In a session examining the specialty pharmaceutical pipeline at AMCP 2024, speakers reviewed recently approved high-impact specialty drugs, the mechanism of action and expected indications of promising agents in development, key considerations and cost implications, relevant FDA and legal developments, and more. 

FDA and Legal Considerations

1. Multiple patients on some medications may create a barrier to competition;
2. Biosimilar manufacturers may or may not launch when there is patient risk involved; and
3. The legal process of brand and biosimilar companies reaching settlement agreements on when competition may launch. 

Several FDA challenges were discussed regarding biosimilar and specialty launches, such as inspection delays and authorized unbranded biologics that create competition. Interchangeable exclusivity can also pose problems because of the incentive for biosimilar manufacturers to bring generics to market. 

Complete response letters (CRL) can affect specialty brand launches due to safety, efficacy results, or manufacturing issues, as well as incomplete FDA applications. Health professionals in the special pharmacy sector should also prepare for the potential of FDA advisory committee meetings. The FDA approved 55 New Molecular Entity (NME) in 2023 and the number of advisory meetings only seem to be increasing, according to the speakers.  

Reviewing the Data

The speakers reviewed data on FDA brand approvals between 2009 and 2023 and noted an increase in overall approvals. In 2009, there were only 35 total approvals, but in 2023, there were 72. 

Speakers discussed the drugs approved in 2023. The most notable were respiratory syncytial virus (RSV) vaccines Arexvy, the first vaccine approved to prevent RSV, and Abrysvo. Other specialty drugs highlighted were lecanemab (Leqembi), the first fully approved Alzheimer disease modifier; tofersen (Qalsody), the first therapy to target a genetic cause of amyotrophic lateral sclerosis; and zuranolone (Zurzuvae), which was the first oral option to treat postpartum depression. 

“In 2023, there were a lot of firsts. Moving into 2024, similar to many recent years, about 80% of the approvals are specialty,” said speaker Jeffrey Casberg, MS, RPh, Senior Vice President of Pharmacy, IPD Analytics, LLC. 

Another trend in the 2024 specialty pipeline was that many potential specialty drugs are for rare and orphan diseases. The overall leading drugs in the specialty pipeline are in the areas of neurology, oncology, endocrine and metabolic drugs, dermatology, hematology, and gastroenterology. Gene therapy cost was also noted, and the cost of drugs in this category are between $23,000 and$1,000,000 a year (as well as $1-3 million per treatment). 

To put things in perspective, the speakers provided a detailed overview of the drugs currently in the specialty pharmacy pipeline. Drugs discussed include biologics and biosimilars.

Specialty Drugs in the Pipeline

Biosimilar Specialty Drugs in the Pipeline

Cost Estimates

Speakers concluded with a few calculated estimates regarding the biosimilar market over the next 10 years. According to the speakers, the therapeutic area that will most likely produce the most biosimilar savings is inflammatory disease with treatments like ustekinumab and dupilimab (2023 estimated annual sales of $18 billion and $9 billion respectively). Biosimilars for inflammatory conditions could have a massive impact on the specialty medicine landscape. According to a press release from Express Scripts, inflammatory medications represented almost 25% of total drug spend across its commercial book of business in 2022, and competition created by biosimilars could save between $225 billion and $375 billion in total for the US pharmacy spend over the next decade. 

Other top candidates for savings was the oncology drug pembolizumab, the opthamolic drug aflibercept, and the type 2 diabetes drug dulaglutide. 
Speakers also noted that change could be coming for the FDA guidance on interchangeable biosimilars. 

“Currently, there are 10 products with interchangeability designation,” said Mr Casberg. “And the FDA is starting to wobble a little bit on their stance and trying to de-emphasize [interchangability] and making it just something that allows pharmacists to substitute at the pharmacy and a substitution issue instead of a clinical issue.” 

Reference 
Casberg J, Fish L. Specialty pharmaceutical pipeline 2024. Presented at: AMCP 2024; April 18, 2024; New Orleans, LA.