ICER Update 01.29.20

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

Here is this week’s ICER recap:

  1. ICER released a draft evidence report on three treatments for sickle cell disease: crizanlizumab (Adakveo®, Novartis), voxelotor (Oxbryta™, Global Blood Therapeutics), and L-glutamine (Endari®, Emmaus Life Sciences). Public comment is open until February 20. ICER’s preliminary draft notes that sickle cell disease has been an historically underserved area for research, with no new treatments for decades. While assessing the value of these treatments, policymakers will need to consider the substantial lifelong burden associated with sickle cell disease, as well as the potential for these treatments to reduce the disparity in life expectancy between black and white Americans. This preliminary draft marks the midpoint of ICER’s eight-month process of assessing these treatments, and the findings within this document should not be interpreted to be ICER’s final conclusions.
    EXCELSIOR ACTION POINT: As noted by ICER, sickle cell disease has been underserved by research efforts for a very long time. These new products have the potential to reduce the burden of this disease among a relatively large population of patients. Plans should undertake efforts to quantify the number of sickle cell patients in their respective populations as the uptake of these new medications may be higher than what is typical for new medications, owing to the prolonged research drought. Also, additional medications – including at least one gene therapy – are continuing to work their way through the development pipeline. Finally, the draft evidence report takes a dim view of the current pricing of these products, but as ICER hints, this is merely an interim report at the midpoint of the drafting process.
  2. ICER also posted a Draft Scoping Document outlining their planned review of the gene therapy valoctocogene roxaparvovec (BioMarin Pharmaceutical, Inc.) and emicizumab (Hemlibra®, Genentech), for the potential treatment of hemophilia A. The draft scope is open to public comment until February 13.
    EXCELSIOR ACTION POINT: As news begins to leak out that, at least in the case of valoctocogene, it may be priced between $2 million and $3 million for what is hoped to be a one-time dose, Plans should be doing two things: (1) Quantifying the number of hemophilia A patients in their populations, and (2) stratifying their hemophilia A patients by severity using the criteria specified by the National Hemophilia Foundation. The reason for the quantification step is to determine the scope of hemophilia prevalence; the reason for stratification by severity is to anticipate the possibility that certain of these new hemophilia A treatments, depending upon their final cost, may only be cost-effective vs. currently available treatments for hemophilia patients whose disease severity is higher.

As always, if you would like to discuss the potential impact of these, or any prior, ICE reports, or any other aspects of your Pharmacy Benefit Plan, simply reach out to you Excelsior Solutions account team and we will be happy to quickly set up a call.

Until next time!

Bob Kordella, RPh, MBA

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