Michelle Skornicki, MPH, Natalie Schibell, MPH, and Rohan Shah
Inflammatory bowel disease (IBD) presents significant challenges in drug development due to its heterogeneous etiology and unpredictable disease progression. Traditional real-world data (RWD) sources—such as claims and electronic health records (EHRs)—offer valuable insights but often lack the depth needed to capture IBD's full clinical and biological complexity. Critical data elements—such as molecular biomarkers, biosamples, and patient-reported outcomes (PROs)—are essential for defining patient subgroups, characterizing disease trajectories, and assessing treatment response.
To bridge these gaps, Aetion’s experts—including data scientists, epidemiologists, and analysts—partnered with the Crohn’s & Colitis Foundation and its IBD Plexus Disease Progression Working Group to conduct research using the Aetion Evidence Platform® (AEP). This collaboration led to developing a Crohn’s disease progression model and identifying key risk factors associated with disease progression.
Transforming Data into Actionable Evidence
IBD Plexus is a comprehensive data ecosystem specifically designed for IBD research. Unlike traditional claims or EHR datasets, IBD Plexus integrates high-dimensional clinical, molecular, and patient-reported data to better understand disease progression and treatment response.
Key data elements include:
- Molecular Data: Enable biomarker-driven research, supporting precision medicine approaches and refining patient stratification for clinical trials.
- Biosamples: Provide access to high-quality biological specimens linked to clinical and molecular data, facilitating translational research.
- Electronic Case Report Forms (eCRFs): Ensure structured, consistent patient outcomes and therapeutic response tracking through standardized data collection.
- Patient-Reported Outcomes (PROs): Leverage survey question-and-answer fields to integrate direct patient insights, bridging the gap between clinical endpoints and real-world disease burden assessment.
- Laboratory Test Results: Link biomarker levels with disease activity and treatment efficacy, strengthening the evidence base for therapeutic decision-making.
IBD Plexus combines diverse data sources to generate decision-grade RWE, equipping researchers with the evidence needed to enhance clinical decision-making and strengthen regulatory and reimbursement submissions.
Leveraging Real-World Data to Define Disease Progression
Despite the progressive nature of Crohn’s Disease (CD), no standardized real-world data-based definition for disease progression currently exists, making it difficult to assess the impact of long-term treatment. Other CD progression scoring methods, such as the Lémann Index, rely on imaging or procedural data, which limits their applicability in RWE studies. The Crohn’s & Colitis Foundation, the IBD Plexus Disease Progression Working Group, and Aetion developed a progression score to address this gap, providing a clinically grounded, real-world approach to tracking disease progression. This score classifies Crohn’s Disease (CD) progression into three tiers—low, moderate, and high—representing the likelihood of progressing to a surgical outcome. It leverages linked data from the SPARC IBD and IBD Qorus cohorts, integrated with claims and laboratory data from HealthVerity, another Aetion® Data Network Partner.
Our analysis found several strong predictors of disease progression (p<0.05). Abdominal abscess, C. difficile testing, viral or fungal infection, high-dose steroid use, perianal disease, and weight loss were all associated with increased likelihood of progression. For example, each viral or fungal infection or abdominal abscess diagnosis within a patient’s current risk category increases the likelihood of progression by 50% and 61%, respectively.
Scaling Real-World Applications in IBD
The CD progression score demonstrated its potential for patient segmentation, supporting clinical research and treatment decision-making. The next phase will expand validation in additional cohorts and integrate the score into broader regulatory and clinical frameworks.
This success highlights the power of fit-for-purpose real-world data in improving disease understanding, optimizing clinical trial design, and informing market access strategies. With the Aetion Evidence Platform®—the industry’s only enterprise RWE solution—and IBD Plexus data, researchers can enhance disease characterization, accelerate targeted therapy development, and drive more effective treatment strategies for IBD.
Driving Innovation in IBD Drug Development with Real-World Evidence
Developing new therapies for inflammatory bowel disease (IBD) requires a detailed understanding of disease progression, patient heterogeneity, and treatment response. Aetion’s expertise in real-world evidence (RWE) generation, combined with the Crohn’s & Colitis Foundation’s IBD Plexus data, provides biopharmaceutical companies with the tools to:
1. Identify and Validate Novel IBD Biomarkers
Molecular data and biosamples present an opportunity to support biomarker discovery, providing insights into disease mechanisms, patient segmentation, and treatment response predictions. As data availability and analytic capabilities advance, validated biomarkers could enable the development of targeted therapies and accelerate precision medicine approaches in IBD.
2. Optimize Patient Stratification for Clinical Trials
Integrating molecular, clinical, and patient-reported data enables precise patient segmentation, ensuring that trials can identify subpopulations based on disease subtypes, biomarker profiles, and treatment history. For instance, patients with high inflammatory markers and specific genetic mutations could be prioritized for biologic therapies, allowing for more targeted treatment approaches. This level of granularity enhances trial efficiency, improves response rates, and generates high-quality evidence to support regulatory approvals.
3. Strengthen Regulatory and Payer Engagements with High-Quality Evidence
Regulatory bodies and payers require transparent, scientifically rigorous evidence on treatment effectiveness, safety, and real-world outcomes. Integrating the Aetion Evidence Platform® with IBD Plexus data enables the generation of robust, decision-grade RWE—real-world evidence that adheres to the highest methodological and regulatory standards. This level of rigor ensures confidence in treatment impact, supports regulatory submissions, informs reimbursement strategies, and strengthens engagement with health authorities and payers to support regulatory submissions and demonstrate therapeutic impact, reinforcing confidence in treatment strategies and informing reimbursement decisions.
Take the Next Step in IBD Research
Partner with Aetion today to harness real-world evidence and drive the next generation of IBD therapies forward.