The role of real world evidence in the cost-effectiveness analyses for pharmacological treatments in overactive bladder

Nazir, Mohammed Jameel (2017) The role of real world evidence in the cost-effectiveness analyses for pharmacological treatments in overactive bladder. Doctoral thesis (PhD), Manchester Metropolitan University.

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Abstract

The aim of this thesis is to evaluate the extent to which cost-effectiveness of a recently launched drug in the treatment of overactive bladder is supported by real world evidence. The demand for real world effectiveness data by Payers and decision-makers is increasing to better manage the uncertainty at the time of making reimbursement decisions. Real world data can help to fill the knowledge gap between clinical trials and actual clinical practice. Overactive bladder is one of many chronic conditions likely to impact people as they age. It is a common condition characterised by a group of lower urinary tract storage symptoms, which has a profound and measurable negative effect on patient health-related quality of life (HRQoL). The economic burden of OAB is also considerable. Antimuscarincs have been the mainstay pharmacological treatment for OAB for over 30 years, but adverse events and persistence on medication remain a key issue. Mirabegron, a ß3 adrenoceptor agonist provides an alternative option. The thesis presents and critiques 9 peer-reviewed publications to demonstrate the value evidence generated pre-health-technology assessment (HTA) and that generated post-health -technology assessment in clinical practice. Each publication adds a new building block to the value proposition, and a sophistication of methods that help illustrate the value of the new drug compared to competing alternatives. The impact of OAB on patient HRQoL is explored through analyses of both disease specific and generic validated patient reported outcome (PRO) instruments. Utility values are also derived for the purposes of health economic modelling. The comparison of efficacy and safety of mirabegron with other competing alternatives is assessed using network meta-analysis (NMA), a requirement from most HTA bodies in the absence of head-to-head evidence. The outputs from the NMA and the PRO analyses are then applied to a series of trial based and NMA based HE models to assess the cost-effectiveness of mirabegron. This is followed by an assessment of effectiveness through analysis of a large retrospective database to see if indeed cost-effectiveness is supported in the clinical practice. The thesis concludes that cost-effectiveness of mirabegron is broadly supported by Real world evidence in terms of persistence and adherence while also highlighting the strengths and weaknesses of the current research and making recommendations for future research.