Practice Management Toolbox

Making a case for patient-reported outcomes in clinical inflammatory bowel disease practice


 

Patients seek medical care when they perceive a deterioration in their health. Gastroenterologists and health care providers are trained to seek out clinical, laboratory, radiologic, and endoscopic evidence of pathology. Conventional endpoints in inflammatory bowel disease (IBD) clinical trials and clinical care may fail to capture the full health status and disease experience from the patient perspective. The Food and Drug Administration has called for the development of coprimary endpoints in research trials to include an objective measure of inflammation in conjunction with patient-reported outcomes (PROs). The objective is to support labeling claims and improve safety and effectiveness in the drug approval process.1,2 There is also growing recognition that high-value care includes management of biologic and psychosocial factors to enable patients with chronic diseases to regain their health. Clinicians might follow suit by incorporating valid, reliable PRO measures to usual IBD care in order better to achieve patient-centered care, inform decision making, and improve the care provided.

What are patient-reported outcomes?

The FDA defines a PRO as “any report of the status of a patient’s health condition that comes directly from the patient, without interpretation of the patient’s response by a clinician or anyone else.” Two PROs are used to measure various aspects of health including physical, emotional, or social domains. PROs have emerged as tools that may foster a better understanding of the patient’s condition, which may go beyond disease activity or symptoms. In effect, incorporating PROs into clinical practice enables a model of “coproduction” of health care, and may contribute to a more reciprocal patient-provider interaction where the needs of the patient may be more fully understood and incorporated into decision-making that may lead to improved patient satisfaction and outcomes.3,4

Dr. Erica R. Cohen

There are hundreds of available PROs in gastroenterology,5 ranging from simple (characterizing pain with a basic numeric rating scale) to complex multidomain, multi-item instruments. PROs may cover symptom assessment, health-related quality of life, and adherence to and satisfaction with treatment, and may be generic or disease specific. Numerous PROs have been developed for patients with IBD. Commonly used PROs in IBD include severity scales for pain, defecatory urgency, and bloody stool, and several disease-specific and generic instruments assessing different health-related quality-of-life domains have been used in research studies for patients with IBD.

The current approach to patient-centered care for IBD is limited

IBD is a difficult disease to manage – in part because there is no known biomarker that accurately reflects the full spectrum of disease activity. Numerous indices have been developed to better quantify disease activity and measure response to treatment. Among the most frequently used indices in clinical trials are the Crohn’s Disease Activity Index (CDAI) and (for ulcerative colitis [UC]) the Mayo Clinic Score. These endpoints incorporate signs and symptoms, laboratory findings (in the CDAI), and endoscopic assessments. The CDAI is a suboptimal instrument because of a lack of correlation with endoscopic inflammation and potential confounding with concomitant gastrointestinal illnesses, such as irritable bowel syndrome.6 The Mayo Clinic Score is difficult to interpret because of some subjective elements (what is considered a normal number of stools per day?); vagueness (mostly bloody stools more than half the time?); and need for a physician assessment, which often does not correspond with the patient’s perception of their disease.7 From a research perspective, this disconnect can compromise the quality of trial data. Clinically, it can negatively impact patients’ satisfaction and impair the patient-provider relationship.8

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