A new global collaboration has been formed to utilise clinical trial data standards for VL to optimise and harmonise medical product research and ultimately reduce the burden of the disease. At a meeting in February in New Delhi, India, co-organised by the Infectious Diseases Data Observatory (IDDO) and the Drugs for Neglected Diseases Initiative (DNDi), senior scientists from institutions around the world involved in VL agreed to work together to create a freely available guidance on applying CDISC data standards to VL data.
Required by the United States Food and Drug Administration (FDA) and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA), recommended by the China National Medical Products Administration (NMPA) and adopted by the world’s leading research organisations, CDISC standards enable the entire research community to maximise the value of data for more efficient and meaningful research that has invaluable impact on global health.
In addition to IDDO and DNDi scientists, researchers from Kala-Azar Medical Research Centre (KAMRC), Rajendra Memorial Research Institute of Medical Sciences (RMRI), Indian Council for Medical Research (ICMR), Novartis, the European Medicines Agency (EMA), Médecins Sans Frontières (MSF), Institute of Postgraduate Medical Education & Research (IPGMER), World Health Organization (WHO), PATH India, University of Gondar, global health research institute International Centre for Diarrhoeal Disease Research (icddr,b,) University of Khartoum, and the University of Brasilia as well as from CDISC.
Currently there are no agreed upon standards for VL and different terminology is used across different countries. This collaboration, involving representatives from across the VL research community, aims to set guidelines that will benefit current and future research into the disease.
As a next step, Working Groups have been set up to agree upon standard parameters for variables, such as biomarkers, core outcome measures, HIV, and signs and symptoms. This work will be collated into a VL case record form (CRF), which will be annotated using CDISC standards. Later this year, this work will be used in a clinical trial run by DNDi.
Professor Shyam Sundar, Programme Director at the Kala-Azar Medical Research Centre, said: “This is an important collaboration involving the VL research community in affected areas and we will be working closely together to create data standards. Once these have been implemented, they will available for use by the VL research community to enable the consistent collection of data. This will have a major impact on improving research and ultimately, on reducing the burden of this neglected poverty-related disease.”
Dr Dinesh Mondal, Senior Scientist at icddr,b, said: “The purpose of creating standards is to give guidance of what to collect, so the Working Groups will focus on agreeing key variables to help ensure the necessary data is collected on the ground. This will be a live standard, so that we can continue to incorporate future developments on data captured in future trials.”
Dr Gustavo Romero, Principal Investigator, University of Brasilia, said: “There’s a lack of standards for VL and it’s good to have CDISC’s support and guidance with this initiative. Regulators are keen to see an accepted set of research community standards rather than many different parties producing their own unique versions and this collaboration can really push that work forward.”
Dr Sakib Burza, Medical Advisor Asia, MSF, said: “This sort of adaptive and consensus based-initiative is quite important in accelerating the process of data generation through to drug delivery and approval on the field. With falling numbers of cases of VL, particularly in Asia, yet a strong need for better treatments remaining, this consolidative and collaborative approach is very timely. In particular, standardising approaches to the rarer manifestations of VL, such as VL-HIV infection, will be critical in maximising the utility of evidence generated through clinical trials.’’
The guidance will also be applied to data already submitted to IDDO to enable retrospective, consistent use of the CDISC standards. Through collaborations with research partners, IDDO’s VL platform now holds almost a fifth of the world’s individual patient-level data (IPD) from clinical trials. Thirty-two studies have already been shared with the platform, representing over 7,000 IPD that will be standardised and curated by IDDO’s data team using the new data standards. This work will facilitate re-use for detailed analysis to generate evidence on the safety and efficacy of existing medicines, inform the development of new treatments, and advance understanding of the disease.
The VL data have been identified by systematic review, updated twice each year by IDDO’s Science team. IDDO’s work with the existing data from VL clinical trials and the VL research community has led to its involvement in the initiative to create a data standard for VL.
The VL data repository forms part of the wider IDDO infrastructure, which includes scientific networks, technical and governance infrastructure, standards development and advocacy for evidence-based treatment, and capacity building.
The VL Scientific Advisory Committee (SAC) is currently working with the VL community to prioritise research questions that could be addressed using the assembled data.
Find out more about Contributing Data to the VL Data Platform