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.
Recommended instruments for adult populations were identified through an evidence-informed psychometric review evaluating validity, reliability, and responsiveness across candidate PROMs (see Rahib, Basadre, and Palermo, 2026). Recommended instruments for pediatric populations were identified by a Pediatric Working Committee — comprising pediatric gastroenterologists, a pediatric pain psychologist, and patient/parent and nonprofit stakeholders — through a structured review of validated instruments spanning the full pediatric age range.
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
Pediatric
Numerical Rating Scale (0–10 NRS)
Age range: 8–18
Abdominal Pain Index (API) – Child test
Abdominal Pain Index (API) – Child
Age range: 7-17
Faces Pain Scale – Revised (FPS-R)
Age range: 4-16
FLACC (Face, Legs, Activity, Cry, Consolability)
Age range: 0-7
Pain Interference with Daily Living
How much pain interferes with engagement in social, physical, and recreational activities
Pediatric
FDI (Functional Disability Inventory)
Age range: 7-17
CALI-9 (Child’s Activity Limitations Interview)
Age range: 8-18 (self-report or parent-proxy)
PROMIS Pediatric Pain Interference Short Form 8a
Age range: 8-17 (self-report) or 5-17 (parent-proxy)
Pain Interference with Daily Living
How much pain interferes with engagement in social, physical, and recreational activities
Pediatric
FDI (Functional Disability Inventory)
Age range: 7-17
CALI-9 (Child’s Activity Limitations Interview)
Age range: 8-18 (self-report or parent-proxy)
PROMIS Pediatric Pain Interference Short Form 8a
Age range: 8-17 (self-report) or 5-17 (parent-proxy)
Ability to Participate in Social Roles & Activities
Being able to do regular activities, go to school, spend time with friends, and/or work.
Pediatric
PedsQL 4.0 (Pediatric Quality of Life Inventory)
Age range: 5-18 (self-report) or 2-18 (parent-proxy)
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).
Pediatric
PGIC (Patient Global Impression of Change)
Age range: 12-17 (self-report) or <12 (parent-proxy)
*Added based on expert clinician recommendation; not included in the formal Delphi/OMERACT core outcome set development process.
Mandatory Domains
Not measured through Patient Reported Outcome Measurements (PROMs)
Disease Sequelae
Long-term disease related consequences or effects.
Exocrine pancreatic insufficiency, diabetes
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
Important But Optional Domains
Measured through Patient Reported Outcome Measurements (PROMs)
Diabetes Management
Keeping blood sugar in a healthy range and manage pancreatitis-related diabetes (e.g., type 3c), including the burden of monitoring and treatment and episodes of high or low blood sugar.
Age range: 5-18 (self report) or 2-18 (parent proxy)
Diabetes Quality of Life for Youth (DQOLY)
Age range: 10-18 (self report)
Diabetes Distress Scale (DDS17)
Age range: ≥18 (self report)
Opioids
Use of opioid pain medicines to manage pancreatitis pain.
Mental Health
Emotional and psychological well-being, including symptoms such as anxiety and depression and how pancreatitis affects mood, stress, and coping.
PROMIS Pediatric Depressive Symptoms
Age range: 8-17 (self report) or 5-17 (parent proxy)
Age range: 8-17 (self report) or 5-17 (parent proxy)
Ability to Eat
Being able to eat and tolerate food without triggering or worsening symptoms (e.g., pain, nausea, vomiting, early fullness) and without needing to avoid meals or restrict diet excessively.
About Your Child’s Eating (AYCE) questionnaire
Age range: 8-16 (parent proxy)
Age Ranges, Translations, and Access
This table summarizes the instruments referenced above, including recommended age ranges, known translation availability, and where to go to access or request licensing for the official instrument materials. Because licensing terms and available language versions can change, please use the “Next step” link for each instrument for the most up-to-date information on access, permissions, and translations.
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.
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