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Related Concept Videos

Clinical Trials: Overview01:11

Clinical Trials: Overview

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Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
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Clinical Trials01:16

Clinical Trials

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Clinical trials are prospective experimental studies conducted on humans to determine the safety and efficacy of treatments, drugs, diet methods, and medical devices. Using statistics in clinical trials enables researchers to derive reasonable and accurate conclusions from the collected data, allowing them to make wise decisions in uncertain situations. In medical research, statistical methods are crucial for preventing errors and bias.
There are four phases in a clinical trial. A phase one...
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Preclinical Development: Overview01:28

Preclinical Development: Overview

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Preclinical development consists of a series of tests that ensure the safety and efficacy of a new therapeutic compound before it is tested in humans. There are four main phases to this process. First, safety pharmacology tests are conducted to ensure the drug does not produce any acutely harmful effects. These tests examine parameters such as bronchoconstriction, cardiac dysrhythmias, blood pressure changes, and ataxia. Next, preliminary toxicological testing is performed to determine the...
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Crossover Experiments01:16

Crossover Experiments

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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.
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Study Designs in Epidemiology01:20

Study Designs in Epidemiology

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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...
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Hazard Ratio01:12

Hazard Ratio

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The hazard ratio (HR) is a widely used measure in clinical trials to compare the risk of events, such as death or disease recurrence, between two groups over time. It reflects the ratio of hazard rates—the instantaneous risk of the event occurring—between a treatment group and a control group. This measure provides valuable insights into the relative effectiveness of a treatment by assessing how the risk of an event differs between the two groups.
For example, in a clinical trial...
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Updated: Apr 15, 2026

A Clinical Trial Assessing the Safety, Efficacy, and Delivery of Olive-Oil-Based Three-Chamber Bags for Parenteral Nutrition
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Phase II design: history and evolution.

Larry Rubinstein1

  • 1Biometric Research Branch, NCI, Bethesda, MD, USA. rubinsteinl@mail.nih.gov.

Chinese Clinical Oncology
|April 6, 2015
PubMed
Summary
This summary is machine-generated.

Phase II oncology trials are evolving. While historically focused on tumor response rates, modern trials increasingly use progression-free survival (PFS) or overall survival (OS) to evaluate targeted therapies.

Keywords:
Phase II trialphase II/III trialrandomized phase II trialrandomized screening trialrandomized selection design trial

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Area of Science:

  • Clinical Trials
  • Oncology Drug Development
  • Biostatistics

Background:

  • Phase II oncology trials traditionally used single-arm designs focused on tumor response rates.
  • This paradigm assumed a 20% response rate was necessary for subsequent Phase III success.
  • Recent oncology drug development trends challenge this historical approach.

Purpose of the Study:

  • To review the history and evolution of phase II trial designs in oncology.
  • To highlight the shift towards alternative endpoints like progression-free survival (PFS) and overall survival (OS).
  • To provide a primer on phase II trial designs for investigators.

Main Methods:

  • Review of historical and contemporary phase II oncology trial designs.
  • Analysis of endpoint selection, including tumor response rate, PFS, and OS.
  • Discussion of statistical considerations for endpoint choice.

Main Results:

  • Phase II trial designs have evolved from solely focusing on tumor response rates.
  • Progression-free survival (PFS) is often preferred over overall survival (OS) due to statistical efficiency.
  • Overall survival (OS) may be chosen when salvage therapy is ineffective or progression assessment is complex.

Conclusions:

  • The traditional phase II oncology trial paradigm is being updated.
  • Molecularly targeted agents may improve PFS or OS through mechanisms other than direct tumor shrinkage.
  • Careful endpoint selection in phase II trials is crucial for assessing novel oncology agents.