FROM SCOPING TO SCALING: COLLABORATIVE INDIVIDUAL PATIENT DATA PLATFORMS FOR NEGLECTED INFECTIOUS DISEASES: A thesis submitted in partial fulfilment of the requirements for the degree of Doctor of Philosophy [PhD by publication]

Abstract

This thesis presents the rationale and theoretical context for a portfolio of published work examining the clinical study landscape across five different neglected and poverty-related diseases (NPRDs). It critically analyses the limitations of traditional aggregate meta-analysis for synthesising evidence for these diseases, demonstrating how challenges such as limited volume of trials and enrolled participants, methodological heterogeneity and inconsistent reporting necessitate alternative approaches. Meta-analysis of individual patient data (IPD) is proposed as an alternative; however, access to IPD raises legal, ethical, and technical barriers, which I critically examine, drawing on the experiences gained through stakeholder engagement. I engaged with the disease specific research communities to assess the feasibility of building global collaborative data platforms which can provide solutions to the many challenges of sharing IPD. The common difficulties identified in my publications across the NPRDs, together with the limited global attention, funding, and resources available, strongly reinforced the need for a unified data-sharing infrastructure to enable economies of scale. My work directly informed the evolution of the Infectious Diseases Data Observatory (IDDO) Roadmap, a framework of tools, templates, and resources to streamline platform scoping and development, reduce duplication, and adapt processes efficiently to new disease areas. I also demonstrate how systematic review outputs from my publications evolved into dynamic, openaccess tools that enhance the discoverability of IPD and promote data-sharing. I describe the development of a research community consensus-driven process for generating research agendas that enable the meaningful re-use of IPD to address critical knowledge gaps in these under-researched disease areas. My contribution helped shape and operationalise the vision of IDDO as a researcher-driven initiative to build data platforms, supporting the identification, collation, and harmonisation of IPD to promote meaningful data re-use and advance the treatment and control of neglected infectious diseases.