Over the past 5 months on this Phase 1 grant, we have established the feasibility of non-invasive quantitative Magnetic Resonance Imaging for obese children – the Paediatric LiverMultiScan test.
Towards Objective 1, we identified a top clinical trials unit and statistician: Dr Rebollo Mesa, Senior Lecturer in Trials, King’s Clinical Trials Unit, Biostatistics Department, Kings College London; and as clinical lead investigator, Professor Dhawan, a world authority on paediatric liver disease. We established a network of five leading clinical study sites in four EU countries, i.e. children’s hospitals in London, Birmingham, Hannover, Warsaw and Rome. We obtained statistics on the numbers of liver biopsies performed in children, ensuring that sufficient patient recruitment at each site is possible. Together with the study investigators we developed a detailed study design and power calculations, aiming to assess, over two years, 250 children with suspected chronic liver disease referred for liver biopsy, and to compare biopsy with LiverMultiScan results. We established the technical feasibility of MRI measurements at each site, ensuring the available MRI equipment and expertise is suitable for the trial and that data obtained are comparable across sites. We precisely costed the phase 2 clinical trial at £1,740,044.
For Objective 2, we analysed demand and user base and obtained precise estimates of market size and growth rates, as well as the economic benefit to users. This shows that the Total Accessible Market will grow to 16M people by year 5 of the project, leading to projected annual revenues for Perspectum Diagnostics of 13.5M€, and (subject to final decisions on pricing) substantial EBITDA. We performed a detailed competitor analysis and comparison including developing proposals for work flow that potentially integrate our quantitative MRI approach and Fibroscan, showing that our test has clear advantages over all existing competitors in terms of ability to detect early disease, able to assess even the most obese subjects, and ability to provide high throughput at relatively low cost without additional hardware requirements.
