ICER Update 07.15.19

Hello, everyone, and welcome to this edition of Excelsior Solutions’ ICER Update. Here is this week’s ICER recap:

  1. ICER published its evidence report assessing the comparative clinical effectiveness and value of two exon-skipping therapies to treat Duchenne muscular dystrophy (DMD) — eteplirsen (Exondys 51™, Sarepta Therapeutics) and golodirsen

(Sarepta Therapeutics) — as well as deflazacort (Emflaza®, PTC Therapeutics), a corticosteroid. Previewing the conversation that will happen at its July 25 public meeting, ICER’s chief medical officer Dr. David Rind stated:

“DMD is a progressive neuromuscular disorder that devastates the lives of children and the families who care for them, and better therapies are

desperately needed… This apparent mismatch between evidence and price raises important concerns for patients, caregivers, and all other stakeholders in the health care system.”

EXCELSIOR ACTION POINT: Many of the concerns voiced at the time that the FDA approved Exondys 51 revolved around whether pressure from DMD advocacy groups, in the absence of any prior treatments for DMD, caused FDA to reach

an approval decision based on a lower standard of proof than they would have applied under normal circumstances. ICER is bringing an objective standard to that ongoing conversation surrounding DMD treatments.

  1. Earlier this week, and based on its June public meeting, ICER issued its Final Evidence Report assessing the clinical and economic value of two investigational technologies that treat children with peanut allergy by building their immune tolerance to peanuts: Viaskin® Peanut (DBV Technologies) and AR101 (Aimmune Therapeutics).

EXCELSIOR ACTION POINT: The major takeaway from this Final Evidence Report is that the clinical evidence does not yet demonstrate that long-term benefits of peanut desensitization outweigh short-term risks. This fundamental

concern ought to place the near-term prospects for adoption of these peanut desensitization technologies under some reasonable amount of skepticism.

  1. Finally this week, ICER provided a sneak peek into potential topics they are considering for value assessments next year (calendar year 2020). From gene therapies to PrEP to digital apps, there’s a little something for everyone.

If you would like to discuss these ICER activities, or any other aspects of your Pharmacy Benefit Plan, simply reach out to your Excelsior Solutions account team and we will be happy to quickly set up a call.

Until next time!

Bob Kordella, RPh, MBA

More related articles

More Articles