Breakthrough treatments will contribute to a 12% compound annual growth rate for orphan drugs through to 2024, according to a report by Evaluate Pharma. The group predicts these products, in addition to recently marketed cell and gene therapies, will cause the increase. Non-orphan treatments are also expected to grow by 2024, but by only 6% each year.
“Seven of the top 20 R&D orphan products by net present value are either a cell or a gene therapy,” said report author Karen Pomeranz. “However, we may not see the full market impact of these drugs by 2024, which means that the long-term growth and patient benefits could be even more pronounced.”
It is expected that bigger companies tapping smaller companies for orphan drugs will continue to dominate merger and acquisition activity, in line with recent trends.
“Growth rates approximately double those of the overall prescription drugs market and the mean cost for every patient a 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,” says the report.
This has resulted in increased scrutiny of industry prices, and highly favourable incentives for orphan drugs. The report warns the promise of innovative products “will only become reality if the innovation of drug companies is matched by innovation in the drug pricing and reimbursement systems”.
The recent explosion of cell and gene therapies has come with very high prices, with a number of drug pricing watchdog organisations advocating for fair pricing strategies.