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These approaches are compared head\u2010to\u2010head for clinical trial situations in rare diseases. Specifically, we consider 3 case studies of rare diseases (Lyell disease, adult\u2010onset Still disease, and cystic fibrosis) with the aim to plan the sample size for an upcoming clinical trial. We outline in detail the reasonable choice of parameters for these approaches for each of the 3 case studies and calculate sample sizes. We stress that the influence of the input parameters needs to be investigated in all approaches and recommend investigating different sample size approaches before deciding finally on the trial size. Highly influencing for the sample size are choice of treatment effect parameter in all approaches and the parameter for the additional cost of the new treatment in the decision\u2010theoretic approach. These should therefore be discussed extensively.<\/jats:p>","DOI":"10.1002\/pst.1848","type":"journal-article","created":{"date-parts":[[2018,1,11]],"date-time":"2018-01-11T04:31:11Z","timestamp":1515645071000},"page":"214-230","source":"Crossref","is-referenced-by-count":21,"title":["Approaches to sample size calculation for clinical trials in rare diseases"],"prefix":"10.1002","volume":"17","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4161-7851","authenticated-orcid":false,"given":"Frank","family":"Miller","sequence":"first","affiliation":[{"name":"Department of Statistics Stockholm University  Stockholm Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sarah","family":"Zohar","sequence":"additional","affiliation":[{"name":"INSERM, U1138, Team 22, Centre de Recherche des Cordeliers Universit\u00e9 Paris 5, Universit\u00e9 Paris 6  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nigel","family":"Stallard","sequence":"additional","affiliation":[{"name":"Statistics and Epidemiology, Division of Health Sciences, Warwick Medical School University of Warwick  Coventry UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jason","family":"Madan","sequence":"additional","affiliation":[{"name":"Clinical Trials Unit, Warwick Medical School University of Warwick  Coventry UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Martin","family":"Posch","sequence":"additional","affiliation":[{"name":"Section for Medical Statistics, Center for Medical Statistics, Informatics, and Intelligent Systems Medical University of Vienna  Vienna Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0415-263X","authenticated-orcid":false,"given":"Siew Wan","family":"Hee","sequence":"additional","affiliation":[{"name":"Statistics and Epidemiology, Division of Health Sciences, Warwick Medical School University of Warwick  Coventry UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Pearce","sequence":"additional","affiliation":[{"name":"Complexity Science University of Warwick  Coventry UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M\u00e5rten","family":"V\u00e5ger\u00f6","sequence":"additional","affiliation":[{"name":"Swedish Orphan Biovitrum  Stockholm Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Simon","family":"Day","sequence":"additional","affiliation":[{"name":"Clinical Trials Consulting and Training Limited  Buckingham UK"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2018,1,10]]},"reference":[{"key":"e_1_2_7_2_1","doi-asserted-by":"publisher","DOI":"10.1198\/000313001317098149"},{"key":"e_1_2_7_3_1","doi-asserted-by":"publisher","DOI":"10.1002\/sim.1783"},{"key":"e_1_2_7_4_1","doi-asserted-by":"publisher","DOI":"10.1002\/sim.5381"},{"key":"e_1_2_7_5_1","doi-asserted-by":"publisher","DOI":"10.1002\/pst.175"},{"key":"e_1_2_7_6_1","doi-asserted-by":"publisher","DOI":"10.1002\/pst.232"},{"key":"e_1_2_7_7_1","doi-asserted-by":"publisher","DOI":"10.1002\/sim.3551"},{"key":"e_1_2_7_8_1","doi-asserted-by":"publisher","DOI":"10.1002\/0470033312"},{"key":"e_1_2_7_9_1","doi-asserted-by":"publisher","DOI":"10.1002\/pst.450"},{"key":"e_1_2_7_10_1","doi-asserted-by":"publisher","DOI":"10.1002\/pst.1552"},{"key":"e_1_2_7_11_1","doi-asserted-by":"publisher","DOI":"10.1177\/1740774512453784"},{"key":"e_1_2_7_12_1","doi-asserted-by":"publisher","DOI":"10.1177\/0962280215588245"},{"key":"e_1_2_7_13_1","doi-asserted-by":"publisher","DOI":"10.1093\/biomet\/90.4.923"},{"key":"e_1_2_7_14_1","doi-asserted-by":"publisher","DOI":"10.1002\/bimj.201500228"},{"key":"e_1_2_7_15_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.burns.2009.06.213"},{"key":"e_1_2_7_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.dsi.2013.09.008"},{"key":"e_1_2_7_17_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.autrev.2014.01.058"},{"key":"e_1_2_7_18_1","first-page":"2345","article-title":"Interleukin I inhibition with anakinra in adult\u2010onset Still disease: a meta\u2010analysis of its efficacy and safety","volume":"8","author":"Hong D","year":"2014","journal-title":"Drug Des Devel Ther"},{"key":"e_1_2_7_19_1","doi-asserted-by":"publisher","DOI":"10.3899\/jrheum.111549"},{"key":"e_1_2_7_20_1","unstructured":"Cystic Fibrosis Foundation. 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