Navigating Complexity: Rescuing a Biomarker-Driven Breast Cancer Trial

Conducting global oncology trials in rare, genetically defined populations is a formidable challenge—one that demands operational agility, scientific rigor, and deep collaboration.

This case study explores how Precision rescued a multinational, randomized, double-blind Phase 2–3 trial evaluating a targeted therapy in patients with locally advanced or metastatic breast cancer (mBC) characterized by a specific genetic mutation. The story highlights the strategic approaches, operational hurdles, and key learnings that shaped the trial’s success.

Study Overview

The trial enrolled over 400 patients across more than 140 sites in 16 countries, spanning North America, Europe, APAC, and Australia. The study design included:

• Phase 2: Focused on objective response rate (ORR) across two cohorts, with sites in France, Germany, Spain, the UK, and the US.
• Phase 3: Expanded to progression-free survival (PFS) as the primary endpoint, with sites in 16 countries and a broader patient population.

Precision’s involvement ranged from additional site identification, CRO coordination through rescue of project management activities, plus health informatics integration and clinical study report (CSR) preparation.

Key Challenges

1. Slow Enrollment in a Niche Population

Recruiting patients with a specific genetic mutation who had already received prior therapy for metastatic disease proved challenging. Enrollment rates were modest—0.10 patients/site/month in Phase II and 0.09 in Phase III—reflecting the complexity of the population and the competitive landscape.

2. Rescuing Global Coordination Across Regions

Rescuing the study and managing three CROs across diverse geographies required robust communication, harmonized processes, and a shared commitment to data integrity and timely reporting.

3. Site Education and Readiness

Educating sites on the investigational therapy’s unique mechanism of action was essential to reinvigorate the study. Precision leveraged genomic sequencing data and health informatics platforms to match patients to trials, ensuring sites were both scientifically prepared and operationally ready.

Precision’s Strategic Approach

Leveraging Networks and Informatics

• Site Networks: Precision’s established relationships with oncology sites and key opinion leaders (KOLs) enabled rapid additional site activation and predictable startup processes for study expansion.
• Health Informatics: Access to ICD-10 claims data, genomic sequencing databases, and competitive landscape analysis helped identify new sites with the right patient population and operational capacity.
• CRO Coordination: Precision rescued global coordination and managed three CROs, ensuring consistent execution and alignment across regions.

Operational Excellence

• Competitive Landscape Analysis: Understanding site-level experience and competition was as important as trial staff expertise, allowing for targeted site selection and efficient enrollment
• CSR and Regulatory Support: Precision’s involvement in the CSR process and regulatory preparation streamlined trial documentation and submission.

Outcomes and Impact

The trial met its primary endpoint, demonstrating the clinical value of targeted therapy in a genetically defined population. Precision’s integrated approach enabled operational agility and success across diverse geographies, validating the importance of strategic site selection, genomic targeting, and collaborative execution.

Lessons Learned

• Strategic Relationships Matter: Access to KOLs and established site networks is critical for niche populations.
• Data-Driven Patient Identification: Health informatics and genomic data integration streamline patient matching in biomarker-driven studies.
• Operational Agility is Key: Rescuing global trials requires flexible processes, robust coordination, and a willingness to adapt to evolving challenges.
• Site-Level Experience Counts: Trial success depends not only on staff expertise but also on site-level competitive dynamics and readiness.

Winning the Battle in Biomarker-Driven Trials

This global, biomarker-driven breast cancer trial underscores just how much expertise, collaboration, and adaptability matter when the stakes are high and a Co-ordinating CRO change is enabled mid-study. From navigating slow enrollment and complex logistics to harnessing the power of health informatics and strategic site selection, every step required precision and partnership. The lessons learned here—about the importance of data-driven approaches, operational agility, and trusted relationships—continue to shape how we tackle genetically defined cancers.

As we look ahead, the commitment to innovation and excellence remains at the heart of every trial. If you’re interested in learning more about our approach or exploring how strategic partnerships can drive success in your own studies, click the button below to learn more about Precision Oncology services or connect with our team to get started.