Medical Device Expanded Access: Emergency Use, Compassionate Use & Treatment IDE

When a patient faces a serious or immediately life-threatening condition and cannot participate in an ongoing clinical trial, treating physicians and device sponsors face an urgent regulatory question: How can an unapproved, investigational medical device be legally accessed for treatment outside the clinical trial protocol?

Unlike pharmaceutical expanded access—which is frequently channeled through individual-patient Investigational New Drug (IND) mechanisms and simplified forms—investigational medical devices operate under a distinct statutory and regulatory framework governed by Title 21 of the Code of Federal Regulations (CFR) Part 812 (Investigational Device Exemptions), Part 56 (Institutional Review Boards), and Part 50 (Protection of Human Subjects).

This guide provides a comprehensive operational roadmap for clinical investigators, hospital Institutional Review Boards (IRBs), clinical affairs teams, and regulatory sponsors navigating US expanded access for investigational medical devices. It details the three primary US mechanisms, exact submission pathways, filing clocks, IRB reporting exemptions, statistical insights from FDA Center for Devices and Radiological Health (CDRH) submission data, and critical distinctions from adjacent pathways such as Emergency Use Authorizations (EUA), Humanitarian Device Exemptions (HDE), the Right to Try Act, and EU MDR Article 59 national derogations.

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Quick-Reference Decision Matrix: The Three US Mechanisms

The US Food and Drug Administration (FDA) recognizes three distinct expanded-access mechanisms for investigational medical devices. Determining the correct mechanism depends on the immediacy of the life-threatening situation, whether an active Investigational Device Exemption (IDE) is already on file with FDA, and whether access is intended for an individual patient or a larger treatment cohort.

Mechanism	Patient Scope	Clinical Urgency	Prior FDA Approval Required?	Prior IRB Approval Required?	Primary Regulatory Vehicle	Key Follow-Up Reporting Deadline
Emergency Use	Individual patient	Immediately life-threatening; treatment cannot wait for FDA review	No (post-use reporting)	No (exempt under 21 CFR 56.104(c) if reported in 5 working days)	IDE 5-day report (if IDE exists) or Physician 5-day DCC letter (if no IDE)	5 working days to FDA & IRB
Compassionate Use	Individual patient or small group	Serious or life-threatening; time exists to request prior approval	Yes	Yes (Full IRB approval or Chair concurrence if permitted)	IDE Supplement under 21 CFR 812.35(a) or CDRH Non-IDE Request	45 days post-treatment follow-up report to FDA
Treatment IDE	Widespread patient population / cohort	Serious or immediately life-threatening; trials ongoing or completed	Yes (30-day default clock)	Yes (Full IRB review at each treating site)	Treatment IDE Application under 21 CFR 812.36	Semi-annual progress reports (Annual post-marketing application)

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What Is Expanded Access for Medical Devices, and How Does It Differ from a Clinical Trial?

Expanded access (often colloquially referred to as "compassionate use") refers to the legal pathway by which patients with serious or immediately life-threatening diseases or conditions can obtain access to an investigational medical device outside of a clinical trial when no comparable or satisfactory alternative therapy exists.

It is critical to distinguish expanded access from standard clinical investigations:

1. Primary Intent: The primary objective of a clinical investigation under an approved Investigational Device Exemption (IDE) is to collect safety and effectiveness data to support a premarket submission (such as a PMA, De Novo request, or 510(k)). The primary intent of expanded access is patient diagnosis, monitoring, or treatment, not hypothesis testing or evidence generation.
2. Subject Status: Emergency-use and individual compassionate-use patients are treated outside the IDE trial protocol and are not enrolled as protocol subjects in the pivotal study database. A licensed physician who treats patients under a Treatment IDE is an “investigator” under 21 CFR 812, and those patients remain investigational subjects for consent, IRB, and reporting purposes.
3. Data Utilization: Safety and outcome data collected during expanded access must be tracked and reported to FDA, but expanded access data cannot replace formal, controlled pivotal trials required for commercial approval.

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Mechanism 1: Emergency Use of an Unapproved Investigational Device

When Does Emergency Use Apply?

Emergency use applies strictly when an individual patient confronts an immediately life-threatening condition that requires immediate treatment with an investigational device, where no commercially available alternative exists, and where there is no time to obtain prior FDA approval.

Under FDA guidance and statutory interpretations of 21 CFR Part 812, emergency use is a post-hoc notification mechanism. FDA does not grant "pre-approval" or "clearance" on the spot; rather, the treating physician exercises clinical judgment under severe urgency, and compliance is evaluated through mandated post-use filings.

Pre-Procedure Patient Protection Requirements

Even though prior formal FDA approval is not required, FDA and human subject protection standards require the treating physician to implement as many patient safeguards as feasible before implanting or using the device:

1. Independent Physician Assessment: Concurrence from an independent physician who is not participating in the investigation, confirming in writing that the patient's condition is immediately life-threatening and that no standard acceptable alternative therapy is available.
2. Informed Consent: Full informed consent obtained from the patient or legally authorized representative (LAR) under 21 CFR 50.20 and 50.25. If obtaining consent is impossible due to the patient's acute incapacitation, the emergency consent exception criteria under 21 CFR 50.23 must be strictly satisfied and documented by both the treating physician and the independent physician.
3. Institutional & IRB Notification: While prior convened IRB review is exempt, the physician should notify the IRB Chair or administrative office prior to use if time allows, or secure concurrence per institutional policy.
4. Manufacturer Agreement: The device manufacturer must agree to release the device for emergency clinical use.

The 5-Working-Day Reporting Clocks

Following emergency use, two independent 5-working-day reporting obligations are triggered:

1. FDA Notification (Within 5 Working Days)

• If an IDE already exists for the device: The IDE sponsor notifies FDA. 21 CFR 812.35(a)(2) is the emergency-deviation reporting rule for an approved investigational plan (report to FDA within 5 working days after the sponsor learns of the deviation; see also 21 CFR 812.150(a)(4) for the investigator’s 5-working-day notice to the sponsor and IRB). FDA’s device expanded-access page uses that same 5-day IDE-report clock when emergency expanded access occurs and an IDE already exists.
• If no IDE exists for the device: The treating physician must submit a detailed follow-up report within 5 working days directly to the FDA CDRH Document Control Center (DCC). The report must include a comprehensive clinical summary, patient condition, justification for emergency use, device information, adverse events, independent physician concurrence, and a copy of the signed informed consent form (or 50.23 certification).

2. IRB Notification (Within 5 Working Days)

Under 21 CFR 56.104(c), emergency use of a test article is exempt from the requirement for prior IRB review, provided that the emergency use is reported to the IRB within 5 working days.

Subsequent-use trap (21 CFR 56.104(c)): Emergency use of a test article is exempt from prior IRB review provided the emergency use is reported to the IRB within 5 working days. Any subsequent use of the test article at that institution is subject to IRB review. A later patient with a similar condition cannot automatically reuse the same exemption; the institution needs prior IRB review (and, where applicable, a compassionate-use or Treatment IDE path).

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Mechanism 2: Compassionate Use (Individual Patient or Small Group)

Purpose and Scope

Compassionate use (also referred to as single-patient or small-group expanded access) applies when an individual patient—or a small, defined group of patients—has a serious disease or condition, is ineligible for any existing clinical trial, has no comparable alternative therapy, but the clinical situation is not an immediate emergency where treatment cannot wait for FDA review.

Unlike emergency use, prior FDA approval and prior IRB approval (or chair concurrence) are strictly required before the investigational device can be shipped or administered.

Filing Pathways: IDE on File vs. No IDE on File

The administrative procedure depends on whether an approved IDE is already active for the device:

1. Confirm the patient has a serious or life-threatening condition, cannot enroll in the trial, and has no satisfactory alternative—and that there is time to obtain prior FDA approval.
2. If an active US IDE exists: the IDE sponsor files a compassionate-use IDE supplement under 21 CFR 812.35(a) to the CDRH Document Control Center.
3. If no US IDE exists: the physician or manufacturer files a single-patient compassionate-use request to CDRH DCC and may coordinate via CDRHExpandedAccess@fda.hhs.gov.
4. FDA reviews IDE-supplement requests on the statutory 30-day cycle; CDRH states compassionate-use requests are reviewed on average within 15 days of receipt, and in as little as one day.
5. Obtain IRB approval or chair concurrence per institutional SOP (some IRBs wait for the FDA letter).
6. Execute 21 CFR Part 50 consent, ship under 21 CFR 812.5 labeling, and treat only after FDA approval.
7. Submit the follow-up report to FDA within 45 days of device use (and report problems to the reviewing IRB as soon as possible).

Pathway A: When an IDE Exists (21 CFR 812.35(a))

The sponsor of the active IDE submits an IDE Supplement for compassionate use under 21 CFR 812.35(a). The submission packet must contain:

1. Description of the patient's clinical history, diagnosis, and severity.
2. Clinical rationale explaining why alternative approved therapies are unsatisfactory or contraindicated.
3. Rationale for why the patient cannot participate in the ongoing IDE study.
4. Discussion of the device design, mode of operation, and risk-benefit profile.
5. Specific treatment plan and clinical monitoring protocol.
6. Draft informed consent document tailored to the expanded-access use.
7. Independent physician statement of concurrence.
8. Device accountability and tracking protocol.

FDA Review Timeline: While standard IDE supplements are governed by a statutory 30-day review cycle, CDRH prioritizes compassionate use IDE supplements. FDA states that compassionate-use requests are reviewed on average within 15 days of receipt, and in some cases in as little as one day.

Pathway B: When No IDE Exists

If no active IDE exists in the United States, either the treating physician or the device manufacturer submits an original compassionate use packet to the CDRH Document Control Center (and coordinates directly via CDRHExpandedAccess@fda.hhs.gov). The submission contains the same clinical, technical, and consent documentation as an IDE supplement.

Mandatory 45-Day Post-Treatment Follow-Up

Following compassionate use treatment, the treating physician or sponsor must submit a written Follow-Up Report to FDA within 45 days of device use (FDA’s device expanded-access page; the page does not distinguish working days from calendar days). The follow-up report must document:

• Patient clinical outcome and therapeutic response.
• Any Adverse Device Effects (ADEs), Unanticipated Adverse Device Effects (UADEs), or device malfunctions.
• Final device accountability (including return, destruction, or ongoing patient implantation status).
• Any deviations from the proposed treatment plan.

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Mechanism 3: Treatment IDE (21 CFR 812.36)

Purpose: Widespread Cohort Access During Active Development

A Treatment IDE is a formal regulatory mechanism established under 21 CFR 812.36 that allows for the widespread treatment use of an investigational device in a broader population while controlled clinical trials are ongoing or completed, and while the sponsor actively pursues premarket approval or clearance with due diligence.

Unlike single-patient compassionate access, a Treatment IDE functions as a multi-center treatment protocol operating under defined eligibility criteria.

The Four Statutory Criteria (21 CFR 812.36(b))

Under 21 CFR 812.36(b), FDA shall consider treatment use of an investigational device if all four of the following criteria are met (these are the gate for consideration, not an automatic approval):

1. Intended use: The device is intended to treat or diagnose a serious or immediately life-threatening disease or condition.
2. No comparable alternative: There is no comparable or satisfactory alternative device or other therapy available to treat or diagnose that stage of the disease or condition in the intended patient population.
3. Same-use investigation: The device is under investigation in a controlled clinical trial for the same use under an approved IDE, or such clinical trials have been completed.
4. Due diligence: The sponsor of the investigation is actively pursuing marketing approval/clearance of the investigational device with due diligence.

Disease Stage Timing Rules: Serious vs. Immediately Life-Threatening

21 CFR 812.36 establishes a distinct timing rule based on disease severity:

• Immediately Life-Threatening Diseases: Under 812.36(a), an “immediately life-threatening” disease is a stage of disease in which there is a reasonable likelihood that death will occur within a matter of months, or in which premature death is likely without early treatment. In that category, a device may be made available for treatment use before all clinical trials have been completed. FDA may still disapprove or withdraw a Treatment IDE if the evidence, taken as a whole, does not support a reasonable conclusion that the device may be effective or that it would not expose patients to an unreasonable and significant additional risk (21 CFR 812.36(d)(2)(iv)).
• Serious Diseases: For serious diseases that are not immediately life-threatening, treatment use is ordinarily available only after all clinical trials have been completed, during the window while the premarket submission is under FDA review.

The 30-Day Default Approval Clock (21 CFR 812.36(d))

Under 21 CFR 812.36(d), treatment use may begin 30 days after FDA receives the Treatment IDE submission, unless FDA notifies the sponsor in writing earlier than those 30 days that treatment use may or may not begin. FDA may approve the treatment use as proposed or approve it with modifications. “Clinical hold” is an IND concept; device Treatment IDEs are approved, approved with modifications, or disapproved/withdrawn under 21 CFR 812.36(d) and 812.30.

Reporting Obligations: Semi-Annual and Annual Clocks

A Treatment IDE incurs rigorous ongoing reporting requirements:

• Semi-Annual Progress Reports: Under 21 CFR 812.36(f), the sponsor must submit progress reports on a semi-annual basis to all reviewing IRBs and FDA until a marketing application is filed. Those reports must include the number of patients treated under the Treatment IDE, the names of participating investigators, and a brief description of the sponsor’s efforts to pursue marketing approval/clearance. Other reports required by 21 CFR 812.150 (including unanticipated adverse device effects) still apply.
• Annual Reports: After a marketing application is filed, progress reports are submitted annually in accordance with 21 CFR 812.150(b)(5) until marketing authorization is granted or denied.

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Regulatory Misconceptions: Pathways That Do NOT Apply to Medical Devices

One of the most frequent sources of compliance errors and submission rejections is the misapplication of drug-specific or emergency-specific regulations to medical device expanded access.

Pathway sometimes mixed in	Why it is not device expanded access
Form FDA 3926	Individual-patient expanded access INDs for drugs and biologics only. FDA’s Form FDA 3926 guidance states that the guidance and form do not apply to medical-device expanded-access requests.
Federal Right to Try Act (21 U.S.C. § 360bbb-0a)	Limited by statute to eligible investigational drugs. It creates no device authorization mechanism.
Emergency Use Authorization (FD&C Act § 564)	Commercial distribution authorization during a declared CBRN or infectious-disease public-health emergency. It is not a named-patient IDE tool. See the EUA guide.
Humanitarian Device Exemption (21 CFR 814 Subpart H)	A marketing authorization for a HUD (not more than 8,000 US patients per year), not investigational expanded access. See the HDE guide.
EU MDR Article 59	A Member State market-access derogation from CE marking, not a US-style IDE supplement. See the Article 59 guide.

1. Form FDA 3926 Does NOT Apply to Medical Devices

In pharmaceutical expanded access, physicians routinely use Form FDA 3926 (Individual Patient Expanded Access IND) for simplified single-patient drug requests.

However, FDA’s official guidance document, Individual Patient Expanded Access Applications: Form FDA 3926 (October 2017; page current as of 2024-01-29), states in its scope:

“This guidance and Form FDA 3926 do not apply to other types of expanded access requests, including request for expanded access for medical devices.”

Submitting Form FDA 3926 to CDRH for a medical device request is an administrative error that will delay review. Device compassionate use must be submitted as an IDE supplement or formal CDRH DCC expanded-access packet.

2. The Federal Right to Try Act Does NOT Apply to Devices

The Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017 (codified at 21 U.S.C. § 360bbb-0a) created an alternative pathway for terminally ill patients to access investigational medical products without FDA oversight.

However, the statutory text of 21 U.S.C. § 360bbb-0a is strictly limited to "eligible investigational drugs" (defined as drugs that have completed a Phase 1 clinical trial and are under active investigation in an IND). The Right to Try Act contains no provision for investigational medical devices. Any expanded access to an investigational device must proceed through FDA under 21 CFR Part 812.

3. Emergency Use Authorization (EUA) vs. Expanded Access

Emergency Use Authorizations (EUAs) under Section 564 of the FD&C Act (21 U.S.C. § 360bbb-3) operate on a completely different legal plane:

• EUA Requirement: Requires a formal declaration by the Secretary of Health and Human Services (HHS), Department of Defense (DoD), or Department of Homeland Security (DHS) of a chemical, biological, radiological, nuclear (CBRN), or infectious disease public health emergency.
• Scope: EUAs authorize the commercial distribution, importation, and clinical use of unapproved devices (e.g., diagnostic test kits, respirators, PPE) across the entire healthcare system during a declared crisis.
• Expanded Access: In contrast, Part 812 expanded access applies to individual patients or clinical cohorts on a named-patient basis during routine times, with no emergency declaration required.

4. Humanitarian Device Exemption (HDE) vs. Treatment IDE

A Humanitarian Device Exemption (HDE) under 21 CFR Part 814 Subpart H is a premarket marketing authorization, not an investigational study mechanism:

• An HDE authorizes commercial marketing of a Humanitarian Use Device (HUD) intended to benefit patients with rare diseases affecting fewer than 8,000 individuals in the US per year.
• Once an HDE is approved by FDA, the device can be sold commercially (subject to local IRB oversight at each hospital under 21 CFR 814.124).
• A Treatment IDE under 21 CFR 812.36 is an investigational bridge prior to marketing authorization.

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What CDRH Submission Data Reveal: Request Volume and Approval Trends

FDA CDRH publishes annual statistics on expanded-access submissions. Analyzing historical submission tables (FY2019 through FY2023) reveals significant trends in how investigational devices are actually accessed in US clinical practice.

CDRH Expanded Access Submissions: FY2019–FY2023

Fiscal Year	IDE requests received	IDE evaluable	IDE approved	IDE approval rate	Non-IDE single-patient received	Non-IDE evaluable	Non-IDE approved	Non-IDE approval rate	Total receipts
FY2019	76	69	67	97.1%	352	335	332	99.1%	428
FY2020	92	89	89	100%	384	372	372	100%	476
FY2021	106	103	102	99.0%	502	497	493	99.2%	608
FY2022	101	94	94	100.0%	587	580	578	99.66%	688
FY2023	104	101	101	100.0%	741	732	722	98.63%	845

Source: FDA Expanded Access (Compassionate Use) Submission Data, CDRH IDE and non-IDE tables (page current as of 2024-05-02). Approval rates are FDA’s percentages of evaluable requests, not of all receipts. FY2024–FY2025 device tables were not posted as of 24 August 2026.

Key Operational Insights from the Data

1. Volume Growth: Total CDRH expanded access receipts grew from 428 in FY2019 to 845 in FY2023, representing a 97.4% increase over five years.
2. Dominance of Non-IDE Requests: In FY2023, non-IDE single-patient requests accounted for 87.7% (741 of 845) of all expanded-access submissions to CDRH. This demonstrates that the vast majority of device compassionate use occurs when no formal US IDE is on file, requiring physicians and manufacturers to use the direct CDRH Document Control Center pathway.
3. High Approval Rates for Evaluable Submissions: FDA approval rates for evaluable requests consistently exceed 98% to 100%. This indicates that when clinical teams prepare complete, compliant packets adhering to 21 CFR 812 criteria, CDRH reviewers work expeditiously to grant access.
4. Evaluable denominator, not “file and treat”: Approval rates are calculated on evaluable requests (FDA: a substantive grant/denial review). Combined, 24 of 428 FY2019 receipts (5.6%) and 12 of 845 FY2023 receipts (1.4%) sat outside that denominator. High approval rates also say nothing about whether the manufacturer will ship the device.

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Why ClinicalTrials.gov Under-Counts Medical Device Expanded Access

A frequent question among clinical researchers is why ClinicalTrials.gov lists so few medical device expanded-access records despite hundreds of FDA submissions each year.

The Registration Disparity: Data Analysis (August 2026)

A live query of the National Library of Medicine (NLM) ClinicalTrials.gov API reveals a stark divergence between registered studies and real-world CDRH expanded-access activity:

• Total Expanded Access Records on ClinicalTrials.gov: 1,067 records across all intervention types.
  • Drug Expanded Access Records: 772
  • Biological Expanded Access Records: 168
  • Device Expanded Access Records: Only 81 lifetime records
• Status Breakdown of the 81 Device Records:
  • AVAILABLE (actively accepting patients): 19
  • NO_LONGER_AVAILABLE: 38
  • APPROVED_FOR_MARKETING: 20
  • TEMPORARILY_NOT_AVAILABLE: 4
• Context: In comparison, ClinicalTrials.gov lists 63,581 registered interventional medical device studies.

Why the Gap Exists

1. No Legal Requirement for Single-Patient Access: Under the Food and Drug Administration Amendments Act (FDAAA 801) and 42 CFR Part 11, mandatory clinical trial registration applies to Applicable Clinical Trials (ACTs) of FDA-regulated devices. Single-patient emergency use and individual compassionate use requests do not meet the statutory definition of an Applicable Clinical Trial and are not required to be registered on ClinicalTrials.gov.
2. Protocol vs. Clinical Care: Treatment IDEs (multi-patient programs) are occasionally registered, but single-patient filings (which comprise ~88% of all CDRH volume) are treated as individualized patient-care episodes rather than public research registries.
3. Pharma vs. Device Transparency Mandates: The 21st Century Cures Act mandated expanded-access policy disclosure for investigational drugs treating serious diseases, but this statutory mandate did not impose equivalent public registry listing requirements on investigational medical devices.

These figures are different units of analysis: ClinicalTrials.gov counts registered expanded-access studies, while CDRH counts expanded-access requests. Lifetime DEVICE expanded-access registrations (81 as of 24 August 2026) are far smaller than a single fiscal year of CDRH requests (845 in FY2023) and smaller than the 3,045 combined CDRH receipts in FY2019–FY2023. ClinicalTrials.gov is therefore not a census of US device expanded access.

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European Comparison: EU MDR Article 59 Derogation Is NOT US Expanded Access

Medical device manufacturers operating globally frequently conflate US expanded access with European market access exemptions under Regulation (EU) 2017/745 (EU MDR).

The Legal Distinction

	US 21 CFR Part 812 expanded access	EU MDR Article 59
Legal object	Investigational clinical authorization for unapproved device use in named patient(s) or a treatment cohort	National market-access derogation from CE-marking conformity assessment
Who grants it	FDA (federal)	Individual Member State competent authorities (for example BfArM, ANSM, AEMPS, Infarmed)
Governing rules	IDE / clinical-investigation rules under 21 CFR 812, 50, and 56	National distribution and public-health safety-valve law
Typical focus	Protocol, consent, IRB, and investigational labeling	Public-health interest, patient protection, and lack of an alternative CE-marked device

Under EU MDR Article 59 (National Derogation), a national competent authority may authorize the placing on the market or putting into service of a non-CE-marked medical device within its sovereign territory in the interest of public health or patient safety.

Key operational differences include:

• Territorial Limitation: An Article 59 derogation issued in Germany (by BfArM) applies strictly within German territory and does not permit shipment to France, Italy, or other Member States.
• Article 59(2) Single-Patient Carve-Out: When an Article 59 derogation is granted for an individual patient, the Member State is exempt from notifying the European Commission and other Member States under Article 59(2).
• Not a Clinical Investigation: Article 59 is a market-access exemption, not an authorization to conduct an MDR Article 62–82 clinical investigation.

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Manufacturer Supply, Cost Recovery, and Investigational Labeling

1. FDA Cannot Compel a Manufacturer to Supply a Device

A critical legal and ethical reality of expanded access is that FDA approval of an expanded-access request does not compel or obligate the device manufacturer to provide the device.

Under federal law, the decision to manufacture, release, and ship an investigational device for compassionate or emergency use rests entirely with the device manufacturer. Manufacturers may decline expanded-access requests due to:

• Limited inventory allocated for clinical trials;
• Concerns regarding product liability or off-label complications;
• Resource constraints associated with training and proctoring; or
• Potential adverse event data confounding ongoing pivotal trial reviews.

Treating physicians must secure written confirmation from the manufacturer’s clinical/regulatory department agreeing to supply the device before submitting a non-IDE compassionate use request to FDA.

2. Cost Recovery and Charging Rules (21 CFR 812.7(b))

Under 21 CFR 812.7(b), a sponsor or investigator may not charge a subject for an investigational device a price larger than that necessary to recover costs of manufacture, research, development, and handling.

Commercialization beyond cost recovery is prohibited. Unlike drug charging under 21 CFR 312.8, Part 812 does not create a separate compassionate-use charging-authorization form. If a Treatment IDE device is to be sold, 21 CFR 812.36(c)(1)(x) requires the application to state the price and that the price is based on manufacturing and handling costs only. The 812.7(b) ceiling still applies to emergency and compassionate use.

3. Investigational Labeling Requirements (21 CFR 812.5)

Any investigational device shipped for emergency, compassionate, or treatment IDE use must strictly comply with 21 CFR 812.5 labeling requirements:

• The device label must bear the prominent statement:
  "CAUTION—Investigational device. Limited by Federal (or United States) law to investigational use."
• The labeling must describe all relevant contraindications, hazards, adverse effects, and precautions.
• The labeling must not contain any commercial claims or statements implying the device is safe or effective for the intended use.

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Documentation and Archiving: What Goes in the TMF and ISF

When a sponsor and IDE already exist, expanded-access approvals, 5-day reports, 45-day follow-ups, and device-accountability records belong in the Trial Master File (TMF) and Investigator Site File (ISF). For no-IDE physician-directed emergency or compassionate use, the hospital’s investigational-use / IRB file is the primary record set; there is not always a sponsor TMF to populate. ISO 14155 does not itself create a US expanded-access pathway.

Sponsor TMF (when a sponsor/IDE exists)	Investigator Site File / hospital investigational-use file
FDA IDE supplement or non-IDE request and FDA correspondence	Signed informed consent (original)
Redacted case summary and 45-day follow-up	IRB approval, chair concurrence, or 5-working-day emergency notice
Independent-physician concurrence (copy)	Independent-physician concurrence (original)
Shipment manifests and device reconciliation	Device accountability and implant/explant log
Safety / UADE reports (de-identified as appropriate)	Unredacted medical records

Privacy firewall. Original signed consent forms and identifiable medical records stay in the hospital file or ISF. The sponsor TMF, when one exists, should receive only redacted clinical summaries and safety dossiers.

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Step-by-Step Checklists: Emergency and Compassionate Use

Checklist A: Emergency Use Workflow (No Prior FDA Approval)

1. Clinical assessment. Confirm an immediately life-threatening condition, no acceptable approved alternative, and no time to obtain prior FDA approval.
2. Independent concurrence. Obtain a written assessment from a physician who is not participating in the case.
3. Consent. Obtain 21 CFR Part 50 consent from the patient or legally authorized representative, or document a 21 CFR 50.23 emergency exception.
4. Institutional and manufacturer clearance. Notify the IRB chair or office if time permits; confirm the manufacturer will release the device.
5. Use the device. Maintain lot/serial accountability.
6. Five working-day reports. Report to the IRB under 21 CFR 56.104(c). Report to FDA through the IDE sponsor if an IDE exists, or directly to CDRH DCC if it does not.
7. Follow-up. Monitor for UADEs under 21 CFR 812.150 and file records in the ISF or hospital investigational-use file.

Checklist B: Compassionate Use Workflow (Prior FDA Approval Required)

1. Screening. Confirm a serious or life-threatening condition, trial ineligibility, and no satisfactory approved alternative.
2. Manufacturer commitment. Obtain written agreement to supply the investigational unit before filing.
3. Packet. Assemble clinical history, treatment plan, risk-benefit rationale, device description, draft consent, and independent-physician letter.
4. FDA filing. IDE on file: sponsor IDE supplement under 21 CFR 812.35(a). No IDE: physician or manufacturer request to CDRH DCC (CDRHExpandedAccess@fda.hhs.gov).
5. Approvals. Wait for the FDA letter (CDRH average about 15 days; sometimes one day). Obtain IRB approval or written chair concurrence per local SOP.
6. Consent and treatment. Execute 21 CFR Part 50 consent; ship with 21 CFR 812.5 investigational labeling.
7. Follow-up. Submit the follow-up report to FDA within 45 days of device use, and report problems to the IRB as soon as possible.

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Frequently Asked Questions (FAQs)

Is prior FDA approval required before emergency use of an unapproved device?

No. For a true emergency in an individual patient confronting an immediately life-threatening situation where treatment cannot wait for FDA review, prior FDA approval is not required. However, the treating physician must obtain independent physician concurrence, obtain informed consent (or execute a 21 CFR 50.23 emergency exception), secure manufacturer supply, and submit written follow-up reports to both the IRB and FDA within 5 working days of device use.

What is the difference between compassionate use and a Treatment IDE?

Compassionate use is designed for an individual patient or small group of identified patients requiring treatment outside an active clinical trial, requiring case-by-case FDA approval (via an IDE supplement or CDRH submission). A Treatment IDE under 21 CFR 812.36 is a multi-patient expanded-access protocol that makes an investigational device available to a broader patient cohort across multiple clinical sites while pivotal clinical trials are ongoing or completed and marketing approval is actively pursued.

Can I use Form FDA 3926 for a medical device expanded-access request?

No. Form FDA 3926 is strictly designated for individual-patient expanded access to investigational drugs and biologics (INDs). FDA guidance explicitly states that Form FDA 3926 does not apply to medical devices. Device requests must be submitted as IDE supplements under 21 CFR 812.35(a) or formal CDRH DCC expanded-access packets.

Does the federal Right to Try Act apply to investigational medical devices?

No. The federal Right to Try Act (21 U.S.C. § 360bbb-0a) applies exclusively to eligible investigational drugs that have completed Phase 1 clinical testing. It contains no statutory provisions for medical devices. All compassionate and emergency access to investigational medical devices must proceed under 21 CFR Part 812.

How fast does CDRH review compassionate-use IDE supplements?

While statutory review cycles for standard IDE submissions allow up to 30 days, CDRH prioritizes compassionate-use requests. FDA states that compassionate-use requests are reviewed on average within 15 days of receipt, and in some cases in as little as one day.

What must be reported to the IRB after emergency use under 21 CFR 56.104(c)?

The treating physician must submit a written report to the IRB within 5 working days following the emergency use. The report must detail the patient's condition, clinical justification, outcome, copy of the signed informed consent (or 50.23 emergency consent exception), and independent physician concurrence. Under 21 CFR 56.104(c), any subsequent use of the investigational device at the institution requires prior IRB review.

Can FDA force a manufacturer to provide an investigational device for compassionate use?

No. FDA cannot compel or mandate a medical device manufacturer to provide an investigational device. The manufacturer must voluntarily agree to manufacture, release, and ship the product. If a manufacturer declines access due to supply constraints or liability concerns, the expanded-access request cannot proceed.

Is EU MDR Article 59 the European equivalent of US compassionate use?

No. EU MDR Article 59 is a national market derogation that allows an individual EU Member State competent authority (such as BfArM in Germany or ANSM in France) to authorize the placing on the market of a non-CE-marked device in the interest of public health or patient safety. It is a territorial market-access safety valve governed by national law, not a US-style clinical IDE protocol supplement.

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Summary and Key Takeaways

1. Three Distinct US Mechanisms: Choose Emergency Use for immediately life-threatening crises where treatment cannot wait (5-day post-use reports to FDA and IRB); choose Compassionate Use for individual patients or small groups when time permits FDA review (~15-day average CDRH turnaround; 45-day follow-up); choose Treatment IDE (21 CFR 812.36) for widespread cohort access during premarket development.
2. Avoid the Drug Traps: Do not file Form FDA 3926, do not cite the Right to Try Act, and do not confuse expanded access with public health Emergency Use Authorizations (EUAs) or Humanitarian Device Exemptions (HDEs).
3. Data Realities: CDRH expanded access volume has grown significantly, reaching 845 requests in FY2023, with 87.7% filed through the non-IDE single-patient pathway. Approval rates for complete, evaluable files exceed 98%.
4. ClinicalTrials.gov Under-Registration: Only 81 lifetime device expanded-access records exist on ClinicalTrials.gov because single-patient compassionate access is exempt from mandatory public trial registration.
5. Rigorous Compliance Governance: Maintain strict 21 CFR 812.5 investigational labeling, enforce 21 CFR 812.7(b) cost-recovery ceilings, comply with 21 CFR 50 informed consent (or 50.23 emergency exceptions), and maintain an airtight privacy firewall between the Sponsor TMF and Investigator Site File.