INTEGRATE stands for Integrated Care of New Therapies through Telemedicine, Empowerment, Gene Therapies, Registries, Drug (“Arzneimittel”) Safety, Structured Treatment Pathways, and Reimbursement (“Erstattung”). The INTEGRATE-ATMP project has been approved by the Innovation Fund of the Federal Joint Committee (“Gemeinsamer Bundesausschuss (G-BA)”) and statutory health insurance funds for four years, with total funding of €13.64 million.
Homogeneous and harmonized care structures are important for the use of advanced therapy medicinal products (ATMPs). 24 academic German treatment centers have joined forces for the INTEGRATE-ATMP project, which is currently funded by the Innovation Fund of the Federal Joint Committee (G-BA). The project will develop harmonized and quality-assured instruments to ensure the best possible quality of treatment for patients treated with ATMP and test them at the participating centers. The success of these instruments will be evaluated, among other things, by recording the health-related quality of life and satisfaction of the patients and the treatment providers. If successful, INTEGRATE-ATMP will make a significant contribution to creating a binding framework for the quality-assured application of existing and future ATMPs in Germany, thus ensuring the best possible care for patients.
The project addresses adult patients suffering from diseases of the lymphatic system who are treated with chimeric antigen receptor (CAR) T-cells. It also includes children with spinal muscular atrophy (SMA), metachromatic leukodystrophy (MLD), or severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), who are being treated with ATMPs.
ATMP Therapy
ATMPs (Advanced Therapy Medicinal Products) are innovative medicinal products composed of nucleic acids, cells, or tissues. Gene therapies aim to repair, replace, or regulate defective genes, while somatic cell and tissue therapies involve cells that have been genetically modified in vitro to acquire specific functions, such as immune defense against cancer or tissue regeneration.
High Quality Standards in the Use of ATMPs
In recent years ATMP therapies have increasingly been approved in Germany. Since these therapies involve modifications to the genome, high quality standards are set for ATMPs and their application. This presents several challenges: The administration of ATMPs in medical centers introduces new requirements. To meet these demands, existing healthcare structures at the hospitals must be adapted for outpatient pre- and post-treatment care. This also includes financial and structural adjustments to align with the effort involved in the application of ATMPs. Experience shows that the treatment effort for patients receiving ATMPs is significantly higher compared to other therapies.
INTEGRATE-ATMP aims to help establish binding frameworks for the quality-assured and cost-effective application of existing and future ATMPs in Germany, thereby ensuring optimal care for patients. Ideally, this will lead to an improvement in the individual quality of life of the patients.
INTEGRATE-ATMP Structural Measures
The optimization of care for ATMP patients in the project will be achieved through the phased introduction of several new healthcare structures, referred to as structural measures:
1. Standardized ATMP Treatment Plans for Structured Pre- and Post-Care
In Germany, there are quality assurance guidelines for the administration of ATMPs developed by the Federal Joint Committee (G-BA). However, there is a lack of standardized, nationwide structured treatment plans for therapy preparation and post-treatment care following the administration of ATMPs. Additionally, there is no uniform, cost-covering reimbursement for the resource-intensive pre- and post-care associated with ATMP therapies at specialized medical centers.
This is why, within the project, structured treatment plans for pre- and post-care are being developed and implemented, tailored to the specific disease. These plans are being implemented uniformly across all involved medical centers and are being funded accordingly. Furthermore, the consortium and the Scientific Advisory Board will develop a financing plan for the structured treatment plans. This is intended to allow an evaluation of the cost-effectiveness of the project-related measures after the project concludes.
The treatment plans and financing models developed in the project are also intended to serve as a blueprint for the care of ATMPs that are currently under development and will be approved in the future.
2. Cross-Disease INTEGRATE-ATMP Registry for a Comprehensive Data Collection
Medical registries collect data on diseases, disease progressions, therapies, and/or patient groups. These registries aim to address medical-scientific or epidemiological questions alongside quality assurance aspects.
Registries are essential for rare diseases, as they collect standardized data across locations and borders, enabling a better description of the diseases and the impact of new therapies on disease progression. The more data a registry contains, the greater the information gain and the ability to assess existing therapies in terms of their safety and effectiveness. Most commonly, registries focus on a specific disease. Patients with the relevant condition are observed over an extended period. Currently, there are over 400 different medical registries in Germany, but most of them cannot be linked with other registries, which limits the exchange and joint analysis of data to enhance their validity.
The INTEGRATE-ATMP registry aims to overcome this isolated data collection. The registry developed within the project is designed to capture data on various diseases treated with ATMPs. It will not replace existing disease registries but will be linked with them and can be expanded to include new ATMP approvals in the future.
3. Telemedicine INTEGRATE-ATMP Platform and App for Targeted Communication
A telemedicine communication platform and app will facilitate direct communication between all parties involved in ATMP treatment (patients, doctors, case managers). The goal is to alleviate the everyday burdens on patients in their therapy and on medical center in their daily care operations. For example, treating physicians can have the therapy data of their patients presented in a clear format. A further advantage is the integration of the telemedicine platform with the INTEGRATE-ATMP registry.
The telemedicine platform is available to patients as an app for smartphones and tablets. The app allows patients to input health data, view appointments in a structured manner, and quickly contact their healthcare providers.
4. Side Effect Management Platform for Children with Spinal Muscular Atrophy
As part of the project, the side effect management platform offers parents of children who have received gene therapy for spinal muscular atrophy the opportunity to record any unwanted side effects of the therapy. Based on this data, the project will develop suitable therapy recommendations for these side effects.
If severe side effects occur, the medical staff must report them to the regulatory authorities. This legal reporting obligation remains in place within the INTEGRATE-ATMP project.
Reporting doctors can consult an expert group that will advise on the respective case. The goal is to receive a joint therapy recommendation based on the best available current knowledge. This is done in consultation with the treating physicians, who can implement the therapy recommendation. The INTEGRATE-ATMP project coordination will document the success of the therapy for the side effect. This will help improve knowledge about side effects and their treatment.
5. Various Treatment Options for Optimal Care
ATMP therapy may involve a treatment decision board consisting of doctors from different specialties. Outpatient specialists can present patients who may be suitable for ATMP treatment at the respective medical center. In the context of the INTEGRATE-ATMP project, this treatment decision board can be convened through the telemedicine platform. After the ATMP treatment at the medical center, post-care for patients should be provided in cooperation with outpatient specialists. The telemedicine platform of the INTEGRATE-ATMP project enables continuous, secure, and structured information exchange between the medical center and outpatient physicians.