Secondary use of patient data within decentralized studies using the example of rare diseases in Germany: A data scientist's exploration of process and lessons learned

Digital Health, vol. 10, pp. 20552076241265219

Abstract

Objective: Unlocking the potential of routine medical data for clinical research requires the analysis of data from multiple healthcare institutions. However, according to German data protection regulations, data can often not leave the individual institutions and decentralized approaches are needed. Decentralized studies face challenges regarding coordination, technical infrastructure, interoperability and regulatory compliance. Rare diseases are an important prototype research focus for decentralized data analyses, as patients are rare by definition and adequate cohort sizes can only be reached if data from multiple sites is combined. Methods: Within the project "Collaboration on Rare Diseases", decentralized studies focusing on four rare diseases (cystic fibrosis, phenylketonuria, Kawasaki disease, multisystem inflammatory syndrome in children) were conducted at 17 German university hospitals. Therefore, a data management process for decentralized studies was developed by an interdisciplinary team of experts from medicine, public health and data science. Along the process, lessons learned were formulated and discussed. Results: The process consists of eight steps and includes sub-processes for the definition of medical use cases, script development and data management. The lessons learned include on the one hand the organization and administration of the studies (collaboration of experts, use of standardized forms and publication of project information), and on the other hand the development of scripts and analysis (dependency on the database, use of standards and open source tools, feedback loops, anonymization). Conclusions: This work captures central challenges and describes possible solutions and can hence serve as a solid basis for the implementation and conduction of similar decentralized studies.

Authors 19

1. Michéle Zoch corresponding

   University Hospital Carl Gustav Carus · Technische Universität Dresden

   Affiliation as printed

   Institute for Medical Informatics and Biometry, Faculty of Medicine and University Hospital Carl Gustav Carus, TUD Dresden University of Technology, Dresden, Germany

2. Christian Gierschner

   University Hospital Carl Gustav Carus · Technische Universität Dresden

   Affiliation as printed

   Institute for Medical Informatics and Biometry, Faculty of Medicine and University Hospital Carl Gustav Carus, TUD Dresden University of Technology, Dresden, Germany

3. Anne-Katrin Andreeff

   University Hospital Carl Gustav Carus · Technische Universität Dresden

   Affiliation as printed

   Institute for Medical Informatics and Biometry, Faculty of Medicine and University Hospital Carl Gustav Carus, TUD Dresden University of Technology, Dresden, Germany

4. Elisa Henke

   University Hospital Carl Gustav Carus · Technische Universität Dresden

   Affiliation as printed

   Institute for Medical Informatics and Biometry, Faculty of Medicine and University Hospital Carl Gustav Carus, TUD Dresden University of Technology, Dresden, Germany

5. Martin Sedlmayr

   University Hospital Carl Gustav Carus · Technische Universität Dresden

   Affiliation as printed

   Institute for Medical Informatics and Biometry, Faculty of Medicine and University Hospital Carl Gustav Carus, TUD Dresden University of Technology, Dresden, Germany

6. Gabriele Müller

   University Hospital Carl Gustav Carus · Zentrum für Evidenzbasierte Gesundheitsversorgung · Technische Universität Dresden

   Affiliation as printed

   Center for Evidence-based Healthcare, Faculty of Medicine and University Hospital Carl Gustav Carus, TUD Dresden University of Technology, Dresden, Germany

7. Jenny Tippmann
   Affiliation as printed

   Thiem-Research GmbH, Cottbus, Germany

8. Helge U. Hebestreit

   University of Würzburg · Universitätsklinikum Würzburg

   Affiliation as printed

   Center for Rare Diseases – Reference Center Northern Bavaria, University Hospital, Julius-Maximilians University, Würzburg, Germany

9. Daniela Choukair

   University Hospital Heidelberg

   Affiliation as printed

   Center for Rare Diseases, University Hospital Heidelberg, Heidelberg, Germany

10. Georg Friedrich Hoffmann

    University Hospital Heidelberg

    Affiliation as printed

    Center for Rare Diseases, University Hospital Heidelberg, Heidelberg, Germany

11. Fleur Fritz

    University Hospital Heidelberg

    Affiliation as printed

    Institute of Medical Informatics, University Hospital Heidelberg, Heidelberg, Germany

    Center for Rare Diseases, University Hospital Heidelberg, Heidelberg, Germany

12. Nicole Toepfner

    University Hospital Carl Gustav Carus · Technische Universität Dresden

    Affiliation as printed

    University Centre for Rare Diseases and Department of Pediatrics, Faculty of Medicine and University Hospital Carl Gustav Carus, TUD Dresden University of Technology, Dresden, Germany

13. Reinhard Berner

    University Hospital Carl Gustav Carus · Technische Universität Dresden

    Affiliation as printed

    University Centre for Rare Diseases and Department of Pediatrics, Faculty of Medicine and University Hospital Carl Gustav Carus, TUD Dresden University of Technology, Dresden, Germany

14. Stephanie D. Biergans

    Universitätsklinikum Tübingen

    Affiliation as printed

    University Hospital Tübingen

    Medical Data Integration Center (meDIC), University Hospital Tübingen, Tübingen, Germany

15. Raphael Verbücheln

    Universitätsklinikum Tübingen

    Affiliation as printed

    University Hospital Tübingen

    Medical Data Integration Center (meDIC), University Hospital Tübingen, Tübingen, Germany

16. Jannik Schaaf

    Goethe University Frankfurt · University Hospital Frankfurt

    Affiliation as printed

    Goethe University Frankfurt

    Institute of Medical Informatics, Goethe University Frankfurt, University Hospital, Frankfurt, Germany

17. Felix Nikolaus Wirth

    Berlin Institute of Health at Charité - Universitätsmedizin Berlin · Charité - Universitätsmedizin Berlin

    Affiliation as printed

    Berlin Institute of Health (BIH) at Charité Universitätsmedizin Berlin, Berlin, Germany

18. Josef Schepers

    Berlin Institute of Health at Charité - Universitätsmedizin Berlin · Charité - Universitätsmedizin Berlin

    Affiliation as printed

    Berlin Institute of Health (BIH) at Charité Universitätsmedizin Berlin, Berlin, Germany

19. Fabian Praßer

    Berlin Institute of Health at Charité - Universitätsmedizin Berlin · Charité - Universitätsmedizin Berlin

    Affiliation as printed

    Berlin Institute of Health (BIH) at Charité Universitätsmedizin Berlin, Berlin, Germany