1.  Background

Gene and cell therapies are on the rise. On June 12, 2024, the German Federal Government was handed the strategy paper for a National Strategy for Gene and Cell Therapies. The paper is intended to serve as a basis for policymaking to give Germany a leading role in the field of gene and cell therapies (GCT) in Europe. The German Government recognizes that the age of GCT has started but that there are many legal, regulatory and practical shortcomings that impedes research and development of GCTs in Germany.

Back in the fall of 2022, the German Federal Ministry of Education and Research (BMBF) had commissioned the Berlin Institute of Health (BIH) to coordinate and moderate the development of a National Strategy for GCT. Eight working groups were created to develop the National GCT Strategy, with a total of about 150 experts from various stakeholder groups. The result of their work is a document divided into eight fields of action, in which various measures are proposed to achieve strategic goals in the field of GCT.

The National GCT Strategy is one of several highly targeted measures with which the German Government aims to make Germany more attractive as a location for pharmaceutical and healthcare innovation. Just six months ago, in December 2023, the Federal Ministry of Health (BMG) presented a strategy paper for the new National Pharma Strategy. We reported on this in detail in an earlier Covington blog.

Unlike the National Pharma Strategy, which was developed under the Social Democrat-led Federal Ministry of Health (BMG), the National Strategy for GCT is an initiative led by the Federal Ministry of Education and Research which is led by the liberal party FDP. The BMBF appears keen to play a leading role in the establishment of GCT in Germany. Industry stakeholders may welcome this as the BMBF is known to be a more industry-friendly part of the German Government than the BMG.

2.  The National Pharma Strategy as a possible role model

The example of the National Pharma Strategy and its rapid implementation already indicates what the next steps in the National GCT Strategy may be. Shortly after the National Pharma Strategy was agreed upon, the first draft of the “Medical Research Act” was presented on 26 January 2024 to implement key elements of the Pharma Strategy, including amendments in the areas of clinical trials, ATMPs and pharmaceutical pricing and reimbursement (AMNOG). We reported on this in two earlier blogs that discussed the proposed changes for clinical trials and drug pricing. The draft Medical Research Act is expected to come into force in the fall of 2024. Hence, the current German Government is keen to act fast to strengthen Germany as a place for pharmaceutical innovation and R&D.

As the German Government recognizes gene and cell therapies are a key strategic area, a similarly fast and determined implementation in the form of new policies and legislation could also be expected for the National GCT Strategy.

3.  Key Elements of the National Strategy for Gene and Cell-based Therapies

The National GCT Strategy as reflected in the new strategy paper is divided into eight “fields of action” in which the participating stakeholders see a need for action in order to put Germany in a leading position. Within each field of action, the paper defines strategic goals and proposes specific measures. It also indicates the time frame in which the implementation of the measure is expected, and which circumstances are considered to be indicators of success. In the following, we provide an overview of the eight fields of action, the respective strategic objectives and some proposed implementation measures:

Field of Action 1: Connecting and supporting stakeholders

Strategic objectives:

  • Coordinated implementation of the measures of the National GCT Strategy.
  • Strengthening political responsibility for the national future topic of gene and cell-based therapies.
  • Strengthening national GCT networks.
  • Establishing and expanding national and international networking activities in the field of GCT.

Selected proposed measures:

  • Establishing a governance structure for implementing the National GCT Strategy with the participation of the relevant stakeholders.
  • The creation of a “GCT network map” intended to provide a structured overview of the relevant GCT stakeholders, including those from research, healthcare, industry and government.
  • Exchange of information within the framework of networks with international and European clinical research groups, international regulators, as well as investors and donors.

Field of Action 2: Training and strengthening of skills in the area of GCT

Strategic objectives:

  • Development of training and professional development programs for junior and senior professionals, as well as improvement of the infrastructure necessary for training and professional development.
  • Development of appropriate career, bonus and interaction concepts.

Field of Action 3: Technology Transfer

Strategic objectives:

  • Improving the conditions for the early recognition and realization of the innovative potential of scientific findings.
  • Holistic advice and evaluation of transfer projects that cover the entire development process of an innovative investigational medicinal product (IMP), from production to application in patients.
  • Facilitating the utilization of the social and/or economic potential of scientific findings.
  • Establishing the recognition of technology transfer achievements as part of the scientific reputation of individual scientists and institutions.

Selected proposed measures:

  • Establishing a product development unit focused on GCT that can train, advise and support technology transfer institutions and key players in translational projects.
  • Enabling start-ups by providing clear, standardized terms and conditions for participation and licensing, as well as access to the necessary GCT infrastructure.
  • Successful technology transfer and support for spin-offs should be anchored in the state university laws as a goal of medical universities. Successful technology transfer achievements are also to be included in the academic institutions’ ratings.

Field of Action 4: Standards, Regulations and Regulatory Framework

Strategic objectives:

  • Defragmentation and standardization of competencies and processes in clinical R&D of GCT, as well as strengthening of the higher federal authority and its resources as a single point of contact.
  • Continuous adaptation of regulatory processes to developments in the GCT sector.
  • Increasing the availability of low-threshold regulatory advice.

Selected proposed measures:

  • Concentration of GCT-related regulatory procedures and responsibilities, including the manufacturing authorization, at the Paul Ehrlich Institute (PEI). According to this proposal, the approval of applications for clinical trials and the evaluation of medical devices with GCT-related applications, as well as expertise for products from bioreactor processes (GCT, antibodies, mRNA, etc.), are to be coordinated by a central point.
  • Strengthening the PEI with more resources to avoid a loss of expertise.
  • The introduction of sandbox procedures as innovation space. For new developments, such as gene and cell therapies, a procedure should be created that enables quick and flexible assessments.  

Field of Action 5: Expansion of quality and capacities in the area of GMP production

Strategic objectives:

  • Need-based development and expansion of qualified GMP infrastructures (manufacturing and quality control capacities), in particular for starting materials and for complex GCT products.
  • Ensuring the necessary personnel capacities and expertise for GCT production and quality control.
  • Increasing efficiency and accelerating processes in manufacturing.
  • Further development and risk-based streamlining of the framework conditions.

Selected proposed measures:

  • Creation of a central GCT-GMP and regulatory committee, consisting of all relevant stakeholders (including, for example, the BMG, PEI, industry), which continuously analyzes progress towards the strategic goals and drives it forward.
  • The targeted provision of sufficient financial resources from the German Government, the German States (Länder) and other capital providers for the establishment, expansion and operation of GMP infrastructures.
  • Creation of a central national production facility for the manufacture of critical starting materials for GCT for delivery to all commissioning GMP facilities.

Field of Action 6: Research and Development

Strategic objectives:

  • Improving the structural conditions for technology transfer research and development.
  • Identification and promotion of future GCT topics.
  • Improving the organizational and regulatory framework for preclinical and clinical GCT studies.
  • Ensuring the involvement of patients, patient advocates and patient organizations.
  • Changing attitudes and strengthening a bio-entrepreneurial spirit in the German GCT community.

Selected proposed measures:

  • The establishment of a national GCT network.
  • The establishment of new flexible funding formats with short lead times to cover currently not adequately addressed needs.  
  • The definition of standards for patient involvement in project budgets and the remuneration of patient representatives for their involvement in projects and selection procedures.

Field of Action 7: Market authorization and transition to healthcare provision

Strategic objectives:

  • Easier access to and targeted selection of patients for a GCT.
  • Flexibilization of reimbursement and care models in the application of GCT.
  • High-quality treatment of patients with innovative therapies by establishing interdisciplinary GCT treatment centers.
  • Optimization and establishment of the data landscape for the purpose of versatile usability in research and tracking of long-term data of GCT.

Selected proposed measures:

  • Ensuring quality assurance, including the establishment of therapy decision boards for the diagnosis and treatment of patients, by adapting reimbursement models.
  • Maintaining the necessary flexibility in the benefit assessment and pricing in the German AMNOG process in order to maintain access to and availability of GCT for patients.
  • Standardization of the collection and documentation of clinical data by linking existing registries and establishing a method-specific national GCT registry.

Field of Action 8: Interaction with Society

Strategic objectives:

  • Informing society about GCT by providing reliable and target group-specific information.
  • Support decision-makers by strengthening interactions with politics and open-minded social discourse.
  • Targeted promotion of the benefits of GCT through increased involvement and participation of research funding organizations, foundations and civil society.

Selected proposed measures:

  • Establishing a central, web-based point of contact for providing quality-checked information.
  • Compiling or creating target-group-specific information for various stakeholder groups.

4.  What will be the Next Steps?

The fast speed of the recent legislative proposals in the field of German healthcare and pharma laws has shown that the practical implementation of elements of the National Strategy for Gene and Cell Therapies could come similarly fast. This would also be in line with the approach by the German Government reflected in the new National Pharma Strategy.

However, there will still be some steps to take from the just presented strategy paper for a National Strategy for Gene and Cell Therapies up to the point when this will be implemented. It is likely that the implementation of the National GCT Strategy will be accompanied by significant legislative and policy changes. It will also be important to see how this new legislation will fit in with the overarching National Pharma Strategy and other currently ongoing legislation activities such as the Medical Research Act.

As the areas of gene and cell therapies and ATMPs are very specific and complex, it is likely that the German Government and other policymakers will be open to consider the input of the relevant industry stakeholders to convert the National GCT Strategy into new laws and policies. Insofar, the life sciences industry companies and stakeholders now have a window of opportunity to weigh in and get involved in the next steps of the policymaking and implementation of the new German Strategy for Gene and Cell Therapies.

The Life Sciences Team of Covington & Burling LLP in Frankfurt (Germany) will continue monitoring the developments in this area and is well positioned to assist clients in navigating through the various ongoing and upcoming legislative projects.

***

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Photo of Dr. Dr. Adem Koyuncu Dr. Dr. Adem Koyuncu

Adem Koyuncu is double qualified as a lawyer and medical doctor and a partner in our Brussels and Frankfurt office. He is a chair of the firm’s “Food, Drug & Device” practice group and also a member of our Compliance practice. Adem is…

Adem Koyuncu is double qualified as a lawyer and medical doctor and a partner in our Brussels and Frankfurt office. He is a chair of the firm’s “Food, Drug & Device” practice group and also a member of our Compliance practice. Adem is recognized as a “leading lawyer for pharma and medical devices law” (JUVE 2021).

Adem helps clients on a wide range of EU and German law issues, including regulatory, compliance, privacy and liability matters. He also provides strategic advice. He knows the life sciences sector also from his earlier work in the pharmaceutical industry and as a medical doctor. He represents clients before courts and authorities and assists them in contract negotiations, investigations and transactions. For years, Adem is listed in various lawyer rankings.

See some Accolades from Clients and Surveys:

  • “He is one of the most detail-oriented and client-focused partners I have ever encountered.” (Client, Chambers 2021)
  • „Great professional and human competence, good team player.“ (Client/Adverse Party, JUVE 2022)
  • “I find him to be one of the most pragmatic regulatory lawyers. He was a doctor before a lawyer, has been in-house, worked on lots of stuff that I have to handle in-house, which helps when getting advice. He is really good at saying it’s a complex situation and your best option is to do this.” (Chambers 2022)
  • “He always comes through with extremely helpful advice. He brings a unique understanding and experience to his practice as both a lawyer and medical doctor.” (Chambers 2021)
  • “Adem Koyuncu is not only a lawyer but also a doctor and has worked in industry. He knows the perspectives that also move in-house lawyers.” (Legal 500 2022)
  • “He is very sharp and quick, while at the same time having a good sense of humor and nerves of steel. Very pleasant to work with.” (Legal 500 2022)
  • He is described as “versatile competent, reliable and high quality” (JUVE 2021) and “incredibly fast.” (JUVE 2018)
  • Provides advice at “an outstanding level.” (Legal 500 2015)
  • “Very strong negotiation skills.” (JUVE 2011)
  • Clients appreciate his “very broad knowledge and long-standing expertise” (JUVE 2021/22) and that “he is approachable, knowledgeable and really easy to talk to over the various issues. He is calm and has seen most problems before.” (Chambers 2020)
  • Peer lawyers described him as “highly competent” and a “very good and pleasant lawyer” (JUVE 2014) and as “the off-label-guru, substantively very good, creative.“ (JUVE 2022)

Adem is the author of numerous publications (e.g., in leading books on pharma law, product liability and clinical trials) and frequent speaker at different events. As such, he will soon speak at following events:

  • “Medical Device Liability and Risk Transfer 2.0 | A liability issue for the Management?!,” BVMed, Webinar (3/24/2023)
  • “Data Protection Requirements and Open Issues in the Pharma Sector – Status quo,” Speech at conference, 26. Marburger Gespräche zum Pharmarecht, Marburg (3/16/2023)
  • “Vendor Oversight & Vendor (Quality) Management in the Pharma Industry,” FORUM-Institut, Online Seminar (4/19/2023)
Photo of Maximilian Aretz Maximilian Aretz

Maximilian Aretz is an associate in Covington’s Frankfurt office and a member of our Food, Drug and Device Practice. He advises clients on regulatory and compliance matters.

His advisory work covers all aspects of pharmaceutical and medical device regulation, clinical trials, advertising and…

Maximilian Aretz is an associate in Covington’s Frankfurt office and a member of our Food, Drug and Device Practice. He advises clients on regulatory and compliance matters.

His advisory work covers all aspects of pharmaceutical and medical device regulation, clinical trials, advertising and other regulatory aspects over the entire product lifecycle. In addition, he advises pharmaceutical companies on EU market access matters including the German AMNOG procedure. Furthermore, Maximilian provides legal advice on Freedom of Information Act (FOIA) cases, data protection laws and contractual matters. He represents clients before authorities and in court.

Maximilian received his law degree from the University of Marburg with a focus on medical and pharmaceutical law. He also obtained an LL.M. degree in Dispute Resolution from the University of Cape Town, South Africa.

He completed his legal clerkship at the Berlin Court of Appeals. During his clerkship, he has worked at the Berlin Public Prosecutor’s Office and at the German federal health agency Robert Koch Institute.