HEARD BUT NOT SEEN: PATIENT AND CLINICIAN INPUT IN THE FIRST ORPHAN JCA
Author(s)
Dávid Márk Gyorbiró, MSc1, Amira Marzouk, MSc2, Krzysztof Kloc, MSc3, Mondher Toumi, MSc, PhD, MD4.
1Clever Access, Kraków, Poland, 2Clever-Access, Tunis, Tunisia, 3Clever-Access, Kraków, Poland, 4Aix-Marseille University, Marseille, France.
1Clever Access, Kraków, Poland, 2Clever-Access, Tunis, Tunisia, 3Clever-Access, Kraków, Poland, 4Aix-Marseille University, Marseille, France.
OBJECTIVES: Evidence-based medicine rests on a tripod of evidence, clinicians, and patients. Does the JCA? Using the first orphan oncology JCA (tovorafenib for paediatric low-grade glioma), this analysis examined whether documented patient and clinician input visibly shaped the assessment.
METHODS: A case analysis examined how carer and clinical-expert input was solicited, recorded, and used in the published JCA report, and assessed the weight given to evidence relative to clinician and patient contributions across scoping and assessment.
RESULTS: Input from a carer and clinical experts was solicited and documented at the scoping and draft-report stages, satisfying the procedural requirement. However, the report does not show that this input changed any assessment conclusion: contributions were recorded but their influence was not traced. The assessment leaned heavily on the evidence leg of the tripod, with limited visible room for clinician and patient experience, and the factual-accuracy stage further restricted how non-evidentiary perspectives could enter. In a paediatric rare cancer — where lived experience and clinical judgement are especially informative about meaningful outcomes — input that is heard but not demonstrably weighed risks becoming procedural rather than substantive. Whether participation altered the assessment cannot be determined from the published output.
CONCLUSIONS: Participation should be visible in the result, not just the record. Documenting how patient and clinician input was weighed — and where it changed conclusions — would make engagement meaningful and align the JCA more closely with evidence-based medicine.
METHODS: A case analysis examined how carer and clinical-expert input was solicited, recorded, and used in the published JCA report, and assessed the weight given to evidence relative to clinician and patient contributions across scoping and assessment.
RESULTS: Input from a carer and clinical experts was solicited and documented at the scoping and draft-report stages, satisfying the procedural requirement. However, the report does not show that this input changed any assessment conclusion: contributions were recorded but their influence was not traced. The assessment leaned heavily on the evidence leg of the tripod, with limited visible room for clinician and patient experience, and the factual-accuracy stage further restricted how non-evidentiary perspectives could enter. In a paediatric rare cancer — where lived experience and clinical judgement are especially informative about meaningful outcomes — input that is heard but not demonstrably weighed risks becoming procedural rather than substantive. Whether participation altered the assessment cannot be determined from the published output.
CONCLUSIONS: Participation should be visible in the result, not just the record. Documenting how patient and clinician input was weighed — and where it changed conclusions — would make engagement meaningful and align the JCA more closely with evidence-based medicine.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
PCR27
Topic
Patient-Centered Research
Topic Subcategory
Patient Engagement
Disease
Oncology, Pediatrics