Regenerative Medicine: S&T Committee Report

Lord Willis of Knaresborough Excerpts

Thursday 13th March 2014 Lords Chamber

[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”.

--- Later in debate ---

[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.