Alzahra Hamdan received the Hans-Günter Schäfer Science Award 2025

The Hans-Günter Schäfer Science Award 2025 goes to Alzahra Hamdan. She is PhD student in her third year of studies in Pharmacometrics at the Uppsala University/Sweden and was awarded for the publication „Longitudinal Analysis of Natural History Progression of Rare and Ultra-Rare Cerebellar Ataxias Using Item Response Theory”. This paper (Clin. Pharm. Ther., doi:10.1002/cpt.3466) highlights the role of natural history studies and innovative methodologies in enhancing trial-readiness for rare diseases.

Degenerative cerebellar ataxias are a heterogeneous group of rare and ultra-rare genetic disorders. While disease-modifying therapies are under development, robust study designs and analysis methods are still lacking.

This publication leverages data from a natural history study to present a powerful methodological framework that enables reliable assessment of disease progression and treatment effects. A longitudinal Item Response Theory (IRT) model was developed to describe the progression of ten rare ataxia genotypes, using data from a large registry of autosomal recessive ataxias.

This award is sponsored by the Frankfurt Foundation Quality of Medicines (FFQM) and endowed with 1,000 €. Alzahra Hamdan received this award on May 16 at the PKPD expert meeting in Bad Dürkheim/Germany, an annual conference organized by the Arbeitsgemeinschaft für angewandte Humanpharmakologie (AGAH).

The certificate was handed over to the awardee by the AGAH president, Dr. Joachim Höchel (Bayer AG/Berlin), the meeting chair Dr. Nele Müller-Plock (Certara/Berlin) and Prof. Henning Blume, chairman of FFQM’s board.

We congratulate Alzahra on this work, which demonstrates how natural history analyses can support the planning of future treatment trials in genetic ataxias.