Contents
Introduction 3
1. 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... 6
2. The Health Research Authority (HRA) has made some positive first steps and it must now demonstrate its effectiveness by streamlining the macro regulatory environment... 6
3. 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... 8
4. 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)... 9
5. We recommend that the phase Ⅱ disease teams of the TSB regenerative medicine platform, and other regenerative medicine funding programmes, specifically require researchers to invole manufacturing and scale-up experts in their development process... 10
6. 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... 10
7. 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... 11
8. 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). 11
9. 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... 11
10. 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... 12
11. 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... 13
12. Furthermore, given the large number of potential funders, the TSB, research councils and NIHR should produce and online funding guide, regularly updated, to help researchers and SMEs know where they should apply at each stage of research... 13
13. 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... 14
14. 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... 14
15. 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... 14
16. 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). 15
17. 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... 15
18. 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... 16
19. 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... 17
20. The UK Government must ensure that ist pricing and reimbursement systems are fit for purpose otherwise companies will base themselves in other countries (paragraph 150). 17
21. 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... 17
22. 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... 18
23. 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... 19
24. 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... 20