What to Expect with Expanded Access
An investigational product (IP) is a drug, biologic, or medical device that is currently being studied as a potential treatment but is not approved by the Food and Drug Administration (FDA). Clinical trials are the only way to formally evaluate the efficacy of investigational products and discover new treatments for people living with ALS. However, not all patients are able to participate in a clinical trial. This is when Expanded Access (EA) can offer a potential pathway for the purpose of accessing an investigational product.
What is Expanded Access?
Expanded Access (EA), also referred to as Compassionate Use, is a pathway for patients with a serious and life-threatening disease to access an investigational product (IP) that is not yet approved by the FDA. EA is an option for patients who do not qualify to participate in a clinical trial. An IP offered through EA is experimental, so its effectiveness as a treatment for ALS is not yet known. At this time, EA programs tend to be smaller programs with limited availability. To read more about EA, visit the FDA's website.
How is Expanded Access Different from Clinical Trials?
The purpose of clinical trials is to formally evaluate investigational products (IPs). Clinical trials are designed to gather data about IPs that can lead to FDA approval if the data generated from the trial supports the safety and efficacy of the IP. Clinical trials have specific eligibility criteria, frequent in-person study visits, and typically involve a chance of being on placebo. Clinical trials are separate from clinical care.
On the other hand, the purpose of Expanded Access (EA) is to provide access to an investigational product. EA 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 participation in a clinical trial. The criteria for participation in an EAP are broad and inclusive, in-person visits may be infrequent, and there is no placebo. EA is considered an extension of a patient’s clinical care.
How do I learn more about participating in an Expanded Access Protocol?
Any licensed medical doctor can approach a pharmaceutical company to request an Expanded Access Protocol (EAP) on behalf of his/her patient. There are a range of factors, such as financial resources and staff support, that determine whether a doctor or institution can offer an EAP. In addition, pharmaceutical companies may or may not have enough investigational product (IP) available for EAPs, or companies may have policies preventing them from offering IP for an EAP. The best way to learn about EAP opportunities is to have a discussion with your ALS doctor and clinical care team.
Why would I participate in an Expanded Access Protocol?
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 research by providing safety and biomarker data that may benefit the ALS community. 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.
How are Expanded Access Protocols funded?
Expanded Access Protocols (EAPs) are typically funded by multiple sources, including contributions from philanthropy and fundraising. The costs associated with EAPs may include the investigational product itself and the administration and storage of the product. EA funding also supports the staff (clinicians, nurses, and research coordinators) responsible for clinical monitoring of the EAP participant through in-person appointments and virtual visits. It is always best to talk to your clinical care team about the specific costs that may be associated with an EAP.
Learn More About EAPs
- Watch an educational webinar about EAPs hosted by the Northeast ALS Consortium (NEALS)
- Visit the NEALS website
- Visit the US FDA website
- Read the scientific journal article Accessing Investigational Products Outside of a Trial: Considerations for Neuromuscular Providers
Key to Acronyms/Abbreviations
- ALS: Amyotrophic Lateral Sclerosis
- EA: Expanded Access
- EAP: Expanded Access Protocol
- FDA: Food and Drug Administration
- IP: Investigational Product
- NEALS: Northeast ALS Consortium
Transforming Medicine Through Innovation
Expanded Access Protocol programs at the Sean M. Healey and AMG Center for ALS are funded by multiple sources, including contributions from donors like you. Thank you for partnering with us in the quest to discover life-saving therapies for individuals affected by ALS.