Abstract
BackgroundCell therapies present an exciting potential but there is a long history of expensive translational failures in stroke research. Researchers engaged in cell therapy research would benefit from a practical framework that can help in planning research and development of investigational cell therapies into viable medical products.MethodsWe developed a checklist using a mixed methodology approach to evaluate the impact of study design, regulatory policy, ethical, and health economic considerations for efficient implementation of early phase cell therapy studies.ResultsThe checklist comprises a series of questions arranged under four domains: the first concerns study design such as characterization of target study population, trial design, endpoints and operational fit of dosage, time, and route of administration to target populations. A second domain addresses the data package required for regulatory approval relevant to the intended use (allogeneic/autologous; homologous/non-homologous; nature of cell processing). The third domain comprises patient involvement to ensure relevant data is collected via targeted study design. The final domain requires the team to determine the critical data elements that could be built into study design to enable health economic data collection to be started at an early phase of the study.ConclusionsThe CT2S checklist can help to determine areas of expertise gaps and enable research groups to appropriately allocate resources for capacity building. Use of this checklist will allow identification of key areas where trial planning needs to be optimized, as well as helping to identify resources that need to be secured. The CT2S checklist can also serve as a general cell therapy research decision aid to improve research output and accelerate new cell therapy development.
Highlights
Cell therapies present an exciting potential but there is a long history of expensive translational failures in stroke research
Groups engaged in cell therapy research would benefit from an expanded and practical framework that can help accelerate research and development of investigational cell therapies (CTs) into a viable medical product
They were integrated into a practical framework in checklist form organized under four domains: study design, regulatory, ethical, and health economic considerations, proposed as a tool for research teams to efficiently plan operationalization of early clinical studies of cell therapy
Summary
Cell therapies present an exciting potential but there is a long history of expensive translational failures in stroke research. The landmark success in the clinical translation of reperfusion strategies in stroke, thrombolysis and endovascular thrombectomy, represents significant mortality and morbidity benefit [1]. These therapies are limited by a narrow window of opportunity [1, 2]. Groups engaged in cell therapy research would benefit from an expanded and practical framework that can help accelerate research and development of investigational CTs into a viable medical product
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