Publications

 

New and improved methods for individual trial design (WP2 )

• Jansen-van der Weijde M, Gaasterland C, Roes K, Pontes C, Vives R, Sancho A, Nikolakopoulos S, Vermeulen E, van der Lee J.
  Rare disease registries: potential applications towards impact on development of new drug treatments
  Orphanet Journal of Rare Diseases 2018

• Ristl R, Urach S, Rosenkranz G, Posch M. 
  Methods for the analysis of multiple endpoints in small populations: A review
  Journal of Biopharmaceutical Statistics 2018

• Urach S, Gaasterland C, Posch M, Jilma B, Roes K, Rosenkranz G, van der Lee J. Ristl R.​​​
  Statistical analysis of Goal Attainment Scaling endpoints in randomised trials
  Statistical Methods in Medical Research 2018

• Ristl R, Xi D, Glimm E, Posch M.
  Optimal exact tests for multiple binary endpoints
  Computational Statistics & Data Analysis 2018

• Brakenhoff TB, Roes K, Nikolakopoulos S.    
  Bayesian sample size re-estimation using power priors 
  Statistical Methods in Medical Research 2018

• Nikolakopoulos S, van der Tweel I, Roes K. 
  Dynamic borrowing through empirical power priors that control type I error
  Biometrics 2017

• Lasch F, Weber K, Chao M, Koch A. 
  A plea to provide best evidence in trials under sample size restrictions: the example of pioglitazone to resolve leukoplakia and erythroplakia in Fanconi anemia patients
  Orphanet Journal of Rare Diseases 2017

• Spineli LM, Jenz E, Großhennig A, Koch A.
  Critical appraisal of arguments for the delayed start design proposed as alternative to the parallel-group randomized clinical trial design in the field of rare disease
  Orphanet Journal or Rare Diseases 2017

• Eichler HG, Bloechl-Daum B, Bauer P, Bretz F, Brown J, Hampson LV, Honig P, Krams M, Leufkens H, Lim R, Lumpkin MM, Murphy MJ, Pignatti F, Posch M, Schneeweiss S, Trusheim M, Koenig F.
  'Threshold crossing': A useful way to establish the counterfactual in clinical trials?
  Clinical Pharmacology and Therapeutics 2016

• Hilgers RD, Roes K, Stallard N. 
  Directions for New Developments on statistical design and analysis of small population group trials
  Orphanet Journal of Rare Diseases 2016

• Nikolakopoulos S, Roes K, Van der Tweel I.
  Sequential designs with small samples: Evaluation and recommendations for normal responses
  Statistical Methods in Medical Research 2016

• Derhaschnig U, Gilbert J, Jäger U, Böhmig G, Stingl G, Jilma B.
  Combined integrated protocol/basket trial design for a first-in-human trial 
  Orphanet Journal of Rare Diseases 2016

• Magirr D, Jaki T, Koenig F, Posch M.
  Sample Size Reassessment and Hypothesis Testing in Adaptive Survival Trials .
  PloS One 2016

• Urach S, Posch M. 
  Multi-arm group sequential designs with a simultaneous stopping rule 
  Statistics in Medicine 2016

• Ristl R, Frommlet F, Koch A, Posch M.
  Fallback tests for co-primary endpoints 
  Statistics in Medicine 2016

• Hlavin G, Koenig F, Male C, Posch M, Bauer P.
  Evidence, eminence and extrapolation
  Statistics in Medicine 2016

 

New methods for prospective design and analysis of series of studies (WP3)

 

• Pateras K, Nikolakopoulos S, Roes K. 
  Combined assessment of early and late‐phase outcomes in orphan drug development
  Statistics in Medicine 2021

• Pateras K, Nikolakopoulos S, Roes K. 
  Prior distributions for variance parameters in a sparse‐event meta‐analysis of a few small trials
  Pharmaceutical Statistics 2020

• Weber K, Lasch F, Koch A. 
  New research strategy with ambiguous implications: A comment on "Planning future studies based on the conditional power of a meta-analysis"
  Statistics in Medicine 2018

• Weber K, Hemmings R, Koch A. 
  How to use prior knowledge and still give new data a chance?
  Pharmaceutical Statistics 2018

• Pateras K, Nikolakopoulos S, Roes K.   
  Interval estimation of the overall treatment effect in a meta-analysis of a few small studies with zero events
  Contemporary Clinical Trials Communications 2018

• Pateras K, Nikolakopoulos S, Roes K.  
  Data-generating models of dichotomous outcomes: Heterogeneity in simulation studies for a random-effects meta-analysis
  Statistics in Medicine 2017

• van der Tweel I, Pateras K, van Baal C, Roes K.
  A review of frequent methods for combining results or series of trials  - to appear

• Gonnermann A, Framke T, Großhennig A, Koch A.
  No solution yet for combining two independent studies in the presence of heterogeneity
  Statistics in Medicine 2015                 

• Gonnermann A, Kottas M, Koch A.
  Biometric Entscheidungsunterstützung in Zulassung und Nutzenbewertung am Beispiel der Implikationen von heterogenen Ergebnissen in Untergruppen der Studienpopulation
  Bundesgesundheitsblatt - Gesundheitsforschung - Gesundheitsschutz 2015

 

Improved use of patient level information and perspectives (WP4 )

• Gaasterland C, Jansen-van der Weijde M, du Prie-Olthof MJ, Donk M, Kaatee MM, Raczmarek R, Lavery C, Leeson-Beevers K, O'Neill N, Timmis O, van Nederveen V, Vroom E, van der Lee J.
  The patient's view on rare disease trial design - a qualitative study
  Orphanet Journal of Rare Diseases 2019

• Rosenkranz G.
  Can we identify patients at high risk of harm under a generally safe intervention? 
  International Journal of Clinical Biostatistics and Biometrics 2017

• Hasford J, Koch A.
  Ethische Aspekte der klinischen Prüfung bei seltenen Erkrankungen  (in German).
  Bundesgesundheitsbl 2017

• Gaasterland C, Jansen-van der Weijde M., Weinreich S, van der Lee J.
  A systematic review to investigate the measurement properties of goal attainment scaling, towards use in drug trials
  BMC Medical Research Methodology 2016

• Koenig F, Slattery J, Groves T, Lang T, Benjamini Y, Day S, Bauer P, Posch M.
  Sharing clinical trial data on patient level: opportunities and challenges
  Biometrical Journal 2015

 

Validation of new methods within clinical as well as regulatory settings (WP5 )

• Pontes C, Fontanet JM, Vives R, Sancho A, Gomez-Valent M, Rios J, Morros R, Martinalbo J, Posch M, Koch A, Roes K, Oude Rengerink K, Torrent-Farnell J, Torres F.
  Evidence supporting regulatory-decision making on orphan medicinal products authorisation in Europe: methodological uncertainties 
  Orphanet Journal of Rare Diseases 2018

• Mitroiu M, Oude Rengerink K, Pontes C, Sancho A, Vives R, Pesiou S, Fontanet JM, Torres F, Nikolakopoulos S, Pateras K, Rosenkranz G, Posch M, Urach S, Ristl R, Koch A, Spineli L, van der Lee J, Roes K. 
  Applicability and added value of novel methods to improve drug development in rare diseases
  Orphanet Journal of Rare Diseases 2018

• Roes K.
  ASTERIX - Advances in Small Trials Design for Regulatory Innovation and Excellence - FP7
  Impact 2017

• Roes K.
  Editorial on special topic ISCB 2015: "Clinical research design, rare diseases, and personalized medicine" Biometrical Journal 2017

• Hofer MP. Hedman H, Mavris M, Koenig F, Vetter T, Posch M, Vamvakas S, Regnstrom J, Aarum S. 
  Marketing Authorization of orphan medicines in Europe from 2000 to 2013
  Drug Discovery Today 2017

• 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, Thomson A, Torres F, Zohar S, Ayme S, Day S, Editors.
  Small Population Clinical Trials: Challenges in the Field of Rare Diseases
  IRDiRC Workshop Report and Recommendations July 2016

• Roes K.
  A framework: make it useful to improve clinical trial design in smaller populations
  BMC Medicine 2016

• Koch A, Ziert Y. 
  Does it help that efficiency has been proven once we start discussing (added) benefit? 
  Biometrical Journal 2015

• Pontes C
  Heterogeneity, the problem with the averages and the new regulatory challenge: "whom to treat"
  Clinical Therapeutics 2015

• Pontes C, Fontanet JM, Gomez-Valent M, Rios Guillermo J, Vives Vilagut R, Morros R, Martinalbo J, Torrent-Farnell J, Torres F
  Milestones On Orphan Medicinal Products Development: The 100 First Drugs For Rare Diseases Approved Throughout Europe
  Clinical Therapeutics 2015

• Pontes C, Fontanet JM, Gomez-Valent M, Rios Guillermo J, Vives Vilagut R, Morros R, Martinalbo J, Torrent-Farnell J, Torres F.
  Clustering of rare medical conditions based on clinical features to their study
  Clinical Therapeutics 2015

• Pontes C, Fontanet M, Gómez-Valent M, Ríos J, Vives R, Morros R, Torrent-Farnell J, Torres F 
  Systematic approach to the description of the evidence supporting EMA regulatory opinions on orphan medicinal products
  Basic & Clinical Pharmacology and Toxicology 2014