Drugs Don’t Work If People Can’t Afford Them: The High Price Of Tisagenlecleucel
By Paul Kleutghen, David Mitchell, Aaron S. Kesselheim, Mehdi Najafzadeh, and Ameet Sarpatwari,
HealthAffairs
| 02. 08. 2018
In a system in which life-saving drugs are developed with direct and indirect taxpayer support and afforded market protection through government-granted exclusivities, patients deserve to know how drug manufacturers are arriving at ever-higher prices for their products. Without such information—and subsequent policy reforms based on it—treatment will become increasingly unaffordable. Sadly, for many Americans, it already has; 21 percent of 1,204 respondents in a December 2016 Henry J. Kaiser Family Foundation survey reported that they or a family member did not fill a prescription in the past year because of cost.
Why Tisagenlecleucel Matters
One of the most recent examples highlighting the prescription drug price debate is the chimeric antigen receptor T-cell (CAR-T) immunotherapy tisagenlecleucel (Kymriah). Widely hailed as a groundbreaking treatment, it was approved by the Food and Drug Administration (FDA) in August 2017 for the treatment of pediatric and young adult acute lymphoblastic leukemia. In coming years, tisagenlecleucel is expected to receive marketing approval in other countries and for other indications, including adult acute lymphoblastic leukemia, chronic lymphocytic leukemia, diffuse large B-cell lymphoma, and small lymphocytic lymphoma...
Related Articles
By Ian Sample, The Guardian | 07.04.2024
Biological models of human embryos that can develop heartbeats, spinal cords and other distinctive features will be governed by a code of practice in Britain to ensure that researchers work on them responsibly.
Made from stem cells, they mimic, to...
By Kevin Davies, Genetic Engineering & Biotechnology News | 06.27.2024
Physician-scientist Matthew Porteus, MD, PhD, has been a mainstay in the genome editing field for more than two decades. He trained at Stanford University Medical School before completing his residency and hematology/oncology fellowship at Boston Children’s Hospital/Dana Farber Cancer Institute...
By Peter Aldhous, Scientific American | 07.02.2024
In June a notice posted on the website of the journal Nature set a new scientific record. It withdrew what is now the most highly cited research paper ever to be retracted.
The study, published in 2002 by Catherine Verfaillie...
By Robert F. Service, Science | 07.04.2024
Image by Ed Uthman from Flickr
Last year, the U.S. Food and Drug Administration (FDA) approved two gene therapy procedures that can treat and, in some cases essentially cure sickle cell disease, a genetic blood disorder that causes pain and...