The VDGH sees GCT as a "project of the century" for medicine and biomedical research. It is of central importance that Germany takes a leading position here, both in basic research and in application. The establishment of a national strategy is essential in order to achieve this goal.
The VDGH is in favor of an open consultation process for the development of a national strategy. At the same time, the association sees itself as a key stakeholder in gene- and cell-based therapies and is therefore calling for its permanent involvement in the implementation of the strategy process. The sectors represented by
VDGH, i.e. the life science research industry and the diagnostics industry, enable GCT along the entire value chain with their technologies, services and products - from basic research to patient-specific laboratory diagnostics.
Securing Germany as a location for science and business is seen as particularly relevant. Outstanding academic foundations must be maintained and expanded in Germany. At the same time, the translation of academic findings into value creation must not be forgotten, because promoting translation secures technologies, jobs and thus Germany as a business location. Interest in and acceptance of gene- and cell-based therapies in society lay the foundation for trust in novel therapeutic options and must therefore be promoted at an early stage.
On the draft of the BIH et al. of May 15, 2023
The VDGH, together with the Life Science Research (FA LSR) department organized within the association, represents the common interests of more than 120 companies operating in Germany. The LSR companies have particular expertise in the development and production of instruments, reagents, test systems and consumables for research in the life sciences. LSR products are tailored precisely to the needs of scientists in basic and applied research. The diagnostics companies represented in the VDGH produce examination systems and reagents for the diagnosis of human diseases. Such in-vitro diagnostics (IVD) increasingly include biomarker-based and molecular genetic tests. IVDs are indispensable for the development of GCT, but especially for use on patients. Even more than in the conventional understanding of personalized medicine and companion diagnostics, GCT require extensive individualized in-vitro diagnostics. The companies in both sectors are networked worldwide through scientific collaborations.
The LSR and IVD industry covers the entire value chain from basic research in the life sciences to routine diagnostics in the medical laboratory. The products and expertise of the member companies therefore represent all process steps in the research, development, production and application of gene and cell therapies.
About the fields of activity
I. Networking and supporting stakeholders
The VDGH advocates the establishment of a permanent political dialog at the top level between the BMBF, BMG and BMWK. Activities in terms of intensifying research funding, business development (especially start-ups and SMEs), regulatory compliance (e.g. Genetic Engineering Act) and the establishment of suitable reimbursement models for diagnostics and therapy as a SHI benefit are the most important points. They must be coordinated interministerially at an early stage in order to become an effective whole. Reference is made to comparable dialog formats of the past (e.g. National Strategy Process Innovations in Medical Technology).
The VDGH supports the establishment of a network office that links all relevant stakeholders and does not neglect the international perspective. The network office should, for example, collect obstacles to development from other countries and formulate strategies for Germany in an adapted form. The establishment of a digital platform with information on timetables, milestones, GMP specifications, etc. creates transparency for all stakeholders involved. An advisory platform must also be created to support international stakeholders in particular in their interaction with German authorities.
II Training and capacity building
The development of training and further education programs for young talent and skilled workers is the basis for securing Germany as an attractive labour market.
In order to create a better foundation and do justice to the handling of GCT, relevant topics must be included in university and technical education and training curricula at an early stage. In addition, dual study programs promote further links between industry and academia. Hybrid training and work models also make it easier to recruit and retain top international talent.
The companies associated with the VDGH have a great deal of technological expertise to actively participate in the training and further education of academic staff. Post-doctoral positions, 50 percent of which are in industry and 50 percent in academia, are solution-oriented.
III Technology transfer
The association considers the establishment of a technology transfer program to be a priority in order to promote value creation following academic research and development. Only by translating academic success into economic success can patients benefit from innovative and safe therapies in the long term.
To this end, further funding programs should be created by the ministries and public-private partnerships in particular should be used to combine public and private funding. The German start-up sector currently still suffers from many bureaucratic hurdles and is characterized by financing difficulties, as a healthy German capital ecosystem is lacking.
IV Standards and regulation
The VDGH considers the development of a regulatory framework to be very important in order to establish guidelines for the development, approval and monitoring of cell and gene therapies. In Germany, there is a lack of nationwide harmonization of requirements in the areas of data protection and ethics, which significantly complicates the study conditions. The optimization of clinical trials, e.g. conducting them at several locations, ultimately leads to faster approvals.
The lack of uniform federal regulations creates legal uncertainty if, for example, the competent state authorities interpret the term "manufacturing" differently (e.g. the collection of suitable cells for CAR-T cell therapy is a separate process that is independent of the manufacturing process for the advanced therapy medicinal product). Approval procedures must also be implemented more quickly in future to ensure that Germany is internationally competitive as a GCT location.
The initiatives on research and health databases must be further developed and genomic, phenotypic and therapy progression data on GCT must be connected. The establishment of a European research database promotes the exchange of international data, which is particularly important for clinical studies. Access to data must be granted equally to public and private research.
In order to set the course for GMP-compliant production at an early stage, certain quality assurance measures for working materials such as cell culture products must also be introduced at the academy. The scalability of methods and technologies must also be taken into account in basic research.
In general, care must be taken to bring the national strategy for Germany in line with European regulations and the European market, or even to harmonize it internationally. For this reason, networking with other stakeholders such as the EMA, MedTech Europe, IHI Call ERD2 consortium and the FDA makes sense (see field of action I.).
V. Expansion of quality and capacities in the area of GMP production
The definition of GMP criteria must be an elementary component of the national strategy. This includes, for example, the GMP-compliant scalability of processes or quality assurance with new methods such as next generation sequencing. In addition, GMP-compliant production differs between local and central GCT production centers. The formulation of standards must take this into account.
Necessary capacities should be precisely planned and adapted at an early stage in order to secure and further develop Germany as a location for GCT. Currently, the production capacities for GCT in Europe are clearly too low to be internationally competitive.
Ensuring quality and quantity in the supply chain can help to avoid bottlenecks. Attention should also be paid to the scalability of supply chains at an early stage in order to secure raw material and production capacities.
VI Research and development
Funding formats for initial academic applications and proof-of-concept are important, but suitable funding measures for translation into value creation must not be forgotten, such as funding measures to improve the scalability and automation of industrial processes.
In order to reduce manufacturing costs and make GCT available to many patients, automation and digitalization technologies for GCT production must be expanded and promoted. Automation in the area of cell collection from donors, modular production technologies and the final formulation and filling of the product require new developments and therefore special research funding. The need for development lies not only in the area of hardware but also in the area of software solutions. For the actual production of GCT and the associated data, technologies such as integrated process controls, process monitoring using "digital twins" or analysis and process control using AI must be taken into account in the future in order to exploit the full automation potential.
The VDGH suggests formulating section VI. e) as a separate field of action or, alternatively, integrating it into section VII.
The manufacturers of in-vitro diagnostics represented in the VDGH have extensive expertise in biomarker-based and molecular genetic tests. Support for the development/improvement of structures for companion diagnostics - better therapy-accompanying diagnostics - the monitoring and aftercare of patients is expressly welcomed. In the opinion of the VDGH, companion diagnostics to identify suitable recipients of GCT go beyond the concept of classic companion diagnostics (CDx). Classic CDx, as used in oncology, for example, identify patient groups and stratify treatment processes, whereas diagnostics for GCT should be individualized and applicable to a single person. Current regulations for CDx are anchored in the European IVD Regulation (IVDR 2017/746) and are extremely complex and time-consuming to approve. The traditional reimbursement models for companion diagnostics are likely insufficient for individualized diagnostics in GCT; a more detailed analysis and consideration of alternative models is required.
VII Transition to care
GCT are generally approved for small patient populations and cost coverage for treatment centers is currently inadequate. Reimbursement models for GCT must take into account the special nature of novel therapies and thus be completely rethought and created, as current procedures of medical self-administration are not tailored to GCT. In addition to therapy, new steps in diagnostics must also be taken into account: In addition to initial diagnostics, quality control of the genetic modification after application and longer-term therapy monitoring are essential for successful therapy. Development and production processes that are implemented using life science research technologies must also be taken into account in remuneration models in line with performance. Then the costs for GCT can fall in the long term.
VIII. Interaction with society
This field of action must be prioritized. Interest and acceptance for GCT must be created in society at an early stage in order to lay the foundations for trust in novel treatment options for future generations. In the VDGH's opinion, the starting point could be a broad-based information and education campaign by the German government. For example, clear and low-threshold communication of success stories could increase public acceptance of GCT.
On the open questions
In your opinion, are there any other fundamental fields of action that should be taken into account in the development of the national strategy? If so, which ones?
Diagnostics as a separate field of action (see section VI.)
The envisaged Health Data Utilization Act and the Research Data Act are important legislative procedures that are relevant to the national GCT strategy. It is desirable to define the design of these laws and their significance for JCT as a separate field of action.
Should further priorities beyond the aspects outlined so far be integrated into the listed fields of action? - If so, which ones?
In the future, therapy options should also be implemented in the outpatient sector. For example, satellite centers specializing in GCT could be set up for this purpose. This will increase the number of patients treated and reduce the burden on hospitals at the same time.
Germany needs extensive lighthouse projects, such as the Berlin Center for Translation, because technology locations in Germany must exceed a critical size in order to remain competitive. Corresponding mergers and clusters must be increasingly promoted.
With the increasing establishment of GCT, issues relating to the scalability of production and the automation of laboratory and production processes are becoming more important.
Are there aspects within the fields of action where you see different priorities in terms of design and implementation?
In your opinion, what potential challenges in the timeline, development and implementation of the national strategy should be taken into account in addition to the points addressed in the attached documents?
Prompt establishment of a permanent strategy dialog between the BMBF, BMWK and BMG (see section I.)
In your view, are there addressees/actors beyond the stakeholders involved so far who should be included in the further discussion? - If so, which ones?
The VDGH is not yet explicitly included in the stakeholder list (appendix to the BIH paper) and would like to be included.
Around 30 life science research companies are organized as specialist departments in the VDGH. As research suppliers, they support training, research and development, translational work, scaling, trials, logistics and manufacturing as well as accompanying diagnostics. The industry is also able to support the GCT community in Germany and Europe as consultants with global experience in this field.
The VDGH member companies (around 120 companies in total) with a focus on IVD production are leaders in the manufacture of biomarker-based and molecular genetic tests and thus support the care of patients through accompanying diagnostics, prognostics, therapy monitoring and aftercare.
