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Patient-Reported Outcomes in Oncology: Is JCA Helping or Hurting?

  • November 4, 2026
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At the end of October I had the pleasure of attending the European Society for Medical Oncology (ESMO) congress in Madrid. It’s inspiring to be around so much science and super smart people all focused on improving patient outcomes for people with cancer. There were some particularly interesting presentations on cancer vaccines, the role of AI in biomarker evaluation, and tailored/precision- medicine. But I was bitterly disappointed about how little direct patient experience data (PED) was on display. There was not a single dedicated session on PED, despite recent initiatives from SISAQOL 1 and the PROTEUS consortium OncoPRO initiative 2 . And whilst there was a handful of posters focusing on PED, these represented a significant minority (<2%) of those being presented; see Table 1. There were even fewer posters presented on the regulatory and payer landscape (see Table 2), despite that EMA PED guidance and the first reports from the EU-wide joint clinical assessment (JCA) have been published since the last ESMO congress. This was disappointing given that almost all companies claimed “patient centricity” or “patient focused” as a core part of their messaging.

From patient voice to value

I was therefore super grateful to Regeneron for sponsoring a satellite symposium titled “From Voice to Value: Advancing Actionable Patient-Reported Outcome (PRO) Evidence in Oncology” and inviting me to be one of the speakers. In this session we discussed why patient experience matters in medicines development, how to define patient-relevant clinical trial outcomes, and how PROs shape regulatory, payer and clinical decision-making. Professor Pilar Garrido started by explaining the role of PROs in cancer research and care while simultaneously highlighting the lack of standardization in concepts measured, and instruments used, across trials (citing Kim et al, 2023 3 ). Merel Hennink – a lung cancer survivor and patient advocate gave an impassioned plea to medical professionals and researchers to routinely use PROs in healthcare to capture information that can drive patient-focused discussions about treatment. She gave some clear advice to clinicians – tell patients why you are asking them to complete PROs, who reads the responses that they give, and what will happen based on their responses. Without this, she warned, engagement is likely to be low – or worse, completion will be unreliable! Professor David Gandara thereafter talked about how he uses PROs in his clinical practice and discussed his work exploring the prognostic value of PROs in lung cancer. This research shows that symptoms, physical functioning and HRQoL can all predict survival outcomes 4-6 . Then Professor Olivier Chassany and I discussed the European regulatory and JCA environment for PROs. We talked about the 3 oncology products that have been through the JCA process, the role of PROs during review, and the potential for JCA to increase use (and harmonization) of PROs in medicines development. It was fun. But the discussion also got me thinking…..is there a risk that JCA could lead to less collection of PROs in the future?

What are the JCAs telling us?

Three cancer medicines have been through the JCA process as of October 2026 – Tovorafenib (Ojemda) for the treatment of low grade gliomas, and Tarlatamab (Imdylltra) and Lurbinectedin (Zepzelca) for extensive stage small cell lung cancer. For all three products, the EU HTA Secretariat requested the inclusion of PROs in the dossiers – health-related quality of life, health status, and symptoms. This is great news. However, the data submitted by the companies was of poor quality. Two of the three products relied on indirect treatment comparisons and/or meta-analyses to support part of their submissions, and thus the PRO data was either missing or only partially presented. For the third product, the data was rated as low quality by the reviewers in part due to missing data, PRO implementation challenges (including variable assessment frequencies and follow-up time), and trial design causing interpretation challenges (e.g. open-label). None of the JCA reports give a clear “conclusion” or “recommendations” to member states for how to use the PRO data to inform decisions about incremental effectiveness vs standard of care treatment options. That means that each HTA agency are likely to look at the PRO data in different ways that suit their own appraisal system, and history tells us that this will be quite variable, and somewhat unpredictable!

The PRO problem isn’t new

The JCA critique should not have been a surprise. We have known for a long time that there are challenges in interpreting PRO data in oncology owing the preponderance of single-arm trials and open-label studies, the breadth of PRO instruments used variably across studies, a lack of standardized guidance of interpreting PRO data, and a chronic de-prioritization by site staff to robustly collect PRO data during trial conduct (both before and after progression). But regulators and payers know this too, and they have been actively engaged in scientific and operational work in recent years to improve data completeness, quality and interpretability. Given the increasing requests for PRO data from regulators and payers, and the increasing use of PROs in pivotal oncology trials 7 , I thought that the HTA reviewers would take a pragmatic approach to looking at the data. Please do not think that I am advocating for consideration of data that lacks scientific scrutiny and rigour –I am the first to criticise PRO data that has not been adequately considered or has been collected using poorly-performing instruments in poorly-considered trials; but the member states must have anticipated this when requesting the PRO data from the sponsors.

Could JCA have the opposite effect?

Perhaps – and I hope this is the case – they are using these initial appraisals to set out their stall. We REALLY want you to collect PRO data. We REALLY want to use that data in our appraisal of comparative clinical effectiveness. But, we REALLY need that data to be better than it is right now. That will require sponsors to reconsider their approaches to trial design to generate robust PRO data. This is a laudable thought. But I fear that it risks having the opposite effect. Sponsors are unlikely to fundamentally change their approach to trial design “just” to ensure that HTA agencies can examine PRO data that they may or may not value. And so, there is a real risk that sponsors start to decrease their use of PROs in clinical trials, doubling down on survival and progression as the outcomes that are clinically important, and ignoring that quality of life, symptom management, and tolerable treatment options are the patient priorities. This would be a big failing of the system, and against all the things that Merel, Pilar and David spoke about in our symposia. And in this case, future ESMO conferences may have even less PED being presented.

Search of the poster presentations at the 2026 ESMO conference for generation of PED (electronic and presented posters; total N=4014)

Word/term in poster title
Quality of life (QoL)
Patient-reported outcome (PRO)
Preference
Qualitative*
Shared decision-making^
Engagement*
Number of posters
34
23
7
5
3
1
% of all available posters
0.85%
0.57%
0.17%
0.12%
0.08%
0.03%
* results presented that relate to qualitative research with patients/patient engagement rather than qualitative research with HCPs and engagement with regulators, process, etc.

^ There was one excellent satellite symposia on this topic, sponsored by Lilly

Search of the poster presentations at the 2026 ESMO conference for regulator and payer research focus (electronic and presented posters; total N=4014)

Word/term in poster title
Food and Drug Administration (FDA)
European Medicines Agency (EMA)
Regulator(y)
Health technology assessment (HTA)
Payer(s)
NICE
IQWiG
AMNOG
HAS
Number of posters
2
1
0
1
0
0
0
1
0
% of all available posters
0.05%
0.03%
0%
0.03%
0%
0%
0%
0.03%
0%
* results presented that relate to qualitative research with patients/patient engagement rather than qualitative research with HCPs and engagement with regulators, process, etc.

References

  1. SISAQOL. https://www.sisaqol.org/. Accessed October 2026.
  2. Proteus Consortium. OncoPRO Resources. https://theproteusconsortium.org/about-oncopro/oncopro-resources/. Accessed October 2026.
  3. Kim Y et al. Clinical outcome assessment trends in clinical trials- Contrasting oncology and non-oncology trials. Cancer Med. 2023;12(16):16945-16957.
  4. Gandara D et al. Prognostic value of patient-reported outcomes for survival in patients with advanced lung cancer receiving immune checkpoint inhibitors. Front Immunol. 2025;16:1640595.
  5. Gandara D et al. Composite patient-reported outcomes and risk prediction for overall survival in advanced non-small cell lung cancer with first-line cemiplimab. Front Oncol. 2026;16:1676687.
  6. Gandara D et al. Association Between Patient-Reported Outcomes and Overall Survival in Patients with Advanced NSCLC Treated with First-Line Cemiplimab-Based Therapy. Cancers (Basel). 2026;18(6):916.
  7. Militzer B et al. Integration of patient experience data (PED) into regulatory and payer decision-making. Chapter 12 in Reaney M & Skaltsa K (eds). Using Patient Experience Data to Evaluate Medical Interventions. 2026: New York: IQVIA
Sam Llewellyn

Matt Reaney

Chief Scientific Officer

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