Drug Discovery and Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Genomics/Proteomics
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • Orphan Drugs
  • R&D 100 Awards

Estimating the Efficacy and Cost of Curative Gene Therapy for Beta-Thalassemia

By Mary Ann Liebert, Inc./Genetic Engineering News | May 10, 2019

Gene therapy offers the promise of a cure for beta-thalassemia and a new study has shown that it is associated with fewer complications and hospital admissions over 2 years than treatment by allogeneic hematopoietic stem cell transplantation (HSCT). The study, which analyzes and compares the effectiveness and cost of gene therapy versus (HSCT) in patients with major beta-thalassemia is published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. Click hereto read the full-text article free on the Human Gene Therapy website through May 18, 2019.

Séverine Coquerelle, URC Eco-Assistance Publique Hôpitaux de Paris, Université Paris Diderot (Sorbonne Paris Cité), and CRESS, INSERM UMR (Paris), France led a team of French researchers in publishing the article entitled “Innovative Curative Treatment of Beta Thalassemia: Cost-Efficacy Analysis of Gene Therapy Versus Allogenic Hematopoietic Stem-Cell Transplantation.” Patients treated with HSCT had 3 times more frequent infectious complications. Gene therapy was shown to be about 2.8 times more costly, with nearly half the cost of gene therapy accounted for by preparation of the delivery vector.

“There has been much discussion and controversy about the high cost of gene therapy, but what has been lacking is a direct comparison to alternative therapies, which them-selves are also often very costly and may produce suboptimal outcomes,” says Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Med-ical School, Worcester, MA. “This study does a critical comparison of both cost and out-comes for patients with beta-thalassemia, whose primary alternative to gene therapy would be hematopoietic stem cell transplantation. These data may enable a more rational debate of the overall value of gene therapy for this relatively common genetic disease.”

Related Articles Read More >

FDA releases guidance to speed personalized drug development
Pigs
FDA OKs intentional genomic alteration in pigs — with potential therapeutic applications
Oxford Gene Technology
Oxford Gene Technology expands next-generation sequencing line
cleveland-clinic-disruptors-2021
The top 10 medical disruptors of 2021

DeviceTalks Tuesdays

DeviceTalks Tuesdays

MEDTECH 100 INDEX

Medtech 100 logo
Market Summary > Current Price
The MedTech 100 is a financial index calculated using the BIG100 companies covered in Medical Design and Outsourcing.

Need Drug Discovery news in a minute?

We Deliver!
Drug Discovery & Development Enewsletters get you caught up on all the mission critical news you need. Sign up today.
Enews Signup

R&D Twitter

Tweets by @RandDWorld
Drug Discovery and Development
  • Enews Signup
  • Contact Us
  • R&D World
  • Pharmaceutical Processing
  • Drug Delivery Business News

Copyright © 2021 WTWH Media LLC. All Rights Reserved. The material on this site may not be reproduced, distributed, transmitted, cached or otherwise used, except with the prior written permission of WTWH Media
Privacy Policy | Advertising | About Us

Search Drug Discovery

  • Home Drug Discovery and Development
  • Drug Discovery
  • Genomics/Proteomics
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • Orphan Drugs
  • R&D 100 Awards