The Patient Perspectives on Future Therapeutic Options in NASH and Patient Needs

Abstract

Background: Non-alcoholic steatohepatitis (NASH) is a chronic liver disease with severe complications and without approved therapies. Currently, there is limited data on the overall burden of the disease for patients or on patient needs and preferences. This study investigates patient preferences in relation to potential future therapies for NASH. In addition, the factors that are relevant to patients and their importance in relation to future treatment options are explored. Method: Telephone in-depth interviews (TDIs) preceded an online 30-min quantitative survey. The online survey included (1) multiple choice questions (MCQs) on NASH diagnosis and disease background. (2) An exercise to determine patients' satisfaction levels with information provided at diagnosis, and to explore symptomatology in detail. (3) Exercises to evaluate potential new products and product attributes, including a "drag and drop" ranking exercise, and an adaptive choice-based conjoint exercise (ACBC). (4) The EQ-5D-5L questionnaire and the Visual Analog Scale (VAS), which measures patients' health status. (5) Collection of socio-demographic data, and (6) Questions to measure patient satisfaction with the survey. Results: There were 166 patients included in this study from Canada [n = 36], Germany [n = 50], the UK [n = 30], and USA [n = 50]. Fifty seven percent of patients [n = 94] had had a liver biopsy for confirmation of NASH. Patients were often unable to link their symptoms to NASH or other conditions. ACBC results showed that efficacy, defined as "impact on liver status" was the single most important attribute of a potential future NASH therapy. Other attributes considered to have secondary importance included impact on weight, symptom control and the presence of side effects. The EQ-5D utility score was 0.81 and VAS = 67.2. Conclusion: "Impact on liver status" is the primary outcome sought. Patients demonstrate a general lack of understanding of their disease and appeared to be unfamiliar with longer-term consequences of NASH. It is necessary to improve patient understanding of NASH and its progressive nature, and there is a need for improving confirmatory diagnosis and monitoring.

Keywords: EQ5D-5L; adaptive choice-based conjoint; liver disease; non-alcoholic fatty liver disease (NAFLD); non-alcoholic steatohepatitis (NASH); patient preference; patient-based evidence; patient-reported outcomes.

Figure 1

Methodology summary.

Figure 2

Overview of clinically confirmed or suspected NASH diagnosis across countries.

Figure 3

Patient satisfaction with information provided about NASH by their physician on diagnosis.

Figure 4

Overall reported symptoms of NASH patients across the four countries of this survey.

Figure 5

Overall summary of analysis of symptoms attributed to NASH. Patients were asked to indicate which symptoms they are experiencing, and then asked to indicate what was causing their symptoms.

Figure 6

(A) Importance scores of a hypothetical product attributes as seen by patients. The total importance score is out of 100, if an attribute of a profile receives a score of 50 that means that half of all the importance of a profile is allocated to this attribute. (B) Summary of simulated patient preference based on the patient ACBC exercise responses.

Figure 7

Overall summary of EQ-5D data across all countries.

Figure 8

Overall results of survey experience across all four countries.