Background The past 10 years witnessed a significant increase in the approval of cellular and gene therapy products worldwide. The US Food and Drug Administration (FDA) approved 3 gene therapy products within the last 4 months of 2017. The objective of this study was to examine the approval characteristics, discontinuations and cost of all cellular and gene therapy products approved worldwide. Data and Methods We conducted an electronic search of approved cell and gene therapy products from the databases of the main drug regulatory agencies including the US Food and Drug Administration, the European Medicines Agency (EMA), the Korea Ministry of Food and Drug Safety (MFDS), and Japan’s Pharmaceuticals and Medical Devices Agency, China Food and Drug Administration, Ministry of Health and Social Development-Russia, Health Canada, and the Food and Drug Administration of the Philippines. We also searched the literature using MEDLINE/PubMed, Cochrane Library, Google Scholar and EMBASE databases. Cost information from the US was derived from the Reb Book (Truven Health Analytics). Costs from Europe were derived from Health Technology Assessments and from public sector, financial news and company’s web pages. Where available, we also obtained cost for products approved in other countries from public sector, financial news and company’s web pages. All cost data were converted to USD. Results There were 52 cell and gene therapy products marketed in the world as of September 2018. Of these, 39 (75.0%) were cell therapy medicinal products and 13 (25.0%) were gene therapy medicinal products. Of the approved products, 29 (74.3%) cell and 8 (61.5%) gene therapy products were first approved in the past 10 years (2008-present). Korea had the greatest number of approved cellular therapy-19 followed by US-12, Japan -4, Europe -3 and Canada -1. While Europe had the highest number of approved gene therapies - 5 followed by US - 3, China -2 and Korea, Russia and Philippines one each. Overall, 8 (66.7%) of the approved gene therapy products were granted orphan designation. Three products approved by the EMA were withdrawn or discontinued from the market. Of them, 2 remain currently marketed in the US. Cost of treatment from Gene therapy in US ranged from $447600 to $1,020,000 while in Europe the range was $370000 to $962890. Gene therapies outside US and Europe were relatively cheaper. Cell therapies in North America had a price range from $30,370-$200,000, European cell therapies ranged from $17,658-$105,000. Conclusion In the study period, more autologous cell therapy products were approved than allogenic agents. The cost of gene and cell therapy drugs is much higher in the EU and North America in comparison to Asia. Majority of the gene therapies received orphan designation by the regulatory authorities and had conditional approvals while less than a quarter of cellular therapies received the orphan designation. Most of the gene therapies are approved for rare diseases with smaller patient population, companies find it difficult to make profits which results in market withdrawal of the therapies.
Shukla V, Seoane-Vazquez E, Fawaz S, Brown LM, Rodriguez-Monguio R. The landscape of cellular and gene therapy products: Cost, approvals, and discontinuations. Hum Gene Ther Clin Dev. 2020;30(3):102-113 https://doi.org/10.1089/humc.2018.201
Mary Ann Liebert
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This is a pre-copy-editing, author-produced PDF of an article accepted for publication in Human Gene Therapy Clinical Development, volume 30, issue 3, in 2019 following peer review. The definitive publisher-authenticated version is available online at DOI:10.1089/humc.2018.201.