Expanded Access Programs (EAPs) are a compelling commercialization strategy due to the many benefits they offer prescribers and patients. An EAP program is typically offered before the commercial launch of a new drug, once clinical trials have concluded and the NDA has been filed.
In the U.S., EAPs allow manufacturers to support patients who have life-threatening diseases with no other satisfactory treatment options available. They provide an ethical, compliant, and controlled mechanism of access to investigational drugs, at no charge. The potential benefit to the patient taking the investigational drug must outweigh the risk of taking the medication prior to regulatory approval.
For manufacturers, the benefits of a U.S. EAP Program include:
Today, more and more manufacturers are actively working to establish strategies for converting U.S. EAP patients to commercial patients once their product is approved. There are many good options for vendor support in these efforts, making the EAP investment a no-brainer for companies focused on rare and orphan disease products.
Before establishing an EAP, it’s vital to align internally around several crucial program decisions. These five are key:
1. Dates. When will your program start and how long will it run? Will all patients have access for the duration of the program, or will there be time limits? Will the program end immediately after approval and/or launch? Keep in mind that vendors will need at least 6-9 months to build, staff-up, and train for your EAP, making it essential to start the vendor selection up to 12 months prior to your desired program launch.
2. Population. Which patients will be given access via your EAP? All patients with the condition? Just those who were previously participating in the clinical trial? Perhaps they need to be treated by certain physicians or medical centers? The inclusion/exclusion criteria should be mapped prior to program design because it will influence many factors.
3. IRB/Regulatory Approval. FDA mandates some IRB oversite of all EAPs. How will your organization manage? Typically, manufacturers work with CROs to manage this vital requirement, but other options are available.
4. Data. If patient-level data is important to your strategy, planning to obtain consent is a vital step of program design. Being fully aligned on which data points to capture will help the program build move more smoothly.
5. Enrollment. How will you communicate EAP availability to patients and providers? Do you need vendor support in building a web portal or establishing other lines of communication?
If your commercialization strategy includes an EAP, it’s important to consider these factors in your planning. Archbow Consulting can help you develop the right expanded access approach for your unique product and patient population, laying the groundwork for a successful launch. Contact us today to get started.