Regenerative Medicine: S&T Committee Report
(10 years, 2 months ago)
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Lord Patel
To move that this House takes note of the Report of the Science and Technology Committee on regenerative medicine (1st Report, HL Paper 23).
Lord Patel (CB)
My Lords, I invite noble Lords who have just taken part in the education debate to stay, if they want to enrich their education. However, while they make a decision on that, I am pleased to introduce this debate on the Science and Technology Committee’s inquiry into regenerative medicine.
Our chairman of the committee and of the inquiry, the noble Lord, Lord Krebs, could not be here but, on behalf of the committee, I thank him for his brilliant chairmanship. I also thank our special adviser, Professor Fiona Watt FRS. The committee was well and expertly advised by her. I also thank our clerk, Mr Chris Atkinson, all the staff of the committee and all its members, some of whom are taking part in this debate. I thank all noble Lords who are taking part, particularly the Minister, the noble Earl, Lord Howe, and the noble Lord, Lord Hunt, on the Opposition Front Bench.
I declare my relevant interests. I am a professor and chancellor of the University of Dundee, a fellow of the Academy of Medical Sciences and of the Royal Society of Edinburgh, and I have previously chaired various stem cell committees in the United Kingdom.
Regenerative medicine is an umbrella term for the medical specialty of the regeneration of human tissue, organs and cells. It has the potential to treat or cure disease. Possible treatments range from curing neurological disorders to eventually repairing hearts. Our inquiry sought to pinpoint the UK’s strengths in regenerative medicine, identify barriers to translation and commercialisation—in the case of commercialisation, primarily dealing with treatments in the healthcare market—and recommend solutions.
The UK has an enviable potential resource in the National Health Service, with access to hundreds of thousands of patients in one system and a strong science base in this field. The Government have also been paying significant attention to developing this field. Together, these factors could combine to benefit patient well-being and the health of the UK economy. Basic science translation and commercialisation in this field are being well supported in other countries. However, there are growing concerns that, despite positive progress so far, the UK could fall behind in this area and miss out on opportunities to translate basic science into commercially viable treatments as the science develops. The opportunity cannot be missed. The UK could and should be a world leader in this field.
It is for that purpose that the committee chose to limit its inquiry to the regulatory framework for the translation of science and commercialisation. The key areas of inquiry were the research base, the application of science, barriers to translation, barriers to commercialisation and international comparison. The call for evidence was issued in July 2012. The committee was informed by a seminar held prior to our inquiry at King’s College, and during the inquiry some members of the committee visited the California Institute for Regenerative Medicine in San Francisco, often referred to as CIRM, for three days of intensive seminars and discussions. CIRM is an impressive organisation and, in many aspects, is quite special—in its inception, its funding and its delivery and scale—and is likely to deliver regenerative treatments in the near future.
We published our inquiry report and the voluminous evidence in July 2013 in HL Paper 23, and I now turn to the report and its findings and recommendations. The term “regenerative medicine” is used to refer to methods to replace or regenerate human cells, tissues or organs in order to restore or establish normal function. This includes cell therapies, tissue engineering, gene therapy and biomedical engineering, as well as more traditional treatments involving pharmaceuticals, biologics and devices.
Perhaps I may give some examples. Bone marrow transplantation, which is well understood by many, is the original stem cell therapy. Another is the use of pancreatic islet transplantation for certain types of difficult-to-control glycaemia in type 1 diabetes. Another is the use of skin cells to treat burns. Less well known perhaps is the use of gene therapy to treat lipoprotein lipase deficiency and autologous cell therapy to treat cartilage defects in knees. Treatments likely to be available in the next four or five years are those that support the body’s own regeneration and repair mechanisms. Others are treatments using cells, including embryonic stem cells, for certain eye conditions, such as retinitis pigmentosa and age-related macular degeneration, which affects 30% of the population over the age of 60. The hope is that eventually there will be treatments for Parkinson’s disease, cardiovascular disease and diabetes, and cures for diseases for which we currently have no treatment.
Through their various publications on regenerative medicine and life science strategy, and funding for research, the Government have recognised the potential of regenerative medicine and life sciences generally to improve both health and the UK economy. This is valued, and our report says that we value the Government’s support. The UK has a strong science base, reflected in the number of much-cited publications, multiple academic centres of excellence and the three Nobel laureates of recent times: Sir Martin Evans, who is still working in the field of cell biology, Sir Robert Edwards and Sir John Gurdon.
The UK currently has nearly 40 early-phase clinical trials involving stem cells. There are more than 76 projects funded in basic science and early-phase trials. So far, it is good news. However, when it comes to translation, the theme that permeated our inquiry was that of uncertainty. Those who gave evidence asked for proportionate regulation and a clearer path from bench to bedside. The current system was described as a sort of great frustration. We make recommendations for improvement. Similar comments were made about clinical trials, despite the recognition that the NHS affords the best environment for clinical trials. The Government need to address the issue to make the UK more effective.
The Cell Therapy Catapult reported delays in starting trials, and similar comments were made by others. Furthermore, the delivery of regenerative medicine treatments, particularly involving living cells, produces challenges for manufacture and delivery on a large scale. If the UK is to be competitive and be attractive to companies from outside, both investing in the UK and using UK facilities, the Government need to support and invest in infrastructure development. The committee recognised that setting up the Cell Therapy Catapult has significant potential but to achieve it, alternative funding channels will need to be explored.
We make some suggestions. We make several recommendations for evaluation and pricing of treatments and look to NICE to devise suitable models, including value-based pricing. Covering all the areas, we make 24 recommendations to help ensure that the potential of regenerative medicine is realised. To bring it all together, we asked that an expert working group be set up with an independent chair. On the whole, we welcome the Government’s positive response to our report and hope that they will ensure that the recommendations will be taken forward. However, I do have some questions.
What progress has been made to ensure that the regulatory process for clinical trials is simplified? What plans do the Government have to encourage investment in large-scale manufacturing facilities for regenerative medicine products? What action has UKTI taken to improve the chances of the UK being the location of the development and manufacture of regenerative medicine therapies?
Although the Government did not accept our recommendation of an independent chair for the expert working group, they accepted the establishment of such a group, and we are pleased that Sir Michael Rowlands is to chair it. What terms of reference have been given to Sir Michael’s committee, and when is it expected to report?
In conclusion, regenerative medicine has the potential not only to save lives but, with the NHS as a resource, also to support the UK economy. We can be the world’s centre for developing regenerative medicine. I commend the report and beg to move.
The Earl of Selborne (Con)
My Lords, the whole House will be grateful to the noble Lord, Lord Patel, who speaks with great authority on the subject and was a fellow member of the committee. He speaks as a very distinguished professor in this area; I speak as a layman, but one who learnt a lot during the course of this inquiry into a fascinating area of very fast-moving technology and science.
This is a highly appropriate subject for a Select Committee report. Where science moves so fast, the regulatory framework will inevitably lag behind and it is a challenge for Administrations to ensure that there is the appropriate regulatory framework and fiscal support for what everyone will recognise has enormous potential. The technology has enormous potential in the longer term to produce new treatments for the plethora of diseases mentioned in paragraph 18 of the report, which lists Parkinson’s disease, cardiovascular disease and diabetes. I am sure that many people will wish to add to that list.
In the shorter term, a number of treatments can be seen coming over the horizon or are indeed almost available. One example, in paragraph 15, is a therapy which seeks to reverse the damage caused by a stroke. Given an ageing population—which is common to pretty well every developed economy—given that healthcare as practised at present with the tools available to us is forecast to require ever larger proportions of our economic resources and given the international interest, let alone our own national interest, in novel and innovative treatments that may have great economic and social benefits, there will be enormous rewards to those countries that put in place successful policies to promote these opportunities.
As the noble Lord, Lord Patel, reminded us, we have a strong science base at present and we need alongside it a suitable regulatory framework which gives confidence to patients, investors and, of course, the scientists and those conducting trials. We will need to ensure that public funds can complement funding from other sources, such as charities and commercial interests. We will have to negotiate the so-called valley of death, which is the difficulty, so often experienced in this country, in commercialising research findings. That is being addressed by the Cell Therapy Catapult, about which more anon. We will have to ensure that we make best use of those advantages derived from the National Health Service. The noble Lord, Lord Patel, reminded us that we have the great advantage of access to much valuable data on a scale not available to some of our competitors.
The Government have identified regenerative medicine as one of their eight great transforming technologies. Other reports, as well as our own, have alerted the Government to the critical importance of identifying barriers to development. The Government’s own report of July 2011, Taking Stock of Regenerative Medicine in the United Kingdom identified,
“steep technological, regulatory and strategic barriers to realising regenerative medicine’s significant potential”.
Our report emphasised the frustration that some scientists and clinicians have experienced negotiating the various regulatory hurdles. The Government response recognised the need for regulatory simplification in the long term and help to get through the existing minefield—perhaps I should call it labyrinth—in the shorter term. The establishment of the Regenerative Medicine Expert Group, tasked with developing a regenerative medicine delivery readiness strategy and action plan, is certainly a positive response to the report and is to be welcomed. However, at the risk of seeming grudging in my praise for the Government’s response, I draw attention to the rapidly changing international competition for recognition as a global leader. We refer in our report to the high level of investment in the United States and to the rapid progress in countries such as China and India.
Since we published our report, Japan passed legislation in November last year that revised its pharmaceutical affairs law, with the intention of establishing Japan as a global leader in regenerative medicine while continuing to protect patient safety and confidence. Last month at the World Economic Forum in Davos, the Japanese Prime Minister made it clear that this legislation redefines the regulatory framework and gives the opportunity for new therapies to move more rapidly from an early-stage clinical trial towards conditional approval, which enables the product to be brought to market and therefore to obtain reimbursement for the product in an accelerated manner. In the light of such responses from our competitors, it may well prove necessary to look more fundamentally at our own regulatory framework, as indeed we recommended.
On pages 42 and 43 we say that we will revisit the regulatory aspect of the inquiry to ensure that progress has been made. I am absolutely sure that it will be essential to monitor progress, although we all hope that the Regenerative Medicine Expert Group can facilitate simplification without, at least for the present, the need for further legislation.
I want to say a word in support of the catapult centres in general and the Cell Therapy Catapult centre in particular. For years we have complained in this country that we allow others to reap the rewards of commercialising scientific research. The previous Administration are to be congratulated on asking Hermann Hauser to make recommendations on how we should address this long-standing problem. He concluded that what was missing from the United Kingdom’s innovation landscape was a network of centres working at the commercialisation stage of technology, matching scientific research to industrial needs. His recommendation was that we should follow the model of the Fraunhofer institutes in Germany.
One of the seven new catapult centres is the Cell Therapy Catapult established in 2012 through the Technology Strategy Board and is designed to create a world leading cell therapy industry in the United Kingdom through innovation and collaboration. It is early days and we do not yet know how successful the centre will be—we will not know, probably, for a decade or so—but meanwhile we should allow the centre to build up its dedicated cell therapy teams, bringing together scientists, investors, manufacturing interests, regulatory experts and other interested parties from around the world. We must resist all temptation to interfere or change—it will need a good long period for bedding down—but it must be a highly appropriate subject for a catapult centre.
What is now needed is continuity of funding and support from us all. This catapult centre is exactly what is needed if we are going to emerge in the next decade as a global leader in this exciting sector.
Lord Turnberg (Lab)
My Lords, I am delighted to reiterate the remarks of the noble Lord, Lord Patel, in congratulating the noble Lord, Lord Krebs, on chairing our Select Committee so ably, and on the support of his expert adviser and secretariat. I also commend the noble Lord, Lord Patel, for introducing this topic and for explaining so clearly the enormous potential of regenerative medicine to cure diseases that are hitherto incurable, and for pointing out the considerable benefit we have from our expert scientific base in the UK.
I express my interests as scientific adviser to the Association of Medical Research Charities, many of whose members have strong interests and involvement in regenerative medicine.
It is worth noting that our report was produced last summer after taking evidence for more than a year and that things have moved on since then. It is a rapidly moving field and some things have changed for the better while others have been thrown into starker relief. There have been more advances in the science; clinical applications are being developed; the mood in the investment community, oddly enough, has improved—I am told that there is a greater appetite among venture capitalists to take the risks needed to invest in the field; and there is some hope that we will see some of the recommendations of our report being put into action.
However, I want to focus on only three aspects: the complex regulatory framework; the MHRA and MEA approval processes; and our capacity to manufacture and scale up production.
First, on the regulatory framework, I hope to build on the words of the noble Earl, Lord Selborne, and I know that the noble Lord, Lord Willis, will also take up the cudgels on regulation. Under the regulatory framework, a researcher or a small biotech company wanting to take a discovery forward for further development in a clinical trial or commercialisation will be faced with no fewer than 11 regulatory bodies that they may have to apply to. This morass of bodies, with a mix of acronyms from the HFEA to the HTA, from GTAC to the MHRA, from the EMA to the HRA—to say nothing of having to jump through the hoops of NICE—is extremely confusing, and not only to the novice. The UK has many more regulators than virtually any other country in the world, and certainly more than the USA, which seems to have one.
Our recommendations focus on the need to take a grip of this complexity and suggest that the Health Research Authority should expand its current role in streamlining the regulatory process. The HRA is doing an admirable job within its limited resources. It is under the expert guidance of Professor Jonathan Montgomery, and in a pilot study that it has already carried out, it has demonstrated that it could do much more. The authority has shown that it could provide a sort of one-stop shop for researchers so that a single application made to the HRA would be fed through a gateway for approval by the authority where it has the competence to do so or distributed to those other bodies that need to give their approval. This would be a remarkable achievement if it could be done and would transform the atmosphere for researchers. However, of course, it requires more funding for the HRA. It would not need vast sums, and could indeed be achieved with a modest investment, while the gains made, both financially and in saving wasted time, would be enormous. My first question for the noble Earl is whether he will examine whether there is some way to find the modest extra money needed. I know that a bid from the authority has gone in to the department, and it would be helpful if he could tell us how far it has got.
I turn now to the processes by which new treatments are assessed by the Medicines and Healthcare products Regulatory Agency. Here the timescale is almost always very long, and sometimes it can several years and involve large and expensive phase III trials. However, for treatments such as those using regenerative medicines, stem cells and the like, such a lengthy process is quite inappropriate. This has been recognised in Japan and the USA, where a much more flexible approach has been taken. In Japan, as the noble Earl, Lord Selborne, mentioned, the law has recently been changed so that approval for regenerative medicines can be based on phase II trial evidence alone, without the need for phase III trials. In the USA, the FDA has introduced what is called a breakthrough therapy designation that provides a similar phase II-only requirement. I know that the MHRA recognises the need for something similar here, and it would be extremely helpful if the noble Earl could indicate how far the expert group set up by the MHRA has progressed in its efforts to develop an adaptive licensing system to speed up approval of these types of innovative treatment. The Government are paying much more attention to the need for innovation in healthcare, and certainly those in the field would find the efforts of the MHRA encouraging. It would allow us to keep up with our rivals around the world.
Finally, I come to another concern. Our report described a reluctance among venture capitalists to invest in biotech in general and in regenerative medicine in particular, and I mentioned earlier that the situation here may be changing. The so-called valley of death between invention and commercialisation may not be as deep as we thought, even though we remain way behind the more adventurous investors in the USA. However, as prospects for investors are now improving and more cell-based therapies appear to be coming on stream, the problem of the lack of manufacturing capacity to take these advances to the market has been shown to be much more obviously rate-limiting than had been thought. We have drawn attention to this problem in several of our recommendations and we have had supportive responses from the Government and others, but much more needs to be done. Our ability to scale up the production of these highly specialised treatments so that they can become available to large numbers of patients is sorely lacking. I feel that the UK Regenerative Medicine Platform, which has been asked to take this on and which could have had all this in hand, has been just a touch complacent. In this light, can the noble Earl tell us how far the Ministerial Industry Strategy Group, which met in November, got with its discussions on manufacturing capacity? What recommendations, if any, emerged as a result of that meeting?
I note that the Cell Therapy Catapult is gathering evidence on capacity, but that alone will not solve the problem if we do not offer some inducement to those who need to build up our manufacturing capacity. It is also the case that the resources available to the catapult are limited and will only go a little way in offering this inducement. Is there any prospect that the Technology Strategy Board will offer more support for this purpose? Will the UKTI Life Science Investment Organisation play a role in helping fill this gap? It will certainly be offering advice and information to potential investors overseas. What practical encouragement will it be able to offer companies that they will be supported if they come here?
The potential for regenerative medicine to transform healthcare in the next few decades is enormous. We must take advantage of the lead we have in basic research and convert it into therapies for patients and economic benefits for the UK. There are encouraging signs, and the Government are clearly aware of the importance of investing in this area, but there is much that remains to be done. In particular, we must make sure that we have a regulatory environment that is efficient and fit for purpose, that we keep up with the competition with a responsive and speedy approval system, and that we are well prepared with the capacity to manufacture to scale these potentially remarkable treatments.
Lord Willis of Knaresborough (LD)
My Lords, as a member of the committee that produced this report, it is a pleasure for me, too, to speak in this debate. I thank the noble Lord, Lord Patel, for the way in which he introduced it and for the very expert advice and guidance he gave to members of the committee throughout the inquiry. It was like having your own personal adviser at your side. In particular, I echo his comments about Professor Fiona Watt, who I thought was an outstanding adviser to the committee. Her standing in the international community gave the report real aplomb when it was produced.
Having sat as chair of the Science and Technology Select Committee in the House of Commons, and now as member of this committee in your Lordships’ House, I have to say that, although most of our inquiries are interesting—some more so than others, which I find quite difficult—this one afforded us the opportunity to examine an area of medical science that promises significant breakthroughs in the way in which we treat patients with a wide range of medical conditions, where there are currently no effective treatments or no treatments at all. Faced with that sort of scenario, you can understand the huge hope that regenerative medicine gives to tens of thousands of people around our nation.
Equally, the NHS is faced with a funding crisis that will get worse as each year goes by, as an ageing population with multiple long-term morbidities makes increased demands on a decreasing real-terms budget. The need to introduce disruptive technologies into the NHS to treat patients therefore becomes ever more urgent and there are two drivers for supporting regenerative medicine.
The stark evidence contained in the report showing the increase in the number of people with long-term conditions—diabetes up by 25% in the five years to 2011, chronic kidney failure up by 45% over five years and dementia up by 25%—is really sobering. The escalation of these figures over the next five, 10 and 15 years will put a huge burden on our health service but also on the Exchequer. The King’s Fund estimates that by 2070, 20% of the UK’s GDP will be spent managing long-term conditions. That is simply not affordable, nor is it acceptable, unless we can introduce some new disruptive technologies to address the situation.
Will regenerative medicine change this landscape? Probably, but not in the short term. Our report makes the point very forcefully that this is not a short-term fix—this is a long journey. Many of the technologies that the noble Lord, Lord Patel, spoke of in his introduction will not come about within five, 10 or 15 years, but could take even longer than that. However, it is important that efforts by our researchers, funders, regulators, manufacturers, government departments and industry have a clear steer. For me, the central theme of this report is certainty—in terms of the regulatory framework and the funding framework and, irrespective of which Government are in power, that we are going in a particular direction and we are going to keep to it.
The report assessed where we are at present, recognised the global competition and suggested ways in which we could move swiftly and effectively to get promising technologies into clinics. Global competition is strong and that is good. We are aware that in Japan, Germany, South Korea and, particularly, the US, there is a recognition that regenerative medicine has huge potential both for domestic use and in terms of its wider economic impact. Research shows that by 2050, 37% of US GDP will be needed for health and healthcare at current rates of growth. That is totally unsustainable, so the emphasis is on finding solutions because the US cannot afford not to, and I think that is the situation in the UK.
It was interesting that when we were in California, so great was the reputation of our research base that American researchers—even in CIRM, with $3 billion over 15 years at its disposal—were looking to UK research groups to add to their expertise. Indeed, our Professor Fiona Watt was revered among the researchers that we met.
To be fair, the Government have played their cards well—as did the previous Government, who recognised that this was an emerging destination. Allocating £180 million to the biomedical catalyst fund, the biomedical research centres and units established at leading universities with an £800 million investment, research councils continuing to fund very basic research, and the establishment of the Regenerative Medicine Platform to address technical and scientific discoveries are all going in the right direction. The UK Stem Cell Bank gives us a unique advantage, as does our NHS database. The establishment of the Cell Therapy Catapult, which we have just heard about, with a vision for global leadership, are all things that the Government rightly deserve credit for.
I suspect that our resources will never match those of our competitors, particularly the US, but it is interesting that other sectors, particularly the charitable sector, are beginning to shift their funding into regenerative medicine. The Association of Medical Research Charities, which I chair and of which the noble Lord, Lord Turnberg, is the science adviser, spends 15% of its R&D budget—remember, we raise £1.3 billion every year—on regenerative medicine research. That was in 2011; it has probably gone up since then. It is not surprising that Research Councils UK, the MS Society, the British Heart Foundation, Fight for Sight, the Alzheimer’s Society and, of course, the Wellcome Trust all see regenerative medicine as the real hope for their future as they struggle to find treatments for the most hard-to-reach diseases.
However, if we are to get regenerative medicine treatments into clinics, we have to address the issue of cost. There is, I am sad to say, a somewhat complacent air about the Government’s response to our various recommendations about costing novel treatments. This is a not inconsiderable issue; it is the very essence of getting early treatment for patients. It will not be the NHS or the British Government who actually fund putting the treatments into clinics; it will be the private sector, and we have to make it sustainable and attractive in order for private investment to take those things past the valley of death and into phase 3 trials and patients.
That is why the point in our report about looking at value-based pricing is crucial. Yes, we were perhaps naive to say that within one year we would like to have a report on what the Government are doing on value-based pricing, but the principle is right, and I hope that when the Minister responds he will say when we can expect to see a review and whether regenerative medicine technologies will be part of it.
In their response to the report the Government have been partly helpful, but there is a long way to go. The noble Lord, Lord Turnberg, and the noble Earl, Lord Selborne, have said many of the things that I wanted to say, so I shall not repeat them. UK regulation is rightly prized and valued across the world as being the yardstick by which other countries judge their regulatory frameworks. We are rightly proud of that, but I remain to be convinced that, despite the great efforts which the Government have made to make the regulatory framework less complex, it is fit for purpose.
Let us remember that our regulatory framework has grown rather like Topsy; it has been built in parts to respond to new developments in science and medicine. Regenerative medicine needs a bespoke regulatory framework to drive it through. We cannot go on saying that we have a complicated regulatory system and that somebody will help you with it. I found it quite depressing when representatives from the MHRA came to our committee and said, “Well, it’s very complicated science. Therefore, you need very complicated regulation”. That does not follow. With complicated science, you have to be able to drive through that science and have essential regulation which is easy to follow, because many companies and research groups that develop these technologies will not be the large pharma companies of yesterday but small groups with relatively small budgets that need an awful lot of hand-holding. To have, as the noble Lord, Lord Turnberg, said, up to 11 regulatory frameworks to go through—and sometimes you have to go back over the hurdle as well—to satisfy Europe as part of the deal means that we have got to help.
Our proposal that there should be a particular group that looks at regulation was turned down; the Government said that they wanted instead a Regenerative Medicine Expert Group. To be fair, one of the three main strands of that will be a work stream on regulation and licensing. I welcome that; it is a reasonable response. Getting Mike Rawlins to chair that is an excellent move. But who will the expert group report to? I understood that the HRA was going to be the authority which looked at all that, yet what we now have is another expert group with another remit in terms of streamlining regulation. When the HRA comes up with a groundbreaking scheme to bring together ethics permissions and local NHS permissions, and it has sat on a desk at the Department of Health since October awaiting an answer when everybody else feels that it is the right way forward, I genuinely feel that we are missing a trick. When the Minister responds, I hope that he will respond to the question that I have asked today. This is an excellent report. I give the Government at least seven out of 10 for their response, but at the bottom of their report, I would always say, “Could do better”.
Baroness Greenfield (CB)
I add my voice to those of the other noble Lords in congratulating the noble Lord, Lord Patel, on bringing attention to this timely and important report. He and others have already spoken eloquently on the wider issues surrounding stem cell research, so I shall restrict my comments to my own particular area: diseases of the central nervous system.
While other conditions such as heart disease and cancer are devastating, we all fear in particular the disorders that destroy our brains. The report discusses the wonderful prospect that in the next five years treatments are likely to be available for stroke and multiple sclerosis. However, only under a section on longer-term possibilities is Parkinson’s disease mentioned.
The neurodegenerative diseases of Alzheimer’s and Parkinson’s target the very essence of what it means to be human: what it means to move freely, to smile, to think, to speak and to have memories—indeed, to be a unique individual. The problem is that, as yet, we do not know why key brain areas in each case embark on the initial cycle of self-destruction or why it occurs only in certain brain regions and not in others. Because we do not currently understand the basic mechanisms, we cannot get to the root of the problem. The best that we can do is to combat the symptoms.
As brain cells die, they release less and less of their essential chemical messengers. Current strategies, therefore, are to offset the dwindling level of those naturally occurring chemicals with drugs, but here the problems are several-fold. First, as with all drugs, the treatment will permeate into areas of the brain and body where it is not needed and hence cause side-effects. For example, with Parkinson’s disease, treatment with the drug in current use, L-dopa, will supply the necessary chemical messenger, dopamine, to the area of devastation, but will also raise levels of the same chemical elsewhere in the brain, and this can often result in psychotic side-effects, with disturbing hallucinations. Even when such treatment offers temporary alleviation of the patient’s basic condition—or slowing down of the deterioration, as in the case of the anti-Alzheimer’s drug Aricept—it has proved hard to convince organisations such as NICE that the costs are worthwhile.
The situation is made even worse when we consider how many more of us are going to need such treatment in the future. Today, nearly a million people suffer from Alzheimer’s or Parkinson’s or both, and that number is expected to double by 2050. The total cost of caring for one person with dementia can be up to £30,000 per patient per year—plus the additional costs caused by loss of earnings.
Even more sobering, beyond the mere economics, is the human cost. For every person suffering from either Parkinson’s or Alzheimer’s, let us say there are 10 people who care about that individual. Hence, as the number in the UK reaches almost 2 million by the middle of this century, almost 20 million lives could be affected by those devastating disorders.
So there is a huge and growing need: a need that is currently unmet. Stem cell therapy offers an exciting and realistic alternative. The rationale is completely different from that of conventional treatments. The idea is not to treat the symptoms, but to harness regenerative biological mechanisms so that new cells are created. That would be a real cure. It would not be merely replacing the chemicals that are lost as a result of cell death, but actually replacing the neurons themselves.
Some cases of Parkinson’s disease have been successfully treated using human foetal cells; however, such tissue is hard to obtain, and the ideal would be switch to human embryonic stem cells. Those cells are derived from very early embryos, at the stage when the embryo is a microscopic ball just a few days old and consisting of only one to 200 cells. Not only are they immortal, they can produce every type of cell in the body. By introducing such cells into the appropriate environment within the brain, they will actually become the brain cells that have been lost.
There are, inevitably, potential downsides, As a neuroscientist, I am unable to comment with any authority on the ethical or financial issues, so I will restrict my caveats to technical issues. The first would be immune rejection of the new cells. However, that can be overcome by immunotolerising patients or even by immunosuppression. Such therapies have side-effects, but the risk-benefit ratio compared to that with conventional drugs is greatly shifted in favour of the positive.
A further problem is that stem cells could proliferate out of control in the brain and therefore become a tumour. However, to date, there is no clinical evidence that that has occurred with stem cell therapy and, in any event, it could be circumvented by biochemical chicanery—for example, manipulating stem cells so that they divide at a few degrees hotter than would normally be the case in the living brain.
Another issue is that implanted stem cells may produce excessive amounts of chemical messenger compared to normal levels. In principle, however, once stem cells have repopulated the brain, they should behave like their naturally occurring predecessors and release chemicals within the normal range as and when they are stimulated and interacting in their normal brain environment. In any event, conventional drugs already produce excessive amounts of chemical messenger, but that can be controlled by current treatments.
Finally, we must be careful not to conflate Parkinson’s and Alzheimer’s diseases. They are very different conditions and are differentially tractable to stem cell therapy. Parkinson’s disease is much more localised in the brain than Alzheimer’s, and therefore it will be much easier to locate where the stem cells should be placed. However, there is often a co-pathology—patients presenting with both Alzheimer’s and Parkinson’s diseases—so in these cases perhaps the alleviation of the movement symptoms of Parkinson’s may help in the patient’s quality of life, not least because we know that the better that people can move physically, the more that they can sustain a good blood supply to the brain.
There is now a growing body of evidence that physical exercise can enhance the natural growth of brain cells, a phenomenon known as neurogenesis, as well as the proliferation of blood vessels, therefore bringing more oxygen to the brain, which improves its functioning. There are even some claims that Alzheimer’s disease could be less prevalent in those who exercise routinely. So a treatment for patients suffering from both Alzheimer’s and Parkinson’s that enabled them to move more freely might in the long term be more generally beneficial.
Some might say that introducing the cells into the brain would be problematic, but the brain surgery required is modest. Modern stereotactic surgery is performed under local anaesthetic, with only a small hole made in the skull and a fine needle introduced—a bit like drilling for oil using precise three-dimensional co-ordinates. The area targeted can then be localised.
In summary, we have reason to be confident that, although not without risks or difficulties, stem cell therapy could be a chance to harness the nervous system’s natural mechanisms to regenerate itself. In the case of neurodegenerative disorders, though, much more research needs to be done.
I commend the authors of the report for increasing the chances that we will,
“facilitate the translation of scientific knowledge into clinical practice and encourage its commercial exploitation.”.
Still, far more money needs to be devoted to research into the use of stem cells in brain disease, which at present is a poor relation to heart disease and cancer. If these recommendations are implemented, the horizons could be very bright, not just for those with Alzheimer’s and Parkinson’s, who are currently condemned to a highly disabled life and an even bleaker future, but for everyone who cares about them.
Baroness Sharp of Guildford (LD)
My Lords, as a member of the committee, I thank the noble Lord, Lord Patel, for his excellent introduction to the debate. I repeat the thanks to the noble Lord, Lord Krebs, who cannot be with us today, who was an excellent chairman, and to Professor Fiona Watt, our specialist adviser.
Working on this report took me back to the 1980s when, in my position in the science policy research unit at the University of Sussex, I was an alien social scientist sitting in as a fly on the wall on an experiment in university/industry collaboration called the Protein Engineering Club, which was an attempt to assist the process of carrying science from the laboratory through to industrialisation and commercialisation. Then, as now with regenerative medicine, there were great hopes about what might be achieved. As we were working with proteins and antibodies, we hoped, for example, that regulation would get easier rather than more difficult because we were working with biological entities rather than chemical entities that were alien to the body.
There was also hope that the rise of the venture capital industry in the United States would rapidly spread to the United Kingdom—there was some indication that it was coming—and that this would lead to a wholesale change in the process of coping with, as they put it, the valley of the shadow of death—the process of financing commercialisation—with a wave of new firms that would be well funded, developed and built up, either contracting to or, as in many cases, being bought up by larger firms.
Looking back on it, the work that was done then has been very much the foundation of the current range of biotechnology medicines that are now coming on to the market, but it was 25 to 30 years ago. It has taken a very long time to get many of these medicines on to the market, and indeed some of them are still working their way on to it. The regulatory process, far from getting easier, has if anything become considerably more difficult and complex. The financing is no easier; venture capital waxes and wanes, largely with the macroeconomy. Big pharma itself has waxed and waned. The sort of work that was being done in the Protein Engineering Club has in many senses provided an underpinning for the technology for the two big British pharmaceutical companies, GlaxoSmithKline and AstraZeneca, in their current success. Nevertheless, there have been many ups and downs in the process.
The lesson that comes through clearly in the report is that there are great hopes for these new medicines but much hype. In paragraph 19, we talk about the possibility of regenerative medicine that may provide treatments for long-term, chronic diseases, such as Parkinson’s, cardiovascular disease and diabetes, but we also say:
“Many submissions to the inquiry offered a ‘health warning’, however, that public expectations must be managed as many of these treatments are relatively far from delivery to the wider public”.
As my noble friend Lord Willis said, regenerative medicine is not a short-term fix.
Our report identified the four main challenges to be overcome. Other noble Lords have spoken at some length about some of the issues. One is the science itself. As the noble Earl, Lord Selborne, said, it is moving under our feet. It is a highly innovative area where new ideas are bubbling up and being tested all the time. In no sense is the science stabilised. It is an extremely stimulating environment, but because things do not stand still, it is always a matter of the science moving forward and innovation having to adapt to the new developments that are taking place. The regulatory framework is increasingly complex—perhaps unnecessarily complex, as my noble friend Lord Willis suggested. We were hoping that an expert group could be set up which would manage to find some way to simplify it. Scale up—the shift from the laboratory to larger scale production—is also a considerable problem. There are always completely unforeseen difficulties in such processes. Cells do not behave on a larger scale as they do in a laboratory dish. Finally, there is the business model. It is very important that there should be patient capital. One is looking at 25 years, a whole generation, for such medicines to come on to the market. There has to be capital that is prepared to put its money down and wait for results.
On the whole it seems to me, as it does to others, that the processes the Government have put in place have been appropriate. As my noble friend Lord Willis said, the Government have played their cards well. They have designated regenerative medicine as one of the eight great technologies and have concentrated on the life sciences with the emphasis on investment in the research base, where we have a considerable comparative advantage. In November 2011, they produced their strategy for the life sciences, reinforcing what they call the “life sciences ecosystem”, and, in particular, bringing together R&D in the National Health Service and academic research supported by the research councils and seeking to exploit what other countries see as the UK’s unique advantage in having a unified health service as a platform for assessing the effectiveness of treatments.
Building on this, and into this, indeed, is the Technology Strategy Board. Its April 2012 report A Strategy for UK Regenerative Medicine dealt with the translation from research into commercialisation and concentrated in particular on the regulatory framework, manufacturing and industrial collaboration. Alongside the TSB initiatives is the Cell Therapy Catapult, which was originally a technology and innovation centre to help develop an emerging industry to be a precursor to what could be a £10 billion industry. The catapult is working on a five-year plan pulling academic and industry plans together and ensuring a voice for this new technology within government here and within Europe.
All of this is very positive, except when we come to what is happening in California. The California Institute for Regenerative Medicine—CIRM—has been raising $3 billion in 30-year bonds. The National Institutes of Health is spending $1.3 billion on regenerative medicine. In the UK, TSB is spending £16.25 million over the next three years; approximately £5.5 million a year. Catapult has core funding of £70 million over five years—£14 million a year core funding—and is hoping that the third sector will add another £10 million, and industry another £10 million, making somewhere in the region of £35 million a year. However, much of that is still a matter of hope, although both sectors are beginning to put more money into this area. Putting it all together, we are looking at something in the region of £70 million to £100 million a year for this sector, compared with the $3 billion that the Californians have raised through 30-year bonds.
Our recommendation 13 to the Government was that the ESRC and the Technology Strategy Board should do an evaluation of innovative funding models. The Government must, we said, put their money where their mouth is: we cannot expect the goose to lay golden eggs unless we feed it. The UK public spending and accounting framework is, to my mind, quite unduly risk-averse and focused on the short term in many senses. The centralisation of all capital funding via the Treasury cuts out intermediate authorities as raisers of capital. This compares with the United States, where states such as California, come in and can raise money for innovation. In Germany, the Länder, again, can go to the capital market and raise money for innovation and then work in conjunction with the Landesbank. There is no encouragement here by the Government of highlyconcentrated funding mechanisms at a regional level. The response of the Government to this recommendation was non-committal at best. They said:
“The TSB and Research Councils will respond to the recommendation to evaluate innovative funding models for late stage clinical development”.
Indeed, the TSB and the research councils welcomed the idea. The Government’s response continued:
“The Government is confident that regenerative medicine has enormous potential which is why we invest in the research base through the Research Councils and support commercialisation through the TSB. We cannot commit to adopting policy recommendations on regenerative medicine that might emerge from an Economic and Social Research Council … and TSB study … although we will consider any recommendations of such a study”.
As I say, that is non-committal at best. Unless we are prepared to think big in the way that California does, we shall end up—as we did in some senses with protein engineering—with positive but relatively little gains from this new technology while others grab the really big gains.
Lord Hunt of Kings Heath (Lab)
My Lords, I very much welcome the debate and I congratulate the noble Lord, Lord Patel, on securing it and commend him, the noble Lord, Lord Krebs, and the committee members for the quality of the report itself. I declare my interests as chair of an NHS foundation trust, president of GS1 and consultant and trainer with Cumberlege Connections.
This subject is of considerable interest to me. I am sure that the noble Earl, Lord Howe, will remember our great debate on the order allowing the extension of embryonic stem cell research over 10 years ago, when your Lordships’ House debated the matter for over seven hours. We agreed to it subject to the establishment of a Select Committee. I think that we played our part in laying some of the foundations for the progress that has subsequently been made.
The report itself is a powerful one. It very helpfully points out the many strengths the UK has in regenerative medicine—strengths we always hoped we would be able to take advantage of—but it also points out some of the issues around private investment, regulation, translation and how we are to take forward developments that look particularly promising as far as patient treatments are concerned.
Overall I welcome the Government’s response, which has been positive. However, it leaves four specific questions, which I will put to the noble Earl. They concern: regulation; the funding of research and development; and manufacturing capacity. I will then look a little further down the line towards the role of the National Health Service and the uptake of such new medicines and developments.
Both the noble Lords, Lord Willis and Lord Turnberg, spoke particularly eloquently about the issue of regulations and the problem we may have because of the number of regulatory bodies involved in regulation. The committee itself wanted to give a stronger role to the HRA; that is an important point on which we look forward to a perhaps more considered response from the Government. However, I was particularly interested in the point raised by my noble friend Lord Turnberg about the issue of clinical trials and the approach of the MHRA as compared to regulators in Japan and the US. That is the issue in Japan, for example. My noble friend cited that approval can be based around phase 2 trials and that what is needed is the adoption of what my noble friend called an adaptive licensing system. I am well aware of that issue.
I believe that the MHRA is a very good agency that does very good work. However, I am not yet convinced that it sufficiently recognises the urgency of making changes to its own approach. I understand that this is difficult, and I have no doubt that it has to work within the context of European directives in that area. However, can the noble Earl give us some assurance that the MHRA and the other regulatory bodies recognise that this country has a big stake in regenerative medicine? We cannot allow overbureaucratic regulatory procedures to get in the way of that.
I know that we have debated regulation on many occasions, and I wonder whether the noble Earl might consider taking a more proactive role himself—because I think he has responsibility in this area—to knock heads together between the different regulatory bodies. We have huge potential here; it would be a very great pity to lose it because our regulatory bodies were not able to keep up with the science.
Can the noble Earl respond to the issue of research investment? We will never be able to match the kind of investment that the US makes. However, we have clearly shown that we can produce very good results with investment at UK levels. Hearing the sums—the noble Baroness, Lady Sharp, very helpfully went through them—I think that they are pitiful compared to the kind of sums that need to be invested. I would like to hear some more about how the Government think that they, the medical charities and other sources can increase our effort in this area.
Manufacturing capacity has been raised as an issue. I welcome the Regenerative Medicine Expert Group, but I saw no reference in the work streams to the issue of manufacturing capacity. Can the noble Earl say a little more about how the Government think they ought to be able to encourage more manufacturing capacity in the UK?
Finally, I will ask the noble Earl about uptake by the National Health Service, which again is an issue that we have debated and which will become a big problem in the future. We know that the NHS, wonderful though it is, is very conservative as regards uptake of new medicines and treatments. The reason NICE was created was to encourage the NHS to do better. Yet in a recent report, as regards technology appraisals, which clinical commissioning groups are by law duty-bound to fund, it is clear that the take-up varies 20-fold for some important new technologies in England. We cannot allow the caution of the NHS over the uptake of new medicines to get in the way of making the most of regenerative medicines. I would be interested in the Minister’s view of how we can get the NHS to play a much stronger role.
The Regenerative Medicine Expert Group is very much to be welcomed. All of us respect highly Sir Mike Rawlins, who is an excellent choice as chairman, and I note that the group is expected to conclude its work by the end of 2014. It would be very helpful to the House if the Minister could be clear with us that the Government will take the report seriously and, on the back of it, will be prepared to reconsider some of the points that they have made in answer to the excellent report of your Lordships’ House.
The Parliamentary Under-Secretary of State, Department of Health (Earl Howe) (Con)
My Lords, first, I congratulate the noble Lord, Lord Patel, on securing this debate and congratulate the Select Committee on Science and Technology, chaired by the noble Lord, Lord Krebs, on its excellent work in highlighting the important issues associated with the development of the regenerative medicine sector in the UK. The Government welcome the committee’s report and recommendations and agree that it is very important that the translation and commercialisation of research in this area in the UK is enabled.
As noble Lords recognised, regenerative medicines have enormous potential to treat and cure diseases, including in areas where no cure yet exists, to improve the quality of people’s lives and generate significant economic benefits for the UK. The Government remain committed to developing this important field of medicine, which we recognise as one of the UK’s eight great technologies. As the noble Lord, Lord Patel, acknowledged, the UK, with its strong science base, research funding and regulatory frameworks, and access to patients provided by the NHS, retains a strong international position to support the successful commercial translation of regenerative medicines. That said, more can be done to support and enhance the development of regenerative medicines in the UK.
I am grateful to the committee for the recommendations that it made on ways in which this area of medical science can be better supported. The actions that the Government are taking are set out in detail in our response to the report, but I would like to take this opportunity to highlight a number of areas of important activity.
The Government are continuing to invest to support translational health research on regenerative medicines, and this remains a high priority. A number of noble Lords, including the noble Lord, Lord Patel, asked for some reassurance that the NHS was ready for regenerative medicine clinical trials. As he knows, the Department of Health’s National Institute for Health Research—the NIHR—funds infrastructure in the NHS for translational research in regenerative medicine, in particular, as my noble friend Lord Willis mentioned, through biomedical research centres and units. These are established in leading NHS and university partnerships to drive progress on innovation and translational research in biomedicine into NHS practice. In 2011, the Government announced £800 million of NIHR funding for five years from April 2012 for 11 biomedical research centres and 20 biomedical research units. As part of this, the NIHR is investing more than £9 million annually in research programmes within its BRCs and BRUs that involve significant cutting-edge translational research in regenerative medicine across a range of disease areas.
This approach is already bearing fruit. For example, scientists at the NIHR biomedical research centre at Guy’s and St Thomas’s, and King’s College London have, for the first time, identified the unique properties of two different types of skin cells, including those responsible for repairing skin wounds. This research could pave the way for new and effective treatments to repair injured skin and reduce the impact of ageing on skin.
In addition to these centres and units, the NIHR funds infrastructure for regenerative medicine studies through its clinical research facilities and the Clinical Research Network. The Government’s response to the committee’s report highlighted work to implement changes to the organisational structure of the Clinical Research Network to take effect from 1 April, and I can report that these changes are on target.
The noble Baroness, Lady Greenfield, in her highly informative speech, spoke about the development of regenerative medicines to treat neurodegenerative diseases, including dementia. The Government have established the new National Institute for Health Research Dementia Translational Research Collaboration to pull discoveries from basic science into real benefits for patients. This brings together resources within the NIHR biomedical research centres and units.
As part of the collaboration, the NIHR Queen’s Square Dementia Biomedical Research Unit recently held a workshop on the use of stem cells in dementia and other neurological diseases, without—as the noble Baroness emphasised—conflating very distinct conditions. In addition, the NIHR Clinical Research Network will be responsible for delivering clinical research studies across a full breadth of specialties, which will include dementia and neurodegenerative diseases. Studies in regenerative medicine will be supported within the relevant specialty.
Regenerative medicine research has also benefited from the Regenerative Medicine Platform, the Technology Strategy Board and the Biomedical Catalyst, which have made significant investments in this area. Furthermore, noting the committee’s observation that there are a large number of different research and development funders, I am pleased to report, particularly to my noble friend Lady Sharp, that a single, interactive research “funding portal” is in development. We anticipate that it will be rolled out this spring. This portal will support researchers across academia and industry to identify and access relevant funding opportunities. As noble Lords have suggested, there should be a clear pathway from development to delivery of regenerative medicines in the NHS. This will aid the growth of this sector so that effective regenerative medicines become readily available and provide benefits to patients.
The healthcare system in the UK has already been delivering regenerative medicines successfully for decades, as shown through stem cell or tissue transplantation and through medicines such as erythropoietin. As the noble Lord, Lord Patel, pointed out, it is important to remember the breadth of the therapies that fall under a regenerative medicine definition, ranging from well established bone marrow transplantation procedures through to those at early-stage research. Each regenerative medicine product that is developed will differ by its mode of action, cost and therapeutic application.
My noble friend Lord Selborne referred to some new therapies appearing over the horizon. For new innovative therapies we are implementing recommendations from the Innovation, Health and Wealth report to spread innovation quickly and at scale throughout the NHS to improve outcomes and quality for patients and the NHS. I recognise fully the concern of the noble Lord, Lord Hunt, about the pace of uptake in the NHS of NICE-approved medicines; it is a concern that I share.
NHS England’s Commissioning through Evaluation programme provides an opportunity to strengthen the information we have available to inform commissioning policy for procedures or treatments that show significant future promise, but for which the evidence base on clinical and cost-effectiveness is currently insufficient to support routine commissioning. This is particularly important for rarer or smaller-volume treatments, such as some regenerative medicine treatments, where randomised controlled research evidence may be less readily available. We have also asked NICE to develop multiple-technology appraisal guidance on autologous chondrocyte implantation, a regenerative medicine for repairing symptomatic articular cartilage defects of the knee.
As noble Lords mentioned, in order to develop an NHS pathway for regenerative medicines, we have established a Regenerative Medicine Expert Group, as recommended by the committee, to develop an NHS regenerative medicine delivery readiness strategy and action plan. We are pleased that Professor Sir Michael Rawlins has agreed to chair this group, which is of a multidisciplinary nature with all the key stakeholder groups represented within the membership, including NHS England, the National Institute for Health and Care Excellence, regulators, industry, researchers, patient representatives, NHS Blood and Transplant, the Scottish National Blood Transfusion Service, the Welsh Blood Service and the Cell Therapy Catapult.
The noble Lord, Lord Patel, asked about the group’s terms of reference. These can be found on its website, which is located on the Department of Health’s website. However, in essence, we have tasked the group with considering all the important elements of the pathway of regenerative medicines into the NHS, including licensing and regulation of a product, evaluation, commissioning and reimbursement, as well as practicalities such as manufacturing and distribution. I can tell the noble Lord that the group has been asked to report to the Secretary of State by the end of this calendar year, and its work is well under way. It will deliver an NHS regenerative medicine delivery readiness strategy and action plan.
I am sure that the noble Lord, Lord Turnberg, will be glad to know that as well as NHS delivery we have also tasked the Regenerative Medicine Expert Group with looking at the regulatory system for regenerative medicines. My noble friend Lord Selborne referred to this, as did my noble friend Lady Sharp and other noble Lords. The Government recognise, as did the committee, that regulation in this area may act to stifle innovation and thus that we should endeavour to simplify systems to provide support for those developing regenerative medicines to navigate regulatory pathways. With these aims in mind, the main regulatory bodies are working closely with one another to streamline the regulatory system and provide support to help guide applicants with regulatory submissions—for example, the Health Research Authority is undertaking work to streamline research approvals and has a new website to guide researchers. Also, we can look to the joint working of the MHRA and the Human Tissue Authority as a result of the McCracken report recommendation on the regulation of tissue for applications aimed at developing regenerative medicines. I hope that those initiatives will be of particular comfort to my noble friend Lord Willis.
I should like to emphasise to him, to my noble friend Lady Sharp and to the noble Lord, Lord Hunt, that the Government remain committed to streamlining research approvals.
Lord Willis of Knaresborough
Before my noble friend leaves that point, I asked specifically—as did the noble Lord, Lord Turnberg—when a decision will be made on the initiative of the Health Research Authority to bring together and streamline NHS approvals and local ethics approvals. The decision has been awaited since October.
Earl Howe
My noble friend anticipates some news that I was about to convey. He is right: the Health Research Authority is the organisation created to deliver the streamlining of research approvals. It has completed its feasibility study. The results demonstrated that NHS R&D assessments could be integrated with elements of the research ethics committee review into a single HRA assessment for the approval of all research in the NHS. Department of Health officials are scrutinising the business case submitted by the HRA as part of standard governance processes, and approval of the case is subject to the proposals demonstrating value for money. Consideration of the business case is well advanced and we would anticipate that this process will conclude shortly.
The noble Lords, Lord Patel, Lord Turnberg and Lord Hunt, asked me about support for manufacturing, in particular as regards large-scale trials. The Cell Therapy Catapult has recently completed its survey of regenerative medicine manufacturing capacity in the UK, and an appraisal of national capability is planned on an annual basis to keep abreast of the evolving needs of the area and to ensure that the UK remains globally competitive. The 2013 survey and analyses that the Cell Therapy Catapult compiled have been shared with stakeholders. The key findings of the survey, including a demand forecast, have been shared at various meetings in the UK, including at the Regenerative Medicine Expert Group, and at meetings of the advanced therapeutic products manufacturing community and will be published shortly. The survey output, along with analysis of demand, was used to make a proposal to BIS for further investment to fill the cell manufacturing gap and support late-stage clinical trials. The investment proposal is currently being assessed.
The noble Lord, Lord Patel, asked what action UKTI has taken to improve the chances of the UK being a location for the development and manufacture of regenerative medicine therapies. UKTI has consulted extensively with UK stakeholders and has developed a new UK regenerative medicine sector proposition, which was launched in December 2013 at the World Stem Cell Summit in San Diego. Training on the materials has been rolled out to a number of its overseas teams, with more training to follow in the coming months.
The noble Lord, Lord Hunt, made some criticism of the MHRA in the context of progress on adaptive licensing. This issue was also raised by the noble Lord, Lord Turnberg. The MHRA has been involved with a discussion group at the European Medicines Agency in developing guidance, case studies and draft calls for expressions of interest to go out this year. One has to remember that work on adaptive licensing must be conducted within the context of European law. We had hoped that this would be issued last year but there has been a delay, as the European Commission has wanted to be satisfied that proposals can be accommodated in the existing regulatory flexibilities. I can tell the noble Lord, Lord Hunt, that we continue to be actively involved in pushing the EMA in bringing this work forward, and I have been personally involved in overseeing that.
Distinct from the concept of adaptive licensing is the early access to medicines scheme. This is designed to enable earlier UK patient access to highly promising medicinal products before they are licensed. This is expected to be announced very soon. It will operate within the current regulatory structure, and is voluntary and non-statutory. The MHRA will provide a scientific opinion on promising new medicines that will treat, diagnose or prevent life-threatening or seriously debilitating conditions without adequate treatment options before the medicines are licensed. Further details will be announced in the near future.
My noble friend Lord Willis asked about NICE’s value-assessment process. NICE, in consultation with stakeholders, keeps its methodologies under review to ensure that they remain fit for purpose. Our priority is to make sure that we get the best possible results for all NHS patients with the resources that we have, which means using taxpayers’ money responsibly and getting good value for money. We have asked NICE to look at how drugs are assessed so that patients can get the treatments that they need at the best value for the NHS and so that the price that the NHS pays is more closely linked to the value that a medicine brings. NICE will carry out a full public consultation before implementing any changes. I would just add that NICE is a key member of the Regenerative Medicine Expert Group, which will look at and provide recommendations on the evaluation and commissioning of these novel medicines and their adoption in the NHS.
My noble friend Lord Selborne and the noble Lord, Lord Turnberg, referred to developments in Japan. We are aware of the Japanese plans, and the report on the approach is being considered by the expert group. As noble Lords have mentioned, the Japanese Government are exploring ways in which the regulatory process there might be changed to support earlier evaluation of the clinical effectiveness and adoption of regenerative medicines within their health system following evidence of safety. Earlier this year, a Department of Health official, along with Foreign Office officials, attended a conference in Japan where the plans were discussed. The details have yet to be worked out but a watching brief will be maintained with the contacts that were made. A report of the conference has been made available to the expert group for consideration.
The Government look forward to receiving the Regenerative Medicine Expert Group’s strategy and action plan. We anticipate that this will provide a platform to help ensure that the UK will be in the lead in realising the exciting medical and commercial potential of these cutting-edge treatments.
Lord Patel
My Lords, I thank the noble Earl for his detailed response and all other noble Lords who have taken part in this debate. It has been a very good debate, which went wider than the inquiry report. I look forward to another debate when the report of the expert working group comes out; we will know then whether progress has been made.