@misc{kelter_two_stage_2025,
  author       = {Kelter, Riko and Pawel, Samuel},
  title        = {The Bayesian optimal two-stage design for clinical phase II trials based on Bayes factors},
  year         = {2025},
  eprint       = {2511.23144},
  archivePrefix = {arXiv},
  primaryClass = {stat.ME},
  url          = {https://arxiv.org/abs/2511.23144}
}

@misc{kelter_power_2026,
  author       = {Kelter, Riko},
  title        = {Power and Sample Size Calculations for Bayes Factors in two-arm clinical Phase II Trials with binary Endpoints},
  year         = {2026},
  eprint       = {2603.01715},
  archivePrefix = {arXiv},
  primaryClass = {stat.ME},
  url          = {https://arxiv.org/abs/2603.01715}
}

@misc{kelter_third_2025,
  author       = {Kelter, Riko},
  title        = {Bayesian Power and Sample Size Calculations for Bayes Factors in the Binomial Setting},
  year         = {2025},
  eprint       = {2502.02914},
  archivePrefix = {arXiv},
  primaryClass = {stat.ME},
  url          = {https://arxiv.org/abs/2502.02914}
}

@article{khannaRiociguatPatientsEarly2020,
  title = {Riociguat in Patients with Early Diffuse Cutaneous Systemic Sclerosis ({{RISE-SSc}}): Randomised, Double-Blind, Placebo-Controlled Multicentre Trial},
  shorttitle = {Riociguat in Patients with Early Diffuse Cutaneous Systemic Sclerosis ({{RISE-SSc}})},
  author = {Khanna, Dinesh and Allanore, Yannick and Denton, Christopher P. and Kuwana, Masataka and {Matucci-Cerinic}, Marco and Pope, Janet E. and Atsumi, Tatsuya and Be{\v c}v{\'a}{\v r}, Radim and Czirj{\'a}k, L{\'a}szl{\'o} and Hachulla, Eric and Ishii, Tomonori and Ishikawa, Osamu and Johnson, Sindhu R. and Langhe, Ellen De and Stagnaro, Chiara and Riccieri, Valeria and Schiopu, Elena and Silver, Richard M. and Smith, Vanessa and Steen, Virginia and Stevens, Wendy and Sz{\"u}cs, Gabriella and Truchetet, Marie-Elise and Wosnitza, Melanie and Laapas, Kaisa and Pena, Janethe de Oliveira and Yao, Zhen and Kramer, Frank and Distler, Oliver},
  year = 2020,
  month = may,
  journal = {Annals of the Rheumatic Diseases},
  volume = {79},
  number = {5},
  pages = {618--625},
  publisher = {BMJ Publishing Group Ltd},
  issn = {0003-4967, 1468-2060},
  doi = {10.1136/annrheumdis-2019-216823},
  urldate = {2025-12-11},
  abstract = {Objectives Riociguat is approved for pulmonary arterial hypertension and has antiproliferative, anti-inflammatory and antifibrotic effects in animal models of tissue fibrosis. We evaluated the efficacy and safety of riociguat in patients with early diffuse cutaneous systemic sclerosis (dcSSc) at high risk of skin fibrosis progression. Methods In this randomised, double-blind, placebo-controlled, phase IIb trial, adults with dcSSc of {$<$}18 months' duration and a modified Rodnan skin score (mRSS) 10--22 units received riociguat 0.5 mg to 2.5 mg orally three times daily (n=60) or placebo (n=61). The primary endpoint was change in mRSS from baseline to week 52. Results At week 52, change from baseline in mRSS units was --2.09\textpm 5.66 (n=57) with riociguat and --0.77\textpm 8.24 (n=52) with placebo (difference of least squares means --2.34 (95\% CI --4.99 to 0.30; p=0.08)). In patients with interstitial lung disease, forced vital capacity declined by 2.7\% with riociguat and 7.6\% with placebo. At week 14, average Raynaud's condition score had improved {$\geq$}50\% in 19 (41.3\%)/46 patients with riociguat and 13 (26.0\%)/50 patients with placebo. Safety assessments showed no new signals with riociguat and no treatment-related deaths. Conclusions Riociguat did not significantly benefit mRSS versus placebo at the predefined p{$<$}0.05. Secondary and exploratory analyses showed potential efficacy signals that should be tested in further trials. Riociguat was well tolerated.},
  chapter = {Systemic sclerosis},
  copyright = {\copyright{} Author(s) (or their employer(s)) 2020. Re-use permitted under CC BY. Published by BMJ.. https://creativecommons.org/licenses/by/4.0/This is an open access article distributed in accordance with the Creative Commons Attribution 4.0 Unported (CC BY 4.0) license, which permits others to copy, redistribute, remix, transform and build upon this work for any purpose, provided the original work is properly cited, a link to the licence is given, and indication of whether changes were made. See:~https://creativecommons.org/licenses/by/4.0/.},
  langid = {english},
  pmid = {32299845},
  keywords = {disease activity,systemic sclerosis,treatment},
  file = {/Users/riko/Zotero/storage/RCI3UR8U/Khanna et al. - 2020 - Riociguat in patients with early diffuse cutaneous systemic sclerosis (RISE-SSc) randomised, double.pdf}
}

@misc{FDA_ComplexInnovativeDesignsDecember2020,
  title = {Interacting with the {{FDA}} on {{Complex Innovative Trial Designs}} for {{Drugs}} and {{Biological Products}} - {{Guidance}} for {{Industry}}},
  author = {{U.S. Department of Health and Human Services} and {Food and Drug Administration} and {Center for Biologics Evaluation and Research} and {Center for Drug Evaluation and Research}},
  year = 2020,
  month = dec,
  file = {/Users/riko/Zotero/storage/6SL5X2MQ/U.S. Department of Health and Human Services et al. - 2020 - Interacting with the FDA on Complex Innovative Trial Designs for Drugs and Biological Products - Gui.pdf}
}

@techreport{FDA_UseOfBayesianMethodologyJanuary2026,
  title = {Use of {{Bayesian Methodology}} in {{Clinical Trials}} of {{Drug}} and {{Biological Products}} - {{Guidance}} for {{Industry}}},
  author = {{U.S. Department of Health and Human Services Food and Drug Administration, Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation and Research (CBER)}},
  year = 2026,
  month = jan,
  file = {/Users/riko/Zotero/storage/YMKD6LNZ/_.pdf}
}

@misc{europeanmedicinesagencyICHE20Adaptive2025,
  title = {{{ICH E20}} Adaptive Designs for Clinical Trials - {{Scientific}} Guideline \textbar{} {{European Medicines Agency}} ({{EMA}})},
  author = {{European Medicines Agency}},
  year = 2025,
  month = jun,
  publisher = {European Medicines Agency},
  urldate = {2025-12-23},
  langid = {english},
  file = {/Users/riko/Zotero/storage/YHPGG8Q8/ich-e20-adaptive-designs-clinical-trials-scientific-guideline.html}
}



