ArticleCPT: pharmacometrics & systems pharmacology2013
Comparisons of Analysis Methods for Proof-of-Concept Trials.
Article in CPT: pharmacometrics & systems pharmacology, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 30 papers, 1 of them a synthesis that pooled it.
What it found
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
30 citing papers in PubMed, 1 synthesis or guideline pooled it.
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- Detecting placebo and drug effects on Parkinson's disease symptoms by longitudinal item-score models.CPT: pharmacometrics & systems pharmacology · 2021Trial
- Drug Effect of Clofazimine on Persisters Explains an Unexpected Increase in Bacterial Load in Patients.Antimicrobial agents and chemotherapy · 2020Trial
- Model-Informed Approaches for Pediatric Type 2 Diabetes Evidence Generation: Insights from a Model-Based Longitudinal HbA1c Meta-Analysis from EMA Marketing Authorization Applications.Clinical pharmacology and therapeutics · 2026Article
- Comparing randomized trial designs to estimate treatment effect in rare diseases with longitudinal models: a simulation study showcased by Autosomal Recessive Cerebellar Ataxias using the SARA score.BMC medical research methodology · 2025Article
- Development and comparison of model-integrated evidence approaches for bioequivalence studies with pharmacokinetic end points.CPT: pharmacometrics & systems pharmacology · 2024Article
- Statistical recommendations for count, binary, and ordinal data in rare disease cross-over trials.Orphanet journal of rare diseases · 2023Article
- Comparison of Seven Non-Linear Mixed Effect Model-Based Approaches to Test for Treatment Effect.Pharmaceutics · 2023Article
- Pharmacometrics meets statistics-A synergy for modern drug development.CPT: pharmacometrics & systems pharmacology · 2021Article
- Pharmacometrics-Based Considerations for the Design of a Pharmacogenomic Clinical Trial Assessing Irinotecan Safety.Pharmaceutical research · 2021Article
- Sweet/Fat Preference Taste in Subjects Who are Lean, Obese and Very Obese.Pharmaceutical research · 2020Article
- Item Response Theory Modeling of the International Prostate Symptom Score in Patients with Lower Urinary Tract Symptoms Associated with Benign Prostatic Hyperplasia.The AAPS journal · 2020Article
- Evaluation of non-linear-mixed-effect modeling to reduce the sample sizes of pediatric trials in type 2 diabetes mellitus.Journal of pharmacokinetics and pharmacodynamics · 2020Article
- Dose Finding in the Clinical Development of 60 US Food and Drug Administration-Approved Drugs Compared With Learning vs. Confirming Recommendations.Clinical and translational science · 2019Article
- Drugs' development in acute heart failure: what went wrong?Heart failure reviews · 2018Review
- Study Design Selection in Early Clinical Anti-Hyperglycemic Drug Development: A Simulation Study of Glucose Tolerance Tests.CPT: pharmacometrics & systems pharmacology · 2018Article
- Lessons learned from IDeAl - 33 recommendations from the IDeAl-net about design and analysis of small population clinical trials.Orphanet journal of rare diseases · 2018Review
- Comparison of Model Averaging and Model Selection in Dose Finding Trials Analyzed by Nonlinear Mixed Effect Models.The AAPS journal · 2018Article
Corrections and comments
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Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Drug development struggles with high costs and time consuming processes. Hence, a need for new strategies has been accentuated by many stakeholders in drug development. This study proposes the use of pharmacometric models to rationalize drug development. Two simulated examples, within the therapeutic areas of acute stroke and type 2 diabetes, are utilized to compare a pharmacometric model-based analysis to a t-test with respect to study power of proof-of-concept (POC) trials. In all investigated examples and scenarios, the conventional statistical analysis resulted in several fold larger study sizes to achieve 80% power. For a scenario with a parallel design of one placebo group and one active dose arm, the difference between the conventional and pharmacometric approach was 4.3- and 8.4-fold, for the stroke and diabetes example, respectively. Although the model-based power depend on the model assumptions, in these scenarios, the pharmacometric model-based approach was demonstrated to permit drastic streamlining of POC trials.CPT: Pharmacometrics & Systems Pharmacology (2013) 2, e23; doi:10.1038/psp.2012.24; advance online publication 16 January 2013.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.