Academic Medical Center Amsterdam
The Pediatric clinical Research Office (PRO) is part of the Clinical Research Office of the Department Woman and Child in the AMC. The epidemiologists of the PRO have published about statistical methods for obtaining valid evidence from randomized clinical trials while optimizing the sample size, and about general topics of RCT methodology particularly for trials including children, such as adequate sample sizes, Data Monitoring Committees, and the identification and use of valid and relevant outcome measures.
PRO also contributes to a world-wide network of clinical researchers and methodologists aiming to improve the design, conduct and reporting of clinical trials in children, called StaR Child Health (www.starchildhealth.org), and is a partner in the FP7 funded network of excellence for paediatric medicines called GRiP (Global Research in Paediatrics) (www.grip-network.org).
AMC is the leader of WP4 (Improved use of patient level information and perspectives), in which it closely collaborates with other partners in the consortium, especially UMCU and VSOP/EGAN. AMC will develop methods for the systematic involvement of patients and parents in the issues that are of particular importance in rare diseases, e.g. registration of disease course characteristics that are needed to extract or extrapolate information necessary for estimation of nuisance parameters, weighting of several outcomes within composite outcomes (to be used in WP2), involving patients and parents in decisions about the sequence of particular types of trials (to be used in WP3), and individualized outcome measures. Because of AMC’s close collaboration with many clinician-researchers in subspecialties caring for patients with rare diseases, e.g. paediatricians in metabolic diseases, nephrology and hematology, and with patient organisations, it will also provide the consortium with case studies to validate their newly developed statistical methodology.
Dr. Hanneke van der Lee is head of the Department PRO. She is the convener of the StaR Child Health standard development group on Adequate Sample Sizes and Data Monitoring Committees and has been the methodologist of several (multidisciplinary) consensus procedures in metabolic diseases such as mucopolysaccharidosis I (MPS I). Hanneke has extensive clinimetric experience and is the primary methodologist in the RICH-Q (Renal Insufficiency therapy in Children – Quality assessment and improvement) project. She is lead scientist of the deliverables concerning Adequate Sample Size and Data Monitoring Committees of the EU funded Global Research in Paediatrics project (GRiP).
Dr. Martine Jansen-van der Weide is an epidemiologist involved in several research projects. For her PhD thesis, she conducted research on the ethics of end-of-life decisions, including in-depth interviews with proxies. In the FP7 project GRiP, she contributed to the development of tools on Data and Safety Monitoring Committees and Adequate Sample Size to guide clinical researchers in the design phase of a trial. She acts as a trial statistician in several paediatric trials, and is a consultant in methodological and statistical issues regarding clinical research. Her main interests include trial methodology and patient involvement.
Charlotte Gaasterland, MSc is a PhD student with a background in psychological methodology. In the ASTERIX project, she will investigate the psychometric properties of Goal Attainment Scaling in drug research, and she will develop methods for patient involvement in research design and in the weighing of outcome measures.
Dr. Hanneke van der Lee
Dr. Martine Jansen-van der Weide
Charlotte Gaasterland, PhD student