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Proposal 1637

Title of the Proposed Research

1618 - Optimizing Patient Reported Outcomes by Linking Study Endpoints to Real-World Benchmarks

Lead Researcher

Dr. Jeffrey Lackner

Affiliation

University at Buffalo, SUNY

Funding Source

None

Potential Conflicts of Interest

None

Data Sharing Agreement Date

31 May 2017

Lay Summary

Patient reports about how much their symptoms have or haven’t improved, and what impact those symptoms have on their quality of life, are increasingly important when evaluating the effectiveness of new treatments. This is especially true in cases where diseases lack a clearly identifiable physical basis such as irritable bowel syndrome.A common way to collect reports from patients is to have them fill out multi-point rating scales about the degree of improvement in their symptoms since the treatment began.For instance, the Subjective Global Assessment scale rates symptoms from 1 to 7, where:

1= very much improved
2= much improved
3= minimally improved
4= no change
5= minimally worse
6= much worse
7= very much worse

We want to document the meaning of patient responses as accurately as possible, but we’re concerned that metrics such as these might have different meanings for different patients and other stakeholders that rely on quality outcome data. What exactly does it mean for a patient to say s/he is “minimally improved” and how different is that from saying that one is “much improved”? These are vague terms. We want to track variations in what these terms mean to patients and we want to give them a more precise definition.Traditional testing research examines the reliability (whether a test gives the same outcome every time).and validity (whether a test measures what it is supposed to) of measures like the Subjective Global Assessment scale. It documents the extent to which responses are affected by external factors, such as a patient’s mood at the time the rating is made or momentary lapses in attention on the part of the patient. Our focus, instead, is on elaborating the meaning of one rating category (such as “much improved”) as compared to another rating category (“minimally improved”). We want to know what variations in these meanings imply in terms of actual symptom change and changes in the patient’s quality of life. For one patient, modest changes in some symptoms might be deemed as “minimal improvement.” However, for another patient, larger changes in those same symptoms might be characterized as “minimal improvement.” How do the various symptoms of differ for irritable bowel syndrome patients who say they are “much improved” versus patients who say they are “minimally improved”? How do the experienced irritable bowel syndrome symptoms differ for patients who say they are “minimally worse” compared to patients who say they are “much worse”? The fact is we do not have a good sense of how symptom changes map onto patients’ descriptions of improvement, nor do we know how symptom change map onto their characterizations of their quality of life. We intend to do such mapping in order to gain a better understanding of what a patient means when s/he reports.

Study Data Provided

NOVARTIS-CHTF919A2306: A randomized, double-blind, placebo-controlled, parallel group, multicenter study to assess the efficacy and safety of repeated treatment with tegaserod 6mg bid and placebo in women with irritable bowel syndrome with constipation (IBS-C)

Statistical Analysis Plan

Publication Citation

The publication citation will be added after the research is published.

Summary Results

Results summary or link will be posted when available.