Orphan Drug Sales Projected to Reach $262 Billion by 2024

EvaluatePharma recently released its take on the current state of the market for orphan drugs in its Orphan Drug Report for 2018.

The Orphan Drug Act of 1983 incentivized the development of drugs to treat rare diseases because such drugs were not considered financially sustainable due to the small number of patients who are affected. In the United States, orphan drugs for rare diseases are made to treat fewer than 200,000 patients.

“According to this year’s report, sales of orphan drugs are set to climb by 11% a year all the way through to 2024—this compares with 6.4% growth for the overall pharma market in the same period,” read the report.

Celgene earned the distinction of the pharmaceutical company with the largest orphan drug sales due to lenalidomide (Revlimid), through its multiple myeloma indication, earning the company $8.1 million in 2017 alone. By 2024, the drug is projected to reach $12 billion in sales worldwide.

Though Celgene produced the most sales from its orphan drug, Alexion pharmaceuticals maintained the designation of the most expensive orphan drug, eculizumab (Soliris), for the treatment of paroxysmal nocturnal hemoglobinuria and myasthenia gravis. EvaluatePharma projects the drug to reach $5.3 billion in sales by 2024. Currently, Amgen is developing a biosimilar eculizumab candidate.

In addition, the report found that the mean price of the top 100 orphan drugs in the United States (as ranked by sales) grew at an annual rate of 5.2% from 2013 to 2017, compared with 9.2% for the top 100 non-orphan drugs.

Finally, EvaluatePharma estimates that oncology will be the leading orphan drug therapeutic area in 2024, making up about 50% of the total 2024 market worldwide. The second leading area is blood disorders, which is estimated to account for about 12% of the market. By 2024, orphan drugs are projected to capture one-fifth of worldwide prescription sales, reaching nearly $262 billion.

“The implementation of the FDA’s orphan drug modernization plan has led to an increase in the number of orphan designations granted in the United States. Overall, the FDA has granted more orphan drug designations than any other regulatory body,” read the report.