Digital technology in somatic and gene therapy trials of paediatric patients with ocular diseases: a systematic scoping review protocol

Abstract

Background Pharmacogenomics suggests that diseases with similar symptomatic presentations often have varying genetic causes, affecting an individual patient’s response to a specific therapeutic strategy. Gene therapies and somatic cell therapies offer unique therapeutic pathways for ocular diseases and often depend on increased understanding of the genotype-phenotype relationship in disease presentation and progression. While demand for personalised medicine is increasing and the required molecular tools are available, its adoption within paediatric ophthalmology remains to be maximised in the post-genomic era. To address the individual hurdles encountered in the field of genomic-related clinical trials and facilitate the uptake of personalised medicine, we propose to conduct a review that will examine and identify the digital technologies used to facilitate data analysis in somatic and gene therapy trials in paediatric patients with ocular diseases. Objectives To present an outline of HIT/ICT resources used in somatic and gene therapy clinical trials in children with ocular diseases. This review will enable authors to identify challenges and provide recommendations facilitating the uptake of genetic and somatic therapies as therapeutic tools in paediatric ophthalmology. The review will also determine whether conducting a systematic review will be beneficial. Results Database searches will be initiated in September 2018. We expect to complete the review in December 2018. Conclusions Based on review findings, the authors will summarise methods used for facilitating IT integration in personalised medicine. Additionally, it will identify further research gaps and determine whether conduction of further reviews will be beneficial.