Use of alternative and confirmatory data in support of rare disease drug development.

Authors: Chow SC; Department of Biostatistics and Bioinformatics, Duke University School of Medicine, Durham, North Carolina, USA., Pariser A; International Rare Diseases Research Consortium, Columbus, Ohio, USA., Galson S; Boston Consulting Group, Boston, Massachusetts, USA.

Source: Journal of biopharmaceutical statistics [J Biopharm Stat] 2025 Oct; Vol. 35 (6), pp. 1005-1019. Date of Electronic Publication: 2025 Apr 11.

Publication Type: Journal Article; Review

Journal Info: Publisher: Taylor & Francis Country of Publication: England NLM ID: 9200436 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1520-5711 (Electronic) Linking ISSN: 10543406 NLM ISO Abbreviation: J Biopharm Stat Subsets: MEDLINE

MeSH Terms: Rare Diseases*/drug therapy , Drug Development*/statistics & numerical data , Drug Development*/methods , Orphan Drug Production*/statistics & numerical data , Orphan Drug Production*/methods , Research Design*/statistics & numerical data , Adaptive Clinical Trials as Topic*/statistics & numerical data , Adaptive Clinical Trials as Topic*/methods, Humans ; Data Interpretation, Statistical ; Clinical Trials as Topic/statistics & numerical data ; Clinical Trials as Topic/methods

A Preplanned Multi-Stage Platform Trial for Discovering Multiple Superior Treatments With Control of FWER and Power.

Authors: Greenstreet P; Department of Mathematics and Statistics, Lancaster University, Lancaster, UK.; Exeter Clinical Trials Unit, University of Exeter, Exeter, UK., Jaki T; MRC Biostatistics Unit, University of Cambridge, Cambridge, UK.; Faculty for Informatics and Data Science, University of Regensburg, Regensburg, Germany., Bedding A; Data and Statistical Sciences Department, Roche Products Ltd., Welwyn Garden City, UK., Mozgunov P; MRC Biostatistics Unit, University of Cambridge, Cambridge, UK.

Source: Biometrical journal. Biometrische Zeitschrift [Biom J] 2025 Feb; Vol. 67 (1), pp. e70025.

Publication Type: Journal Article

Journal Info: Publisher: Wiley-VCH Verlag GmbH & Co. KGaA Country of Publication: Germany NLM ID: 7708048 Publication Model: Print Cited Medium: Internet ISSN: 1521-4036 (Electronic) Linking ISSN: 03233847 NLM ISO Abbreviation: Biom J Subsets: MEDLINE

MeSH Terms: Biometry*/methods , Clinical Trials as Topic*, Humans ; Sample Size ; Adaptive Clinical Trials as Topic

Clinical trial design for novel targeted agents in neuro-oncology.

Authors: Lim-Fat MJ; Division of Neurology, Department of Medicine, Sunnybrook Health Sciences Centre, University of Toronto, Toronto, ON, Canada. Maryjane.limfat@sunnybrook.ca., Roberto K; Division of Neurology, Department of Medicine, Sunnybrook Health Sciences Centre, University of Toronto, Toronto, ON, Canada., Wen PY; Center For Neuro-Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA, USA.

Source: Journal of neuro-oncology [J Neurooncol] 2025 Jan; Vol. 171 (1), pp. 21-33. Date of Electronic Publication: 2024 Oct 08.

Publication Type: Journal Article; Review

Journal Info: Publisher: Springer Country of Publication: United States NLM ID: 8309335 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1573-7373 (Electronic) Linking ISSN: 0167594X NLM ISO Abbreviation: J Neurooncol Subsets: MEDLINE

MeSH Terms: Antineoplastic Agents*/pharmacology , Antineoplastic Agents*/therapeutic use , Brain Neoplasms*/drug therapy , Brain Neoplasms*/mortality , Brain Neoplasms*/pathology , Clinical Trials as Topic* , Research Design*, Humans ; Biomarkers, Tumor/analysis ; Biomarkers, Tumor/metabolism ; Molecular Targeted Therapy/methods ; Adaptive Clinical Trials as Topic

Adaptive designs in clinical trials: a systematic review-part I.

Authors: Ben-Eltriki M; Department of Pharmacology and Therapeutics, Max Rady College of Medicine, University of Manitoba, Winnipeg, MB, Canada. mbeneltriki@alumni.ubc.ca.; George and for Fay Yee Centre Healthcare Innovation, Winnipeg, MB, Canada. mbeneltriki@alumni.ubc.ca.; Cochrane Hypertension Review Group, Therapeutic Initiative, University of British Columbia, Vancouver, BC, Canada. mbeneltriki@alumni.ubc.ca., Rafiq A; Department of Pharmacology and Therapeutics, Max Rady College of Medicine, University of Manitoba, Winnipeg, MB, Canada., Paul A; Department of Pharmacology and Therapeutics, Max Rady College of Medicine, University of Manitoba, Winnipeg, MB, Canada., Prabhu D; George and for Fay Yee Centre Healthcare Innovation, Winnipeg, MB, Canada., Afolabi MOS; Department of Pediatrics and Child Health, University of Manitoba, Winnipeg, Manitoba, Canada., Baslhaw R; George and for Fay Yee Centre Healthcare Innovation, Winnipeg, MB, Canada.; Department of Community Health Sciences, University of Manitoba, Winnipeg, MB, Canada., Neilson CJ; Neil John Maclean Health Sciences Library, University of Manitoba, Winnipeg, MB, Canada., Driedger M; Department of Community Health Sciences, University of Manitoba, Winnipeg, MB, Canada., Mahmud SM; Department of Community Health Sciences, University of Manitoba, Winnipeg, MB, Canada., Lacaze-Masmonteil T; Department of Pediatrics, Cumming School of Medicine, University of Calgary, Alberta, Canada., Marlin S; Clinical Trials Ontario, Toronto, Ontario, Canada., Offringa M; Department of Paediatrics, Management & Evaluation, Institute of Health Policy, University of Toronto, Ontario, Canada.; The Hospital for Sick Children, Toronto, Ontario, Canada., Butcher N; The Hospital for Sick Children, Toronto, Ontario, Canada.; Department of Psychiatry, University of Toronto, Toronto, Ontario, Canada., Heath A; The Hospital for Sick Children, Toronto, Ontario, Canada.; Division of Biostatistics, Dalla Lana School of Public Health, Child Health Evaluative Sciences, University of Toronto, ScientistToronto, Ontario, Canada.; Department of Statistical Science, University College London, London, UK., Kelly LE; Department of Pharmacology and Therapeutics, Max Rady College of Medicine, University of Manitoba, Winnipeg, MB, Canada. lauren.kelly@umanitoba.ca.; George and for Fay Yee Centre Healthcare Innovation, Winnipeg, MB, Canada. lauren.kelly@umanitoba.ca.; Children's Hospital Research Institute of Manitoba, Winnipeg, MB, Canada. lauren.kelly@umanitoba.ca.; Departments of Pharmacology and Therapeutics, Community Health Sciences, University of Manitoba, 417-753 McDermot Ave, Winnipeg, Manitoba, R3E0T6, Canada. lauren.kelly@umanitoba.ca.

Source: BMC medical research methodology [BMC Med Res Methodol] 2024 Oct 04; Vol. 24 (1), pp. 229. Date of Electronic Publication: 2024 Oct 04.

Publication Type: Journal Article; Systematic Review

Journal Info: Publisher: BioMed Central Country of Publication: England NLM ID: 100968545 Publication Model: Electronic Cited Medium: Internet ISSN: 1471-2288 (Electronic) Linking ISSN: 14712288 NLM ISO Abbreviation: BMC Med Res Methodol Subsets: MEDLINE

MeSH Terms: Research Design* , Clinical Trials as Topic*/methods , Clinical Trials as Topic*/statistics & numerical data, Humans ; Child ; Adaptive Clinical Trials as Topic/methods ; Adaptive Clinical Trials as Topic/statistics & numerical data ; Adult

Modern trials are most useful when they are pragmatic and explanatory - there is no continuum.

Authors: Janiaud P; Pragmatic Evidence Lab, Research Center for Clinical Neuroimmunology and Neuroscience Basel (RC2NB), University Hospital Basel and University of Basel, Basel, Switzerland; Department of Clinical Research, University Hospital Basel and University of Basel, Basel, Switzerland., Hemkens LG; Pragmatic Evidence Lab, Research Center for Clinical Neuroimmunology and Neuroscience Basel (RC2NB), University Hospital Basel and University of Basel, Basel, Switzerland; Department of Clinical Research, University Hospital Basel and University of Basel, Basel, Switzerland; Meta-Research Innovation Center at Stanford (METRICS), Stanford University, Stanford, California, USA. Electronic address: lars.hemkens@usb.ch.

Source: Journal of clinical epidemiology [J Clin Epidemiol] 2024 Dec; Vol. 176, pp. 111566. Date of Electronic Publication: 2024 Oct 11.

Publication Type: Journal Article

Journal Info: Publisher: Elsevier Country of Publication: United States NLM ID: 8801383 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1878-5921 (Electronic) Linking ISSN: 08954356 NLM ISO Abbreviation: J Clin Epidemiol Subsets: MEDLINE

MeSH Terms: Pragmatic Clinical Trials as Topic*/methods , Research Design*, Humans ; Clinical Trials as Topic/methods

Innovative approaches for vaccine trials as a key component of pandemic preparedness - a white paper.

Authors: Bethe U; Institute of Translational Research, Cologne Excellence Cluster on Cellular Stress Responses in Aging-Associated Diseases (CECAD), Faculty of Medicine and University Hospital Cologne, University of Cologne, Herderstrasse 52, 50931, Cologne, Germany.; Center for Integrated Oncology Aachen Bonn Cologne Düsseldorf (CIO ABCD) and Excellence Center for Medical Mycology (ECMM), Department I of Internal Medicine, Faculty of Medicine, University Hospital Cologne, University of Cologne, Cologne, Germany.; Partner Site Bonn-Cologne, German Centre for Infection Research (DZIF), Cologne, Germany., Pana ZD; Medical School, European University of Cyprus, Nicosia, Cyprus., Drosten C; Institute of Virology, Charité Universitätsmedizin Berlin, Berlin, Germany., Goossens H; Laboratory of Medical Microbiology, Vaccine & Infectious Disease Institute and Biobank Antwerp, University of Antwerp, Wilrijk, Belgium., König F; Center for Medical Data Science, Institute of Medical Statistics, Medical University of Vienna, Vienna, Austria., Marchant A; European Plotkin Institute for Vaccinology, Université libre de Bruxelles, Brussels, Belgium., Molenberghs G; Interuniversity Institute for Biostatistics and Statistical Bioinformatics, Data Science Institute, KU Leuven and Hasselt University, Wilrijk, Belgium., Posch M; Center for Medical Data Science, Institute of Medical Statistics, Medical University of Vienna, Vienna, Austria., Van Damme P; Centre for the Evaluation of Vaccination, VACCINOPOLIS, Vaccine and Infectious Disease Institute, University of Antwerp, Wilrijk, Belgium., Cornely OA; Institute of Translational Research, Cologne Excellence Cluster on Cellular Stress Responses in Aging-Associated Diseases (CECAD), Faculty of Medicine and University Hospital Cologne, University of Cologne, Herderstrasse 52, 50931, Cologne, Germany. oliver.cornely@uk-koeln.de.; Center for Integrated Oncology Aachen Bonn Cologne Düsseldorf (CIO ABCD) and Excellence Center for Medical Mycology (ECMM), Department I of Internal Medicine, Faculty of Medicine, University Hospital Cologne, University of Cologne, Cologne, Germany. oliver.cornely@uk-koeln.de.; Partner Site Bonn-Cologne, German Centre for Infection Research (DZIF), Cologne, Germany. oliver.cornely@uk-koeln.de.

Source: Infection [Infection] 2024 Dec; Vol. 52 (6), pp. 2135-2144. Date of Electronic Publication: 2024 Jul 17.

Publication Type: Journal Article; Review

Journal Info: Publisher: Springer Heidelberg Country of Publication: Germany NLM ID: 0365307 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1439-0973 (Electronic) Linking ISSN: 03008126 NLM ISO Abbreviation: Infection Subsets: MEDLINE

MeSH Terms: Pandemics*/prevention & control , Clinical Trials as Topic*, Humans ; Vaccines ; Research Design ; COVID-19/prevention & control ; COVID-19/epidemiology ; Pandemic Preparedness ; Adaptive Clinical Trials as Topic

Effects of sceptical priors on the performance of adaptive clinical trials with binary outcomes.

Authors: Granholm A; Department of Intensive Care 4131, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark., Lange T; Section of Biostatistics, Department of Public Health, University of Copenhagen, Copenhagen, Denmark., Harhay MO; Clinical Trials Methods and Outcomes Lab, PAIR (Palliative and Advanced Illness Research) Center, Perelman School of Medicine, University of Pennsylvania, Philadelphia, USA.; Department of Biostatistics, Epidemiology, and Informatics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, USA., Perner A; Department of Intensive Care 4131, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark., Møller MH; Department of Intensive Care 4131, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark., Kaas-Hansen BS; Department of Intensive Care 4131, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark.; Section of Biostatistics, Department of Public Health, University of Copenhagen, Copenhagen, Denmark.

Source: Pharmaceutical statistics [Pharm Stat] 2024 Sep-Oct; Vol. 23 (5), pp. 728-741. Date of Electronic Publication: 2024 Mar 29.

Publication Type: Journal Article

Journal Info: Publisher: Wiley Country of Publication: England NLM ID: 101201192 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1539-1612 (Electronic) Linking ISSN: 15391604 NLM ISO Abbreviation: Pharm Stat Subsets: MEDLINE

MeSH Terms: Bayes Theorem* , Research Design* , Adaptive Clinical Trials as Topic*/statistics & numerical data , Adaptive Clinical Trials as Topic*/methods , Computer Simulation*, Humans ; Sample Size ; Data Interpretation, Statistical ; Probability ; Models, Statistical ; Clinical Trials as Topic/methods ; Clinical Trials as Topic/statistics & numerical data

Adaptive clinical trial designs for rapid vaccine development: Developing country vaccine manufacturers' perspective.

Authors: Mahajan M; Zydus Lifesciences, India. Electronic address: Manish.Mahajan@zyduslife.com., Martin LB; Former U.S. Pharmacopeial Convention, Current Affiliation: Independent Consultant, United States. Electronic address: laurabmartin@vacc4impact.science., Gaurav K; Panacea Biotec, India. Electronic address: kumargaurav@panaceabiotec.com., Suri RK; Developing Countries Vaccine Manufacturers' Network (DCVMN), Switzerland. Electronic address: r.suri@dcvmn.net.

Source: Vaccine [Vaccine] 2025 Dec 05; Vol. 68, pp. 127890. Date of Electronic Publication: 2025 Nov 01.

Publication Type: Journal Article; Review

Journal Info: Publisher: Elsevier Science Country of Publication: Netherlands NLM ID: 8406899 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1873-2518 (Electronic) Linking ISSN: 0264410X NLM ISO Abbreviation: Vaccine Subsets: MEDLINE

MeSH Terms: Vaccine Development*/methods , Drug Industry* , Vaccines* , Adaptive Clinical Trials as Topic*/methods , Research Design* , Clinical Trials as Topic*/methods, Humans ; Developing Countries

Advancing Decentralized and Pragmatic Clinical Trials as a Path Toward Improved Representativeness and Greater Generalizability of Clinical Trial Evidence.

Authors: Marra C; Verily Life Sciences LLC, Dallas, Texas. Electronic address: cmarra@verily.com., Chaiyachati KH; Ascension, St. Louis, Missouri., Washington V; Verily Life Sciences LLC, Dallas, Texas., Abernethy AP; Highlander Health, Dallas, Texas.

Source: Clinical therapeutics [Clin Ther] 2025 Nov; Vol. 47 (11), pp. 966-970. Date of Electronic Publication: 2025 Aug 21.

Publication Type: Journal Article

Journal Info: Publisher: Excerpta Medica Country of Publication: United States NLM ID: 7706726 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1879-114X (Electronic) Linking ISSN: 01492918 NLM ISO Abbreviation: Clin Ther Subsets: MEDLINE

MeSH Terms: Pragmatic Clinical Trials as Topic*/methods , Clinical Trials as Topic*/methods , Patient Selection*, Humans ; Research Design

Practical guidance for conducting high-quality and rapid interim analyses in adaptive clinical trials.

Authors: Mossop H; Population Health Sciences Institute, Newcastle University, Newcastle Upon Tyne, UK., Walmsley Z; Population Health Sciences Institute, Newcastle University, Newcastle Upon Tyne, UK.; Newcastle Clinical Trials Unit, Newcastle University, Newcastle Upon Tyne, UK., Wilson N; Population Health Sciences Institute, Newcastle University, Newcastle Upon Tyne, UK., Agbeleye O; NIHR Innovation Observatory, Newcastle University, Newcastle Upon Tyne, UK., Bardgett M; Newcastle Clinical Trials Unit, Newcastle University, Newcastle Upon Tyne, UK., Bevin-Nicholls A; Population Health Sciences Institute, Newcastle University, Newcastle Upon Tyne, UK., Breckons M; Population Health Sciences Institute, Newcastle University, Newcastle Upon Tyne, UK.; NIHR Innovation Observatory, Newcastle University, Newcastle Upon Tyne, UK., Cole M; Population Health Sciences Institute, Newcastle University, Newcastle Upon Tyne, UK., Craig D; Population Health Sciences Institute, Newcastle University, Newcastle Upon Tyne, UK.; NIHR Innovation Observatory, Newcastle University, Newcastle Upon Tyne, UK., Dimairo M; Clinical Trials Research Unit, Sheffield Centre for Health and Related Research (SCHARR), University of Sheffield, Sheffield, UK., Hancock H; Population Health Sciences Institute, Newcastle University, Newcastle Upon Tyne, UK.; Newcastle Clinical Trials Unit, Newcastle University, Newcastle Upon Tyne, UK., Law M; Medical Research Council Biostatistics Unit, University of Cambridge, Cambridge, UK.; Royal Papworth Hospital, Cambridge, UK., Lopes A; CRUK Cancer Trials Centre, University College London, London, UK., Noor NM; Department of Medicine, University of Cambridge, Cambridge, UK., Oparah C; NIHR Innovation Observatory, Newcastle University, Newcastle Upon Tyne, UK., Pallmann P; Centre for Trials Research, Cardiff University, Cardiff, UK., Phillipson J; Newcastle Clinical Trials Unit, Newcastle University, Newcastle Upon Tyne, UK., Robertson DS; Medical Research Council Biostatistics Unit, University of Cambridge, Cambridge, UK., Teare MD; Population Health Sciences Institute, Newcastle University, Newcastle Upon Tyne, UK.; Newcastle Clinical Trials Unit, Newcastle University, Newcastle Upon Tyne, UK., Thomson KH; NIHR Innovation Observatory, Newcastle University, Newcastle Upon Tyne, UK., Yap C; Clinical Trials and Statistics Unit, The Institute of Cancer Research, London, UK., Wason JMS; Population Health Sciences Institute, Newcastle University, Newcastle Upon Tyne, UK. james.wason@newcastle.ac.uk.

Source: BMC medicine [BMC Med] 2025 Oct 01; Vol. 23 (1), pp. 528. Date of Electronic Publication: 2025 Oct 01.

Publication Type: Journal Article; Review

Journal Info: Publisher: BioMed Central Country of Publication: England NLM ID: 101190723 Publication Model: Electronic Cited Medium: Internet ISSN: 1741-7015 (Electronic) Linking ISSN: 17417015 NLM ISO Abbreviation: BMC Med Subsets: MEDLINE

MeSH Terms: Research Design*/standards , Adaptive Clinical Trials as Topic*/methods , Adaptive Clinical Trials as Topic*/standards , Clinical Trials as Topic*/methods , Clinical Trials as Topic*/standards, Humans

An Approach to Design Adaptive Clinical Trials With Time-to-Event Outcomes Based on a General Bayesian Posterior Distribution.

Authors: McGree JM; School of Mathematical Sciences, Queensland University of Technology, Brisbane, Australia., Overstall AM; School of Mathematical Sciences, University of Southampton, Southampton, UK., Jones M; Sydney School of Public Health, University of Sydney, Sydney, Australia., Mahar RK; Melbourne School of Population and Global Health, University of Melbourne, Melbourne, Australia.; Clinical Epidemiology and Biostatistics Unit, Murdoch Children's Research Institute, Parkville, Australia.

Source: Statistics in medicine [Stat Med] 2025 Oct; Vol. 44 (23-24), pp. e70207.

Publication Type: Journal Article

Journal Info: Publisher: Wiley Country of Publication: England NLM ID: 8215016 Publication Model: Print Cited Medium: Internet ISSN: 1097-0258 (Electronic) Linking ISSN: 02776715 NLM ISO Abbreviation: Stat Med Subsets: MEDLINE

MeSH Terms: Adaptive Clinical Trials as Topic*/methods , Research Design* , Clinical Trials as Topic*/methods, Bayes Theorem ; Humans ; Computer Simulation ; Models, Statistical ; Gastroenteritis/prevention & control ; Likelihood Functions ; Treatment Outcome ; Infant

An improved biomarker-guided adaptive patient enrichment design for oncology trials.

Authors: Zhou Z; Amgen, Global Biostatistical Science, Thousand Oaks, CA, USA., Teng Z; Servier Bio-Innovation, Global Biometrics, Boston, MA, USA., Zhu J; Servier Bio-Innovation, Global Biometrics, Boston, MA, USA., Tang RS; Astellas Pharma, Quantitative Sciences and Evidence Generation, Boston, MA, USA.

Source: Journal of biopharmaceutical statistics [J Biopharm Stat] 2025 Oct; Vol. 35 (6), pp. 1227-1243. Date of Electronic Publication: 2025 Apr 23.

Publication Type: Journal Article

Journal Info: Publisher: Taylor & Francis Country of Publication: England NLM ID: 9200436 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1520-5711 (Electronic) Linking ISSN: 10543406 NLM ISO Abbreviation: J Biopharm Stat Subsets: MEDLINE

MeSH Terms: Research Design*/statistics & numerical data , Neoplasms*/drug therapy , Neoplasms*/therapy , Neoplasms*/metabolism , Biomarkers, Tumor* , Clinical Trials as Topic*/methods , Clinical Trials as Topic*/statistics & numerical data , Medical Oncology*/statistics & numerical data , Medical Oncology*/methods , Adaptive Clinical Trials as Topic*/statistics & numerical data , Adaptive Clinical Trials as Topic*/methods, Humans ; Computer Simulation ; Sample Size ; Models, Statistical ; Biomarkers

Sample size recalculation in three-stage clinical trials and its evaluation.

Authors: Bokelmann B; Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Institute of Biometry and Clinical Epidemiology, Charitéplatz 1, Berlin, 10117, Germany. bjoern.bokelmann@gmx.de., Rauch G; Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Institute of Biometry and Clinical Epidemiology, Charitéplatz 1, Berlin, 10117, Germany.; Technische Universität Berlin, Straße des 17. Juni 135, Berlin, 10623, Germany., Meis J; Institute of Medical Biometry, University Medical Center Ruprechts-Karls University Heidelberg, Im Neuenheimer Feld 130.3, Heidelberg, 69120, Germany., Kieser M; Institute of Medical Biometry, University Medical Center Ruprechts-Karls University Heidelberg, Im Neuenheimer Feld 130.3, Heidelberg, 69120, Germany., Herrmann C; Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Institute of Biometry and Clinical Epidemiology, Charitéplatz 1, Berlin, 10117, Germany.

Source: BMC medical research methodology [BMC Med Res Methodol] 2024 Sep 25; Vol. 24 (1), pp. 214. Date of Electronic Publication: 2024 Sep 25.

Publication Type: Journal Article

Journal Info: Publisher: BioMed Central Country of Publication: England NLM ID: 100968545 Publication Model: Electronic Cited Medium: Internet ISSN: 1471-2288 (Electronic) Linking ISSN: 14712288 NLM ISO Abbreviation: BMC Med Res Methodol Subsets: MEDLINE

MeSH Terms: Clinical Trials as Topic*/methods , Clinical Trials as Topic*/statistics & numerical data , Research Design*/statistics & numerical data, Sample Size ; Humans ; Computer Simulation ; Adaptive Clinical Trials as Topic

Similarities and Differences Between Pragmatic Trials and Hybrid Effectiveness-Implementation Trials.

Authors: Fortney JC; Department of Psychiatry and Behavioral Sciences, School of Medicine, University of Washington, Seattle, WA, USA. fortneyj@uw.edu.; Department of Veterans Affairs, Health Systems Research, Center of Innovation for Veteran-Centered and Value-Driven Care, Seattle, WA, USA. fortneyj@uw.edu., Curran GM; College of Pharmacy, University of Arkansas for Medical Sciences, Little Rock, AR, USA.; Department of Nursing and Midwifery, University of Limerick, Limerick, Ireland., Lyon AR; Department of Psychiatry and Behavioral Sciences, School of Medicine, University of Washington, Seattle, WA, USA., Check DK; Department of Population Health Sciences, School of Medicine, Duke University, Durham, NC, USA., Flum DR; Department of Surgery, School of Medicine, University of Washington, Seattle, WA, USA.

Source: Journal of general internal medicine [J Gen Intern Med] 2024 Jul; Vol. 39 (9), pp. 1735-1743. Date of Electronic Publication: 2024 Apr 16.

Publication Type: Journal Article

Journal Info: Publisher: Springer Country of Publication: United States NLM ID: 8605834 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-1497 (Electronic) Linking ISSN: 08848734 NLM ISO Abbreviation: J Gen Intern Med Subsets: MEDLINE

MeSH Terms: Pragmatic Clinical Trials as Topic*/methods, Humans ; Clinical Trials as Topic/methods ; Clinical Trials as Topic/standards ; Evidence-Based Medicine/methods ; Evidence-Based Medicine/standards ; Research Design

Regulatory Issues of Platform Trials: Learnings from EU-PEARL.

Authors: Nguyen QL; Section Data Science and Methods, Paul-Ehrlich-Institut, Langen, Germany.; Department of Medical Informatics, Biometry and Epidemiology, Friedrich-Alexander-Universität Erlangen-Nürnberg, Erlangen, Germany., Hees K; Section Data Science and Methods, Paul-Ehrlich-Institut, Langen, Germany., Hernandez Penna S; Novartis Pharma AG, Basel, Switzerland., König F; Institute for Medical Statistics, Center for Medical Data Science, Medical University of Vienna, Vienna, Austria., Posch M; Institute for Medical Statistics, Center for Medical Data Science, Medical University of Vienna, Vienna, Austria., Bofill Roig M; Institute for Medical Statistics, Center for Medical Data Science, Medical University of Vienna, Vienna, Austria., Meyer EL; Institute for Medical Statistics, Center for Medical Data Science, Medical University of Vienna, Vienna, Austria.; Berry Consultants, Vienna, Austria., Freitag MM; Charité - Universitätsmedizin Berlin, Berlin, Germany., Parke T; Berry Consultants, Abingdon, UK., Otte M; Euroscan, Cologne, Germany., Dauben HP; Euroscan, Cologne, Germany., Mielke T; Statistics and Decision Sciences, Janssen-Cilag GmbH, Neuss, Germany., Spiertz C; Janssen Biologics, Leiden, The Netherlands., Mesenbrink P; Analytics, Development, Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA., Gidh-Jain M; Sanofi, Gentilly, France., Pierre S; Sanofi, Gentilly, France., Morello S; Janssen R&D, Springhouse, PA, USA., Hofner B; Section Data Science and Methods, Paul-Ehrlich-Institut, Langen, Germany.; Department of Medical Informatics, Biometry and Epidemiology, Friedrich-Alexander-Universität Erlangen-Nürnberg, Erlangen, Germany.

Source: Clinical pharmacology and therapeutics [Clin Pharmacol Ther] 2024 Jul; Vol. 116 (1), pp. 52-63. Date of Electronic Publication: 2024 Mar 26.

Publication Type: Journal Article; Review; Research Support, Non-U.S. Gov't

Journal Info: Publisher: Wiley Country of Publication: United States NLM ID: 0372741 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1532-6535 (Electronic) Linking ISSN: 00099236 NLM ISO Abbreviation: Clin Pharmacol Ther Subsets: MEDLINE

MeSH Terms: European Union* , Clinical Trials as Topic*/legislation & jurisprudence , Clinical Trials as Topic*/methods , Clinical Trials as Topic*/standards , United States Food and Drug Administration* , Drug Development*/legislation & jurisprudence , Drug Development*/methods, Humans ; United States ; Research Design ; Guidelines as Topic ; Technology Assessment, Biomedical/legislation & jurisprudence ; Adaptive Clinical Trials as Topic

Graph Based, Adaptive, Multiarm, Multiple Endpoint, Two-Stage Designs.

Authors: Mehta C; Cytel Corporation, Cambridge, MA, USA.; Harvard T.H. Chan School of Public Health, Boston, MA, USA., Mukhopadhyay A; Cytel Corporation, Cambridge, MA, USA., Posch M; Medical University of Vienna, Austria.

Source: Statistics in medicine [Stat Med] 2025 Dec; Vol. 44 (28-30), pp. e70237.

Publication Type: Journal Article

Journal Info: Publisher: Wiley Country of Publication: England NLM ID: 8215016 Publication Model: Print Cited Medium: Internet ISSN: 1097-0258 (Electronic) Linking ISSN: 02776715 NLM ISO Abbreviation: Stat Med Subsets: MEDLINE

MeSH Terms: Research Design* , Endpoint Determination*/methods , Endpoint Determination*/statistics & numerical data , Clinical Trials as Topic*/methods , Clinical Trials as Topic*/statistics & numerical data, Humans ; Computer Simulation ; Sample Size ; Models, Statistical ; Data Interpretation, Statistical

Clinical trials for older cancer patients: A systematic review.

Authors: Fazio R; Department of Gastrointestinal Oncology, Hôpital Universitaire de Bruxelles (HUB), Institut Jules Bordet - Erasme Hospital, Université Libre de Bruxelles (ULB), Brussels, Belgium., Audisio A; Department of Gastrointestinal Oncology, Hôpital Universitaire de Bruxelles (HUB), Institut Jules Bordet - Erasme Hospital, Université Libre de Bruxelles (ULB), Brussels, Belgium., Bregni G; Department of Gastrointestinal Oncology, Hôpital Universitaire de Bruxelles (HUB), Institut Jules Bordet - Erasme Hospital, Université Libre de Bruxelles (ULB), Brussels, Belgium., Sur D; Department of Medical Oncology, University of Medicine and Pharmacy 'Iuliu Haţieganu', Cluj-Napoca, Romania., Daprà V; Department of Gastrointestinal Oncology, Hôpital Universitaire de Bruxelles (HUB), Institut Jules Bordet - Erasme Hospital, Université Libre de Bruxelles (ULB), Brussels, Belgium., Conti C; Department of Gastrointestinal Oncology, Hôpital Universitaire de Bruxelles (HUB), Institut Jules Bordet - Erasme Hospital, Université Libre de Bruxelles (ULB), Brussels, Belgium., Abbassi FZ; Department of Gastrointestinal Oncology, Hôpital Universitaire de Bruxelles (HUB), Institut Jules Bordet - Erasme Hospital, Université Libre de Bruxelles (ULB), Brussels, Belgium., Benhima N; Department of Gastrointestinal Oncology, Hôpital Universitaire de Bruxelles (HUB), Institut Jules Bordet - Erasme Hospital, Université Libre de Bruxelles (ULB), Brussels, Belgium., Assaf I; Department of Gastrointestinal Oncology, Hôpital Universitaire de Bruxelles (HUB), Institut Jules Bordet - Erasme Hospital, Université Libre de Bruxelles (ULB), Brussels, Belgium., Lago LD; Department of Medical Oncology, Chirec Cancer Institute, Brussels, Belgium., Papamichael D; Department of Medical Oncology, Bank of Cyprus Oncology Centre, Nicosia, Cyprus., Laethem JV; Department of Gastrointestinal Oncology, Hôpital Universitaire de Bruxelles (HUB), Institut Jules Bordet - Erasme Hospital, Université Libre de Bruxelles (ULB), Brussels, Belgium., Saad ED; International Drug Development Institute, Louvain-la-Neuve, Belgium., Sclafani F; Department of Gastrointestinal Oncology, Hôpital Universitaire de Bruxelles (HUB), Institut Jules Bordet - Erasme Hospital, Université Libre de Bruxelles (ULB), Brussels, Belgium. Electronic address: francesco.sclafani@hubruxelles.be.

Source: Cancer treatment reviews [Cancer Treat Rev] 2025 Dec; Vol. 141, pp. 103045. Date of Electronic Publication: 2025 Nov 04.

Publication Type: Journal Article; Systematic Review; Review

Journal Info: Publisher: Elsevier Country of Publication: Netherlands NLM ID: 7502030 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1532-1967 (Electronic) Linking ISSN: 03057372 NLM ISO Abbreviation: Cancer Treat Rev Subsets: MEDLINE

MeSH Terms: Neoplasms*/therapy , Clinical Trials as Topic*/statistics & numerical data , Clinical Trials as Topic*/methods , Patient Selection*, Humans ; Aged ; Quality of Life ; Aged, 80 and over ; Geriatric Assessment ; Age Factors

Statistical innovations in clinical trial design with a focus on drug combinations, factorials, and other multiple therapy issues.

Authors: Berry DA; Division of Discovery Science, Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, TX, USA. Electronic address: don@berryconsultants.net.

Source: The journal of prevention of Alzheimer's disease [J Prev Alzheimers Dis] 2025 Dec; Vol. 12 (10), pp. 100392. Date of Electronic Publication: 2025 Oct 27.

Publication Type: Journal Article

Journal Info: Publisher: Elsevier Country of Publication: United States NLM ID: 101638820 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2426-0266 (Electronic) Linking ISSN: 22745807 NLM ISO Abbreviation: J Prev Alzheimers Dis Subsets: MEDLINE

MeSH Terms: Research Design* , Clinical Trials as Topic*/methods , Clinical Trials as Topic*/statistics & numerical data, Humans ; Bayes Theorem ; Drug Combinations ; Drug Therapy, Combination

Medication adherence during the run-in phase of clinical trials: A systematic review of methodological and reporting rigour.

Authors: Davies N; Centre for Health Economics and Medicines Evaluation, North Wales Medical School, Bangor University, Bangor, UK., Vrijens B; AARDEX Group, Liège, Belgium.; Department of Public Health, Liège University, Liège, Belgium., Wright DFB; Sydney Pharmacy School, Faculty of Medicine and Health, University of Sydney, Sydney, Australia.; Department of Clinical Pharmacology & Toxicology, St. Vincent's Hospital, Sydney, Australia., Hughes DA; Centre for Health Economics and Medicines Evaluation, North Wales Medical School, Bangor University, Bangor, UK.

Source: British journal of clinical pharmacology [Br J Clin Pharmacol] 2025 Dec; Vol. 91 (12), pp. 3353-3365. Date of Electronic Publication: 2025 Jul 26.

Publication Type: Journal Article; Systematic Review

Journal Info: Publisher: Wiley-Blackwell Country of Publication: England NLM ID: 7503323 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1365-2125 (Electronic) Linking ISSN: 03065251 NLM ISO Abbreviation: Br J Clin Pharmacol Subsets: MEDLINE

MeSH Terms: Research Design*/standards , Clinical Trials as Topic*/methods , Clinical Trials as Topic*/standards , Medication Adherence*, Humans ; Bias

The reporting of health systems data use in primary results publications of clinical trials: a systematic review.

Authors: Thompson J; MRC Clinical Trials Unit at UCL (MRC CTU), Institute of Clinical Trials and Methodology, UCL, London, UK., Bobou M; MRC Clinical Trials Unit at UCL (MRC CTU), Institute of Clinical Trials and Methodology, UCL, London, UK.; Health Data Research UK (HDR UK), London, UK., Roberts K; MRC Clinical Trials Unit at UCL (MRC CTU), Institute of Clinical Trials and Methodology, UCL, London, UK., Taheri S; MRC Clinical Trials Unit at UCL (MRC CTU), Institute of Clinical Trials and Methodology, UCL, London, UK., Love SB; MRC Clinical Trials Unit at UCL (MRC CTU), Institute of Clinical Trials and Methodology, UCL, London, UK. s.love@ucl.ac.uk.; Health Data Research UK (HDR UK), London, UK. s.love@ucl.ac.uk., Murray ML; MRC Clinical Trials Unit at UCL (MRC CTU), Institute of Clinical Trials and Methodology, UCL, London, UK.; Health Data Research UK (HDR UK), London, UK.

Source: Trials [Trials] 2025 Nov 26; Vol. 26 (1), pp. 549. Date of Electronic Publication: 2025 Nov 26.

Publication Type: Journal Article; Systematic Review; Review

Journal Info: Publisher: BioMed Central Country of Publication: England NLM ID: 101263253 Publication Model: Electronic Cited Medium: Internet ISSN: 1745-6215 (Electronic) Linking ISSN: 17456215 NLM ISO Abbreviation: Trials Subsets: MEDLINE

MeSH Terms: Clinical Trials as Topic*/standards , Clinical Trials as Topic*/statistics & numerical data , Clinical Trials as Topic*/methods , Research Design*/standards, Humans