Regenerative medicine - Science and Technology Committee Contents


Chapter 6: Conclusions and recommendations

The value and importance of regenerative medicine

161.  The weight of evidence to our inquiry was that regenerative medicine has the potential to deliver new, innovative therapies, or even cures, where conventional approaches do not provide adequate solutions (paragraph 19).

162.  Regenerative medicine has the potential to cure or provide more effective treatments for a number of chronic diseases, which would be of major benefit to the UK public purse given rising expenditure on healthcare associated with chronic disease management and related indirect costs (paragraph 21).

Uncertainty

163.  For a regenerative medicine industry to flourish in the UK steps must be taken to clear the path "from bench to bedside" as part of building investor confidence (paragraph 56).

Regulatory environment

164.  A reputation for proportionate regulation is important for the UK both in terms of inspiring confidence of potential patients and encouraging investment (paragraph 57).

165.  The twin challenges of improving perceptions of the regulatory system and streamlining it are so great that both immediate and long-term action are needed (paragraph 70).

166.  We recommend that, as a matter of urgency, the HRA establish a regulatory advice service. This would build on the expertise of the Office for Life Science toolkit, the newly established MHRA Innovation Office and the experience of regulators. Researchers and companies require more than a web-based service. They should be assigned a single point of contact to support them in navigating the regulatory system, directing their queries to others where appropriate, but retaining ownership and oversight of the advice process. Such a service would be of short-term value to this (and the broad healthcare) sector until such a time as the regulatory environment is rationalised (paragraph 71). (Recommendation 1)

167.  The Health Research Authority (HRA) has made some positive first steps and it must now demonstrate its effectiveness by streamlining the macro regulatory environment. We recommend that the HRA commission an independent advisory group, made up of national and international experts in regulation, to develop a designed-for-purpose regulatory system. The UK rightly has a good reputation for its robust regulatory system; it is vital that this reputation be maintained. Similarly, we acknowledge there is significant value in the expertise of some regulators. But patients, business and the taxpayer deserve a modern, designed-for-purpose, efficient regulatory system rather than one that has evolved in a haphazard, piecemeal way. An independent advisory group supporting the HRA will give it the necessary support to focus and clarify the functions of regulators. This group should give special consideration to reducing the overall number of regulators. We recommend that the group make proposals 18 months from its establishment. We will revisit this aspect of the inquiry to ensure that progress has been made. The HRA must simplify the regulatory route so that the development of regenerative medicine, and other innovative therapies, is not hindered (paragraph 73). (Recommendation 2)

Clinical trials

168.  The Government must therefore identify how the UK can become a more attractive venue for clinical trials as, currently, the number of trials does not reflect its significant benefits (paragraph 76).

169.  The evidence received conveys considerable demand for greater support in the design and set-up of clinical trials. There is expertise in clinical trial design and set-up in the NIHR CRN, its BRUs and BRCs, and amongst academics exploring innovative trial design. There is also considerable expertise in NICE which could help inform trial design to ensure outcomes meet its evaluation requirements, the MHRA which already offers an advisory service, and amongst manufacturing experts from both industry and academia, who could provide advice to ensure that therapies are developed in a scalable fashion. Each of these groups would benefit from greater two-way interaction: to inform regulation and guidance making, and product development and trial design (paragraph 88).

170.  Consequently, we recommend that the NIHR establish a regenerative medicine stream of its clinical research network. Such a move would support researchers in addressing the specific needs of regenerative medicine clinical trial design, help overcome difficulties in identifying patients and ensure that doctors interested in such trials could be easily identified. The network could also facilitate dialogue with regulators on future regulatory needs and issues encountered with regulation. The regenerative medicine stream of the network should employ a hub and spoke model for allogeneic treatments, whereby it has one or two co-ordinating centres and regional operations. Given the need for clinical trials of a certain size, this network should span across the UK and build on existing developed infrastructures like NHS Research Scotland (paragraph 89). (Recommendation 3)

171.  The NHS would be a very attractive location for trials with these improvements, and there are reciprocal benefits to the UK in the form of inward investment, gaining further experience, potential for early market adoption and thus availability to NHS patients. The Government must ensure that this opportunity is not missed (paragraph 90).

172.  Therefore, we recommend increased dialogue between regulators and researchers in the form of regular regenerative medicine workshops, and that the MHRA produce a series of guidance notes (to be updated bi-annually) setting out clinical trial endpoint requirements for regenerative medicine, in consultation with the industry and academic researchers. UK regulators should learn from the example of FDA-CIRM workshops and similar efforts in other countries (paragraph 91). (Recommendation 4)

Scale-up and manufacturing

173.  We recommend that the phase II disease teams of the TSB regenerative medicine platform, and other regenerative medicine funding programmes, specifically require researchers to involve manufacturing and scale-up experts in their development process to ensure that translational work is scalable and therefore deliverable to a large number of patients (where the disease area requires this) (paragraph 98). (Recommendation 5)

174.  Recognising the importance of capacity to deliver therapies at scale, both for trials and wider patients populations, and the fast-moving pace of the manufacturing and scale-up field, we recommend that the TSB and EPSRC undertake an annual stock-take of regenerative medicine manufacturing capacity and make recommendations to BIS about future needs, with the first survey informing the Government's review of infrastructure investment. The Cell Therapy Catapult has begun work on such a survey so we recommend that this work is taken as a starting point. BIS must then act to ensure that appropriate infrastructure investment is made to support the field. At the very least, investment should be made in facilities to support the scale-up of treatments in mid to late stage clinical development. Money for this, and other recommendations, should be found by the re-prioritisation of budgets and innovative funding methods (paragraph 100). (Recommendation 6)

175.  UK capacity to manufacture at scale could be attractive to companies considering investing in or expanding operations to this country. We recommend that the UKTI Life Science Investment Organisation use the results of this survey to advise foreign companies on UK capacity to manufacture regenerative products (paragraph 101). (Recommendation 7)

176.  We recommend that the NHS develop a training programme for technical staff to support the development of cell therapies and other regenerative therapies at scale (paragraph 103). (Recommendation 8)

177.  We recommend that the MHRA canvas views from industry on the suitability of current GMP requirements and, if there is significant discontent, take these problems to the European Commission to seek agreement on overcoming them through amendments to the GMP Directive and associated guidance (paragraph 105). (Recommendation 9)

Delivery

178.  It is clear that the national blood and transfusion services have the logistical capability to collect, produce, store and transport components of regenerative treatments. However, we were concerned to see that the NHS is less ready for the provision of regenerative therapies (paragraph 108).

179.  Investors need to see a clear pathway from development to delivery in the NHS if they are to have the confidence to invest in regenerative medicine. It is not sufficient to rely on trail blazing therapies to forge pathways to clinical delivery. The NHS must shift from reacting to regenerative medicine to a state of preparedness to deliver new and innovative treatments (paragraph 109).

180.  We recommend that the Department of Health establish a regenerative medicine expert working group to develop an NHS regenerative medicine delivery readiness strategy and action plan by December 2014. This group should report to the Secretary of State for Health directly and have the support of a high-profile, independent chair. The group must also contain NHS England officials, NHSBTS and devolved blood and transfusion services, regulators, researchers and industry representatives. We consider the role of the chair further in Chapter 5 (paragraph 110). (Recommendation 10)

Business models, venture capital and the funding gap

181.  Investment could be stimulated by reducing associated risk (paragraph 115).

182.  The Cell Therapy Catapult was only set up in May 2012 and we recognise that there is significant potential in the venture. However, we are concerned that it is seeking to achieve too much, too quickly, given the level of funding. We recommend that the TSB and Cell Therapy Catapult prioritise its activities to enable the Cell Therapy Catapult to focus on taking high growth potential projects through clinical trial to be phase III trial ready and developing links with the regenerative medicine community (paragraph 124). (Recommendation 11)

183.  Furthermore, given the large number of potential funders, the TSB, research councils and NIHR should produce an online funding guide, regularly updated, to help researchers and SMEs know where they should apply at each stage of research and development in regenerative medicine (paragraph 125). (Recommendation 12)

184.  There is insufficient TRL 6-8 funding available to support this fast-developing field. It would be unrealistic to depend exclusively upon additional funding coming from venture capitalist or "big pharma" investment. A mechanism must be found to fill this gap. Therefore, we recommend that the ESRC and the TSB commission an evaluation of innovative funding models, which spread risk and most likely will contain a degree of government matched funding or be underpinned by government guarantees, and recommend how additional funding could be provided for late stage clinical development in this field. The Government have said that this field has enormous potential and that they will support it. They must "put their money where their mouth is"; BIS and Her Majesty's Treasury must adopt the policy recommendation of the ESRC and TSB study (paragraph 127). (Recommendation 13)

Intellectual Property

185.  There is significant commercial potential in the enabling tools and technologies, and commercial know-how associated with regenerative medicine—the regenerative medicine community must ensure that investors are aware of this potential. UK Trade and Investment has a specific programme to attract inward investment in regenerative medicine and so we recommend that they support the field by informing investors about the economic potential of investment in the field (paragraph 133). (Recommendation 14)

186.  There is significant scope for patenting within the field and much of the negative publicity around the Brüstle ruling seems to have overstated the implications (paragraph 135).

187.  Concern over the cost of patenting, the sufficiency of support available for innovators and questions about the ability of universities to recognise the potential in regenerative medicine patents lead us to conclude that the TSB should set-up a time-limited support fund for regenerative medicine patents. This fund should be open to university researchers who wish to pursue patents beyond the first stage, so that potential income from regenerative medicine products is not lost. Such a fund would help foster this fledgling industry and be a helpful tool until university patent offices are better placed to deal with the potential value of these products (paragraph 137). (Recommendation 15)

Evaluation and the pricing of treatments

188.  We consider the NICE model for evaluating innovative treatments and cures to be inappropriate. It must devise suitable models that give appropriate consideration to the long-term savings sometimes offered by high up-front cost treatments (paragraph 142). (Recommendation 16)

189.  The first few regenerative medicine products will invariably be more expensive than products further down the line. Other countries, such as France, have evaluation and reimbursement systems which provide for this. NICE must ensure that its evaluation process recognises the higher initial costs of innovative treatments, without compromising its goal of assessing value-for-money in healthcare. Part of its value-for-money consideration should be that early investment in this field could unlock other treatments with significant economic impact, both in terms of savings to the health system and increased potential work productivity (paragraph 143). (Recommendation 17)

190.  Value-based pricing may resolve the difficulties which companies with high up-front cost treatments that provide long-term savings currently experience when seeking approval, but the devil will be in the detail of the system. We recommend that the Department of Health commit to an evaluation of value-based pricing after the first year of operation. We have no doubt that other Parliamentary committees, such as the House of Commons Health Committee, will keep a watching brief on this area—this is vital as appropriate reimbursement is of great importance to the health of both this emerging industry and the established pharmaceutical industry (paragraph 146). (Recommendation 18)

191.  NICE must ensure that it gives guidance to companies developing novel treatments on how to demonstrate comparability. One mechanism for this may be the seminars, developed as part of the life science strategy, which aim to show innovators how to demonstrate value. NICE's processes must allow for difficulties in demonstrating comparability for innovative treatments (paragraph 148). (Recommendation 19)

192.  The UK Government must ensure that its pricing and reimbursement systems are fit for purpose otherwise companies will base themselves in other countries (paragraph 150). (Recommendation 20)

Risks of regenerative medicine tourism

193.  In an era when access to information about these offerings, and ability to travel, is so great, the UK Government must take action to protect its citizens from rogue therapies at home and abroad. The primary tool to combat this is information. Patients must have access to information about the safety and efficacy of these types of treatments. The Government recommend that patients always consult their physicians about the possibility of travelling for treatment—this is, of course, correct. Furthermore, the NIHR has produced guidance for patients considering travelling abroad for treatment. We recommend that the Foreign and Commonwealth Office (FCO) partner with the Department of Health to develop a website, in the same model as FCO travel advice for countries, which, in the first instance, contains summary assessments of the strength of safety measures in place for innovative therapies abroad. In time, they might develop this further, in partnership with organisations such as the International Society for Stem Cell Research (who have begun work in this area), to identify unproven therapies and those who provide them (paragraph 152). (Recommendation 21)

Hospital exemption

194.  The current EU ATMP Regulation is unclear. Terminology used such as "preparation on a non-routine basis" leaves too much room for interpretation. There is also uncertainty about whether a hospital exemption is still permissible when a fully validated, centrally approved Advanced Therapy Medicinal Product (ATMP) is available. We recommend that the UK Government, during the review of the ATMP Regulations, make the case at the European Commission level for clarity on these two points in the revised Regulations (paragraph 155). (Recommendation 22)

Harmonisation

195.  To realise the full potential of this global industry, and to ensure the UK is an attractive location for regenerative medicine companies to invest in and to undertake their clinical trials in, the UK Government must take the lead in promoting harmonisation of regulatory requirements (paragraph 156). (Recommendation 23)

Co-ordination and final conclusion

196.  Regenerative medicine has the potential to save lives and to help support the UK economy. The UK has a great potential resource in the NHS which could make it an attractive place for investment. But the UK is currently underprepared to realise the full potential of regenerative medicine. The many words which have been spoken about regenerative medicine must translate to action, and quickly. We must not miss out on this opportunity to lead the world in this work (paragraph 159).

  1. Accordingly, we recommend that the Government also appoint the chair of the independent regenerative medicine delivery expert working group as the UK's regenerative medicine champion. This person would foster links between the many stakeholders (including, but not limited to, investors, basic scientists, clinicians, manufacturing experts, delivery networks, regulators), drive forward the regenerative medicine agenda and represent the UK's interests on the global stage. This champion should have a budget and support from a Government office (paragraph 160). (Recommendation 24)



 
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