Regenerative Medicine: S&T Committee Report

Lord Turnberg Excerpts

Thursday 13th March 2014 Lords Chamber

[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.