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Related Concept Videos

Randomized Experiments01:13

Randomized Experiments

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The randomization process involves assigning study participants randomly to experimental or control groups based on their probability of being equally assigned. Randomization is meant to eliminate selection bias and balance known and unknown confounding factors so that the control group is similar to the treatment group as much as possible. A computer program and a random number generator can be used to assign participants to groups in a way that minimizes bias.
Simple randomization
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Group Design02:01

Group Design

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The most basic experimental design involves two groups: the experimental group and the control group. The two groups are designed to be the same except for one difference— experimental manipulation. The experimental group gets the experimental manipulation—that is, the treatment or variable being tested—and the control group does not. Since experimental manipulation is the only difference between the experimental and control groups, we can be sure that any differences between...
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Sampling Plans01:23

Sampling Plans

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Sampling is a crucial step in analytical chemistry, allowing researchers to collect representative data from a large population. Common sampling methods include random, judgmental, systematic, stratified, and cluster sampling.
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Sample Size Calculation01:19

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Knowledge of the sample size is the first requirement to conduct random sampling or an experiment. The sample size is the total number of units, observations, or groups (in some cases) used to get the data to estimate a population parameter. As the name suggests, the sample size is that of the sample drawn from the population and differs from the population size.
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Random Sampling Method01:09

Random Sampling Method

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Sampling is a technique to select a portion (or subset) of the larger population and study that portion (the sample) to gain information about the population. Data are the result of sampling from a population. The sampling method ensures that samples are drawn without bias and accurately represent the population. Because measuring the entire population in a study is not practical, researchers use samples to represent the population of interest. Among the various sampling methods used by...
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Stratified Sampling Method01:16

Stratified Sampling Method

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Sampling is a technique to select a portion (or subset) of the larger population and study that portion (the sample) to gain information about the population. The sampling method ensures that samples are drawn without bias and accurately represent the population. Because measuring the entire population in a study is not practical, researchers use samples to represent the population of interest.
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Related Experiment Video

Updated: Sep 10, 2025

A Clinical Trial Assessing the Safety, Efficacy, and Delivery of Olive-Oil-Based Three-Chamber Bags for Parenteral Nutrition
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Randomization in phase II trials: No exemption based on sample size.

Theodor Framke1, Gernot Beutel2, Arnold Ganser2

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British Journal of Clinical Pharmacology
|August 27, 2025
PubMed
Summary

Single-arm trials (SATs) may seem feasible but can lead to misleading results in drug development. Randomized clinical trials (RCTs) are crucial for clear interpretation, even with small patient groups.

Keywords:
AML/MDSclofarabinedrug development programmeevidentiary standardsfludarabinephase 2randomization

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Area of Science:

  • Hematology
  • Clinical Pharmacology
  • Oncology

Background:

  • Randomized clinical trials (RCTs) are challenging, especially in early drug development or rare diseases.
  • Single-arm trials (SATs) are sometimes used instead of RCTs due to perceived feasibility, often employing historical controls.
  • This approach can significantly complicate the interpretation of trial outcomes.

Purpose of the Study:

  • To present results from a stem cell transplantation trial investigating fludarabine and clofarabine for induction chemotherapy.
  • To discuss the planning and assessment of a trial initially designed as a single-arm trial (SAT).
  • To highlight the implications of using SATs versus RCTs for interpreting trial results.

Main Methods:

  • The study involved a trial for high-risk acute myeloid leukemia or advanced myelodysplastic syndrome.
  • The trial investigated fludarabine and clofarabine as induction chemotherapy agents.
  • The focus was on the planning and assessment of the trial from a single-arm perspective.

Main Results:

  • Changes in treatment settings or patient selection in SATs can create 'promising results' that are difficult to validate.
  • These apparent successes may generate unrealistic expectations that subsequent randomized trials cannot confirm.
  • Interpreting SATs is only straightforward when treatment effects are exceptionally large and individual patient outcomes exceed all prior experience.

Conclusions:

  • Randomized clinical trials (RCTs) are essential for straightforward interpretation of results, regardless of sample size.
  • Single-arm trials (SATs) can lead to misinterpretations and unwarranted hopes, particularly when treatment effects are not overwhelmingly large.
  • The use of RCTs is recommended to ensure reliable conclusions in drug development and clinical research.