The theme of ISPOR Europe 2025 was “Powering Value and Access Through Patient-Centered Collaboration”, with the shift in terminology from patient engagement to patient collaboration underscoring that this remains a strong focus within the HEOR community. Patient collaboration was represented in a designated programme of talks, issue panels and research presentation sessions, and the message that healthcare decision-making should be patient-centred was reflected from all sides of the conference hall.
However, despite this clear consensus, big questions remain below the surface: how do we achieve patient-centred decisions? Who should be involved in the decision-making process, and at what point(s)? Have the actions taken to date truly translated into better representation of patient preference and experience within decision-making?
It was positive to hear numerous examples of situations in which patient collaboration is currently leading to tangible outcomes within the HEOR space. Regulatory bodies are moving towards formal incorporation of recommendations relating to patient experience data (PED) within their guidelines, such as the recent publications from the FDA and EMA defining PED, with the EMA publication presenting a very favourable view on early engagement for the collection of PED and stating an expectation that this is collected.
In line with this regulatory perspective, the HTA voice was clear that consideration of patient preference is not a novel concept for decision-makers, though historically limited reporting has meant that the extent to which it has informed or shaped final outcomes has often been unclear. For example, previous research presented by Costello Medical at ISPOR Europe 2023 on the reporting of patient preferences in prior NICE non-oncology appraisals found that, of the 15 appraisals where attributes identified from patient preference evidence were mentioned in the final appraisal document (FAD), only two of these included a standalone patient preference-related section in the FAD summarising committee conclusions.
Furthermore, specific areas of uncertainty where PED and qualitative patient collaboration meaningfully address gaps in the decision-making framework were highlighted. For example, incorporation of the real-life impact for patients, their carers or their families may be supplemented in some areas by robust PED, such as in diseases in which generic HRQoL tools such as the EQ-5D are insensitive to relevant domains, despite being favoured by many HTA bodies such as NICE. Capturing the experiences of patients with rare diseases is another valuable application, where few alternatives exist. For ultra-rare conditions however, it can be challenging to identify patients from particular geographies or other settings. Expert judgement and/or elicitation is crucial in these situations to provide a proxy for patient experience (please see our recent publication for recommendations on clinical expert judgment methods in the context of rare diseases).
The incorporation of patient preference is a nuanced topic, reflecting the diversity in patient experience and preference both within and between diseases. Monday’s plenary session highlighted that patients with multiple sclerosis asked the EMA to retain natalizumab as a treatment option based on its superior efficacy profile, despite its associated increased risk of patients developing progressive multiple leukoencephalopathy (PML), a very serious brain infection that may result in severe disability or death. In contrast, patients in an oncology indication stated they would refuse treatment with a drug which demonstrated improved overall survival but was commonly associated with severe, non-life-threatening side effects such as vomiting and diarrhoea. In both cases, patients were trading efficacy against safety, but advocated for opposing conclusions, emphasising the importance of disease- specific PED. Despite this, research presented by Costello Medical at ISPOR Europe 2025 indicates that to date, many NICE submissions in which a utility adjustment is included within the economic model to reflect patient preference have sourced these PED from general population or mixed population cohorts rather than disease-specific cohorts – see our podium presentation.
It is unlikely that one standardised approach will suitably capture all relevant input, and several talks discussed novel methods for involving patient voices in HEOR research but acknowledged considerable barriers that may limit their uptake. One talk focussed on the interplay between clinical outcome assessments (COAs) and patient-preference studies, such as leveraging COA concept elicitation data to inform study design or using patient-preference information to construct meaningful change thresholds. However, COA concepts, attributes, and levels may not map cleanly to preference attributes. Furthermore, COA scores are often ordinal, meaning that translating this to continuous preference scales is non-trivial.
In addition to the “how”, consideration also needs to be given to the “when”. Discussions were clear that engagement with patients too late in the process decreases the likelihood that their perspective will be meaningfully incorporated into final outcomes like regulatory guidelines, HTA decisions or trial design, and instead risks it becoming a tick-box exercise. Early and open engagement will facilitate a more meaningful and positive impact on healthcare decision-making. For example, our research has found that involvement of patient experts in the design of real-world evidence studies leads to more robust study design and produces more relevant and valuable insights to inform HTA decision-making.
There was strong consensus across delegates from industry to regulatory bodies to decision makers that patient experience matters and should be central to final decision making, but that the ecosystem is complex and multifactorial and any guidance or standardisation should suitably reflect this nuance. As discussed in Monday’s plenary session, just as no single organism within a termite mound has knowledge of how the mound functions as a complex system, so it may be unrealistic that any individual person or decision-making body can know all possible perspectives on decisions, but that does not mean that each individual part does not have a critical role to play.
We need stories in the form of qualitative input as well as quantitative data, and these qualitative inputs may help to define which qualitative data need to be sought. By its nature, data will only ever answer the question asked, so beginning with patient stories is critical to ensure that the right questions are asked at the right time, allowing understanding of what it is that patients really value. From the generation of relevant and appropriate data that follows, a truly patient-centric approach to HEOR can develop. Formalising guidelines or standardising approaches to PED generation and incorporation into decision-making, before qualitative research has been performed to establish preferences and priorities from patients themselves, may put the cart before the horses. This would risk engraining into guidelines approaches that do not accurately or completely reflect the views of patients.
Amongst all of this, the burden of data collection must on patients should be considered, particularly given Joint Clinical Assessment (JCA) is likely to increase data collection demands already, with some talks calling for JCA harmonisation of PRO data assessment accordingly. It must be carefully considered how this burden for participants, particularly those in rare spaces, will be managed. Pragmatic trial design was suggested as one option to address this, as well as a renewed focus on the collection of qualitative data.
Overall, the message throughout the conference was clear: we all know where we want to go, but the pathway of how we will get there remains to be defined.
If you would like any further information on the summary presented above, please get in touch, or visit our Patient Engagement page. Tom Gleeson (Consultant) and Aneka Sowman (Consultant) contributed to this article on behalf of Costello Medical. The views/opinions expressed are their own and do not necessarily reflect those of Costello Medical’s clients/affiliated partners.
