Development and validation of patient- and caregiver-reported outcome parameters in HSP

Subproject 3

Project lead: Stephan Klebe, Karl-Heinz Jöckel (Universitätsmedizin Essen)


Validated clinical outcome measures are an essential prerequisite for clinical trials. Existing outcome measures do not always systematically measure aspects and concepts relevant to patients and caregivers. The concept of Patient-Centered Outcomes (PCOM), designed to provide evidence for the impact of the disease and treatment on patients and their caregivers, tries to remedy this shortcoming.

The aim of this study is the development and validation of patient-centered outcome parameters for Hereditary Spastic Paraplegias (HSP) in a prospective multicenter observational study. Following a design and validation process for PCOM recommended by the “International Rare Diseases Research Consortium” (IRDiRC) we will develop a set of German-language disease-specific PCOMs for HSP and related disorders in an interactive, qualitative and quantitative validation process. Key stakeholders including clinicians, researchers, patients and family members, caregivers and patient organizations will be involved throughout all steps of the process.