Trends to Watch in Specialty Pharmaceuticals in Development

Last year, 51.5% of pharmacy spending was for specialty products as compared to 48.5% for traditional medications, according to a keynote presentation at AMCP Nexus 2023. Specialty drugs represent significantly more new-drug approvals than traditional medications. Therefore, it is important for managed care professionals to understand emerging trends and likely effects of new specialty medications on the market, said Aimee Tharaldson, PharmD, clinical program principal of emerging therapeutics at Evernorth.

During her keynote address, Dr. Tharaldson defined a specialty medication as a treatment that has limited distribution or requires one of the following:

  • Frequent dosing adjustments/intensive clinical monitoring.
  • Intensive patient training/compliance assistance.
  • Specialized handling/administration.

She reviewed the major disease categories that have the highest use of specialty medications and the most robust pipelines of new medications. The top category according to per-member-per-year spending is inflammatory diseases, including a variety of chronic, autoimmune diseases, such as rheumatoid arthritis, psoriasis, psoriatic arthritis, juvenile idiopathic arthritis, ankylosing spondylitis, Crohn’s disease, inflammatory bowel disease, ulcerative colitis, and lupus. Other major categories are multiple sclerosis, HIV, nonalcoholic steatohepatitis (also known as NASH), Alzheimer’s disease, paroxysmal nocturnal hemoglobinuria, Duchenne muscular dystrophy, hemophilia, and gene therapy.

Dr. Tharaldson also identified three key trends to watch in the specialty pharmaceutical space: biosimilar competition, cancer drug development, and treatments for orphan conditions. Biosimilars will become increasingly important in the specialty pharmaceuticals market because there are more than 60 patents scheduled to expire through 2027, she said. Specific brand-name drugs she highlighted that will be affected by biosimilar development are Humira, Remicaid, Lantus, and Rituxan.

The second trend, cancer specialty drugs, comes from the sheer number of Americans affected by the disease, she explained: 18 million Americans have a history of invasive cancer, and 1.9 million new cases are diagnosed each year in the United States. The first two trends overlap, she said, as biosimilars are becoming increasingly common in cancer treatment.

The third trend to watch in specialty pharmaceuticals is development of drugs for “orphan” diseases, defined as a condition that affects 200,000 or fewer people. Although each rare condition has a low prevalence, there are more than 7,000 orphan disease affecting 25 million to 30 million Americans. Drugs for orphan conditions represent 66% of the specialty drug pipeline, she added.


Tharaldson A. Specialty Pharmaceuticals in Development. Session K1. Presented at AMCP Nexus 2023; Oct. 16‒19, 2023; Orlando, Fla.