Related Experiment Video

Updated: May 18, 2026

A Clinical Trial Assessing the Safety, Efficacy, and Delivery of Olive-Oil-Based Three-Chamber Bags for Parenteral Nutrition
04:53

A Clinical Trial Assessing the Safety, Efficacy, and Delivery of Olive-Oil-Based Three-Chamber Bags for Parenteral Nutrition

Published on: September 20, 2019

Novel trial designs for monotherapy

Martin J Brodie1

  • 1Epilepsy Unit, Western Infirmary, Glasgow, Scotland, UK. mjb2k@clinmed.gla.ac.uk

Epileptic Disorders : International Epilepsy Journal with Videotape
|September 15, 2012
PubMed

Insights

Regulatory trials for new epilepsy drugs differ between the US and Europe. This paper explores trial designs for novel antiepileptic drugs (AEDs) in drug-naïve epilepsy patients, aiming for transatlantic regulatory approval.

Area of Science:

  • Neurology
  • Clinical Pharmacology
  • Regulatory Science

Background:

  • Licensing novel antiepileptic drugs (AEDs) for drug-naïve epilepsy patients requires specific regulatory trial programs.
  • Regulatory approaches in the US (FDA) and Europe (EMA) differ significantly for these trials.

Purpose of the Study:

  • To discuss the advantages and disadvantages of current FDA and EMA trial designs for new AEDs.
  • To explore novel trial designs that could satisfy both American and European regulatory authorities.

Main Methods:

  • Comparative analysis of FDA's withdrawal to monotherapy data with historical controls versus EMA's head-to-head randomized studies.
  • Exploration of alternative trial designs, focusing on placebo-controlled studies after a single seizure with EEG/imaging support.

Main Results:

  • FDA requires superiority data against historical controls, while EMA accepts non-inferiority data against established comparators.
  • Current designs present challenges for drug developers seeking simultaneous approval.

Conclusions:

  • There is a need for harmonized or adaptable trial designs to streamline the approval process for new antiepileptic drugs.
  • Novel approaches, such as carefully selected placebo-controlled studies, may offer a viable path to satisfy diverse regulatory requirements.

More Related Videos

In Silico Clinical Trials for Cardiovascular Disease09:09

In Silico Clinical Trials for Cardiovascular Disease

Published on: May 27, 2022

Related Concept Videos

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

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...
Clinical Trials: Overview01:11

Clinical Trials: Overview

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...
Clinical Trials01:16

Clinical Trials

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...
Dosage Regimens: Designs and Approaches01:28

Dosage Regimens: Designs and Approaches

Designing a dosage regimen, which refers to the manner of drug administration, is a complex process involving the selection of drug dose, route, and frequency. This process is underpinned by pharmacokinetic parameters derived from tests and population averages. These parameters are then tailored to patient-specific variables such as diagnosis, demographics, and allergy status. Once therapy commences, therapeutic response monitoring is critical and achieved through clinical and physical...
Crossover Experiments01:16

Crossover Experiments

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.
Bioequivalence Experimental Study Designs: Completely Randomized and Randomized Block Designs01:20

Bioequivalence Experimental Study Designs: Completely Randomized and Randomized Block Designs

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 subjects...