Assessment of health-related quality of life and patient preferences for treatments of overactive bladder

Hakimi, Zalmai (2022) Assessment of health-related quality of life and patient preferences for treatments of overactive bladder. Doctoral thesis (PhD), Manchester Metropolitan University.

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Abstract

Overactive bladder (OAB) is a highly prevalent symptomatic condition characterised by urinary symptoms: urgency and urinary frequency, and is often accompanied by incontinence and nocturia. Although it has no impact on mortality, OAB is a debilitating condition and has a significant impact on health-related quality of life (HRQoL). The patient perspective is becoming increasingly important in the assessment of the value of new treatments by Health Technology Assessment (HTA) bodies as it informs the burden of disease, the clinical relevance of the treatment benefit and can be a predictor of the persistence with treatment which is often an important factor in health economic evaluations. Therefore, it is important to ensure that a robust assessment of the patient perspective is included during the HTA process for OAB therapies. The aim of this thesis was to provide an understanding and a critical assessment of HRQoL for treatments of OAB in relation to HTA. The thesis presented and critically assessed eight peer reviewed publications by the researcher that examined several aspects of patient perspective by applying different methodologies. The studies that were included in this thesis provide a coherent body of evidence that has been used by HTA bodies to assess the value of OAB treatments and has had an influence on reimbursement guidelines for mirabegron, a first in class treatment for overactive bladder. Patient burden was assessed using concept elicitation interviews allowing a comprehensive evaluation of the quality of life dimensions affected by OAB. The patient preferences influencing patients and physician treatment choices were investigated through two discrete choice experiments conducted in three European countries. The clinical relevance of the treatment benefit on OAB symptoms was examined in three large clinical trials by conducting single and double responder analyses involving a response based on both objectively assessed symptoms and PRO instruments. Finally, the estimation of utility values and a robust economic evaluation that were integral part of the single technology appraisal of mirabegron in the United Kingdom were discussed. The thesis concluded that performing a robust and comprehensive assessment of the patient perspective is a critical component of the value demonstration in OAB and has been a critical element informing HTA decisions in OAB. Limitations were discussed and the thesis provides recommendations for future research to further examine the generalisability of this work in other settings