Patient-reported outcomes in overactive bladder: Importance for determining clinical effectiveness of treatment

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Abstract

Overactive bladder (OAB) is a condition defined by its symptoms—urinary urgency with or without urgency urinary incontinence and often with frequency and nocturia. As such, determining the efficacy of OAB treatments using objective measures, such as urodynamic testing, can be difficult. A better means of gauging treatment efficacy for symptom-based conditions is through the use of patient-reported outcomes (PROs). With PROs, clinicians can gain insight into how a treatment affects a patient’s symptoms and whether improvement in symptoms has a positive effect from the patient’s perspective. PROs are increasingly being included as end points in clinical trials, including those of antimuscarinic drugs for OAB. Consequently, clinicians should become familiar with the most commonly used instruments. We provide an overview of instruments used to assess symptoms, health-related quality of life, and treatment satisfaction in patients with OAB and discuss how PROs can be incorporated into clinical trial protocols.

Section snippets

Patient-reported outcomes in overactive bladder

OAB is a chronic condition comprising several symptoms, the principal symptom being urgency,8 an abnormal, compelling desire to urinate in which patients may feel that leakage of urine is imminent; urgency may or may not result in urgency urinary incontinence (UUI).9 OAB is not life threatening and its symptoms typically do not cause overwhelming disability, yet OAB symptoms do greatly affect a patient’s life. Patients may lose sleep because of nocturia, curtail activities, limit travel, and

Incorporating patient-reported outcomes into overactive bladder clinical trials

The importance of PROs in assessing OAB symptom bother, HRQL, and patient satisfaction with treatment makes these measures an essential part of assessing efficacy in OAB clinical trials. Several reviews provide detailed discussion on issues to be considered when incorporating PROs into clinical trial protocols.1, 5, 27, 34, 35, 36 Overall, PROs, whether primary or secondary end points, must meet the same standards as those used for traditional clinical end points.27, 34, 35 Some basic

Conclusion

OAB is a symptom-defined condition, making it important to understand the patient’s perspective in assessing treatment effects. Patient reports of changes in OAB symptoms provide a better indication of treatment efficacy than do objective urodynamic tests, and they are the only means of determining the impact of treatment on HRQL. PRO data complement clinical outcomes in a research setting and allow investigators to determine whether any treatment-related improvement actually made a difference

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  • Cited by (0)

    1

    Linda Brubaker receives research funding from Q-Med, Pfizer Inc, and Allergan and receives consultant/speaker honoraria from Q-Med, Novartis, Pfizer Inc, and Astellas.

    2

    Christopher Chapple is a scientific consultant to Pfizer Inc, Astellas, Novartis, Schwarz, Q-Med, Tanabe, and Allergan; and receives funding from Pfizer, and Allergan.

    3

    Karin S. Coyne is a consultant to Pfizer Inc.

    4

    Zoe Kopp is an employee of Pfizer Inc.

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