Expanded Access Programs

Strategic Planning - Clinical Operations - Expanded Access Programs

Expanded Access Programs and How they Work

What is Expanded Access?

Types of Expanded Access (US FDA)

Expanded Access — also referred to as Compassionate Use, Special Access, or Early Access depending on the country — is a regulatory pathway that allows patients with a serious or immediately life-threatening disease or condition to access investigational drugs, biologics, or medical devices that have not yet received full regulatory approval.

This pathway exists because drug development is a lengthy process. Clinical trials, while essential to establishing safety and efficacy, cannot accommodate every patient who might benefit from a promising new therapy. Expanded Access bridges that gap — providing a structured, regulatory-compliant route for sponsors and physicians to make investigational treatments available to patients outside of a clinical trial setting.

Regulatory oversight varies by country. In the United States, Expanded Access is governed by the US Food and Drug Administration (FDA). In Canada, the equivalent pathway is administered through Health Canada's Special Access Program (SAP). The European Medicines Agency (EMA) and individual member states oversee similar frameworks across Europe, often referred to as Compassionate Use or Named Patient Programs.

Types of Expanded Access (US FDA)

In the United States, the FDA recognises three categories of Expanded Access IND applications, each designed for a different scale of use:

1. Individual Patient — Emergency UseFor a single patient facing an immediately life-threatening condition where there is no time to submit a standard IND application. The FDA can authorise treatment by telephone, with a written submission to follow within 15 days. This is the fastest route available.

2. Individual Patient — Non-Emergency UseFor a single patient who does not meet the criteria for emergency use but has no satisfactory alternative treatment options. A written protocol and treatment plan must be submitted to the FDA and reviewed by an Institutional Review Board (IRB) before treatment can begin.

3. Intermediate-Size Patient PopulationFor a small group of patients — typically more than one but fewer than the number that would be enrolled in a large treatment IND. This is often used when a drug is being actively studied but a formal treatment IND is not yet warranted.

4. Large Population — Treatment IND or Treatment ProtocolFor broader access, typically when a drug is in late-stage development and there is sufficient evidence of safety and efficacy. This type may also be used to facilitate commercial-scale access ahead of formal approval.

Patient Eligibility Criteria

Types of Expanded Access (US FDA)

Expanded Access Around the World

For a patient to receive treatment under an Expanded Access Program, the following criteria must generally be met:

1. No comparable alternatives exist.The patient must have a serious or life-threatening condition for which no comparable or satisfactory approved therapies are available, or for which existing treatments have failed.

2. The benefit justifies the risk.The potential therapeutic benefit must outweigh the known and potential risks of the investigational treatment, taking into account the severity of the condition and the patient's overall clinical situation.

3. The program will not compromise ongoing trials. Access to the investigational drug must not interfere with the sponsor's clinical development program, including the initiation, conduct, or completion of ongoing clinical trials required for market approval.

4. A regulatory submission has been made and authorised.The sponsor must submit an Expanded Access application — as a new or amendment to an existing IND — including a protocol and treatment plan, to the relevant Health Authority (FDA, Health Canada, EMA, etc.), and receive authorisation to proceed.

5. IRB approval has been obtained.In the US, approval from an Institutional Review Board (IRB) is required before treatment can begin. Equivalent ethics committee review is required in other jurisdictions.

Expanded Access Around the World

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Expanded Access Around the World

Regulatory frameworks for Expanded Access vary significantly by country. Understanding the relevant pathway — and its specific requirements — is essential before a program is launched.

United States — FDA Expanded Access. Governed under 21 CFR Part 312 (drugs and biologics) and 21 CFR Part 812 (devices). The FDA maintains a public database of available EA programs and has published extensive guidance for both sponsors and physicians.

Canada — Health Canada Special Access Program (SAP). Canada's SAP allows physicians to request access to drugs not yet approved for sale in Canada for patients with serious or life-threatening conditions. Requests are made by the treating physician directly to Health Canada, with the manufacturer's cooperation required.

European Union — Compassionate Use & Named Patient Programs. The EMA provides a framework for compassionate use of medicines that are under evaluation or authorized in other jurisdictions. Individual EU member states also operate their own Named Patient Program (NPP) frameworks, which vary considerably in process and timelines.

Other Jurisdictions Australia (TGA Special Access Scheme), Japan (MHLW), Brazil (ANVISA), and other regulatory bodies each operate distinct frameworks. Multi-country EA programs require jurisdiction-specific regulatory expertise.

Useful Reference Material - Weblinks

Ready to discuss your Expanded Access Program?

US FDA Expanded Access Programs — Overview

Expanded Access to Investigational Drugs for Treatment Use — FDA Guidance for Industry

US FDA Report on the State of Expanded Access (2018)

Overview of FDA's Expanded Access Program for Investigational Drugs — Therapeutic Innovation & Regulatory Science

Health Canada Special Access Program — Overview

Requesting a Drug Under Special Access — Health Canada

Reagan Udall Foundation — Expanded Access Navigator

MedaSystems — Cloud Platform for Expanded and Managed Access Programs

Ready to discuss your Expanded Access Program?

Whether you are considering launching an EA program for the first time, managing an existing program that has grown beyond your current capacity, or navigating a complex multi-country access situation, Langlands & Associates can help.

We bring hands-on experience across the full EA program lifecycle — from policy development and regulatory submission through to day-to-day operational management and real-world data collection.