Architectural Divergence Across Three Hemophilia Studies: Technical Implications for SDTM/ADaM Conformance, Metadata Governance, and CSR‚ Level Consistency

About this session

Part of Session 6, Track A: Foundational Standards - Validation/Quality (Chair: Harivardhan Jampala, Sumitomo Pharma).

Rare disease programs often progress through multiple studies built on differing CRF architectures, metadata assumptions, and derivation rules. The INH5, INH8, and INH7 hemophilia studies exemplify this challenge, as each was developed using distinct data collection frameworks that produced fragmented metadata, heterogeneous derivation logic, and inconsistent controlled terminology. The most impactful divergences involved non uniform ABR algorithms, differences in dosing schema, heterogeneous laboratory schedules and units, age calculations, and distinct eDiary workflows, all of which propagated into the extension study and resulted in mixed derivation rules and inconsistent assumptions across CSR packages.
These foundational setup issues also created substantial complications during ISS/ISE preparation, where cross study inconsistencies became more pronounced and required extensive remediation.
This paper provides a technical assessment of these architectural gaps and proposes a standards driven framework emphasizing unified CRF design, centralized metadata governance, harmonized derivation specifications, and proactive SDTM/ADaM alignment to ensure CSR ready, compliant outputs.

Speakers

• PK

  Prasanna Kakade

  Ephicacy Consulting Group

  Speaker

• AW

  Achin Wadhwa

  Ephicacy Consulting Group

  Speaker