More Generics, Biosimilars, and Orphan Drugs on the Horizon

Boston—According to a presentation at the AMCP meeting, as patented products expire, >$13 billion in brand-name specialty medications are expected to face generic competition in the next 5 years, and it is projected that there is a $31.8 billion opportunity in the market for biosimilars through 2020. Aimee Tharaldson, PharmD, senior clinical consultant, emerging therapeutics, Express Scripts, presented the information, discussing trends, recent specialty drug approvals, and the specialty drug pipeline.

Specialty medications, which are defined by Express Scripts as drugs that require frequent dosing adjustments, intensive patient training, limited distribution, or specialized handling, are an increasingly prevalent part of the drug industry. In 2013, 19 specialty drugs were approved compared to just 8 traditional drug approvals. “Specialty medications represented 70% of all FDA drug approvals last year,” said Dr. Tharaldson.

The largest area of growth in recent years has occurred in the oncology market, which according to Dr. Tharaldson accounts for approximately 30% of all drugs in the specialty pipeline. Aside from 9 new oncology drug approvals in 2013, an additional 5 drugs have been approved so far this year.

The market is expected to see an increase in competition. According to the presentation, 39 specialty products will have patents expiring at the end of 2018, and by the end of 2020, patents on 51 additional biologic products will also expire, leaving significant opportunities for both generic and biosimilar medications.

While there are currently no biosimilar agents approved in the United States, 2 drugs are pending approval. Filgrastim, a drug used for neutropenia, is expected to be approved in March 2015, while infliximab, a drug used to treat rheumatoid arthritis and other inflammatory conditions, is expected to gain approval in August 2015. According to Dr. Tharaldson, the introduction of biosimilars will not only increase competition, but it will also provide an opportunity for significant cost savings within several specialty therapy classes.

Another trend is the growing prevalence of orphan drugs, or drugs used to treat conditions effecting <200,000 US citizens. These medications currently comprise 35% of the specialty drug pipeline. “Medications to treat orphan conditions are typically very expensive, costing several hundred-thousand dollars per year,” said Dr. Tharaldson. “Approximately 30% of drugs are blockbusters, with annual sales of at least $1 billion per year, so many manufacturers are focusing development efforts on orphan drugs.”

In addition to orphan drugs, the industry is also seeing medications receive breakthrough therapy designation from the FDA to help expedite the development and review of those drugs. To receive the designation, manufacturers must demonstrate that the drug treats a serious or life-threatening disease and has early evidence that suggests it would provide a substantial improvement over existing therapies.

Dr. Tharaldson also discussed recent and pipeline drugs, particularly noting some of the most significant approvals this year. Pembrolizumab was the first PD-1 inhibitor to be approved for treatment of advanced melanoma and is a significant development for the industry. PD-1 inhibitors are used to stimulate a patient’s immune system to attack cancer cells. In addition, the approval of combination ledipasvir/sofosbuvir, which is used to treat genotype 1 hepatitis C through a once daily, single-tablet oral regimen, was significant. Dr. Tharaldson said she believes there will be great demand for this drug, which is priced at $94,500 for a 12-week treatment course. Finally, pirfenidone and nintedanib were both recently approved to treat idiopathic pulmonary fibrosis and are expected to carry an annual cost of $94,000 per patient.

Dr. Tharaldson discussed some pipeline trends, including new injectable biologic agents for inflammatory conditions, leading competition from biosimilars and psoriasis development. In the multiple sclerosis pipeline, the generic drug glatiramir acetate is expected to receive approval within the next few months. Competition from oral drugs is also anticipated in the oncology pipeline, along with breakthrough therapies, targeted therapy, and immunotherapy development.—Jill Sederstrom