Case Study: Delivering Pivotal Data in Phase 3 R/R Multiple Myeloma

Multiple myeloma (MM) remains incurable, with patients frequently progressing through multiple lines of therapy. In relapsed/refractory cases (RRMM), treatment options narrow significantly—especially in later lines. This global Phase 3 trial evaluated a novel regimen against standard care (Vd) in patients with RRMM. 

This case study explores how the sponsor and its partners successfully executed a complex, multinational trial involving >400 patients across 150 sites in 18 countries. Despite the challenges of global coordination, regulatory variability, and a high-risk patient population, the study achieved full enrollment and delivered pivotal data that supported regulatory approval and expanded treatment options for patients with RRMM. 

How the Trial Was Executed Across 18 Countries 

Executing a randomized, comparator-controlled trial across 150 sites in 18 countries required precision planning and operational agility. The sponsor partnered with regional CROs and deployed a hybrid site management model to accelerate activation and maintain enrollment momentum. 

Critical Challenges in RRMM Trial Execution 

• Rapid Disease Progression: Patients needed to be screened and randomized quickly to avoid missing therapeutic windows. 

• Complex Inclusion Criteria: Many patients were in third or fourth line of therapy, increasing screen failure risk. 

• Global Variability: Referral pathways, standard-of-care practices, and regulatory timelines differed across regions. 

To meet these challenges, the sponsor partnered with global CROs and deployed a hybrid site management model, leveraging regional expertise to accelerate activation and enrollment. 

Key Milestones in a Global Oncology Program 

The study launched in May 2017 and completed primary data collection in February 2020. Over the course of nearly three years, the study achieved several critical milestones: 

Precision for Medicine managed 120 of the 150 sites, primarily in Europe and Australia, while the sponsor oversaw North American operations. 

Precision Strategies That Enabled Trial Success in RRMM 

Hybrid Site Management Accelerated Global Activation 

A dual oversight model allowed regional CROs to tailor contracting, regulatory submissions, and site engagement to local requirements. This approach reduced startup timelines and supported sustained enrollment across geographies. 

Real-Time Enrollment Monitoring Informed Resource Allocation 

Dynamic dashboards tracked screening, randomization, and dropout rates. These insights enabled the sponsor to shift resources toward high-performing regions and deprioritize underperforming sites—maintaining enrollment velocity and geographic balance. 

Protocol Flexibility Reduced Screen Failures 

To accommodate diverse treatment paradigms, the protocol allowed flexibility in prior lines of therapy and disease staging. This broadened eligibility and improved enrollment rates, especially in regions with differing standards of care. 

Managing Data Across Borders and Teams 

Global Team Alignment Through Shared Tools and Governance 

With multiple CROs and sponsor teams involved, expectation management was critical. Weekly cross-functional meetings, shared trackers, and clear escalation pathways ensured transparency and timely issue resolution. 

• Weekly global data review meetings 

• Shared dashboards for enrollment and data cleaning 

• Defined escalation protocols for site-level issues 

Regulatory Coordination Across 18 Countries 

Early planning and local expertise were essential to navigate country-specific ethics and health authority requirements. Harmonized templates and centralized oversight ensured consistency across submissions. 

• Country-specific timelines mapped in advance 

• Harmonized submission packages 

• Regional teams empowered to manage local interactions 

Centralized Oversight Maintained Data Quality 

Despite the scale, data integrity remained high. Centralized biostatistics and data management teams oversaw query resolution, form freezing, and interim analysis preparation—delivering clean datasets on schedule. 

• Real-time query tracking 

• Centralized data management 

• On-time interim analysis delivery 

Key Learnings for Future Heme-Oncology Trials 

This Phase 3 program surfaced actionable insights for sponsors managing large-scale oncology trials in relapsed/refractory populations: 

• Use Hybrid Site Models to Accelerate Activation 
  Regional CROs can tailor strategies to local requirements, reducing startup delays. 

• Monitor Enrollment in Real Time to Guide Decisions 
  Dashboards help identify high-performing sites and enable agile resource shifts. 

• Design Protocols with Flexibility for Real-World Populations 
  Broader inclusion criteria reduce screen failures and improve generalizability. 

• Align Global Teams with Shared Tools and Frequent Touchpoints 
  Weekly meetings and centralized dashboards keep data cleaning and enrollment on track. 

• Plan Regulatory Submissions with Local Expertise 
  Country-specific timelines and ethics requirements demand early coordination and regional ownership. 

Strategic Impact of Phase 3 Trials in RRMM 

This trial demonstrated the feasibility of executing a complex, multinational Phase 3 study in a high-risk oncology population. The data supported regulatory approval and expanded treatment options for patients with RRMM. Operational learnings from this program continue to inform future trial designs—especially in relapsed/refractory settings where speed, flexibility, and precision are essential. 

At Precision for Medicine, we specialize in navigating complexity—from biomarker-driven enrollment to global site activation and complex data workflows. Our integrated teams bring deep therapeutic expertise and operational agility to every engagement. 

Planning a late-phase oncology trial? Let’s talk about how Precision can help you deliver with confidence.