Specialty Pharmacy Pipeline Update from AMCP 2019

Specialty pharmacy medications represent about 1.5% to 2.5% of prescriptions but comprise approximately 50% of total prescription costs. The average cost of a specialty drug is approximately $4,000, and specialty cost inflation has run between 11% of 15%. According to a study, 55% of employer respondents rated drug costs as their top concern, and 71% said they do not feel that the current prices of orphan drugs are sustainable.

During a presentation at the AMCP Annual Meeting, Susan Trieu, PharmD, director of enterprise specialty clinical solutions at MedImpact Healthcare Systems, Inc., in Southlake, Texas, gave an overview of the specialty pharmacy pipeline.

Dr. Trieu said that when considering the budget impact, the following components are important:

  • Population
    • What does this new agent offer?
    • How does this compare to current treatment options?
  • Market share
    • Will prescribers and patients want the new treatment?
    • Are there other viable treatment options that payers will require first?
  • Treatment cost
    • Is there anything else to treat this condition?

Other factors to consider include the line of business (commercial, Medicare, Medicaid), size of the plan, whether it’s a genetic disease, regional differences, the need for centers of excellence, and medical versus pharmacy costs.

She then discussed some specialty drugs in the pipeline. An investigational chimeric antigen receptor T-cell therapy, bb2121, is in development for the treatment of relapsed/refractory multiple myeloma. In a phase I study of heavily pretreated patients, there was a 95.5% overall response rate in the 22 patients who received the highest dose, including a 50% complete response rate. The median progression-free survival was 11.8 months. Many patients (63%) experienced cytokine release syndrome, and 33% of patients experienced neurotoxicity. If approved, this drug may launch in 2020.

Risdiplam is an investigational oral agent for the treatment of spinal muscular atrophy (SMA). In the phase II/III FIREFISH study that included patients with type 1 SMA, 95% (n=20/21) were event-free at 10.5 months. In the phase II/III SUNFISH study that included patients with type 2 and 3 SMA, 63% of patients (n=19/30) had improvement of three or more points in motor function measure at 12 months. No patients have discontinued treatment due to adverse events (AEs). In the FIREFISH trial, 10 patients (48%) had at least one serious AE, and two patients died due to disease progression. In the SUNFISH trial, six patients (11.8%) had at least one serious AE. The sponsors plan on filing 2H in 2020.

Valoctocogene roxaparvovec is an adeno-associated virus-factor VIII vector gene therapy in development for the treatment of adults with severe hemophilia A with no inhibitors. In a phase I/II trial of nine adult patients, the high-dose cohort (n=7) saw increased factor VIII levels, which was maintained one year after infusion. The high-dose cohort had an annualized bleeding rate that decreased from 16 events to one event after the gene transfer. No patients developed inhibitors to factor VIII or thrombosis. Seven patients experienced an increase in alanine aminotransferase levels with resolution. The manufacturer plans to file via accelerated approval this year or next year.

Specialty spend will continue to increase due to a very active pipeline, Dr. Trieu concluded, noting that the impact on payers will vary based on the size of the treatable population and plan.