How smarter trial design can derisk and accelerate your malignant hematology asset

By Amy Pace, ScD, Vice President, Biostatistics Consulting

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

Trial design in malignant hematology is being rewritten on two fronts. In early-phase trials, the FDA’s Project Optimus mandate now requires sponsors to justify the optimal dose using randomized data before pivotal trials. In late phase trials, the agency is increasingly willing to accept surrogate endpoints, such as MRD in multiple myeloma, to support accelerated approval in high-unmet-need indications. 

Many small and emerging biotechs still race toward first-in-human (FIH) milestones to secure their next funding round. However, without a comprehensive clinical plan, they often end up repeating dose-optimization work or rethinking endpoints when the FDA raises concerns at the end of Phase 2. 

At Parexel, we advise sponsors to leverage seamless trial designs that accelerate development timelines and reduce rework, applying that logic across the entire developmental pathway of their investigational product. 

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