The Symptom Assessment Project: Building New Endpoints for Clinical Trials

Quotes from interviews of patients living with PSC describing their fatigue, presented as a poster at AASLD's The Liver Meeting 2024.

Patient-reported outcomes (PROs) are used as clinical trial endpoints* for some diseases, but the Food and Drug Administration (FDA) has not yet approved any PRO assessment tools for establishing the effectiveness of a new PSC treatment.

While some PRO tools exist for PSC, they do not meet the FDA’s standards. PSC Partners is leading the Symptom Assessment Project (SAP), an initiative whose goal is to develop FDA-approved PRO tools for three common PSC symptoms that impact quality of life – fatigue, brain fog (cognitive impairment), and pain around the liver.

Lead investigators for the project are Donna Evon, Ph.D., Duke University, and Bryce Reeve, Ph.D., University of North Carolina – Chapel Hill. They are working to create three separate validated questionnaires about the above symptoms. The multi-stage validation process ensures that the assessment tool accurately captures the intended information and is fit for its intended purpose.

“The Symptom Assessment Project is important to the PSC community,” says Dr. Evon, “because [the questionnaires] will capture experiences that can be shared with healthcare providers who may be able to help, and, ultimately, these measures will be used to evaluate the benefits of new treatments for PSC.”

Work on this tool began in 2022 with the input of focus group volunteers, who all belong to the Patient Registry and have personal experience with common PSC symptoms. Group participants helped to identify and rank the symptoms that most affected their quality of life, and they reviewed the PRO tools that were currently being used for PSC.

The Symptom Assessment Project is nearly finished developing the three questionnaires (fatigue, brain fog, and pain around the liver). These tools will become available in a Registry survey later this year. Obtaining real-world data from a large group of people living with PSC (including those in the Registry) is a key step in development of these tools to support their ultimate use in a clinical trial setting.

Drs. Evon and Reeve published an article in the March 2025 edition of Current Opinions in Gastroenterology that gives an overview of FDA guidance regarding PRO tools; reviews existing PRO tools; highlights how the new SAP questionnaires will adhere to FDA guidance; and encourages collaborations among academic, industry, and patient advocacy organizations in the design of these new tools.

The article abstract is available here.

*Endpoint: a measurable outcome used to determine the effect of a treatment in a clinical trial.