The orphan drug market is expected to increase at a 12% compound annual growth rate (CAGR) to reach $224bn by 2024, according to a report from the pharma-analysis group Evaluate Pharma.
The report attributes this growth to breakthrough one-shot treatments such as Novartis’ Zolgensma, and to the development of cell and gene therapy drugs such as Kite’s CAR-T Yescarta.
“Seven of the top 20 R&D orphan products by net present value are either a cell or a gene therapy.”
This growth is also attributed to favourable conditions in the orphan drugs market, due to generous incentives and high prices commanded during reimbursement processes. However there has recently been increased scrutiny of industry prices, with an industry watchdog urging Novartis to revise their pricing strategy for Zolgensma.
“Growth rates approximately double those of the overall prescription drugs market and the mean cost per patient per year of the top 100 orphan products in the US hitting $150,854 in 2018 versus $33,654 for a non-orphan drug, explain much of the allure.”
By contrast, non-orphan drugs are expected to grow by a respectable 6% CAGR until 2024.
For further information, please read Evaluate Pharma’s full report here.