The Approval Process of Personalized Medicine for Chemotherapy in European Union

Personalized medicine (PM) has transformed oncology by tailoring chemotherapeutic interventions to the genetic, molecular, and clinical characteristics of individual patients. Within the European Union (EU), the regulatory framework for such innovative therapies is complex, involving coordination between the European Medicines Agency (EMA) and the In Vitro Diagnostic Regulation (IVDR) (EU 2017/746) for companion diagnostics (CDx). This review provides an in-depth overview of the approval pathway for personalized chemotherapy in the EU, covering regulatory mechanisms, clinical trial regulations, and adaptive approval schemes such as PRIME, conditional marketing authorization (CMA), and accelerated assessments. The EMA’s centralized authorization process, combined with the Clinical Trials Regulation (EU No 536/2014), ensures consistent evaluation and transparency across Member States. Ethical, scientific, and logistical challenges—such as data privacy under the General Data Protection R egulation (GDPR), evidence generation for small biomarker-defined populations, and disparities in patient access—remain major hurdles. Comparative insights between EU and US frameworks highlight the global trend toward harmonized, evidence-based regulatory science. Future developments, including the European Health Data Space (EHDS), DARWIN EU®, and artificial intelligence (AI)-driven pharmacovigilance, are expected to streamline the assessment of personalized chemotherapies. Overall, this review emphasizes that the EU’s evolving regulatory ecosystem continues to balance innovation, patient safety, and equitable access in the era of precision oncology.