Experts have signaled a need for the development of validated patient-reported outcomes for inflammatory bowel disease based on Food and Drug Administration guidance, in the face of a move toward the use of patient-reported outcomes and objective measures of disease as the primary endpoint for clinical trials.
Dr. Nicolas Williet, from the Université Henri Poincaré, Vandoeuvre-lès-Nancy, France, and his colleagues wrote that the FDA was moving away from use of the Crohn’s Disease Activity Index in the assessment of inflammatory bowel disease (IBD) outcomes, and would likely soon require patient-reported outcomes as a coprimary endpoint with objective measures such as endoscopic assessment.
Patient-reported outcomes should be obtained by using a validated, self-administered questionnaire with items that are generated solely by patients, the authors wrote online Feb. 19 in Clinical Gastroenterology and Hepatology.
"A PRO [patient-reported outcome] is any report that comes directly from a patient about a health condition or its treatment without interpretation of the patient’ s response by a clinician or anyone else," they wrote. "The items measured by PRO instruments that are used most often in support of labeling claims refer to a patient symptom, sign, or an aspect of functioning directly related to disease status."
However, the authors said that no existing patient-reported outcomes for IBD, such as the Inflammatory Bowel Disease Questionnaire assessing quality of life and the IBD Disability Index, were developed according to the recently developed FDA guidelines. There is a need for international consensus to develop validated patient-reported outcomes using FDA guidance, they asserted.
"During the development of PROs, the disease characteristics of the patients (type of IBD, extent of the disease, the presence or absence of objectively measured inflammation, presence or absence of disease complications such as stricture, fistula, and abscess, and so forth) must be documented carefully to determine the impact of various disease characteristics on the operating characteristics of the PRO."
Particularly in irritable bowel syndrome, there is still debate about how best to measure patient-reported outcomes, the authors said (Clin. Gastroenterol. Hepatol. 2014 Feb. 19 [doi: 10.1016/j.cgh.2014.02.016]).
"Depending on their structure and format, PROs can have different levels of sensitivity to core IBS symptoms and can be influenced by psychological and somatic complaints."
Patient-reported outcomes are already used in other chronic conditions such as rheumatoid arthritis and multiple sclerosis, with studies showing that patient-reported outcomes offer important endpoints for drug approval in these conditions.
"In the near future, PROs may evolve to be used as the primary or major secondary endpoints in clinical trials in patients with IBD, possibly leading to drug approval and labeling, similar to the JAK inhibitor in the treatment of myelofibrosis," the authors reported.
Two of the three authors disclosed consulting and lecture fees, as well as research support, from numerous pharmaceutical companies.