Introduction to Expanded Access Programs

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Expanded Access, often also referred to as Compassionate Use, describes the route governed by US FDA, Health Canada, and other country specific Health Authority regulations by which patients with an immediately life-threatening or serious disease or condition, may access a treatment with an investigational, unapproved or uncleared drug, biologic or medical device. 

In the US, there are three types of expanded access IND applications, each dependent upon the size of the population intended to be treated:

1. Single patient emergency or non-emergency use. 

2. Intermediate-sized patient populations.

3. Large patient population under a treatment IND.

Several criteria must be met for a patient to be eligible and receive treatment with an investigational drug under an expanded access program:

1. There are no comparable or satisfactory alternative therapies available.
2. The potential benefit justifies the potential risks of treatment, and those risks are not unreasonable in the context of the disease or condition as a whole, 
3. Patient treatment through expanded access will not interfere with the initiation, conduct or completion of ongoing clinical investigations directed towards market approval, or interfere in other ways in the development program; 
4. Submission of an application, expanded access submission, as a new or existing active IND with a protocol and treatment plan, to Country Health Authority for the specific use, and authorization to proceed with treatment; and,
5. Approval from the institutional review board (IRB).

 As an investigational drug enters phase 2 and 3 clinical development with promising early data supporting potential beneficial effects for patients, interest in the investigational drug grows from both the physician and patient. 

Develop an EA policy - make it publicly available

Plan early for an EA program - it takes time to develop a good program

Consider: 

• Drug supply, on-going clinical trials, country requirements, 
• Start-up and close-out plans
• Workflows to process requests seamlessly
• Collection of Real World Data
• Overall value to the development program

• US FDA Report on Expanded Access (2018)

• Overview of FDA’s Expanded Access Program for Investigational Drugs

• US FDA Expanded Access Programs (US FDA)

• Expanded Access to Investigational Drugs for Treatment Use Questions and Answers Guidance for Industry

• Health Canada's Special Access Programs: Overview 

• Requesting a drug under Special Access (Health Canada)

• Reagan Udall Foundation 

• MedaSystems Cloud Platform for Expanded and Managed Access Programs