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LONDON, BOSTON, TOKYO (April 25, 2019) – Scientific advancements will help propel the orphan drug market to a 12% CAGR to 2024, double the 6% rate expected for non-orphan products. In addition to recent breakthrough cell and gene therapy products like Yescarta and Luxturna, a number of “one and done” treatments for orphan diseases are approaching the market, including Novartis’s Zolgensma and bluebird bio’s Lentiglobin.
The explosion of new technologies brings their high price points into greater focus — for example, the $425,000 per eye list price for Spark’s Luxturna is the value-based price agreed on with Spark and payers. In 2018, the mean cost for an orphan drug was 4.5 times that of a non-orphan product.
Despite the clear advancements in treatment, however, those calling for reform of the Orphan Drug Act may gain further ammunition. Big pharma continues to entrench their position in the orphan market, as evidenced by Bristol-Myers Squibb’s bid for Celgene, Roche’s pending acquisition of Spark, and Novartis’s $8.7bn payment for Zolgensma.
“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, PhD. “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.”
Additional report highlights include:
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