How can patient-reported outcomes (PRO) and health-related quality of life (HRQoL) data inform regulatory decisions?
As part of the cancer pathfinder initiative, EMA and EORTC held a joint workshop on 29 February 2024 addressing How can patient-reported outcomes (PRO) and health-related quality of life (HRQoL) data inform regulatory decisions?. Participants included representatives from academia, learned societies, patients, regulatory agencies ((US FDA, PMDA, HC, Swissmedic), health technology assessment bodies (HTA), and industry.
Two twin articles accessible for free by registration to the journal as an “editorial discretion” have just been published in the Lancet oncology on 14 April 2025:
‘Using patient-reported outcomes and health-related quality of life data in regulatory decisions on cancer treatment: highlights from an EMA-EORTC workshop’. The article has been authored by speakers and chairs of the 2024 workshop with an agreement on the recommendations to move forward on concrete proposals.
‘Use of patient-reported outcomes to inform symptom and functional outcomes in cancer drug regulatory decisions: challenges and future directions’ providing the regulators position.
Key findings of the articles:
- Patient-reported outcomes such as symptoms or health-related quality of life are important criteria for the evaluation of cancer drugs. For PROs to be used meaningfully, they must be adequately planned, collected, evaluated and reported, just like all other endpoints.
- Considering what matters most to patients when selecting PROs is crucial.
- From an HTA perspective, it is particularly important that the instruments used to collect PROs enable a fair comparison between a new drug and the standard of care. This also applies across different studies (and over a longer period of time) so that indirect comparisons between different treatment options are possible (a method that is used more frequently by HTA than by regulatory authorities). It is also important for HTA that PROs are collected long enough (and beyond e.g. disease progression) to adequately assess the benefit of a treatment option.
- Initiatives like Setting International Standards in Analysing Patient-Reported Outcomes and Quality of Life Endpoints (SISAQOL) continue to address how PROs should be implemented to meet the needs of all stakeholders, ensuring the patient voice is incorporated in the decision-making process.
The EMA view on the use of PRO measures in oncology studies is described in the Appendix 2 to the guideline on the evaluation of anticancer medicinal products in man. The concept paper to revise the anti-cancer guideline has been adopted by CHMP in April 2025 and has been released for public consultation (the Revision 7 aims to keep the guideline aligned with evolving regulatory and scientific developments and to implement the estimands framework introduced by ICH E9(R1) addendum. Other changes include the addition of sections dedicated to haematological cancers, a revision of the guidance on single-arm trials, and on clinical trials in adjuvant, neoadjuvant and perioperative settings). Stakeholders can provide comments by 31 July 2025.
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