ICER Weekly View: August 16, 2019

From the desk of David Whitrap

Good morning everyone. This week, The New York Times Editorial Board provided some important recommendations for how the US should evaluate rare-disease treatments when the basic clinical evidence is still emerging – to better balance the safety, innovation, and affordability we all want from our health care. In some instances, the US health system is getting incredible pharmaceutical innovations at cost-effective prices. But in other instances, Americans are getting completely ripped off by treatments with unproven benefits and sky-high prices. And historically, the country hasn’t had a good mechanism for distinguishing one situation from the other. ICER is working to fill that gap.

Okay, let’s take a look at the rest of this week’s news…

  • ICER in the News: ICER’s upcoming patient webinar to discuss potential changes to our 2020 methodology, and our final evidence report on treatments for Duchenne muscular dystrophy.
  • Pharmaceutical News: Roche’s latest cancer treatment is approved, Novartis reveals that the data manipulators were actually “exited” back in May, employers are struggling to keep up with rising healthcare costs, the Trump administration’s drug importation plan will not move forward without pharma’s buy-in, Members of Congress want to restart an investigation into price hikes for generic drugs, and Scotland balks at the prices of treatments for cystic fibrosis.

ICER in the News

ICER is inviting all patient organizations and the general public to attend a webinar on September 4th where we will review the suggestions ICER received during the recent public comment period on our 2020 Value Assessment Framework, outline potential changes to these methods that ICER is now considering, and discuss how patients can continue to be part of improving our process. The webinar will be a Q&A session with ICER’s President Steve Pearson, and it will be moderated by Tanisha Carino, the Executive Director at FasterCures. As a reminder, we’ll be posting our proposed refinements to our 2020 framework on August 21st, and we’ll be accepting public comment on those proposals through October 18th.

ICER’s Value Framework and Patient Feedback:
Q&A with Steve Pearson

(Webinar Registration)

And yesterday afternoon, we released our Final Evidence Report assessing the clinical and economic value of three treatments for Duchenne muscular dystrophy: eteplirsen, golodirsen, and deflazacort. Our public meeting on this topic revealed all the tensions inherent around high-priced treatments that lack adequate evidence of efficacy, and yesterday’s report elevates several important policy recommendations that were made throughout that meeting, which was also covered by STAT News and Pink Sheet.

ICER Issues Final Report and Policy Recommendations
Regarding Treatments for Duchenne Muscular Dystrophy


As Sarepta awaits approval of new Duchenne drug,
big questions hang over therapy already on market

(STAT News)

ICER Reviews Duchenne: Exondys 51 Lacks
Net Health Benefit; Emflaza Fairs Better

(Pink Sheet)

Pharmaceutical News

Yesterday, the FDA approved Roche’s cancer drug Rozlytrek for patients with any kind of tumor that tests positive for one kind of genetic alteration, known as NTRK, and for non-small cell lung cancer that tests positive for another genetic alteration, ROS1. It’s the third time that the FDA has approved a cancer drug not based on where the tumor showed up in the body but on its genetic makeup. How much will it cost? $17,050 per month.

Roche drug approval offers a glimpse
at the future of cancer treatment

(STAT News)

The Novartis controversy around data manipulation continues with news that two former AveXis researchers were actually let go back in May, around the time FDA approved Zolgensma. Novartis didn’t notify the FDA about the issue until June.

Novartis’ AveXis shifted top scientific leadership
before Zolgensma data scandal erupted


Data from a new survey from the National Business Group on Health suggests that it may be large employers, feeling the budgeting challenges of rising health care costs, that drive the next wave of innovation around affordable care.

Large Employers Double Down on Efforts to Stem
Rising U.S. Health Benefit Costs which are
Expected to Top $15,000 per Employee in 2020

(National Business Group on Health)

The Trump administration’s plan to export cheaper medications from Canada seems impractical without support from manufacturers. Pharmaceutical companies typically use wholesalers and distributors to expand their reach to non-US markets, and their contractual agreements usually ensure that products will not be exported back to the US.

Trump’s Canada Drug Import Plan
Can’t Happen Without Big Pharma


Canada is introducing reforms to cut the amount spent on prescription drugs. As a part of those efforts, Canada’s Patented Medicine Prices Review Board (PMPRB) will consider the cost-effectiveness of therapies for rare diseases.

Canada’s Patented Medicine Prices Review Board
may be “more forgiving” in setting price
caps for drugs that treat rare diseases


Rep. Elijah Cummings and Senator Bernie Sanders are continuing a previously stalled probe into price hikes for several generics developed by Mylan, Teva, and Heritage Pharmaceuticals.

Sanders and Cummings renew probe into
generic pricing they say was ‘stonewalled’

(STAT News)

The Scottish National Health Service (NHS) has rejected Vertex’s cystic fibrosis drugs, Orkambi and Symkevi, due to the treatments’ inability to demonstrate cost-effectiveness at their current prices. Vertex is also negotiating with the UK’s National Institute for Health and Care Excellence (NICE), which has also declined access based on the current price.

As negotiations with England labor on,
Scotland rejects routine use of Vertex’s cystic fibrosis drugs

(Endpoints News)


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