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Zhaohui Su: Natural History Studies Strengthen Evidence Generation in Rare Disease Drug Development
Zhaohui Su, VP, Strategic Consulting at Veristat, shared a post on LinkedIn:
“One of the most significant challenges in rare disease drug development is demonstrating treatment benefit when traditional randomized controlled trials (RCT) are not feasible. This recent research paper by Samuel H. Hughes, MBA and colleagues shared the following insights
- Data quality matters more than study type. Regulators focus on the rigor of collection, endpoint definition, patient comparability, and completeness, regardless of whether data are collected prospectively or retrospectively.
- Disease characteristics are critical. External controls are most persuasive in severe, progressive diseases with well-characterized natural histories and limited treatment options, where randomization may be unethical or impractical.
- Natural history studies have become strategic assets. Landmark approvals such as Zolgensma, Skysona, and Lenmeldy demonstrate how well-executed natural history datasets can provide the context needed to interpret single-arm clinical trial results.
- Regulators are increasingly embracing a “totality of evidence” framework. External controls are rarely the only source of evidence; they are typically complemented by biomarkers, mechanistic understanding, clinical outcomes, and other supportive data.
- Patient advocacy groups are playing an increasingly important role by helping establish, govern, and sustain natural history datasets that support both research and regulatory decision-making.
For statisticians, clinical developers, regulators, and patient advocacy organizations, this means investing early in high-quality natural history data is a foundational component of the development strategy for future rare disease therapies
At Veristat Strategic Consulting, we work with sponsors to address these challenges—from natural history strategy and external control design to regulatory evidence generation and development planning for rare disease and advanced therapy programs. Building regulatory-grade evidence requires thoughtful planning long before pivotal studies begin. Learn more.”
Title: External controls for rare disease drug development: Lessons for emerging and advanced therapeutic modalities
Authors: Samuel H. Hughes, Lauren A. Beretich, Matthew Fuller, Kimberly Goodspeed, Melissa Penn, Leonard A. Valentino, Caitlin McCombs
Read the article
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Caitlin McCombscancerKimberly GoodspeedLauren A. BeretichLeonard A. ValentinoMatthew FullerMelissa PennOncoDailyOncologyrare diseaseSamuel H. HughesVeristatVeristat Strategic ConsultingZhaohui Su
