well foresight studies opportunities for regenerative medicine in the netherlands royal netherlands academy of arts and sciences

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1 well underway opportunities for regenerative medicine in the netherlands royal netherlands academy of arts and sciences foresight studies

2 5. conclusions and recommendations Scientific research in regenerative medicine has made major progress since the 1990s. New scientific insights such as the reprogramming of cells and the development of degradable materials have led to new potential applications. Up to now, however, clinical studies have only been carried out on a restricted scale, for example involving applications aimed at promoting the regeneration of skin, bone and cartilage. Science is starting to tackle the challenge of regenerating complex three-dimensional cellular structures. Nevertheless, as a field of research, regenerative medicine is well under way. 5.1 Conclusions Progress in regenerative medicine will require additional basic research on stem cells, biomaterials and their interaction. Specifically, this will involve; isolating, differentiating and replicating stem cells; understanding the cellular response in the tissue environment; studying the interaction between biomaterials and the tissue environment. Our advancing knowledge of the natural extracellular matrix serves as a source of inspiration for all this research. Crucial to the development of this field is that the risks should be properly explored, for example the unwanted differentiation of cells, immunogenicity, and the toxicity of cells and materials. 56 conclusions and recommendations

3 The Netherlands has a strong position in the scientific field of regenerative medicine. The country is number ten in the world as regards the volume of publications in this field (from 2000 through 2008), and within Europe, it ranks fifth (after Germany, the United Kingdom, Italy, and France). With an annual average of three percent of the world total of publications, the contribution of the Netherlands in this field is greater than the two and a half percent average contribution made in all Dutch research fields. In recent years, the Dutch government has invested more than 85 million euros in research in regenerative medicine. That money has come entirely from the proceeds from natural gas, and has been matched by private and public partners. A number of programmes were set up on the basis of these subsidies. Typically, they run for a short period (five or six years) and have an emphasis on valorisation. Due to this focus, less attention has been paid to knowledge gaps in fundamental regenerative medicine research. The rate at which science develops is partly dependent on the development of enabling technologies. The Foresight Committee has identified six technologies which it considers to be important in progressing more effectively from basic science to translational research and clinical application: biomarkers; imaging techniques; high-throughput technologies; in vitro and in vivo modelling systems; bioreactors; tools for minimally invasive administration. These technologies are of great importance for investigating the effectiveness and safety of new products in the field of regenerative medicine. In general, developing new enabling technologies or optimising existing systems involves a great deal of time and money. The current financing systems offer insufficient scope for covering these costs. The level of collaboration between the Dutch knowledge institutions in the field of regenerative medicine has increased considerably in recent years. New players have also become active in the field, in particular the universities of technology. These developments are probably related to the start of the first subsidy programme specifically for regenerative medicine, namely the Dutch Program for Tissue Engineering (2004). This has been followed by a number of other incentive schemes within and between universities. For the development of the discipline, it is essential that collaboration involves a continuum that is independent of the source of financing. Regenerative medicine is not yet very visible in university education. Although we are still at an early stage in the development in regenerative medicine, today s students of medicine and pharmacy are the professionals of the future who will need to deal with such developments. For the development of the discipline, it is therefore important that students taking bachelor s and master s degrees have the opportunity 58 conclusions and recommendations

4 to familiarise themselves with regenerative medicine, for example by means of internships or optional courses. In order to guarantee the multidisciplinary nature of the discipline, existing collaboration between medical/biomedical faculties and technical faculties needs to be expanded structurally where teaching is concerned. Formal administrative agreements should form the basis for this. At the moment, there are no research schools or multidisciplinary training programmes at PhD level in the Netherlands that specialise in regenerative medicine. Regenerative medicine is, however, partly covered in existing Dutch research schools. It is essential for PhD students in medical and technical disciplines to learn to speak one another s language. Preparation of regenerative medicine products including in the context of clinical research is increasingly taking place under GMP conditions, given that more and more stringent quality conditions are being set, with specific expertise also being required. Five of the Dutch universities have a GMP facility (or will soon have one). As yet, there is hardly any exchange of expertise between the users and managers of the various facilities. Quality, safety, and cost control make collaboration necessary. This should focus, amongst other things, on pooling legal knowledge and on creating possibilities for preparation at one another s facilities. In addition, pharmacists play an increasing important role; their complementarity of expertise enables them to work with both research departments and clinical departments. They will, however, require support as regards training and continuing education. In a new field of science where the applicable legislation has not yet been fully developed, it is valuable to clarify the legal aspects. In the field of regenerative medicine, researchers and the other parties involved find themselves confronted by a large number of complex rules. In particular, this involves the lack of any cohesive legal framework regarding control of bodily materials. Nor have the authorities adopted a position regarding the tension that exists between the altruism of the donor of bodily material on the one hand and the commercial generation of products on the basis of that material on the other. An European regulation concerning advanced therapy medicinal products has made the rules for researchers who wish to start a clinical study significantly more onerous and complicated. Various researchers now find themselves asking the same legal questions and are confronted by the same problems. It is extremely important that they can familiarise themselves with all of the legal requirements and possible exceptions associated with the clinical use of a certain product. The authorities must also act effectively and consistently in this regard. It is notable that the Dutch authorities have set up an inspectorate for clinical research but that in this country there is no option for consultation or advice, although there is in fact a demand for it. There must be an ethical discussion concerning the potential clinical implications in regenerative medicine, one that involves all stakeholders, for example the authorities, researchers, patient organisations, and doctors associations. It is only by means of an interactive process that a common basis of support can be created. The ethical discussion can also help prevent excessive and unrealistic expectations being raised 60 conclusions and recommendations

5 regarding therapies that may become possible now and in the future by means of regenerative medicine, expectations that may prove in the long run to be detrimental to the development and implementation of this branch of medicine. 5.2 Recommendations Based on its findings, the Foresight Committee has issued the following recommendations. science and finance Regenerative medicine should be financed in a more long-term manner, with attention being paid to closing knowledge gaps in fundamental research, thus allowing the Netherlands to maintain and intensify their leading position in this field. In addition, to concentrate the field the universities should organize special working groups (parties: universities, Ministries of Economic Affairs (EZ), Health, Welfare and Sport (VWS), Education, Culture and Science (OCW); Netherlands Organisation for Scientific Research (NWO), and Netherlands Organisation for Health Research and Development (ZonMW)). Large-scale investment in research in regenerative medicine should apply transparent, verifiable assessment procedures based on peer review with the decisive factor from the start, and these should be based on scientific excellence (parties: universities, Ministries of Economic Affairs (EZ), Health, Welfare and Sport (VWS), and Education, Culture and Science (OCW)). Measures should be put in place to promote the development of enabling technologies so as to more effectively facilitate translational research and clinical application in regenerative medicine (parties: universities, health funds, Netherlands Organisation for Scientific Research (NWO), Netherlands Organisation for Health Research and Development (ZonMW), Ministries of Economic Affairs (EZ), Health, Welfare and Sport (VWS), and Education, Culture and Science (OCW)). collaboration Collaboration should be promoted between universities as regards the preparation of cellular products under GMP conditions from the point of view of cost, quality, and safety. The establishment of a national academic GMP platform could support this (parties: pharmacists, researchers, Dutch Federation of University Medical Centres (NFU)). 62 conclusions and recommendations

6 education Structural collaborative partnerships should be set up between medical/biomedical faculties and technical faculties for bachelor s and master s training (parties: teaching staff and medical/biomedical, pharmaceutical and technical faculties). Multidisciplinary educational programmes should be set up for PhD students by technical and medical faculties. Use should preferably be made of existing modules. The possibility of a national research school should be explored (parties: researchers, graduate schools). legal and ethical aspects The legal complexity regarding the control of bodily materials requires clarification by the authorities. As the use of bodily material affects the constitutional rights of individuals, self-regulation will not suffice; instead, legislation is required (parties: Ministry of Health, Welfare and Sport (VWS)). The government should take a position on the conflict between the altruism of the donor of bodily material on the one hand and the commercial generation of products on the basis of that material on the other hand (parties: Ministry of Health, Welfare and Sport (VWS)). Researchers should have the option of obtaining advice at a national level on the design of clinical trials using innovative medicinal products. This advice should be independent of central inspectorate activities (parties: researchers, national authorities and other bodies involved in clinical research). The complexity of the Dutch legal framework of regenerative medicine makes a thematic legal evaluation necessary. This evaluation should take place well in advance of the evaluation of the European Regulation on Advanced Therapy Medicinal Products (parties: Ministry of Health, Welfare and Sport (VWS)). Public support should be generated by engaging in discussions of the ethical implications of research in regenerative medicine. In order to encourage this, it is important for financial scope to be explicitly created within research programmes (parties: researchers, clinicians, patients, and ethicists). 64 conclusions and recommendations