Myasthenia Gravis is a neuromuscular disorder that impacts over 50 thousand patients in the United States (U.S.).
Of those patients that are diagnosed, approximately 95 percent are treated with immunosuppressants, intravenous immunoglobulin, and/or plasma exchange. The total number of treated patients is expected to grow at an annual rate of 1.7 percent. Of those patients who are treated, it has been reported that 5 to 10 percent will fail treatment with traditional therapies (Figure 1) (http://www.alexion.com/Products/Soliris/Soliris-Generalized-Myasthenia-Gravis (accessed October 25, 2017)).
Figure 1: Comparison of prevalence, treatment, and treatment failure numbers for patients with myasthenia gravis in the U.S. (GlobalData)
On October 23, 2017, the Food and Drug Administration (FDA) approved Soliris® for generalized myasthenia gravis patients who are anti-acetylcholine receptor (AchR) antibody positive.
This approval represents the first FDA approval for this condition in over 60 years and provides an innovative treatment for a subset of myasthenia gravis. Soliris’s approval for this condition was based on the REGAIN and REGAIN Open Label Extension study which evaluated effectiveness in patients who were refractory to treatment with immunosuppressive agents, intravenous immunoglobulin, and plasma exchanges (Table 1). For details on clinical response to therapy please refer to Alexion’s website (http://www.alexion.com/Products/Soliris/Soliris-Generalized-Myasthenia-Gravis ) which has a concise summary of clinical results.Table 1: REGAIN study design and dosing
|Study||Study Type||Population||Population Size||Soliris Dose|
|REGAIN (MG-301)||randomized, double-blind, placebo-controlled trial||Population required to have a confirmed myasthenia gravis diagnosis with positive serologic test for anti-AChR antibodies. In addition, all patients were required to have previously failed treatment with at least two immunosuppressive agents or failed treatment with at least one immunosuppressive agent and required chronic plasma exchange or IVIg, and had an MG-ADL total score ≥6 at study entry.||125||900 mg weekly for 4 weeks followed by 1200 mg one week later, and then 1200 mg every two weeks.|
|REGAIN Open-Label Extension Study (MG-302)||Continuation of REGAIN (MG-301)||125|
Upon approval of Soliris, we started to question what would be the potential total cost of this therapy on a monthly basis and what should a plan potentially expect in PMPM spend due to utilization.
The wholesale acquisition cost of Soliris is $6,522.90 for a 300mg vial as of September 2017, which translates to a HCPCS price of $217.43. For the starting 900 mg dose, the total WAC would be $19,569 and for the maintenance dose of 1,200 mg it would equal $26,092 (Table 2). The final maintenance dose of Soliris would be given on a biweekly basis resulting in a total monthly WAC of $52,183 for the final dose of 1,200 mg.Table 2: Soliris® Coding and Pricing
|HCPCS Descriptor||Injection, eculizumab, 10 mg|
|Vial strength||300 mg per 30 ml|
|WAC per vial||$6,522.90|
|HCPCS units per vial||30 units|
|WAC per HCPCS unit||$217.43|
|Total WAC 900 mg Dose||$19,569|
|Total WAC 1,200 mg Dose||$26,092|
|Total WAC for 2,400 mg (2 doses of 1,200 mg)||$52,183|
Taking the total monthly cost of therapy and estimates prevalence for a 1 million life membership, we estimate that a plan should expect an increase of $0.52 to $1.04 in PMPM utilizing wholesale acquisition cost (Table 3). This represents a significant amount of spend for a small population but also represents an opportunity for increased diligence in ensuring that appropriate criteria are in place supporting a stepwise approach to therapy and reservation of Soliris® for treatment refractory patients.Table 3: Projected cost impact on monthly and annual basis for a 1 million life plan
|Rates||Cost Per Month||Annual Cost|
|Total Members||1 million|
|Total patients with myasthenia gravis (0.02%)||200|
|Traditional therapy treatment failure at 5%||10|
|Traditional therapy treatment failure at 10%||20|
|Total monthly treatment WAC for Soliris®||$52,183|
|Total monthly Soliris® WAC for 5% failure rate||$521,832|
|Total monthly Soliris® WAC for 10% failure rate||$1,043,664|
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