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Non-Invasive Quantitative Magnetic Resonance for Obese Children – the Paediatric LiverMultiScan Test

Periodic Reporting for period 1 - Paediatric LMS (Non-Invasive Quantitative Magnetic Resonance for Obese Children – the Paediatric LiverMultiScan Test)

Reporting period: 2015-06-01 to 2015-10-31

The most serious pandemic facing Europe is fatty liver disease and non-alcoholic steatohepatitis (NASH). The main causal factor is obesity: according to recent statistics (Lancet. 2014 Aug 30;384[9945]:766-81) almost 14M European children are overweight, of which 5M are obese. This is a time bomb with regard to future healthcare resource implications, since obese children tend to impose a disproportionate burden on healthcare systems throughout their adult lives. Paediatric liver diseases are mainly asymptomatic and their diagnosis is based on either incidental observation, blood test values, or on ultrasound examination due to non-specific abdominal pain. Current techniques for the diagnosis and monitoring of liver disease are either insensitive (blood tests, ultrasound) or invasive, risky, costly and not necessarily representative (biopsy from a tiny fraction of liver).

Thus, there is an urgent need to develop a test that can both assess liver disease at early stage and is applicable for 43 million obese children worldwide.

The ultimate goal of this project was to lay the groundwork for developing a non-invasive MRI method to diagnose and monitor paediatric liver disease.
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.
It became clear during the Phase 1 project that there is a need for further, substantial development of the product for paediatric liver disease. In its present form the paediatric study carries too significant a risk, and that the further development time required exceeds the horizon for the SME Instrument.

We believe this work is still extremely important and therefore the project has been recast into a EUROSTARS proposal. This funding stream better suits the technological development level of the paediatric product.

It also became clear during the project that there is a need for substantial commercial development for the existing adult LiverMultiScan product.

We will, therefore, re-purpose the SME Instrument to target a health economic analysis of using adult LiverMultiScan in fatty liver care pathways in multiple European countries.