{"id":127073,"date":"2021-09-01T21:22:19","date_gmt":"2021-09-02T04:22:19","guid":{"rendered":"https:\/\/lifeboat.com\/blog\/2021\/09\/new-gene-therapies-may-soon-treat-dozens-of-rare-diseases-but-million-dollar-price-tags-will-put-them-out-of-reach-for-many"},"modified":"2021-09-01T21:22:19","modified_gmt":"2021-09-02T04:22:19","slug":"new-gene-therapies-may-soon-treat-dozens-of-rare-diseases-but-million-dollar-price-tags-will-put-them-out-of-reach-for-many","status":"publish","type":"post","link":"https:\/\/lifeboat.com\/blog\/2021\/09\/new-gene-therapies-may-soon-treat-dozens-of-rare-diseases-but-million-dollar-price-tags-will-put-them-out-of-reach-for-many","title":{"rendered":"New gene therapies may soon treat dozens of rare diseases, but million-dollar price tags will put them out of reach for many"},"content":{"rendered":"<p><a class=\"aligncenter blog-photo\" href=\"https:\/\/lifeboat.com\/blog.images\/new-gene-therapies-may-soon-treat-dozens-of-rare-diseases-but-million-dollar-price-tags-will-put-them-out-of-reach-for-many2.jpg\"><\/a><\/p>\n<p>Why not eradicate disease for everyone?<\/p>\n<hr>\n<p>\n<a href=\"https:\/\/theconversation.com\/the-most-expensive-drug-in-the-world-how-it-works-and-the-devastating-disease-it-treats-164535\" rel=\"nofollow noopener\" target=\"_blank\" class=\"\">Zolgensma<\/a> \u2013 which treats <a href=\"https:\/\/www.mda.org\/disease\/spinal-muscular-atrophy\" rel=\"nofollow noopener\" target=\"_blank\" class=\"\">spinal muscular atrophy<\/a>, a rare genetic disease that damages nerve cells, leading to muscle decay \u2013 is currently the most expensive drug in the world. A one-time treatment of the life-saving drug for a young child <a href=\"https:\/\/www.npr.org\/sections\/health-shots\/2019\/05\/24\/725404168\/at-2-125-million-new-gene-therapy-is-the-most-expensive-drug-ever\" rel=\"nofollow noopener\" target=\"_blank\" class=\"\">costs US$2.1 million<\/a>.<\/p>\n<p>While Zolgensma\u2019s exorbitant price is an outlier today, by the end of the decade there\u2019ll be dozens of cell and gene therapies, costing hundreds of thousands to millions of dollars for a single dose. The Food and Drug Administration <a href=\"https:\/\/www.fda.gov\/news-events\/press-announcements\/statement-fda-commissioner-scott-gottlieb-md-and-peter-marks-md-phd-director-center-biologics\" rel=\"nofollow noopener\" target=\"_blank\" class=\"\">predicts that by2025it will be approving 10 to 20 cell and gene therapies<\/a> every year.<\/p>\n<p>I\u2019m a <a href=\"https:\/\/www.kevindoxzen.com\/\" rel=\"nofollow noopener\" target=\"_blank\" class=\"\">biotechnology and policy expert<\/a> focused on improving access to cell and gene therapies. While these forthcoming treatments have the potential to save many lives and ease much suffering, health care systems around the world aren\u2019t equipped to handle them. Creative new payment systems will be necessary to ensure everyone has equal access to these therapies.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Why not eradicate disease for everyone? Zolgensma \u2013 which treats spinal muscular atrophy, a rare genetic disease that damages nerve cells, leading to muscle decay \u2013 is currently the most expensive drug in the world. A one-time treatment of the life-saving drug for a young child costs US$2.1 million. While Zolgensma\u2019s exorbitant price is an [\u2026]<\/p>\n","protected":false},"author":621,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[11,412,1495,31],"tags":[],"class_list":["post-127073","post","type-post","status-publish","format-standard","hentry","category-biotech-medical","category-genetics","category-health","category-policy"],"_links":{"self":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/127073","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/users\/621"}],"replies":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/comments?post=127073"}],"version-history":[{"count":0,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/127073\/revisions"}],"wp:attachment":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/media?parent=127073"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/categories?post=127073"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/tags?post=127073"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}