
Who Can Participate in an EAP?
To be considered for expanded access, you must:
-
Have a life-threatening illness, like ALS.
-
Have no other comparable treatment options available.
-
Not be able to participate in a clinical trial. This could be because there are no ongoing trials, you do not have access to a trial site, or you do not meet trial eligibility criteria, among other reasons.
In addition, the therapy’s potential benefits must be determined to outweigh any potential risks.
What Kinds of EAPs Are There?
FDA regulations describe three categories of EAPs based on the number of participants:
-
Expanded access for individuals, including for emergency use.
-
Expanded access for intermediate-size patient populations, which are considered more than one person but fewer than the number of people who would be involved in a typical clinical trial.
-
Expanded access for widespread treatment use, which could involve hundreds of people and is usually reserved for drugs that are being tested in phase 3 clinical trials or have completed a phase 3 trial but are not yet FDA-approved.
Are EAPs the Same as “Right to Try”?
Right to Try is a separate pathway available to people with life-threatening diseases who are unable to participate in clinical trials. Under Right to Try, people living with ALS and their doctor(s) work directly with the company that is developing the experimental therapy to get access, without involving the FDA in the process. Because the FDA is not involved, companies may be less likely to approve Right to Try requests. In contrast, the FDA authorizes nearly all of the Expanded Access requests it receives.
How Do I Find EAPs for ALS?
The best way to learn about EAP opportunities is to have a discussion with your doctor or another member of your care team. Our partners, myTomorrows and the Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS), also provide lists of active EAPs on their websites. (On the NEALS site, you will need to scroll all the way to the bottom of the page.)
How Do I Join an EAP?
People living with ALS are not able to apply for an EAP themselves—only a licensed physician can. The first step is to find a doctor who is willing to do this for you. Once identified, your doctor will need to get four different approvals before you can receive the experimental therapy.
- Company Approval. Your doctor will reach out to the company that manufactures the experimental therapy (or the company sponsoring the clinical trial) to request access through an EAP. The company will review the request, ask questions, and then decide whether to provide the experimental therapy. Companies have no legal obligation to grant the request. The decision to provide access to an experimental therapy through an EAP is solely at the company’s discretion.
- IRB Approval. Your doctor will present the expanded access plan to an Institutional Review Board or IRB, which is an independent group of people who review and monitor biomedical research to ensure the welfare, rights, and privacy of research participants.
- FDA Approval. The FDA reviews and decides if the expanded access request can proceed. The agency has up to 30 days to review EAP applications but often reviews them much more quickly.
- Your Approval. This is also known as informed consent. You will be asked to carefully read an informed consent form, ask questions, and make sure you understand the potential risks associated with the experimental therapy before signing. After you have signed this form, you can still choose to stop treatment at any time.
When deciding whether to approve an EAP application, each individual and organization in this process considers if:
-
There are any other treatment options available.
-
The potential benefits of the experimental therapy outweigh the risks.
-
Providing access to the experimental therapy will interfere with any clinical trials.
Do I Have to Pay to Participate in an EAP?
The costs associated with EAPs, such as the cost of the experimental therapy and any associated medical services, are often not covered by health insurance, including Medicare. People who participate in these programs may have to pay out of their own pocket. In some cases, the pharmaceutical company running the EAP may cover the cost of the experimental therapy. Your doctor or another member of your care team can help answer any questions you have about the cost of participating in an EAP.
Are EAPs Research Studies?
Although IRB approval is required and data about safety and biomarkers may be collected, EAPs are not the same clinical research studies. The primary intent of an EAP is to provide treatment access and may involve differing treatment protocols and dosing regimens, while the primary intent of a clinical trial is to generate evidence about the treatment's efficacy and safety profile using standardized protocols, dosing, and endpoints.
How Does the ACT for ALS Support EAPs?
Thanks to the hard work of ALS advocates, the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS) was signed into law by President Biden in December 2021. This law contains provisions intended to make it easier for companies with experimental ALS treatments to conduct EAPs. The National Institutes of Health (NIH) now offers grants to companies and clinical trial sites to support EAPs for ALS drugs in phase 3 clinical trials. The first of these grants was awarded in 2023.
Where Can I Learn More About EAPs?
You can learn more about EAPs in this episode of the Connecting ALS Podcast, which features Dr. Alison Bateman-House, a medical ethicist and member of NYU Langone’s Working Group on Compassionate Use and Preapproval Access. The FDA and NEALS also provide information about EAPs on their websites.