As It Happens

'Breakthrough' child cancer drug costs $475K, and this advocate says that's way too much

The president of Patients for Affordable Drugs says the price tag on a one-time treatment for childhood leukemia is "excessive".
David Mitchell has the treatable but incurable blood disease multiple myeloma, and says someday this drug may be able to treat him. (Patients for Affordable Drugs)

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It's being hailed as a potentially revolutionary treatment for the most common type of childhood cancer, but the drug recently approved by the FDA comes with a price tag that has the president of the U.S. advocacy group shaking his head. 

The drug, Kymriah, is the first cancer drug that genetically engineers patients' own blood cells into an army of assassins to seek and destroy childhood leukaemia, and it's customized to each patient. 

Patients for Affordable Drugs' David Mitchell, a cancer patient himself, says the $475,000 US cost of this one-time treatment developed by Novartis Pharmaceuticals is "excessive" and reflects the "systemic problem" in the U.S. health-care system. 

​Mitchell spoke with As It Happens guest host Susan Bonner about the new drug, and how its high cost affects cancer patients. Here is part of their conversation. 

Can you tell us a bit about how this treatment works and why it could be a breakthrough for children with cancer?

The treatment is incredibly exciting.

They take your own T cells out of your body, send them to a plant in New Jersey, re-engineer them with a genetic change and give you back those cells with an element that causes those cells to go hunt and go kill cancer cells. It's not just revolutionary for children and their families; it can be revolutionary for all cancers.

The Food and Drug Administration approved the first treatment that genetically engineers patients own blood cells into an army of leukemia-fighting assassins. ( Brent Stirton/Novartis Pharmaceuticals Corp/Associated Press)

It will be used for all blood cancers subsequently and it's very exciting for me because I have an incurable blood cancer and I'm hopeful that within the next couple of years these CAR-T treatments are approved for my blood cancer, which is called multiple myeloma.

What do we know about the effectiveness coming out of those tests?

The results have been very very encouraging. 

Now how long that response lasts remains to be seen. We don't know how long these re-engineered CAR-T cells work and stay in your body. We need longer-term data for that.

And there are toxicities. It's dangerous. It can be dangerous. They recommend that someone who gets this re-engineered cells put into their body hang around a hospital for four weeks.

You talk about how it comes with dangers. It also comes with a pretty high sticker cost. I heard the excitement in your voice when you talked about someday it might help you with your cancer. But how did you react when you heard about the cost which is approaching half a million dollars per a patient?  

Well and it's higher than that actually. The $475,00 is for the drug.

I'm hopeful that within the next couple of years these CAR-T treatments are approved for my blood cancer, which is called multiple myeloma.- David Mitchell 

Many people will land in the hospital. Some will land in the ICU.

So when you think about the travel and the hotels and the hospital days, and the services to extract the T cells and then give you them back, and deal with possible side effects, all add up we estimate between $100-150,000 on top of the price of $475,000.

So it's a very expensive treatment.

Is this properly priced, in your view?  

We think the price is excessive. And we thinks so because of a couple of reasons.

Two years ago, the chief financial officer of a competing company with a CAR-T drug, the company called KITE, estimated that the base price was going to be $150,000 per treatment, not $475,000. And at that time, analysts were estimating up to $300,000, at most, per treatment.

Novartis says that it's spent years developing the drug, preparing it for market, getting it to market. It has to be customized for each patient. It can take weeks for a single treatment. That's an expensive prospect, no?

 think you have to bear in mind, however, that in the United States of America, taxpayers do the research, pay for the research for 50 to 60 per cent of all the new drugs that come to market.

In this case, taxpayers have invested through our National Institutes of Health more than $200 million to develop the basic science that underpins this CAR-T drug.  

Mitchell says that the $475,000 price tag of Kymriah is 'excessive.' (Novartis via AP)

Canada's researchers in this field, are saying: We're working on this. We see the price tag. We think we can do something that would be affordable in the Canadian system. Does that lead you to believe that it is something that can be made affordable?

I sure would like to get pricing in this country like you're able to get in Canada (chuckles).

So what's to be done in the United States then?

I believe we have to change our whole system. We're reflecting a systemic problem of the way we allow drugs to be priced in this country.

With files from The Associated Press. This interview has been edited for length and clarity. For more, listen to our conversation with David Mitchell: 

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