Back to Explorer

Adaptive Designs for Medical Device Clinical Studies: Guidance for Industry and Food and Drug Administration Staff

FinalCenter for Devices and Radiological Health Center for Biologics Evaluation and Research07/27/2016

Description

An adaptive design for a medical device clinical study is defined as a clinical study design that allows for prospectively planned modifications based on accumulating study data without undermining the study’s integrity and validity. Adaptive designs, when properly implemented, can reduce resource requirements, decrease time to study completion, and/or increase the chance of study success.This guidanceprovides sponsors and Food and Drug Administration (FDA) staff with guidance on how to plan and implement adaptive designs for clinical studies when used in medical device development programs.

Scope & Applicability

Product Classes

7
Diagnostic Devices

Special Considerations for Diagnostic Devices

Non-Significant Risk Device

Device studies that may not require an IDE submission to FDA; Devices that do not meet the definition of significant risk

Significant Risk Device

Devices requiring full IDE submission

Therapeutic Device

rationale for adaptive study may differ from diagnostic devices

Diagnostic Device

unique issues with adaptive study designs for diagnostic devices; heterogeneous in scope and include devices that may be invasive

Combination Product

Products combining drug, device, or biological constituents; Generally recommended for Enhanced Documentation; Requires 14971-based framework incorporating ICH Q9; A drug-device combination where the device constituent part detects ingestion.

Medical Device

FDA intends to assess device cybersecurity based on a number of factors; demonstrate or maintain its safety and effectiveness; ensuring cybersecurity has become essential to FDA’s ability to protect the public health; Cyber-resiliency capabilities for medical devices

Stakeholders

6
Sponsor

Entity responsible for submitting applications under section 524B

DMC

Data monitoring committee; Oversight body that may request interim analysis

Data Monitoring Committee

Safety oversight body for interpreting adverse events; Safety oversight body for study design

Investigator

Responsible for qualifications, training, and trial conduct; Individual responsible for trial conduct and data governance at a site.; May delegate tasks but retains overall responsibility; Person responsible for the conduct of the clinical trial at a trial site; Responsible for trial conduct and participant safety; Responsible for trial conduct, data integrity, and investigational product management.; Individual responsible for trial conduct at a site and informing the institution.; maintaining

Data Monitoring Committees

May access unblinded data as described in the DMC Charter.

Institutional Review Boards

Bodies responsible for evaluating trial risks and benefits for pregnant participants.; IRBs experienced in this population may advise on compensation.

Regulatory Context

Attributes

10
Operational Bias

Bias arising from differences in study conduct.

Conditional Power

Probability of a statistically significant treatment effect based on interim results.

Prevalence

Estimated number of people with a disease, used to determine clinical investigation enrollment.

Alpha

The significance level, typically 0.05.; Each vertex is allocated an initial amount of alpha.

Significant Risk

Risk classification for medical device clinical investigations.; A classification for medical device studies.; A risk determination for a clinical study.

Type I error rate

Statistical parameter requiring strong control when testing multiple endpoints

Multiplicity

Approach to handling multiplicity

Type II Error

failing to conclude that a device was effective when it was

Blinded

Pathologists must remain blinded to treatment arm

Statistical Bias

Bias making it difficult to characterize the true effect of the device

Related CFR Sections (5)

Related Warning Letters (10)

  • Institutional Review Board (IRB)

    Armstrong County Memorial Hospital

    2024-11-05
  • Institutional Review Board (IRB)

    Louisiana State University Health Science Center IRB

    2024-10-08
  • Bioresearch Monitoring Program/IRB

    Massachusetts Institute of Technology MIT

    2024-07-16
  • Clinical Investigator (Sponsor)

    Angela D. Ritter, M.D.

    2024-06-18
  • Institutional Review Board (IRB)

    New York State Psychiatric Institute IRB

    2024-03-26
  • Clinical Investigator

    Jeffrey W. Taub, M.D./Children's Hospital of Michigan

    2024-01-09
  • Clinical Investigator

    Luis Javier Pena-Hernandez, M.D., FCCP

    2023-09-29
  • Bioresearch Monitoring Program/Institutional Review Board (IRB)

    Larkin Community Hospital Institutional Review Board

    2023-01-24
  • Clinical Investigator (CI)

    Sabine S. Hazan, M.D.

    2022-03-15
  • Clinical Investigator

    Richard J. Obiso, PhD dba Avila Herbals, LLC

    2022-02-15

See Also (8)