Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches01:23

Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches

340
Biopharmaceutical studies constitute a vital field aiming to enhance drug delivery methods and refine therapeutic approaches, drawing upon diverse interdisciplinary knowledge. In research methodologies, the choice between controlled and non-controlled studies significantly influences the study's reliability and accuracy.
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast,...
340
Bioequivalence Experimental Study Designs: Repeated Measures, Cross-Over, Carry-Over, and Latin Square Designs01:15

Bioequivalence Experimental Study Designs: Repeated Measures, Cross-Over, Carry-Over, and Latin Square Designs

119
Body:Bioequivalence experimental study designs play a pivotal role in testing the effectiveness of various treatments. Key among these are the repeated measures, cross-over, carry-over, and Latin square designs. In the repeated measures design, each subject receives all treatments, allowing for temporal comparisons. This type of design is useful in reducing variability but requires careful planning to avoid bias.The cross-over design, an economical method, involves sequential administration of...
119
Crossover Experiments01:16

Crossover Experiments

4.4K
Crossover experiments, also called the repeated-measurements design, is a study design in which all experimental units are exposed to all treatments in different periods. Crossover experiments are generally used in psychology, the pharmaceutical industry, agriculture, and medicine.
Crossover designs are performed even with smaller sample sizes since the samples can act as their controls. These are better than simple randomized trials since patients are exposed to all the treatments.
4.4K
Bioequivalence Experimental Study Designs: Completely Randomized and Randomized Block Designs01:20

Bioequivalence Experimental Study Designs: Completely Randomized and Randomized Block Designs

161
Body:Bioequivalence experimental study designs are crucial methodologies used in evaluating and comparing the bioavailability of different drug products. These designs are categorized into various types: completely randomized, randomized block, repeated measures, cross and carry-over, and Latin square designs.Completely randomized designs involve randomly allocating treatments to all subjects participating in the experiment. This allocation is achieved by assigning unique random numbers to...
161
Study Designs in Epidemiology01:20

Study Designs in Epidemiology

801
Epidemiological study designs are fundamental tools for investigating the distribution, determinants, and control of health conditions in populations. They help researchers understand the relationships between exposures and outcomes, and they broadly fall into two categories: "observational" and "experimental" studies.
Observational studies are those where the researcher does not intervene but rather observes natural variations. They include cross-sectional, cohort, and...
801
Blind Procedures02:07

Blind Procedures

12.7K
Ideally, the people who observe and record the children’s behavior are unaware of who was assigned to the experimental or control group, in order to control for experimenter bias. Experimenter bias refers to the possibility that a researcher’s expectations might skew the results of the study. Remember, conducting an experiment requires a lot of planning, and the people involved in the research project have a vested interest in supporting their hypotheses. If the observers knew which...
12.7K

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

Evaluating the Environmental Impact of Clinical Research: A Full Life Cycle Analysis of a French Academic Randomised Clinical Trial.

Fundamental & clinical pharmacology·2026
Same author

Accelerating recruitment in clinical trials: A strategic challenge.

Therapie·2026
Same author

Therapie·2026
Same author

Hypnosis for unplanned procedural pain in the intensive care unit: the HYPIC randomized clinical trial.

Critical care (London, England)·2025
Same author

Maintaining ventilation with very low tidal volume and positive-end expiratory pressure versus no ventilation during cardiopulmonary bypass for cardiac surgery in adults: a randomized clinical trial.

Intensive care medicine·2025
Same author

Trends of Publication of Negative Trials Over Time.

Clinical pharmacology and therapeutics·2025
Same journal

Pharmacology of pruritus (pathophysiology, therapeutic targets).

Therapie·2026
Same journal

Barriers to prescribing methylphenidate for adults with ADHD: A qualitative study.

Therapie·2026
Same journal

[Self-medication and its factors amongst Navymen aboard surface ships of the French Navy at sea: A descriptive study].

Therapie·2026
Same journal

Subacute cutaneous lupus erythematosus induced by ticagrelor: Recurrence after rechallenge.

Therapie·2026
Same journal

Multiple HIV virological failures related to bictegravir-valproic acid interaction evidenced by therapeutic drug monitoring: A retrospective study.

Therapie·2026
Same journal

Indication-related misuse of oral fluoroquinolones in adults by general practitioners in France.

Therapie·2026
See all related articles

Related Experiment Video

Updated: Dec 28, 2025

Author Spotlight: Exploring the Impact of Reduced Resistance Exercise Volume on Metabolic Health
06:13

Author Spotlight: Exploring the Impact of Reduced Resistance Exercise Volume on Metabolic Health

Published on: December 1, 2023

1.5K

From single-arm studies to externally controlled studies. Methodological considerations and guidelines.

Michel Cucherat1, Silvy Laporte2, Olivier Delaitre3

  • 1Service de pharmacotoxicologie, hospices civils de Lyon, 16, avenue Lacassagne, 69003 Lyon, France; Laboratoire de biométrie et biologie évolutive, CNRS, UMR5558, université Lyon 1, 69008 Lyon, France.

Therapie
|February 18, 2020
PubMed
Summary
This summary is machine-generated.

Single-arm studies have methodological limitations for evaluating new treatments. Externally controlled trials are preferred over single-arm trials to mitigate bias and improve reliability in treatment assessment.

Keywords:
Control groupExternal control studyIndirect comparisonMatching-adjusted indirect comparisonsNon-comparativeSingle-arm study

More Related Videos

A Within-Subject Experimental Design using an Object Location Task in Rats
09:28

A Within-Subject Experimental Design using an Object Location Task in Rats

Published on: May 6, 2021

5.1K
Transauricular Vagus Nerve Stimulation and Electroencephalographic Assessment in Disorders of Consciousness
04:04

Transauricular Vagus Nerve Stimulation and Electroencephalographic Assessment in Disorders of Consciousness

Published on: July 11, 2025

1.1K

Related Experiment Videos

Last Updated: Dec 28, 2025

Author Spotlight: Exploring the Impact of Reduced Resistance Exercise Volume on Metabolic Health
06:13

Author Spotlight: Exploring the Impact of Reduced Resistance Exercise Volume on Metabolic Health

Published on: December 1, 2023

1.5K
A Within-Subject Experimental Design using an Object Location Task in Rats
09:28

A Within-Subject Experimental Design using an Object Location Task in Rats

Published on: May 6, 2021

5.1K
Transauricular Vagus Nerve Stimulation and Electroencephalographic Assessment in Disorders of Consciousness
04:04

Transauricular Vagus Nerve Stimulation and Electroencephalographic Assessment in Disorders of Consciousness

Published on: July 11, 2025

1.1K

Area of Science:

  • Clinical Trials Methodology
  • Evidence-Based Medicine
  • Pharmaceutical Research

Background:

  • Single-arm studies are often used as pivotal trials but possess inherent methodological limitations.
  • Randomized controlled studies are the gold standard for evaluating new treatments due to higher reliability.
  • Single-arm studies cannot reliably demonstrate treatment benefit without a comparative baseline.

Purpose of the Study:

  • To discuss the limitations of single-arm studies in clinical research.
  • To analyze solutions and propose guidelines for the conduct and assessment of single-arm studies.
  • To emphasize the importance of comparative data in treatment evaluation.

Main Methods:

  • Discussion and analysis of methodological limitations in single-arm studies.
  • Exploration of solutions including externally controlled trials and various comparison methods.
  • Review of guidelines for the conduct and assessment of clinical studies.

Main Results:

  • Single-arm studies are intrinsically inappropriate for demonstrating treatment benefit due to the inability to infer benefit without a counterfactual.
  • Comparison with external data is necessary but faces limitations like post hoc bias, confounding bias, and measurement/attrition biases.
  • Externally controlled trials, with a priori defined controls and comparison methods, are recommended over single-arm trials.

Conclusions:

  • Externally controlled trials should be prioritized over single-arm trials, aligning with ICH E10 guidelines.
  • Formalization of external controls in study protocols, including a priori selection of controls and comparison methods (e.g., MAIC), is crucial.
  • Externally controlled studies are acceptable only when randomization is infeasible and must demonstrate freedom from residual confounding bias, particularly for dramatic effects in predictable diseases.