Real‑world evidence as a strategic architecture for precision hematology

By Mike D’Ambrosio, Senior Vice President and Global Head, Real World Research

Earl Gillespie, Ph.D., Senior Director, Health Advances

Published on: May 29, 2026

This article is part of Parexel's Hematology playbook series that is comprised of five chapters on the strategic decisions that separate hematology programs that launch from those that stall — from first-in-human design to post-approval evidence.

Intro Chapter 1 Chapter 2 Chapter 3 Chapter 4 Chapter 5

Precision hematology has reached a point where evidence strategy must be integrated upfront, not as a downstream step in clinical development. Genetically defined micro‑populations, unstable surrogate endpoints, and rising expectations from regulators and health technology assessment (HTA) agencies mean that real-world evidence (RWE) must shape the target product profile (TPP), trial design, and value narrative from day zero.  

Many small and emerging hematology sponsors are still approaching evidence planning with a legacy mindset; they treat RWE as a data commitment to satisfy post-approval requirements, rather than as an upstream input that shapes the TPP and de-risks clinical development. In hematology, where treatment pathways evolve rapidly in line with regulatory and HTA expectations, by the time the gap emerges, the cost of closing it is exactly what an emerging biotech can least absorb. 

At Parexel, we see the same pattern across programs: the organizations that succeed are those that treat RWE as a strategic filter, a risk‑reduction engine, and a market‑access accelerator and not as a compliance requirement. What follows is a structured, decision‑oriented roadmap for building that evidence architecture. 

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