The European Commission published on Tuesday 9 June the first joint clinical assessment report carried out under the Regulation on health technology assessment (HTA), applicable since January 2025. Approved by all Member States, the document concerns tovorafenib (Ojemda), a treatment developed by the French pharmaceutical company Ipsen for certain low-grade paediatric gliomas, the most common form of brain tumour in children.
Joint clinical assessments allow Member States to cooperate in examining the clinical benefits of new medicines and certain medical devices. According to the European Commission, this mechanism should help reduce duplication between national authorities and facilitate decisions on patients’ access to innovative treatments.
18 joint clinical assessments concerning new cancer treatments and innovative therapy medicines have already been launched since the Regulation entered into application.
The medicine assessed is intended for patients aged six months and over presenting certain alterations of the BRAF gene and whose disease has progressed after at least one previous systemic treatment. The European Commission granted it a conditional market authorisation on 20 April, following the scientific assessment by the European Medicines Agency (EMA).
Only one comparison retained. The assessment group notes that, for the majority of patient populations and comparators retained, no comparative data were available. The results are based mainly on FIREFLY-1, an open-label, multi-centre, non-randomised and non-comparative phase II study.
The only comparative results included concern children over one year of age with a low-grade paediatric glioma associated with a BRAF V600E mutation. Tovorafenib was compared with the dabrafenib-trametinib combination by means of an indirect comparison between studies.
However, the authors of the report identify several limitations in the available data. On several occasions, they indicate that the results presented are associated with “a number of major uncertainties” and that they should “not necessarily be interpreted as causal effects of the treatment”.
The report also points to several shortcomings in certain analyses submitted by the medicine’s developer and considers that these elements must be taken into account when interpreting the results.
Read the report: https://aeur.eu/f/m9a (Original version in French by Nithya Paquiry)