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      The IRDiRC Drug Repurposing Guidebook: Creating an efficient and visible pathway for rare diseases

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            Abstract

            Drug repurposing is an exciting topic in the world of rare diseases, and it has often been suggested as a key approach for developing more therapies for the estimated 6000-8000 rare diseases. This strategy can be an attractive option because it often involves developing therapies in an efficient, potentially cheaper, and innovative way, building on previous knowledge and experience. Drug repurposing can be defined in several ways but in broad terms, can be considered as developing an existing drug in an indication outside the scope of the original indication, with the ultimate purpose of obtaining a new regulator-approved indication. Several tools and incentives have been developed to stimulate and ease the approach for repurposing for rare diseases. Nevertheless, the field still sees quite some challenges, such as intellectual property issues, lack of knowledge on regulatory requirements, the need for additional (re)formulation or obtaining additional safety-efficacy data that may be difficult to collect, and difficulties in commercialization due to the lack of sustainable business models. Consequently, repurposing approaches for rare diseases have, until now, not been as impactful as anticipated.

            We will present the work of IRDiRC’s Therapies Scientific Committee Task Force, following the previously launched Orphan Drug Development Guidebook. We set out to develop a Drug Repurposing Guidebook. This Guidebook is developed for researchers and developers involved in drug repurposing in the rare disease space, specifically academics, startups, small and medium enterprises, and patient-led groups. This Drug Repurposing Guidebook gathered and reviewed tools and created a roadmap to help deliver an efficient development program. This roadmap is integrated with a Gannt chart, highlighting the key repurposing activities for each development phase with checklists to consider the necessary steps to be implemented before starting a repurposing project. As such, this Guidebook can help researchers and developers who want to optimize a repurposing project for rare diseases. By allowing an understanding of the available tools, by asking the developer essential questions at different stages and directing them to the available resources, repurposing for rare diseases can be faster and more efficient, and more aligned with the regulatory processes.

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            Author and article information

            Conference
            REPO4EU
            12 September 2023
            Affiliations
            [1 ] University of Twente, Enschede (The Netherlands) ( https://ror.org/006hf6230)
            [2 ] IRDiRC, Paris, (France);
            [3 ] MHRA, London, (United Kingdom);
            [4 ] Evotec, Verona, , (Italy);
            [5 ] Clinical Trials Consulting & Training, North Marston, (United Kingdom);
            [6 ] EATRIS, Amsterdam (The Netherlands);
            [7 ] FDA, Washington DC (United States);
            [8 ] CBG, Utrecht (The Netherlands) ( https://ror.org/05mv4rb84)
            Author information
            https://orcid.org/0000-0001-5883-7610
            https://orcid.org/0000-0003-3852-9738
            https://orcid.org/0000-0002-7264-5436
            https://orcid.org/0000-0002-4601-5975
            https://orcid.org/0000-0003-0641-3829
            Article
            10.58647/REXPO.23004
            e8afe1e8-faa7-47b4-8bdc-27a73815522f
            Authors

            Published under Creative Commons Attribution 4.0 International ( CC BY 4.0). Users are allowed to share (copy and redistribute the material in any medium or format) and adapt (remix, transform, and build upon the material for any purpose, even commercially), as long as the authors and the publisher are explicitly identified and properly acknowledged as the original source.

            RExPO23
            2
            Stockholm, Sweden
            25-26 October 2023
            History
            Product

            REPO4EU

            Categories

            Data sharing not applicable to this article as no datasets were generated or analysed during the current study.
            Pharmacology & Pharmaceutical medicine
            Drug repurposing,Rare diseases,Guidebook,Academics,SMEs,Patient-led repurposing,Regulatory processes

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