ICER Weekly View 05-25-18

From the desk of Mitchell Stein


Good morning. Imagine a stone tossed in a pond whose ripples continue to spread weeks later, now imagine that stone is the President’s drug price speech. We’ll talk about the continued ripples emanating from that toss leading off today’s Inside the Beltway section. We’ll also talk about ICER news, right-to-try and much more. Then, and only then, you can start thinking about good weather and what you’ll be cooking on the grill over the long weekend. Here’s what’s standing between you and those happy thoughts:

ICER in the news – Pearson on evaluating cures, report on Midwest CEPAC CF meeting, Ollendorf participates in value assessment panel, Pearson on value assessment, and more on migraines.

Inside the Beltway – Reverberations of the President’s drug price speech (many articles), right-to-try heads to the President’s desk, Gottlieb on accelerated reviews for gene therapy, Frakt on the risks and benefits of expedited reviews, industry reacts to FDA guidance on Alzheimer’s, a profile of Azar, and a look at if the FDA can stop product hopping.

Industry Trends – Cancer drug spending climbs, it’s the monopolies, stupid, repurposing inexpensive medication and making them expensive, the copay coupon wars continue, Pfizer pays a fine, pharma well represented in the Fortune 500, it’s complicated part 394 – a look at coverage for ALS drug Radicava, and more good news/bad news for Keytruda.

Now, on to the news.

ICER In The News

ICER’s President, Dr. Steve Pearson, talks with AJMC about evaluating new treatments that are cures.

Dr. Steven Pearson Highlights the Challenge of Evaluating New Interventions That Cure Diseases

Biocentury reported on last week’s Midwest CEPAC meeting reviewing treatments for Cystic Fibrosis.

ICER panel: Low value for Vertex CF drugs

The Midwest Comparative Effectiveness Public Advisory Council (CEPAC) voted that the long-term value was low for three cystic fibrosis drugs from Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) as some patients and payers voiced their frustration at the biotech’s pricing of the therapies.

At an ISPOR panel, ICER’s Dan Ollendorf participated in a panel on personalized medicine (AJMC).

Value Assessments in the Age of Personalized Medicine May Require a Cultural Shift, Panel Says

A report from AJMC on a value assessment panel included this quote from ICER’s Dr. Pearson:

“Speaking from the audience during the panel’s question-and-answer component was Steven Pearson, MD, MSc, founder and president of ICER. Pearson contended that there would be a higher number of citations for his organization’s reports in the SPEC database had more recent data been included in Chambers’ analysis, and added that “we would never envision” that ICER is the only factor that payers should consider in making decisions. Finally, he cited pricing of Amgen and Novartis’ new migraine therapy, erenumab-aooe (Aimovig), as a reflection of the utility of ICER’s work in helping to rein in drug costs.”

Value Assessments Are Just One Tool in the Decision-Maker’s Toolkit, Panel Says

Following last week’s approval of Aimovig, coverage continued, including this piece from Biopharma Dive discussing ICER’s assessment.

Amgen, Novartis’ closely watched migraine drug approved

Amgen and Novartis on Thursday secured Food and Drug Administration approval for Aimovig, the first regulatory thumbs up for a new class of migraine treatments called calcetonin gene-related peptide (CGRP) inhibitors. Aimovig heads to market with a broad label, cleared for the preventive treatment of migraine in adults.

Pharmaceutical News

Inside the Beltway/State Regulatory Developments

Two weeks after the President’s speech on drug prices, we’re still talking about its ramifications. Last week saw HHS Secretary Azar go into more detail, but as The Hill reports, Trump official on defensive as critics scoff at drug plan. That’s the Administration, in Congress, to date Republicans have not been as vocal; Axios reports that: Republicans are quiet about Trump’s drug plan. However, in June The Hill reports the Senate health committee will hold a hearing on Trump drug pricing plan during which Secretary Azar will testify.

People are taking a closer look at some elements of the plan. Here the NY Times looks at including prices in DTC ads: Requiring Prices in Drug Ads: Would It Do Any Good? Is It Even Legal? That’s one idea that some Democrats are getting behind as The Hill reports on some initial efforts to make that happen: Dem senators ask drug companies to list prices in ads.

Among the most complicated of the plan’s ideas is switching coverage for some drugs from Medicare Part B to Part D. Avalere looked at the implications of such a switch, noting that some could face higher drug costs if the proposed changes are made: Highlights Complexities of Transitioning Medicare Part B Drugs into Part D.

Several of the proposals in the plan focus on generic drugs and the stalling tactics used by brand-name manufacturers to keep them off the market. As our final piece on the plan, here is Kaiser’s analysis looking at the cost of delaying generic availability.

Drugmakers Blamed For Blocking Generics Have Jacked Up Prices And Cost U.S. Billions

This story also ran on This story can be republished for free ( USA Today. details). Makers of brand-name drugs called out by the Trump administration for potentially stalling generic competition have hiked their prices by double-digit percentages since 2012 and cost Medicare and Medicaid nearly $12 billion in 2016, a Kaiser Health News analysis has found.

The House passed the Senate version of the right-to-try bill. Donna Young of S&P Global has a detailed review of how the House version of the bill that critics said was worse than the Senate version ended up being the one that will become law. (Remember, some believe this is meant to undermine the FDA and that it won’t make it any easier for patients to access legitimate experimental drugs.)

Trump gets his right-to-try win; FDA chief ‘comfortable’ after initial criticism

Gottlieb lays out the accelerated review pathway for gene therapy through the use of surrogate endpoints.

FDA chief Gottlieb is building a regulatory speedway to accelerate gene therapy development

In a rallying cry for gene therapy, FDA Commissioner Scott Gottlieb says he’s determined to clear the pathway for drug developers in a move to accelerate the first wave of gene therapies pointed to the market. The first therapeutic area to benefit from new surrogate endpoints will be hemophilia, Gottlieb

As we talk about approval speedways, this JAMA forum piece from Austin Frakt looks at the pluses and minuses inherent in the FDA’s existing expedited review process.

JAMA Forum: The Risks and Benefits of Expedited Drug Reviews

The US Food and Drug Administration (FDA) oversees several programs that expedite approval of certain drugs that treat serious conditions and address unmet medical needs. On average, a drug in an expedited program reaches market almost a year sooner than other drugs. However, expediting drug approvals raises concerns that important safety or effectiveness information will be missed,…

Industry likes the FDA’s draft guidance on Alzheimer’s treatments – despite continued failures (RAPS). (And speaking of Alzheimer’s failures, there was another one announced this week.)

Alzheimer’s Disease: Companies Weigh Revised FDA Draft Guidance

Posted 21 May 2018 | By Zachary Brennan Companies developing treatments for early Alzheimer’s Disease submitted another round of positive comments last week on US Food and Drug Administration (FDA) draft guidance, offering a hint of optimism in a space where no company has seen success.

A look at HHS Secretary Azar – In some ways, not what anyone expected (NY Times).

Trump’s New Health and Human Services Secretary Is a Joyful Regulator

In an administration with a deregulatory bent, Alex Azar relishes his policymaking powers. Alex Azar, President Trump’s new man running health policy, was waving a Pilot ballpoint in front of a room full of reporters. “This pen has a lot of power,” he said, smiling at his own joke.

Does the FDA already have the authority to prevent product hopping? The authors of this Health Affairs piece think it does.

A Preferable Path For Thwarting Pharmaceutical Product Hopping

Industry Trends

To the surprise of no one, the price of cancer drugs continues to climb.

Cancer drug spending climbs, with top winners earning lion’s share

Oncology is a lucrative field for pharma, but the payoff is heavily concentrated around just a few dozen medicines, finds a new report from the Iqvia Institute for Human Data Science. The top 35 cancer drugs by sales accounted for 80% of the roughly $110 billion spent globally on oncology therapeutics last year, according to Iqvia.


Dr. David Blumenthal, President of the Commonwealth Fund, thinks he knows why we have a drug price problem.

It’s the Monopolies, Stupid!

Endpoints News takes a look at another case of an old inexpensive medication being turned into a new expensive one.

Another cheap, old drug is being freshened up for brand pricing in the US – and shares soar, briefly

BioRegnum, the view from John Carroll When the execs at Fortress went about setting up their pain drug biotech Avenue Therapeutics $ATXI, they went to some extremes to avoid R&D risk. Instead of trying something new, they tweaked a European IV formulation for tramadol – never approved in the US,

Copay coupon wars escalated they have, as new weapons are being deployed from both sides (FiercePharma).

Amgen, AbbVie ‘ahead of the crowd’ in fighting payers’ latest assault on copay coupons

Copay coupons-pharma loves them, payers hate them. And that battle is getting hotter than ever, thanks to rapid growth of the couponing strategy. With more than 40% of specialty drug costs now supplemented by patient coupons, payers are cracking down anew and pharma’s finding creative ways to thwart them.

Speaking of copay coupons, Pfizer, without admitting any wrongdoing, agrees to pay a $24 million regarding illegal kickbacks. Given the amount, paying the fine is not expected to impact the firm materially (see story below).

Pfizer Settles Kickback Case Related To Copay Assistance For $24M

Pfizer will pay the government nearly $24 million as part of a settlement to resolve allegations that it funneled money through a foundation resulting in illegal kickbacks. The company is not admitting wrongdoing or liability as part of its agreement with the Department of Justice.

The new Fortune 500 list is out, and pharma continues to be well represented (Biopharma Dive).

J&J, Pfizer, Merck again lead pharma in Fortune 500

Johnson & Johnson, Pfizer and Merck & Co. continue to live up to their big pharma titles, having yet again secured top spots among drug companies on the latest Fortune 500 list. By ranking, J&J was 37, Pfizer 57 and Merck 78.

In this week’s chapter of it’s complicated, a long but informative read from STAT’s Ed Silverman on the ALS drug Radicava. Reimbursement issues are complicated as patients are anxious to start the drug despite questions on its efficacy.

ALS patients losing time as they wait for insurers to cover pricey new drug

or the past two years, Sarah Benoit has been getting around with the help of a walker, waiting for a medicine that’s out of reach. Benoit, a former congressional aide, has ALS, a fatal neurological disease that gradually causes muscle weakness and paralysis.

As seems to be the case most weeks, Keytruda was in the spotlight with both good and bad news. Two stories below; FiercePharma with the good news and BiopharmaDive with the bad.

Merck rains on Roche’s parade with double Keytruda win in squamous lung cancer

Merck already controls a big piece of the first-line non-small cell lung cancer market, and it likely just secured most of the rest. Its immunotherapy Keytruda nailed a study in previously untreated patients with the squamous form of the disease, who represent up to 30% of newly diagnosed NSCLC cases.

FDA warns of decreased survival in 2 studies of Keytruda, Tecentriq

The Food and Drug Administration on Friday warned oncologists and clinical trial investigators of early signs from two trials that some bladder cancer patients treated first with either Merck & Co.’s Keytruda or Roche’s Tecentriq alone were dying sooner than those given platinum-based chemotherapy. 
@mhstein  |

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