“RCTs are a foundation—but not the future.”
As tumour-agnostic therapies and rare molecular targets become central to modern oncology, the limitations of traditional evidence pathways and reimbursement models have become increasingly apparent. Randomised controlled trials (RCTs), while essential, are not always feasible or sufficient for therapies targeting ultra-rare biomarkers. Health systems must therefore re-evaluate how evidence is generated, validated, and translated into access.
This session explores the shift toward innovation-ready oncology systems that accommodate real-world evidence, tissue-agnostic regulatory approvals, and universal genomic testing. Case studies from Sweden and Austria, where national strategies have institutionalised molecular profiling at diagnosis, will be presented alongside EU-wide initiatives to align EMA regulatory pathways with real-time data from national registries.
Speakers will discuss the integration of ESMO Scale for Clinical Actionability of molecular Targets (ESCAT) into clinical guidelines, the role of national biobanks in stratified care, and the policy levers needed to make genomic profiling routine for patients with advanced cancers. The session will also tackle equity gaps in access to precision oncology tools across Member States and propose solutions to ensure that tumour biology, not postcode, determines treatment eligibility.
Reflecting EAPM’s work on biomarker access and genomics in public health, this discussion will advance the conversation on how health systems can balance flexibility and rigour in regulatory decision-making, embed profiling into reimbursement pathways, and monitor outcomes through real-world implementation frameworks.
Conclusion:Precision oncology must be supported by precision policy. Only by aligning regulatory agility, reimbursement innovation, and genomic equity can Europe ensure that all patients benefit from the full potential of personalised cancer care.
