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The biomarker validation gap in precision oncology
Biomarker validation definition
Biomarker validation demonstrates that a biomarker can reliably measure or predict a clinically meaningful outcome. It also shows that the biomarker can support decision-making in research, regulatory submissions, and routine care. In precision oncology, validation is critical. Biomarkers must demonstrate clinical validity, clinical utility, and real-world applicability before they can influence treatment decisions or gain adoption across healthcare systems.
Prostate cancer: one of precision oncology’s largest opportunities
According to a 2025 analysis published in European Urology, more than 1.4 million men are diagnosed with prostate cancer each year worldwide. As a result, it remains the second most commonly diagnosed cancer in men.
In Europe, prostate cancer continues to place a significant burden on healthcare systems. Consequently, demand for improved screening, diagnostics, and precision treatment strategies remains high. Population-level analyses published in The BMJ and data from the European Cancer Information System (ECIS) highlight the sustained impact of prostate cancer on healthcare systems across the continent.
Advances in pathology, imaging, surgery, radiotherapy, and systemic treatments have improved patient care. However, predicting how an individual patient’s disease will progress remains difficult.
Patients with seemingly similar clinical and pathological characteristics can experience dramatically different outcomes. Some remain stable for years under active surveillance, while others progress and require earlier intervention.
As a result, clinicians, researchers, and pharmaceutical companies need better ways to identify which patients are likely to progress, respond to treatment, or remain stable over time. This challenge has made biomarkers a major focus in precision oncology. Researchers continue to explore their potential to improve patient stratification, predict disease progression, and support treatment decisions
Why biomarkers matter in prostate cancer
Traditional prostate cancer risk assessment relies on pathology findings, PSA measurements, imaging results, and clinical evaluation. While these tools remain essential, they do not always show how an individual patient’s disease will progress over time.
Biomarkers can complement conventional assessment by providing additional biological insights into disease behaviour. Depending on their intended use, they can help predict disease progression, identify patients most likely to respond to treatment, support active surveillance decisions, and improve risk stratification.
As a result, biomarkers may help answer some of the most important questions in prostate cancer care:
- Which patients can safely remain under active surveillance?
- Which patients are at higher risk of disease progression?
- Which patients are most likely to benefit from a specific therapy?
- Which patients may require earlier intervention?
The value of biomarkers also extends to drug development. For pharmaceutical companies, validated biomarkers can improve patient selection, strengthen clinical trial design, support companion diagnostic development and generate evidence for regulatory and reimbursement decisions.
Researchers have identified numerous promising biomarkers. The greater challenge lies in demonstrating clinical validity and generating the evidence required for adoption by clinicians, regulators, health technology assessment bodies and healthcare systems.
Generating real-world evidence requires access to large, high-quality datasets. These datasets must combine clinical, pathology, imaging, and molecular data collected consistently across institutions and over long follow-up periods.
Why the pharmaceutical industry is investing in biomarker-driven oncology
Validated biomarkers can:
- Identify patients most likely to benefit from a therapy
- Support patient selection and trial enrichment strategies
- Improve clinical trial efficiency and statistical power
- Enable companion diagnostic development
- Strengthen regulatory and health technology assessment submissions
- Support market access and reimbursement discussions
- Increase confidence in precision medicine approaches
A growing market driving investment
Prostate cancer remains one of the most active areas of oncology research and pharmaceutical investment. Industry analyses suggest that more than 140 companies are currently developing over 150 prostate cancer therapies. These efforts span the full disease spectrum, from localized disease and active surveillance to advanced and metastatic prostate cancer.
Industry investment continues to grow
Major pharmaceutical companies continue to invest heavily in biomarker-guided approaches to prostate cancer. AstraZeneca, Bayer, Johnson & Johnson, Pfizer, Astellas and Novartis are among the companies developing therapies and precision medicine programs that rely on molecular testing, patient stratification and companion diagnostics.
The biomarker validation gap in precision oncology
Despite growing investment in biomarker research, many promising candidates never reach routine clinical practice. Researchers have identified numerous potential biomarkers, but generating the clinical, technical and regulatory evidence required to validate them and support adoption across healthcare systems remains a significant challenge.
Robust biomarker validation depends on large longitudinal datasets that combine clinical records, pathology findings, imaging data, genomic information and patient outcomes. These datasets must be collected consistently, standardized across institutions and made suitable for robust analysis.
Biomarker developers also face growing expectations from regulators, healthcare providers and health technology assessment bodies. Evidence must demonstrate not only analytical performance, but also clinical validity, clinical utility and real-world applicability.
Addressing these challenges requires more than individual studies. It requires a framework that can connect biomarker research, evidence generation, data infrastructure and implementation pathways.
Why validation is becoming the real bottleneck in precision oncology
The next phase of precision oncology will not be defined by biomarker discovery alone. It will be defined by whether healthcare and life sciences organizations can generate the evidence needed to demonstrate that biomarkers are clinically valid, clinically useful, and applicable across real-world patient populations.
Generating that evidence remains challenging because candidate biomarkers are emerging faster than the infrastructure needed to validate them. Single-site studies may provide important insights, but they often lack the scale, diversity, longitudinal follow-up, and multimodal context required to support regulatory confidence, reimbursement decisions, and routine clinical adoption.
For biomarker developers, the strategic question is no longer simply whether a marker can be discovered. It is whether clinical, molecular, imaging, and outcomes data can be connected, harmonized, and analyzed in a trusted way across institutions. Organizations that can combine real-world evidence, multimodal data, and secure analytics will be better positioned to move promising biomarkers from research into clinical impact.
This is the challenge that BRECISE was created to address.
BRECISE: bridging the gap between biomarker discovery and clinical adoption
BRECISE (Biomarker Research and Evaluation for Clinical Implementation and Supporting Systems Enhancement) was established to address this challenge. Funded through the Innovative Health Initiative (IHI) and Horizon Europe, the €22.9 million project brings together 25 partners from 15 European countries to accelerate the clinical validation and implementation of next-generation biomarkers in oncology.
The consortium combines expertise from healthcare providers, academic institutions, pharmaceutical companies, technology partners, regulators and health technology assessment organizations. Together, these stakeholders are working to create a more structured pathway for moving biomarkers from research environments into routine clinical use.
Building the evidence foundation for biomarker validation
For the prostate cancer use case, BRECISE partners are evaluating biomarkers that could improve risk stratification and support clinical decision-making for patients managed through active surveillance. One of the project’s clinical use cases focuses on validating the PDE4D7 biomarker to help identify patients at higher risk of progression and reduce unnecessary biopsies.
Researchers must be able to link biomarker findings with clinical outcomes across large and diverse patient populations. This requires access to longitudinal clinical, pathology, imaging, and molecular data collected consistently across multiple healthcare settings.
As a consortium partner, BC Platforms contributes expertise in data integration, harmonization, and evidence generation, helping create the evidence foundation needed to evaluate biomarker performance across diverse patient populations and healthcare settings.
Access to sufficiently large and representative patient cohorts remains a major barrier to biomarker validation. In addition to its data integration and harmonization expertise, BC Platforms contributes its partner network to help support access to relevant clinical and molecular datasets.
This combination of data access, standardized data models and multimodal data integration helps generate the evidence required to evaluate biomarker performance, support regulatory decision-making and accelerate clinical adoption.
Conclusion: from biomarker discovery to clinical impact
The oncology industry has made remarkable progress in biomarker discovery. Yet discovery alone is not enough. To influence clinical decisions, support regulatory approval and enable widespread adoption, biomarkers must be validated against high-quality evidence generated across diverse patient populations.
Prostate cancer highlights both the opportunity and the challenge facing precision oncology. Biomarkers have the potential to improve patient stratification, predict disease progression and support more personalized treatment decisions, but realizing that potential depends on robust clinical validation.
Initiatives such as BRECISE demonstrate the importance of collaboration between healthcare providers, researchers, pharmaceutical companies, technology partners and regulators. By combining clinical expertise, data integration and evidence generation, the consortium aims to accelerate the transition of promising biomarkers from research environments into routine clinical practice.
As precision oncology continues to evolve, organizations that can connect biomarkers, data and evidence generation at scale will be best positioned to transform scientific discovery into meaningful clinical impact.
Better decisions start with the right evidence foundation
Validate biomarkers, accelerate precision oncology research and generate real-world evidence with access to bespoke datasets, custom evidence networks and secure analytics across the product lifecycle.
FAQs
Biomarker validation is the process of demonstrating that a biomarker consistently predicts or measures clinically meaningful outcomes and can be reliably used in research or clinical practice.
Validation helps ensure that biomarkers support accurate patient stratification, treatment selection, and clinical decision-making before they are adopted in routine care.
BRECISE is a Horizon Europe and Innovative Health Initiative (IHI) project that brings together 25 partners across 15 European countries to accelerate biomarker validation and implementation in prostate and bladder cancer.
Real-world data helps researchers evaluate biomarker performance across large and diverse patient populations, generating evidence needed for clinical adoption and regulatory decision-making.
Real-world evidence helps demonstrate whether a biomarker performs consistently across routine clinical settings, diverse patient populations, and longer follow-up periods. This can strengthen confidence in clinical utility, regulatory evidence packages, and adoption by healthcare systems.
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