Joanna IntHout

Group Leader Innovative Clinical Trials for Rare Disease - Radboudumc (The Netherlands)

Joanna IntHout has more than 30 years of experience in biomedical research as biostatistician, with a focus on clinical trials and meta-analysis. Since 12.5 years she has been working at the Radboudumc, where she is regularly involved in trials for the development of therapies for rare diseases, including discussions with regulatory authorities (EMA, FDA). Previously she has worked as a biostatistician and group leader in a hospital, the National Institute for Health, a clinical research organisation, and the pharmaceutical industry.

Currently, in her new role as Group Leader Innovative Clinical Trials for Rare Diseases, her work is fully focused on designing smart and innovative trials that aim to generate valid and reliable evidence on the efficacy and safety of therapies for rare diseases, but with the minimum number of patients. She leads the Radboudumc effort to innovate trial designs for rare diseases and to support the implementation together with a multidisciplinary team.

She is involved in the Horizon Europe SIMPATHIC drug repurposing project, where she and her colleagues are designing a “basket trial” in which one (or more) existing drugs are tested in patients with different neurological disorders but similar clinical symptoms. Another interesting aspect of this trial is the use of a common outcome measure that can be applied to all patients, despite their different disorders and different stages of disease severity, i.e. with a high degree of heterogeneity. This interesting project will be the topic of her talk at IDR24.