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Review
. 2020 Jul;245(13):1155-1162.
doi: 10.1177/1535370220924743. Epub 2020 May 12.

Tackling rare diseases: Clinical trials on chips

Sandra H Blumenrath  1 , Bo Y Lee  2 , Lucie Low  3 , Ranjini Prithviraj  1 , Danilo Tagle  3
Affiliations
Review

Tackling rare diseases: Clinical trials on chips

Sandra H Blumenrath et al. Exp Biol Med (Maywood). 2020 Jul.

Abstract

Designing and conducting clinical trials are extremely difficult in rare diseases. Adapting tissue chips for rare disease therapy development is pivotal in assuring that treatments are available, especially for severe diseases that are difficult to treat. Thus far, the NCATS-led National Institutes of Health (NIH) Tissue Chip program has focused on deploying the technology towards in vitro tools for safety and efficacy assessments of therapeutics. However, exploring the feasibility and best possible approach to expanding this focus towards the development phase of therapeutics is critical to moving the field of CToCs forward and increasing confidence with the use of tissue chips. The working group of stakeholders and experts convened by NCATS and the Drug Information Association (DIA) addresses important questions related to disease setting, test agents, study design, data collection, benefit/risk, and stakeholder engagement-exploring both current and future best use cases and important prerequisites for progress in this area.

Keywords: Tissue chips; clinical trials on chips; microphysiological systems; rare diseases; therapy development.

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References

    1. Ronaldson-Bouchard K, Vunjak-Novakovic G. Organs-on-a-chip: a fast track for engineered human tissues in drug development. Cell Stem Cell 2018; 22:310–24 - PMC - PubMed
    1. Park SE, Georgescu A, Huh D. Organoids-on-a-chip. Science 2019; 364:960–5 - PMC - PubMed
    1. US Food and Drug Administration. Rare diseases: common issues in drug development. Guidance for industry, www.fda.gov/media/120091/download (2019, accessed 15 January 2020)
    1. US Food and Drug Administration. Human gene therapy for rare diseases. Draft guidance for industry, www.fda.gov/media/113807/download (2018, accessed 15 January 2020)
    1. Nony P, Kurbatova P, Bajard A, Malik S, Castellan C, Chabaud S, Volpert V, Eymard N, Kassai B, Cornu C, CRESim, Epi-CRESim. A methodological framework for drug development in rare diseases. Orphanet J Rare Dis 2014; 9:164. - PMC - PubMed

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