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

1:1
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 (pdf)
Pharmacometrics

Year of publication

2025

Source

CPT: Pharmacometrics & Systems Pharmacology

Link to cite

Acces to Link >

Author

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

You might also be interested in

Task-Forces-IRDiRC

IRDiRC launches call for experts for four new rare disease activities

Rare disease researchers, clinicians, patient advocates and data experts are invited to help shape four new IRDiRC activities linked to the new IRDiRC Roadmap, with applications closing on 20 February 2026.
Picture for the website (1)

New survey launched to improve rare diseases clinical trial enrolment

The RealiseD project launched a new multi-stakeholder survey that invites the rare disease community to share insights and help improve clinical trial enrolment
Advancement of Treatments for Rare Diseases

Advancement of Treatments for Rare Diseases

The two-day event will focus on translating research and policy initiatives into tangible benefits for Rare Disease patients through better prevention, diagnosis, and treatment.
RealiseD webinars

RealiseD webinar series: Helping re‑shape rare disease clinical trials

13 January – 10 February 2026 | Online webinar series to advance best practices in clinical trial design for rare and ultra rare diseases.

ERDERA's

search form