Using Patient-Reported Outcomes to Understanding the Effects of Treatment on Health-Related Quality of Life

September 2013

Reporting the effects of treatments using outcomes as reported by patients now is a crucial part of the evidence required for pricing and reimbursement in many countries.  The pharmaceutical industry is responding by incorporating measures of patient-reported outcomes (PROs) into clinical trials in addition to more conventional clinical endpoints. An example of good practise in this area is provided by Astellas, a company  committed to evidence on patient-reported outcomes. The EQ-5D, a widely-used generic measure of patient self-reported health, is routinely included in clinical trials of its products, alongside disease-specific measures, thus providing a rich source of data on patients’ responses to treatment.

OHE has been working with Astellas to provide analyses of PRO data that generate robust evidence on the effectiveness of products as measured by improvements in patient-reported outcomes. A recent example of this research has been published in the Journal of Medical Economics[1]. This paper presents a wide range of statistical analyses performed on EQ-5D data collected in international clinical trials for a treatment for overactive bladder, mirabegron.

EQ-5D data quite often are analysed simply by applying a ”value set” to generate the utilities that are required by health economics models. However, the data provided by the EQ-5D facilitates a much richer understanding of the effects on patients’ quality of life. For example, by examining the way patients report their health on each of the five dimensions (their EQ-5D profile), we can identify which aspects of their health are affected by their condition, and how that changes with treatment. Those changes in health can be summarised in a variety of ways, using both the profile data and patients’ own overall assessment of their quality of life using the EQ-VAS scale. These insights from patients can sometimes get lost when EQ-5D data are summarised using value sets based on the preferences of the general public. The Pavesi et al (2013) study demonstrates the importance of looking more closely at PRO data. This perspective has provided Astellas with valuable evidence on mirabegron; the study also has suggested a number of important issues that can be explored in future studies of health-related quality of life in urology.

[1]Pavesi, M., Devlin, N., Hakimi, Z., Nazir, J., Herdman, M., Hoyle, C. and Odeyemi, I., 2013 Understanding the effects of HR-QoL of treatment for overactive bladder: A detailed analysis of EQ-5D clinical trial data for mirabegron. Journal of Medical Economics. 16(7), pp.866-876.

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"The feedback on your presentation was excellent! I am happy to say that you converted a few sceptics who had previously been quite negative about PROs and EQ-5D. Not only does our team now have a good basic understanding of EQ-5D and QoL measures generally, but it was also felt that your presentation helped them to understand the importance of collecting this data earlier. Most of all, they really appreciated your presentation style and delivery and how you simplified a lot of the complexity in to something that they could understand and relate to. This is a skill I have to say many health economists lack in the Pharma world."
-- Director, Health Economics and Outcomes Research, Astellas Pharma Europe Ltd.

To learn more about OHE Consulting’s services, please contact Dr Jorge Mestre-Ferrandiz  • • +44 (0)20 7747 8860

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