Five strategies to build a better non-malignant hematology drug by listening to patients

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

Non-malignant hematology assets frequently enter an increasingly saturated market. When commercial performance fails to meet expectations, retrospective analyses often trace the problem to a patient profile that was never rigorously defined. This represents a strategic misstep that emerging biotechs can no longer afford in today’s competitive landscape.

Recent gene therapies for sickle cell disease (SCD) (Casgevy, Lyfgenia) and hemophilia (Hemgenix, Roctavian), approved between 2023 and 2025, have not achieved the level of commercial uptake that their underlying science and clinical promise initially suggested. Programs for beta thalassemia and bleeding disorders face the same risk: complex treatment procedures, high costs, infrastructure limits, and patient hesitancy keep eligible patients on the sidelines.

Biotechs frequently shape hematology development around the most severe patient groups, influenced by the advocacy voices most visible due to disease burden. Yet working-age patients with mild-to-moderate conditions, who feel generally well, often are far less motivated to pursue complex or burdensome treatments. Default regulatory endpoints may support approval but fail to capture outcomes that matter to patients, creating a mismatch that can contribute to underperformance at launch.

At Parexel, we advise sponsors to pressure-test trial designs against patient reality before the protocol locks. Our goal is to reach beyond the most engaged advocates to design programs that earn both regulatory approval and long-term patient support.