Titlebook: Innovative Designs and Analyses for Small Population Clinical Trials; Development Strategi Jingjing Ye,Lei Nie Book 2024 The Editor(s) (if

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The Case Study of NCI-COG Pediatric MATCH Trial,various age groups presents a significant challenge for drug development program as compared to those for adults. Furthermore, modern targeted and precision cancer drug development typically are only effective in a subtype of disease, which further shrinks the targeted patient population. As a resul

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Non-Oncology Case Study of Lonafarnib,roteins like progerin. Lonafarnib, a farnesyltransferase inhibitor, emerged as a potential treatment for HGPS. This chapter outlines lonafarnib’s development program, supported by pivotal trials comparing treated patients to a natural history cohort. Utilizing the Progeria Research Foundation Intern

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Natural History, Patient Registry, and Patient Voice,ural history of the condition and the degree of unmet medical needs. The natural history studies and the patient’s voice are critical components to rare disease drug development. In recent years, there is also strong regulatory support to incorporate natural history and patient voice into rare disea

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Design and Analysis Considerations,ive and effective drugs. We start by describing regulatory and development pathways for rare disease drug developments..We attribute the issue of chance findings in clinical trials of noneffective drugs to small sample sizes. To mitigate this, we advocate for the adoption of measures such as minimum

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Crossover Design,lly significant results. These designs capitalize on repeated measurements from the same subjects under different treatments, potentially enhancing the study’s efficiency and sensitivity. However, their suitability depends on specific trial characteristics, such as disease stability and reversibilit

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Use of Real-World Data (RWD) and Real-World Evidence (RWE), of validated endpoints. To address these challenges, regulatory agencies like the FDA have introduced flexibilities in evidence requirements and provided guidance specifically aimed at rare disease drug development. This paper reviews the regulatory landscape and challenges in rare disease drug dev