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.

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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

Experienced Clinical Research Partner: REAgency+ Facilitates Biotech Industry Advancements and Product Introductions

Experienced Clinical Research Partner: REAgency+ Facilitates Biotech Industry Advancements and Product Introductions

REAgency+ had the pleasure of working with a variety of clients from the biotech industry, as well as others, for clinical research projects, supporting them to advance their research efforts and introduce new products to the market. We are an excellent option for companies that need specialised expertise with limited resources available to assist their research efforts. This post summarises some of the benefits in working with each other that were experienced over the past years. Here explaining what advantages REAgency+ can be a highly valuable resource for companies that are expanding.  

REAgency+ is flexible and cost effective. On a project basis, we can enable companies to access expertise without long-term commitment or having to manage complex, intransparent service structures. The focus of REAgency+ is to enable an efficient value creation chain from and for experts and to support companies with an individual approach, all in one.  This can be particularly beneficial for small or medium-sized companies that have no resources or access to the relevant expertise. REAgency+ provides a flexible, cost-effective and transparent cost control structure, which can be essential for companies managing their budgets effectively.

REAgency+ is a specialist. Another key advantage for clinical research projects is access to expertise. With REAgency+, you have the opportunity to cover specific areas of expertise. This allows REAgency+ to provide targeted solutions tailored to specific business needs. We also frequently experience a collaborative partnership, with new perspectives on project goals. This is especially appreciated. 

REAgency+ is available at short notice. We enable fast start-up times to get projects up and running quickly and efficiently. Especially in terms of planned timelines and KPIs, this is very valuable in collaboration. REAgency+ is committed providing practical expertise at a high quality level as fast as possible and in a target-oriented approach to the projects. Because of our extensive practical experience, we are very quick to integrate into work processes, structures and requirements. Also in terms of steakholder and vendor support and excellent understanding of practical workflows, REAgency+ enables companies to focus on core competencies while outsourcing sub-areas on an individualised basis. This can in combination accelerate companies‘ research efforts and help develop products and advance project goals more effectively.

REAgency+ is a solution to support growing corporate organisations with clinical research projects, hands-on, specialised with expertise, flexible and cost effective. Clients are provided with targeted solutions consistently, with a fast start-up time and a cost effective approach. 

A Case Study: Phase III Clinical Trial Impacts Survival of a Biotech Company

A Case Study: Phase III Clinical Trial Impacts Survival of a Biotech Company

As the clinical trials industry continues to grow and evolve, the role of contract research organisations (CROs) in managing clinical trials becomes increasingly important. CROs can provide valuable support to biotech companies by managing various aspects of clinical trials, including study design, patient recruitment and data analysis.

A case study in collaboration with a German biotech company demonstrated the importance of a specific analysis of requirements and objectives when working with service providers. Effective management of a clinical trial can have serious consequences for a small biotech company. In this case study, a biotech company partnered with a large service provider to conduct a phase III clinical trial for a promising oncology therapy. The biotech company expected the trial to be successful in generating financing for future research and development work and, more importantly, to obtain regulatory approval for this drug.

However, the partnership between the company and the contracted service provider unfortunately resulted in several serious errors that ultimately resulted in the breakdown of the study, the company’s objectives and also the continuation of the company’s business. 

In cooperation with independent consultants and experts, in SWAT function, the biotech company wanted to bring the study back into its internal administration in order to rescue what remained to be restored. To Date, the biotech had very carefully and with great effort and expense coordinated a workshop lasting several days, in which they discussed details of the further procedure and what they needed for this very demanding task.

In the course of the process of return the project to in-house administration , it turned out that the communication between the company and the contracted service provider already showed considerable and multi-layered deficits. As a result, only incomplete information was available regarding sponsor oversight, or contractually agreed work requirements were generally not performed. In an extensive procedure, missing documents, work processes and data from the last 2 years had to be retrospectively revised, compared and processed.

There was also a lot of uncertainty among the study sites, involved vendors and stakeholders. Study sites and vendors were not managed effectively and it was not ensured that the study protocol was adhered to. As a result, the study data were not considered to be of sufficient quality to demonstrate the efficacy of the drug. Many of the data were not valid or were not adequately verified. A substantial backlog of data became evident. With a great effort and the support of the study sites, all data had to be retrospectively assessed, transferred to the database, verified again and evaluated. 

The biotech company was not sufficiently informed about the status of the study until this date, which led to misunderstandings and delays in decisions and effective work processes. This had a significant impact on compliance processes, the regression of the study and the retrospective processing of the study data.

As a result of these errors, the Phase III trial failed and the biotechnology company was no longer able to secure the funding it needed to continue its research and development efforts. In the end, the focus was on saving data and properly closing the trial. The overall situation was a fatal hit to the company, which was finally forced to reduce its staff, downsize its operations and ultimately be liquidated. 

This case study demonstrated the importance of carefully selecting external service providers. As a start-up or growing company, it is important to ensure that their CRO partners have demonstrated success and expertise in the therapeutic area and are able to effectively manage all aspects of a clinical trial. In this specific case (which is not the only case in the past 10 years), it turned out that significant additional costs were planned for insufficient resources. The biotech company was no longer able to oversee the study status. An unbalanced dependence of the biotech company on the service commitment of the CRO occurred. This was an insidious process and resulted in the complete breakdown of the study targets. Finally, the financial resources ran out and the CRO was no longer able to provide specific results of work activities. 

Biotech companies with specialised, exploratory clinical trial requirements need to actively track trial progress and ensure that their service partners are meeting their specific expectations and milestones as expected. Effective communication can be essential for the success of the clinical trial.

REAgency+ is happy to advise you on the strategic planning and implementation of clinical trials, also in a CRO context as a second-tier partner or independent consultancy.

The power of engineered T cells – a new treatment option for cancer patients ….and more !

The power of engineered T cells – a new treatment option for cancer patients….and more !

The use of T cell receptor-directed T cells to target cancer antigens is an exciting area of cancer research. Realising the full potential of a personalised therapy option, it still presents challenges.

There is a clear need for collaboration between researchers, clinicians and industry leaders to overcome these challenges and advance the field of T-cell receptor-directed therapy opportunity. The potential benefits of this approach are significant, and with continued research and development, there is potential for personalised and effective treatments.

CAR-T cell therapies are a promising new approach to cancer treatment that is gaining increasing attention around the world. In Germany, the introduction of CAR-T cell therapies has been relatively slow, but the country is starting to make up ground. 

For CAR-T cell therapies, the patient’s own T cells are genetically modified to recognise and target cancer cells. For this purpose, a chimeric antigen receptor (CAR) is added to the surface of the T cells, allowing them to recognise and bind to specific proteins on the surface of cancer cells. Once the CAR T cells have bound to the cancer cells, they can destroy them.

The first CAR-T cell therapy was approved by the FDA in the United States in 2017, and since then several more CAR-T cell therapies have been approved in the US and Europe. These treatments have shown remarkable results in clinical trials. By the end of 2021, three autologous anti-CD19 CAR-T cell products, Yescarta, Kymriah and Tecartus, received marketing approval based on positive clinical data from phase I/II trials. In the EU, all three products have been subject to an accelerated review (PRIME designation process) to facilitate the development and clinical use of advanced therapy medicinal products that meet an unmet medical need.

CAR-T cell therapies are not yet widely used in Germany, but the country is starting to catch up with other countries in terms of acceptability. Several clinical trials of CAR-T cell therapies are currently in progress in Germany, including trials for the treatment of lymphoma, leukaemia and multiple myeloma.

Challenges to introducing CAR-T cell therapies in Germany include the financial investment required for these treatments. The production and administration of CAR-T cell therapies are still quite cost-intensive, so health insurance companies are still hesitant to cover the costs of these treatments. However, as more data on the efficacy of these therapies becomes available, it is likely that more health insurance providers will cover the costs.

Despite the challenges, the potential benefits of CAR-T cell therapies are significant. CAR-T cell therapies are a highly personalised treatment that is tailored to each individual patient. This treatment offers versatile and personalised ways to treat different types of cancer. The potential for other personalised immunotherapies is also of great interest. For patients who have exhausted all other treatment options, CAR-T cell therapies offer hope.

CAR-T cell therapies are an exciting new development in cancer treatment and more. While the introduction of these therapies in Germany has been slow, the country is starting to make up ground with several clinical trials currently ongoing. As more and more data becomes available on the effectiveness of these treatments, it is likely that CAR-T cell therapies will become an important treatment option.

It remains exciting…

#clinicalresearch #outsource #clinicaltrials #consulting #research #researchanddevelopment #share #like #healthcare
#biotech #pharma #medicine #oncology #lifescience #neurology #autoimmune #immunology #clinicaldevelopment #clinicaltrial #ClinicalResearchOrganisation #celltherapy #cartcelltherapy #biotech #biotechnology #cancerresearch #cancer #immunology #immunooncology #immunotherapy #collaboration

The Future for Clinical Research and Development in Germany – What’s Next ?

The Future for Clinical Research and Development in Germany

What’s Next ? Reduce dependencies, take more risks!

Prof. Bernd Raffelhüschen is a famous German economist who has been drawing attention to the demographic changes in Germany for years.  
In his lectures he often suggests and calls for a more independent position from the demographic problems in Germany.

In the scope of many conferences and network cooperations, REAgency+ is a representative for the promotion of young academics and a contact person for business advice and development in clinical research. In the interaction with young research academics, innovators and industry, there is a great demand for business and personal development opportunities, added value in collaboration and an effective value chain.

Bernd Raffelhüschen, for example, once recommended in one of his lectures to focus on future-relevant areas and to push the development of new industries and technologies. Economic growth should be supported and the quality of life in Germany improved.

Providing research funding, especially for areas relevant to the future, can help researchers develop their skills and expertise.
Above all, and REAgency+ is committed to and supports this approach, it is particularly important to promote a culture of innovation. Raffelhüschen suggests to be encouraged to take risks and explore new ideas.

International collaboration also creates opportunities for researchers to work with international partners.
Business entrepreneurship needs to be boosted and researchers need to be supported more. Start-ups and small businesses could be supported more by a supportive legal environment.

What is your opinion on the topic?

Here is one of the available lectures by Prof. Bernd Raffelhüschen

#clinicalresearch #outsource #clinicaltrials #consulting #research #researchanddevelopment #healthcare
#biotech #pharma #medicine #oncology #lifescience #neurology #autoimmune #immunology #clinicaldevelopment #clinicaltrial #ClinicalResearchOrganisation  #regulatoryaffairs #demographics #futureofhealthcare # #entrepreneurship #work #innovation #opportunities #funding #growth #future #development #academics #researchers