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The "One and Done" Era for Orphan Drugs: Evaluate Predicts 12% Growth, Driven by New Technologies, but Benefits Go to Big PharmaScientific 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 repot highlights include:
Download your complimentary copy of the "EvaluatePharma® Orphan Drug Report 2019" at http://www.evaluate.com/OrphanDrug2019. About Evaluate Ltd. Evaluate provides trusted commercial intelligence for the pharmaceutical and medical device industries. Our EvaluatePharma® and EvaluatePharma Vision online subscription services provide a seamless view of the past, present and future of the global pharmaceutical market in a single, standardised platform. Vantage - our award-winning, independent editorial team - provides thought-provoking news and insights into the current and future developments in the industry. Evaluate has been a partner to industry-leading organisations for over 20 years. For more information on how we give our clients the time and understanding to drive better decisions, visit www.evaluate.com. Follow us on Twitter (News - Alert): @EvaluatePharma, @Vantageanalysis
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