Professors Manuel Romero Gómez and Jörn M. Schattenberg, leading experts in liver disease and members of the HCD Economics Expert Reference Group for the GAIN study (Global Assessment of the Impact of NASH (Non-Alcoholic SteatoHepatitis), a socio-economic study), addressed a global audience of over 30 delegates in a webinar intended to share the design and results of the GAIN study (published in 2020 in JHEP Reports the companion Journal to the highly respected Journal of Hepatology) and provide a forum for a live Q and A session, open to all webinar delegates. In his opening remarks Manuel confirmed that GAIN was part of ‘the main game’ to improve the outcome of NASH patients.

Manuel opened the webinar, stating the importance of increasing awareness of the burden of NASH and the objectives of GAIN, to study the social face of NASH. Specifically, to contribute to the understanding of the global impact of NASH, by observing the socio-economic and health related quality of life burden and the clinical economic demographics of this growing epidemic. A recording of this event is available now.

Described by Jörn as an ambitious, multi-disciplinary study, he confirmed that GAIN was a collaborative effort by experts and (‘this is very important’) patient representatives. Manuel confirmed the strict recruitment criteria (as defined by the Expert Reference Group) and explained that cost categories were collected from participating physicians and patients. The study was conducted in Europe and the US and recruited an impressive large number of patients - 3754 patients, of which 767 contributed patient and public involvement and engagement data (patient reported health related quality of life and self-reported health economic data). The sample included early and advanced disease, with probable NASH diagnosis being confirmed by biopsy (43% of the sample) or non-invasive methods.

Jörn confirmed, that when compared to other large studies, the GAIN population felt, in his opinion, representative. The results replicate earlier findings, reporting more impaired quality of life in advanced disease, drawing upon data from the generic HRQoL instrument EQ-5D-5L and the disease specific CLDQ-NAFLD instrument. The results are clinically meaningful differences, thus are valuable to inform understanding of the NASH landscape, to contribute to a better outcome in the future for NASH patients.

Questions occupied half of the available time, including that the application of the data in informing the payer landscape was confirmed, within a recent ICER report (full reference available in the recording). Optimism for future management to include drugs currently in development was confirmed, Jörn stated ‘the disease burden is real and these patients have impaired quality of life’ – Manuel commented ‘new drugs could totally change the treatment landscape’.

The context of COVID-19 in this population was discussed, although not reported in GAIN which was conducted before the pandemic, the experts shared their experience from clinic and the impact both had seen on outreach work amongst primary care to improve NASH diagnosis and NASH research generally, now compromised because of the overwhelming COVID-19 related demand in primary and secondary care.

In response to a question, HCD Economics confirmed their intention to undertake a longitudinal socio-economic study in NASH in 2021 (GAIN II). This will be one of a number of studies being designed by HCD Economics as part of the organisations commitment to liver disease, including rare liver diseases. The crucial role and valued partnership of ERG members in the pursuit of these research objectives was confirmed in the concluding remarks – which included the following statement from Manuel ‘congratulations for this work’

For more information on the GAIN study, and the research plans in liver disease including rare liver disease, please contact Alison Rose, RWE Director, HCD Economics –