New perspectives for patients: National Strategy for Gene- and Cell-based Therapies
Around 150 experts from various stakeholder groups—including a number of scientists from the network of the German Center for Infection Research (DZIF)—have developed a roadmap for improving healthcare and strengthening Germany as a hub for gene and cell therapies. The German Federal Ministry of Education and Research (BMBF) had commissioned the Berlin Institute of Health (BIH) at Charité to coordinate and oversee the drafting of the strategy. Some 250 stakeholders from science, industry, politics and society from across Germany attended the official handover of the National Strategy for Gene- and Cell-Based Therapies to Federal Research Minister Bettina Stark-Watzinger on 12 June at the Futurium in Berlin.
Gene- and cell-based therapies (GCTs) are some of the key technologies driving innovation in biomedical research and patient care. They are not only used to modulate disease processes and alleviate symptoms, but also directly address the genetic causes of diseases. This opens promising perspectives for patients suffering from severe and rare diseases for which no treatment currently exists.
In order to improve patient access to gene and cell therapies and to strengthen Germany as an international hub for research and innovation in this field, the German Federal Ministry of Education and Research (BMBF) commissioned the Berlin Institute of Health (BIH) at Charité in fall of 2022 to coordinate and oversee the development of a National Strategy for Gene- and Cell-Based therapies. This strategy was presented to Federal Research Minister Bettina Stark-Watzinger (FDP) at the Futurium in Berlin on 12 June.
Federal Research Minister Bettina Stark-Watzinger: “The National Strategy for Gene- and Cell-Based Therapies is an important step towards securing and expanding Germany's position as a centre of biomedical innovation. Our declared aim is to create new treatment options for patients in the long term. I am very pleased that we have succeeded in bringing together so many stakeholders from different areas and jointly developing the National Strategy. This collaboration between science, industry, the public sector and society is an important key to success. I would like to thank all those involved for their great commitment. With this spirit of optimism, we should now move forward together in a national network."
Interdisciplinary measures in eight action areas
The National Strategy is chiefly characterised by its multi-stakeholder approach, which brought together a wide range of views and interests from science, business, politics, society and patient groups. In total, more than 150 experts—including Prof. Dirk Busch, Chair of the Executive Board of the German Center for Infection Research (DZIF), and other scientists in the DZIF network—contributed to the formulation of concrete goals and measures to be implemented in eight action areas. These action areas were determined in advance in consultation with stakeholders from various relevant fields by holding discussions at public meetings and providing the opportunity to submit written feedback, and were then finalised at a round table event. They address the following topics:
I. Stakeholder networking and support
II. Training and development of skills
III. Technology transfer
IV. Standards, norms and regulatory framework
V. Improvement of quality and capacity for good manufacturing practice (GMP) production
VI. Research and development
VII. Marketing authorisation and translation into patient care
VII. Interaction with society
The strategy emphasises the tremendous potential that the cutting-edge field of gene and cell therapy has for improving patient care and the healthcare industry and for strengthening Germany as a location for pharmaceuticals and innovation. Yet, measures must be taken to accelerate the translation of findings from Germany’s strong basic research programmes into clinical practice, while at the same time making gene and cell therapies not only safe and efficient but also affordable and widely accessible.
“Gene and cell therapies are the future of medicine. They will make it possible to treat major diseases with targeted therapies tailored to the individual patient. At the same time, there is a great need for concerted research and development efforts with regard to efficiency, safety and health economics. The strategy paper primarily aims to benefit future patients across Germany. The past few months have shown what can be achieved when all stakeholders take joint responsibility: the formulation of a strategy with concrete measures that must now be adopted and implemented by policymakers. In my view, this can only succeed if we think regionally and not federally,” says Prof. Christopher Baum, Chair of the BIH Board of Directors and Spokesperson of the National Strategy for Gene and Cell Therapies.
What’s next for the National Strategy for Gene and Cell Therapies?
The proposed measures will now be implemented step by step in collaboration with all stakeholders. A number of other activities have already been initiated alongside the drafting of the strategy. These include the creation of a National Network Office for Gene and Cell Therapies, the establishment of the nationwide entrepreneurship programme GeneNovate, the offering of low-threshold advice on regulatory issues and the laying of the groundwork for funding to support researchers and projects in the field of gene and cell therapy. The Network Office is tasked with building a national GCT community that brings together all stakeholder groups through independent, cross-site information exchange and networking.
“Gene and cell therapies represent a quality of life that was previously not available to many people. Today kicks off the creation of a strong GCT network in Germany and beyond. We are delighted to see the keen interest shown by the various stakeholders so far and look forward to continuing to drive forward networking in line with our mission and to supporting the joint implementation of the measures,” says Dr. Elke Luger, Head of the National Network Office for Gene and Cell Therapies.
A total of €48 million in funding is being provided from 2023 to 2026 to support the design and implementation of the strategy’s measures, with the Federal Government contributing 90 percent of the funds and the State of Berlin contributing 10 percent.
Dr. Ina Czyborra, Senator for Higher Education and Research, Health and Long-Term Care of the State of Berlin, says: “The National Strategy for Gene and Cell Therapies comprehensively integrates all relevant perspectives and highlights the enormous potential that exists for supporting research over the long term and for significantly improving healthcare. From the very beginning the Berlin Senate has been committed to realising this dual approach. We look forward to playing our part in making Germany the leading location for biotechnology.”
The press release of the Berlin Institute of Health and download links to the National Strategy for Gene- and Cell-Based Therapies and the expert committees can be found here.
