{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,1]],"date-time":"2026-08-01T03:53:48Z","timestamp":1785556428476,"version":"3.56.0"},"reference-count":24,"publisher":"Wiley","issue":"11","license":[{"start":{"date-parts":[[2024,10,3]],"date-time":"2024-10-03T00:00:00Z","timestamp":1727913600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["ascpt.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["CPT Pharmacom &amp;amp; Syst Pharma"],"published-print":{"date-parts":[[2025,11]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>Quantitative model\u2010based clinical trial simulation tools play a critical role in informing study designs through simulation before actual execution. These tools help drug developers explore various trial scenarios in silico to select a clinical trial design to detect therapeutic effects more efficiently, therefore reducing time, expense, and participants' burden. To increase the usability of the tools, user\u2010friendly and interactive platforms should be developed to navigate various simulation scenarios. However, developing such tools challenges researchers, requiring expertise in modeling and interface development. This tutorial aims to address this gap by guiding developers in creating tailored R Shiny apps, using an example of a model\u2010based clinical trial simulation tool that we developed for Duchenne muscular dystrophy (DMD). In this tutorial, the structural framework, essential controllers, and visualization techniques for analysis are described, along with key code examples such as criteria selection and power calculation. A virtual population was created using a machine learning algorithm to enlarge the available sample size to simulate clinical trial scenarios in the presented tool. In addition, external validation of the simulated outputs was conducted using a placebo arm of a recently published DMD trial. This tutorial will be particularly useful for developing clinical trial simulation tools based on DMD progression models for other end points and biomarkers. The presented strategies can also be applied to other diseases.<\/jats:p>","DOI":"10.1002\/psp4.13246","type":"journal-article","created":{"date-parts":[[2024,10,3]],"date-time":"2024-10-03T06:24:02Z","timestamp":1727936642000},"page":"1765-1774","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":5,"title":["A model\u2010informed clinical trial simulation tool with a graphical user interface for Duchenne muscular dystrophy"],"prefix":"10.1002","volume":"14","author":[{"ORCID":"https:\/\/orcid.org\/0009-0006-0459-8574","authenticated-orcid":false,"given":"Jongjin","family":"Kim","sequence":"first","affiliation":[{"name":"Department of Pharmaceutics, Center for Pharmacometrics and Systems Pharmacology, College of Pharmacy University of Florida  Orlando Florida USA"},{"name":"Department of Statistics and Data Science University of Central Florida  Orlando Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1588-9167","authenticated-orcid":false,"given":"Juan Francisco","family":"Morales","sequence":"additional","affiliation":[{"name":"Department of Pharmaceutics, Center for Pharmacometrics and Systems Pharmacology, College of Pharmacy University of Florida  Orlando Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6222-7479","authenticated-orcid":false,"given":"Sanghoon","family":"Kang","sequence":"additional","affiliation":[{"name":"Department of Pharmaceutics, Center for Pharmacometrics and Systems Pharmacology, College of Pharmacy University of Florida  Orlando Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0005-1706-6289","authenticated-orcid":false,"given":"Marian","family":"Klose","sequence":"additional","affiliation":[{"name":"Department of Pharmaceutics, Center for Pharmacometrics and Systems Pharmacology, College of Pharmacy University of Florida  Orlando Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3383-0759","authenticated-orcid":false,"given":"Rebecca J.","family":"Willcocks","sequence":"additional","affiliation":[{"name":"Department of Physical Therapy University of Florida  Gainesville Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9856-9486","authenticated-orcid":false,"given":"Michael J.","family":"Daniels","sequence":"additional","affiliation":[{"name":"Department of Statistics University of Florida  Gainesville Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3380-4972","authenticated-orcid":false,"given":"Ramona","family":"Belfiore\u2010Oshan","sequence":"additional","affiliation":[{"name":"Critical Path Institute  Tucson Arizona USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7346-2244","authenticated-orcid":false,"given":"Glenn A.","family":"Walter","sequence":"additional","affiliation":[{"name":"Department of Physiology and Aging University of Florida  Gainesville Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0617-1540","authenticated-orcid":false,"given":"William D.","family":"Rooney","sequence":"additional","affiliation":[{"name":"Advanced Imaging Research Center Oregon Health &amp; Science University  Portland Oregon USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0007-8183-1381","authenticated-orcid":false,"given":"Krista","family":"Vandenborne","sequence":"additional","affiliation":[{"name":"Department of Physical Therapy University of Florida  Gainesville Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9179-0735","authenticated-orcid":false,"given":"Sarah","family":"Kim","sequence":"additional","affiliation":[{"name":"Department of Pharmaceutics, Center for Pharmacometrics and Systems Pharmacology, College of Pharmacy University of Florida  Orlando Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,10,3]]},"reference":[{"key":"e_1_2_11_2_1","unstructured":"U.S. Food and Drug Administration.Pediatric rare diseases \u2013 a collaborative approach for drug development using gaucher disease as a model; draft guidance for industry. 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