Compassionate Use is a regulatory pathway that lets a patient with a serious or life-threatening condition access an investigational medical device outside a clinical trial when no satisfactory approved alternative exists. In the US, it is a form of FDA expanded access; in the EU, it relies on national derogations under EU MDR Article 59.
What is compassionate use?
Compassionate use sits in the late investigational stage of the device lifecycle, after a device has entered clinical study but before it has FDA clearance or approval, or a CE mark. It is one branch of a wider set of early-access mechanisms that regulators allow for products still under evaluation.
In the US, compassionate use is a category of expanded access under the Investigational Device Exemption (IDE) regulations. It covers individual patients or small groups who do not qualify for an ongoing trial yet have no comparable option. The EU has no central program of this kind for devices. Instead, a national competent authority can authorize a specific non-CE-marked device under Article 59 of EU MDR 2017/745 when public health or patient safety justifies it.
Why compassionate use matters in medical device development
For a manufacturer, a compassionate use request is rarely just a goodwill gesture. It creates real regulatory and quality obligations. The device is still investigational, so its safety and performance are not established, and any use generates data that regulators and review boards will scrutinize.
Several stakes stack up. Patient safety comes first: the requesting physician and the sponsor share responsibility for informed consent and patient protection. Compliance exposure is next, because each use must follow the applicable regulation, with follow-up reporting on outcomes, including any adverse device effects. There is also a strategic dimension. Early use can surface usability or design issues before a formal clinical trial, but poorly documented access can complicate a later submission. Teams that treat these requests as one-off favors, rather than controlled events under the quality system, tend to pay for it during audits.
How compassionate use works
The mechanics differ by jurisdiction, but the trigger is the same: a serious or immediately life-threatening condition, no satisfactory alternative, and a physician willing to take responsibility for the patient.
In the US, the FDA recognizes three forms of device expanded access:
- Emergency use, when there is no time to obtain prior FDA approval. The physician acts first, then submits a follow-up report, generally within five working days, under 21 CFR 812.150.
- Compassionate use, for a single patient or a small group. If an IDE already exists, the sponsor files an IDE supplement under 21 CFR 812.35(a). The FDA typically reviews the request within about 30 days before treatment begins.
- Treatment use, for wider access to patients outside a trial while the device moves toward market, is governed by 21 CFR 812.36 with periodic progress reports.
All three require concurrence from an Institutional Review Board (IRB), informed consent under 21 CFR Part 50, and adherence to the IDE responsibilities in 21 CFR Part 812. The device still operates within its established risk controls under ISO 14971 and, where applicable, ISO 13485 process discipline.
In the EU, there is no equivalent federal program. Article 59 of EU MDR 2017/745 lets a member state’s competent authority authorize a specific device that has not completed conformity assessment, limited to that country. Single-patient authorizations do not have to be notified to the European Commission. Related routes include custom-made devices and in-house devices manufactured and used within a single health institution.
Common challenges and best practices
The recurring mistake is handling compassionate use outside the quality system. A request arrives, a clinician is under pressure, and the device ships without the documentation a regulator will later expect. Good practice ties every request back to controlled records.
A few patterns separate teams that manage this well:
- Define a written procedure ahead of demand, so the regulatory, clinical, and quality steps are not improvised case by case.
- Confirm eligibility honestly. The pathway is for serious conditions with no satisfactory alternative, not a shortcut around a trial or a marketing route.
- Keep risk files current. Each use should map to the device risk management file, with residual risks disclosed in consent.
- Plan the follow-up. Outcome and adverse-event reporting is mandatory, and the data may feed a future submission, so capture it cleanly.
- Coordinate early with the IRB or competent authority. Review windows like the FDA’s are fixed and cannot be compressed by urgency alone.
Frequently Asked Questions
Not exactly. Expanded access is the FDA’s umbrella term for using an investigational product outside a trial. Compassionate use is one category within it, aimed at individual patients or small groups with serious conditions and no satisfactory alternative. The other device categories are emergency use and treatment use. People often use the two terms interchangeably, but compassionate use is the narrower one.
There is no central EU compassionate use program for devices, unlike the system for medicines. Instead, Article 59 of EU MDR 2017/745 lets a national competent authority authorize a specific non-CE-marked device when public health or patient safety requires it. Each authorization is country-specific, and single-patient cases do not need to be reported to the European Commission. Custom-made and in-house device routes can also apply.
In the US, a licensed physician initiates the request, usually working with the device sponsor or manufacturer that holds the Investigational Device Exemption. The physician takes on investigator duties, including informed consent and reporting. Institutional Review Board concurrence and, in most cases, prior FDA authorization are required before the device is used, except in genuine emergencies where action precedes formal approval.
It can contribute, but it is not a substitute for a controlled clinical investigation. Expanded access is meant to treat patients, not to prove safety and effectiveness, so the data is observational and uncontrolled. Regulators may still review it, and adverse-event reports are required. Well-documented outcomes can add supporting evidence to a marketing submission, though definitive evidence normally comes from a dedicated trial.
Related Terms
- Investigational Device Exemption (IDE)
- Clinical Investigation
- Adverse Event
- ISO 14155
- EU MDR 2017/745
