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A newly approved gene therapy for children with spinal muscular atrophy will cost more than $2 million for a one-time infusion.Medscape Reader Polls
Source: Medscape Business of Medicine Headlines - Category: Pharmaceuticals Tags: Internal Medicine News Source Type: news

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CONCLUSION: Gene therapies have the potential to significantly influence the course of neuromuscular diseases. First positive intermediate results have been published and the first treatment has recently been approved in the USA. Long-term data on sustained effects and toxicity of gene therapies are not yet available. These novel treatment options will present new challenges for the healthcare systems concerning diagnosis, treatment and reimbursement. PMID: 31286145 [PubMed - as supplied by publisher]
Source: Der Nervenarzt - Category: Neurology Authors: Tags: Nervenarzt Source Type: research
AbstractOnasemnogene abeparvovec (onasemnogene abeparvovec-xioi; formerly AVXS-101; ZOLGENSMA®) is an adeno-associated viral vector-based gene therapy designed to deliver a functional copy of the humansurvival motor neuron (SMN) gene to the motor neuron cells of patients with spinal muscular atrophy (SMA). It has been developed by AveXis, a Novartis company, and was approved in May 2019 in the USA for the treatment of paediatric patients aged  
Source: Drugs - Category: Drugs & Pharmacology Source Type: research
Gene Therapy, Published online: 26 June 2019; doi:10.1038/s41434-019-0085-4AAV9 Vector: a Novel modality in gene therapy for spinal muscular atrophy
Source: Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Abstract Introduction Genetic neuromuscular diseases (NMDs) constitute a heterogeneous group of rare conditions, including some of the most disabling conditions in childhood. Over the last decade, early diagnosis, multidisciplinary care and anticipatory treatment strategies have improved survival and quality of life of several conditions. Recently, advanced technologies have greatly expanded preclinical and clinical research, and specific therapies have been introduced for three diseases, namely enzyme replacement therapy for Pompe disease (PD), gene expression modulation and gene therapy for Duchenne muscular dys...
Source: Pharmacological Reviews - Category: Drugs & Pharmacology Authors: Tags: Expert Rev Clin Pharmacol Source Type: research
Novartis said it plans to price its recently approved gene therapy at $2.125 million, making it the world’s most expensive medicine. www.wsj.com Novartis slaps $2M-plus pricetag on newly approved gene therapy Zolgensma—and cost watchdogs approve Industry watchers have been speculating for months about just how high Novartis would price gene therapy Zolgensma—and just how payers would cover the "transformative" spinal muscular atrophy trea...
Source: Student Doctor Network - Category: Universities & Medical Training Authors: Tags: Topics in Healthcare Source Type: forums
With the recent FDA approval, Zolgensma became the  world’s most expensive medication. Priced at $2.125 million per patient, the one-dose gene therapy is a potential life-saver for children with spinal muscular atrophy (SMA). Now, the treatment is at the center of an intensifying debate over the rising price of medications. Industry watchdogs ar e outraged. They say Zolgensma […]Find jobs at  Careers by KevinMD.com.  Search thousands of physician, PA, NP, and CRNA jobs now.  Learn more.
Source: Kevin, M.D. - Medical Weblog - Category: General Medicine Authors: Tags: Meds Medications Neurology Source Type: blogs
The University of Rochester Medical Center has been tapped as one of the first institutions in the U.S. to offer a new gene replacement therapy to treat spinal muscular atrophy. The new treatment, which is being marketed by Novartis under the brand name Zolgensma, can be delivered within weeks of birth and clinical trials have shown that it dramatically changes the course of the disease.
Source: University of Rochester Medical Center Press Releases - Category: Universities & Medical Training Authors: Source Type: news
Zolgensma: First Gene Therapy for Pediatric Spinal Muscular Atrophy (SMA) Zolgensma (onasemnogene abeparvovec-xioi) from AveXis (a Novartis company), is now approved for children less than 2 years of age with spinal muscular atrophy (SMA). SMA...
Source: Drugs.com - Pharma News - Category: Pharmaceuticals Source Type: news
At $2,125,000, it is the most expensive drug in the world
Source: Chemical and Engineering News - Category: Chemistry Authors: Source Type: research
AskBio continues to lead the path for gene therapy development and cost-effective manufacturing methods based on the contributions of its co-founder, Dr. Jude Samulski.(PRWeb May 30, 2019)Read the full story at https://www.prweb.com/releases/newly_approved_spinal_muscular_atrophy_gene_therapy_zolgensma_validates_askbio_gene_therapy_platform/prweb16344912.htm
Source: PRWeb: Medical Pharmaceuticals - Category: Pharmaceuticals Source Type: news
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