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 Experiment Videos

FDA perspective on trials with interim efficacy evaluations.

Robert Temple1

  • 1Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, MD, USA. robert.temple@fda.hhs.gov

Statistics in Medicine
|July 19, 2006
PubMed
Summary

Adaptive trial designs, including sample size adjustments and enrichment strategies, offer efficient ways to conduct clinical research. These methods aim to improve trial outcomes by focusing on specific patient populations and optimizing data analysis for better therapeutic targeting.

Related Concept Videos

You might also read

Related Articles

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

Sort by
Same author

Closing the gap: A United States perspective on enhancing drug evaluation in older adults.

Journal of the American Geriatrics Society·2024
Same author

Participation of Older Adults in Clinical Trials for New Drug Applications and Biologics License Applications From 2010 Through 2019.

JAMA network open·2022
Same author

Effect of Mailing Educational Material to Patients With Atrial Fibrillation and Their Clinicians on Use of Oral Anticoagulants: A Randomized Clinical Trial.

JAMA network open·2022
Same author

Roadmap to 2030 for Drug Evaluation in Older Adults.

Clinical pharmacology and therapeutics·2021
Same author

U.S. racial and ethnic participation in global clinical trials by therapeutic areas.

Journal of clinical pharmacy and therapeutics·2021
Same author

Encouragement of subgroup assessment by the FDA.

Pharmaceutical statistics·2021

Area of Science:

  • Clinical trial methodology
  • Biostatistics
  • Pharmaceutical research

Background:

  • The U.S. Food and Drug Administration (FDA) has observed various adaptive trial designs over time.
  • Adaptive approaches, such as repeated p-value calculations and sample size adjustments based on event rates, are increasingly of interest.
  • These methods can be implemented with blinded or unblinded analyses, and group sequential methods are common in large trials.

Purpose of the Study:

  • To review and discuss adaptive clinical trial designs and their applications.
  • To highlight the benefits and considerations of different adaptive strategies in pharmaceutical research.
  • To emphasize the importance of careful design in maximizing trial efficiency and therapeutic targeting.

Main Methods:

Related Experiment Videos

  • Discussion of adaptive approaches including repeated p-value calculations.
  • Exploration of sample size adjustments based on event rates (blinded or unblinded).
  • Review of group sequential analytic approaches and early stopping criteria, particularly for cardiac trials focusing on survival endpoints.
  • Consideration of adaptive randomization and enrichment designs.

    • Main Results:

    • Adaptive designs can potentially reduce sample sizes and target therapies more effectively.
    • Enrichment designs focus on populations with higher event rates or better response likelihood.
    • Early stopping, while reducing data, requires careful consideration of endpoints, especially survival in cardiac trials.

    Conclusions:

    • Adaptive strategies offer flexibility and efficiency in clinical trial design.
    • Enrichment designs can enhance the likelihood of detecting treatment effects and targeting therapies.
    • Careful selection of adaptive methods and endpoints is crucial for successful clinical research and drug development.