Clinical Trial Endpoints
Well-defined endpoints that capture patient-reported outcomes are critical in advancing care in recurrent acute (RAP) and chronic pancreatitis (CP). Mission: Cure worked with patients and providers to identify clinically meaningful outcomes that should be measured in future pancreatitis trials. We encourage researchers planning clinical studies to incorporate the following endpoints. For details on the methodology used, see the summary below.
Delphi Process Overview
To identify the most important outcomes, we conducted a two-round Delphi consensus survey with four stakeholder groups: adult patients, pediatric patients/parents, adult care providers, and pediatric care providers. Through repeated rounds of rating and feedback, participants helped prioritize and refine potential outcomes.
This process followed the OMERACT framework, an internationally recognized approach for creating standardized, consensus-based outcome measures. Using OMERACT helps ensure that research outcomes are meaningful, practical to measure, and reflect both patient and clinical priorities.
Identifying the Core Outcome Set
After completing the Delphi survey, the steering committee reviewed and organized the prioritized outcomes within the OMERACT framework.
The OMERACT "Onion": Organization of Domains
The OMERACT (Outcome Measures in Rheumatology) framework is an internationally recognized approach for creating standardized, consensus-based outcome measures. Using OMERACT helps ensure that research outcomes are meaningful, practical, consistent to measure, and reflect both patient and clinical priorities.
The committee categorized each outcome as:
Mandatory domains in all trials
Mandatory in specific trials
Important but optional
Research agenda domains
These categories form the foundation of the Core Outcome Set for Recurrent Acute and Chronic Pancreatitis, guiding consistent and patient-centered outcome measurement across future studies.
Core Outcomes Assessment and Measures in Clinical Trials
To promote consistency across future studies, recommended approaches for measurement have been defined for core outcome domains for recurrent acute and chronic pancreatitis. These outcomes are grouped by whether they are assessed through patient-reported outcome measures (PROMs) or non–patient-reported assessments.
- Patient-reported outcome measures (PROMs): capture patients’ own experiences, such as pain severity or social participation, using standardized questionnaires or rating scales.
- Non–patient-reported measures: rely on clinical or laboratory assessments to evaluate outcomes such as disease progression or hospitalization
The tables below list the mandatory domains for all clinical trials in recurrent acute and chronic pancreatitis, along with their definitions and the recommended measurement tools for adults and children.
Mandatory Domains
Measured through Patient Reported Outcome Measurements (PROMs)
Pain Severity
Perception of the severity of pain, including how intense the pain is and how frequently it occurs
Pain Interference with Daily Living
How much pain interferes with engagement in social, physical, and recreational activities
Ability to Participate in Social Roles & Activities
Being able to do regular activities, go to school, spend time with friends, and/or work.
Pancreatic Enzyme Insufficiency
Not being able to take in adequate nutrition or manage Exocrine Pancreatic Insufficiency (EPI) to support health and weight, and/or not growing properly.
Pain Treatment Satisfaction​
How much a person thinks their pain has improved from the treatment (symptoms are better).
Mandatory Domains
Not measured through Patient Reported Outcome Measurements (PROMs)
Disease Sequelae
Long-term disease related consequences or effects
ER Visits/Hospitalization
Needing to visit the ER or being hospitalized due to pancreatitis-related problems.
AP Attacks/Flares
Having attacks of pancreas-related abdominal pain lasting for several hours or days.
Time to first adjudicated flare, reduction in flare frequency over a defined treatment period, average flare duration, proportion of patients flare-free during treatment
See Mission: Cure memo on flares as a clinical trial endpoint
References
- Palermo TM, Li R, Birnie KA, Crombez G, Eccleston C, KashikarZuck S, et al. Updated recommendations on measures for clinical trials in pediatric chronic pain: a multiphase approach from the Core Outcomes in Pediatric Persistent Pain (CoreOPPP) Workgroup. PAIN. 2024;165(5).
- Li R, Gibler RC, Rheel E, Slack K, Palermo TM. Recommendations for Patient-Reported Outcomes Measurement Information System pediatric measures in youth with chronic pain: a COnsensus-based Standards for the selection of health Measurement INstruments systematic review of measurement properties. PAIN. 2024 Feb 1;165(2):258–95.
Other Resources
Preclinical Models
Here, researchers and drug developers can find preclinical pancreatitis models. Each category of pre-clinical model has advantages and disadvantages that should be considered when designing pre-clinical studies.
Share A Research Opportunity
We provide services to researchers in order to support the development of effective therapies and treatments for recurrent acute pancreatitis and chronic pancreatitis. Click here to learn more, or share your research opportunity by clicking the button below.