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Humanitarian Device Exemption (HDE) Program: Guidance for Industry and Food and Drug Administration Staff

FinalCenter for Devices and Radiological Health Center for Biologics Evaluation and Research09/06/2019

Description

FDA developed thisguidance documentto provide clarity to industry and FDA staff about the current review practices for the Humanitarian Device Exemption (HDE) Program. This programmatic guidance addresses commonly asked questions about HDEs and Humanitarian Use Devices (HUDs), including FDA actions on HDE applications, post-approval requirements, and special considerations for devices marketed under the HDE Program. This guidance document reflects changes in the HDE Program resulting from statutory amendments made by the 21st Century Cures Act (Cures Act) and explains the criteria FDA considers to determine if  “probable benefit” has been demonstrated as part of the Agency’s decision-making process regarding marketing authorization for a HUD. This guidance document also reflects amendments made to the HDE provision of the Federal Food, Drug, and Cosmetic Act (FD&C Act) by the FDA Reauthorization Act of 2017 (FDARA).

Scope & Applicability

Product Classes

3
Humanitarian Use Devices

Medical devices intended to benefit patients in the treatment or diagnosis of diseases affecting not more than 8,000 individuals in the U.S. per year.; Devices intended for diseases occurring in fewer than 8,000 individuals; devices approved under an HDE

Humanitarian Use Device

Medical device intended for small populations

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.

Stakeholders

9
HDE holder

Entity responsible for HDE submissions.

care-partner

Care-partner perspectives may be considered where it is not feasible to obtain PPI.; Perspective considered during benefit and risk assessment

applicant

entity submitting marketing applications

Clinical Investigator

Veterinarian responsible for selecting anesthetic regimens and conducting field studies

Institutional Review Board

Governs top dose in clinical studies

patient advocacy groups

consulting in advance with patient advocacy groups; Entities that may review consent forms to ensure they facilitate understanding

HDE applicant

Entity submitting the HDE application

Appropriate local committee

Alternative to IRB for approving HUD use at a facility; Standing committee for the facility that has expertise in clinical care

Independent certified public accountant

Prepares report verifying device costs

Regulatory Context

Attributes

10
Probable benefit

The standard used by FDA to determine if a HUD outweighs the risk of injury or illness.; Standard for HDE approval weighing benefit against risk; Standard for HDE approval where patients are likely to benefit

Indications for Use

conformity with an FDA-recognized standard can help demonstrate that an NGS-based test developer has performed the activities necessary to identify the indications for use; number and types of samples used in the study should be statistically justified for the test’s indications for use; If a modification expands the indications for use beyond aiding in the diagnosis of symptomatic individuals; Acceptable performance levels depend on these

risk classification

reasonably foreseeable risks or side effects

Reasonable assurance of effectiveness

requirement from which HDEs are exempt

Probable benefit-risk assessment

evaluation factor for HDE approval

Valid Scientific Evidence

Standard for data supporting an HDE

Shelf Life

Determined based on stability data; Established through stability testing; also called dating period.; Established through formal stability studies; Establishing the period during which a drug product is expected to remain within specifications; Period for drug products; The period during which a drug product is expected to remain within specifications; Duration product remains within acceptance criteria; Period during which product remains within specifications; Established for intermediates pu

Non-Significant Risk

The IRB would need to make a SR/NSR determination

Significant Risk

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

geographic dispersion

Factor considered for 'highly impracticable' determination

Identified Hazards

Hazards

5
Unreasonable risk of illness or injury

Safety standard evaluated during substantive review

false-negative results

Risk assessment factor for diagnostic devices

false-positive results

Risk assessment factor for diagnostic devices

risk of injury or illness

FDA must determine that the device will not expose patients to an unreasonable or significant risk of illness or injury.

unreasonable or significant risk of illness or injury

Criteria for determining if pediatric development is unsafe

Related CFR Sections (12)

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See Also (8)

Humanitarian Device Exemption (HDE) Program: Guidance for Industry and Food and Drug Administration Staff | Guideline Explorer | BioRegHub