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International Clinical Trials

Early Engagement

Patient-reported outcomes (PROs) are any report of the status of a patients’ health condition that come directly from the person, without interpretation of their response by a clinician or anyone else (1). PRO measures (typically surveys) may be generic, measuring, for example, health-related quality of life (HRQoL), or they may be disease-specific, providing information about symptoms experienced by a patient. For generic PROs, another sub-category of preference-based PRO instruments exists, such as the EQ-5D (2). These generate a profile score that summarise patients’ responses to items designed to tap into different domains of HRQoL, for example, ‘mobility’ and ‘self-care’. Therefore, the profile score represents a given health state, which is then converted to an index score using an algorithm that applies weights to the profile score based on population preferences for that specific health state. This is referred to as a health utility value and can be used in cost effectiveness analyses.

Collection of PROs in clinical trials usually serves one of two main purposes. First, generic and disease-specific PRO data are used in labelling claims and product summaries, providing evidence of treatment benefits not easily measured using clinical tools. This can be particularly useful in therapy areas without well-defined physiological markers, such as pain and mental health. Even where robust clinical indicators are available, PROs can demonstrate additional benefits, such as improvements in functioning and alleviation of symptoms. In rare or orphan diseases where treatments are commonly expensive, demonstration of HRQoL or functioning improvements can be particularly important factors for regulatory and reimbursement agencies in the decision-making process. For treatments that aim to alleviate symptoms rather than to cure, PRO evidence may constitute a primary efficacy, whereas for the endpoint of curative or life-extending treatments, PRO evidence may constitute a secondary endpoint, providing evidence of additional treatment benefits. The second way in which PRO trial data are used applies specifically to the use of preference-based PRO instruments. Information from these tools is used to calculate health utilities for cost-effectiveness models. These are one of the cornerstones of health-technology appraisals (HTAs) in the UK and are given prominence in HTA guidelines around the world (3,4). Given that appropriate utility measures are essential for accurate economic modelling, utility collection is a crucial part of regulatory and reimbursement approval.

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Dr Jenny Retzler is a Senior Research Consultant at York Health Economics Consortium, with a PhD in psychology and an MSc in cognitive neuroscience and neuroimaging, both from the University of Nottingham, UK. As a senior member of the outcomes research team, she works on a variety of projects, specialising in the measurement of treatment benefit. Her work includes the provision of evidence-based strategic advice on the optimal selection, implementation and analysis of outcome measures, and the development and implementation of utility
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Dr Jenny Retzler
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