Commentary

Video

Challenges With Access, Delivery of New ALS Therapeutics: Richard Bedlack, MD, PhD, MS

The director of the Duke ALS Clinic at Duke Health provided perspective on the issues with getting newly approved ALS therapies to patients who need them the most. [WATCH TIME: 4 minutes]

WATCH TIME: 4 minutes

"All these medications require a prior authorization, which is not a big deal. The problem with edarvone and Relyvrio (AMX0035)—and this is going to be this way with Qalsody (tofersen)—is that these are now all immediately getting denied, resulting in necessary appeals. The initial appeals are easy, but then they become more complicated and ultimately often require a peer to peer review, which takes time to schedule."

After decades of little to no successful drug development, the amyotrophic lateral sclerosis (ALS) community has been invigorated in recent years, fueled by the approvals of AMX0035 (Relyvrio; Amylyx) and tofersen (Qalsody; Biogen). AMX0035, a coformulation of sodium phenylbutyrate-taurursodiol, became the third approved therapy to help slow disease progression or mortality in ALS, following riluzole (Rilutek) in 1995 and edaravone (Radicava; MT Pharma) in 2017. Tofersen, an antisense oligonucleotide, became the first approved agent to treat patients with SOD1 mutation-mediated ALS, a genetic form of the disease for which there were no other approved products.

Despite the benefits these medications may bring patients, accessing them remains a significant issue, says Richard Bedlack, MD, PhD, MS. At the 2023 International Congress on the Future of Neurology (IFN), held in Jersey City, New Jersey, from September 22-23, Bedlack presented on the advances in ALS treatment and the process of incorporating these new therapeutics. Bedlack, director of the Duke ALS Clinic at Duke Health, also spoke about a recently conducted survey study in which results revealed a significant proportion of patients with ALS who were not on FDA-approved medications despite being eligible.

At the conference, Bedlack sat down to discuss his presentation and the change in ALS treatment management. In the interview, he provided commentary on the issues with accessing newly approved agents and ensuring patients who need treatment receive it. He also spoke on the process of administering these therapies, the time it takes, and the need to find new creative ways to effectively utilize neuromuscular specialists and their time.

Click here for more coverage of IFN 2023.

Related Videos
1 KOL is featured in this series.
1 KOL is featured in this series.
Li Gan, PhD
Sarah Anderson, PharmD, NBC-HWC
© 2024 MJH Life Sciences

All rights reserved.