FDA Issues Comprehensive Guidance for Industry and Academic Sponsors Developing Ex Vivo-Manufactured CAR T Cell Products in Oncology: Expert Recommendations for CMC, Pharmacology, and Clinical Trial Design

FDA Issues Comprehensive Guidance for Industry and Academic Sponsors Developing Ex Vivo-Manufactured CAR T Cell Products in Oncology: Expert Recommendations for CMC, Pharmacology, and Clinical Trial Design

The guide includes recommendations tailored to both autologous and allogeneic CAR T-cell products. In addition, it provides guidance on comparative analytical studies for CAR T-cell products. Although the focus is on CAR T-cell products, certain information and recommendations may also be relevant for other genetically modified lymphocyte products, such as CAR natural killer (NK) cells or T-cell receptor (TCR)-modified T-cells. It is important to note that for these related product types, further considerations beyond those suggested in this guidance may be required depending on the product and manufacturing process.


Germany Takes the Lead in Cancer Innovation Patents Across Europe !

Germany Takes the Lead in Cancer Innovation Patents Across Europe !

This is the conclusion of a report published by the European Patent Office on Thursday.

The future of cancer-related innovation looks promising, with the United States leading the world. Recent data from the Patent Office report shows that American companies led around 46 percent of patent applications for cancer-related inventions between 2002 and 2021. In Europe, Germany leads the field, followed by the United Kingdom, and topping both France and Switzerland in the area of cancer-related advances. This underlines the prospects for further developments and breakthroughs in the fight against cancer.


Fluoropolymers at a Crossroads: A Possible Ban that Shocks the Industry

Fluoropolymers at a Crossroads: A Possible Ban that Shocks the Industry

A proposal has emerged suggesting the potential ban of fluoropolymers, raising concerns and discussions within the industry. While it is currently just a proposal, if the European Chemicals Agency (ECHA) concludes at the end of its decision-making process that fluoropolymers lack economic necessity, a ban on these materials could become a possibility. There will be more clarity in the next review conducted by REACH (Registration, Evaluation, Authorization, and Restriction of Chemicals Regulation) and the European Chemicals Agency (ECHA), which is scheduled for September 2023.

The proposal to ban fluoropolymers revolves around the assessment of their economic necessity. Fluoropolymers are widely used in various industries due to their unique properties, including heat resistance, chemical resistance, and low friction. The proposal raises questions about whether alternative materials or technologies could adequately replace fluoropolymers without compromising functionality and performance.

A ban on fluoropolymers seems inconceivable due to their irreplaceable properties and the extensive use of these materials in critical applications. Fluoropolymers play a vital role in several sectors and especially of course in healthcare for various medical devices and medicines, where their exceptional properties are critical to product performance, safety and reliability.

While the proposal emphasizes economic necessity, it is of course important to consider the environmental aspects associated with fluoropolymers. As with any material, their production, use, and disposal can have environmental implications. However, the focus should be on improving sustainability and implementing responsible practices rather than imposing a complete ban. Collaborative efforts between industry and regulatory bodies can help address environmental concerns without compromising the functionality and performance benefits offered by fluoropolymers.

When assessing the economic necessity of fluoropolymers, a comprehensive evaluation should consider factors such as the availability of alternative materials, potential impact on various industries, technological feasibility, and the overall economic implications of a ban. Collaboration between industry stakeholders, scientific experts, and regulatory authorities is crucial to ensure a well-informed decision that strikes a balance between economic and environmental considerations.

The proposal to ban fluoropolymers is currently under consideration and has raised discussions and concerns in the industry. It is difficult to imagine a scenario where fluoropolymers are deemed economically unnecessary. These materials play an important role in critical applications in various industries and alternative solutions may not offer comparable performance and functionality. Considering that the Medical Device Regulation is already having a significant impact on the market, this represents a further challenge to ensure the availability of urgently needed products. 

As the decision-making process continues, it is important for industry professionals, regulatory authorities, and environmental advocates to engage in constructive dialogue. Together, we can explore sustainable practices, enhance environmental responsibility, and find innovative solutions that address concerns without sacrificing the benefits fluoropolymers provide.

We should closely monitor the outcome of the decision-making process, taking into account both economic and environmental factors to ensure a balanced approach that promotes innovation, safety and environmental protection in the industry.

What challenges do you see and/or have you already considered alternatives to overcome the “ forever chemicals“?

Further references can be found here:




Exciting news for medical device manufacturers!

Exciting news for medical device manufacturers!

The amendment of Regulation (EU) 2023/607 opens up new opportunities for those transitioning their medical devices to the MDR. Legacy devices can now have the validity of their directive certificates extended (until the end of 2027/2028, depending on the device classification) if certain conditions are met.

To take advantage of this, you need to ensure that you set up an MDR-compliant QMS and submit a formal application for MDR conformity assessment to a notified body by 26 May 2024. Remember that signing a formal agreement with the Notified Body is crucial and must be done by 26 September 2024 at the latest.

Do not hesitate and submit your application to the notified body in time and preferably within the above-mentioned deadlines.

After the expiry of the certificates issued under the Directives and without a valid MDR certificate, manufacturers are no longer allowed to place these medical devices on the EU market. The overall goal of the proposed amendments is to maintain patients’ access to a wide range of medical devices while ensuring the transition to the new framework. The extension will be staggered depending on the risk class of the device, i.e. until December 2027 for devices with a higher risk and until December 2028 for medium and lower risk devices.

Official information can be found here:  https://health.ec.europa.eu/system/files/2023-01/mdr_proposal.pdf


Foundation for the National Institutes of Health (FNIH) Announces AMP BGTC’s Selection of Eight Rare Diseases for Clinical Trials, Paving the Way for Streamlined Gene Therapy Approvals

Foundation for the National Institutes of Health (FNIH) Announces AMP BGTC’s Selection of Eight Rare Diseases for Clinical Trials, Paving the Way for Streamlined Gene Therapy Approvals

Exciting news from the Foundation for the National Institutes of Health (FNIH) as the Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (AMP® BGTC) selects eight rare diseases for its clinical trial portfolio. This innovative approach to gene therapy development for rare diseases demonstrates the potential for streamlined approval pathways through manufacturing and testing standards. By establishing minimum standards and providing information to the public at no cost, the BGTC aims to expedite the delivery of customized gene therapies. This milestone enables further options for treating patients with rare genetic diseases and helps prepare the way for approved therapies. We congratulate the FNIH and the AMP BGTC for their commitment to advancing medical research and improving outcomes for patients.

Read more: https://lnkd.in/ewmz-nhW


Promotion of Authentic Healthcare Innovations and Treatments

Promotion of Authentic Healthcare Innovations and Treatments

In the constantly evolving landscape of healthcare, innovation is the driving force behind breakthrough treatments and solutions. However, not all innovations are created equal. It is crucial to distinguish between advances in healthcare that are truly replicable and authentic, and those that do not bring benefits to patients in a concrete way. Appropriate laws and regulations play a crucial role in this regard, ensuring that patients continue to benefit from high-quality and cost-effective treatments.

The GKV Financial Stabilisation Act (GKV-FSG) in Germany has a lot of potential to advance clinical research and the development of innovations in a targeted manner. One of the main benefits of the GKV-FSG is to promote financial support for clinical research. Closer cooperation with health insurance funds and sponsors enables more studies to be conducted in Germany under the highest standards, corresponding quality and efficiency.

The GKV-FSG reinforces and takes into account the cost-effectiveness of clinical research. Studies that demonstrate a clear benefit for patients receive more attention. With the focus on more efficiency, real-world data can be used to help provide more accurate data on the effectiveness of treatments in real-world situations.

The introduction of new laws and regulations, promote and improve the quality and safety of clinical research in Germany. For example, the GKV-FSG Act has introduced new requirements for adverse event reporting that can help improve patient safety and the overall quality of clinical research studies.

In total, the GKV-FSG entails an increased administrative burden. In terms of increased financial support, a stronger focus on cost-effectiveness and the introduction of new regulations, the quality and safety of clinical research studies in Germany will be improved still further. It becomes evident what impact the GKV-FSG will have on Germany, as an important, strategic research location, and on clinical research and development. The focus is on ensuring that patients continue to benefit from high-quality, cost-effective treatments.

#healthcareinnovation #clinicalresearch #outsource #clinicaltrials #consulting #research #researchanddevelopment  #healthcare #biotech #pharma #medicine  #lifescience #neurology #autoimmune #immunology #clinicaldevelopment #clinicaltrial #ClinicalResearchOrganisation #celltherapy #cartcelltherapy #biotech #biotechnology #cancerresearch #cancer #immunology #immunotherapy #collaboration #rarediseases #rarediseasetrials #rarediseaseawareness

Rare Disease Treatment: Exciting Advances in Clinical Research

Rare Disease Treatment: Exciting Advances in Clinical Research

Rare diseases affect millions of people worldwide, but diagnosis and treatment can be challenging due to low prevalence. The lack of available data and limited understanding of these diseases often leads to misdiagnosis and delayed treatment, resulting in significant morbidity and mortality. Meanwhile, with the increasing focus on clinical research on rare diseases, there are innovative treatment options for patients with rare diseases.

Clinical trials have an important role in the development of new treatments for these diseases. Safety and efficacy of new treatments, as well as the optimal dose, administration and duration of treatment, need to be determined. Phase I and II studies provide important information for further clinical development.

In Germany, clinical research on rare diseases is at the leading edge of scientific innovation. The health care system offers nationwide coverage and access to high-quality medical treatment options. Germany as a location offers highly attractive opportunities for clinical trials.

The German Federal Ministry of Education and Research (BMBF) has launched several initiatives to promote research on rare diseases, including funding for clinical trials. For example, the BMBF works with organisations and sponsors to ensure that the latest scientific research and technological advances are incorporated into clinical research in Germany.

Clinical research on rare diseases in Germany includes studies on Pompe disease, a rare genetic disorder that affects muscles and other tissues. Studies on the safety and efficacy of a new enzyme replacement therapy achieved promising results.

A current phase III trial is testing a new oral drug in patients with Niemann-Pick type C disease, a rare lipid storage disorder that affects the nervous system. Initial results suggest that the treatment may slow the progression of the disease.

These trials are just a small sample of the innovative clinical research on rare diseases conducted in Germany. With its strong scientific fundament, favourable regulatory environment and access to advanced technologies and care, Germany is an ideal location for clinical research.

Rare diseases are a major challenge for patients and healthcare providers worldwide. With an increasing focus on clinical research into rare diseases, improved diagnosis, treatment and outcomes for patients with these diseases can be made possible. Germany’s commitment to rare disease research, its strong scientific infrastructure and widespread access to the healthcare system make it an ideal location for clinical trials that can benefit patients around the world.

#rarediseases #rarediseasetrials #rarediseaseawareness #clinicalresearch #outsource #clinicaltrials #consulting #research #researchanddevelopment  #healthcare #biotech #pharma #medicine  #lifescience #neurology #autoimmune #immunology #clinicaldevelopment #clinicaltrial #ClinicalResearchOrganisation #celltherapy #cartcelltherapy #biotech #biotechnology #cancerresearch #cancer #immunology #immunotherapy #collaboration

Unlocking the Power of the 5HT-2A Receptor: Natural Ways to Enhance Mood and Cognition for Optimal Brain Function

Unlocking the Power of the 5HT-2A Receptor: Natural Ways to Enhance Mood and Cognition for Optimal Brain Function

The 5HT-2A receptor plays a crucial role in regulating mood and cognition. Discover natural ways to strengthen this receptor to improve brain function !!! Great to see that research is breaking new ground.


The Future of Life Science Research: Targeted Solutions and Collaborative Expertise with REAgency+

The Future of Life Science Research: Targeted Solutions and Collaborative Expertise with REAgency+

The business world is changing: REAgency+ stands for more flexibility and targeted resourcing solutions. We offer life science expertise for research and development in the true sense of the job description. Performance competence and also an understanding of business development are essential for efficient cooperation. From EXPERTS for EXPERTS, REAgency+ disruptively builds a bridge for a new and sustainable way of working together. In a perfect working environment, we create targeted solutions that meet the growing needs of people, exploratory business structures, young scientific experts and explorative thinking. Sustainable, diverse, efficient and with added value.

It is simply no longer contemporary or acceptable to provide or communicate expertise without added value and understanding in collaboration.

Let’s find new solutions together instead of just thinking about them.


CTIS Submission Planning and Implementation Expert at REAgency+ Available to Offer Support and Solutions

CTIS Submission Planning and Implementation Expert at REAgency+ Available to Offer Support and Solutions

The Clinical Trial Information System (CTIS) is an electronic system that supports the management and submission of clinical trials to regulatory authorities. With the new European Union Clinical Trials Regulation (CTR) come into force in 2022, companies conducting clinical trials in the EU will have to use the CTIS to submit their trial data. As a result, companies are currently integrating CTIS submissions into their processes for clinical trials. Some of the CTIS experiences of the REAgency+ experts in collaboration with a biotech client are presented here.

The company in the case is a US-based biotech company specialising in oncology pharmaceuticals. The company had previously conducted clinical trials in the EU, but had not yet implemented CTIS submission into the company’s processes. Here are some key learnings and lessons learned in the collaboration:

Start early and include delays: implementing CTIS submission processes can be a prolonged process that requires a considerable effort in planning and preparation. The company started planning more than a year before the next clinical trial. Nevertheless, unexpected delays and drawbacks occurred, so that the realisation went on a bit longer than expected.

Working with REAgeny+ experts: Implementing the CTIS submission requires a dedicated team of people who are familiar with the system and regulatory requirements. The company found that it was necessary to allocate additional resources to the CTIS submission to ensure that it could be completed in a timely and efficient manner.

CTIS submission can be a complex process and companies may not have the required expertise. The company found it helpful to work with external experts from REAgency+ who had experience with CTIS implementation and regulatory requirements.

Understanding of legal requirements: The CTIS system is designed to help companies comply with legal requirements, but it is important that companies understand these requirements to ensure that their CTIS submission is successful. The company found it helpful to work with REAgency+ experts to understand the regulatory requirements and timelines. Also, working with investigative sites, required OMS registrations, even though still with some system errors, were completed swiftly and required documents were obtained.

Communication and collaboration with stakeholders: CTIS submission can involve multiple parties within a company such as clinical teams, data management teams, regulatory affairs teams as well as involved stakeholders and vendors. The company found it beneficial, in cooperation with REAgency+ experts, to communicate and collaborate with these stakeholders on a regular basis to ensure that all requirements are met in a timely manner.

In conclusion, CTIS submission can be a complex and time-consuming process, but it is an important step for companies conducting clinical trials in the EU. In conclusion, the standardisation via CTIS submission is considered an advantage for the evaluation and timeline expectation. There are also significantly less documents needed for the submission (Part II). By starting early, allocating resources appropriately, working with experts, understanding regulatory requirements, and communicating and collaborating with stakeholders, companies can successfully incorporate CTIS requirements into their regulatory processes.

#clinicalresearch #outsource #clinicaltrials #consulting #research #researchanddevelopment   #healthcare #biotech #pharma #medicine #lifescience
#clinicaldevelopment #clinicaltrial #ClinicalResearchOrganisation #CTR #CTIS #regulatoryaffairs #CRO #biotechnology