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Project Results

Submitted Publications

Toumazi, A., Comets, E., Alberti, C., Friede, T., Lentz, F., Stallard, N., Zohar, S. and Ursino, M. (May 2017). An R-package for Bayesian Dose-Finding Design using Pharmacokinetics (PK) for Phase I Clinical Trials.

Miller F, Zohar S, Stallard N, Madan J, Posch M, Hee SW, Pearce M, Vågerö M, Day S. (May 2017) Approaches to sample size calculation for clinical trials in rare diseases

Shing, L.M., Ursino, M., Cheung, Y.K. and Zohar, S. (March 2017). Dose-finding designs for cumulative toxicities using multiple constraints.

Ursino, M., Yuan, Y., Alberti, C., Comets, E., Fevrais, G., Friede, T., Lentz, F., Stallard, N., and Zohar, S. (March 2017). A Dose-finding Design for Seizure Reduction in Neonates.

Thall, P.F., Ursino, M., Baudouin, V., Alberti, C. and Zohar, S. (February 2017). Bayesian Treatment Comparison Using Parametric Mixture Priors Computed from Elicited Histograms.

Day S, Hechtelt Jonker A, Lau L, Hilgers R-D, Irony I, Larsson K, Roes K, Stallard N. (Jan 2017) Recommendations for the design of small population clinical trials

Messroghli DR, Pickard T, Fischer M, Opgen-Rhein B, Papakostas K, Böcker D, Jakob A, Khalil M, Mueller GC, Schmidt F, Kaestner M, Floris E. A. Udink ten Cate, Wagner R, Ruf B, Kiski D, Wiegand G, Degener F, Ulrike M. M.Bauer, Friede T and Schubert S. Trial Design Towards evidence-based diagnosis of myocarditis in children and adolescents: Rationale, design, and first baseline data of MYKKE, a multi-centre registry and study platform

Wandel S, B Neuenschwander B, Friede T, Röver C (September 2016) Using phase II data for the analysis of phase III studies: an application in rare diseases

Konietschke F T, Friede T, Pauly M (2016) Analysis of over-dispersed count data with varying follow up times: asymptotic theory and small sample approximations


Daniel R. Messroghli , Thomas Pickardt , Marcus Fischer , Bernd Opgen-Rhein , Konstantin Papakostas , D Boecker , A Jakob , Markus Khalil , Goetz C. Mueller , Florian Schmidt , Michael Kaestner , Floris E.A. Udink ten Cate , Robert Wagner , Bettina Ruf , Daniela Kiski , Gesa Wiegand , Franziska Degener , Ulrike M.M. Bauer , Tim Friede , Stephan Schubert (2017) Toward evidence-based diagnosis of myocarditis in children and adolescents: Rationale, design, and first baseline data of MYKKE, a multicenter registry and study platform. American Heart Journal 2017 May;187:133-144. doi: 10.1016/j.ahj.2017.02.027. Epub 2017 Feb 24

Hee SW, (further citation needed) (May 2017) Decision-theoretic designs for a series of trials with correlated treatment effects using the Sarmanov multivariate beta-binomial distribution Biom. J doi:10.1002/bimj.201600202

Ursino M, Zohar S, Lentz F, Alberti C, Friede T, Stallard N and Comets E (2017) Dose-finding methods for Phase I clinical trials using pharmacokinetics in small populations. Biom. J.. doi:10.1002/bimj.201600084

Hee SW, Willis A, Smith CT, Day S, Miller F, Madan J, Posch M, Zohar S, Stallard N (2 March 2017) Does the low prevalence rate affect the sample size of interventional clinical trials of rare diseases? An analysis of data from the Aggregate Analysis of Orphanet Journal of Rare Diseases 12:44 Doi: 10.1186/s13023-017-0597-1

Röver C, Friede T (Jan 2017) Discrete approximation of a mixture distribution via restricted divergence Journal of Computational and Graphical Statistics Vol. 26/Issue 1 doi: 10.1080/10618600.2016.1276840

Petit C, Samson A, Satoshi M, Ursino M, Guedj J, Jullien V, Comets E, Zohar S, (Published online before print October 4, 2016) Unified approach for extrapolation and bridging of adult information in early-phase dose-finding paediatric studies Stat Methods Med Res 0962280216671348 doi: 10.1177/0962280216671348

Kunz, CU, Stallard N, Parsons N, Todd S, FriedeT (2016) Blinded versus unblinded estimation of a correlation coefficient to inform interim design adaptations Biometrical Journal, doi: 10.1002/bimj.201500233

Chataway J , Friede T (2016) The N-MOmentum trial: Building momentum to advance trial methodology in a rare disease Mult Scler June 2016 22: 852-853, doi:10.1177/1352458516643399

Friede, T., Röver, C., Wandel, S., and Neuenschwander, B. (2016) Meta-analysis of few small studies in orphan diseases. Res. Syn. Meth., doi: 10.1002/jrsm.1217

Friede T, Röver C , Wandel S and Neuenschwander B, 2016 - Published online before print 18 October 2016) Meta-analysis of two studies in the presence of heterogeneity with applications in rare diseases, Biometrical Journal 2016 doi: 10.1002/bimj.201500236

Varges D, Manthey H, Heinemann U, Ponto C, Schmitz M, Schulz-Schaeffer WJ, Krasnianski A, Breithaupt M, Fincke F, Kramer K, Friede T, Zerr (Published online before print 2 November 2016) Doxycycline in early CJD: a double-blinded randomised phase II and observational study, Journal of Neurology, Neurosurgery and Psychiatry 2016 doi: 10.1136/jnnp-2016-313541

Ondra, T, Jobjörnsson, S, Beckman, R A, Burman, C-F, König, F, Stallard, N and Posch, M (2016) Optimizing trial designs for targeted therapies. PLoS One, 11: e0163726

Hilgers, R.-D., Roes, K. and Stallard, N. (2016) Directions for new developments on statistical design and analysis of small population group trials. Orphanet Journal of Rare Diseases, 11, 78.

Unkel S, Röver C, Stallard N, Benda N, Posch M, Zohar S and Friede S, (20 Feb 2016) Systematic reviews in paediatric multiple sclerosis and Creutzfeldt-Jakob disease exemplify shortcomings in methods used to evaluate therapies in rare conditions. Orphanet Journal of Rare Diseases, 201611:16, doi: 10.1186/s13023-016-0402-6

Ondra T, Dmitrienko A, Friede T, Graf A, Miller F, Stallard N, Posch M. (2016), Methods for identification and confirmation of targeted subgroups in clinical trials: a systematic review. Journal of Biopharmaceutical Statistics, 26, 99-119

Petit C, Jullien V, Samson A, Guedj J, Kiechel J-R, Zohar S, Comets E (2015) Designing a Pediatric Study for an Antimalarial Drug by Using Information from Adults. Antimicrob Agents Chemotherapy, 2015 Dec 28;60(3):1481-91 doi:10.1128/AAC.01125-15

Röver, C, Knapp, G and Friede (2015), Hartung-Knapp Sidik Jonkman Approach and its modification for random-effects meta-analysis with few studies. BMC Medical Research Methodology, Vol 15:99. doi: 10.1186/s12874-015-0091-1

Hee, S W, Hamborg, T, Day, S, Madan, J, Miller, F, Posch, M, Zohar S and Stallard, N (2015), Decision
theoretic designs for small trials and pilot studies: a review.
doi: 10.1177/0962280215588245
 (Published online before print June 5, 2015)

Graf, A. C., Posch, M. and Koenig, F. (2014), Adaptive designs for subpopulation analysis optimizing utility functions. Biom. J.. doi: 10.1002/bimj.201300257 (Published 14 November 2014)

Koenig, F., Slattery, J., Groves, T., Lang, T., Benjamini, Y., Day, S., Bauer, P. and Posch, M. (2014), Sharing clinical trial data on patient level: Opportunities and challenges. Biom. J.. doi: 10.1002/bimj.201300283 (Published 18 June 2014)



Artemis Toumazi , Moreno Ursino and Sarah Zohar, INSERM, Paris. (10 August 2016). R package dfpk "A Bayesian Dose-Finding Design using Pharmacokinetics (PK) for Phase I Clinical Trials". Available at: 

Christian Roever, University Medical Centre, Gottingen. (16 December 2015). Available at: The bayesmeta package provides a collection of functions to facilitate easy Bayesian inference in the generic random-effects meta-analysis model. It allows to derive the posterior distribution of the two parameters (effect and heterogeneity), and provides the functionality to evaluate joint and marginal posterior probability distributions, predictive distributions, shrinkage, etc.

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