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Technology Considerations for Enabling eSource in Clinical Research: Industry Perspective.

ABSTRACT: BACKGROUND:The technological complexities and broad operational scope of eSource impede coordinated, inter-organizational action on advancing at-scale solutions. METHODS:We introduce an architectural framework for articulating technological considerations across organizations. The architecture neither implies nor endorses solution implementations; rather, it proposes solution functionality based upon principles and good clinical practices. RESULTS:Key technology considerations include patterns of anticipated use, implications to the current state of clinical trial operations, and the need for new technologies (i.e., IoT, Big Data, Predictive Analytics). CONCLUSION:Technology considerations drive implications beyond technology-influencing regulatory, process, and ethical realms of clinical research.

SUBMITTER: Jennings DG

PROVIDER: S-EPMC7458892 | biostudies-literature | 2020 Sep

REPOSITORIES: biostudies-literature

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Technology Considerations for Enabling eSource in Clinical Research: Industry Perspective.

Jennings Donald G DG Nordo Amy A Vattikola Aruna A Kjaer Jesper J

Therapeutic innovation & regulatory science 20200311 5

<h4>Background</h4>The technological complexities and broad operational scope of eSource impede coordinated, inter-organizational action on advancing at-scale solutions.<h4>Methods</h4>We introduce an architectural framework for articulating technological considerations across organizations. The architecture neither implies nor endorses solution implementations; rather, it proposes solution functionality based upon principles and good clinical practices.<h4>Results</h4>Key technology considerati ...[more]

PMID: 32865798

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Project description:Despite the development of novel pharmacological treatments, cardiovascular disease morbidity and mortality remain high indicating an unmet clinical need. Viral gene therapy enables targeted delivery of therapeutic transgenes and represents an attractive platform for tackling acquired and inherited cardiovascular diseases in the future. Current cardiovascular gene therapy trials in humans mainly focus on improving cardiac angiogenesis and function. Encouragingly, local delivery of therapeutic transgenes utilising first-generation human adenovirus serotype (HAd)-5 is safe in the short term and has shown some efficacy in drug refractory angina pectoris and heart failure with reduced ejection fraction. Despite this success, systemic delivery of therapeutic HAd-5 vectors targeting cardiovascular tissues and internal organs is limited by negligible gene transfer to target cells, elimination by the immune system, liver sequestration, off-target effects, and episomal degradation. To circumvent these barriers, cardiovascular gene therapy research has focused on determining the safety and efficacy of rare alternative serotypes and/or genetically engineered adenoviral capsid protein-modified vectors following local or systemic delivery. Pre-clinical studies have identified several vectors including HAd-11, HAd-35, and HAd-20-42-42 as promising platforms for local and systemic targeting of vascular endothelial and smooth muscle cells. In the past, clinical gene therapy trials were often restricted by limited scale-up capabilities of gene therapy medicinal products (GTMPs) and lack of regulatory guidance. However, significant improvement of industrial GTMP scale-up and purification, development of novel producer cell lines, and issuing of GTMP regulatory guidance by national regulatory health agencies have addressed many of these challenges, creating a more robust framework for future adenoviral-based cardiovascular gene therapy. In addition, this has enabled the mass roll out of adenovirus vector-based COVID-19 vaccines. KEY MESSAGES: First-generation HAd-5 vectors are widely used in cardiovascular gene therapy. HAd-5-based gene therapy was shown to lead to cardiac angiogenesis and improved function. Novel HAd vectors may represent promising transgene carriers for systemic delivery. Novel methods allow industrial scale-up of rare/genetically altered Ad serotypes. National regulatory health agencies have issued guidance on GMP for GTMPs.

Dataset Information

Technology Considerations for Enabling eSource in Clinical Research: Industry Perspective.

Publications

Technology Considerations for Enabling eSource in Clinical Research: Industry Perspective.

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