Specialty Medications Nearing 50 Percent of Spend

Nov 27, 2018

Specialty pharmacy drug spend continues to rise. Express Scripts' Aimee Tharaldson reviews the current and future specialty medication landscape.

Rph Article

While traditional drug spend is decreasing, specialty pharmacy drug spend continues to rise. Now at 41% of overall pharmacy drug spend, specialty is estimated to reach close to 50% in the next two years.

Within the specialty pipeline, orphan drugs make up 45% of those in development.

Specialty medications managed through the pharmacy benefit account for 41% of plans’ total pharmacy spend. It’s important for plans to understand the pipeline of specialty medications and create a strategy to manage these new, costly therapies, including reducing the burden on patients with rare diseases.

This year, the U.S. Food and Drug Administration (FDA) has approved 34 new specialty medications. And in 2019, expect to see more specialty drugs gain approval.

As we near the end of 2018, let’s take a look at some of the approvals this year and what’s ahead for 2019.


  • Erleada™ (apalutamide) gained approval February 14, 2018 and is indicated to treat prostate cancer, the most common form of cancer among men.
  • On June 27, 2018, Braftovi™/Mektovi® (braftoviencorafenib/binimetinib) received approval for the treatment of metastatic melanoma.
  • When Libtayo® (cemiplimab-rwlc) was approved on September 28, 2018, it became the first FDA approval of a drug specifically for advanced cutaneous squamous cell carcinoma.

Multiple cancer medications are pending approval in the first half of 2019, including:

  • Sacituzumab govitecan for the treatment of patients with metastatic triple-negative breast cancer who previously received at least two prior therapies for metastatic disease. The prescription drug user fee act target action date is January 18, 2019. If approved, sacituzumab govitecan would be the first antibody-drug conjugate approved for the treatment of metastatic triple-negative breast cancer.
  • Selinexor for the treatment of patients with penta-refractory multiple myeloma has a prescription drug user fee act target action date of April 6, 2019.


Multiple medications for the treatment of human immunodeficiency virus (HIV) have been approved in 2018, including:

  • Biktarvy® (BIC/FTC/TAF), a once-daily tablet regimen which is a combination of a novel unboosted integrase strand transfer inhibitor (INSTI)—bictegravir—and Descovy (FTC/TAF, Gilead) dual nucleoside reverse transcriptase inhibitor (NRTI) backbone, approved Feb. 7, 2018.
  • Trogarzo™ (ibalizumab-uiyk), in combination with other antiretroviral(s), is indicated for the treatment of human immunodeficiency virus type 1 (HIV-1) infection in heavily treatment-experienced adults with multidrug resistant HIV-1 infection failing their current antiretroviral regimen. Trogarzo received FDA approval on March 6, 2018.

One of the HIV drugs in the pipeline for 2019, leronlimab (PRO-140) is a new antibody viral-entry inhibitor that is given as a monthly subcutaneous injection

Specialty Drugs to Watch

  • AVXS-101 is a DNA based gene therapy that delivers a functional copy of a human survival motor neuron 1 (SMN1) gene to treat children with spinal muscular atrophy (SMA) Type 1. SMA Type 1 is a life-threatening neurodegenerative disease in which babies have difficulty breathing and swallowing; they also cannot sit, crawl or walk. Most children with SMA Type 1 do not survive or need permanent ventilation support to breathe by 20 months of age. AVXS-101 has demonstrated strong efficacy in an early-phase study. According to AveXis, a subsidiary of Novartis, approximately 300 children per year in the U.S. will be candidates for treatment with AVXS-101. It is expected to cost between $1million and $3 million for the one-time IV infusion. While the FDA action date is June 18, 2019, early approval is expected.
  • Tafamidis meglumine is expected to be the first drug approved for the treatment of cardiomyopathy caused by transthyretin-mediated amyloidosis. It is for patients with the hereditary form of the disease as well as the wild-type form of the disease, which may occur as people age. This is a rare condition that causes progressive heart failure and premature death. Tafamidis is an oral medication that is taken once daily. It works as a transthyretin stabilizer to reduce deposits of amyloid fibrils in the heart. In a pivotal clinical study, tafamidis demonstrated a 30% reduction in the risk of death and a 32% reduction in the rate of heart-related hospitalization compared to placebo. Approval of tafamidis is expected in mid-2019; it could rapidly become the standard-of-care for these patients.
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