ICER Weekly View: July 12, 2019

From the desk of David Whitrap

Good morning everyone. This week, influential minds from both ends of the political spectrum delivered new recommendations for how the US could lower drug costs. And while there is predictable disagreement between their respective approaches, both sides feature ICER as part of the solution…

  • The left-leaning New York Times Editorial Board published a column in Sunday’s paper, “Sound, Fury and Prescription Drugs,” recommending three strategies for the US government to lower drug prices. Recommendation #1 is to align a drug’s price with its benefits for patients, similar to the approach ICER takes: The nonprofit Institute for Clinical and Economic Review already [evaluates drugs’ cost-effectiveness] and has had success in getting some drug companies to lower some of their prices.”
  • On the other end of the political spectrum, experts from the conservative American Enterprise Institute this week authored an editorial for Health Affairs, “A Market-Oriented Framework For Reforming Medicare Part B Drug Payment,” recommending that Congress eschew the notion of an International Price Index and instead pass legislation that would rely on a competitive private sector to deliver lower drug prices in the US. Specifically, AEI foresees an HHS-established advisory panel that would: “Analyze available clinical information on the therapies lacking effective competition… determine the range of clinical value estimates [from sources to include PCORI and ICER, as well as HTA agencies in other countries]… and publish a report on its findings that vendors could use to negotiate pricing under Part B.”

Other than that, lots of activity this week to dive into:

  • ICER in the News: Our Evidence Report on treatments for Duchenne muscular dystrophy; policy recommendations related to the investigational therapies for peanut allergy; and a look ahead to potential ICER assessments in 2020.
  • Pharmaceutical News: Washington DC’s chaotic week that saw the end of the “rebate rule,” the end of drugmakers disclosing list prices in TV ads, a promise of some sort of “most favored nation” approach to future US drug price negotiation, and the latest small hint of potential bipartisan compromise; the repercussions of the FDA’s “fast-track” approval approach becoming more routine; Boston biotech’s frustration with its home state seeking accountable drug pricing; the latest round of mid-year drug price increases; and so much more.

ICER in the News

Yesterday, ICER published our evidence report assessing the comparative clinical effectiveness and value of two exon-skipping therapies to treat Duchenne muscular dystrophy (DMD) — eteplirsen (Exondys 51™, Sarepta Therapeutics) and golodirsen (Sarepta Therapeutics) — as well as deflazacort (Emflaza®, PTC Therapeutics), a corticosteroid. Previewing the conversation that will happen at our July 25 public meeting, ICER’s chief medical officer Dr. David Rind stated:

“DMD is a progressive neuromuscular disorder that devastates the lives of children and the families who care for them, and better therapies are desperately needed… This apparent mismatch between evidence and price raises important concerns for patients, caregivers, and all other stakeholders in the health care system.”

ICER Publishes Evidence Report on Treatments for Duchenne Muscular Dystrophy

Earlier this week, and based on our June public meeting, we issued our Final Evidence Report assessing the clinical and economic value of two investigational technologies that treat children with peanut allergy by building their immune tolerance to peanuts: Viaskin® Peanut (DBV Technologies) and AR101 (Aimmune Therapeutics).

ICER’s Final Report on Peanut Allergy Treatments: Clinical Evidence Does Not Yet Demonstrate That Long-Term Benefits of Desensitization Outweigh Short-Term Risks

Finally this week, we provided a sneak peek into potential topics we are considering for value assessments next year. From gene therapies to PrEP to digital apps, there’s a little something for everyone.

ICER Lists Potential Topics for Assessments in 2020

Pharmaceutical News

Last Friday, President Trump said his administration would soon issue an executive order mandating a “favored nations” policy in which U.S. payments for drugs are capped at the lowest price paid by either a manufacturer or a developed country. It was not immediately clear to what extent Trump’s announcement differs from one of the White House’s signature drug pricing proposals: an international price index that would similarly cap U.S. drug payments based on an average of prices paid in an index of developed nations.

Trump plans drug pricing executive order
aimed at ensuring U.S. pays less than other nations

(STAT News)

And on Monday, a federal judge blocked a major White House initiative that would have forced drugmakers to disclose their list prices in TV ads, saying the Trump administration lacks the legal authority to force companies to do so without Congressional legislation.

Judge strikes down rule requiring drug ads to reveal prices
(The Associated Press)

And then in a closed-door Capitol Hill briefing on Tuesday, HHS Secretary Alex Azar joined President Trump’s top policy adviser to encourage GOP Senators to pursue bipartisan legislation that would cap some drug price hikes at the rate of inflation. After the meeting, not all of the Republicans seemed convinced.

Top Trump advisers hint at support for
progressive proposal to cap drug price hikes

(STAT News)

And then just yesterday, the Trump administration announced that it would drop its proposal to ban the rebates drugmakers pay the insurers and PBMs that manage Medicare and Medicaid plans. The “rebate rule” had been a centerpiece of President Trump’s blueprint to lower drug costs.

Trump Administration Drops Plan to Curb Drug Rebates
(The Wall Street Journal)

The FDA is approving a rising proportion of expedited cancer drugs without ample evidence they extend patients’ lives. And in response, some health care officials are now proposing that a third party set prices for expedited drugs and that manufacturers get paid only after the treatments have demonstrated they actually work.

Fast-Track Drug Approval, Designed for Emergencies,
Is Now Routine

(The Wall Street Journal)

The Boston Globe discusses how a proposal by Massachusetts Governor Charlie Baker to lower Medicaid drug prices now angers the biotech leaders who have benefited this past decade from more than $677 million in public grants, loans, tax incentives, and other local support. Former Governor Deval Patrick argues that “state support for biotech should not exempt the industry from accountability.” (Related, one expert busts the industry’s myth that lowering drug prices will hurt the local economy.)

Charlie Baker’s plan to curb drug prices
strains his relationship with biotechs

(The Boston Globe)

Under the cover of the 4th of July holiday, drugmakers initiated a new round of price increases on 104 drugs – including different doses and package sizes of the same medicines – with some of them affecting generic hospital-administered injectable drugs that are in short supply. (Shameless plug: later this year, ICER will publish our list of the most significant price increases that are not supported by new clinical evidence.)

Drugmakers Push Their Prices Higher
(The Wall Street Journal)

One week after former FDA Commissioner Scott Gottlieb was appointed to Pfizer’s board of directors, U.S. Senator Elizabeth Warren urged him to step down. Gottlieb stated he will respond to Warren privately.

Senator Warren asks former FDA chief Gottlieb
to resign from Pfizer board


Amid ongoing concerns that clinical trial data is too often kept under wraps, an analysis found that most large drug makers continue to do a poor job of sharing study data after new medicines were approved, adding further pressure on the pharmaceutical industry to improve disclosure practices.

Pharma continues to get poor grades when it comes
to sharing clinical trial data

(STAT News)

A growing number of insurers and health systems are offering tools that give doctors drug pricing information in real time, but the uptake has been slow.

Doctors Slow To Adopt Tech Tools
That Might Save Patients Money On Drugs

(NPR / Kaiser Health News)


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