Hello, everyone, and welcome to this week’s edition of Excelsior Solutions’ ICER Update.
Here is this week’s ICER recap:
ICER released a Final Evidence Report and Report-at-a-Glance assessing the comparative clinical effectiveness and value of the following targeted immune modulators (TIMs) that treat moderate-to-severe ulcerative colitis:
- Adalimumab (Humira®, AbbVie)
- Golimumab (Simponi®, Janssen)
- Infliximab (Remicade®, Janssen)
- Infliximab-abda (Renflexis®, Merck)
- Infliximab-dyyb (Inflectra®, Pfizer)
- Tofacitinib (Xeljanz®, Pfizer)
- Ustekinumab (Stelara®, Janssen)
- Vedolizumab (Entyvio®, Takeda)
ICER’s report on these therapies was reviewed at the September 2020 public meeting of the California Technology Assessment Forum (CTAF), one of ICER’s three independent evidence appraisal committees.
“Ulcerative colitis can create a lot of suffering for patients and their families and caregivers,” said Pamela Bradt, MD, MPH, ICER’s Chief Scientific Officer. “Fortunately, for patients not receiving adequate response to conventional immune modulator treatment, the evidence clearly demonstrates that targeted immune modulators often produce durable periods of clinical remission of ulcerative colitis symptoms, allowing patients to resume normal activities. However, there is very little head-to-head data comparing treatment options in this increasingly crowded, and expensive, therapy class. Prices are generally too high, even for the significant clinical benefits obtained, but the advent of biosimilars for infliximab has led to dramatic price decreases for this one agent and its biosimilars, displaying the potential cost savings that biosimilars may be able to deliver more broadly in the US. In the meantime, insurers use prior authorization, step therapy, and switching policies to try to manage the inappropriate pricing in this area, but they need to take specific actions to ensure that these policies do not prevent patients from being able to get appropriate care at a price they can afford.”
EXCELSIOR ACTION POINT: This Final Evidence Report provides support for two tactics that we have been advocating that PBMs and Carriers undertake: (1) Indication-based formularies among the biologic anti-inflammatory products, and (2) Formulary restrictions to limit coverage to the one or two products for each indication that provide optimal cost-effective outcomes. I encourage you to refer to the Report-at-a-Glance that contains a very nice scorecard of the various products used to treat UC which enables you to quickly identify which products stack up well and not so well under ICER’s methodology.
ICER also released an updated Evidence Report assessing the comparative clinical effectiveness and value of valoctocogene roxaparvovec (Roctavian™, BioMarin Pharmaceutical) and emicizumab (Hemlibra®, Genentech) for the treatment of hemophilia A. In August 2020, BioMarin received a Complete Response Letter from the FDA requesting an additional two years of follow-up safety and efficacy data on valoctocogene roxaparvovec, and that process may delay the gene therapy’s US approval decision until at least 2022. ICER previously assessed emicizumab when the treatment was approved by the FDA in 2017 with an indication of treating the subset of individuals with hemophilia A who have inhibitors to the clotting protein factor VIII; reflecting emicizumab’s expanded indication in 2018, this year’s report assesses the value of the treatment in individuals who do not have those inhibitors.
“Treatments to reduce the risk of bleeds for patients with hemophilia have profoundly improved the lives of patients and caregivers,” said David Rind, MD, ICER’s Chief Medical Officer. “Recently, exciting new strategies for prophylaxis like emicizumab have become available, and gene therapies have even raised the possibility of a cure on the not-too-distant horizon. Our analysis of the evidence suggests that emicizumab is comparable to or better than factor VIII prophylaxis and does save money overall. But our report notes that this is because the prices for factor VIII, despite recombinant technologies and apparent competition in the marketplace, have not decreased, making prophylaxis with factor VIII an extremely expensive treatment required for a lifetime.
“Patients and patient groups have fought to get access to higher doses and multiple preparations of factor VIII so as to reduce the time each week that they are at increased risk for bleeding. We look forward to our public meeting at which all stakeholders can discuss the tensions created by the pricing of factor VIII options, and the extent to which the high costs for prophylaxis should drive considerations of fair pricing for treatments like emicizumab and the gene therapies that will hopefully soon be available.”
This Evidence Report will be reviewed at a virtual public meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC) on October 30, 2020. The New England CEPAC meeting is one of ICER’s three independent evidence appraisal committees comprising medical evidence experts, practicing clinicians, methodologists, and leaders in patient engagement and advocacy.
EXCELSIOR ACTION POINT: I continue to recommend that you participate in ICER’s activities should your schedule permit. You can register here to watch the live webcast of the virtual meeting as this evidence report continues to be modified into its final form. Recall that Roctavian was expected to be launched with a price ranging between $2 million and $3 million for a one-time infusion so there is no telling what impact the passage of two years’ time will have on those numbers.
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