Hello everyone, and welcome to this edition of Excelsior Solutions’ ICER Update.
ICER released a Final Evidence Report assessing the comparative clinical effectiveness and value of AMX0035 (Amylyx Pharmaceuticals, Inc.) and oral edaravone (Mitsubishi Tanabe Pharma Development America, Inc.) for the treatment of amyotrophic lateral sclerosis (ALS, or “Lou Gehrig’s Disease”).
(Links to ICER’s Final Evidence Report and Policy Recommendations)
EXCELSIOR TAKEAWAYS:
- Evidence of the effectiveness of these therapies vs. standard of care is average at best and insufficient at worst.
- The approximate $170,000 per year cost for both therapies is too high despite the overwhelming need for treatments for this devastating disease.
- The evidence suggests that AMX0035 extends life, and it is clear that oral edaravone is far less burdensome than its infused version, but prices need to be greatly reduced to align with benefits.
- The cost of AMX0035 would have to be reduced to between $9,000 to $31,000 per year to meet ICER’s Annual Health-Benefit Price Benchmark.
- The cost of oral edaravone would have to be reduced to between $1,400 to $3,200 per year to meet ICER’s Annual Health-Benefit Price Benchmark.
- Despite the high unmet medical need, at current pricing neither of these products would appear to be worth their materially high costs according to the ICER analysis.
ICER also released a Final Evidence Report assessing the comparative clinical effectiveness and value of betibeglogene autotemcel (Zynteglo, bluebird bio) for the treatment of beta thalassemia.
(Links to ICER’S Final Evidence Report and Policy Recommendations)
EXCELSIOR TAKEAWAYS:
- It is worth noting that ICER completed its analysis before Zynteglo was approved by the FDA, and used an estimated cost of $2.1 million in its analysis vs. the actual price of $2.8 million announced upon Zynteglo’s FDA approval.
- All ICER panelists (12-0) found the evidence is adequate to demonstrate a net health benefit when Zynteglo is compared to standard clinical management, and a majority (9-3) of ICER panelists found that Zynteglo represents “high” long-term value for money.
- Zynteglo’s manufacturer, Bluebird Bio, has launched a program to support payers and families, and the actual site can be found here.
- Zynteglo is being launched with an outcomes-based arrangement that will refund up to 80% of its cost if a patient fails to achieve and maintain transfusion independence up to two years after infusion.
- The best estimate is that there are approximately 1,500 prospective Zynteglo patients in the U.S.
As always, if you would like to discuss the potential impact of these or any prior ICER reports, 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