These therapies have become the focus of major public research programmes in the US and Europe, and have stimulated the growth of a new sector of the biotechnology industry. However, research carried out at the University of Nottingham's Institute for Science and Society suggests that it might take much longer than anticipated for this breakthrough science to be translated into improvements in patient care. This raises major questions about how best to manage the high levels of hype and hope that surround this emerging field.
The research, funded by the EPSRC (Engineering and Physical Sciences Research Council) as part of the wider remedi Grand Challenge (4), which is charged with exploring the technical, economic, regulatory and social issues of creating a viable regenerative medicine industry, found two overarching problems. First is the difficulty that small biotechnology firms are having in making a business out of stem cells. This is important, as it is the private sector that has historically been the developer of most medical products. Secondly, even when commercial products become available there are a number of barriers to the adoption of these innovations into routine healthcare
1) The risk of market failure
Based on a global survey, our research found that the cell therapy industry had grown rapidly in recent years and had been successful in getting new products on the market, but was faced by poor product sales and a lack of investment from the pharmaceutical industry. As a consequence, there is a significant risk of market failure for a number of types of cell therapy. Unless this situation changes, the industry is likely to rapidly contract and the progress needed to take new therapies into late stage development will be adversely affected. The main difficulties facing the industry are largely structural rather than technical. These include establishing closer collaboration with clinical end-users, the funding of clinical studies, greater regulatory certainty and clearer reimbursement policies. In addition, there is the need to develop enabling technology platforms that could lower manufacturing costs.
2) Barriers to adoption
While the underlying science of regenerative medicine has grown exponentially over recent years, this new knowledge has not been translated into clinical practice. In particular, a dearth of evidence on (cost) effectiveness is leading to problems with utilisation and reimbursement. This is compounded in the UK by an NHS culture that is considered to be unsupportive in utilising innovative technologies. These all result in an unattractive environment for the commercialisation of regenerative medicine products. However, perhaps the key reason why regenerative medicine has not been taken up is because clinical utility and clinical need have not been designed into first generation cell therapy products to a sufficient extent. This has resulted in invention without adoption.
The main problem facing the future development of stem cell therapy is that major institutional and structural barriers will need to be overcome in order to facilitate its adoption, and this will take considerable time and effort. Technical change in medicine is slow and incremental. In our reports, we argue that there is a need for a comprehensive set of polices and we outline a number of key recommendations (5). If policy can systematically overcome the barriers we have identified by taking a more integrated and holistic view of translation that involves both innovation and adoption, then the promise of stem cell therapy can be fully realised in the longer term.
As a consequence of this analysis, it is clear that talk of stem cells revolutionising medicine is misplaced. Successful translation will require sustained public and private investment over many years and concerted policy initiatives leading to major changes in a number of institutions, as well as the time-consuming process of clinical development and adoption. This will not happen quickly and it is vital that a much more realistic set of expectations based on a sober assessment of the evidence is created by scientists, clinicians, policy makers and the media. This will enable a more carefully balanced appreciation of the importance of this field, while not over hyping the promise of the technology and its progress, or by making claims that cannot be fulfilled.