Orphan Drug Sales Set to Soar During Next 4 Years, Report Says

The top orphan drug therapeutic areas are blood, central nervous system, and respiratory, which account for more than 50% of the nononcology orphan drug market, according to a report from pharmaceutical intelligence firm Evaluate. In addition, the orphan drug market will grow from a 12% compound annual growth rate (CAGR) to 2024, double the 6% rate expected for nonorphan products, driven by new advancements.

Looking ahead, the recently released report predicts that sales of Roche Holding AG’s reference product, Rituxan (rituximab), will fall at the highest CAGR (—15.3%), from $6.9 billion in 2018 to $2.6 billion in 2024, as biosimilar competition takes hold and some patent protections begin to expire.

Upcoming competition includes CT-P10, which will be sold under the name Truxima by Celltrion and Teva for oncology indications only—a so-called “skinny label.” Roche retains patent exclusivity on some indications; those patents begin expiring through 2029.

The report said a number of “one and done” treatments for orphan diseases are approaching the market, including Novartis’s AVXS-101 (Zolgensma), a treatment for a rare, progressive, fatal neuromuscular disease in infants called spinal muscular atrophy type 1, and bluebird bio’s treatment for a type of sickle cell disease.

By 2024, orphan drugs are expected to reach $242 billion in sales and capture one-fifth of worldwide prescription sales.

In the United States, the median price differential decreased between orphan and nonorphan drugs by almost 50% in the last 4 years, but the mean orphan drug cost per patient of the top 100 US orphan drugs was almost 4.5 times greater than the nonorphan drug cost last year. The mean cost per patient per year of the top 100 orphan products was $150,854 in 2018 compared with $33,654 for nonorphan drugs.

One of the most expensive orphan drug products, reference eculizumab (Soliris), had the highest revenue per patient per year in 2018.

By 2024, the report predicts that reference ibrutinib (Imbruvica) will become the world’s top-selling orphan drug.