Anjali Nagpal1, Austin G Milton2,3, Simon A Koblar1,4, M Anne Hamilton-Bruce5,6,7. 1. Stroke Research Programme, Adelaide Medical School, University of Adelaide, Adelaide, South Australia, 5005, Australia. 2. Stroke Research Programme, The Queen Elizabeth Hospital, Woodville South, South Australia, 5011, Australia. 3. Royal Adelaide Hospital, Central Adelaide Local Health Network (CALHN), Adelaide, South Australia, 5000, Australia. 4. Stroke Research Programme, Neurology, Central Adelaide Local Health Network, Adelaide, South Australia, Australia. 5. Stroke Research Programme, Adelaide Medical School, University of Adelaide, Adelaide, South Australia, 5005, Australia. anne.hamilton-bruce@sa.gov.au. 6. Stroke Research Programme, Neurology, Central Adelaide Local Health Network, Adelaide, South Australia, Australia. anne.hamilton-bruce@sa.gov.au. 7. Stroke Research Programme, Neurology 5C, The Queen Elizabeth Hospital, Central Adelaide Local Health Network, Woodville South, South Australia, 5011, Australia. anne.hamilton-bruce@sa.gov.au.
Abstract
BACKGROUND: Cell 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. METHODS: We 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. RESULTS: The 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. CONCLUSIONS: The 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.
BACKGROUND: Cell 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. METHODS: We 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. RESULTS: The 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. CONCLUSIONS: The 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.
Entities:
Keywords:
Cell therapies in stroke; Checklist; Clinical translation; Health economic; Patient and Public Involvement in Research (PPIR); Regulatory; Study design
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