Case study

International clinical trial in an ultra-rare paediatric genetic disease with a major impact on the quality of life of affected children

Background

The international rare disease clinical trial was supported by Pharmaspecific through monitoring, regulatory submissions and clinical trial agreement management in France and the United Kingdom. The teams also supported the sponsor with site set-up, quality follow-up and compliance with regulatory requirements. This coordination ensured consistent organisation of activities in both countries and rigorous traceability of the data collected throughout the study.

essai clinique international en maladie rare projet national de centralisation de données clinique en vie réelle, étude clinique de bioéquivalence, Étude clinique sur le diabète étude clinique sur la sécurité d'un agent de contraste

étude observationnelle multicentrique en oncologie

Challenges identified

This international rare disease clinical trial presented several scientific and operational challenges:

extremely limited recruitment of eligible patients, due to the rarity of the disease
defining and validating specific endpoints suited to a paediatric population and a rare disease
international multicentre coordination, requiring precise organisation and continuous support for investigator sites.

Pharmaspecific’s role

The international rare disease clinical trial was supported by Pharmaspecific through monitoring, regulatory submissions and clinical trial agreement management in France and the United Kingdom. The teams also supported the sponsor with site set-up, quality follow-up and compliance with regulatory requirements, in line with Good Clinical Practice (GCP). This coordination helped ensure consistent management of activities in both countries, as well as rigorous traceability of the data collected throughout the study.

étude observationnelle multicentrique en oncologie essai clinique de phase III en myélome multiple ; étude de syndromes coronaires aigus

Results

étude observationnelle multicentrique en oncologie essai clinique de phase II en oncologie thoracique étude clinique de sécurité post-autorisation essai clinique international de phase III en lymphome

Key takeaways

This project illustrates Pharmaspecific's international expertise in conducting an international rare disease clinical trial, as well as its ability to adapt to complex scientific and operational settings. Through agile organisation, well-managed multicentre coordination and a personalised approach, Pharmaspecific effectively supports clinical research projects in rare diseases that address specific medical needs.

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