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April 1, 2026JCO oncology advances.Open Access

Balancing Rigor and Urgency: The Development of Trastuzumab Deruxtecan in Rare and Ultra-Rare Sarcomas

Damon R. Reed Michael V. Ortiz Laney Berman Andrew L. Kung Julia Glade Bender Emily K. Slotkin

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Key Points

This research discusses the challenges of developing trastuzumab deruxtecan for rare and ultra-rare sarcomas, focusing on overcoming barriers to clinical trial completion.
Analysis of current frameworks for clinical trial access and drug development in rare diseases.
Discussion on off-label use of trastuzumab deruxtecan in osteosarcoma and desmoplastic small round cell tumor.
Evaluation of stakeholders' roles in drug approval and accessibility.
Identified significant barriers in drug development for rare sarcomas, including low commercial incentive and fragmented expertise.
Off-label use of trastuzumab deruxtecan can enhance access to investigational drugs while formal trials are being designed.
Collaborative frameworks among stakeholders are necessary to expedite translation of promising therapies.

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Implication

Randomized trial investigates trastuzumab deruxtecan's effectiveness in rare sarcomas, suggesting improved patient access to therapies.

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Cite This Study

Reed et al. (2026) studied this question.

synapsesocial.com/papers/6a2e44e4b1cc60ccdea8a52a https://doi.org/10.1200/oa-26-00042

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