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Incorporating Voluntary Patient Preference Information over the Total Product Life Cycle: Draft Guidance for Industry, Food and Drug Administration Staff, and Other Interested Parties

DraftCenter for Devices and Radiological Health Center for Biologics Evaluation and Research09/06/2024

Description

FDA is issuing this draft guidance to communicate when and what methods could be used to collect and submit patient preference information (PPI) across the total product life cycle. This will include helping explain when PPI may be helpful, and in what context. Without this re-issuance, sponsors and other patient preference study developers will not be aware of updated methodologies and considerations, which could result in costly studies that may not be appropriate to inform benefit-risk decision making. This update, when finalized, will provide more clarity in when and in what contexts different methods are best applied.

Scope & Applicability

Product Classes

8
CDRH-regulated devices

Medical devices regulated by the Center for Devices and Radiological Health

Pediatric device

includes smaller prosthesis sizes and is inserted via a surgical procedure

Implantable device

Telephone follow-up after surgery

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

CBER-regulated devices

Medical devices regulated by the Center for Biologics Evaluation and Research

Weight-loss device

Devices used for obesity treatment where PPI informed benefit-risk tolerance.

Hemodialysis device

Device cleared for solo home use based on PPI study results.

Pediatric ear tube system

Novel device where PPI established the performance goal for effectiveness.

Stakeholders

6
Sponsors

Assist sponsors in the nonclinical evaluation

Advisory Committee

Convened to provide advice on withdrawal issues

IRB

Institutional Review Board providing study approvals

Sponsor

Entity responsible for submitting applications under section 524B

Care-partners

Individuals such as parents whose preferences may be considered; involved in early stage qualitative discussions

Healthcare Professionals

Users whose characteristics impact user needs and communication format.; Intended users of the AI-enabled device; Intended User: Healthcare professionals

Regulatory Context

Attributes

9
Benefit-risk profile

Assessment for both mother and child

Minimum clinically meaningful benefit

quantitative PPI can be used to help define the minimum clinically meaningful benefit

Benefit-risk tradeoff

Balance between potential gains and harms

Benefit-risk tradeoffs

context defined in patient preference studies; parents surveyed prefer the benefit-risk tradeoff of this new device; patient willingness to accept risks in exchange for benefits

Generalizability

Applicability of HCEI from one health care setting to another.

Heterogeneity

Variations in patient preferences even within the same disease state

Preference Sensitive

When patient decisions regarding options vary based on personal values

Risk Tolerance

A patient's willingness to accept identified risks

Body Mass Index

expressed as kilograms of weight divided by height in meters squared (kg/m²); percentage change in body weight equals percentage change in BMI

Identified Hazards

Hazards

1
Cognitive bias

potential biases such as framing, anchoring, or ordering effect

Related CFR Sections (7)

Related Warning Letters (10)

  • Clinical Investigator

    Shirish M. Gadgeel, M.D.

    2025-09-09
  • Clinical Investigator

    Mark J. Savant, M.D

    2025-07-15
  • Bioresearch Monitoring Program/Institutional Review Board (IRB)

    United Health Products, Inc.

    2025-05-20
  • Clinical Investigator

    Americo F. Padilla, M.D.

    2025-03-25
  • Institutional Review Board (IRB)

    Armstrong County Memorial Hospital

    2024-11-05
  • Clinical Investigator

    Namita A. Goyal, M.D.

    2024-10-22
  • 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

See Also (8)