Regenerative Medicine: S&T Committee Report

Lord Patel Excerpts

Thursday 13th March 2014 Lords Chamber

Moved by

[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.

--- Later in debate ---

[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.

Motion agreed.