Gene Therapy for Rare Cancer, Miracle Cures at Enormous Prices. - Gina Kolata - NY Times, Medicine and Science Correspondent - Wednesday 10/4

Gene Therapy for Rare Cancer, Miracle Cures at Enormous Prices.
Gina Kolata
NY Times, Medicine and Science Correspondent
Wednesday 10/4

Here’s a theme I stole from a quote Medical researcher and administrator Aaron Kesselheim gave our guest today, NY Times Medicine and Science reporter Gina Kolata.

Let’s say your house catches on fire and you call the fire department, and within minutes they are there.  While his men are attaching their hoses to the nearest hydrant, the fire lieutenant says to you…nice house ya got there.  Mind telling me what it’s worth? Because this week we’re having a half-price special and for half the honest market value we’ll save your home.

But you splutter, my kids are still inside.  Oh, he says, I’m glad you told me that.  The price just tripled.  You gonna pay or do my people put the hoses back in the truck?

After the house and kids are saved, you go down to the fire house to get some things off your chest, but before you can say a word, the captain of the firehouse interrupts:  look, sir, how do you think we pay for our trucks and hoses and gas.  We need your money.  And by the way, think of your kids, think of the value of the service we rendered.

Very recently, the FDA approved a revolutionary new treatment for a rare form of leukemia that kills young people.  The drug, brand-named Kymriah, has little track record, but the promise it is, it kills the cancer, cures the young patient, and does so in a single dose.  Novartis, the maker of Kymriah, is pricing it at $475,000.

Kymriah was tested on 63 leukemia patient and within 3 months had put more than 50 of them into remission.  And that $475,000 price tag comes with a money back guarantee.  Novartis says there will be no charge if the patient does not respond to the treatment within a month.

Still, it’s a lot of money, and another new drug to prevent blindness, caused by another very rare disorder, is mulling it’s one-dose-cure price at between $700,000 and $900,000.

Who can pay these prices?  Sure, the very, very rich can, but more likely the economic decisions will have be made by insurance companies, including the biggest healthcare provider of  ‘em all, the US Government –and its Medicare and Medicaid programs.

So what would be a fair price, taking into account the literal life and death value of the medication, and the expensive research and development that went into it?  This is no abstract issue, your wallet and your tax bill are directly involved, because gene therapy, the breakthrough which Kymriah represents, is about to explode onto healthcare practices and budgets…dozens of new gene therapies are in the final stages of testing…somewhere close to 500 have begun the testing process.  Most of the drug-Makers involved hope to charge these mega-high prices.



Gina Kolata is a reporter at The New York Times, focusing on science and medicine. Her training is in science: She studied molecular biology on the graduate level at M.I.T. for a year and a half and has a master’s degree in applied mathematics from the University of Maryland.

Her work at The Times has led her to be a Pulitzer finalist twice — for investigative reporting in 2000 and for explanatory journalism in 2010. Other writing awards include ones in 2010 from the Silurian Society for a series on the war on cancer and from The Associated Press Sports Editors for writing about the Caster Semenya intersex controversy at the world track championships.

She is the author of several books, including Rethinking Thin: The New Science of Weight Loss and the Myths and Realities of Dieting (Farrar, Straus and Giroux, 2007), which was a finalist for the Quill book awards in 2007.


New Gene-Therapy Treatments Will Carry Whopping Price Tags – The New York Times

F.D.A. Approves First Gene-Altering Leukemia Treatment, Costing $475,000 – The New York Times

With a Simple DNA Test, Family Histories Are Rewritten – The New York Times 

What Does It Cost to Create a Cancer Drug? Less Than You’d Think – The New York Times





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