Expanded Access Questions & Answers

The purpose of expanded access is to provide access to an investigational product (IP). Expanded Access Protocols (EAPs) should not interfere with the completion of clinical trials; therefore, EAPs are meant to be an option for patients who do not qualify for clinical trials. The criteria for participation in an EAP are broad and inclusive, in-person visits may be infrequent, and there is no placebo. Expanded access is considered an extension of clinical care. Visit our webpage to explore available EAP opportunities or use the search tool on ClinicalTrials.gov.

On the other hand, the purpose of clinical trials is to formally test investigational products (IPs). Data gathered from clinical trials may lead to FDA approval of new treatments if an IP is found to be safe and effective. Clinical trials have specific eligibility criteria, frequent in-person study visits, and typically involve a placebo group. Clinical trials are separate from clinical care. View clinical trial opportunities at the Healey & AMG Center for ALS.

EAP stands for Expanded Access Protocol, which is an opportunity for people with life-threatening conditions or serious diseases who are not eligible for clinical trials to gain access to an investigational product that has been shown to be safe and tolerable in early clinical trials. Investigational products offered through EAPs are currently being studied in clinical trials and are not yet approved by the FDA. To read more about expanded access, visit the FDA website.  

Eligibility criteria will differ from one EAP to another depending on the unique properties of the investigational product being offered. To find out if you may be eligible to participate in an EAP, please talk to your ALS doctor or contact a research center that is offering an EAP of interest to you. Visit our webpage to explore available EAP opportunities or use the search tool on ClinicalTrials.gov

Expanded Access Protocols (EAPs) give people living with ALS who are not eligible for clinical trials the opportunity to take an investigational product (IP) while the IP is being formally tested. Participation in an EAP may also contribute to ALS research by providing safety and biomarker data. If you are interested in participating in an EAP, it is helpful to have a conversation with your ALS doctor to discuss the potential risks and benefits.

Expanded Access Protocols (EAPs) are typically funded by multiple sources, including contributions from grants, industry partners, philanthropy, and fundraising. The costs associated with an EAP include the supply, administration, and storage of the investigational product. Funding also supports the staff (clinicians, nurses, and research coordinators) who oversee clinical monitoring, in-person appointments, and virtual visits for each EAP participant. For specific information about patient care costs associated with an EAP, please talk to your clinical care team.

The best way to learn about Expanded Access Protocol (EAP) opportunities is to have a discussion with your ALS doctor and clinical care team. Your care team may be offering an EAP or may be able to provide you with information about multicenter EAPs near you.

It may be possible for a licensed medical doctor to approach a pharmaceutical company to request an EAP on behalf of his/her patient. FDA approval and factors such as financial and staffing resources may determine whether a doctor can offer an EAP at your care center. Additionally, a pharmaceutical company must be able to supply the investigational product (IP) requested for an EAP.”