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Medizinische Hochschule Hannover

Description

Hannover Medical School (MHH) is one of the largest university medical centres in Germany with 1,500 beds and more than 70 clinics and institutes. The Institute of Biostatistics is in charge of teaching medical students, providing biostatistical advice to doctoral theses, research projects, and particularly for supporting the conduct of clinical trials at Hannover Medical School (MHH). Biostatistical methodology for drug trials, therapeutic trials and diagnostic trials, as well as meta-analyses are the main focus of research. MHH is one of the largest transplant centres in Germany and treatment in the field of transplant medicine and post-transplant disease is, per definition, orphan. The Institute is involved in the planning of numerous clinical trials in very frequent as well as in very rare disease, and has successfully achieved public funding for three multi-centre studies in rare conditions (diagnosis and treatment of kidney rejection episodes, cochlear implantation) and one multi-centre study in children (treatment and diagnosis of haemodynamic insufficiency in neonates).

Main tasks

MHH Biostatistics department has substantial experience in planning trials in rare disease and has identified key problems for further methodological research. In WP2, MHH will contribute to the development of standards for adaptive generation of evidence, randomisation methods for clinical trials in small populations, and methods for combination of endpoints. Simulation studies, theoretical considerations and considerations from the experimental design perspective will be utilized. In WP3, MHH will contribute simulation studies comparing developments across multiple studies with concepts, where only one pragmatic study per research question is planned and conducted. In WP5, MHH will contribute with scenario simulations with input from (blinded) real-life data from its clinical trials data and wishes to adapt standard regulatory assessment strategies to the need in rare disease. In WP6, MHH will (beyond publishing articles in relevant journals) bring research outcome to the regulatory discussion about requirements for trial design in drug licensing for orphan drugs.

Key personnel

The PI, Prof. Dr. Armin Koch, has been involved in the development of regulatory guidelines for many years and will contribute to find commensurate rules for drug development in rare disease. He has been heavily involved in the discussion of the European Points to Consider Document on Applications with (i) Meta-Analysis and (ii) one pivotal trial (CPMP/EWP/2330/99), is one of the main authors of the European Reflection on Confirmatory Clinical Trials with an Adaptive design (CHMP/EWP/2459/02) and has led the discussion with all stakeholders in this context to proceed to new methodology in the sensitive area of confirmatory evidence for drug licensing on safe grounds (i.e. without lowering of regulatory standards). Currently he is one of the authors of the European Guidance document on assessment of subgroups of clinical trials. The PI has ample practical experience in study design and assessment of clinical trials for rare diseases related to drug licensing and counselling for regulatory requirements on the national (former Biostatistics Group leader at the German competent authority (BfArM)) and the European level (member of the Scientific Advice Working Party (SAWP) and Biostatistics Working Party (BSWP) since 2007).

Dipl. Stat Annika Müller-Heine is head of the clinical trials group in the Institute of Biostatistics having extensive experience in research designs for rare disease RCTs and registries and will supervise PhD students that work on topics in experimental design for orphan disease studies.

Dr. Anika Großhennig is deputy director of the Institute and will supervise the methodological research.


Prof. dr. Armin Koch
Co-chair and WP2

Dipl. Stat. Annika Müller-Heine
Researcher, WP2

Kristina Weber
Researcher, WP2

Lukas Aguirre D'Avila
Researcher, WP2
 

Theodor Framke
Researcher, WP2

Loukia Spineli, PhD
Researcher, WP2

 

Eva Budde
Researcher, WP2