation. The Strategy for UK Life Sciences states:
“It has become increasingly challenging for life sciences companies, particularly SMEs, to discover, develop and commercialise medical innovation”.
AHSNs, as the noble Lord rightly said, have therefore been set up as a key response to these challenges, acting as the link between the NHS and industry. I think that the universal coverage of AHSNs has had multiple benefits in terms of potential UK growth.
The noble Lord, Lord Walton, and my noble friend Lady Thomas mentioned ultra-orphan drugs. We are aware of the challenges posed by treatments for the very rare conditions, with small patient populations, for which these drugs are made. My noble friend expressed particular worry about eculizumab, or Soliris. From April 2013, NICE has been responsible for the evaluation of selected highly specialised health technologies. It issued draft guidance on 27 February which does not recommend the drug’s use. Stakeholders had until 25 March to submit comments to NICE. I must stress that NICE has not yet issued its final guidance on eculizumab to the NHS and I understand that that is expected in July. While NICE undertakes its evaluation, NHS England has developed an interim commissioning policy to enable patients with aHUS to receive eculizumab. To clarify, AGNSS found that eculizumab was clinically effective but that further information was needed to demonstrate its cost-effectiveness. That is why we tasked NICE with evaluating it.
All candidate medicines have the potential to induce toxicity. I will address the interesting speech of my noble friend Lord Colwyn by reminding noble Lords that medicines must undergo a series of rigorous assessments, progressing from small cohorts of patients in rigorously controlled conditions to larger and more diverse groups of patients, thus ensuring quality, safety and efficacy. We have three phases of clinical trials to ensure that those things are assured.
It is possible to run adaptive licensing design studies that allow for modifications as the trial progresses; for example, the numbers of different treatment arms can be tried out. Such designs have the potential to speed up clinical development and can use resources more efficiently. There is regulatory guidance on adaptive designs. I respond to my noble friend Lady Thomas by welcoming many features of the new clinical trials regulation, which provides for a more streamlined approach, with the introduction of combined clinical trial and ethical approval and a single portal for all EU applications.
I share the enthusiasm and excitement of the noble Lord, Lord Walton, about the developments in genetic medicine. That is why we have established Genomics England, a development that has put us firmly at the head of the field.
The noble Lord, Lord Hunt, asked what we are doing to ensure the rapid uptake of NICE-recommended drugs in the NHS. Innovation, Health and Wealth, a document that we published in 2011, set out a range of measures to support the rapid uptake of NICE-approved medicines in the NHS, including the establishment of the NICE Implementation Collaborative, the automatic incorporation of NICE-recommended drugs into local formularies and the introduction of an innovation scorecard to compare local uptake. Good progress has been made in delivering those commitments, but we recognise that more can and should be done. We are committed to seeing Innovation, Health and Wealth progress.
There are variations in drug usage among CCGs. As the report points out, there can be many reasons for variation. Different areas may have different health needs and it is right that the treatments used should be decided by doctors and patients. As I have said to the noble Lord before, we are committed to tackling unjustified variation in the usage of medicines and we encourage NHS organisations to consider the findings of the report in the context of the needs of their populations.
As my time is running out, I will have to write to noble Lords to cover those issues to which I have not yet managed to reply. However, in conclusion, I was pleased to announce yesterday my approval of the business case presented by the Health Research Authority and the funding that goes with that to enable it to fulfil its remit. The HRA will provide a single approval for research in the NHS to radically streamline and simplify how studies are set up. I believe that the UK’s approach of allowing patients access to promising but as yet unlicensed medicines while encouraging greater use of European licensing flexibilities will provide much earlier access to a number of innovative new medicines, in particular in areas of unmet need. We can be proud of the leading role that we play in ensuring that the UK remains one of the leading countries in which to develop medicines and to see them reach the patient’s bedside in clinical use.