Evidence Generation for Targeted Therapy in Advanced Breast Cancer
Overview
A global pharmaceutical company with a targeted therapy for advanced breast cancer that demonstrated strong clinical efficacy sought to generate real-world evidence to support inclusion in NCCN clinical guidelines- a key driver for physician adoption and payer reimbursement. Despite strong clinical efficacy, the therapy faced slow uptake due to lack of real-world data on its impact across diverse patient populations. Without guideline inclusion, physicians were reluctant to prescribe the therapy over established standards of care, limiting its market penetration.
The company wanted to initiate a study that evaluates the clinical benefits of the targeted therapy compared to guideline recommended alternatives. Their goal was to publish the results in a peer-reviewed journal through engaging a reputable principal investigator. However, they faced challenges in acquiring data sources with the necessary depth to support the study. Without the ability to validate data coverage or assess feasibility upfront, the company struggled to make a business case to justify the investment.
Challenges
Limited Depth of Existing Data Sources: Existing datasets lacked longitudinal and deep datasets to generate robust evidence, particularly molecular and imaging data.
Resource Intensive: Finding, onboarding, and contracting with the right data sources and PI was time consuming and costly. Without knowing whether the necessary data existed to support their planned study, the team struggled to define a clear business case or justify the investment.
Standardization Issues: Data varied widely across institutions, requiring extensive manual curation before analysis.
Regulatory and Compliance Complexities: To comply with HIPAA, the company needed a privacy-preserved approach to data access that still granted the granularity needed.
Why They Chose datma
After evaluating multiple data providers, the pharma company selected datma based in part on two factors: upfront visibility and execution support. datma.FED enabled them to assess, in advance, whether participating sites held sufficient data coverage to support their proposed study design. This provided the internal evidence needed to justify their investment in moving forward with study execution.
Once feasibility was confirmed, datma facilitated seamless study execution by handling investigator identification, onboarding, documentation, ensuring a streamlined study execution from beginning to end, allowing the pharma team to focus on analysis rather than operational logistics.
Solution: datma.FED
datma.FED provided a federated, privacy-preserving infrastructure that enabled the pharmaceutical company to conduct a multi-site study. By leveraging health system data directly within their own environments, pharma could access real-world patient insights without requiring data transfers. The study incorporated structured EHR data, molecular data, and pathology images, allowing pharma to analyze treatment response. Additionally, datma handled the study setup, site engagement, and investigator onboarding, reducing operational burden on the pharma team. The findings provided robust- real-world data supporting the therapy’s effectiveness.