Hearing Patients Loud and Clear: The Growing Importance of Patient-Centred Evidence and the Patient Voice in Health Economics and Outcomes Research (HEOR)

ISPOR Europe 2022 saw a continued focus on patient centricity in the context of decision-making, with particular attention given to the topic of data on patients’ experiences across all aspects of their lives (known as patient experience data) as an important tool in making evidence-based health care decisions. Beyond this, speakers at this year’s conference also focused on a range of further key patient-centric topics, including patient-reported outcomes (PROs), patient preferences and patient engagement. This built on themes discussed at ISPOR International earlier this year in Washington, which highlighted challenges associated with the incorporation of patient preference information into drug development strategies and decision-making and strategies to address these (see our report on this here).

Is Health-Related Quality of Life (HRQoL) Measured Appropriately Throughout the Patient Lifespan?

Speakers at the conference showed an increased recognition of the importance of a consistent and co-ordinated approach for measurement of HRQoL across the patient lifespan, to ensure adequate capture of the patient perspective across different resource allocation decisions. However, a lack of guidance on utilising instruments both within and across age ranges has raised hurdles in the measurement of HRQoL in a standardised fashion. Dr Janine Verstraete of the University of Cape Town highlighted that multiple instruments were recommended for use in certain age ranges, raising questions around whether HRQoL is measured in a consistent way within these populations and the circumstances under which each tool should be considered most appropriate. For example, EQ-5D-3L, EQ-5D-Y, CHU9D, PedsQLTM and HUI are all suitable for use in adolescent populations (aged 12–15; see Figure 1). In England and Wales, the National Institute of Health and Care Excellence (NICE) does not recommend specific performance-based measures in the paediatric population, likely contributing towards heterogeneity in HRQoL measures used across appraisals in this population; current guidance only states that such a measure should be generic and have a good psychometric performance in the specified age range.1

Figure 1: A summary of the appropriate age ranges and respondent types for common paediatric HRQoL measures

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Dr Michael Herdman further noted that the existence of separate tools for children, adolescents and adults may lead to inconsistent HRQoL measurement in trials with mixed paediatric and adult populations, and can also introduce discontinuities in measurement at ‘transition points’ as a patient moves into the next age range. Such inconsistencies may be amplified by differences in respondent types (self-report vs proxy-report), modes of administration (self-completed vs interview-assessed) and geographical setting. Therefore, if HRQoL is to be measured consistently within and across age ranges, both appropriate choice of measure and comparability of results at transition points between different measures will be needed.

Challenges and Strategies Associated with Using Paediatric HRQoL Values in Decision-Making

Speakers at an Issue Panel entitled ‘Are Existing Methods of Paediatric Health-Related Quality of Life Fit for Purpose for Use in Health Technology Assessment?’ acknowledged ongoing challenges associated with health technology assessment (HTA) in paediatric indications, largely stemming from the lack of standardisation in HRQoL measurement discussed above, and also from a lack of established value sets to calculate utilities.2 Ongoing research is aimed at supporting the future recommendation and use of specific instruments from decision-making bodies and drug developers. At the Issue Panel, Professor Kim Dalziel reported on the QUOKKA study, a head-to-head comparison of the psychometric performance of various widely used generic paediatric HRQoL instruments in an Australian setting. Meanwhile, Sophie Cooper, Scientific Advisor at NICE, reported on a range of NICE-supported research initiatives to generate research on the appropriate choice of instrument used to measure HRQoL, the choice of value set used to calculate utilities, and the impact of age, perspective and method on generating these utility values.

Although challenges with considering paediatric HRQoL in HTA remain, it is apparent that there is a growing institutional focus on developing new strategies to address these challenges and enable the capture of paediatric HRQoL in an appropriate way for decision making.

An ISPOR Roadmap for Increasing the Utility and Impact of Patient Preference Studies

Reflective of a desire to increase the utility and relevance of patient-centred research in healthcare decision-making, the ISPOR ‘Using Patient Preferences to Inform Decision Making’ Taskforce unveiled the new ISPOR Patient Preference Roadmap at this year’s congress (Forum 115).3 This framework has been designed to ensure the utility and impact of patient preference studies in healthcare decision-making, intending to achieve this by promoting high-quality research and best practice.

The framework spans five domains for conducting patient preference studies (Figure 2).

Figure 2: Five domains to be considered when conducting patient preference studies

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Source: Adapted from ISPOR Forum 115.

This roadmap is likely to be well-received from both a decision-maker and patient perspective. Jacoline Bouvy from NICE (representing a HTA body perspective) noted that, by making explicit the questions which matter most to decision-makers, the roadmap will enable patient preference studies to better align with the decision-making context and subsequently have the most impact.

In addition to acknowledging the rise of patient-centred research, Eric Low, an independent market access and patient engagement consultant providing a patient perspective, highlighted that a key benefit of the roadmap was the emphasis on the nature of patient engagement during the preference elicitation process. Specifically, the roadmap calls out the importance of recognising the value of patient input, sharing results and resources with patient groups, or otherwise ‘giving back’ to patients. Finally, Eric Low noted that this roadmap represents a movement towards an environment in which the patient perspective is central to decision-making. Whilst questions remain regarding the extent to which patient preference data would influence the final outcome of the multi-factorial reimbursement decision, it may provide weight to recommendations in ‘preference-sensitive’ situations, for example where distinctly different technologies offer similar health outcomes. Over the coming months and years, it will be interesting to observe implementation of this framework and how it shapes inclusion of patient perspectives in decision-making.

Patient Voice in the Decision-Making Process

The increased recognition of the need to incorporate patient preferences in decision-making in a way that enhances the utility and impact of such research, and the research into consistent and appropriate measurement of HRQoL for children and adults, both point towards the growing importance placed on the voice of the patient in HTA. It is clear that focus is being shifted towards patients, and that they ought to be at the centre of decision-making.

HTA body representatives as well as manufacturers and patient groups were active at ISPOR in conveying how patients can provide valuable input throughout the HTA process, from helping to fill evidence gaps to the interpretation of evidence for decision-making. Val Buxton, Chief Executive, Association of Glycogen Storage Disorders noted the particular value of early partnerships between pharmaceutical companies and rare disease patient groups, which are small and benefit from collaboration to achieve large-scale, ambitious goals. Val stressed that patients need to be listened to from early on to facilitate a smooth and unobstructed HTA process, and this starts with the formation of an established process to involve patients, elicit their feedback and ensure their feedback is implemented.

In collaboration with Eric Low, Costello Medical have developed a guide aimed at pharmaceutical companies looking to submit to NICE, which summarises the opportunities for patient involvement throughout the single technology assessment (STA) process.


  1. National Institute for Health and Care Excellence. NICE Health Technology Evaluations Manual (PMG36). Available here. Last accessed: 25 November 2022.
  2. Session 220: Are Existing Methods of Paediatric Health-Related Quality of Life Fit for Purpose for Use In Health Technology Assessment. ISPOR Europe 2022. Vienna, Austria.
  3. Session 115: A Roadmap for Increasing the Usefulness and Impact of Patient-Preference Studies in Decision-Making in Health: The Final Recommendations from the ISPOR Using Patient Preferences to Inform Decision Making Good Practices Task Force. ISPOR Europe 2022. Vienna, Austria.

If you would like any further information on the themes presented above, please do not hesitate to contact Elysia Upton, Consultant (LinkedIn), Keval Haria, Senior Analyst (LinkedIn) or Naman Kochar, Senior Analyst (LinkedIn). Elysia, Keval and Naman are employees at Costello Medical. The views/opinions expressed are their own and do not necessarily reflect those of Costello Medical’s clients/affiliated partners.