Eteplirsen: $300,000 per patient per year

Controversy Continues Over Muscular Dystrophy Drug, Despite FDA Approval
September 24, 2016
http://www.npr.org/sections/health-shots/2016/09/24/495174472/controversy-continues-over-muscular-dystrophy-drug-despite-fda-approval

Just 12 boys had been involved in the key study, and just about everybody agreed that the research was deeply flawed

In the meantime, Sarepta can start charging $300,000 per patient per year for treatment with eteplirsen.

Ovarian freezing

Twin Sisters Try To Get Pregnant With Ovaries They Froze In 2009
July 19, 2016
http://www.npr.org/sections/health-shots/2016/07/19/485838529/twin-sisters-try-to-get-pregnant-with-ovaries-they-froze-in-2009

Dr. Glenn Schattman, an associate professor of reproductive medicine at the Weill Medical College of Cornell University, says recommending ovarian freezing for women who don’t have cancer is “irresponsible.”

related:
http://www.npr.org/sections/health-shots/2012/12/24/167705397/chance-to-pause-biological-clock-with-ovarian-transplant-stirs-debate

Row over human embryo gene editing

Row over human embryo gene editing
24 April 2015
http://www.bbc.com/news/health-32446954

A controversial Chinese study that reveals genes in human embryos have been modified for the first time has sparked fierce debate.

The research looked at genetic editing techniques – which in theory can be used to snip out faulty bits of genetic material that would otherwise lead to serious inherited diseases.

This is the first time it is known to have been attempted on early human embryos. But the results suggest it can cause new, unintended genetic errors.

Experts are questioning whether the procedure – which, if taken further, could lead to genetic changes being passed on to future generations – has crossed ethical, moral and legal lines.

Gilead’s $1,000 Pill

Gilead’s $1,000 Pill Is Hard for States to Swallow
Hepatitis C drug Sovaldi strains programs for the poor; Texas didn’t pay for it last year
April 8, 2015
http://www.wsj.com/articles/gileads-1-000-hep-c-pill-is-hard-for-states-to-swallow-1428525426

A pricey pill made by Gilead Sciences Inc.  caused Medicaid spending on hepatitis C treatments to soar last year, even as most states restricted access to the drug, leaving many low-income patients untreated.

State Medicaid programs spent $1.33 billion on hepatitis C therapies through the third quarter of last year, or nearly as much as the states spent in the previous three years combined, a Wall Street Journal analysis of federal data shows.

The growth was primarily driven by Gilead’s Sovaldi, a highly effective therapy that has a wholesale cost of $84,000 per person over the course of treatment, or $1,000 per pill. The price has sparked an outcry from insurers, members of Congress and others worried about the cost of treating an estimated three million Americans with hepatitis C, which can lead to cirrhosis or cancer of the liver.

The data show patient access to Sovaldi varied widely state by state, reflecting different coverage of the drug and also long-standing disparities in how states deliver health benefits to the poor. Many states limited Sovaldi’s availability to the very sickest patients, primarily those with severe liver scarring.

Videos Ease Tough Conversation

Videos On End-Of-Life Choices Ease Tough Conversation
March 29, 2015
http://www.npr.org/blogs/health/2015/03/29/394087394/videos-on-end-of-life-choices-ease-tough-conversation

… these videos, produced by Dr. Angelo Volandes of Harvard Medical School. She thought maybe they could help. So she brought Volandes to Hawaii to give a little show-and-tell for some health care providers.

“I frankly was astounded,” Seitz says, “at how excited people became when they saw these videos.”

Volandes thinks they were excited and — maybe — a little bit relieved.

“Physicians and medical students aren’t often trained to have these conversations,” says Volandes. “I, too, had difficulty having this conversation, and sometimes words aren’t enough.”

Volandes is the author of a book called The Conversation, which tells the stories of some of the patients he encountered early in his career and their end-of-life experiences. He describes aggressive interventions performed on patients with advanced cases of cancer or dementia. In the book, they suffer one complication after another. There is never a happy ending.

related:

Medicare Says Doctors Should Get Paid To Discuss End-Of-Life Issues
August 18, 2015
http://www.npr.org/sections/health-shots/2015/08/18/427041879/medicare-says-doctors-should-get-paid-to-discuss-end-of-life-issues

http://www.npr.org/sections/health-shots/2016/06/11/481422813/this-doctor-wants-to-help-california-figure-out-aid-in-dying

Scientists Urge Temporary Moratorium On Human Genome Edits

Scientists Urge Temporary Moratorium On Human Genome Edits
March 20, 2015
http://www.npr.org/blogs/health/2015/03/20/394311141/scientists-urge-temporary-moratorium-on-human-genome-edits

A new technology called CRISPR could allow scientists to alter the human genetic code for generations. That’s causing some leading biologists and bioethicists to sound an alarm. They’re calling for a worldwide moratorium on any attempts to alter the code, at least until there’s been time for far more research and discussion.

The big worry is that CRISPR and other techniques will be used to perform germline genetic modification.

Another concern is that this could open the door to what people call designer babies.

see also:
https://franzcalvo.wordpress.com/2015/03/19/scientists-sound-alarm-over-dna-editing-of-human-embryos

http://www.npr.org/sections/health-shots/2016/06/02/480466940/scientists-say-they-hope-to-create-a-human-genome-in-the-lab

CRISPR gene editing produces unwanted DNA deletions
DNA-cutting enzyme used for genetic modification can create large deletions and shuffle genes.
16 July 2018
https://www.nature.com/articles/d41586-018-05736-3

Scientists sound alarm over DNA editing of human embryos

Scientists sound alarm over DNA editing of human embryos
Exper ts call for halt in research to work out safety and ethics issues.
12 March 2015
http://www.nature.com/news/scientists-sound-alarm-over-dna-editing-of-human-embryos-1.17110

Amid rumours that precision gene-editing techniques have been used to modify the DNA of human embryos, researchers have called for a moratorium on the use of the technology in reproductive cells.

NPR version:
https://franzcalvo.wordpress.com/2015/03/29/scientists-urge-temporary-moratorium-on-human-genome-edits

[BOOK] What Doctors Feel

What Doctors Feel: How Emotions Affect the Practice of Medicine
February 22, 2014
http://www.podcast.de/episode/237988910/Encore%3A+What+Doctors+Feel%3A+How+Emotions+Affect+the+Practice+of+Medicine

Danielle Ofri argues in her newest book “What Doctors Feel: How Emotions Affect the Practice of Medicine,” that the idea that doctors don’t have feelings, or that they can ignore those feelings, negatively affects patient care.

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http://www.npr.org/sections/health-shots/2016/02/27/468273093/keeping-up-with-the-joneses-latest-medical-procedure

“writer and doctor” site:npr.org

 

Who Gets First Dibs On Transplanted Liver?

Who Gets First Dibs On Transplanted Liver? Rules May Change
August 14, 2014
http://www.npr.org/blogs/health/2014/08/14/339579923/who-gets-first-dibs-on-transplanted-liver-rules-may-change

“Either it’s a jackpot and you have very, very easy access to a lifesaving liver — if you lived in Indiana or if you lived in Louisiana or Florida,” says Dr. David Mulligan, a transplant surgeon at Yale University. “But if you live in California or New York or New England the chances are significantly worse.”

That’s because the country is divided into 11 regions and some regions have more livers for transplant than others.

“So in the Southeast, for example, or in parts of the Midwest, where there’s a higher death rate,” Mulligan says, “there are more donor organs.”

UNOS is holding a public hearing in Chicago on Sept. 16 to discuss how best to distribute the limited supply of livers for transplantation.