coreNASH: Multi-stakeholder Consensus on Core Outcomes for Decision Making About Nonalcoholic Steatohepatitis...

Area of Science:

• Hepatology and Gastroenterology
• Clinical Trial Design
• Health Outcomes Research

Background:

• Nonalcoholic steatohepatitis (NASH) is increasingly prevalent, necessitating standardized outcomes for treatment evaluation.
• Existing treatment decision-making processes require a prioritized set of outcomes reflecting diverse stakeholder perspectives.

Purpose of the Study:

• To develop a consensus-based core outcome set (COS) for nonalcoholic steatohepatitis (NASH) clinical trials.
• To incorporate multi-stakeholder perspectives in defining the most important outcomes for NASH treatment evaluation.

Main Methods:

• A modified Delphi consensus process involving a multi-stakeholder panel of 53 participants.
• Literature review and key informant interviews to generate an initial candidate outcome list.
• Three rounds of online voting and an in-person meeting to prioritize and finalize outcomes, with special weighting for patient opinions.

Main Results:

• The coreNASH initiative developed two distinct core outcome sets (COS): one for NASH without cirrhosis (F2-F3) and one for NASH with cirrhosis (F4).
• The panel agreed on two core outcomes for NASH without cirrhosis and nine core outcomes for NASH with cirrhosis.
• The initial list of 86 candidate outcomes was refined to a consensus-driven COS.

Conclusions:

• A consensus-based core outcome set (COS) for NASH has been established, applicable across the treatment lifecycle.
• The coreNASH COS will aid decision-making for regulatory approval, market access, and post-market evaluations.
• Incorporating the coreNASH COS into clinical development will improve treatment comparisons and value assessment for new NASH therapies.