What is an expanded access program?
Expanded access programs (EAPs), sometimes known as “compassionate use,” are a potential pathway for people with a serious or immediate life-threatening disease or condition to help patients with serious diseases or conditions get access to medication.
EAPs provide access to an investigational medical product (e.g., drug, biologic, or device) that is not yet approved by the Food and Drug Administration (FDA) for treatment. They are intended for people (individuals or groups) who have no other available treatment options when there is a lack of FDA-approved treatment options and they are not eligible for current clinical trials.
Within an EAP, there is no placebo group. Everyone gets access to the drug.
Expanded Access Programs are funded and operated by pharmaceutical/ biotechnology companies. Funding for an EAP is traditionally provided by the sponsor of the investigational drug or treatment which generally is a biotech/pharmaceutical company. They provide the treatment at no cost to the patient and sometimes cover some of the program expenses. Often, there can be administrative costs that are the responsibility of the patient. Providing an EAP is at the discretion of the investigational drug sponsor/ pharmaceutical company.
In 2021, Congress passed bipartisan legislation, the ACT for ALS that created the first government funded expanded access programs for ALS. We will dive into this program more in our next update of this webpage.
For more on Expanded Access, watch this incredible video presentation by I AM ALS’ Clinical Trials Team:
For a PDF summary of the above information, click here.
How do I know if I qualify for an EAP or a Clinical Trial? And how do I enroll in one?
To find clinical trials and EAPs, please review these resources with your neurologists:
ALS TDI Trial Navigator
A clinical trial navigator tool from the ALS Therapy Development Institute, the world’s largest drug discovery lab focused on ending ALS.
ALS Signal
Click here to explore I AM ALS’s Clinical Research Dashboard, a resource for people living with ALS to learn more about clinical trials and other therapies.
Synapticure
Connect with a neurologist through Synapticure’s telehealth program to discuss clinical trials and EAPs that may be right for you
More EAPS, not less!
There are now several ALS EAPs in the United States. This has not always been the case. Thanks to…
- the hard work of I AM ALS’ Legislative Team passing ACT for ALS
- the Clinical Trial team advocating for more EAPs in Clinical Trials using the PaCTD rating system
- and countless other advocates,
…the ALS community has more EAPs than ever before. But the continued growth and investment in ALS EAPs is not guaranteed.
Let’s Take Action!
ACT for ALS, signed into law in 2021, needs to be reauthorized every five years. This means that, starting this year, the ALS community has to once again show its power and tell Congress to pass and fund ACT for ALS in 2026.
While it’s still 2025, we need Congress to know that ACT for ALS is important to us.
You can help by contacting your senators and representative and telling them how much you appreciate Congress. (We promise this helps! They’re so rarely thanked!)
Send your Elected Officials a Thank-You Tweet
Thank your elected officials who supported ACT for ALS with a pre-drafted post on Twitter/X. Entering your zip code will automatically populate and tag your elected officials. Easy peasy!
Send your Elected Officials a Thank-You Email
Thank your elected officials who supported ACT for ALS with a pre-drafted email. Entering your zip code will automatically populate email addresses for your elected officials. Easy as 1, 2, 3!
Share Your EAP Experience
If you’ve participated in an expanded access program, we’d like to know more. Please share your story with us. Your words and experience will help stregthen the case for support of ACT for ALS!