Building global real-world evidence for a rare disease affecting pregnancy outcomes

Executive summary

A leading global pharmaceutical company partnered with BC Platforms to better understand how a rare disease affects pregnancy outcomes and how clinicians manage these high-risk pregnancies in routine clinical practice. With no global consensus on treatment, practices varied across countries, hospitals, and clinical teams.

To close this evidence gap, BC Platforms delivered a multi-country retrospective observational real-world data study spanning Europe, the United States, and Australia. The program captured structured clinical data, applied consistent quality checks, and securely delivered a robust evidence resource for a rare disease population.

As a result, the client gained a clearer view of treatment practices, care pathways, and pregnancy outcomes across routine clinical settings. The evidence resource also provided a foundation for regulatory, market access, and other milestone discussions.

Project snapshot

Key metrics

Background 

Our client, a top 20 global pharma company, is developing a compound for a rare disease associated with high-risk pregnancies and needed real-world context for ongoing clinical development. Limited evidence existed outside the clinical trial setting. Therefore, the client sought to understand how clinicians managed at-risk pregnancies in routine care, which interventions they used, and what outcomes they observed across different healthcare settings.

To address this gap, our client initiated a real-world data study to characterize treatment practices, care pathways, and pregnancy outcomes across diverse healthcare settings.

Challenge 

The central challenge was translating fragmented, real-world clinical practice into a consistent evidence base while preserving the variations in care that made the study valuable.

Identifying patients with a rare condition

Rare disease studies often involve highly dispersed patient populations that can be difficult to find. Participating sites needed to identify pregnancies considered at risk based on prior disease-affected pregnancies while ensuring eligibility criteria were applied consistently.

Capturing variation in clinical practice

Care sites often managed these pregnancies in different ways, making it difficult to understand which approaches were being used and how outcomes varied between sites. The study needed to document how treatment decisions, monitoring approaches, and interventions differed across healthcare organizations without imposing a predefined model of care.

Maintaining data quality across sites

Because data would be collected across multiple sites and geographies, the study needed a shared approach to data capture, review, and validation before analysis could begin. Without consistent quality checks, differences in documentation practices could reduce the reliability and comparability of the evidence.

Delivering analysis-ready datasets

Raw site data needed review, cleaning, validation, harmonization, integration, and preparation for downstream analysis.

Solution 

BC Platforms translated the study requirements into a cohesive evidence-generation approach, coordinating site engagement, structured data capture, data quality review, dataset preparation, and secure access.

Standardized data collection across sites

BC Platforms designed a dedicated data collection database to support consistent collection of patient-level clinical information across participating sites and countries. Project and data teams also provided site training to support alignment.

Data consistency and evidence quality

We supported consistent data review across participating sites. As a result, collected information remained complete, comparable, and ready for downstream analysis.

Delivering analysis-ready datasets

We reviewed, cleaned, validated, and standardized collected data before delivery. This process ensured consistent, analysis-ready datasets that supported future research activities.

Secure data delivery

We delivered validated datasets through a dedicated secure research environment built for the client. The environment provided controlled access to study data while supporting ongoing research and collaboration. In addition, the project expanded an existing secure environment already used for related rare disease programs.

Coordinated evidence delivery

A multidisciplinary BC Platforms team coordinated the data, site, and delivery workflows needed to support the study, helping our client maintain alignment across sites and countries while staying focused on scientific objectives.

Impact 

The project will give our client a clearer, evidence-based view of current practice in an area where limited real-world data and the absence of a global consensus have constrained decision-making.

Greater visibility into real-world clinical practice

The study created a multi-country view of how high-risk pregnancies are managed across different healthcare systems, providing insight into treatment variation and current clinical practice.

Evidence to support future research

The resulting dataset supported future analyses of treatment approaches, management strategies, and outcomes, helping our client place clinical development findings in a broader real-world context.

High-quality data for downstream analysis

Standardized data collection, review, and validation processes improved consistency across sites and facilitated delivery of analysis-ready data.

Foundation for future clinical development

By clarifying how care was delivered in the absence of a globally established standard, the study gave our client a stronger evidence base for planning future research, publications, and stakeholder discussions.

Why BC Platforms 

We were selected for our ability to deliver complex real-world evidence programs across multiple sites, countries, and stakeholders. In this study, that meant helping our client understand treatment practices and outcomes while creating a high-quality evidence foundation for research, publications, and clinical development.

Specifically, BC Platforms:

• Built on experience supporting rare disease research programs for the client and other global pharmaceutical organizations.
• Supported site identification and engagement through our global data partner network spanning 35+ countries.
• Designed the database for data collection.
• Implemented standardized data collection across 20 sites.
• Supported consistent data review and quality checks across participating sites.
• Delivered standardized, analysis-ready datasets.
• Provided secure research environments for controlled data access, building on infrastructure already used in related programs.
• Supported research in a rare disease area with limited existing data and no globally established standard of care.

Conclusion

Meaningful rare disease research requires more than access to patient data. It depends on finding dispersed patient populations, collecting consistent information across sites, and transforming clinical data into research-ready evidence.

BC Platforms helped establish a global evidence program that captured how high-risk pregnancies are managed in routine clinical practice, giving our client a stronger foundation for future research, publications, and clinical development.