Cytori responds to House of Lords in UK...along with 60 other big players
Cytori Therapeutics is very pleased to have the opportunity to respond to the Call for Evidence in relation to the on-going inquiry into regenerative medicine. Cytori is one of the world’s leading regenerative medicine companies, manufacturing medical devices for the extraction, concentration and storage of adult stem cells derived from adipose tissue.
We thought that it might be helpful to emphasise at the outset that the regenerative medicine sector covers a wide variety of therapies including gene therapy, the use of embryonic stem cells, the use of ‘adult’ cells derived from third parties (allogeneic cells), and the use of a patient’s own (autologous) cells. These different uses and sources each give rise to their own challenges and opportunities which are sometimes markedly different.
Cytori manufactures Celution®, a medical device which enables stem cells to be derived from the patient’s own adipose tissue for therapeutic use. Celution is a closed, automated system which can be used to prepare a regenerative medicine product based on the patient’s own cells at the point of care (in theatre, at the bed side, or within a hospital). As the cells are generally extracted and processed within one hour, the Celution device enables clinicians to extract and use adipose-derived stem cells within the same surgical procedure.
Stem cells derived from a patient’s own adipose (fat) tissue are recognised as one of the best sources of therapeutically relevant stem cells. There are a large number of clinical trials underway globally investigating various therapeutic applications of such cells, and Cytori is actively pursuing a number of these therapeutic applications with a number of clinical partners globally.
Cytori has also developed a system (known as the StemSource Cell Bank) to allow patients to store their own cells for future use.
We have answered select questions in the Call for Evidence, addressed in turn below.
. Research Base
How does the UK rank internationally in the field of regenerative medicine?
The UK is a global leader in the application of and research in regenerative medicine. The UK’s reputation in this field was a decisive factor in first attracting Cytori (a US-based company) to the UK.
The UK’s scientific track record in pioneering scientific advances in regenerative medicine and the basic science underpinning it is well known internationally. Less well known is the fact that, in principle, the UK’s healthcare landscape allows for faster and more effective market entry than other systems. The NHS, as a national healthcare programme, presents a clearer path to market entry and coded reimbursement than many other systems in the world. Device adoption and handling within the NHS is also more stratified and rigorous compared to many private systems.
. Application of the Science
Cytori Therapeutics – Written evidence
Is the science being translated into applications? What are the current applications of the science of regenerative medicine in the UK and internationally?
Cytori’s Celution device has been available for use in Europe since 2006, during which time more than 5,000 patients have been treated using adipose-derived stem cells obtained using the Celution device without any serious safety concerns or adverse incidents.
We have been involved in a number of clinical trials to examine the use of adipose-derived cells in a variety of clinical settings, including:
The treatment of cardiovascular disease for patients who have undergone myocardial infarction or suffer from chronic myocardial ischaemia; and
Post-mastectomy breast reconstruction.
More details about our on-going clinical trials are enclosed as Appendix 1 (not published here).
Beyond these trials, we are aware that a number of clinicians are independently using the Celution device to investigate the use of adipose-derived stem cells to assist the treatment of (for example) wound healing, renal failure and peripheral artery disease. We enclose a summary of studies involving adipose-derived cells at Appendix 2.
. Barriers to Translation and Commercialisation, and International Comparisons
Rather than addressing individual questions in the remaining categories, we thought it would be useful to provide a brief overview of our experience and approach which will answer many of the questions raised.
In our view, the regulatory framework established by various EU Directives and Regulations ensures that autologous stem cells derived from a patient and used within the same surgical procedure are appropriate. Medical device laws require Cytori to:
ensure that the Celution device satisfies applicable ‘Essential Requirements’, as set out in the Medical Device Directive, addressing the quality and safety of the Celution device;
btain a certificate of conformity from a Notified Body (the organisation which has responsibility for confirming that a medical device conforms to the applicable standards) to confirm that the technical, scientific and clinical evidence supports the conclusion that the device satisfies the applicable Essential Requirements;
propose specific clinical indications for the Celution device, which are also #$%$ed by the Notified Body;
establish and pursue a post-marketing surveillance plan (which itself must be verified by the Notified Body) to #$%$ the clinical use of the device in the real world. In the case of the Celution device, Cytori has committed to conducting a number of Post-Marketing Clinical Follow-Up studies; and
Cytori Therapeutics – Written evidence
report any serious adverse incidents123 to regulatory authorities.
Further, the clinicians using the Celution device must do so in accordance with applicable clinical and professional standards.
In our experience to date, the UK regulatory and commercial environment facilitates rather than obstructs the translation and commercialisation of regenerative medicine. As set out above, the framework is demanding, but clear and consistent.
Our positive experience of the UK environment is reflected in the fact that Cytori recently decided to appoint UK-based BSI as its European notified body in preference to our former notified body (which was based in a different European jurisdiction). This decision was made specifically as a result of the regulatory environment in the UK and the approach of this particular UK notified body. We found that BSI was not only more knowledgeable about the application of advanced therapies in this field, but also applied a more contextual, risk-based #$%$ment which combined a focus on patient safety with a practical understanding of the clinical context and clinician-led application of our device.
Similarly, our experiences of dealing with the MHRA and the HTA have also been positive. On the whole, we have found both agencies to be constructive and balanced in their application of UK and EU law in a manner that is both consistent and pragmatic, while balancing risks according to the principles of proportionality. We have had no difficulties or obstacles in the conduct of our UK clinical trials, nor have we encountered barriers to the commercialisation of our product in the UK.
Unfortunately, EU Regulations and Directives are not been implemented or applied harmoniously throughout all EU Member States, and we have found certain other national regulators to be inconsistent and unduly restrictive in their approach to the regulation of regenerative medicine. In this dynamic and fluid field, regulators can adopt an overly restrictive approach, and misapply rules intended for other scenarios or products. In some contexts and countries, this has led to regulatory inertia, with officials unwilling to issue clear and practical guidance, focusing instead on the rigid application of rules. In contrast, the UK has published guidance on some key points of interest to Cytori, and this has greatly helped us shape our plans for the future.
We know that the UK is perceived by other EU Member States as a pre-eminent regulator in this sector. We would therefore encourage the HTA and MHRA to continue to take an approach with European colleagues that pursues a collaborative, risk-based, evidence-led approach which focuses on reducing regulatory burdens and unwarranted bureaucratic barriers wherever possible without ever compromising the safety of patients. This could also have the effect of ensuring that the UK is the preferred point of entry into Europe for companies based in the US and elsewhere.
While good and proportionate regulatory oversight is crucial, a clear route to market is the main driver of technological progress and patient benefit.
In the UK, the existing systems, while not specifically oriented towards new advances in regenerative medicine, nonetheless provide sufficient flexibility in our experience. The
123 The obligations relating to serious adverse incidents are set out in the Medical Device Directive. 108
Cytori Therapeutics – Written evidence
over regulation of regenerative medicine runs the risk of stifling innovation and forcing the technology, the associated companies and their jobs to other, more favorable locations. In our experience, however, the UK has maintained one of the most attractive and facilitative environment in the EU.
The over-regulation of regenerative medicine (or even an inconsistent approach to regulation) runs the risk of stifling innovation and forcing the technology, the associated companies and their jobs to other, more constructive locations. This could also have the effect of depriving patients of the opportunity to access the therapeutic potential of their own cells. We enclose (at Appendix 3) a number of testimonials from patients who have been treated by doctors in the UK with their own cells obtained from the Celution system.
In our experience, however, the UK has maintained one of the most attractive and facilitative environments in the EU. We hope that the Committee will support the regulatory framework and encourage UK regulators to continue to adopt a proportionate and pragmatic approach to the regulation of stem cells.
20 September 2012