IDeAl Output – IDEAL

Documents for Download

In this section, you can download the files that have been produced as official output in the project.

Articles in peer-reviewed journals

Reetz, K., Hilgers, R.-D., Isfort, S., Dohmen, M., Didszun, C., Fedosov, K., Kistermann, J., Mariotti, C., Durr, A., Boesch, S., Klopstock, T., de Rivera Garrido, F.J.R., Schöls, L., Klockgether, T., Pandolfo, M., Korinthenberg, R., Lavin, P., Molenberghs, G., Libri, V., Giunti, P., Festenstein, R., Schulz, J.B., and the EFACTS or NICOFA study group: Protocol of a randomized, double-blind, placebo-controlled, parallel-group, multicentre study of the efficacy and safety of nicotinamide in patients with Friedreich ataxia (NICOFA). Neurological Research and Practice, 1:33, October 2019.
Collignon, O., Schritz, A., Senn, S. J., and Spezia, R.: Clustered allocation as a way of understanding historical controls: Components of variation and regulatory considerations. Statistical Methods in Medical Research, October 2019.
Rückbeil, M.V., Hilgers, R.-D., Heussen, N.: Randomization in survival studies: An evaluation method that takes into account selection and chronological bias. PLOS ONE, 14(6): e0217946, June 2019.
Hilgers, R.-D., Manolov, M., Heussen, N., Rosenberger, W.F.: Design and analysis of stratified clinical trials in the presence of bias. Statistical Methods in Medical Research, May 2019.
Van der Elst, W., Alonso Abad, A., Geys, H., Meyvisch, P., Bijnens, L., Sengupta, R., and Molenberghs, G.: Univariate versus multivariate surrogates in the single-trial setting. Statistics in Biopharmaceutical Research, May 2019.
Loingeville, F., Nguyen, T.T., Riviere, M.-K., Mentré, F.: Robust designs in longitudinal studies accounting for parameter and model uncertainties – Application to count data. Jounal of Biopharmaceutical Statistics, April 2019.
Senn, S.: Childhood asthma exacerbations and ADRB2 polymorphism: Caution needed. Journal of Allergy and Clinical Immunology, 141(5): pp. 1954-1955, May 2018
Nassiri, V., Lovik, A., Molenberghs, G., Verbeke, G.: On using multiple imputation for exploratory factor analysis of incomplete data. Behavior Research Methods. Behaviour Research Methods, 50(2): pp. 501-517, April 2018.
Jobjörnsson, S., Christensen, S.: Anscombe’s model for sequential clinicl trials revisited. Sequential Analysis, 13(1): pp. 115-144, March 2018.
Meeting Abstracs of the 9th European Conference on Rare Diseases & Orphan Products (ECRD Vienna 2018) in Vienna, Austria, 10-12 May 2018. Orphanet Journal of Rare Diseases, 13 (Suppl 2) :167, September 2018.
Day, S., Hechtelt Jonker, A., Lau, L.P.L., Hilgers, R.-D., Irony, I., Larsson, K., Roes, K.C.B., Stallard, N.: Recommendations for the design of small population clinical trials. Orphanet Journal of Rare Diseases, 13:195, November 2018.
Berger, T., Hilgers, R.-D., Heussen, N.: Observations below multiple lower limits of quantification: how to estimate the mean and variance. Biometrical Journal, September 2018.
Dosne A.G., Bergstrand M., Karlsson M.O.: An automated sampling importance resampling procedure for estimating parameter uncertainty. Journal of Pharmacokinetics and Pharmacodynamics, 44(6): pp. 509-520, December 2017.
Sugitani T., Posch M., Bretz F., Koenig F.:Flexible alpha allocation strategies for confirmatory adaptive enrichment clinical trials with a prespecified subgroup. Statistics in Medicine, 37(24): pp. 3387-3402, October 2018.
Uschner, D., Schindler, D., Hilgers, R.-D., Heussen, N.: randomizeR: An R Package for the Assessment and Implementation of Randomization in Clinical Trials. Journal of Statistical Software, 85(8): pp. 1-22, June 2018.
Molenberghs, G., Hermans, L., Nassiri, V., Kenward, M.G., Van der Elst, W., Aerts, M., and Verbeke, G.: Clusters with unequal size: maximum likelihood versus weighted estimation in large samples. Statistica Sinica, 28(3): pp. 1107-1132, January 2018.
Hilgers R.-D., Bogdan M., Burman C.F., Dette H., Karlsson M., König F., Male Ch, Mentré F, Molenberghs G, Senn S. Lessons learned from IDeAl — 33 recommendations from the IDeAl-Net about design and analysis of small population clinical trials. Orphanet Journal of Rare Diseases, 13:77, May 2018.
Ondra, T., Jobjörnsson, S., Beckman, R.A., Burman, C.F., König, F., Stallard, N., Posch, M. Optimized adaptive enrichment designs. Statistical Methods in Medical Research, December 2017.
Uschner, D., Hilgers, R.-D., Heussen, N.: The impact of selection bias in randomized multi-arm parallel group clinical trials. PLoS ONE, 13(1), January 2018.
Alonso, A., Bigirumurame, T., Burzykowski, T., Buyse, M., Molenberghs, G., Muchene, L., Perualila, N.J., Shkedy, Z., and Van der Elst, W.: Applied Surrogate Endpoint Evaluation with SAS and R. Boca Raton: Chapman & Hall/CRC Biostatistics Series, 2017.
Nassiri, V., Molenberghs, G., and Verbeke, G.: Fast precision estimation in high-dimensional multivariate joint models. Biometrical Journal, 59(6): pp. 1221-1231, November 2017.
Su, W., Bogdan, M., Candes, E.J.: False Discoveries Occur Early on the Lasso Path. Annals of Statistics, 45(5): pp. 2133-2150, October 2017.
Hilgers, R.-D., Uschner, D., Rosenberger, W. F., Heussen, N.: ERDO – a framework to select an appropriate randomization procedure for clinical trials. BMC Medical Research Methodology, 17:159, December 2017.
Brzyski, D., Gossmann, A., Su, W., Bogdan, M.: Group SLOPE-adaptive selection of groups of predictors. Journal of the American Statistical Association, 114(525): pp. 419-433, January 2018.
Senn, S. : Sample size considerations for n-of-1 trials. Statistical Methods in Medical Research, 28(2): pp. 372-383, February 2019.
Sobczyk, P., Bogdan, M., and Josse, J.: Bayesian dimensionality reduction with PCA using penalised semi-integrated likelihood. Journal of Computational and Graphical Statistics, 26(4): pp. 826-839, October 2017.
Hofer, M. P., Hedman, H., Mavris, M., Koenig, F., Vetter, T.,Posch, M., Vamvakas, S., Regnstrom, J., Aarum, S.: Marketing authorisation of orphan medicines in Europe from 2000 to 2013. Drug discovery today, 23(2): pp. 424-433, February 2018.
Miller, F., Burman, C. F.: A decision theoretical modeling for Phase III investments and drug licensing. Journal of Biopharmaceutical Statistics, 48(4): pp. 698-721, October 2017.
Hermans, L., Nassiri, V., Molenberghs, G., Kenward, Michael G., Van der Elst, W., Aerts, M., Verbeke, G.: Fast, Closed-form, and Efficient Estimators for Hierarchical Models with AR(1) Covariance and Unequal Cluster Sizes. Communications in Statistics Part – Simulation and Computation, 47(5): pp. 1492-1505, July 2017.
Hlavin, G., Hampson, L. and Koenig, F.: Many-to-one comparisons after safety selection in multi-arm clinical trials. PLoS ONE, 12(6), June 2017.
Hilgers, R.-D., Bogdan, M., Burman, C.F., Dette, H., Karlsson, M., König, F, Male, C., Mentré, F., Molenberhgs, G., Senn, S.: Integrated Design and Analysis of small population group trials – IDEAL – FP7. Impact, Number 4, pp. 14-16, May 2017.
Senn, S.: Contribution to the discussion of “A critical evaluation of the current p-value controversy”, Biometrical Journal, 59(5): pp. 892-894, September 2017.
Lonergan, M., Senn, S., McNamee, C., Daly, A. K., Sutton, R., Hattersley, A., Pearson, E., and Pirmohamed, M.: Defining drug response for stratified medicine. Drug discovery today, 22(1): pp. 173-179, January 2017.
Dette, H., Möllenhoff, K., Volgushev S., and Bretz F.: Equivalence of regression curves. Journal of the American Statistical Association, 113(522): pp. 711-729, March 2018.
Ueckert, S. and Mentré, F.: A new method for the evaluation of the Fisher information matrix for discrete mixed effect models using Monte-Carlo sampling and adaptive Gaussian quadrature. Computational Statistics and Data Analysis, 111(1): pp. 203-219, July 2017.
Bretz, F., Möllenhoff, K., Dette, H., Liu, W., and Trampisch, M.: Assessing the similarity of dose response and target doses in two non-overlapping subgroups. Statistics in medicine, 37(5): pp. 722-738, February 2018.
Szulc, P., Bogdan, M., Frommlet, F., and Tang, H.: Joint genotype- and ancestry-based genome-wide association studies in admixed populations. Genetic epidemiology, 41(1): pp. 555-566, June 2017.
Brzyski, D., Peterson, C., Sobczyk, P., Candes, E., Bogdan, M., and Sabatti, C.: Controlling the rate of GWAS false discoveries. Genetics, 205(1): pp. 61-75, January 2017.
Strömberg, E.A., Hooker, A.C.: The effect of using a robust opti- mality criterion in model based adaptive optimization. Journal of Pharmacokinetics and Pharmacodynamics, 44(4): pp. 317-324, April 2017.
Rückbeil, M., Hilgers, R.-D., Heussen, N.: Assessing the impact of selection bias on test decisions in trials with a time-to-event outcome. Statistics in Medicine, 36(17): pp.2656-2668,
Senn, S.: Letter to the Editor: Don’t take it personally. Significance, 14(1): pp. 46-47, February 2017.
Bird, S. M., Bailey, R. A., Grieve, A. P., Senn, S.: Statistical issues in first-in-human studies on BIA 10-2474: Neglected comparison of protocol against practice. Pharmaceutical Statistics, 16(2): pp. 100-106, March 2017.
Alonso, A.A., Van der Elst, W., Meyvish, P.: Assessing a surrogate predictive value: a causal inference approach. Statistics in Medicine. 36(7): pp. 1083-1098, March 2017.
Bauer, P.: Comments on ‘Estimands in clinical trials – broadening the perspective’. Statistics in Medicine, 36(1): pp. 22-23, January 2017.
Wang, S., Karlsson, K., Kjellsson, M., Karlsson, M.O., and Hooker, A.C.: A proof-of-principle example for identifying drug effect from a mechanistic model with a more parsimonious model. Journal of Pharmacokinetics and Pharmacodynamics, 43: p. S35, October 2016.
Hilgers, R.-D., König, F., Molenberghs, G., and Senn, S.: Design and analysis of clinical trials for small rare disease populations. Journal of Rare Diseases Research & Treatment, 1(3): pp. 53-60, November 2016.
Dosne, A.-G., Niebecker, R., and Karlsson, M.O.: IdOFV distributions: a new diagnostic for the adequacy of parameter uncertainty in nonlinear mixed-effects models applied to the bootstrap. Journal of Pharmacokinetics and Pharmacodynamics, 43(6): pp. 597-608, October 2016.
Dosne, A.-G., Bergstrand, M., Harling, K., and Karlsson, M.O.: Improving the estimation of parameter uncertainty distributions in nonlinear mixed effects models using sampling importance resampling. Journal of Pharmacokinetics and Pharmacodynamics, 43(6): pp. 583-596. October 2016.
Araujo, A., Julious, S., and Senn, S.: Understanding Variation in Sets of N-of-1 Trials. PLoS ONE, 11(12): e0167167, September 2016.
Ondra, T., Jobjörnsson, S., Beckman, R.A., Burman, C.-F., König, F., Stallard, N., and Posch, M.: Optimizing Trial Designs for Targeted Therapies. PLoS ONE, 11(9): e0163726, September 2016.
Deng, C., Plan, E.L., and Karlsson, M.O.: Approaches for modeling within subject variability in pharmacometric count data analysis: dynamic inter-occasion variability and stochastic differential equations Journal of Pharmacokinetics and Pharmacodynamics. 43(3): pp. 305-314, June 2016.
Reetz, K., Dogan, I., Hilgers, R.-D., Giunti, P., Mariotti, C., Durr, A., Boesch, S., Klopstock, T., Rodriguez de Rivera, F., Schöls, S., Klockgether, T., Bürk, K., Rai, M., Pandolfo, M., and Schulz, J.: Progression characteristics of the European Friedreich’s Ataxia Consortium for Translational Studies (EFACTS): a 2 year cohort study, The Lancet Neurology, 15: pp. 1346-1354, December 2016.
Eichler, H.-G., Bloechl-Daum, B., Bauer, P., Bretz, P., Brown, J., Hampson, L.V., Honig, P., Krams, M., Leufkens, H., Lim, R., Lumpkin, M.M., Murphy, M.J., Pignatti, F., Posch, M., Schneeweiss, S., Trusheim, M., and König, F.: ‘Threshold-crossing’: A useful way to establish the counterfactual in clinical trials? Clinical Pharmacology & Therapeutics, 100(6): pp. 699-712, December 2016.
Van der Elst, W., Molenberghs, G., Hilgers, R.-D., Verbeke, G., and Heussen, N.: Estimating the reliability of repeatedly measured endpoints based on linear mixed-effects models. A tutorial. Pharmaceutical Statistics, 15(6): pp. 486-493, November 2016.
Ueckert, S., Karlsson, M., and Hooker, A.: Accelerating Monte Carlo power studies through parametric power estimation. Journal of Pharmacokinetics and Pharmacodynamics, 43(2): pp. 223-234, April 2016.
Gewandter, J., McDermott, M., McKeown, A., Hoang, K., Iwan, K., Kralovic, S., Rothstein, D., Gilron, I., Katz, N., Raja, S., Senn, S., Smith, S., Turk, D., Dworkin, R.: Reporting of cross-over clinical trials of analgesic treatments for chronic pain: ACTTION systematic review and recommendations. PAIN. 157(11): pp. 2544-2551, November 2016.
Bauer, P. and König, F.: Adaptive paediatric investigation plans, a small step to improve regulatory decision making in drug development for children. Pharmaceutical Statistics, 15(5): pp. 384-386, September 2016.
Hilgers, R.-D., Roes, K., and Stallard, N.: Directions for new developments on statistical design and analysis of small population group trials. Orphanet Journal of Rare Diseases, 11(1): p. 78, June 2016.
Collignon, O. and Monnez, J.-M.: Clustering of the Values of a Response Variable and Simultaneous Covariate Selection Using a Stepwise Algorithm. Applied Mathematics, 7: pp. 1639-1648, September 2016.
Dette, H., Schorning, K. and Konstantinou, M.: Optimal designs for comparing regression models with correlated observations. Computational Statistics & Data Analysis, 113: pp. 273-286, January 2016.
Auffray, C., Balling, R., Barroso, I., Bencze, L., Benson, M., Bergeron, J., Bernal-Delgado, E., Blomberg, N., Bock, C., Conesa, A., Del Signore, S., Delogne, C., Devilee, P., Di Meglio, A., Eijkemans, R., Flicek, P., Graf, N:, Grimm, V., Guchelaar, H.-j., Guo, Y., Glynne Gut, I., Hanbury, A., Hanif, S., Hilgers, R.-D., Honrado, A., Hose, D., Houwing-Duistermaat, Jeanine, Hubbard, T., Janacek, S. H., Karanikas, H., Kievits, T., Kohler, M., Kremer, A., Lanfear, J., Lengauer, T., Maes, E., Meert, T., Muller, W., Nickel, D., Oledzki, P., Pedersen, B., Petkovic, M., Pliakos, K., Rattray, M., Redon i Mas, J., Schneider, R., Sengstag, T., Serra Picamal. X., Spek, W., Tome, M., Vaas, L., van Batenburg, O., Vandelaer, M., Varnai, P., Volloslada, P., Vzicaino J. A., Wubbe, J., Zanetti, G.: Making sense of big data in health research: Towards an European Union action plan. Genome Medicine. Genome Medicine, 8(1):71, June 2016.
Lee, S., Brzyski, D., Bogdan, M.: Fast Saddle-Point Algorithm for Generalized Dantzig Selector and FDR Control with the Ordered l1-Norm. In: Proceedings of the 19th International Conference on Artificial Intelligence and Statistics, JMLR:W&CP Volume 51, pp. 780-789, 2016.
Dette, H. and Schorning, K.: Optimal Designs For Comparing Curves. The Annals of Statistics, 44(3): pp. 1103-1130, June 2016.
Schorning, K., Bornkamp, B., Bretz, F., and Dette, H.: Model Selection versus Model Averaging in Dose Finding Studies. Statistics in Medicine, 35(12): pp. 4021-4040, September 2016.
Riviere, M.-K., Ueckert S., and Mentré, F.: A MCMC-method for the evaluation of the Fisher information matrix for nonlinear mixed effect models. Biostatistics, 17(4): pp. 737-750, May 2016.
Bauer, P., Bretz, F., Dragalin, V., Koenig, F., and Wassmer, G.: Authors’ response to comments. Statistics in Medicine, 35(3): pp. 364-367, February 2016.
Bauer, P., Bretz, F., Dragalin, V., Koenig, F., and Wassmer, G.: Twenty-five years of confirmatory adaptive designs: opportunities and pitfalls. Statistics in Medicine, 35(3): pp. 325-347, February 2016.
Hlavin, G., Koenig, F., Male, C., Posch, M., and Bauer, P.: Evidence, eminence and extrapolation. Statistics in Medicine, 35(13): pp. 2117-2132, June 2016.
Magirr, D., Jaki, T., Koenig, F., and Posch, M., Sample Size Reassessment and Hypothesis Testing in Adaptive Survival Trials. PLoS ONE, 11(2): e0146465, February 2016.
Alonso, A., Van der Elst W., Molenberghs, G., Buyse, M., and Burzykowski, T.: An information-theoretic approach for the evaluation of surrogate endpoints based on causal inference. Biometrics, 72(3): pp. 669-677, September 2016.
Rübben, A., Hilgers, R.-D., Leverkus, M.: Hedgehog Blockade for Basal Cell Carcinoma. Coming at a (Secondary Neoplastic) Price. JAMA Dermatology, 152(5): pp. 521-523, May 2016.
Van der Elst, W., Hermans, L., Verbeke, G., Kenward, M. G., Nassiri, V., and Molenberghs, G.: Unbalanced cluster sizes and rates of convergence in mixed-effects models for clustered data. Journal of Statistical Computation and Stimulation, 86(11): pp. 2123-2139, October 2016.
Jobjörnsson S., Forster M., Pertile P., Burman C.-F.: Late-Stage Pharmaceutical R&D for Rare Diseases under Two-Stage Regulation. Journal of Health Economics, 50: pp. 298-311, December 2016.
Senn, S.: Mastering variation: variance components and personalised medicine. Statistics In Medicine, 35(7): pp. 966-977, March 2016.
Hermans, L., Molenberghs, G., Kenward, M.G., Van der Elst, W., Nassiri, V., Aerts, M., and Verbeke, G.: Clusters with random size: maximum likelihood versus weighted estimation. In: Friedl, H., and Wagner, H. (eds.) Proceedings of the 30th International Workshop on Statistical Modelling. Volume 1, pp.215-220, July 2015.
Hecksteden, A., Kraushaar, J., Scharhag-Rosenberger, F., Theisen, D., Senn, S., and Meyer, T.: Individual response to exercise training – a statistical perspective. Journal of Applied Physiology, 118(12): pp. 1450-1459, June 2015.
Bogdan, M., van den Berg, E., Sabatti, C., Su, W., Candès, E.J.: SLOPE — Adaptive Variable Selection via Convex Optimization. Annals of Applied Statistics. 9(3): pp. 1103-1140, September 2015.
Alonso Abad, A., Van der Elst, W., Molenberghs, G., Burzykowski, T., and Buyse, M.: On the relationship between the causal-inference and meta-analytic paradigms for the validation of surrogate endpoints. Biometrics, 71(1): pp. 15-24, March 2015.
Van der Elst, W., Molenberghs, G., Alonso, A. A.: Exploring the relationship between the causal-inference and meta-analytic paradigms for the evaluation of surrogate endpoints. Statistics in Medicine, 35(8): pp. 1281-1298, April 2016.
Lendrem, D., Senn, S.J., Lendrem B.C., Isaacs, J.D.: R& D productivity rides again? Pharmaceutical Statistics, 14(1): pp. 1-3, January 2015.
Van der Elst, W., Molenberghs, G.: Surrogate endpoints in rare diseases. In: Applied surrogate evaluation methods with SAS and R. 1st Edition, Chapman & Hall/CRC Biostatistics Series, July 2016.
Greenland, S., Senn, S., Rothman, K., Carlin, J., Poole, C., Goodman, S. and Altman, D.: Statistical tests, P values, confidence intervals, and power: a guide to misinterpretations. European Journal of Epidemiology, 31(4): pp. 337-350, April 2016.
Alonso, A., Van der Elst, W., and Molenberghs, G.: Validating predictors of therapeutic success: a causal inference approach. Statistical Modelling, 15(6): pp. 619-636, December 2015.
Kennes, L.N., Rosenberger, W.F., and Hilgers, R.-D.: Inference for Block Randomization Under a Selection Bias Model. Biometrics, 71(4): pp. 979-984, December 2015.
Graf, A., Posch, M., and Koenig, F.: Adaptive designs for subpopulation analysis optimizing utility functions. Biometrical Journal, 57(1): pp. 76-89, January 2015.
Klinglmueller, F., Posch, M., and Koenig, F.: Adaptive graph-based multiple testing procedures. Pharmaceutical statistics, 13(6): pp. 345-356, October 2014.
Tamm, M., and Hilgers, R.-D.: Chronological bias in randomized clinical trials arising from different types of unobserved time trends. Methods of Information in Medicine, 53(06): pp. 501-510, 2014.
Senn, S.: A note regarding meta-analysis of sequential trials with stopping for efficacy. Pharmaceutical Statistics, 13(6): pp. 371-375, November 2014.
Koenig, F., Slattery, J., Groves, T., Lang, T., Benjamini, Y., Day, S., Bauer, P., and Posch, M.: Sharing clinical trial data on patient level: Opportunities and challenges. Biometrical Journal, 57(1): pp. 8-26, January 2015.
Gewandter, J.S., Dworkin, R.H., Turk, D.C., McDermott, M.P., Baron, R., Gastonguay, M.R., Gilron, I., Katz, N.P., Mehta, C., Raja, S.N., Senn, S., Taylor, C., Cowan, P., Desjardins, P., Dimitrova, R., Dionne, R., Farrar, J.T., Hewitt, D.J., Iyengar, S., Jay, G.W., Kalso, E., Kerns, R.D., Leff, R., Leong, M., Petersen, K.L., Ravina, B.M., Rauschkolb, C., Rice, A.S., Rowbotham, M.C., Sampaio, C., Sindrup, S.H., Stauffer, J.W., Steigerwald, I., Stewart, J., Tobias, J., Treede, R.D., Wallace, M., White, R.E.: Research designs for proof-of-concept chronic pain clinical trials: IMMPACT recommendations. PAIN, 155(9): pp. 1683-1695, September 2014.
Graf, A., Bauer, P., Glimm, E., Koenig, F.: Maximum type 1 error rate inflation in multiarmed clinical trials with adaptive interim sample size modifications. Biometrical Journal, 56(4): pp. 614-630, July 2014.

E-Publications

Ondra, T., Jobjörnsson, S., Beckman, R., Burman, C.-F., König, F., Stallard, N. and Posch, M.: Optimizing Trial Designs for Targeted Therapies. [June 13. 2016]
Brzyski, D., Christine, P., Sobczyk, P., Candes, E., Bogdan, M. and Sabatti, C.: Controlling the rate of GWAS false discoveries. [June 10. 2016]
Dette, H., Schorning, K. and Konstantinou, M.: Optimal designs for comparing regression models with correlated observations. [January 25. 2016]

Presentations

Various settings in ERDO – a framework to select the best practice randomization procedure in clinical trials. R.-D. Hilgers 2018 May 1, Design of Experiments: New Challenges Plans d’ expériences: nouveaux défis CIRM, Luminy
Optimal designs for trials with discrete longitudinal data analyzed by nonlinear mixed effect models. F. Mentré, 2018 May 3, Design of Experiments: New Challenges Plans d’ expériences: nouveaux défis CIRM, Luminy
Biostatistical Challenges of Trials in Rare Diseases, IDDI WebinarRalf-Dieter Hilgers and Everardo Saad 2018 April 26
IDeAl – New developments on Design and Analysis of Small Population GroupR.-D. Hilgers 2018 March 27 64. Biometrisches Kolloquium, Frankfurt am Main
ERDO – A framework for scientic evaluation of the best practice randomization procedure in clinical trialsR.-D. Hilgers and N. Heussen 2018 March 27 64. Biometrisches Kolloquium, Frankfurt am Main
Small Population Clinical Trials – Past, Present and Future R.-D. Hilgers 2017 October 16 Annual Novartis Basel Biostatistics Conference, Basel
Using Hamiltonian Monte-Carlo to design longitudinal studies with discrete outcomes accounting for parameter and model uncertainties Florence Loingeville, Jérémy Seurat, ThuThuy Nguyen,Marie-Karelle Riviere,France Mentré 2017 September 8 PODE, Basel
Thinking Statistically. S. Senn. 2017 May 17. Invited Talk. 37th Conference on Applied Statistics in Ireland (CASI 2017).
Randomisation isn’t perfect but doing better is harder than you think. S. Senn. 2017 May 3. Invited Talk. Fourth Bayesian, Fiducial, and Frequentist Conference (BF F4).
Personalised medicine? Don’t hold your breath. S. Senn. 2017 April 27. Invited Talk. SAS Analytics conference Luxembourg.
P-Value Wars. S. Senn. 2017 April 6. Invited Talk. Cardiff University, UK.
Historical controls: think cluster, not parallel. S. Senn, O. Collignon, A. Schritz 2017 March 30. Invited Talk FP7 Small-population research methods projects and regulatory application workshop. European Medicines Agency. London, UK.
Surrogate marker evaluation when data are small, large, or very large. G. Molenberghs, O. Collignon, A. Schritz 2017 March 30. Invited Talk FP7 Small-population research methods projects and regulatory application workshop. European Medicines Agency. London, UK.
Improved methodology for use of non-linear mixed effect models (NLMEM) in decision making. M.O. Karlsson, A.C. Hooker 2017 March 30. Invited Talk FP7 Small-population research methods projects and regulatory application workshop. European Medicines Agency. London, UK.
Optimal design for trials with discrete longitudinal studies with uncertainty on model and parameters. F. Mentré, F. Loingeville, M.K. Rivière, T.T. Nguyen 2017 March 30. Invited Talk FP7 Small-population research methods projects and regulatory application workshop. European Medicines Agency. London, UK.
Overview on IDeAl Integrated Design and AnaLysis of small population group trials. R.-D. Hilgers 2017 March 30. Invited Talk FP7 Small-population research methods projects and regulatory application workshop. European Medicines Agency. London, UK.
Threshold-crossing: A Useful Way to Establish the Counterfactual in Clinical Trials? DIA 29th Annual Euromeeting. König, F., Invited Talk. 30 March, 2017, Glasgow, UK.
Evidence synthesis. S. Aymé 2017 March 29. Discussion FP7 Small-population research methods projects and regulatory application workshop. European Medicines Agency. London, UK.
Combining evidence: Purpose is everything. S. Senn 2017 March 29. Invited Talk FP7 Small-population research methods projects and regulatory application workshop. European Medicines Agency. London, UK.
Equivalence of dose-response curves. H. Dette, K. Möllenhoff, S. Volgushev, F. Bretz 2017 March 29. Invited Talk FP7 Small-population research methods projects and regulatory application workshop. European Medicines Agency. London, UK.
Evidence and Decisions. C.-F. Burman 2017 March 29. Invited Talk FP7 Small-population research methods projects and regulatory application workshop. European Medicines Agency. London, UK.
Does the level of evidence depend on randomization? R.-D. Hilgers, N. Heussen, D. Uschner 2017 March 29. Invited Talk FP7 Small-population research methods projects and regulatory application workshop. European Medicines Agency. London, UK.
Adaptive Levels of Evidence: An extrapolation framework to specify requirements for drug development in children. F. König 2017 March 29. Invited Talk FP7 Small-population research methods projects and regulatory application workshop. European Medicines Agency. London, UK.
The Challenge of “Small Data” – Rare Diseases and Ways to Study Them. S. Senn. 2017 March 22. Invited Talk. Joint AstraZeneca-MRC conference ‘All data great and small’, Cambridge, UK.
Statistical Methodology for comparing curves. Dette, Holger. Plenary Speaker at 13th Workshop on Stochastic Models, Statistics and Their Applications, 2017 February 20-24, Berlin, Germany.
Innovative statistical design methodologies for clinical trials in small populations focussing on rare diseases. R.-D. Hilgers. 2017 March 20. Invited talk, Committee for Orphan Medicinal Products (COMP) Strategic Review and Learning Meeting, Valletta, Malta.
Minimally important differences: definitions, ambiguities and pitfalls. S. Senn. 2017 March 8. Invited talk, 2nd EuroQol Academy Meeting, Nordwijk, Netherlands.
IDeAl Designing a Clinical Trial – a Case Study. R.-D. Hilgers, G. Molenberghs. 2017 Februar. Invited talk, 3rd IRDiRC Conference, Paris, France.
P-value wars. S. Senn. 2017 February 23. Invited talk, EMA, London, UK.
Respone, Quality and Variation – What drug development may be missing. S. Senn. 2017 February 22. Invited talk, Statsols, Cork, Ireland.
Does Randomization protect against bias? What can be done to improve the level of clinical evidence of effectiveness. R.-D. Hilgers. 2017 Februar. Invited talk, Heidelberger Kolloquium, Heidelberg, Germany.
What randomisation can and cannot do for you. S. Senn. 2017 January 27. Invited talk, Drug Safety, Probabilistic Causal Assessment, and Evidence Synthesis, LMU Munich, Germany.
On being Bayesian. WBS Herbst Seminar 2016. Senn, S., Invited Talk. 9 November 2016. Vienna, Austria
Academic and Regulatory Aspects of Data Sharing. Clinical Forum for Operational Excellence. König, F., Invited Talk. 27-28 October 2016. Düsseldorf, Germany
Adaptive Designs Today, Key Principles and Outlook. Clinical Forum for Operational Excellence. König, F., Invited Talk. 27-28 October 2016. Düsseldorf, Germany.
The selection of a randomization procedure to avoid the impact of bias on the test result in clinical trials – A case study! WBS Herbst Seminar 2016. Hilgers, R.-D., Invited Talk, 9 November 2016, Vienna, Austria
On Being Bayesian. S. Senn. 2016 October 13. Invited talk, 24th Annual Meeting of the Belgian Statistical Society, Palais de Congres, Namur, Belgium.
Optimizing Trial Designs for Targeted Therapies – A decision theoretic approach comparing sponsor and public health. Seminar der Wiener Biometrischen Sektion der Internationalen Biometrischen Gesellschaft Region Österreich – Schweiz, Vienna, Austria. Ondra, T. und Jobjörnsson, S., Invited Talk. 2016 September 29.
Clinical trials: three statistical traps for the unwary. S. Senn. 2016 September 15. Invited talk, Challenges in drug development : bridging scientific excellence with patients need, University of Geneva, Geneva, Switzerland.
P-values and the art of herding cats RSS conference of the royal statistical society, Senn, S., Invited Talk. 2016 September 07. Manchester, UK.
Predicting patient’s response to the treatment based on high dimensional genetic data. M Bogdan. 2016 August 26. International Conference on Computational Statistics 2016, Contributed talk, Oviedo, Spain.
Approximate analysis of covariance in trials in rare diseases, in particular rare cancers. S. Senn. 2016 August 25. International Society for Clinical Biostatistics Conference 2016, Mini-Symposium: Statistical methods in rare diseases and small populations, Birmingham, UK.
Randomization based Inference – How to deal with missing observations. N. Heussen. 2016 August 25. International Society for Clinical Biostatistics Conference 2016, Birmingham, UK.
Estimability and bias of the treatment effect in a classical cohort stepped wedge design under consideration of carry-over effects. Foldenauer, A. 2016 August 24. International Society for Clinical Biostatistics Conference 2016, Birmingham, UK.
A Decision Theoretic Approach to Optimize Clinical Trial Designs for Targeted Therapies. M. Posch. 2016 August 23. Contributed talk International Society for Clinical Biostatistics Conference 2016, Birmingham, UK.
Multi-armed platform trials in small populations using a two-step testing procedure incorporating efficacy and safety. G. Hlavin. 2016 August 23. Contributed talk International Society for Clinical Biostatistics Conference 2016, Birmingham, UK.
Response-Adaptive Randomization and Adaptive Design in Small Population Clinical Trials: Pitfalls and Advantages. F. König. 2016 August 22. Contributed talk International Society for Clinical Biostatistics Conference 2016, Birmingham, UK.
N of 1 Trials, Statistical Inference and Rare Diseases. S. Senn. 2016 August 22. Contributed talk International Society for Clinical Biostatistics Conference 2016, Birmingham, UK.
Assessment of randomization procedures with respect to multiple objectives. D. Schindler. 2016 August 22. Contributed talk International Society for Clinical Biostatistics Conference 2016, Birmingham, UK.
A Selection Bias Adjusted Randomization Test. D. Uschner. 2016 August 22. Contributed talk International Society for Clinical Biostatistics Conference 2016, Birmingham, UK.
A framework for scientific evaluation of randomization procedures in small clinical trials. R.-D. Hilgers. 2016 August 22. Contributed talk International Society for Clinical Biostatistics Conference 2016, Birmingham, UK.
The impact of selection and chronological bias on test decisions in survival analysis. M. Rückbeil. 2016 August 22. Contributed talk International Society for Clinical Biostatistics Conference 2016, Birmingham, UK.
Efficient tests to demonstrate the similarity of dose response curves with regard to small sample size. K. Möllenhoff. 2016 August 22. Contributed talk International Society for Clinical Biostatistics Conference 2016, Birmingham, UK.
Flexibility in confirmatory clinical trials – what is for free?. P. Bauer. 2016 June 27. Summer Symposium 2016 of the International Biometric Society Viennese Section.
Optimal trial designs for Targeted Therapies. S. Jobjörnsson. 2016 June 24. Contributed talk, EFSPI Biomarkers and Subgroups workshop, Leiden, Netherlands.
Optimal Designs for Comparing Curves. K. Schorning. 2016 June 16. Contributed talk, mODa 11. Hamminkeln-Dingden, Germany.
Assessment of Randomization Procedures with Respect to the Influence of Bias on Type 1 error Elevation. R.-D. Hilgers. 2016 June 14. Contributed talk, mODa 11. Hamminkeln-Dingden, Germany.
Linked Assessment Criterion for the Choice of a Randomization Procedure. D. Schindler and R.-D. Hilgers. 2016 June 14. Contributed talk, mODa 11. Hamminkeln-Dingden, Germany.
An extrapolation framework to specify requirements for drug development in children. F. König, G. Hlavin, P. Bauer. 2016 May 25. PSI Conference, Berlin, Germany.
Pseudo-likelihood and split-sample methods in small populations/small trials: IDEAL & ExaScience. G. Molenberghs. 2016 May 23. Invited presentation, Salvador, Brazil.
FDASA presents: Herausforderungen der individualisierten Medizin (Challanges of Personlized Medicine). R.-D. Hilgers, and N. Heussen. 2016 May 19. Fachvertreterkonferenz, Münster, Germany
FDASA presents: Does Randomization protect against bias? R.-D. Hilgers, and N. Heussen. 2016 May 6. FDA, Silver Spring, Maryland, USA.
The Challenge of “Small Data” Rare Diseases and Ways to Study Them. Senn, S., Invited Talk. 2016 May 5.
Regulatory and methodological issues in adaptive designs for confirmatory trials. F. König, M.Posch. 2016 May 4. BBS Seminar Adaptive Designs, Basel, Switzerland.
An extrapolation framework to specify requirements for drug development in children. F. König, P. Bauer, G. Hlavin. 2016 May 3. Novartis Basel, Switzerland.
Evidence, Eminence and Extrapolation – the background . F. König, P. Bauer, C. Male, G. Hlavin. 2016 May 3. Novartis Basel, Switzerland.
A little bit me, a little bit you: N of 1 trials, random effects and shrinkage estimators. S. Senn. 2016 May 2. SAS sponsored Symposium, Raleigh Durham USA.
Strategies for dealing with missing data in randomization tests. N. Heussen. 2016 April 29. Invited talk. Clifton Bailey Seminar Series Spring 2016, George Mason University, Fairfax, Virginia, USA.
The Challenge of “Small Data” Rare Diseases and Ways to Study Them. S. Senn. 2016 April 28. Vanda Pharmaceuticals
Can we optimize rare disease trials?. C.-F. Burman. 2016 April 27. DIA/FDA Statistics.
P-values? The problem is not what you think. S. Senn. 2016 April 22. Key Note Invited talk, FLAMES Annual Meeting 2016, Antwerp, Belgium.
Aspects for the scientific evaluation of randomization procedures in small clinical trials R.-D. Hilgers. 2016 April 22. Invited talk. Clifton Bailey Seminar Series Spring 2016, George Mason University, Fairfax, Virginia, USA.
How to Adjust for Multiplicity if you must: The Casino Approach. C.-F. Burman. 2016 April 20. Health Metrics Seminar, Gothenurg, Sweden.
The Challenge of Small Data. S. Senn. 2016 March 24. Invited talk. Luxembourg Analytics Summit. Hamm, Luxembourg.
Validating the similarity of regresion curves with regard to small samples. K. Möllenhoff, H. Dette. 2016 March 16. Contributed talk, DAGStat 2016. Göttingen, Germany.
The impact of bias on different randomization procedures. D. Schindler, N. Heussen, D. Uschner, R.-D. Hilgers. 2016 March 15. Contributed talk, DAGStat 2016. Göttingen, Germany.
Optimizing trial designs for targeted therapies – A decision theoretic approach comparing sponsor and public health perspectives. T. Ondra, S. Jobjörnsson. 2016 February 11. Contributed talk to Seminar der Wiener Biometrische Sektion der Internationalen Biometrischen Gesellschaft Region Österreich – Schweiz. Medical University of Vienna, Austria.
Precision medicine? Don’t hold your breath. S. Senn. 2016 February 2. Royal College of Surgeons, Dublin, Ireland.
Variations on a theme of variances. S. Senn. 2016 January 27. Astra Zeneca Gothenburg, Sweden.
P-values? The problem is not what you think. S. Senn. 2016 January 26. Chalmers University, Gothenburg, Sweden.
Late-Stage Pharmaceutical R&D for Rare Diseases under Two-Stage Regulation. S. Jobjörnsson, M. Forster, P. Pertile, C.-F. Burman. 2016 January 18. Contributed talk by M. Forster, York Economics Workshop, York, Great Britain.
Precision medicine? Don’t hold your breath. S. Senn. 2016 January 15. European Medicine Agency.
Pseudo-likelihood and split-sample methods in small populations/small trials: IDEAL & ExaScience. G. Molenberghs. 2016 January 13. Contributed Talk, London, UK.
Take it to the limit: quantitation, likelihood, modelling, inference and other matters. S. Senn. 2015 December 17. BPS Pharmacology, London, England.
P-values? The problem is not what you think. S. Senn. 2015 December 16. University of Cambridge, England.
Computation of the Fisher information matrix for discrete nonlinear mixed effect models. F. Mentre. 2015 December 12-14. 8th International Conference in Computational and Methodological Statistics, London, UK.
From Optimal Design Theory to Optimizing Designs of Clinical Trials. C.-F. Burman. 2015 December 9. Symposium (celebrating Hans Nyquist’s 65 years) at Stockholm Univerisity, Sweden.
Integrated Design and Analysis of Clinical Trials in Small Population Groups (The IDeAl Project). R.-D. Hilgers. 2015 November 30. Invited talk. Clinical Trials in Small Populations Workshop, University of Lancaster. Lecture Theatre, Royal Statistical Society, London.
The crisis of reproduceibility. P-values are not the problem. S. Senn. 2015 November 24. Invited talk. University of Zurich, Switzerland.
Evaluation of the Bayesian Individual Information Matrix in Nonlinear Mixed Effect Models using Monte Carlo integration. Workshop on Population Optimal Designs of Experiments. Loingeville, F., Nguyen, T., Rivière, M.-K. & Mentré, F., Contributed Talk. 2015 November 20. Uppsala, Sweden.
randomizeR – An R-package that implements randomization and considers biases. D. Uschner. 2015 October 28. Contributed talk and live demonstration. National Institute of Health, Rockville, Maryland, USA.
Evidence, Eminence and Extrapolation. F. König, P. Bauer, C. Male, G. Hlavin. 2015 October 17. 20th Young Statisticians Meeting, Vorau, Austria.
How to use randomizeR to investigate randomization tests in the presence of selection bias. D. Uschner. 2015 October 16. Invited talk, Statistics Seminar Series, George Mason University, Fairfax, Virginia, USA.
Late-Stage Pharmaceutical R&D for Rare Diseases under Two-Stage Regulation. S. Jobjörnsson, M. Forster, P. Pertile, C.-F. Burman. 2015 October 15-16. AEIS, Alghero, Italy.
New developments in the design and analysis of small population group trials. R.-D. Hilgers. 2015 October 12. Invited talk. Annual Novartis Biostatistics Conference, Basel, Switzerland.
Selecting an appropriate randomization procedure for a small population group trial on the basis of a linked optimization criterion. D. Schindler, R.-D. Hilgers. 2015 September 24. Contributed talk. International Workshop on Simulation, Vienna, Austria.
A software tool for the design and analysis of small population group trials. D. Uschner, D. Schindler, R.-D. Hilgers. 2015 September 24. Contributed talk, International Workshop on Simulation, Vienna, Austria.
Evidence, Eminence and Extrapolation. F. König, P. Bauer, C. Male, G. Hlavin. 2015 September 24. Contributed talk, International Workshop on Simulation, Vienna, Austria.
Adaptive designs for confirmatory model based decisions using MCP-Mod. S. Krasnozhon. 2015 September 24. Contributed talk, International Workshop on Simulation, Vienna, Austria.
Evaluation of the expected Fisher information matrix without linearization, in nonlinear mixed effect models for discrete and continuous outcomes. M.-K. Riviere. 2015 September 24. Contributed talk, International Workshop on Simulation, Vienna, Austria.
Statistical issues in the design of n-of-1 trials in small populations. A. Araujo. 2015 September 21. Life Sciences PhD Days 2015. Luxembourg.
Strategies for Dealing with Missing Values in Randomization Tests Under Different Randomization Procedures. 9th International Conference on Multiple Comparison Procedures, Heussen, N., Contributed Talk. 2015 September 2-5. Hyderabad, India.
A Unified Criterion for Assessing the Biased Test Decision in Cases of Selection and Chronological Bias. 9th International Conference on Multiple Comparison Procedures, Hilgers, R.-D., Contributed Talk. 2015 September 2-5. Hyderabad, India.
Some Aspects of Clinical Trials in Small Population Groups. 9th International Conference on Multiple Comparison Procedures, Hilgers, R.-D., Invited Talk. 2015 September 2-5. Hyderabad, India.
Science, logic and patients. On inference in small disease populations. C.-F. Burman. 2015 September 2-5. Invited talk and followed by pannel discussion. MCP, Hyderabad, India.
Two-Step Procedures Using Safety for Multiplicity Adjustments in Many-One Tests. F. König. 2015 September 2-5. MCP, Hyderabad, India.
Strategies for dealing with missing data in randomization tests. N. Heussen. 2015 August 24. Contributed talk, ISCB Utrecht 2015, Utrecht, Netherlands.
Understanding variation in n-of-1 trials A. Araujo, S. Julious, S. Senn, . 2015 August 24. Contributed talk, ISCB Utrecht 2015, Utrecht, Netherlands.
New developments in integrated design and analysis of small population group trials (IDeAl) R.-D. Hilgers. 2015 August 24. Invited talk, ISCB Utrecht 2015, Utrecht, Netherlands.
Baselines as possible controls in trials in small populations S. Senn. 2015 August 24. Contributed talk, ISCB Utrecht 2015, Utrecht, Netherlands.
The effect of selection bias on the test decision of the randomization test in small population group trials. D. Uschner. 2015 August 24. Contributed talk, ISCB Utrecht 2015, Utrecht, Netherlands.
Desirability of restricted randomization procedures in small population group trials in case of selection and chronological bias. D. Schindler. 2015 August 24. Contributed talk, ISCB Utrecht 2015, Utrecht, Netherlands.
Efficient tests for the similarity of dose response curves using a bootstrap approach in regard to small sample sizes. K. Möllenhoff, H. Dette. 2015 August 24. Contributed talk, ISCB Utrecht 2015, Utrecht, Netherlands.
Evaluation of the fisher information matrix without linearization, in nonlinear mixed effect models for discrete and continuous outcomes. M.K. Riviere, S. Ueckert, F. Mentré. 2015 August 24. Contributed talk, ISCB Utrecht 2015, Utrecht, Netherlands.
Randomisation: Misunderstanding, myths, and truth. S. Senn. 2015 August 5. Brussel Summer School of Mathematics Université Libre de Bruxelles, Brussels, Belgium.
Designing an Adaptive Trial with Treatment Selection and a Survival Endpoint. Design and Analysis of Experiments in Healthcare. Jennison, C., 2015 July 6. Design and Analysis of Experiments in Healthcare Cambridge, UK.
Computation of the Fisher information matrix for discrete nonlinear mixed effects models. Design and Analysis of Experiments in Healthcare. Ueckert, S., Riviere, M.-K., Mentré, F., 2015 July 07. Design and Analysis of Experiments in Healthcare Cambridge, UK.
Design for dose-escalation trials. Bailey R., 2015 July 08. Design and Analysis of Experiments in Healthcare Cambridge, UK.
Efficient study designs. S. Julious. 2015 July 10. Design and Analysis of Experiments in Healthcare Cambridge, UK.
Randomization for small clinical trials. Rosenberger, W. F., 2015 July 10. Design and Analysis of Experiments in Healthcare Cambridge, UK.
Optimal designs for correlated observations H. Dette. 2015 July 10. Design and Analysis of Experiments in Healthcare Cambridge, UK.
Assessment of randomization procedures based on single sequences under selection and chronological bias R.-D.Hilgers, S. Langer. 2015 July 10. Design and Analysis of Experiments in Healthcare Cambridge, UK.
Confirmatory Testing for a Beneficial Treatment Effect in Dose-Response Studies using MCP-Mod and an Adaptive Interim Analysis F. König. 2015 July 9. Design of Experiments in Drug Development, Cambridge, UK.
IDeAl (Integrated design and analysis of small population group trials)- A Collaborative R.-D.Hilgers. 2015 July 9. Design of Experiments in Drug Development, Cambridge, UK.
Adaptive MCPMod -Adaptive Contrast Selection and confirmatory testing F. König. 2015 July 8. Addplan User Consortium Meeting – Cambridge, UK.
10 years of progress in population design methodology and applications. F. Mentré. 2015 July 7. Design and Analysis of Experiments in Healthcare Cambridge, UK.
Evaluation of the Fisher information matrix in nonlinear mixed effect models without linearization S. Ueckert, F. Mentré, M.-K. Riviere. 2015 July 7. Design and Analysis of Experiments in Healthcare Cambridge, UK.
Regulatory and methodological issues in adaptive designs for confirmatory trials. F. König, M.Posch. 2015 July 2-3. Novartis China Biostatistics Workshop. Shanghai, China .
Repeat after me! Bayes will not cure “the crisis of reproducibility “. S.Senn. 2015 June 29. International Society for Biopharmaceutical Statistics & Drug Information Association Meeting, Beijing, China.
Exploring subgroups: when is enough, enough?. S. Senn. 2015 June 29. International Society for Biopharmaceutical Statistics & Drug Information Association Meeting, Beijing, China.
Confirmatory adaptive designs using closed-test MCP-Mod. F. König. 2015 June 27. International Society for Biopharmaceutical Statistics & Drug Information Association Meeting, Beijing, China.
Response-Adaptive Randomization and Adaptive Combination Test for Clinical Trials with Limited Number of Patients: Practical Guide. S. Krasnozhon, D. Schindler, R.-D. Hilgers, N. Heussen, W.F. Rosenberger, F. König. 2015 June 25. Adaptive Design and Multiple Testing Procedures Workshop – Cologne, Germany.
Adjusting multiplicity using safety data in many-one comparisons. F. König, P.Bauer, G. Hlavin. 2015 June 25. Adaptive Design and Multiple Testing Procedures Workshop – Cologne, Germany.
Evidence, Eminence and Extrapolation – Adjusted Levels of Evidence in Small Populations. F. König, P. Bauer, C. Male, G. Hlavin. 2015 June 18. IROeS 2015 – Mailand, Italy.
Optimizing the biomarker subpopulation strategy in late stage clinical development. C.-F. Burman. 2015 June 14-18. Invited talk, DIA 51st Annual Meeting. Washingtion DC, USA.
Pseudo-likelihood and split-sample methods in small populations/small trials: IDEAL & ExaScience. G. Molenberghs. 2015 June 11. Invited presentation, Utrecht, the Netherlands.
Clusters with random size: maximum likelihood versus weighted split-sample pseudo-likelihood. G. Molenberghs. 2015 June 5. Contributed presentation, Brussels, Belgium.
Alternative to resampling methods in maximum likelihood estimation for NLMEMs by borrowing from Bayesian methodology. S. Uekert. 2015 June 4. Invited talk, Population Approach Group in Europe, Hersonisos, Greece.
Late-Stage Pharmaceutical R&D for Rare Diseases under Two-Stage Regulation. S. Jobjörnsson, M. Forster, P. Pertile, C.-F. Burman. 2015 May 28-29. Contributed talk by P. Pertile, 16th European Health Workshop, Toulouse, France.
Pharmaceutical Phase III investments with uncertain reimbursement. S. Jobjörnsson. 2015 May 27. Contributed talk. Kiel University, Germany.
‘Repligate.’ Why and How Should We Care About Reproducing Study Results? S. Senn. 2015 May 23. 27th Convention of the Association for Psychological Science, New York, USA.
European Research Projects on Statistical Methodology to Enhance Clinical Trial Designs and Analysis. König, F., Pharmaceuticals and Medical Devices Agency, Japan (PMDA) 2015 March 23.
Adaptive graph-based multiple testing procedure. F. König 2015 May 21. PSI & RSS Webinar.
Foundations of adaptive and group sequential designs. F. König. 2015 May 19. Internal Seminar at Institut für Medizinische Statistik, Uniklinik RWTH Aachen, Germany.
Meta-analysis of rare (binary) events S.Senn. 2015 May 11. PSI Annual Conference. London, UK.
Adaptive designs for confirmatory model based decisions using MCP-Mod. Krasnozhon, S., WBS Winter Seminar 2014 “Innovative Statistical Approaches in Drug Development”. December 2014. Vienna, Austria.
Multiplicity issues in adaptive clinical trials. König, F., Pharmaceuticals and Medical Devices Agency, Japan (PMDA). 2015 March 23.
Regulatory and methodological issues in adaptive designs for confirmatory trials. F. König. 2015 Mar 30. Joint Stat Conference, Tokyo, Japan.
Sharing clinical trial data on patient level. König, F., Posch, M., 2015 Mar 12. Workshop ‘Health outcomes in an era of cost containment Improving efficiency of interventional research: decreasing costs, increasing quality’. Paris, France.
An overview of new methods and tools for model building, evaluation and utilization. Karlsson, M., International Society of m (ISoP), Lambertville/NJ/USA, 2015 March 18.
Efficient tests for the similarity of dose response curves. K. Möllenhoff, H. Dette. 2015 Mar 18. 61. Biometrisches Kolloquium. Dortmund, Germany.
SLOPE – Adaptive Variable Selection via Convex Optimization. Bogdan, M., van den Berg, E., Sabatti, C., Su, W., Candes, E. 12^th German-Polish Workshop on Stochastic Models, Statistics and Their Applications. 2015 February.
Some Aspects of Clinical Trials in Small Population Groups with Special Interest in Randomization. Hilgers, R.D., WBS Winter Seminar 2014 “Innovative Statistical Approaches in Drug Development”. 2. December 2014. Vienna, Austria.
Seven myths of randomization. Stephen S., WBS Winter Seminar 2014 “Innovative Statistical Approaches in Drug Development”. 2. December 2014. Vienna, Austria.
Joint Genotype and Ancestry based Genome-wide Association Studies in Admixed Populations. Bogdan, M., Frommlet, F., Szulc, P., Tang, H., 12^th German-Polish Workshop on Stochastic Models, Statistics and Their Applications. 2015 February.
A Linked Optimization Criterion for the Assessment of Selection and Chronological Bias in Randomized Clinical Trials. D. Schindler, R.-D. Hilgers. 2015 March 18. Contributed talk, 61. Biometrisches Kolloquium. Dortmund, Germany.
Pseudo-likelihood methodology for partitioned large and complex samples. G. Molenberghts. 2015 January 16. Contributed talk, Leuven, Belgium.
Statistical inference for comparing dose-response curves. Dette, H., WBS Winter Seminar 2014 “Innovative Statistical Approaches in Drug Development”. 2. December 2014. Vienna, Austria.
Adapted levels of Evidence for small populations. Evidence, Eminence & Extrapolation. Hlavin, G., WBS Winter Seminar 2014 “Innovative Statistical Approaches in Drug Development”. 2. December 2014. Vienna, Austria.
Model selection approach for genome wide association studies. M. Bogdan, F. Frommlet, P. Szulc, H. Tuang. 2014 Dec 6-8. 7th International Conference of the ERCIM WG on Computational and Methodological Statistics. Pisa, Italy.
Slope – Sorted L-One Penalized Estimation. Bogdan, M., van den Berg, E., Su, W., Candes, E., Biostatistics Seminar of Indiana University School of Public Health. 2014 November.
Big thunder, little rain? F. König. 2014 November 13. Invited Talk, Joint BBS-EFSPI Seminar Data Sharing in Clinical Development. Basel, Switzerland.
Open Data and closed minds. S. Senn. 2014 October, Kopenhangen, Denmark
Variabilty in Drug Response. S. Senn. Defining Drug Response for Stratified Medicine. 2014 October 23. UK Pharmacologenetics and Stratified Medicine Network. University of Liverpoool in London, UK.
Chronological bias caused by unobserved time trends in randomized clinical trials. M. Tamm. 2014 Oct 11. AISC Greensboro, North Carolina, USA.
Subgroup analysis: trying to get more from less? S. Senn, G. Molenbergh (Presenter), F. Koenig, R.-D. Hilgers. IDeAl presentation at European Medicines Agency workshop on the investigation of subgroups in confirmatory clinical trials. 2014 November 07. London, UK.
Stakeholder perspectives on drug development decision making. C.-F. Burman. Biometric Colloquium, 2014 November 12. Hannover, Germany.
Confirmatory testing for a beneficial treatment effect in dose-response studies using MCP-Mod. F. König, B. Bornkamp, F. Bretz, E. Glimm. Contributed Talk, 2014 August 24-27. ISCB 2014 Vienna, Austria.
Mastering variation: variance components and personalised medicine. S. Senn. Contributed Talk, 2014 August 25. ISCB 2014 Vienna, Austria.
Assessment of chronological bias in randomized controlled clinical trials. M. Tamm. Contributed Talk. 2014 Aug 25. ISCB 2014 Vienna, Austria.
Lightning Talks – various topics. G. Hlavin, S. Krasnozhon, K. Kerkmann, D. Uschner, D. Schindler, M. Tamm, A. Araujo, D. Brzyski, P. Sobczyk, M.-K. Riviere, C. Louenan, K. Karlsson, A.-G. Dosne, S. Jobjörnsson. 2014 Jun 30. IDEAL YSM Vienna, Austria.
Pharmacometrics – a useful methodology in small clinical trials. Karlsson, K.E., Symposium on Small Populations, Vienna, Austria. 2014 July.
Overcoming Resistance: Trials, Tribulations and Treatments. S. Senn. Invited Talk. 2014 Jul 02. Freiburg, Germany.
A picture is worth a 1000 words – usually. Communicating Complex Statistical Concepts. S. Senn. Invited Talk.
Flexibility in confirmatory clinical trials – what is for free? Workshop “Adaptive Designs and Multiple Testing Procedures” of the Joint Working Group (Section) of the ROeS and the German Region of the IBS. P. Bauer. Invited Keynote Talk. 2014 Jun 05-06. Basel, Switzerland.
Adaptive designs for subgroup analysis optimizing utility functions. Workshop “Adaptive Designs and Multiple Testing Procedures” of the Joint Working Group (Section) of the ROeS and the German Region of the IBS. F. König. Invited Keynote Talk. 2014 Jun 05-06. Basel, Switzerland.
A Bayesian model for the selection of sample size in clinical trials. S. Jobjörnsson. Contributed Talk. 2014 June 5. Nordstat, Turkey.
Flexibility in confirmatory clinical trials – what is for free? International Clinical Trials’ Day. P. Bauer. Invited Talk. 2014 May 20. Luxembourg.
IDEAL Integrated Design and Analysis of small population group trials. R.-D. Hilgers, C.-F. Burman, F. Koenig. DIA ADSWG Meeting. 2014 May 20.
Slope – Sorted L-One Penalized Estimation. Bogdan, M., van den Berg, E., Su, W., Candes, E., Statistics Seminar of the Agrocampus Ouest, Rennes, France. 2014 March.
Model selection approach for genome wide association studies in admixed populations. Bogdan, M., Biostatistics Seminar of the University of Washington, USA. 2014 February.

Input to Regulatory Documents

Comments on ‘Draft Guideline on evaluation of anticancer medicinal products in man’ (EMA/CHMP/205/95 Rev.5) issued by Xavier Paoletti, Thomas Jaki, Ralf-Dieter Hilgers, and Franz König on behalf of IDEAS and IDeAl . [submitted 2016-11-15]
Discussion of “Framework of collaboration between the European Medicines Agency and academia”(EMA) participation of M. Rückbeil on behalf of IDeAl. [2016 June 15]
Small Population Clinical Trials: Challenges in the Field of Rare Diseases issued by A. H. Jonker, A. Mills,L.P.L. Lau, Y. Ando, P. Baroldi, F. Bretz, C.-F. Burman, O. Collignon, M. Hamdani, R. J. Hemmings, R.-D.Hilgers, I. Irony, M. Karlsson, J. Kirschner, J.P. Krischer, K. Larsson, K. Leeson-Beevers, G. Molenberghs, D. O’Connor, M. Posch, K. C. Roes, F. Schaefer, J. Scott, S. Senn, N. Stallard, A. Thompson, A. Torres, S, Zohar, S. Aymé, S. Day. [published 2013-07]
Comment on „Draft Guideline on the investigation of subgroups in confirmatory clinical trials (EMA/539146/2013)” issued by S. Senn, F. König, R.-D. Hilgers, and G. Molenberghs.[submitted 2014-07-25]
Comment on ‘Draft guideline on adjustment for baseline covariates’ (EMA/295050/2013) issued by R.-D. Hilgers. [submitted 2013-12-31]
Comment on ‘Draft qualification opinion of MCP-Mod as an efficient statistical methodology for model-based design and analysis of phase-II dose-finding studies under model uncertainty’ (EMA/CHMP/SAWP/592378/2013) issued by S. Senn. [submitted 2013-11-21]

Reports

Jonker AH, Mills A, Lau LPL, Ando Y, Baroldi P, Bretz F, Burman CF, Collignon O, Hamdani M, Hemmings RJ, Hilgers RD, Irony I, Karlsson M, Kirschner J, Krischer JP, Larsson K, Leeson-Beevers K, Molenberghs G, O’Connor D, Posch M, Roes KC, Schaefer F, Scott J, Senn SJ, Stallard N, Thompson A, Torres F, Zohar S, Aymé S, Day S Eds.: Small Population Clinical Trials: Challenges in the Field of Rare Diseases. July 2016.

Dissertations & Theses

Sebastian Jobjörnsson: On the Optimisation and Regulation of Clinical Trials PhD Thesis, Göteborgs Universitet, Sweden. 2018.
Diane Uschner: The assessment of bias in randomized controlled clinical trials. PhD Thesis, RWTH Aachen University, Germany, October 2018.
Sebastian Jobjörnsson: Optimization of Clinical Trials using Bayesian Decision Theory Licentiate Thesis, Göteborgs Universitet, Sweden. 2016.
Tanja Berger: Estimation Approaches for Distribution Parameters in the Case of Observations Under Multiple Lower Limits of Quantification. Master’s Thesis. RWTH Aachen University. March 2016.
Marcia Rückbeil: The impact of selection bias on test decisions in survival analysis. Master’s Thesis. RWTH Aachen University. September 2015.
Anne-Gaëlle Dosne: Improved Methods for Pharmacometric Model-Based Decision-Making in Clinical Drug Development PhD Thesis, Uppsala Universiteit, Sweden. December 2016.
David Schindler: Assessment of Randomization Procedures in the Presence of Selection and Chronological Bias PhD Thesis, TU Dortmund, Germany. August 2016.
Simon Langer: The modified distribution of the t-test statistic under the influence of selection bias based on random allocation rule. Master’s Thesis. RWTH Aachen University. September 2014.
Damian Brzyski: Selecting relevant groups of explanatory variables via convex optimization methods with the false discovery rate control.. April 2016.
Miriam Tamm: The impact of selection and chronological bias on clinical trials using permuted block randomization (Aachen, Techn. Hochsch., Diss., 2014)

Short Courses / Workshop organization

Introduction to N of 1 Trials . S. Senn. Invited Course. 24-02-2017, University of Sheffield, UK.
PSI Course: The Design and Analysis of N of 1 Trials . S. Senn. 05-06 July 2016. Hayes, UK.
Statistical Issues in Drug Development. S. Senn. 10-11 May 2016. Luxembourg.
Workshop on analysing and planning n-of-1 trials. S. Senn. 2016 May 5. Biogen.
The Challenge of “Small Data”. Rare Diseases and Ways to Study Them. S. Senn. 2016 May 5. Biogen.
Group Sequential and Adaptive Study Design. F. König. 29 June 2016. Summerschool organized by Universität Salzburg, International Biometric Society (German Region and Region Austria-Swiss), Austrian Statisical Society, Austria.
Population Optimum Design of Experiments (PODE). A. Hooker. 20 June 2016. at Uppsala University, Sweden.
Longitudinal and incomplete data. G. Molenberghs. 16-18. March 2016. at University Vienna, Austia.
Tutorial on Adaptive Designs for Confirmatory Clinical Trials. F. König. 8 March 2016. ENAR 2016 Spring Meeting. Austin, Texas, USA.
Joint Workshop on Small Population Clinical Trials Challenges in the Field of Rare Diseases organized by IRDiRC-EMA Small Population Clinical Trials Task Force. F König. 03. March 2016.
Pseudo-likelihood and Split-sample Methods in Small and Very Large Studies G. Molenberghs. 13. January 2016. at London School of Hygiene and Tropical Medicine, London, UK.
Frontiers of Confirmatory Inference in Small Populations. F König. 02. September 2015. 9th International Conference on Multiple Comparison Procedures in Hyderabad, India.
ISCB Workshop in Stratification and randomization in clinical trials. R.-D. Hilgers, D. Uschner and D. Schindler in collaboration with Armin Koch. 23 August 2015. Utrecht, Netherlands.
Regulartory Panel Discussion. F. König. 27 June 2015.ISBS-DIA Joint Symposium on Biopharmaceutical Statistics. Beijing, China.
Trends in adaptive design and statistical approaches to small population group trials in EU. F. König. 23 March 2015. PMDA – Pharmaceuticals and Medical Devices Agency. Tokyo.
ISCB Short Course in “Novel approaches to multiple test problems, with applications to adaptive designs and dose finding”. F. König. 27-28 November 2014. Budapest, Hungary.
Summer school “Adaptive Designs and Multiple Testing in Clinical Trials”. F. König, and M. Posch. Universtat Politécnica de Catalunya. 8-10 July, 2014. Bacelona, Spain.
Surrogate marker evaluation in clinical trials. W. van der Elst. Symposium on Small Populations. July 2014. Vienna. Austria
Randomisation and Stratification in clinical trials. L. Aguirre Dávila, E. Budde, S. Ernst, T. Framke, A. Gonnermann, R.-D. Hilgers, A. Koch, M. Kottas, D. Schindler, L. Spineli, D. Uschner, K. Weber. Symposium on Small Populations. 2014 July 3. Vienna. Austria
Adaptive Clinical Trial Designs. F. König, M. Posch, G. Wassmer. Symposium on Small Populations. 2014 July 3. Vienna. Austria
Supporting the pathways to trials for rare diseases: Clinical trial design and other considerations. Part 1: Clinical trial designs for small population group trials. R.-D. Hilgers. ECRD Invited Workshop. 2014 May 9. Berlin, Germany.

Conference Posters

Influence of clinical trial design to detect drug effect in systems with within subject variability. Karlsson, M. 2015 June 2. – 5. The Population Approach Group in Europe (PAGE) conference, Hersonissos, Crete, Greece.
Perl speaks NONMEM (PsN) and Xpose. Harling, K., Nordgren, R., Hooker, A.C., Karlsson, M. 2016 June 7. – 10. The Population Approach Group in Europe (PAGE) conference, Lisboa, Portugal.
Quantifying drug effects in phase 2a anti-diabetic studies : Power of four HbA1c models. Wellhagen, G.J., Karlsson, M.O., Kjellsson, M.C. 2015 June 2. – 5. The Population Approach Group in Europe (PAGE) conference, Hersonissos, Crete, Greece.
Parameter estimations for various distributed data with observations below a lower limit of quantification. T. Berger, R.-D. Hilgers, Conference Poster. ISCB 2016. 2016 August 21-25.
Determination of Appropriate Settings in the Assessment of Parameter Uncertainty Distributions using Sampling Importance Resampling (SIR). Conference Poster.
Marketing authorisation of orphan medicines in Europe 2000-2013: a 13-year experience. Conference Poster.
Fast Saddle-Point Algorithm for Generalized Dantzig Selector and FDR Control with the Ordered l1-Norm. S. Lee, D. Brzyski, M. Bogdan. Conference Poster.
Minimierungsmethode bei binärem Outcome und kleiner Fallzahl. C. Fitzner, L. A. Dávila, R.-D. Hilgers. Conference Poster. DAGStat 2016. March, 14-18. Göttingen, Germany.
Adaptive two-stage bioequivalence trials with early stopping and sample size re-estimation. F. König, M. Wolfsegger, T. Jaki, H. Schütz, G. Wassmer. Conference Poster. ISCB 2014. 2014 Aug, 24-27. Vienna, Austria.
Adaptive designs for confirmatory model based decisions using MCP-Mod. S. Krasnozhon, A. Graf, B. Bornkamp, F. Bretz, G. Wassmer, F. König. Conference Poster. ISCB 2014. 2014 Aug, 24-27. Vienna, Austria.
Comparison of different allocation procedures in clinical trials in small population groups with respect to accidental and selection bias. D. Schindler, D. Uschner, M. Tamm, N. Heussen, R.-D. Hilgers. Conference Poster. ISCB 2014. 2014 Aug, 24-27. Vienna, Austria.
Split-sample based and multiple imputation estimation & computation for data hierarchies (Meta-analysis, clinical trials, and beyond.). Mohlenberghs, G., Spring Meetings of the Eastern North American Region of the International Biometric Society, Miami, FL, USA. 2015 March 15.
Evidence, Eminence,and Extrapolation: Adapted Levels of Evidence for Small Populations. G. Hlavin. Conference Poster. ISCB 2014. 2014 Aug, 24-27. Vienna, Austria.
Accelerating Monte-Carlo power studies through parametric power estimation. Ueckert, S., Karlsson, M.O., Hooker, A.C., Population Approach Group in Europe (PAGE), Alicante, Spain. 10^th June 2014.
Integrated Design and Analysis of small population group trials. R.-D. Hilgers. Conference Poster. ECRD 2014. 2014 May 8. Berlin, Germany.

Comments

Comment on errorstatistics.com: Being a statistician means never having to say you are certain. by S. Senn. [posted on 2017-01-13]
Comment on errorstatistics.com: Automatic for the people? Not quite. by S. Senn. [posted on 2017-04-23]
Statsols Blog: Making it personal: N-of-1 trials, allowing for individuality but not overdoing it by S. Senn. [posted on 2017-01-11]
Comment on errorstatistics.com: Placebos: it’s not only the patients that are fooled by S. Senn. [posted on 2016-12-19]
Comment on errorstatistics.com: Painful dichotomies by S. Senn. [posted on 2015-08-02]
Comment on errorstatistics.com: Double Jeopardy?: Judge Jeffreys Upholds the Law (sequel to the pathetic P-value) by S. Senn. [posted on 2015-05-09]
Comment on errorstatistics.com: The pathetic p-value by S. Senn. [posted on 2015-03-16]
Comment on IMI 2 Strategic Research Agenda: Aiming to Make a Real Impact on Everyday Healthcare and Patients by S. Senn. [submitted 2014-07-09]
Comment on errorstatistics.com: Responder despondency: myths of personalized medicine by S. Senn. [posted on 2014-07-26]

Conferences

Wilson Aarhus Symposium 2019 [Date: 2019-05-09 to 2019-05-12]
WBS-Winterseminar on Innovative Statistical Approaches in Drug Development.
[Date: 2014-12-2]
The WBS-Winterseminar is also mentioned explicitly on page 7 of the Biometric Bulletin of the International Biometric Society IBS.
IDeAl Annual Meeting [Date: 2014-11-03 to 2014-11-04]
Symposium Vienna. [Date: 2014-07-01 to 2014-07-02]
Kick-Off Meeting [Date: 2013-11-06 to 2013-11]

Statistical Software Programs

R and SAS Code for Statistical Issues in the Design and Analysis of Series of N-of-1 Trials [published 2019-01-30]
R code for the Statistical Analyses of Series of N-of-1 Trials Using R and R-Programms.zip [published 2018-09]
SAS code for the Statistical Analyses of Series of N-of-1 Trials Using SAS and SAS-Programms.zip [published 2018-09]
R, GenStat and SAS Code for Sample Size Considerations in N-of-1 trials [published 2017-05-20]
R application for joint genotype and admixture mapping in admixed populations [published 2016-07-16]
R package for optimization of Bayesian Decision Problems [published 2016-03-30]
R package for genome-wide association studies with SLOPE [published 2015-09-16]
R package for testing similarity of dose response curves [published 2015-09-16]
R package on Randomization for clinical trials [published 2015-09-09]
R package “MIXFIM” for the evaluation and optimization of the Fisher Information Matrix in NonLinear Mixed Effect Models using Markov Chains Monte Carlo for both discrete and continuous data [published 2015-09-01]
R package for the estimation of within subject correlations based on linear mixed effects models [published 2015-04-29]
R package on the Prediction of Therapeutic Success [published 2015-04-27]
R package for dimensionality reduction via variables clustering [published 2014-12-06]
R-Code to calculate worst case type I error inflation in multiarmed clinical trials [published 2014-04-23]
R package on Surrogate Markers [published 2014-03-18]

Press Articles

Gerald Hlavin receives Arthur Linder Award [released 2015-08-07]
Pädiatrie: Die Kindermedizin führt zumeist ein Schattendasein [released 2015-06-06]
IDEAL mentioned in April newsletter of Japanes agency PMDA [released 2015-05-01]
FP7 Symposium on Small Populations [released 2014-02-28]
Project Start [released 2013-09-06]

Awards & Distinctions

- Nicole Heussen has been offered a W2 professorship at University of Rostock. November 2019, Rostock, Germany.
- Friedrich Wilhelm Prize is awarded by RWTH Aachen University to Diane Uschner. October 2019, Aachen, Germany.
- Doctoral Prize of the Medical Faculty donated by Grünenthal is awarded to Diane Uschner. May 2019, Aachen, Germany.
- Diane Uschner, Ralf-Dieter Hilgers and Nicole Heussen received the Best Poster Award for the poster entitled “The choice of a randomization procedure for randomized controlled clinical trials” at the ISCB 2017, Vigo, Spain.
- Damian Brzyski earns distinguished paper award for his work on SLOPE. 17th March 2017.
- Arthur Linder Award of the International Biometric Society is awarded to Gerald Hlavin. 7th August 2015, Vienna, Austria.
- Nicole Heussen has been offered a professorship in biostatistics at Sigmund Freud University Vienna. February 2015, Vienna, Austria.

Commemorative Publications

Burman, C.F. in Festschrift in Honor of Hans Nyquist on the Occasion of His 65th Birthday: From optimal design theory to optimizing designs of clinical trials. Department of Statistics, Stockholm University. 2015. Edited by Ellinor Frackle-Fornius. ISBN: 978-91-87355-19-6.

Webinars & Videos

Clinical trials: three statistical traps for the unwary by Stephen Senn.
Confirmatory Adaptive Designs.
Numbers needed to mislead, meta-analysis and muddled thinking by Stephen Senn.
Generate Randomization Sequences for a Clinical Trial with randomizeR R Package by Diane Uschner.
IDeAl Webinar Series – 01 – Introduction by Ralf-Dieter Hilgers [uploaded 07-Oct-2016]. Download slides.
IDeAl Webinar Series – 02 – Selection of a Randomisation Procedure by Ralf-Dieter Hilgers [uploaded 11-Oct-2016]. Download slides.
IDeAl Webinar Series – 03 – A sampling importance resampling procedure for estimating parameter uncertainty by Mats Karlsson [uploaded 14-Oct-2016]. Download slides.
IDeAl Webinar Series – 04 – Statistical inference for comparing small population groups by Holger Dette [uploaded 14-Oct-2016]. Download slides.
IDeAl Webinar Series – 05 -Pseudo-likelihood and Split-sample Methods in Small and Very Large Studies by Geert Molenberghs [uploaded 20-Oct-2016]. Download slides.
IDeAl Webinar Series – 06 – Adaptive Levels of Evidence by Franz König [uploaded 27-Oct-2016]. Download slides.
IDeAl Webinar Series – 07 – A little bit me, a little bit you: N of 1 trials, random effects and shrinkage estimators by Stephen Senn [uploaded 27-Oct-2016]. Download slides.
IDeAl Webinar Series – 08 – Using Hamiltonian Monte Carlo to design clinical trials with longitudinal data by France Mentré. Download slides.
IDeAl Webinar Series – 09 – Genetic factors influencing the response to the therapy by Malgorzata Bogdan. Download slides.
IDeAl Webinar Series – 10 – Optimal decisions and stakeholder interactions by Carl-Frederik Burman. Download slides.
IDeAl Webinar Series – 11 – Dissemination by Franz König and Ralf-Dieter Hilgers. Download slides.

Newsletter

All newsletters, issued throughout the project can be downloaded from the links below.

Issue 1 – September 2014

Issue 2 – December 2014

Issue 3 – August 2015

Issue 4 – December 2015

Issue 5 – June 2016

Issue 6 – October 2016

Issue 7 – April 2017

Related Research

The following papers are related to IDeAl, either because they complement the work of IDeAl in close coorporation with IDeAl researchers, or they cite research that was conducted in the context of the IDeAl project without directly being part of IDeAl.

Meta-research quoting IDeAl

Tudor Smith, Catrin, Williamson, Paula R., and Beresford, Michael W.: Methodology of clinical trials for rare diseases.Best Practice & Research Clinical Rheumatology, 2, 247-62, 2014. Gives a detailed overview about IDeAls aims and research topics, and states that the research conducted in this project contributes to improving the efficiency of rare disease trials.
O´Connor, Daniel J., and Hemming, Robert James: Coping with small populations of patients in clinical trials. Expert Opinion on Orphan Drugs, 2, 765-768, 2014. Quotes IDeAl as one of three research initiatives funded by the European Unions Seveth Framework Programme dedicated to the development of new methodology for small population group trials.

Sideline Research

Carl-Frederik Burman is part of the organizing committee of the EFSPI workshop on Biomarkers and Subgroups on 24th June, 2016, Leiden.
Carl-Frederik Burman is invited speaker for a session on rare diseases at ISCB in Birmingham from 21th till 25th August, 2016, Birmingham.
S. Jobjörnsson published the R Package bdpopot: Optimisation of Baysian Decision Problems (March 2016).
A.G. Dosne, Bergstrand M., and Karlsson M.O.: A strategy for residual error modeling incorporating scedasticity of variance and distribution shape. Journal of Pharmacokinetics and Pharmacodynamics. 43, 2, 137-151, 2016.
M. Bogdan and P. Szulc are working with D. Siegmund from Stanford University and R.W. Doerge from Purdue University on explaining/preventing false discoveries in gene mapping due to polygenic effects (April 2016).
J. Korell, S.W. Martin, M.O. Karlsson, and Ribbing, J.: A model-based longitudinal meta-analysis of FEV1 in randomized COPD trials. Clinical Pharamcology & Therapeutic. 99, 3, 315-324. 2016.
Y. Aoki, R. Nordgren, Hooker A.C.: Preconditioning of Nonlinear Mixed Effects Models for Stabilisation of Variance-Covariance Matrix Computations. AAPS Journal. 18, 2, 505-518. 2016.
Frommlet, F., Bogdan, M., and Ramsey D.: Phenotypes and Genotypes – search for influential genes. Springer. 2016.
Ralf-Dieter Hilgers, Nigel Stallard and Kit Roes submitted a poster to the ERCD 2016 in Edinburgh (January, 2016).
S. Choy, M.C. Kjellsson, M.O. Karlsson, de Winter W.: Weight-HbA1c-insulin-glucose model for describing disease progression of type 2 diabetes. CPT Pharmacometrics Syst Pharmacol. 5, 1, 11-19. 2016.
Ralf-Dieter Hilgers coauthored an editorial “Hedgehog blockade for basal cell carcinoma: does it come at a (secondary neoplastic) price?“, which shows the connection of small population trials to big data published in JAMA Dermatology.
- Rübben A., Hilgers, R.-D., and Leverkus, M., Hedgehog Blockade for Basal Cell Carcinoma: Coming at a (Secondary Neoplastic) Price. PMID: 26913776. 2016.
Holger Dette has worked with Kirsten Schroning, Wenge Kee Wong, Frank Bretz and Kathrin Kettelhake on: Optimal designs for active controlled dose finding trials with efficacy-toxicity outcomes (January, 2016)
Christina Fitzner started working on the topic “Evaluation of categorical data after covariate adaptive randomization for small samples” at Uniklinik Aachen (January, 2016).
Ralf-Dieter Hilgers, Nigel Stallard and Kit Roes are writing a position paper about “Directions for new developments on statistical design and analysis of small population group trials” to be submitted to Orphante (from December 2015 til March 2016).
Ralf-Dieter Hilgers, Franz König, Geert Molenberghs and Stephen Senn were invited to write an expert opinion paper entitled “Design and analysis of clinical trials for small rare disease populations“ to be submitted to Expert Opinion for Orphan Drugs (from December 2015 til March 2016).
Ralf-Dieter Hilgers coauthored a paper “Making sense of big data in health research – Towards an EU action plan in systems medicine and citizen science“, which shows the connection of small population trials to big data. The paper was submitted to Genome in Medicine in 2016.
Tanja Berger started working on the topic “Estimation approaches for distribution paramters in the case of observations under multiple lower limits of quantification” for small samples supervised by Ralf-Dieter Hilgers and Nicole Heussen (October, 2015).
Carl-Frederik Burman is invited speaker for a session on statistical methodology for clinical research in rare diseases at ISCB in Utrecht from 23th till 27th August, 2015, Utrecht.
A Bayesian model for the selection of sample size in clinical trials. S. Jobjörnsson. Contributed Talk. 2014 June 5. Nordstat, Turkey.
Nicole Heussen, William F. Rosenberger and Ralf-Dieter Hilgers are investigating “Randomization tests with respect to missing values”. Since January 2016, their work is supported by Kai Fuge (June, 2016).
Bram Burger investigates “Randomization tests with longitudinal (generalized) linear mixed models for small samples” supervised by Geert Molenberghs, Wim van der Elst, Bill Rosenberger, Ralf-Dieter Hilgers and Nicole Heussen (June, 2015).
Ann Christina Foldenauer investigates the “Evaluation of Stepped Wedge Design under consideration of carry-over effects for small sample clinical trials” supervised by Ralf-Dieter Hilgers and Nicole Heussen (April, 2014).
In 2014, the Drug Information Associations work stream on Adaptive Programmes, led by Carl-Fredrik Burman, decides to launch a working group on Small Populations. This working group is led by Prof. Bob Beckman, Georgetown University. The Small Population working group is collaborating with IDeAl WP 4 and 9 on designs when biomarker-defined subpopulations are present. The working group also conducts research on other topics around trial design for small population groups, without a formal link to IDeAl.