If a drug designed to treat a life-threatening illness shows early promise in clinical testing, 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. For individuals living with ALS, their caregivers, and their loved ones, Expanded Access not only offers the chance to try new innovative medications that may be successful, but it also offers hope.

Established decades ago by the FDA, Expanded Access, or “compassionate use” as it is often referred, allows patients with a terminal diagnosis early access to new therapeutics that show promise – even if the patient is not involved in the ongoing clinical trial – or if the medication has not yet been approved by the FDA.

To qualify for Expanded Access consideration, a person must:

• Have a life-threatening illness, like ALS, and there cannot be any other comparable treatments available.
• The 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.

However, even if a person meets all the above criteria, there are still some hurdles to gaining experimental therapeutics through the program. On a recent Connecting ALS podcast, Dr. Alison Bateman-House, assistant professor in the division of medical ethics at NYU Langone Health and a member of Langone's Working Group on Compassionate Use and Preapproval Access, shared her thoughts based on her experience.

While the FDA supports Expanded Access, people must exhaust all available therapies and clinical trial options first because clinical trials offer more protections if a drug turns out to be unsafe or ineffective. “The first route should always be a clinical trial. And that in of itself is complicated,” she said. “Not every clinician knows about what clinical trials are happening. It takes some leg work on the part of the patient, unfortunately, to figure out what their options are.”

Obtaining access to what Dr. Bateman-House calls, “investigational, not yet FDA approved medicines” requires not only the support of the patient, but that of a physician, the FDA itself and pharmaceutical or medical device manufacturer as well. Since these decisions are determined on a case-by-case basis, the process is rarely straightforward.

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“As a patient, what you need to do is you need to get your request to that company, and you yourself cannot do that.” Dr. Bateman-House continues, “You have to do that through a physician intermediary. So, your first step is to find a physician who is willing to do this for you. This is time-consuming. It's something that not every physician knows how to do or is willing to do, or perhaps they think the product that you're trying to get access to is not a good choice for you.”

Once a willing physician has been identified, the physician must reach out to the pharmaceutical or medical device manufacturer on behalf of their patient and formally request the product, and then it's up to the company to decide. Dr. Bateman-House went on to explain there are more procedural steps after the request is made, “but those are really the two most challenging ones.”

To make matters even more complicated, “products in pre-FDA approval belong to the company developing them, and you may ask for access, but it is up to that company to decide if it wants to give access or not, and it doesn't really have to justify to anyone on what grounds it makes those decisions,” she said.

Most notably, the patient’s licensed physician, the FDA’s Institutional Review Board (IRB), and the company that manufacturing the product need to agree that expanded access is appropriate in order for a patient to receive the investigational therapy. If the patient meets the criteria, and the patient’s physician, the IRB and the pharmaceutical company all agree, then Expanded Access may be an option.

Dr. Bateman-House reiterates that the true decision-maker for Expanded Access therapeutics is the manufacturer themselves. “The gatekeeper is the company and there are misperceptions out there that it's FDA or various in other roadblocks. It really comes down to, does the company want to give you access or not?”  

“Oftentimes you feel like you're on a path that no one has tread before you, and you're having to learn things yourself,” says Dr. Bateman-House. “I can promise you, people have walked this path before and there are guides out there. And unfortunately, it's just much more difficult to find them than it should be.”

For more information and detail about Expanded Access, visit our website here. The Northeast ALS Consortium (NEALS) recently shared a webinar exploring this topic from a clinical and patient perspective, The Latest on Expanded Access Protocols in ALS. You can view the recording here.

To continue to learn more about ALS and follow stories about people living with the disease in the community, follow our blog at ALS.org/blog.