Digital Technology in Somatic and Gene Therapy Trials of Pediatric Patients With Ocular Diseases: Protocol for a Scoping Review

Edward Meinert; Abrar Alturkistani; Tasnime Osama; Celine-Lea Halioua-Haubold; Josip Car; Azeem Majeed; Glenn Wells; Robert E MacLaren; David Brindley

doi:10.2196/10705

Digital Technology in Somatic and Gene Therapy Trials of Pediatric Patients With Ocular Diseases: Protocol for a Scoping Review

JMIR Res Protoc. 2019 Feb 7;8(2):e10705. doi: 10.2196/10705.

Authors

Edward Meinert^{1

2}, Abrar Alturkistani¹, Tasnime Osama¹, Celine-Lea Halioua-Haubold³, Josip Car¹, Azeem Majeed⁴, Glenn Wells⁵, Robert E MacLaren³, David Brindley⁶

Affiliations

¹ Global Digital Health Unit, Department of Primary Care and Public Health, School of Public Health, Imperial College London, London, United Kingdom.
² Healthcare Translation Research Group, Department of Paediatrics, University of Oxford, Oxford, United Kingdom.
³ Clinical Ophthalmology Research Group, Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, United Kingdom.
⁴ Department of Primary Care and Public Health, School of Public Health, Imperial College London, London, United Kingdom.
⁵ Oxford Academic Health Science Centre, Oxford, United Kingdom.
⁶ Healthcare Translation Group, Department of Paediatrics, University of Oxford, Oxford, United Kingdom.

PMID: 30730295
PMCID: PMC6383115
DOI: 10.2196/10705

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 personalized medicine is increasing and the required molecular tools are available, its adoption within pediatric ophthalmology remains to be maximized in the postgenomic era.

Objective: The objective of our study was to address the individual hurdles encountered in the field of genomic-related clinical trials and facilitate the uptake of personalized 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 pediatric patients with ocular diseases.

Methods: This paper aims to present an outline for Healthcare Information Technology and Information and Communication Technology 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 pediatric 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 2019.

Conclusions: Based on review findings, the authors will summarize methods used for facilitating IT integration in personalized medicine. Additionally, it will identify further research gaps and determine whether conducting further reviews will be beneficial.

International registered report identifier (irrid): PRR1-10.2196/10705.

Keywords: clinical trial; gene therapy; genomics; health care; personalized medicine.

©Edward Meinert, Abrar Alturkistani, Tasnime Osama, Celine-Lea Halioua-Haubold, Josip Car, Azeem Majeed, Glenn Wells, Robert E MacLaren, David Brindley. Originally published in JMIR Research Protocols (http://www.researchprotocols.org), 07.02.2019.