A multi-country longitudinal cohort enabled biomarker-defined population insights, regulatory readiness, and launch planning planning
Executive summary
A global biopharmaceutical company developing targeted oncology therapies partnered with BC Platforms to generate real‑world evidence (RWE) to support the development and planned launch of a biomarker‑driven therapy in non‑small cell lung cancer (NSCLC).
The work focused on how patients were identified and treated in clinical practice, with particular attention to the testing‑to‑treatment cascade, including when biomarker testing occurs, how results are returned, and whether results are available at the time of treatment decision. These factors directly influenced time to treatment and access to targeted therapies.
The objective was to address key evidence gaps relevant for HTA and EU Joint Clinical Assessment (EU JCA), including biomarker testing uptake, patient identification, and treatment pathways and sequencing across lines of therapy, to support launch readiness, scientific engagement, and evidence strategy.
To achieve this, BC Platforms designed and operationalised a multi‑country cohort combining data feasibility assessment, cohort build, and structured data delivery with ongoing refreshes, creating a scalable, regulatory‑relevant evidence asset to support decision‑making across the product lifecycle.
Biomarker testing, cohort identification, treatment pathways, and sequencing across lines of therapy
Use cases
Regulatory and HTA evidence (including EU Joint Clinical Assessment), launch strategy
Design
Ongoing data refreshes
Key metrics
3
countries
12
data partners
~1,200
patients
Background
Treatment decisions for NSCLC depend on identifying actionable molecular subpopulations, requiring timely biomarker testing and integration of results into clinical decision-making.
In real-world clinical practice, biomarker testing pathways varied across countries and institutions, including how tests are ordered, performed, and returned. Differences in testing rates, turnaround times, specimen availability, and whether results are available at the time of treatment decision directly affected how patients were identified for targeted therapies.
Real-world data also reflected incomplete or inconsistently captured biomarker information, creating challenges in accurately identifying molecularly defined populations.
These factors made it difficult to understand biomarker-defined populations and their management in routine care, particularly across European markets where heterogeneity in diagnostic and treatment pathways across health systems was high.
To support the development and planned launch of a targeted therapy, our client, a leading global pharmaceutical company, required a longitudinal view of biomarker-defined NSCLC patients across stages I to IV, including cohort identification, treatment pathways and sequencing across lines of therapy, and patient management in clinical practice.
Challenge
Limited visibility into biomarker testing and patient identification
Biomarker testing practices varied across countries and institutions in terms of availability, timing, and integration into the care pathway. Differences in when testing was performed and whether results were available at the time of treatment decision directly affected patient identification for targeted therapies, which limited the company’s ability to reliably estimate biomarker-defined patient populations and understand how testing translates into treatment decisions in clinical practice.
Beyond single biomarkers: defining clinically meaningful molecular subpopulations
Molecular characteristics, including co-mutation patterns, can influence disease progression and treatment response in NSCLC. However, these data were not consistently captured or structured across real-world data sources, with varying levels of completeness and missing data across countries.
As a result, defining clinically meaningful molecular subpopulations beyond single biomarkers and ensuring consistency across countries remained challenging, hindering the ability to generate evidence aligned with payer and regulatory requirements.
Lack of cross-country comparability
Relevant data was distributed across multiple sites and countries, with differences in data structure, completeness, clinical documentation practices, and regulatory and consent requirements. This made it difficult to generate consistent and comparable evidence across countries, and limited analysis of treatment pathways and outcomes for cross-country decision-making and HTA use.
Meeting regulatory and HTA evidence requirements
To support HTA and regulatory engagement, the client required longitudinal patient-level data across disease stages, consistent cohort definitions, and evidence that could withstand regulatory scrutiny. Ensuring consistent follow-up, cohort definitions, and endpoint calculation across countries remained a key challenge.
Solution
Designing a multi-country real-world evidence cohort
BC Platforms partnered with our client to design and operationalize a multi-country retrospective observational cohort study of biomarker-defined NSCLC patients across the UK, Germany, and Spain, with potential expansion to additional European markets.
The cohort captured patient journeys across clinical trial stages I to IV, enabling analysis of biomarker testing, treatment pathways and sequencing across lines of therapy, and clinical outcomes over time.
Assessing data feasibility and cohort viability
BC Platforms consultants worked with the client to translate the existing study protocol into a fit‑for‑purpose real‑world data model, ensuring that required variables, cohort definitions, and outcomes could be supported by available data across countries.
Leveraging BC Platforms’ European data partner network and understanding of local regulatory landscapes, the team assessed site readiness, data availability, and cohort viability, aligning operational feasibility with scientific objectives and HTA‑relevant expectations.
Cohort build, data harmonization, and initial delivery
BC Platforms enabled secure access to real‑world oncology data across multiple European countries and ingested data from participating sites into a standardized common data model.
Longitudinal data from EHRs and lab and pathology systems — including biomarker information where available — were harmonized to support consistent cohort definitions and cross‑country comparability. Data quality controls and validation checks were applied to ensure reliability for regulatory and HTA‑relevant analyses.
Longitudinal follow-up and continuous evidence generation
The cohort was designed as a longitudinal real‑world evidence asset, rather than a one‑time data extract. Patients are followed over time, enabling repeated observation of biomarker testing, treatment pathways, sequencing across lines of therapy, and outcomes as they evolve in routine clinical practice.
Using BC Platforms’ trusted research environment (TRE) and harmonized data model, the study supports planned data refreshes that incorporate newly diagnosed patients and are updated follow‑up for existing patients. This enables ongoing descriptive and time‑to‑event analyses, where appropriate, and supports evidence generation across the product lifecycle.
This approach provides a durable, scalable evidence foundation that can adapt as biomarker use, treatment standards, and clinical practice change over time.
Governance, compliance, and secure data access
Data were accessed and analyzed within BC Mosaic, BC Platforms’ TRE, a secure and controlled research environment used for real‑world evidence generation. The TRE supports de‑identified patient‑level data, role‑based access controls, and full audit trails, and operates in compliance with GDPR and applicable local regulatory requirements.
Standardized data structures and documentation enabled consistent cross‑country analyses suitable for HTA and regulatory use.
Impact
Quantifying biomarker-defined patient populations
The cohort provided robust insight into real‑world biomarker testing practices and patient identification across European markets. This gave clearer visibility into testing uptake, timing, and the prevalence of biomarker‑defined subpopulations, reducing uncertainty around the true addressable patient population and improving confidence in population sizing assumptions.
Supporting HTA and regulatory evidence strategy
The structured, longitudinal dataset provided an evidence base aligned with HTA and regulatory expectations, including cross-country comparability and patient-level longitudinal data. This supports EU Joint Clinical Assessment and national HTA engagement, strengthening the ability to demonstrate unmet need, define target populations, and provide real-world context on treatment pathways, sequencing, and comparators across markets.
Informing launch and market strategy
By providing a clear view of treatment pathways and sequencing across lines of therapy – and how patients moved from testing to treatment – the cohort supported critical launch decisions, including market prioritization, evidence generation strategy, and scientific positioning. It enabled a clearer understanding of real-world patient identification and treatment dynamics and where gaps in testing or treatment pathways may impact access to targeted therapies.
These insights also supported more targeted scientific engagement, helping Medical Affairs teams refine country-level narratives, identify evidence gaps, and align internal stakeholders.
Establishing a longitudinal evidence asset for ongoing use
The cohort created a reusable, multi-country real-world evidence asset that can be updated over time to reflect changes in biomarker testing, treatment pathways, and clinical outcomes. In doing so, teams can generate vital evidence across the product lifecycle, including post-launch monitoring and future oncology research.
Why BC Platforms
For this project, BC Platforms combined deep oncology and real-world evidence expertise with the capabilities required to deliver a longitudinal, multi-country patient-level dataset across Europe.
BC Platforms leveraged a strong European data partner network and experience navigating country‑specific regulatory and access requirements to enable data collection across key markets. Working from the client’s existing protocol, BC Platforms ensured that the data collected, structured, and delivered were aligned with defined research questions, scientific objectives, and HTA‑relevant expectations.
Through the use of a secure TRE, harmonized data structures, and a common data model, BC Platforms enabled consistent cohort definition, cross‑country comparability, and ongoing dataset refreshes — supporting regulatory engagement, HTA readiness, and launch planning over time.
Conclusion
Generating robust real-world evidence on biomarker-defined NSCLC populations is challenging, particularly across heterogeneous European healthcare systems.
By establishing a longitudinal, multi-country clinical evidence base, our client gained a clearer understanding of real-world patient populations and treatment dynamics, and could make more confident decisions across HTA, launch planning, and evidence strategy. This project provides a foundation to adapt as biomarker use, treatment pathways, and clinical practice continue to evolve.
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