Specialty drugs are those that involve frequent dosing adjustments and intensive clinical monitoring, as well as intensive patient training and compliance assistance, limited distribution, and specialized handling and administration, according to Aimee Tharaldson, PharmD, of Express Scripts, who discussed the current specialty pharmacy pipeline.
In the specialty pharmaceutical industry, many generic options will become available in the next few years, as 64 patents will expire through 2022, with a $25 billion opportunity for revenue. Some of the key drugs losing patent protection include abiraterone acetate, fingolimod, and enalidomide.
Biosimilars will also play a large role in the future as more patent protections end. To date, nine biosimilars have been approved for six therapeutic areas, but only three have launched in the United States. Much of the delay has been due to the legal issues surrounding the patent of the reference agents. The U.S. Food and Drug Administration (FDA) is working on policies to increase competition in biosimilar space to address issues that are holding up the launches. In addition, these agents are finding trouble capturing market share. “This is taking longer than we would like,” said Dr. Tharaldson, “but we will get to cost savings eventually.” Ten additional biosimilars could be approved this year—including two for Herceptin and two for Neulasta.
In 2017, the FDA approved 15 new cancer drugs, and despite the competition in the cancer space, this is not driving down costs, said Dr. Tharaldson.
Much of the market is focused on orphan drugs, as two-thirds of all specialty drugs in development are for orphan drugs. The leading specialty therapy pharmacy spending classes (per 2017 per-member, per-year costs) are inflammatory conditions, oncology, multiple sclerosis, HIV, and hepatitis C. Despite much of the oncology spending going through the medical benefit, this therapeutic area remains at the No. 2 spot for pharmacy spending, she noted.
In 2017, the FDA approved a “record” 36 specialty medications. The big approval was for two new chimeric antigen receptor (CAR) T-cell agents, and Dr. Tharaldson said we will begin to see allogeneic—or “off-the-shelf”—options, which could bring down the costs.
So far this year, 11 specialty drugs have been approved. Dr. Tharaldson forecast some of the coming approvals that are likely to come down the specialty pharmacy pipeline. Some highlights of her talk include:
- Cladribine is in the multiple sclerosis pipeline, with an expected approval date of 2019, despite being rejected twice by the FDA.
- Mogamulizumab, binemetinib/encorafenib, iobenguane I-131, lorlatinib, and ivosidenib are oncology drugs that are all expected to be approved by late summer.
- Lisocabtagene maraleucel is expected to be the next approval CAR T-cell therapy, indicated for B-cell non-Hodgkin lymphoma.
- A number of CGRP inhibitors for migraines are in the pipeline, with three expected for approval this year: erenumab, fremanezumab, and galcanezumab. “Up to 80% of patients discontinue available treatments for migraine within a year,” said Dr. Tharaldson. “So, there’s definitely a need for new agents in this space.”
- The hematology pipeline has a number of gene therapies in development, which are projected to receive approval between 2020 and 2022. These agents could eliminate the need for clotting factors, which could, over time, reduce the cost of this disease, said Dr. Tharaldson.
- Cannabidiol—purified cannabinoid—is in development for Lennox-Gastaut syndrome and Dravet syndrome, rare seizure disorders that develop in young children. Severe antiepileptic drugs can be used to treat these conditions, but they are not specifically approved for these indications. This agent could be approved in late June.
Presentation P1: Specialty Pharmaceuticals in Development. AMCP Annual Meeting 2018.