Guidelines on Target Assessment for Innovative Therapeutics(GOT-IT)
Continuous biomedical research and development of new drug compounds contribute to the maintenance of good medical care through innovative therapies. The basis for the development of new drug compounds is the identification of disease-modulating molecules, so called targets. This kind of research is usually conducted at academic institutions.
The validation of these molecules based on solid quality criteria is a pivotal step for the development of potential new drug compounds, before they can be considered for use in clinical studies. In addition, however, the successful translation of basic research findings into new therapeutics often requires that further areas and aspects are addressed like target-related safety issues, druggability and assayability, as well as differentiation from established or emerging therapies, which are summarized as 'target assessment' in the context of the GOT-IT project.
To support the shift from purely academic exploration to the initiation of formal drug discovery the GOT-IT recommendations help in identifying and prioritizing relevant target assessment activities and in defining a critical path to reach scientific as well as goals related to licensing, partnering with industry or initiating clinical development programs. Based on sets of guiding questions for different areas of target assessment, the GOT-IT framework is expected to stimulate academic scientists' awareness of which factors are important to generate robust and high-quality research outcomes and to facilitate academia-industry collaborations.
The GOT-IT recommendations have been refined through communication with the experts from the broad research community (including pharma, biotech, technology transfer, funders), especially also with the individual 'Target Validation' teams within the BMBF Target Validation for Drug Development program. Dissemination and sustainability of the recommendations will be facilitated via an Educational as well as Online Expert Platform.
The GOT-IT team consists of working groups from the Department of Experimental Neurology, Charité - Universitätsmedizin Berlin, the Translational Drug Validation and Preclinical Disease Models group at the Fraunhofer Institute for Molecular Biology and Applied Ecology (IME-TMP) in Frankfurt a.M. and the PAASP GmbH (Partnership for Assessment and Accreditation of Scientific Practice) in Heidelberg.
Emmerich CE, Martinez-Gamboa L, Hofmann MCJ, Bonin-Andresen M, Arbach O, Schendel P, Gerlach B, Hempel K, Bespalov A, Dirnagl U, Parnham MJ. Improving target assessment in biomedical research: the GOT-IT recommendations. Nature Reviews Drug Discovery (2020) Guidelines and recommendations PMID: 33199880
Academic research plays a key role in identifying new drug targets, including understanding target biology and links between targets and disease states. To lead to new drugs, however, research must progress from purely academic exploration to the initiation of efforts to identify and test a drug candidate in clinical trials, which are typically conducted by the biopharma industry. This transition can be facilitated by a timely focus on target assessment aspects such as target-related safety issues, druggability and assayability, as well as the potential for target modulation to achieve differentiation from established therapies. Here, we present recommendations from the GOT-IT working group, which have been designed to support academic scientists and funders of translational research in identifying and prioritizing target assessment activities and in defining a critical path to reach scientific goals as well as goals related to licensing, partnering with industry or initiating clinical development programmes. Based on sets of guiding questions for different areas of target assessment, the GOT-IT framework is intended to stimulate academic scientists' awareness of factors that make translational research more robust and efficient, and to facilitate academia-industry collaboration.
Critical Path Generator
The Critical Path Generator tool will assist scientist in planning and structuring a target assessment project. The CP Generator will help to arrange important target assessment blocks into a project-specific Critical Path. Using a query form and different sets of guiding questions (Critical Path Questions (CPQs) & Experimental Approach Questions (EAQs)), the CP Generator identifies strengths and weaknesses of a translational project and provides support how to invest critical resources in an optimal way.
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The Educational Tool for young scientists informs about important aspects of target assessment. The target assessment process is depicted as a train journey. In the Central Station, information is provided for planning a target assessment project. Being on the Journey, you will be taken through different Train Stations that provide additional information about important core aspects of target assessment. In addition, illustrative examples for potential Roadblocks, Delays and Project Goals are provided.
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GOT-IT Expert Platform
The GOT-IT recommendations consider the complexity of tasks associated with bridging the gap between basic science research findings and the identification of clinically effective products. The guidelines suggest the need for an environment where academic research teams focusing on newly identified drug targets can connect with experienced and qualified expert industry teams in support of successful drug discovery and development. The GOT-IT Expert Platform is designed to facilitate these interactions. Based on an innovative match-making algorithm, the platform helps academic scientists recruit drug discovery experts, receive timely and well-informed advice, and develop a more extended network of partnerships to ultimately increase the exchange between Industry and Academia. A set of onboarding questions ensures that project-specific goals and individual needs are taken into account so that productive collaborations can provide the most relevant and meaningful support for early-phase translational drug discovery programs.
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