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Understanding Edaravone Use, Adherence Among VA Patients With ALS

March 04, 2021

By Julie Gould

Fran Cunningham, PharmD, associate chief consultant for the Pharmacy Benefits Management Services in VAFran Cunningham, PharmD, associate chief consultant for the Pharmacy Benefits Management Services in VA, discusses the findings of her recent study, highlighting a pharmacovigilance surveillance initiative looking at edaravone and monitoring utilization as well as the safety and effectiveness of edaravone among patients in the VA with ALS.

First, please tell us about yourself and your health care background.

My name is Fran Cunningham. I'm Associate Chief Consultant for the Pharmacy Benefits Management Services in VA. I'm the Director for the Center for Medication Safety.

Our Center for Medication Safety is pretty much responsible for overseeing pharmacovigilance. It's a comprehensive pharmacovigilance program for all of VA. What does that mean? That means that we, for all practical purposes, track the safe and appropriateness of use of medications and pertinent vaccines in VA.

What existing data led you and your co‑investigators to conduct your research?

This was, in essence, part of our pharmacovigilance efforts. It was a surveillance initiative that was conducted as part of our standard pharmacovigilance program, specifically where we assess use of medications. In this instance, this is a medication that was requiring prior authorization in the veteran patient population.

We typically will do that where there is a paucity of clinical trial data or which clinical trial data is limited or lacking in the patient population that resembles our veterans. These types of evaluations are typically conducted by us to assess the safe and appropriate use of new molecular entities in our health care system.

Can you please briefly describe your study and the findings? Of those findings, were any of them particularly surprising?

Let me first state that this was a pharmacovigilance initiative and not a true study. We emphasize that throughout the paper.

The goal of this evaluation was to describe a pharmacovigilance surveillance initiative where we were looking at edaravone and monitor edaravone's utilization as well as the safety and effectiveness of edaravone. It was a descriptive assessment initially to look at edaravone utilization, and then for more of the evaluation of the safety and effectiveness.

We used a propensity score match retrospective cohort evaluation where we could compare edaravone to riluzole. Our results showed that the majority of patients were older white males, which was expected in our patient population.

The utilization assessment showed that a large percentage of our patients discontinued edaravone by the end of the evaluation period with almost half receiving approximately one to three cycles of treatment as part of their overall treatment.

In regard to the outcomes, where we were looking at ALS medications used, also with the mortality, we looked at full discontinuation as well as all‑cause hospitalization. We looked at that in both the acute and chronic users and assessed that accordingly.

When we looked at that, we saw that there was significantly more acute all‑cause hospitalization events that occurred with edaravone versus riluzole, both alone or when used in combination with edaravone. I'm referring to the riluzole.

In our chronic users, our edaravone patients also had an increased risk, and this is as we refer to it as an increased hazard ratio of ALS‑associated hospitalizations. That was pretty reflective of our results.

The death rate, however, was lower with the edaravone group, but the difference was not statistically significant.

What did we see overall? Although some of the beneficial outcomes were somewhat less than expected ‑‑ if you were to ask what we found surprising ‑‑ we still believe that the findings should not raise any major concerns.

What should happen is that, based on our evaluation, we should encourage more detailed studies to be conducted by other investigators to determine the effectiveness of edaravone in ALS patients.

That leads us nicely into this next question. What are some of the possible real-world applications of these findings in clinical practice?

Now this is my favorite question. It is so important to use real world data, especially in pharmacovigilance efforts such as this, so that you can assess medications in your own health care system. That's paramount, so that you can better understand utilization, safety, and effectiveness in the health care system in which one is practicing.

These data, for all practical purposes, assist providers, and in this instance doctors, as well as patients in a shared decision-making process. This, overall, provides realistic expectations of how a drug, and specifically in this case, how edaravone performs in real world settings rather than what you see in a clinical trial setting.

Our real-world assessment of this drug for ALS patients demonstrated that more effective treatments are needed for the treatment of ALS at this time. The use of edaravone has potential risks, but it also has benefits, as we found.

ALS, as a disease, is heterogeneous in nature. Our analysis of our large patient cohort pretty much highlighted the need to conduct these type of pharmacovigilance efforts and why one should do post‑marketing evaluations with newer molecular entities in a real-world population.

It's good for the patient. It's good to understand more about the drug. It helps define the safety and efficacy of the drug, and in this instance of edaravone, outside of a highly selective clinical trial population.

Do you hope to expand upon this research?

What we really wanted to do was, again, I keep going back to that term of pharmacovigilance, and surveillance, and to assess the safe and effectiveness of this drug. What we'd really like to do is see other investigators conduct an analysis and do more with this particular agent.

We will continue to track the safe and appropriate use, as we do other agents as I stated at the top of the interview. This is something we do with other new molecular entities. We will encourage investigators to conduct more detailed evaluations, specifically to better assess edaravone effectiveness.

What we do think, however, is that it will require detailed evaluations, not necessarily available through observational data analysis alone. It may require more of a detailed chart review when other investigators move forward to assess this medication in more detail.


Vu M, Tortorice K, Zacher J, et al. Assessment of Use and Safety of Edaravone for Amyotrophic Lateral Sclerosis in the Veterans Affairs Health Care System. JAMA Netw Open. 2020;3(10):e2014645. Published 2020 Oct 1. doi:10.1001/jamanetworkopen.2020.14645

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