FDA Pediatric Medical Device Regulation: Pathways, Section 515A, and Waivers

A guide to US FDA pediatric device regulation: Section 515A submissions, premarket pathways, MDUFA user-fee waivers, extrapolation, and PDC grants.

Introduction: The Pediatric Device Development Challenge

Developing medical devices for pediatric populations is one of the most persistent challenges in the medical technology (medtech) sector. Because children differ from adults in anatomy, physiology, growth rates, and activity levels, devices designed for adults cannot simply be scaled down. An implant must accommodate a child's skeletal growth; a diagnostic tool must account for distinct pediatric reference ranges; and a delivery system must navigate smaller blood vessels.

Despite this clear clinical need, the market for pediatric medical devices is chronically underserved. The FDA reports that only a small fraction of premarket submissions are labeled for pediatric use. The primary barrier is commercial: the pediatric patient population is often too small to offer a return on the high capital investment required for traditional premarket clinical trials, which we discuss in our medical device clinical trials and IDE guide. Furthermore, conducting clinical trials in children presents major ethical and operational hurdles, including securing parental consent, child assent, and managing trial sites across limited pediatric specialty centers.

To address this gap, the US Congress and the FDA have built a multi-layered regulatory framework. This system imposes a mandatory reporting requirement on high-risk submissions under Section 515A of the FD&C Act, while offering financial and developmental incentives to sponsors. These incentives include standard premarket pathway adjustments, MDUFA premarket user-fee waivers, clinical data extrapolation allowances, and non-dilutive funding through the Pediatric Device Consortia (PDC) Grant Program.

This guide details how the FDA regulates pediatric medical devices, how to satisfy the Section 515A submission requirements, how to select the right premarket pathway and claim user-fee waivers, and how to leverage clinical data and consortia support to bring pediatric innovations to market.

What FDA Counts as "Pediatric": Definition and Subpopulations

The FDA defines the pediatric population as patients who are 21 years of age or younger at the time of diagnosis or treatment. This definition is codified in Section 520(m)(6)(E) of the Food, Drug, and Cosmetic Act (21 U.S.C. § 360j(m)(6)(E)) and applies across all premarket regulatory pathways, including Section 515A reporting.

The broad pediatric population is divided into four distinct subpopulations based on physiological and developmental stages. Sponsors must use these specific categories when compiling premarket data:

Subpopulation	Age Range	Clinical & Regulatory Considerations
Neonate	From birth to 1 month of age	High physiological volatility; rapid changes in cardiac, pulmonary, and renal function; requires specialized neonatal intensive care unit (NICU) design controls.
Infant	Greater than 1 month to 2 years of age	High growth rates; development of motor skills; transitioning diet; biocompatibility testing must account for developing immune systems.
Child	Greater than 2 years to 12 years of age	Active skeletal growth; broad variations in height and weight; cognitive development requires child-specific user interfaces and instructions.
Adolescent	Greater than 12 years to 21 years of age (up to, but not including, the 22nd birthday)	Transition through puberty; hormonal changes; adult-like anatomical dimensions but distinct psychosocial factors; potential for high-impact sports/activity.

Why the Age 21 Threshold Matters

A common point of confusion for regulatory affairs teams is the difference between the legal definition of adulthood (typically 18) and the FDA pediatric regulatory limit (21). The FDA utilizes the 21-and-under threshold because many physiological systems—particularly skeletal growth plates, neurological pathways, and hormonal systems—continue to mature past the age of 18. When evaluating a device for pediatric use, sponsors must ensure that clinical protocols and labeling specifically address the 21-and-under cohort, rather than cutting off data collection at 18.

Section 515A: The Mandatory Pediatric-Subset Submission

Section 302 of the Food and Drug Administration Amendments Act of 2007 (FDAAA) created Section 515A of the FD&C Act (21 U.S.C. § 360e-1). This section imposes a mandatory, non-waivable requirement on sponsors of specific premarket submissions to provide data on pediatric use.

On January 10, 2014, the FDA published the final rule, "Medical Devices; Pediatric Uses of Devices; Requirement for Submission of Information on Pediatric Subpopulations and Number of Affected Pediatric Patients" (79 FR 1735). This rule codified the Section 515A requirements under 21 CFR Parts 801, 814, and 860.

Which Submissions are Covered?

The Section 515A requirement applies to every:

Premarket Approval Application (PMA)
PMA Supplement (specifically those proposing a change in design, manufacturing, or labeling that affects clinical performance)
Humanitarian Device Exemption (HDE)
Product Development Protocol (PDP)

Note: Section 515A does not apply to standard 510(k) premarket notifications, De Novo classification requests (unless subsequently transitioned to a PMA), or Investigational Device Exemptions (IDEs).

What Information Must Be Provided?

Under the final rule, sponsors must submit "readily available" information regarding:

Pediatric Subpopulations: Whether the disease or condition that the device is intended to treat, diagnose, or cure affects any pediatric subpopulations.
Patient Estimates: The number of affected pediatric patients in the United States who suffer from the disease or condition.

The FDA defines "readily available" information as data that can be found in public databases, peer-reviewed literature, national registries, and standard medical textbooks. Sponsors are not required to conduct new clinical trials to satisfy Section 515A; however, they must perform a systematic literature search and document their findings.

The Section 515A Submission Checklist

Every PMA, HDE, or PDP must include a dedicated Section 515A section containing:

A systematic literature search protocol: Database names (e.g., PubMed, Embase, Cochrane), search terms used, date ranges, and exclusion/inclusion criteria.
Incidence or prevalence estimates: Quantitative estimates of the annual number of US patients age 21 or younger who suffer from the indicated disease or condition, stratified by the four pediatric subpopulations (neonate, infant, child, adolescent) if data is available.
A search for pediatric-specific device alternatives: Identification of any cleared or approved devices currently marketed in the US for the same pediatric indications.
A conclusion statement: A clear determination of whether the device is intended for pediatric use, or if the sponsor is proposing adult-only labeling despite the presence of the disease in pediatric populations.

Consequences of Omission

If a sponsor fails to include the Section 515A pediatric information, the FDA will issue a Refusal to Accept (RTA) notification or place the submission on hold. The sponsor will be forced to submit a formal PMA amendment, delaying the start of the substantive regulatory review.

Premarket Pathways for Pediatric Devices

A sponsor bringing a pediatric device to market can utilize the standard FDA premarket pathways, but must adapt their regulatory strategy to account for pediatric-specific rules and exceptions.

                  ┌─────────────────────────────────────────┐
                  │      Is the device Class I, II,         │
                  │            or Class III?                │
                  └────────────────────┬────────────────────┘
                                       │
         ┌─────────────────────────────┼─────────────────────────────┐
         ▼ (Class I/II)                ▼ (Class II Novel)            ▼ (Class III)
  ┌──────────────┐              ┌──────────────┐              ┌──────────────┐
  │   510(k)     │              │   De Novo    │              │     PMA      │
  │  Standard    │              │  User-Fee    │              │  Pediatric   │
  │ Substantial  │              │  Waiver (If  │              │  User-Fee    │
  │ Equivalence  │              │  Sole Use)   │              │  Waiver (If  │
  └──────────────┘              └──────────────┘              │  Sole Use)   │
                                                              └──────┬───────┘
                                                                     │
                                                                     ▼ (Orphan Route)
                                                              ┌──────────────┐
                                                              │     HDE      │
                                                              │  Probable    │
                                                              │   Benefit    │
                                                              └──────────────┘

1. The 510(k) Pathway (Substantial Equivalence)

If a pediatric device is Class II and a predicate device exists that is cleared for pediatric use, the sponsor can file a standard 510(k) premarket notification.

The Challenge: Finding an appropriate pediatric predicate. If the only available predicates are labeled for adult use, expanding the indications for use to include pediatrics constitutes a "new technology" or "different intended use," which often triggers the need for clinical data.
The Strategy: Leverage the FDA's Special 510(k) or Abbreviated 510(k) programs if the modifications are limited to anatomical scaling without changing the core technological characteristics.

2. The De Novo Pathway (Novel Moderate-Risk Devices)

For novel pediatric devices with no predicate, but which present low-to-moderate risk, the De Novo classification process is the primary route. We outline the mechanics of this request in our FDA De Novo classification guide.

The Advantage: The De Novo pathway allows sponsors to establish special controls (such as pediatric-specific bench testing or labeling requirements) to regulate the device, avoiding the onerous clinical trial requirements of a PMA.
Pediatric Incentive: De Novo requests intended solely for a pediatric population are exempt from the standard premarket user fee, as detailed in the fee section below.

3. The PMA Pathway (Class III High-Risk Devices)

For high-risk Class III devices (e.g., pediatric heart valves, neural implants, or automated insulin pumps), a PMA is required.

The Evidentiary Standard: PMA approval requires "reasonable assurance of safety and effectiveness" supported by valid clinical evidence.
The Obstacle: Recruiting enough pediatric patients for a statistically powered clinical trial is frequently impossible. To mitigate this, sponsors must utilize clinical extrapolation or real-world evidence. For a broader analysis of Class III timelines, see our FDA PMA Class III high-risk device approval landscape report.

4. The Humanitarian Device Exemption (HDE) Pathway

The HDE pathway is the primary regulatory route for rare disease devices. An HDE is exempt from the effectiveness requirements of the FD&C Act. Instead, the sponsor must demonstrate that the device is safe and that its probable benefit to patients outweighs the risk of injury or illness.

HUD Designation First: The sponsor must first obtain a Humanitarian Use Device (HUD) designation from the FDA's Office of Orphan Products Development (OOPD). This requires proving that the device treats or diagnoses a disease that affects fewer than 8,000 individuals in the US per year.
The Pediatric Profit Exception: Historically, HDE regulations prohibited sponsors from selling HUDs for a profit. However, under FD&C Act Section 520(m)(6), Congress established a profit exception for pediatric HUDs. A sponsor can sell a pediatric HUD for a profit if the device is intended for the treatment or diagnosis of a disease that occurs in pediatric patients, provided the number of devices distributed annually does not exceed the Annual Distribution Number (ADN), typically defined as the number of devices needed to treat 8,000 individuals. For a detailed breakdown of these rules, see our Humanitarian Device Exemption (HDE) guide.

The Pediatric User-Fee Waiver: Mechanics and the "Adult Trap"

Under the Medical Device User Fee Amendments (MDUFA), the FDA charges substantial fees to review premarket submissions. To incentivize pediatric development, Congress established a statutory user-fee exemption for submissions intended solely for pediatric use.

The Premarket User-Fee Waiver Table

For FY2026 (October 1, 2025 through September 30, 2026), the MDUFA premarket user fees and the savings for pediatric-only submissions are structured as follows:

Submission Type	Standard FY2026 Fee	Small-Business Fee	Pediatric-Only Exemption Savings
PMA (Original)	$579,272	$144,818	Saves up to $579,272
De Novo Request	$173,782	$43,446	Saves up to $173,782
510(k) Notification	$26,067	$6,517	Saves up to $26,067
PMA Supplement (Panel Track)	$463,418	$115,855	Saves up to $463,418
513(g) Request	$7,820	$3,910	No Exemption (Not exempt from fees)

Source: FDA Medical Device User Fee Amendments (MDUFA) Fees page, FY2026 schedule. Fees are adjusted annually; MDUFA VI takes effect October 1, 2027.

Eligibility for the Exemption

To qualify for the MDUFA pediatric user-fee waiver:

The premarket submission must be for a device intended solely for a pediatric population.
The labeling must contain clear limitations restricting the use of the device to patients age 21 or younger.
The sponsor must submit a formal user-fee cover sheet selecting the "Pediatric Exemption" option.

The "Adult-Use Change" Trap

Sponsors must be aware of a critical regulatory clawback: the adult-use change trap. If a manufacturer secures a pediatric user-fee waiver for a device, and subsequently files a premarket submission to expand the indications for use to include adults (or any portion of the adult population), the FDA is legally required to collect the original waived fee.

The manufacturer must pay the full, current fee for the new submission plus the fee that was waived for the original pediatric submission. For a small business, this clawback can trigger an unexpected invoice of over $100,000, which must be paid before the FDA will begin reviewing the adult-labeling submission. Therefore, if a company plans to clear a device for both adult and pediatric populations, it is often more cost-effective to submit the combined label initially and pay the standard fee, rather than using the pediatric waiver as a temporary step.

Clinical Evidence & Extrapolation: Leveraging Adult Data

Because clinical trials in children are logistically and ethically difficult, the FDA allows sponsors to leverage existing clinical data from adult populations or other pediatric subgroups to support pediatric indications.

The Extrapolation Guidance

On June 21, 2016, the FDA issued the final guidance, "Leveraging Existing Clinical Data for Extrapolation to Pediatric Uses of Medical Devices." This guidance defines when and how adult data can be extrapolated to support a pediatric premarket submission:

[Adult Clinical Data Available]
              │
              ▼
[Analyze Course of Disease & Device Effects]
              │
              ├─► Are they similar in Adults & Pediatrics?
              │     ├─► Yes (Full Extrapolation Allowed)
              │     └─► No (Partial/No Extrapolation Allowed)
              ▼
[Determine Data Gaps (e.g., Pediatric Anatomy)]
              │
              ▼
[Fill Gaps with Targeted Bench, Animal, or Small Clinical Study]

To use extrapolation, sponsors must compile evidence demonstrating:

Similarity of Disease Course: The pathophysiology of the disease or condition must be similar in both adult and pediatric populations.
Similarity of Device Effects: The biological and mechanical response of the device must be expected to perform similarly in pediatric tissue.

Types of Extrapolation

Full Extrapolation: If the disease course and device effects are identical, the FDA may approve the pediatric indication based entirely on the adult clinical trial data, supplemented by pediatric-specific bench testing (e.g., scaling fatigue testing) and anatomical analysis.
Partial Extrapolation: If there are minor differences (e.g., pediatric patients have higher metabolic rates or different vascular dimensions), the sponsor can use the adult data to establish a baseline of safety and effectiveness, and then conduct a small, targeted pediatric clinical study (often utilizing a single-arm design) to verify performance in children.

Real-World Evidence (RWE) and Registries

The FDA actively encourages the use of registries and RWE to support pediatric clearances. For details on how to compile these registries, see our real-world evidence for medical devices guide. In pediatric orthopedics and cardiology, registries like the National Cardiovascular Data Registry (NCDR) or international pediatric registries contain years of off-label device usage data. Sponsors can analyze this registry data to compile retrospective clinical evidence packages, avoiding the need to run prospective, de novo clinical trials.

The Pediatric Advisory Panel Requirement

Under the Medical Device User Fee and Modernization Act (MDUFMA) Section 210, which amended Section 515(c) of the FD&C Act, the FDA is required to ensure that medical device advisory panels include or consult with one or more pediatric experts when reviewing submissions that include pediatric indications. This ensures that the clinical extrapolation models and pediatric safety margins are evaluated by clinicians who specialize in pediatric care.

The Pediatric Device Consortia (PDC) Grant Program

To address the lack of commercial funding for pediatric medtech, the FDA's Office of Orphan Products Development (OOPD) administers the Pediatric Device Consortia (PDC) Grant Program. Established by Congress under the Pediatric Medical Device Safety and Improvement Act of 2007, the program was reauthorized by the Food and Drug Administration Reauthorization Act (FDARA) of 2017 and the Food and Drug Omnibus Reform Act (FDORA) of 2022, which extended funding through Fiscal Year 2027.

Program Structure & Funding

The PDC program funds nonprofit, academic-clinical consortia across the United States. These consortia do not design devices themselves; instead, they act as regional accelerators, providing advisory services, clinical trial connections, regulatory pathways planning, and direct seed grants to early-stage pediatric device innovators.

Cumulative Impact: Since its inception in 2009, the PDC program has awarded approximately $37 million in grants to consortia.
Projects Assisted: The consortia have advised or assisted more than 1,000 medical device projects.
Market Success: As of September 2018, the program had successfully helped 19 pediatric devices transition from early concept to FDA clearance or approval.

The Five Currently Funded Consortia

Sponsors can contact any of the five FDA-funded consortia for support:

West Coast CTIP (Center for Technology and Innovation in Pediatrics): Led by Children's Hospital Los Angeles (CHLA) and the University of Southern California (USC). Focuses on digital health, diagnostics, and neonatal devices.
UCSF-Stanford Pediatric Device Consortium: Jointly managed by the University of California, San Francisco and Stanford University. Focuses on surgical instruments, pediatric implants, and engineering design.
Pennsylvania Pediatric Device Consortium: Managed by the Children's Hospital of Philadelphia (CHOP) and the Pennsylvania Pediatric Medical Device Consortium network. Focuses on orthopedic implants, biomaterials, and pulmonary devices.
National Capital Consortium for Pediatric Device Innovation (NCC-PDI): Led by Children's National Hospital and the University of Maryland. Focuses on cardiovascular, orthopedic, and pediatric surgical robotics.
Southwest-Midwest National Pediatric Device Innovation Consortium (SWPDC): Led by Texas Children's Hospital and Baylor College of Medicine. Focuses on clinical trial design, engineering prototyping, and regional market access.

Services and Seed Grants

The consortia provide critical support for medtech startups:

Regulatory Advising: Helping sponsors plan Pre-Submissions, draft Section 515A reports, and select between 510(k), De Novo, and HDE pathways.
Clinical Trial Matching: Connecting sponsors with pediatric clinical investigators and trial sites.
Seed Grants: The consortia host annual pitch competitions, awarding non-dilutive seed grants of up to $50,000 per project to fund prototype development, biocompatibility testing, or pilot clinical studies.

Pediatric Review Timelines: FDA Performance Metrics

Sponsors evaluating the financial viability of a pediatric device must factor in regulatory review timelines. The table below compiles historical performance metrics from the FDA's Annual Report on the Premarket Approval of Pediatric Uses of Devices:

Pediatric Submission Review Performance (FY2017)

Pathway	Number of Pediatric Submissions	Median FDA Review Days	Median Total Elapsed Days	Primary Clinical Data Characteristics
PMA (Pediatric Indication)	16	179 Days	327.5 Days	Heavily relied on adult clinical data extrapolation combined with pediatric registry analysis and bench testing.
HDE (Orphan Pediatric)	2	220.5 Days	Not separately reported	Evaluated under the "probable benefit" standard; clinical trials were limited to small, single-arm safety cohorts.

Source: FDA Annual Report on Premarket Approval of Pediatric Uses of Devices, FY2017. The FY2017 report breaks out pediatric PMA and HDE review times; De Novo pediatric review metrics are not separately reported in that annual report.

Frequently Asked Questions

Does Section 515A apply to 510(k) submissions or only to PMA/HDE/PDP?

Section 515A applies only to PMAs, PMA supplements, HDEs, and PDPs. It does not apply to standard 510(k) premarket notifications. However, if your 510(k) predicate is labeled for adult use and you are expanding the label to include children, the FDA will review the submission for substantial equivalence. While you will not file a Section 515A report, you must still provide clinical or bench data to support the pediatric indication.

What happens if we omit the Section 515A pediatric information from our PMA?

If you omit this information, the FDA will refuse to accept (RTA) your submission during the initial administrative screening. Your file will be placed on hold, and the substantive review will not begin until you submit a PMA amendment containing the required literature search and patient population estimates.

If our device is labeled solely for pediatric use, do we still pay an MDUFA user fee?

No. An application for a device intended solely for pediatric use is exempt from premarket user fees across 510(k), De Novo, and PMA pathways. However, this waiver does not apply to 513(g) requests for classification information. Additionally, you must place strict pediatric limitations in your device labeling to qualify.

Can we extrapolate adult clinical data to support a pediatric indication, and under what conditions?

Yes, under the FDA's 2016 extrapolation guidance. You must demonstrate that the disease course (pathophysiology) and the device effects (biological and mechanical responses) are similar between adult and pediatric populations. Any data gaps (such as pediatric anatomical dimensions or tissue differences) must be addressed through targeted bench testing, animal models, or small clinical safety studies.

What can a Pediatric Device Consortium actually do for an early-stage pediatric device company?

A consortium provides non-dilutive seed grants (up to $50,000), connects you with pediatric clinical investigators, helps design clinical trial protocols, and offers regulatory consulting to help you prepare Pre-Submissions and plan your FDA pathway.

Disclaimer

This guide is for educational and informational purposes only. The information provided does not constitute legal, regulatory, or clinical advice for any specific medical device or company. The regulatory requirements, user-fee schedules, and guidance documents issued by the US Food and Drug Administration are subject to change. Sponsors should consult qualified regulatory affairs professionals and legal counsel, and engage the FDA through the Pre-Submission process, before finalizing their pediatric device development plans.