Autologous and allogeneic stem cell transplantation (SCT) is the treatment of choice for many haematological diseases. Within healthcare provision, SCT is a high risk, high cost procedure that has been at the forefront of translational medicine for the past 35 years.
It is a field of medicine in which its practitioners have an outstanding track record in the critical evaluation and validation of their activities by a real commitment to the collection and scientific analysis of outcome data, as is exemplified by the transmission of outcome data from individual centres to a central database held by the EBMT for further analysis and reporting.
With the advent however of new, effective and very expensive pharmaceutical and biotechnical products for the management of haematological malignancies, future progress in SCT must evaluate best practice in the context of prospective randomised studies. These innovations have the potential to improve patient outcome but also to increase the cost considerably. SCT physicians have always been enthusiastic exponents of clinical trials and are ready to test new hypotheses and to compare SCT with other treatments
Although many individual SCT units and National Transplant Societies have conducted clinical trials in the past, this has become increasingly difficult in the academic setting with the recent introduction of legislation satisfying the EU Directive on Clinical Trials. Although introduced with laudable intentions the effect of the Directive has been to increase the resources and therefore the expense of clinical trials whilst at the same time national differences in interpretation of the new legislation has rendered international studies extremely difficult.
It is intended through this project to support the development of the infrastructure to perform academic-led international prospective studies in SCT and to facilitate the conduct of such studies across Europe by providing a trans-European prospective clinical trials network under the auspices of the EBMT. This will hasten the evaluation of new treatment strategies and improve the outcome for European citizens.
In this endeavour the EBMT represents 530 transplant centres (mostly situated in large teaching and university hospitals), all the existing National Transplant Societies in Europe and has an active collaboration with the Unrelated Donor Registries, all of whom recognise the need to work together to overcome the identified obstacles to ongoing research in STC (see Table 1). The EBMT is able to benefit from the experience of a similar initiative in the USA, through a partnership with the Centre for International Blood and Marrow Transplant Research (CIBMTR). The project is also well supported by the Centre for Professional Ethics, University of Central Lancashire (UCLAN) and by the Project Management team at Imperial Collage.
Table 1: Recognised obstacles in trans-national collaboration in SCT
- Difficulties in the implementation of the EU Directive on Clinical Trials for both academic and multinational studies
- Differences in national resources available for the infrastructure for SCT and clinical trial participation
- Difficulties in exchange of patient derived information and material across national boundaries
- National differences in approaches to the ethics of clinical trial participation and of collection and use of tissue for research
- National differences in establishing informed consent
- Lack of systems to exchange information
- Fragmentation of databases and the information stored therein
- Ill-defined statistical methodologies across Europe
- Lack of dissemination of information to all interested parties