Significance

Fluctuating symptoms in chronic diseases are often poorly captured by traditional clinical trial endpoints. Digital health technologies (DHTs) offer a transformative opportunity to redefine endpoints that are rooted in real-world, patient-reported experiences. By shifting toward patient-centric flare assessment, researchers can improve the clinical relevance, accuracy, and impact of trial outcomes—leading to better treatments and enhanced quality of life.

Issue

How can we strike a balance between patient-centric endpoint development and clinical relevance in drug development with the emergence and adoption of a diverse pool of digital health technologies (DHTs)? In this panel, industry executives and clinical research experts will discuss potential to define and develop better endpoints of disease with flares, on the basis of what is experienced and reported by patients themselves. This panel will debate best practices to minimize participant burden while still capturing the full range of endpoint concepts via patient-centric instrument development, best technological approaches, and, importantly, the emerging trend of creating endpoints on the basis of how patients most accurately experience their condition.

Overview

Patient-reported outcomes (PROs) are leveraged in clinical trials to measure and determine whether a treatment being tested is effective on the basis of patients’ experience. However, current criteria for selecting PROs may not incorporate the presence of fluctuating disease experiences that matter most to patients in their daily lives. As a result, patients with chronic conditions where disease activity can fluctuate, such as sickle cell disease or atopic dermatitis, may find that current patient outcome assessments do not overlap with their most significant flare experiences. Patient-defined flare endpoint development, captured via real-world evidence in DHTs, may be a solution. Development of such endpoints includes: direct involvement from patients, creation of a valid and reliable assessment tool, and the use of this assessment to create an endpoint that is meaningful to patients.

DHTs can significantly enhance the long-term development of patient-centered flare endpoints by providing more accurate, timely, and comprehensive data on patient experiences. Utilizing the general process outlined above, DHTs provide a pathway to enhance patient engagement, continuous monitoring, and capture patient perspectives in a flexible manner. By leveraging DHTs, researchers can develop more accurate, patient-relevant flare endpoints that reflect the real-world experiences of patients with the goal of having better clinical outcomes and improved quality of life for patients.

Conclusion

DHTs present a compelling path forward in building more responsive, relevant, and patient-centered endpoints for clinical research. By integrating real-world, patient-driven insights—particularly around episodic disease activity—researchers can better align drug development with patient needs. This panel will illuminate how such approaches can reduce burden, elevate the patient voice, and ultimately lead to more impactful clinical outcomes.