The House of Lords inquiry into regenerative medicine: mapping the UK route for the commercialisation of cell therapies
The UK House of Lords Science and Technology Select Committee (1) has launched an inquiry into cell therapy and regenerative medicine to take place this autumn. The focus of this inquiry will be whether the UK is sufficiently well placed for the successful translation and commercialisation of research into patient-ready treatments. The inquiry will also ask whether the UK can integrate its efforts into facilitating this process.
The Select Committee analyses Government policy on a wide range of topics relating to science and technology, including a variety of public policy areas. The Committee currently has 14 members from across the political spectrum and is chaired by Lord Krebs, the acclaimed zoologist.
This is not the first time that the House of Lords has examined issues in this sector. In 2002, a Select Committee on Stem Cell Research published a notable report on matters relating to stem cell research and human cloning. The latest inquiry, however, is broader and more focused on translation and more specifically commercialisation than research. It also reaches beyond stem cells to the wider cell therapy and regenerative medicine sectors.
'Regenerative medicine' is the replacement or regeneration of human cells, tissues or organs to restore or establish normal function (2). It includes cell therapies, gene therapies, tissue engineering and biomedical engineering techniques.
A large amount of world-class research and development in regenerative medicine takes place in the UK, including work on cell therapies, on small molecules to trigger in vivo responses to disease, and early developmental-stage research. Internationally, there is a wealth of regenerative medicine and cell therapy developments underway. This fierce pace of development has advanced the field but also revealed new challenges including how best to commercialise the technology. In this context, the focus of the inquiry on regenerative medicine, and how best to facilitate UK translation and commercialisation, is timely.
Significant developments in the UK over the past few years have improved the funding environment for those working in this sector. The recent establishment of the Technology Strategy Board (TSB)-backed Cell Therapy Catapult at Guy's Hospital in London, and the announcement of a £25 million cross-research council UK Regenerative Medicine Platform fund are two prime examples. In addition, the £180 million TSB/Medical Research Council Biomedical Catalyst Fund launched earlier this year and the £650m interdisciplinary medical research initiative (the Francis Crick Institute) are both likely to include regenerative medicine-focused translation projects within their remit.
However, the later stages of the process from lab bench to patient bedside remain particularly important. Gaining regulatory approval, negotiating reimbursement with healthcare providers, improving access to patients, and establishing long-term clinical uptake all pose challenges.
Regenerative medicines face unique issues with regards to many of these steps. For example, clinical trials that require large numbers of identical batches of cells to be used in treating patients present challenges in relation to proven reproducible manufacturing. In addition, the regulatory approval for a clinical trial will often be such that it allows treatment of patients only in the late phase of their condition. This often results in the trial therapy being tested on the target condition but with associated complex multisystem pathology and thus little real opportunity to demonstrate significant patient benefit.
At the same time, therapies need to remain cost-effective to garner recommendation from the National Institute for Health and Clinical Excellence (NICE). Making such highly-personalised medicines, such as autologous cell therapies, available on the NHS, is essential for their reaching large numbers of patients. Then, of course, their price has to be sufficient to ensure commercial viability.
The Select Committee inquiry presents an excellent opportunity for those involved with and interested in the sector to express their views and help shape the future of policy and regulation in this field. The Committee has published a call for evidence inviting written submissions by 20 September 2012. The call covers topics including the research base, the application of science, barriers to translation and commercialisation, and international comparisons.
With the UK being a world leader on cell therapy development, stem cell research and personalised medicine, we now have the opportunity to identify and resolve the bottlenecks in the translation and commercialisation process, and move efficiently towards economic growth from the sector. It is to be hoped that there will be broad and enthusiastic support for this Select Committee as it will be the UK's last chance to become a global leader in the commercialisation of cell therapies.