A Pharmacometrics-Informed Trial Simulation Framework for Optimizing Study Designs for Disease-Modifying Treatments in Rare Neurological Disorders

A new simulation framework leverages pharmacometrics to optimise clinical trial designs for disease-modifying treatments in rare neurological conditions.

The authors present a framework using trial simulations to improve the design of studies targeting rare neurological disorders. By integrating pharmacometric data, the model helps in selecting optimal endpoints and sample sizes to increase the probability of trial success.

Acces this publication
Pharmacometrics

Year of publication

2025

Source

CPT: Pharmacometrics & Systems Pharmacology

Author

Ryeznik Y, Hilgers RD, Heussen N, Comets E, Mentré F, Hendrickx N, et al.

You might also be interested in

Cover of the 16-country rare disease policy assessment, October 2026.

Reckoning With the Rare Disease Landscape. A 16 Country Policy Landscape Assessment across the Life Course.

The assessment compares diagnosis, treatment access and national governance across 16 countries, examining gaps in rare disease support throughout the life course.
erdera policy brief data OCT2026

ERDERA Policy Brief: Making all data count for rare disease research and health outcomes – from policy recommendations to implementation

Making all data count for rare disease research and health outcomes – from policy recommendations to implementation
genera una imagen similar a esta con el fondo blan (2)

The FAIR principles: making rare disease data work better

Responsible data management can help accelerate research and improve collaboration in rare diseases.
Genetic Mapping

New clinical trial guidance supports research beyond the hospital

New international guidance supports decentralised clinical trials, pragmatic designs and the use of real-world data.

ERDERA Search