M. Kaatee

The patient’s view on rare disease trial design – a qualitative study

J. H. van der Lee, C. Gaasterland, M. C. van der Weide, 2019, Orphanet Journal of Rare Diseases.

Position statement on access to care in rare liver diseases: advancements of the European reference network (ERN) RARE-LIVER

A. Lohse, J. Drenth, David E. J. Jones, 2019, Orphanet Journal of Rare Diseases.

The POWER-tool: Recommendations for involving patient representatives in choosing relevant outcome measures during rare disease clinical trial design.

C. Gaasterland, M. Kaatee, R. Kaczmarek, 2018, Health policy.