EU responses examine first published JCA report

Responses to the first published EU joint clinical assessment report have focused less on the individual medicine than on how the new process handles evidence uncertainty and how Member States may use the report in later national decisions.

The European Commission published the joint clinical assessment report on tovorafenib, Ojemda, on 9 June 2026. The package included the report, the summary report, the developer dossier and the annex covering the factual accuracy check and assessors’ response. The report had been endorsed by the Member State Coordination Group on Health Technology Assessment, HTACG, on 30 April 2026 and had passed Commission procedural review on 19 May 2026.

Ipsen, the marketing authorisation holder, welcomed completion of the assessment, but placed the report in the clinical context of relapsed or refractory BRAF-altered paediatric low-grade glioma, where patient numbers are small, disease biology is heterogeneous and treatment practice is not defined by a single established comparator.

The assessment scope contained eight PICO questions. Ipsen submitted single-arm clinical data and indirect comparative analyses for two of the eight questions. The report used one of the indirect comparisons, while treating it as highly uncertain, and did not use the other because the comparator study was not documented in enough detail. Ipsen argues that the limited comparative evidence reflects the small, heterogeneous paediatric rare-disease population and the absence of a clear standard comparator in much of the indication.

The assessment scope included quality of life and several symptom and functional outcomes, but some endpoints could not be assessed for the relevant PICO because results were not available in the dossier for the assessed population. Carer comments in the annex also noted the absence of quality-of-life data and limited discussion of growth and developmental effects.

The European Federation of Pharmaceutical Industries and Associations, EFPIA, focused on the link between the EU report and national processes. It welcomed completion of the first report after 14 months of work and called on Member States to use the joint assessment without duplicating it in national clinical review. EFPIA’s response places the first report within the original purpose of the EU HTA Regulation: reducing repeated clinical assessment while leaving economic evaluation and reimbursement decisions at national level.

The European Confederation of Pharmaceutical Entrepreneurs, EUCOPE, focused on the demands placed on developers during the process. It raised concerns about the number of PICO questions, limited interaction between companies and assessors, and the need for clearer handling of orphan-specific evidence constraints before orphan medicines enter mandatory JCA from 13 January 2028.

Patient organisations responded through a joint statement signed by 15 European patient organisations, including the European Patients’ Forum. The statement welcomed the involvement of a carer in scoping and draft-report review, and called for earlier patient and carer input, clearer reporting of how patient evidence affects the assessment, stronger involvement of patient organisations and summary reports that are easier to use across the EU.

The joint statement also argued that patient and carer input should be reflected more clearly in the assessment record. The patient organisations called for greater attention to quality of life, treatment burden and other outcomes that may be prominent for patients and families, particularly where conventional comparative evidence is limited.

Specialist access commentary has noted that the assessment leaves substantial interpretation for national authorities. The report used only one of the two submitted indirect comparisons, and treated that comparison as highly uncertain. National HTA bodies will therefore still have to decide how to handle missing or incomplete comparative evidence when they apply the JCA report in their own systems.

The first responses indicate the issues likely to recur as the process scales. Developers will need to prepare for a broader set of comparator questions at EU level. Member States will need to decide how far the shared clinical assessment can replace national clinical review. Patient organisations are pressing for their input to be reflected in the assessment record, not only in the procedure leading to it. The tovorafenib report provides the first working example of that shared evidence record, but its reception shows that national interpretation of uncertainty remains a major part of the access pathway.

Source: European Commission; Ipsen; EFPIA; EUCOPE; European Patients’ Forum
Link:
Joint clinical assessment report on tovorafenib (Ojemda);
Ipsen welcomes the European Union’s publication of the Joint Clinical Assessment for Ojemda® (tovorafenib);
EFPIA welcomes the publication of the very first Joint Clinical Assessment (JCA) report developed under the frame of the EU HTA Regulation;
EUCOPE on First Joint Clinical Assessment (JCA) Report: Critical Milestone – Readiness for Orphan Drug Assessments Needed;
Patient Organisations Welcome Publication of First Joint Clinical Assessment Under EU HTA Regulation
Date: 9 June 2026 and 17 June 2026