As projects on promising targets progress towards the clinical development of new drug candidates, additional aspects become critical. Target validation can be understood in numerous ways depending on the context (Box 1), but usually describes the technical evaluation of whether a target has a key role in a disease process and whether pharmacological modulation of the target could be effective in a defined patient population 5. As a result, there is common consensus that robust target validation is a crucial part of drug discovery and deserves greater emphasis in order to facilitate the development of new therapies. Indeed, it was predicted a decade ago that more effective target validation as well as early proof-of-concept studies could reduce attrition in phase II clinical trials by ~24%, lowering the cost of developing new molecular entities by ~30% (ref. Importantly, insufficient validation of drug targets at an early stage has been linked to costly clinical failures 1 and low drug approval rates 2, 3. Once a new target has been identified, various strategies are used to provide validation of the target and to support decisions, such as initiating an extensive drug discovery programme, conducting a proof-of-concept trial in humans or partnering with another organization. ![]() ![]() The discovery and development of first-in-class drugs often begins with the identification of a new drug target, such as an enzyme linked to a disease process. 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. 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. ![]() 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. 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. Academic research plays a key role in identifying new drug targets, including understanding target biology and links between targets and disease states.
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