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Many people living with ALS who want to participate in a clinical trial find out they are ineligible due to strict inclusion and exclusion criteria. Some clinical trials only enroll people who have had ALS symptoms for 18-24 months or less.
The FDA established Expanded Access Programs (EAPs) to provide a potential pathway for people with a serious disease to try an experimental drug if they do not qualify for a clinical trial and if there are no other satisfactory treatment options. |
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Expanded Access Programs can provide additional access to experimental drugs for people living with ALS. However, EAP opportunities are very limited, there can be multiple barriers to participation, and there is no guarantee that the experimental drugs will be effective.
There are two distinct categories of EAPs for people living with ALS:
- Traditional EAPs are extremely rare. They involve a multi-step application process, requests are often denied, and the cost of the experimental drug and related medical care is not typically covered by insurance.
- ACT for ALS EAPs are greater in number though still limited. Multiple sites administer EAPs, the cost of the drug and related visits are typically covered, and research data is collected.
Understanding the Terms
- Expanded Access Program – An FDA-regulated pathway for a person with a serious illness to receive an experimental drug outside of a clinical trial
- Compassionate Use – Another term for Expanded Access
- Right to Try – A separate pathway that allows a single patient and their doctor to request access to an experimental drug directly from the manufacturer. In practice, manufacturers rarely provide access through this pathway.
Traditional EAPs
If you do not qualify for a clinical trial and have no other treatment options, you can apply for a single-patient EAP to access the experimental drug that is being tested in that trial. The process can be lengthy, and few people living with ALS have been successful going this route.
First, you need to ask your doctor—ideally your neurologist at a multidisciplinary ALS clinic—to help you apply. Next, the trial sponsor (often a pharmaceutical or biotech company) needs to agree to provide the experimental drug. Then, approval from the FDA and an Institutional Review Board (IRB) are needed.
There can be roadblocks at any step of the process. Not all doctors have experience with EAPs, and some may not have the time to do all the paperwork involved with individual applications. Sponsors are not obligated to say yes, and they often say no for a variety of reasons, including risk and cost.
If a trial sponsor does agree, they can choose to charge full price for the drug, offer a discount, or provide the drug at no cost. Private insurance and Medicare don’t usually cover traditional EAPs, so you would likely need to pay for the drug, related medical visits, and associated travel costs.
If your application gets approved, your doctor will receive the experimental drug, possibly administer it, monitor your safety, and report any adverse health events. With traditional EAPs, little to no research data is collected.
If you want to apply for access to an experimental drug under a traditional EAP, keep in mind that a drug in Phase 3 of a clinical trial will have more safety data and efficacy data than drugs still in Phase 1 or Phase 2.
To learn more about the steps you would need to take, this Expanded Access Information for Patients page from the FDA goes into greater detail.
First, you need to ask your doctor—ideally your neurologist at a multidisciplinary ALS clinic—to help you apply. Next, the trial sponsor (often a pharmaceutical or biotech company) needs to agree to provide the experimental drug. Then, approval from the FDA and an Institutional Review Board (IRB) are needed.
There can be roadblocks at any step of the process. Not all doctors have experience with EAPs, and some may not have the time to do all the paperwork involved with individual applications. Sponsors are not obligated to say yes, and they often say no for a variety of reasons, including risk and cost.
If a trial sponsor does agree, they can choose to charge full price for the drug, offer a discount, or provide the drug at no cost. Private insurance and Medicare don’t usually cover traditional EAPs, so you would likely need to pay for the drug, related medical visits, and associated travel costs.
If your application gets approved, your doctor will receive the experimental drug, possibly administer it, monitor your safety, and report any adverse health events. With traditional EAPs, little to no research data is collected.
If you want to apply for access to an experimental drug under a traditional EAP, keep in mind that a drug in Phase 3 of a clinical trial will have more safety data and efficacy data than drugs still in Phase 1 or Phase 2.
To learn more about the steps you would need to take, this Expanded Access Information for Patients page from the FDA goes into greater detail.
EAPs Funded by the ACT for ALS
The limited availability of traditional Expanded Access Programs combined with restrictive eligibility criteria for clinical trials motivated the ALS community to find a way to provide greater access to EAPs. Thanks to advocacy and lobbying efforts by ALS organizations and individual advocates, the ACT for ALS became law in 2021.
In addition to funding research and drug development infrastructure, under the ACT for ALS, the National Institutes of Health (NIH) provides federal research grants so trial sponsors and participating sites can administer intermediate-sized group Expanded Access Programs at little to no cost to people living with ALS.
Once a grant is awarded for an experimental drug, which must already be in an active clinical trial, participating sites open enrollment for people with an ALS diagnosis who are not able to enroll in the clinical trial for that drug. Applicants will need to meet additional eligibility criteria. Some EAPs are administered remotely.
In addition to funding research and drug development infrastructure, under the ACT for ALS, the National Institutes of Health (NIH) provides federal research grants so trial sponsors and participating sites can administer intermediate-sized group Expanded Access Programs at little to no cost to people living with ALS.
Once a grant is awarded for an experimental drug, which must already be in an active clinical trial, participating sites open enrollment for people with an ALS diagnosis who are not able to enroll in the clinical trial for that drug. Applicants will need to meet additional eligibility criteria. Some EAPs are administered remotely.
These NIH-funded EAPs improve on traditional EAPs in three major ways:
Despite these improvements, there are still barriers to participation:
- Many more people living with ALS are able to try experimental drugs.
- The cost of the drug and related medical visits are generally covered (though participants may still need to pay out of pocket for travel and other expenses).
- The ACT for ALS requires that research teams collect safety and efficacy data, which is important because this helps advance ALS research and understanding.
Despite these improvements, there are still barriers to participation:
- Though significantly greater in number than traditional EAPs, EAPs funded through the ACT for ALS can only enroll a small percentage of all people living with ALS.
- EAPs funded by the ACT for ALS are not always enrolling, and they tend to fill up.
- Though there are multiple sites that administer these EAPs, geographic gaps still exist, which means participation for some would require long trips for each visit.
Note: The ACT for ALS is currently awaiting reauthorization and must be reauthorized by Congress by September 30, 2026. You can learn more and check for updates on webpages from organizations like I AM ALS and ALS United.
How to Search for Expanded Access Programs
If you want to apply for a traditional single-patient EAP, you will need to approach your neurologist about applying to take the experimental drug in a clinical trial for which you are ineligible.
When searching for Expanded Access Programs in general, start by asking your neurologist if your ALS clinic is a participating EAP site, if there are any EAP sites in your area, or if they can recommend another EAP for you.
You can also use online search tools to try to find all available EAP opportunities. Though these tools can be very helpful, some listings may be missing or incomplete, so you may need to be proactive and search in multiple ways.
When searching for Expanded Access Programs in general, start by asking your neurologist if your ALS clinic is a participating EAP site, if there are any EAP sites in your area, or if they can recommend another EAP for you.
You can also use online search tools to try to find all available EAP opportunities. Though these tools can be very helpful, some listings may be missing or incomplete, so you may need to be proactive and search in multiple ways.
CLINICALTRIALS.GOV
All ACT for ALS EAPs are supposed to be listed in the ClinicalTrials.gov database. For each EAP, you should be able to find eligibility criteria, participating locations, and contact information.
Start by filling out these fields:
Important: To narrow your search to just EAPs, you must click the “More Filters” tab, find Study Type, and check the “Expanded Access” box.
Start by filling out these fields:
- Condition/disease: Type in “Amyotrophic Lateral Sclerosis”.
- Other terms: If you know what you’re looking for, try typing it here—or you can leave it blank.
- Intervention/treatment: If you know what you’re looking for, try typing it here—or you can leave it blank.
- Location: Type in your city or state to see what is closest to you. You can also type in your country if you are willing to travel farther.
- Study Status: Leave “All Studies” checked to see all options.
Important: To narrow your search to just EAPs, you must click the “More Filters” tab, find Study Type, and check the “Expanded Access” box.
ALS TRIAL NAVIGATOR
The ALS Trial Navigator’s Trial Browser has a specific filter for EAPs.
3. Click “Apply” and close out the filters box.
For more detailed guidance on using online search tools in general, visit our Finding Trials and Studies page.
- Select the Interventional tab on top.
- Click the Filters button and then fill out the following:
- Country: Select a country to narrow down the search
- Recruitment Status: Select “Recruiting”. If you want to see upcoming opportunities, select “Not yet recruiting”.
- Expanded Access: Open this tab and check “Only Expanded Access Programs”.
3. Click “Apply” and close out the filters box.
For more detailed guidance on using online search tools in general, visit our Finding Trials and Studies page.
What questions should I ask?
Once you’ve found a specific EAP that may be a good fit, share the information with your ALS clinic or medical team and ask for their input. Discuss whether the potential benefits outweigh the potential risks.
If you are still interested, you will need to reach out to the EAP research team using the contact information you find. Here are some ideas for questions you could ask:
If you are still interested, you will need to reach out to the EAP research team using the contact information you find. Here are some ideas for questions you could ask:
- What are the eligibility criteria?
- What type of drug will I be taking?
- What are the potential benefits?
- What are the potential risks?
- How often will I need to take the drug?
- Will the EAP team coordinate with my ALS care team?
- How will the drug be delivered (e.g. pill, injection, IV, intrathecal (spinal) injection)?
- Where is the EAP site?
- Is there a remote participation option?
- How often will I need to travel to the site?
- How long is each visit?
- Will there be reimbursement for travel or other expenses?
Before your call or visit, add any other questions you may want to ask.
Making the Decision
If you’ve identified a specific EAP, been informed that you are eligible, and had your questions answered, you will need to decide if participating is right for you.
In addition to getting input from your ALS clinic team, ALS support organization, and loved ones, it may be helpful to reflect on the following questions:
In addition to getting input from your ALS clinic team, ALS support organization, and loved ones, it may be helpful to reflect on the following questions:
- Do the potential benefits outweigh the potential burdens?
- Does participating align with my priorities?
- Am I able to commit to the required time?
- Will I have enough support from caregivers or others?
- Will I be able to travel to the site now and in the future?
It is common for people living with ALS to enroll in clinical trials or EAPs because they hope to get early access to an experimental drug that could slow, stop, or reverse their symptoms. Though this is possible, historically the statistical chances are low. It is also true that ALS researchers understand more about the disease now and are designing more targeted and sophisticated clinical trials.
People also enroll in clinical trials and EAPs because they want to help advance ALS research. In addition, ALS clinical research participants often experience a better quality of life because they have more frequent interactions with ALS professionals who can answer questions and provide additional support between clinic visits.
Potential downsides to participation include side effects, adverse health events, time commitment, out-of-pocket costs, and travel to the trial site, which may become more difficult if your ALS is progressing.
People also enroll in clinical trials and EAPs because they want to help advance ALS research. In addition, ALS clinical research participants often experience a better quality of life because they have more frequent interactions with ALS professionals who can answer questions and provide additional support between clinic visits.
Potential downsides to participation include side effects, adverse health events, time commitment, out-of-pocket costs, and travel to the trial site, which may become more difficult if your ALS is progressing.
Learn More
- The Expanded Access Programs webinar hosted by NEALS features three researchers who provide an overview of EAPs for ALS.
- The What is an expanded access program? webpage from I AM ALS includes an informative webinar about EAPs.
- The Sean M. Healey & AMG Center for ALS runs multi-center EAPs and lists them on their Expanded Access Opportunities webpage.
- The FDA’s Expanded Access Information for Patients webpage provides detailed information for people wanting to apply for a traditional single-patient EAP.
- This Guide to Single-Patient Expanded Access from the Reagan-Udall Foundation goes into greater detail on traditional single-patient EAPs.
