THIS WEEK'S POWER PERSPECTIVE

 

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The Challenge and the Opportunity to Put CAR T Before the Price

 

"I think this is the most exciting thing I've seen in my lifetime,” oncologist Tim Cripe, with Nationwide Children’s Hospital.

"We're entering a new frontier in medical innovation,” FDA commissioner, Scott Gottlieb.

They’re talking about the revolutionary new approach to treating cancer called CAR-T cells. Instead of chemotherapy, this removes a patient’s own cells, sends them to a laboratory and reprograms them to fight the cancer when injected back into the patient. It may have application for several blood cancers like B-cell lymphomas and CLL.

This is the ultimate in personalized or precision medicine. 

Initially it will be used for children and young adults with ALL, acute lymphoblastic leukemia, who have relapsed after standard treatment.

These patients have few other options. HAD few other options. Now after a single dose of these re-engineered cells, 83 percent of participants in a clinical trial were cancer-free after three months. It has potential side effects and not everyone responds. But for those who do it’s the long-awaited CURE for this aggressive blood cancer.

Sam Fazeli, a Bloomberg Intelligence analyst says we’re not used to drugs curing people with cancer. He calls it a “a paradigm shift.”

STICKER SHOCK

But no doubt you’ve also seen the other shoe drop: a course of treatment marketed as Kymriah from Novartis, costs $475,000.

That’s generated nearly as much angst over the price as excitement over its potential. So what are patients to do?

We are not apologists for the pharmaceutical industry. Our goal in this analysis is to help determine what is best for patients. So here’s our PERSPECTIVE.

First, the cost could have been much higher. Wall Street analysts who monitor the business end of pharmaceuticals predicted a price tag of $750,000 for a dose. And STAT news also reports even UK regulators who keep some of the tightest reigns on drug prices of regulators anywhere in the world, thought $700,000 would be fair. 

Second, it is approved for a rare cancer with just 3,100 new cases a year. But nearly three-quarters of them can benefit from standard treatment, leaving just a few hundred patients each year eligible for CAR-T.  Remember, it costs as much to develop a drug for a few hundred patients as a few hundred thousand patients. With fewer patients to share the costs, the price per patient goes up. And it’s a one- time treatment; not an IV or a pill patients take every month for years.

But most important from our PERSPECTIVE, this is the first of many similar drugs for other cancers. We don’t want to be so focused on price that we discourage this breakthrough line of research and development. 

BUT CAN WE AFFORD IT?

At least Novartis has recognized the concern and has developed a unique program that means patients in government programs will pay only for results. There is no charge unless patients show signs that the treatment is working within a month of treatment.

Still, there are nay-sayers who say this part of pharmaceutical price creep. (One anti-pharma activist claims the company used $200 million in taxpayer dollars, but trade publications say that is based on “keyword internet search,” and the issue is much more complicated than that.)

So let’s leave you with this PERSPECTIVE. If we asked you which healthcare sector costs the most money, given the current outcry, I think most of us would say pharmaceuticals.

But that’s actually incorrect.

Cost of Healthcare News reports, “according to new data from the Altarum Institute”

  •        annual HOSPITAL expenditures totaled $1.133 trillion through June.
  •        PHYSICIAN and CLINIC spending was second with $708 billion
  •        and PRESCRIPTION DRUGS came in third with $359 billion.

Put another way pharmaceuticals are less than 2 percent of our entire economy, and the least expensive part of the healthcare system.

As for CAR-T treatment, Stephan Grupp, at Children’s Hospital of Philadelphia said, “We’ve never seen anything like this before.” At Patient Power, we want to make sure we see innovation like this again … and again where companies can invest and have a fair return and where the patients who need lifesaving treatments can get them. As you can see, it’s complicated and a rational discussion on how we spur affordable new science is required. We’ll keep bringing you news about this and always happy to hear your perspective too.

~Andrew Schorr

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Page last updated on September 14, 2017