About the project
Medical recommendations are based on high-quality evidence, which is ideally generated by randomised controlled trials (RCTs). In order to collect sufficient data to assess a therapy, a new RCT is usually set up for each new therapeutic approach. This procedure is associated with long implementation times, high costs and a lack of flexibility. Adaptive platform studies are considered a practicable solution to the above-mentioned problems, especially when new evidence is generated quickly in the relevant area, e.g. in the context of a pandemic. NAPKON-TIP aims to establish an infrastructure for adaptive clinical platform studies in Germany. Using the recruitment network established in NAPKON (National Pandemic Cohort Network) and the NUKLEUS (NUM Clinical Epidemiology and Study Platform) infrastructure, successful concepts from already internationally successful study platforms are to be integrated and adapted to the legal national context (regulatory, data protection) in the Network University Medicine (NUM).
In the context of the COVID 19 pandemic, the study landscape in Germany was strongly challenged and also critically scrutinised. In an international comparison, it became clear that Germany has conducted significantly fewer clinical studies and, above all, adaptive platform studies on COVID-19 compared to France and the United Kingdom, for example. In this respect, it is noticeable that there is a lack of central coordination for studies of national importance in the field of infection in Germany, as well as a lack of comprehensive infrastructures for adaptive platform studies. In order to meet the challenges of current and future pandemics as well as current research questions with high medical demands, a network is needed that can quickly focus the capabilities of Network University Medicine on the particularly urgent and important tasks and conduct complex clinical studies itself. The aim of the project is therefore to establish a Therapeutic Intervention Platform (NAPKON-TIP) based on the recruitment network established in the National Pandemic Cohort Network (NAPKON) and the NUM infrastructures, in particular the NUM Clinical Epidemiology and Studies Platform (NUKLEUS), and to demonstrate its performance in a concrete study. If successful, NAPKON-TIP aims to provide the NUM and external parties with the necessary services for conducting adaptive platform studies as part of the NUM infrastructure.
In NAPKON-TIP, therapy studies in which drugs, devices/software (medical devices) and non-pharmacological interventions are to be tested are planned independently of the first use case. The plan is to stratify patients based on individual markers or a combination of clinical profiles, biomarkers, imaging markers and psychometric data. Data collected by digital health applications will also be used for this purpose. Regular interim analyses and mathematical modelling are required to enable continuous adjustments of the clinical studies to the latest state of knowledge using the available data and digital information (principle of the adaptive study). Master protocols and routines are to be developed in structured working groups - organised in clearly defined work packages - which must be regularly communicated and, above all, harmonised between the working groups.
Coordinated by an interdisciplinary Steering Committee, NAPKON-TIP will initially create the necessary organisational and structural conditions for conducting clinical trials in accordance with the AMG and MPG, particularly with regard to adaptive platform studies, and demonstrate their capabilities in a specific study, the first use case. NAPKON-TIP comprises six work packages (WP). WP 1 includes the overall project management, which deals with management and communication at the overall project level and develops the integration of NAPKON-TIP into the existing structures of NUKLEUS, NAPKON and NUM. WP 2 will develop and implement an infrastructure for the continuous identification and prioritisation of urgent research questions. For the adaptation of clinical trials to the latest state of knowledge (principle of the adaptive trial), therapeutic strategies must be constantly revised. WP 3 therefore aims at continuous stratification based on individual markers or a combination of clinical profiles, biomarkers, imaging markers and psychometric data. WP 4 aims to facilitate and accelerate ethical and regulatory aspects related to platform studies, while WP 5 will establish a complete service infrastructure for the implementation of adaptive studies. WP 7 completes the project and develops recruitment strategies for hard-to-reach study populations in emergency, intensive care and general medicine.
We expect the adaptive structure to offer the following advantages over a classic study design:
- Accelerated assessment and translation of basic research into clinical practice in terms of stratification markers, interventions and outcomes
- Advantages for the patients participating in the platform study, who benefit directly from the treatments provided in the study
- Rapid transferability of quickly obtained results on the safety and efficacy of a treatment at a generalised level to all patients involved in the study
Methodologically high-quality and sufficiently large clinical trials are a necessary condition for generating clinical evidence, creating or influencing guidelines to improve treatment outcomes for patients. NAPKON-TIP gives the NUM the opportunity to roll out practice-changing clinical trials nationally and thus offer a key infrastructure for the revitalisation of the clinical trial landscape in Germany.
People in the NUM
Highlights
Study on treatment options for post-COVID syndrome
The first use case RAPID_REVIVE(Randomised adaptive assessment of post-COVID syndrome treatments_Reducing Inflammatory Activity in Patients with post-COVID syndrome) is an adaptive study on a treatment option for post-COVID syndrome and is expected to start recruitment in the third quarter of 2024.