ICER Weekly View 05-04-18

From the desk of Mitchell Stein

 

Good morning. First, my apologies for the delay in publishing this morning. Second, before we dive into the week’s news let me wish you a Happy Star Wars Day. May the fourth be with you. (May the 4th, may the force… get it?)

With those important matters out of the way, let’s get to the other stuff. ICER was all over the news this week with the Regeneron/Sanofi announcement of their deal with Express Scripts on Praluent’s pricing. We also have several stories related to the President’s upcoming speech on drug pricing, and of course lots more. Here’s the full rundown for this week: 

ICER in the news – Regeneron/Sanofi/Express Scripts Praluent agreement involving ICER’s work, evidence report on CF, draft condition update report on plaque psoriasis, new council members named, Dr. Bach on NY Medicaid drug review, Dr. Bach in JAMA on value pricing, and coverage of ICER’s asthma work

Inside the Beltway – The President’s upcoming speech on drug pricing (many stories), Novo Nordisk’s reaction to insulin pricing pressure, and CDC Director takes a salary cut

Industry Trends – CRISPR pioneer’s next step, CAR-T development, are there too many cancer drugs, Bill Gates worried about pandemics, some shareholders think prices are too high, CRISPR court update

Company/Drug Specific – Merck, Novartis, Bristol-Myers, and Roche

Weekly View In-depth – Drug development meets Sci-fi. Big data and AI, are they game changers or the latest buzzwords?

Now, on to the news.


ICER In The News

Regeneron/Sanofi announced a new deal with Express Scripts regarding their PCSK9 inhibitor, Praluent. In exchange for a lower price aligned with ICER’s recent Preliminary New Evidence Update, Express Scripts will greatly reduce the approval process for the drug. ICER issued the following statement:

“Following a decades-long trend toward dysfunction and finger-pointing, the US health care system is beginning to address its drug pricing problem through the emergence of a ‘grand bargain.’ When a manufacturer is willing to responsibly price an innovative medicine in line with its clinical benefits, payers should reciprocate by removing the hurdles that can prevent patients from getting the drug. We commend the Regeneron and Sanofi team for seeking ICER’s independent assessment on what a fair, value-based price would be for Praluent, and we endorse Express Scripts’ decision to make it financially and administratively easier for eligible patients to access this life-saving therapy. As the nation strives to help all Americans achieve sustainable access to high-value care, this sort of grand bargain – a win-win-win for manufacturers, payers, and patients – is a model for the rest of the industry to contemplate.”

The announcement prompted a flurry of media coverage including the following stories that highlighted ICER’s role:

 

ICER released an Evidence Report assessing the comparative clinical effectiveness and value of cystic fibrosis transmembrane conductance regulator (CFTR) modulators, including combination therapies tezacaftor/ivacaftor (Symdeko, Vertex Pharmaceuticals) and lumacaftor/ivacaftor (Orkambi, Vertex Pharmaceuticals), as well as the monotherapy ivacaftor (Kalydeco, Vertex Pharmaceuticals), for the treatment of cystic fibrosis (CF). This Evidence Report will be the subject of an upcoming public meeting of the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC) in St. Louis, MO on May 17, 2018.

“Our report summarizes the evidence demonstrating that CFTR modulator therapies, paired with best supportive care, provide clinical benefit for patients who have not been treated previously with disease-modifying therapy,” noted Dan Ollendorf, PhD, ICER’s Chief Scientific Officer. “The current prices for these drugs, however, produce overall costs that are far in excess of those needed to reach commonly cited cost-effectiveness thresholds. Our analysis suggested that discounts of up to 77% would be needed to bring the prices into alignment with their clinical value to patients. Our public meeting will bring stakeholders together to discuss strategies to achieve sustainable patient access to these therapies while supporting the innovation that will bring further progress in treating diseases like CF.”

Institute for Clinical and Economic Review Finds that at Current Prices CFTR Modulators for Cystic Fibrosis Improve Patient Outcomes at Costs that Far Exceed Standard Cost-effectiveness Levels

 

ICER released a Draft Evidence Report assessing the comparative clinical effectiveness and value of treatments for moderate-to-severe plaque psoriasis. The review will be open to public comment until May 25, 2018. Findings contained in this report are preliminary and subject to change based on additional stakeholder input and further analysis of data. ICER previously reviewed treatments for plaque psoriasis in 2016. In addition to incorporating emerging clinical data for previously reviewed therapies, this condition update report includes new analyses of guselkumab (Tremfya, Johnson & Johnson), tildrakizumab (Ilumya, Sun Pharma/Merck), certolizumab pegol (Cimzia, UCB), and risankizumab (AbbVie).

Institute for Clinical and Economic Review Releases Draft Condition Update Report on Treatments for Plaque Psoriasis

 

ICER has appointed new members to each of its three independent evidence appraisal councils: the California Technology Assessment Forum, the Midwest Comparative Effectiveness Public Advisory Council, and the New England Comparative Effectiveness Public Advisory Council. “Our evidence appraisal councils play a vital role in ICER’s work, representing an open, transparent forum to receive input from patients, clinical experts, and other stakeholders, and then to deliberate and vote on the evidence regarding the comparative clinical effectiveness and value of health interventions,” noted Steven D. Pearson, MD, MSc, President of ICER. “The discussions during each of ICER’s public meetings reflect the dedication and rigor of each member of these councils, and we look forward to the perspectives that this year’s distinguished new members will add.”

The Institute for Clinical and Economic Review Appoints New Members to Each of Its Three Independent Evidence Appraisal Councils

 

Dr. Peter Bach discusses last week’s NY Medicaid Drug Utilization Review Board meeting, ICER’s role in the meeting, and the price of Orkambi.

Medicaid Is Right to Demand Lower Drug Prices

Last week, as President Donald Trump delayed announcing his plan to bring down high drug prices, about a dozen physicians, pharmacists, actuaries and economists in Albany, New York, tried to make a difference. The state Medicaid program’s Drug Utilization Review Board voted 10-0 to lower the price New York would pay for Orkambi, Vertex Pharmaceuticals’ cystic fibrosis drug, to $83,000 per year, from $250,000.

Dr. Bach had a busy week; he also published this piece in JAMA providing details on various “value” pricing concepts. His section on value-based pricing refers to ICER’s work. (The piece also includes a very helpful chart describing the five most talked about concepts.)

Defining and Redefining Value-Based Drug Pricing

In December 2017, the US Food and Drug Administration approved voretigene neparvovec-rzyl (Luxturna), a one-time genetic treatment manufactured by Spark Therapeutics that must be injected into the eye and can improve the vision of individuals with biallelic RPE65 mutation-associated retinal dystrophy, a rare genetic condition that affects approximately 1000 to 2000 people in the United States each year.

AJMC reported on ICER’s Asthma topic announcement.

ICER to Review Five Biologic Drugs Used for Severe Asthma


Pharmaceutical News

Inside the Beltway/State Regulatory Developments

It’s time to invoke my spirit animal Shruggie ¯\_(ツ)_/¯ as we talk about the President’s upcoming speech on drug prices. Initially, not much new was expected after the Administration laid out several strategies in their budget proposal. However, this week in speeches from Azar and Gottlieb, more robust strategies were hinted at. We’ll have to wait for the speech to see what happens, meanwhile, several stories on what we might see.

First a report on Azar’s speech at the World Health Care Congress.

HHS secretary: Trump drug pricing plan will go ‘much further’ than budget proposal

Health and Human Services Secretary Alex Azar said the administration is working on a plan to tackle high drug prices that would go “much further” than the proposals in President Trump Donald John Trump Poll: Republican support for Kanye West grew after rapper expressed support for Trump Giuliani calls on Sessions and Rosenstein to shut down Mueller investigation Fox News’ Ingraham calls out Giuliani for contradicting Trump on Stormy Daniels’ payments: ‘That’s a problem’ MORE ‘s budget.

Next, a report on Gottlieb’s speech.

Kickback law might be used to bring down drug prices, FDA’s Gottlieb says

ASHINGTON – The commissioner of the Food and Drug Administration, Scott Gottlieb, on Thursday suggested that a key federal law on kickbacks could be reinterpreted by the government to help rein in the prices of prescription drugs.

Will the future state of the largest PBMs not being independent impact how the industry adapts? Cigna CEO (and future owner of Express Scripts) doesn’t seem to have a problem with a changed landscape.

Cigna CEO Says Insurer Can Adapt to Drug-Price Shift After Deal

The top executive of health insurer Cigna Corp., which recently agreed to take over one of the largest pharmacy-benefit managers, said he’s ready for changes in how prescription drugs are priced and sold in the U.S.

STAT asks if the President has already had an impact on drug prices (spoiler alert: their answer is no).

Can Trump use the bully pulpit to lower drug prices? Don’t bet on it – STAT

ASHINGTON – Is there a Trump effect on drug prices? President Trump has not yet made his much-ballyhooed speech on drug prices, and a rumored executive order never came to pass.

And finally, on this topic, a reminder it’s still Washington as the Hill interviews PhRMA’s CEO Steve Ubl on their efforts to guide the conversation.

Drug industry faces Trump-fueled storm over prices

Drugmakers are trying to navigate a growing storm over high drug prices as President Trump Donald John Trump Poll: Republican support for Kanye West grew after rapper expressed support for Trump Giuliani calls on Sessions and Rosenstein to shut down Mueller investigation Fox News’ Ingraham calls out Giuliani for contradicting Trump on Stormy Daniels’ payments: ‘That’s a problem’ MORE prepares to unveil new actions on the issue.

And speaking of it’s still Washington; what did Novo Nordisk do when they were under pressure for high insulin prices? Raise their campaign contributions and lobbying expenditures.

How A Drug Company Under Pressure For High Prices Ratchets Up Political Activity

This story also ran on This story can be republished for free ( Fortune. details). Business looked challenging for Novo Nordisk at the end of 2016. As pressure mounted over the pharma giant’s soaring insulin prices, investors drove its stock down by a third on fears that policymakers would take action, limit prices and hurt profits.

After reporting that the CDC Director was making nearly twice what previous directors were paid, his salary will be reduced (by an undisclosed amount).

C.D.C. Director’s $375,000 Salary Will Be Cut

The government will lower the $375,000 salary of the new director of the Centers for Disease Control and Prevention, Dr. Robert R. Redfield, after reports that he was being paid considerably more than previous directors, the Department of Health and Human Services confirmed on Monday, though it declined to say what his new pay will be.

Industry Trends

George Church, a CRISPR pioneer, will use an older technology instead for his next endeavor.

George Church, CRISPR Pioneer, Embraces Alternative Tech In Project To Recode A Human Genome

George Church, a Harvard scientist who pioneered the use of the enzyme CRISPR to edit the genes of human cells, will use an alternative, older technology in an effort to recode an entire human genome in hundreds of thousands of locations in order to make it immune to viruses.

The next step in CAR-T development?

Senti scientific co-founder Wilson Wong designs the SUPRA – a new, high performance model CAR-T

Wilson Wong, one of the scientific co-founders behind Timothy Lu’s new synthetic biology startup Senti, has designed a new CAR-T that he believes has the potential to overcome some of the big issues that continues to plague the first generation of drugs now on the market. And he’s tricked it

Is it possible there is too much cancer drug development?

Too many cancer drugs? Crowded market gives investors pause

LONDON (Reuters) – In London’s world-famous Great Ormond Street children’s hospital, Dr. Karin Straathof is excited about a new cell-based medicine that offers hope for toddlers with incurable nerve tissue cancer. Her progress with a handful of children for whom standard care does not work reveals the promise of modern cancer drugs, an increasingly crowded pharmaceuticals field from which investors must try to select future winners.

Bill Gates is worried about the next pandemic. I am too, but Bill has more resources than I do and is using some of them to do something about it. He’ll fund additional work on the development of a universal flu vaccine.

Thinking of developing a universal flu jab? Bill Gates and Larry Page challenge you to do it

Bill Gates, with support from Google co-founder Larry Page, is so set on staving off a deadly flu pandemic that he’s set up a challenge to develop a universal shot. What will it take? Hint: Innovative concepts or technologies not now used in the flu vaccine field are key.

The call is coming from inside the house. In a new twist, critique of pharma pricing is now coming from shareholders. “…22 percent of Bristol-Myers Squibb shareholders voted in favor of a proposal that requires the drug maker to compile reports about the risks created by high prices and examine the extent to which pricing strategies propel executive compensation”.  (Subscription required for STAT Plus article below.)

In rebuke to pharma, one-fifth of Bristol-Myers shareholders favor proposal tying pricing risks to executive pay – STAT

The vote, which is the first of several that shareholders in other drug companies will consider this spring, comes as concerns mount over rising prices, an issue that has put the entire industry on the defensive. For this reason, the outcome was seen as a key temperature reading of public sentiment.

Round 2 of the CRISPR patent battle court case was this week; here’s a report.

UC Berkeley struggles to find sympathetic court in CRISPR patent appeal

ASHINGTON – The University of California, which has been losing the fight over U.S. patents on the revolutionary genome-editing technology CRISPR-Cas9, did not pull any rabbits out of a hat during oral arguments Monday in its appeal of the case.

Company/Drug Specific

Merck publishes study data that may signal that the amyloid theory, the foundation of so much drug development, may be wrong.

Merck study may signal doom for a broad group of pivotal Alzheimer’s studies

The BACE theory in Alzheimer’s R&D is simple. Cut off the flow of amyloid beta to the brain and you can eliminate what is widely believed – though not proven – to be a cause of the disease. Do that, and you could bend the course of this devastating illness

Novartis CAR-T therapy approved for second indication.

Watch out, Gilead-Novartis got the FDA nod it needs to steal your CAR-T market

Novartis’ CAR-T cancer therapy Kymriah expanded its reach with a second FDA approval Tuesday night-an approval that puts it in direct competition with Gilead’s Yescarta. If the rivalry in relapsed large B-cell lymphoma comes down to price, it will be a close race.

Bristol-Myers has more bad news for IDO inhibitors.

Xconomy: Bristol-Myers Latest to Drop IDO Studies in Wake of Incyte Failure

Xconomy New York – [ Updated 5/1/18, 12:34 p.m. See below.] Drug giant Bristol-Myers Squibb is the latest to feel the shockwave caused by the failure of a widely watched cancer immunotherapy nearly a month ago. Xconomy has learned that Bristol (NYSE: BMY) is curtailing work on three late-stage studies testing an experimental cancer drug that it bought for $800 million three years ago.

Bad news for biosims means good news for Roche.

FDA’s back-to-back punches against biosims from Celltrion, Novartis leave Roche’s big Rituxan franchise untouched

A month after the FDA kicked backed Celltrion’s application to market a biosimilar for Roche’s big blockbuster Rituxan, the agency made it a clean sweep Thursday by doing the same for Novartis’ copycat. Novartis didn’t say why Sandoz’s application got the boot, remarking only that it “stands behind the robust

Weekly View In-depth

Drug development meets Sci-fi. Big data and AI, are they game changers or the latest buzzwords?

In a very real expression of faith in big data, NIH seeks to build a million person database.

NIH seeks health data of 1 million people, with genetic privacy suddenly an issue

Private efforts are also underway to combine previously discrete data sets.

What do you get when you integrate healthcare data from multiple sources into one comprehensive, global package? Travis May says he’s on track to find out

While Travis May was running LiveRamp, which he describes as a kind of escrow service for multi-sourced marketing data that became popular with a wide swath of the Fortune 500, he had plenty of time to think about the ways data could change the world. Then he sold the company

The field of artificial intelligence has been making leaps and bounds – is it about to have an impact on drug development?

Out to ‘rethink the drug design process,’ AI star Daphne Koller is creating a new machine learning venture at insitro with some heavyweight backers

Daphne Koller has been busy. Just two months since the high profile AI expert exited Google’s anti-aging biotech Calico Labs, where she was chief computing officer, Koller has gathered a group of marquee investors to back a tech upstart – insitro – with plans to develop a machine learning platform

 

www.icer-review.org 
@icer_review
@mhstein  |  https://www.linkedin.com/in/mitchellhstein/


Sign-up to have the Weekly View delivered to your inbox every Friday morning.

SIGN UP