Work

Regulatory pathways for targeted and non-targeted oncology therapies at the European Medicines Agency

Year: 2026

Type: article

Abstract: BACKGROUND: The approval of oncology therapies has increasingly incorporated biomarker-driven precision medicine. However, the influence of mandatory biomarker selection on regulatory pathways remains poorly understood. We compared the regulatory characteristics of targeted and non-targeted oncology therapies first authorised in the European Union between 2015 and 2025. METHODS: We conducted a retrospective analysis of drug-indication pairs for malignant solid tumours first authorised following EMA assessment and European Commission marketing authorisation between January 2015 and December 2025. Therapies were classified as targeted when mandatory biomarker testing was required for patient selection in the approved indication. Regulatory characteristics and timelines were obtained from publicly available EMA information. The primary regulatory outcome was Conditional Marketing Authorisation (CMA). RESULTS: Eighty drug-indication pairs were included: 45 targeted (56.3%) and 35 non-targeted (43.8%). No significant linear temporal trend in the proportion of targeted therapies was detected (OR per year 1.15; 95% CI 1.00-1.33; p = 0.051). CMA was granted to 42.2% of targeted versus 5.7% of non-targeted therapies (OR 12.06; 95% CI 2.57-56.53; p < 0.001). The association remained directionally consistent across sensitivity analyses accounting for calendar year, pharmacological class, tumour type, and multiple indications within the same application. No significant difference in total elapsed time to marketing authorisation was detected (median 419 vs 427 days; median difference - 8days; 95% bootstrap CI -52-21). CONCLUSIONS: Targeted oncology therapies requiring mandatory biomarker selection were substantially more likely than non-targeted therapies to receive CMA, although the magnitude of the association was imprecisely estimated. POLICY SUMMARY: Greater use of CMA among targeted therapies means that more indications enter the post-authorisation period with confirmatory evidence obligations outstanding. This may be relevant to HTA and reimbursement decisions and warrants longitudinal research on how these obligations and the evidence base evolve after authorisation.

Source: Journal of Cancer Policy

DOI: 10.1016/j.jcpo.2026.100830

Authors: G. Puig, I. Danés, C. Sans-Pola, A. Vallano