ICER Weekly View: May 31, 2019

From the desk of David Whitrap

I was heartened this week to read Alex Trebek’s announcement that he’s in “near remission” of advanced pancreatic cancer. As the ASCO Annual Meeting kicks off today in Chicago, let’s all hope for many more of these “mind-boggling” success stories in the future.

This morning, we’ll look at:

  • ICER in the News: Our Evidence Report on treatments for peanut allergy; and our updated assessment on the SMA gene therapy Zolgensma.
  • Pharmaceutical News: Why Zolgensma should not represent a reference price for future gene therapies; the current landscape for cancer treatments; approved drugs that have side effects creep up several years later; and the debate over disclosing drug prices in TV ads.

ICER in the News

This week, ICER released an Evidence Report assessing the comparative clinical effectiveness and value of new technologies that build immune tolerance to peanuts. To achieve commonly cited thresholds for cost-effectiveness, the annual price would need to be between $4,800-$7,200 for Aimmune Therapeutics’ AR101 and $3,000-$4,500 for DBV Technologies’ Viaskin Peanut.

ICER’s Assessment Highlights the Risk-Benefit Tradeoffs of Investigational Treatments for Peanut Allergy

In light of additional data from ongoing trials and the final FDA label for Novartis’ Zolgensma, last Friday ICER published an addendum to our Final Evidence Report on treatments for spinal muscular atrophy. Given the new efficacy data for the presymptomatic SMA population, the $2.1 million price for the one-time gene therapy falls within the upper bound of our updated value-based benchmark range.

This New Treatment Could Save the Lives of Babies. But It Costs $2.1 Million.
(The New York Times)

At $2M, priciest ever medicine treats fatal genetic disease
(The Associated Press)

At $2 Million, New Novartis Drug Is Priciest Ever
(The Wall Street Journal)

Novartis $2 million gene therapy for rare disorder is world’s most expensive drug (Reuters)

Pharmaceutical News

Bloomberg’s columnist Max Nisen understands that Zolgensma’s $2.1 million price tag can be “sky-high” AND “cost-effective.” But he worries about the dozens of other incoming gene therapies that will use this price as a reference point. I agree: Zolgensma provides an unusually large benefit for children who have an unusually devastating disease, not all gene therapies will be this impressive, and therefore not all gene therapies deserve a price even close to Zolgensma’s.

A $2.1 Million Drug Price Record Is Made to Be Broken

Cancer drugs that speed onto the market based on encouraging preliminary studies often don’t show clear benefits when more careful follow-up trials are done, according to research published Tuesday.

Cancer Drugs Approved Quickly Often Fail To Measure Up Later

Meanwhile, a new analysis from IQVIA shows that the amount spent on cancer medicines reached nearly $150 billion in 2018, up about early 13%. That’s the fifth consecutive year of double-digit growth.

More new cancer therapies are being launched than ever before,
but spending is skyrocketing

(STAT News)

Roughly 1% of patients receiving immunotherapy drugs experience the same irreversible side effect: diabetes. Making matters worse, oncologists have little clue why.

A lifesaver with a catch: Powerful new cancer drugs
can trigger diabetes — and no one is certain why

(STAT News)

Over the past 15 years, more than 500,000 serious adverse events were reported in people using biologic drugs, with Humira and Enbrel leading the pack.

Biologic medications for arthritis and psoriasis have
flooded the market — and been linked to 34,000 deaths

(Milwaukee Journal Sentinel)

Will forcing drug companies to disclose list prices actually lead them to lower prices? That’s a matter of fierce debate. (But wouldn’t it be more illuminating if drug companies had to disclose whether or not their net prices have been deemed cost-effective by an independent third party?)

Will drug prices in TV ads really be transparent?

The Wall Street Journal created an animated video to help explain how the flow of money, drugs, and rebates may drive up the price of prescription medicine for US consumers.

How Drug Prices Work
(The Wall Street Journal)

And finally, for any of you who feel you’ve already seen all the dysfunction in how US drug prices are set, I leave you with this…

Tylenol For Infants And Children Is The Same.
Why Does 1 Cost 3 Times More?



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