Last week, the FDA gave the green light to Novartis (NYSE:NVS) subsidiary AveXis's gene therapy Zolgensma, which treats spinal muscular atrophy (SMA). James Love is director of Knowledge Ecology International, a not-for-profit organization that advocates for access to medical treatments at affordable prices, and is currently a member of the board of directors of the Union for Affordable Cancer Treatment. 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But there’s more to the story. Along the way, all of these entities took on considerable financial risks, because of the potential for generating solid returns on their investment. Approved by the US Food and Drug Administration (FDA) in late May, Zolgensma treats infants suffering from a deadly genetic disease known as spinal muscular atrophy (SMA). But its record-high price of $2.125 million has sparked a broader conversation about drug pricing characterized by bemused practicality, scathing indignation, and everything in between. Founded in 1993 by brothers Tom and David Gardner, The Motley Fool helps millions of people attain financial freedom through our website, podcasts, books, newspaper column, radio show, and premium investing services. That's almost certainly an overstatement. Zolgensma From Novartis Is The Most Expensive Drug Ever Approved : Shots - Health News The Food and Drug Administration approved a new gene therapy for … But if you're still bothered by Zolgensma's extraordinary price tag, there's some good news. However, drugmakers actually weigh several factors in establishing those prices. That’s where Novartis’ Zolgensma comes into the picture. “She will always have SMA and she will always live with a disability but hopefully now she will have the best chance to have a healthy life,” Lucy’s parents wrote Thursday. Around 30 babies are born each month with SMA. Knowledge Ecology International and the Union for Affordable Cancer Treatment have both petitioned the NIH and other federal agencies to use their rights regarding federally funded inventions to curb excessive prices. Though the numbers are big, this looks like a straightforward math problem. But unlike Zolgensma, which is administered just once, Spinraza must be taken for life. © 2020 Global News, a division of Corus Entertainment Inc. Lucy needed access to the very expensive drug to stop the progression of SMA. Fewer than 10% of all drugs that begin phase 1 clinical testing go on to win regulatory approval. The American Council on Science and Health is a research and education organization operating under Section 501(c)(3) of the Internal Revenue Code. Read all about the drug, its use, side effects on FDA's site. Had Novartis priced Zolgensma much higher, say $20 million per patient, it seems likely that it would have encountered significant pushback, with insurers opting to cover only Biogen's Spinraza. Thank You, Trial Lawyers, For Protecting Us from the Scourge of Baby Powder. vs Borussia Dortmund - Match 198, Paris Saint-Germain beat Borussia Dortmund (2 - 0), International Clubs UEFA Champions League 19/20, Atletico Madrid beat Liverpool FC (3 - 2), Atalanta Bergamasca Calcio beat CF Valencia (4 - 3). 95% of Baby Food Tainted with Toxic Metals? In his second article of a series, Harvard's Dr. Chris Gerry expands the discussion of his first piece, "The Science of Zolgensma, The World’s Most Expensive Medicine" and considers whether Zolgensma merits a price tag of $2.125 million. Thank You, Trial Lawyers, For Protecting Us from the Scourge of Baby Powder. Children’s Foundation and keep some of the funds for Lucy’s future medical needs. Zolgensma is Novartis’ new gene therapy drug for treating paediatric patients of SMA who are less than two years of age. Helping Smokers Quit: The Science Behind Tobacco Harm Reduction, Foods Are Not Cigarettes: Why Tobacco Lawsuits Are Not a Model for Obesity Lawsuits, The Prevention and Treatment of Osteoporosis: A Review. Let’s hope we don’t learn the exact answers to those questions for a very, very long time. These 4 Drugmakers Are Using AI to Improve Their Products. The risk of failure was very high. It is designed to halt progressive spinal muscular atrophy (SMA) – motor neurone loss and muscle wasting – in people who have a defective version of the gene SMN1. In the U.S., government, private, and public health insurance providers are limiting coverage for Zolgensma because of its record-breaking and access-blocking price. At what price does innovation become too costly? Instead, NCH licensed its SMA program to AveXis in exchange for stock. At Harvard, Dr. Gerry contributed over 30 articles to the Science in the News blog, and he served as its Co-Editor-in-Chief from 2017-2019. Pet-Friendly Diwali 2020 Tips: Know How to Take Care of Pet Animals and Strays During This Festive Celebrations, #TwitterBan Trends on Twitter, Netizens Share Funny Memes and Jokes After India Serves Notice to Micro-Blogging Site For Showing Leh in Jammu and Kashmir on Map, Dinosaurs Are Back? Unsurprisingly, the drug’s $2.125 million price tag—the highest for any medicine in the world—has prompted the raising of eyebrows and wringing of hands across the country. While the consequences of SMA are dramatic and widespread, the fact that it’s caused by harmful mutations in a single gene renders it vulnerable to one of the most rapidly evolving fields of medicine: gene therapy. The family had started the fund with the goal of raising $3 million for Zolgensma, a drug often described as the most expensive in the world. One thing this hefty sum won’t get you, however, is a new drug called Zolgensma. More than 200 drugs for rare diseases have been approved since 1983, signaling an industry-wide shift in focus. The Ascent is The Motley Fool's new personal finance brand devoted to helping you live a richer life. Don't Panic, Everything Goes to Pot: Myths Are Driving FDA, CDC Vaping Policy, What the Hulk? SMA is the top genetic cause of death in infants and toddlers. A highly specialized tool used only on a few specific aircraft will cost a lot more than a regular screwdriver. Another key factor pharmaceutical companies use as a guide in setting prices is the value provided by the drug. Should You Worry About Artificial Sweeteners? At Harvard, Dr. Gerry contributed over 30 articles to the Science in the News blog, and he served as its Co-Editor-in-Chief from 2017-2019. Copyright © Latestly.com All Rights Reserved. Get a roundup of the most important and intriguing stories, delivered to your inbox, B.C. These incentives appear to be working, but perhaps too well. Image: Muscular Dystrophy Association, Why Americans Hate Big Pharma More Than Ever, Genetically Modified People: Gene Editing Arrives in the Clinic, Metastasis: Cancer's Invasion of the Body, When Cancer Strikes Back: Fighting Drug Resistance, Why Pfizer's Vaccine Must Be Stored in the Deep, Deep Freeze, Governors Cuomo, Newsom Play Politics with Pfizer's Life-Saving COVID Vaccine. Spinal muscular atrophy is a rare genetic neuromuscular disease: In the U.S., between 10,000 and 25,000 people have the condition. Lucy Van Doormaal received a dose of Zolgensma at B.C. Doing so targets the root cause of genetic diseases directly rather than alleviating their symptoms. We're used to a system of a chronic medication where we spread things out over years if not decades.". The United States spends an exorbitant amount on healthcare, and the high price for prescription drugs—especially compared to other countries—is a one of the primary contributors. babies who need $3-million life-saving drug – Aug 9, 2020, Ujiri hopes Raptors can play in Toronto as COVID-19 cases spike, Are the holidays cancelled? Answer #3: The ability to set a high price for new drugs incentivizes biomedical innovation. And when lawsuits are brought, the court system isn’t exactly known for its speed. Dr. Christopher Gerry was recently awarded a Ph.D. from the Department of Chemistry & Chemical Biology at Harvard University, where he studied the science of therapeutics using a combination of synthetic organic chemistry, chemical biology, and small-molecule screening. The only other treatment for SMA is a drug called Spinraza (made by Biogen). Novartis Gets US Approval for USD 2.1 Million Gene Therapy. Novartis said the drug is priced at half of the estimated $4 million-plus cost of managing the disease with therapy for one decade. But “least expensive” is a relative term. After five years of treatment, Spinraza's total cost exceeds that of Zolgensma. The first year of Spinraza costs $750,000, and all subsequent years cost $375,000, so five years of Spinraza ($2.25 million) costs more than one dose of Zolgensma ($2.125 million). We raise our funds each year primarily from individuals and foundations. Thus, the big reason why Novartis set the price for Zolgensma so high: Its addressable market is minuscule. There's a new drug that just won approval from the Food and Drug Administration (FDA) that will run more than $2.1 million per patient, making it the most expensive drug ever. Both Novartis and Biogen could face a new rival in the not-too-distant future. This photo provided by Novartis shows its gene therapy medicine, Zolgensma. (The above story first appeared on LatestLY on May 27, 2019 12:45 PM IST. COPYRIGHT © 1978-2020 BY THE AMERICAN COUNCIL ON SCIENCE AND HEALTH. People with certain types of SMA can’t stand or walk without being aided. Probably the best solution for controlling high drug prices is increased competition. In general, the fewer people who could benefit from the drug, the higher its price tag will be. If there’s one thing this exercise has taught me so far—and if I haven’t used up my allowance of Oscar Wilde quotes already—it’s that “the truth is rarely pure and never simple.”. Jerry Mendell, a doctor involved in the trial said, "The level of efficacy, delivered as a single, one-time therapy, is truly remarkable and provides a level of unprecedented hope for families.". The first year of Spinraza costs $750,000, and all subsequent years cost $375,000, so five years of Spinraza ($2.25 million) costs more than one dose of Zolgensma ($2.125 million). His background includes serving in management and consulting for the healthcare technology, health insurance, medical device, and pharmacy benefits management industries.