Expanded Access

To prove that their medications are safe and effective before they reach the clinic, pharmaceutical companies must complete extensive clinical testing. Once a drug candidate begins this process, it could take up to 15 years for it to achieve FDA approval. Only after that time are doctors allowed to prescribe the drug to their patients.

But sometimes, a drug shows promise early on, even before it finishes clinical testing. If the drug is designed to treat a life-threatening illness, and if patients have no other options, the FDA might allow the pharmaceutical company to give patients early access to the drug outside of the clinical trial.

Expanded access (or “compassionate use”) gives people access to investigational drugs under certain conditions outside of a clinical trial. The FDA always prefers that people participate in a clinical trial first to ensure they truly have no other options. Today, several pharmaceutical companies are running expanded access programs (EAPs) for ALS drugs. Below, we’ve answered some questions you might have about EAPs and what it means to participate.

  1. What kinds of EAPs are available and how do I get involved?
  2. Who qualifies for expanded access?
  3. Who makes the decision to offer an EAP?
  4. Are there any EAPs for drugs intended to treat ALS?
  5. Are there any risks associated with EAPs?
  6. Does it cost any money to participate in an EAP?
  7. Other Resources on EAPs

What kinds of EAPs are available and how do I get involved?

There are several different types of EAPs. Pharmaceutical companies can request Expanded Access from the FDA to bridge the gap between the end of a clinical trial and when the drug receives marketing approval for those enrolled in the trial. Sometimes, Expanded Access will allow access to a drug that is not being actively investigated for clinical use. The FDA might also give an individual doctor permission to give someone an unapproved drug in an emergency situation.

In any case, a physician can request Expanded Access to an investigational therapy after they have determined the patient is eligible.

Learn more here.

Who qualifies for expanded access?

To qualify for Expanded Access:

  • Someone has to have a life-threatening illness like ALS, and there cannot be any other comparable treatments available.
  • This person is not able to be a part of a clinical trial because there are no ongoing trials, the person does not have access to a trial, or is not eligible for clinical trials.
  • They cannot take the drug if it will interfere with an ongoing clinical trial.
  • The drug’s potential benefits must outweigh the potential risks.

Who makes the decision to offer an EAP?

Even if you meet the criteria under the law and FDA regulations, the licensed physician, the Institutional Review Board (IRB), and the company all need to agree that expanded access is appropriate for you in order for you to receive the investigational therapy.  

Submission of a request for an EAP usually starts with your licensed physician who will formally request a product and submit paperwork to the FDA. Then the FDA decides to approve or deny the request. Then the decision goes the IRB at the institution where you would be receiving the EAP. Finally, the company decides if they want to provide the experimental therapy.

Are there any EAPs for drugs intended to treat ALS?

Because ALS is life-threatening and there are no effective treatments for the condition approved by the FDA, certain drugs under investigation may qualify for expanded access. Companies post their Expanded Access programs on their website as public information.

Here are just some examples of open EAP programs in ALS:

  • Biogen is running an EAP for anyone with a SOD1 mutation (learn more about the genes involved in ALS here). Through this program, Biogen is supplying the drug at no charge to patients. Please talk to your physician to find out if you are eligible. If you are, they can submit a request on your behalf to join the program. Read more on Biogen’s website and learn more about the tofersen expanded access program here.
  • Amylyx Pharmaceuticals opened an EAP program for AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO; also known as ursodoxicoltaurine]) in the U.S. for people living with ALS that meet program eligibility criteria. Learn more here. Also, visit https://www.clinicaltrials.gov, study A35-006. For questions email the Clinical Research Organization overseeing the program at patient.access@bionical-emas.com

Are there any risks associated with EAPs?

The FDA fully supports Expanded Access, but people must exhaust available therapies and clinical trial options first because clinical trials offer more protections if a drug turns out to be unsafe or ineffective. Also, the more quickly a pharmaceutical company can recruit people for their clinical trials, the faster they can complete clinical testing and bring the drug to more patients in need.

The FDA is hesitant to allow EAPs because they involve giving people drugs that have not been a proven safe or to work. Consequently, these drugs may produce unexpected side effects. Also, there is less of a guarantee that the drug will work versus one that is FDA approved.

Does it cost any money to participate in an EAP?

Often, the costs associated with EAPs are not covered by health insurance. People who participate in these programs may have to pay for the experimental drug out of their own pockets. In some cases, the pharmaceutical company running the EAP will cover the cost of the drug. In addition, there may be costs not covered by third-party payers such as private insurance or Medicare. If you are considering entering an EAP, ask your doctor about the costs involved in participating.

Other Resources on EAPs:

ALS Association Connecting ALS Podcast:
Expanded Access to Promising Treatments
March 3, 2022

Features Dr. Alison Bateman-House, a medical ethicist and member of NYU Langone’s Working Group on Compassionate Use and Preapproval Access, to uncover the work being done to expand the ethical allocation of drugs that are currently in development.

Learn more about the Working Group on Compassionate Use and Preapproval Access here.

NEALS webinar:
The Latest on Expanded Access Protocols in ALS
March 18, 2022

Conversation about Expanded Access Protocols (EAPs) in ALS, including recent progress in setting EAPs up alongside clinical trials. This webinar will be moderated by Dr. Jinsy Andrews and will feature Dr. Merit Cudkowicz, Dr. Sabrina Paganoni, Philip Green, and Sandy Morris.

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