ICER Weekly View 03-16-18

From the desk of Mitchell Stein


Good morning. Years from now when you look up the phrase “too much news” you’ll find this past week as the case study. True on the national front (cabinet shake-ups, special elections, student protests, the loss of one of the brightest among us), and true for ICER, whose staff has been VERY busy. This week in ICER In The News we have ICER reevaluating alirocumab in light of the ODYSSEY study results, a Health Affairs blog co-authored by ICER’s President, another Health Affairs blog that compares ICER to NICE, appearing in the report from the President’s Cancer Panel, and much more. Then of course, there is all the other industry news. So grab another cup of coffee because we’ve got a lot to cover. Here’s this week’s full lineup:

ICER in the news – New evidence update for alirocumab in light of the Odyssey results, new evidence report on emicizumab, new draft evidence report on CFTRs, two new draft scoping documents, ICER’s Dr. Pearson co-authors Health Affairs blog, second Health Affairs blog asks the question is ICER becoming America’s NICE?, President’s Cancer Panel recommendations include ICER, patient groups tackle affordability and engage with ICER

Inside the Beltway – Senate 340B hearing, right-to-try bill fails in the House (for now), bill introduced to prevent “gag clauses,” Oregon’s Gov signs transparency bill

Industry Trends – PBMs with a target on their backs, a look behind the curtain at a PBM contract, patients overpaying, is there evidence for “off-label” use?, primer on drug pricing, Theranos slap on the wrist penalty, new technologies, Centene finds a PBM to invest in, from manufacturer CEO to PBM chief

Company/Drug Specific – Walgreens, Sarepta, Solid Biosciences, and AstraZeneca

Weekly View In-depth – Direct-to-Consumer Advertising

Now, on to the news.

ICER In The News

Preliminary New Evidence Update for alirocumab

ICER released a Preliminary New Evidence Update for alirocumab (Praluent®, Regeneron/Sanofi), based on results from the ODYSSEY outcomes trial presented at last weekend’s American College of Cardiology’s 2018 Scientific Session. Under ICER’s established protocols for accepting in-confidence data, an agreement with Regeneron and Sanofi allowed ICER to evaluate the new evidence and, working with a team of academic faculty from the University of California, San Francisco, to update its cost-effectiveness analyses and associated value-based price benchmarks for this drug. ICER’s value-based price benchmarks suggest a price range that aligns fairly with the added benefits of new treatment options for patients and the health care system.

ICER has calculated two updated value-based price benchmarks, net of rebates and discounts, for alirocumab in patients with a recent acute coronary event: $2,300-$3,400 per year if used to treat all patients who meet trial eligibility criteria, and $4,500-$8,000 per year if used to treat higher-risk patients with LDL cholesterol ≥ 100 mg/dL despite intensive statin therapy.

Both the ODYSSEY results and ICER’s updated work were heavily covered by the media. Here are links to some of the stories:

Institute for Clinical and Economic Review Updates Value-Based Price Benchmark for Alirocumab, a PCSK9 Inhibitor for Treatment of High Cholesterol


ICER released an Evidence Report assessing the comparative clinical effectiveness and value of emicizumab (Hemlibra®, Genentech), finding that it offers important improvements in outcomes for people with hemophilia A and inhibitors to Factor VIII, while lowering costs associated with the treatment of this condition. This Evidence Report will be the subject of an upcoming public meeting of the New England Comparative Effectiveness Public Advisory Council on March 29, 2018.

Institute for Clinical and Economic Review Finds Emicizumab Reduces Treatment Cost While Improving Health of People with Hemophilia A


ICER released a Draft Evidence Report assessing the comparative clinical effectiveness and value of cystic fibrosis transmembrane conductance regulator (CFTR) modulators, including combination therapies tezacaftor/ivacaftor (SymdekoTM, Vertex Pharmaceuticals) and lumacaftor/ivacaftor (Orkambi®, Vertex Pharmaceuticals), as well as the monotherapy ivacaftor (Kalydeco®, Vertex Pharmaceuticals), for the treatment of cystic fibrosis. The Draft Evidence Report and Draft Voting Questions are now open to public comment until April 12th.

Institute for Clinical and Economic Review Releases Draft Evidence Report on CFTR Modulator Therapies for Cystic Fibrosis


ICER posted two Draft Scoping Documents outlining planned reviews of 1) enzalutamide (Xtandi®, Astellas and Pfizer), abiraterone acetate (Zytiga®, Janssen), and apalutamide (Erleada™, Janssen) for certain types of high risk prostate cancer, and 2) inotersen (Ionis Pharmaceuticals, Inc.) and patisiran (Alnylam Pharmaceuticals) for hereditary transthyretin-related (hATTR) amyloidosis. The scoping documents will be open to public comment for three weeks until April 2, 2018 at 5pm ET.

The release was covered by AJMC (ICER Outlines Planned Review of Antiandrogen Therapies for nmCRPC)

Institute for Clinical and Economic Review Requests Public Comment on Two Draft Scoping Documents: Treatments for High-Risk Prostate Cancer and Amyloidosis


ICER President Dr. Steve Pearson co-authored this blog post with Len Nichols and Amitabh Chandra. It is one of three new Policy Options Papers examining factors driving prescription drug prices published in Health Affairs.

Policy Strategies For Aligning Price And Value For Brand-Name Pharmaceuticals


Thoughtful Health Affairs blog asks the question is ICER becoming America’s NICE?

America’s “NICE”?


On Tuesday, the President’s Cancer Panel released their report on value, affordability, and innovation. The report recommendations center on ensuring alignment of drug prices with their value, and ICER figures prominently in how to make that happen. The following coverage discussed ICER’s role: The Pharma Letter – President’s Cancer Panel: action on drug prices “national priority”, BioPharmaDive – Federal panel calls for ‘urgent action’ on cancer drug prices and Inside Health Policy – Trump’s Advisory Board Suggests National Academies Panel Lead Rx Value-Based Efforts (subscription required).

Promoting Value, Affordability, and Innovation in Cancer Drug Treatment

This report recommends actions to ensure alignment of drug prices with their value, promote use of high-value drugs, and support continued innovation in cancer drug development.

As patient groups become more active on the affordability front, some are engaging with ICER and participating in ICER’s process.

Xconomy: Despite Pharma Ties, Some Patient Groups Protest High Drug Prices

Xconomy National – For 20 years, Deborah Long (pictured) has been relying on powerful drugs to keep her multiple sclerosis in check. Her out-of-pocket costs were pretty reasonable until two years ago, she says, when she had to switch insurance companies. Her new insurer made her switch to a different drug, Copaxone.

Pharmaceutical News

Inside the Beltway/State Regulatory Developments

Report on Thursday’s Senate hearing on the 340B program.

Senate 340B Hearing Puts Spotlight on Differing Statistics

The dividing line between Senate Democrats and Republicans on the 340B drug pricing program was set on Thursday, with Democrats siding with the hospitals, saying the savings from the program is desperately needed for the poorest populations, while Republicans took the side of drugmakers, saying the program is being abused and needs to be reformed.

The right-to-try bill failed to pass the House this week. The bill was supported by the Administration but many patient groups came out against it once the final text was posted over the weekend. It was voted on under a suspension of rules meaning it required 2/3 to pass. It was announced on Thursday that the Rules Committee would discuss the legislation on Monday (3/19). When the bill is brought up under regular order, it is sure to pass leading to a conference committee to iron out major differences with the previously passed Senate version.

Right to Try fails amid warning from regulators, patient groups

A Republican-led effort to allow so-called Right to Try legislation failed to move forward late Tuesday in the House of Representatives, amid opposition – both muted and loud – by regulators, patient groups and the some in the pharmaceutical industry.

A new bill is introduced targeting “gag clauses.”

Senators target ‘gag clauses’ that hide potential savings on prescriptions

A bipartisan group of senators introduced a bill Thursday to ban “gag clauses” that can hide potential savings on prescriptions from consumers at the pharmacy counter. The clauses, which keep pharmacies from proactively telling customers they could save money on a prescription if they paid out of pocket instead of through insurance, has sometimes been included in contracts between pharmacies, insurers and companies that manage drug benefits for employers called prescription benefit managers (PBMs).

The Governor of Oregon signed the state’s new drug price transparency bill.

Oregon Governor Signs Drug Price Transparency Bill

Posted 13 March 2018 | By Zachary Brennan States are increasingly seeking more information about high drug prices, and Oregon just became the latest to turn a drug price transparency bill into a law. Gov. Kate Brown (D) signed the bill Tuesday, Kate Kondayen, press secretary for Brown, told Focus via email.

Industry Trends

PBMs are being targeted as a solution to high drug prices – but the coming lack of stand-alone PBMs as well as the fact that you can only save so much cutting out the middleman means the results of this targeting will be limited.

The first target on drug prices: pharmacy benefit managers

Top Democrats tell me that if they take back the House in November, a restoration of Speaker Nancy Pelosi is no longer guaranteed. In fact, some well-wired House Democrats predict she will be forced aside after the election and replaced by a younger, less divisive Dem.

In a rare look behind the curtain, Axios’ Bob Herman got his hands on a template Express Scripts contract. Following publication of this article, Express Scripts posted a response.

Inside a drug pricing contract

A contract template used by Express Scripts, the largest pharmacy benefit manager in the U.S., provides a window into how pharmacy benefit managers – middlemen that manage drug coverage for businesses throughout the country – steer negotiations with drug companies to benefit their own financial interests.

No one will be surprised to learn that patients sometimes pay more for drugs with their insurance than they would without – the surprising part is the extent: it happens 23% of the time.

Patients Overpay For Prescriptions 23% Of The Time, Analysis Shows

As a health economist, Karen Van Nuys had heard that it’s sometimes cheaper to pay cash at the pharmacy counter than to put down your insurance card and pay a copay. So one day, she asked her pharmacist how much her prescription would cost if she didn’t use her health coverage and paid cash.

Drugs are often used “off-label” if they appear in professional guidelines (compendiums). However, research shows that those compendium recommendations often have no evidence to back them up.

Taking stock of the evidence for ‘off-label’ use of cancer drugs

Looking for a primer on prescription drug pricing? The American Academy of Actuaries has you covered.

Prescription Drug Spending in the U.S. Health Care System An Actuarial Perspective

Although her “misrepresentations” led to $700 million in investments, the fine agreed to was only $500,000.

Elizabeth Holmes, Theranos C.E.O. and Silicon Valley Star, Accused of Fraud

Elizabeth Holmes, the founder and chief executive of the blood-testing company Theranos, was charged with fraud by the Securities and Exchange Commission on Wednesday for raising more than $700 million from investors by falsely promoting a key product, the commission said. In announcing the charges, the S.E.C.

Interest in new technologies continues to grow.

From app store to drug store, digital health is redefining pharma’s pipeline

he pitches always sounded promising: A new software app could track glucose levels for people with diabetes or soothe the brains of insomniacs. Most pharma executives would politely smile and nod, but then park their money somewhere else. Not anymore.

Centene, feeling left out, has found its own PBM to invest in.

Centene partnering with PBM RxAdvance

Centene, a Medicaid managed care and increasingly Affordable Care Act (ACA) plan payer, announced that it’s investing in RxAdvance , a cloud-based pharmacy benefit manager (PBM). The St. Louis-based payer said the partnership is both a “customer relationship and strategic investment in RxAdvance.” Centene said it may expand its equity investment in the company down the road.

This is an interesting switch – from CEO of GlaxoSmithKline to head of a PBM (with a one year retirement in-between).

From drugmaker to drug manager: Ex-GSK chief Andrew Witty jumps to the helm of giant Optum

Over a period of 10 years, Andrew Witty made his reputation selling drugs around the world as CEO of London-based GlaxoSmithKline. Now, a year after his retirement, he’s taking a leadership role in managing drug benefits in one of the largest, fastest growing outfits in the US. Witty has been

Company/Drug Specific

Class action suit against Walgreen’s generic drug pricing moves forward.

Walgreen Must Face Lawsuit Over U.S. Generic Drug Pricing

(Reuters) – A federal judge on Friday ordered Walgreen Co to face most of a proposed class action lawsuit accusing the drugstore chain of fraudulently overcharging customers for a decade when they bought generic drugs through private insurance, Medicare or Medicaid.

Sarepta gets good news from the FDA. The new drug’s impact on patients is unclear.

Sarepta Says FDA Welcomes Review Of New Muscular Dystrophy Drug

Sarepta Therapeutics says regulators at the Food and Drug Administration have told it they were open to reviewing its next experimental treatment for Duchenne muscular dystrophy, meaning that it could file an application for the new medicine by the end of this year.

Solid Biosciences’ very bad week continued as the FDA put a hold on their new drug and their investment banker expressed concern.

UPDATED: FDA slaps a hold on Solid Bio’s gene therapy for Duchenne MD in wake of safety alert

Just a few weeks after Solid Biosciences $SLDB put out an 11th hour notice that its gene therapy for Duchenne muscular dystrophy had been put on a partial clinical hold due to safety concerns, just ahead of its $125 million IPO, the biotech is back Wednesday evening with the news

Results from the Mystic trial will be delayed. It’s unclear if this is good or bad news.

AstraZeneca’s Mystic trial hits another snag, delaying Imfinzi’s lung cancer expansion

AstraZeneca’s immuno-oncology drug Imfinzi fell short in a closely watched lung cancer trial, Mystic, last year, but company executives pointed out that Imfinzi could later prove it helps patients live longer. But later just got even later, and at a time when Merck’s Keytruda is racing ahead.

Weekly View In-depth

Direct To Consumer Advertising

So, remember that episode of Black-ish where Dre was diagnosed with diabetes? You have Novo Nordisk to thank for that.

Ask Your Doctor. Until Then, Here’s a Word From Our Sitcom.

A reader recently wrote in about drug advertising. “I used to work in production of TV commercials,” she emailed. “Unless I am mistaken, the U.S. is one of only 2 countries IN the WORLD that allow television advertising of pharmaceuticals.

On a related note, recent study finds most DTC ads are not fully compliant with regulations.

Pharma taking liberties with DTC ads, says study

Drugmakers face guardrails governing marketing their products in TV ads, including a bar on teasing indications not approved by the FDA – but a new study finds pharma companies may be bending the rules. The study, published in the Journal of General Internal Medicine earlier this month, found no ads examined described all medication risks “quantitatively,”and very few were compliant with Food and Drug Administration guidelines.

The FDA is looking into several aspects of DTC ads.

FDA Lays Out Plans for Future Research on Pharmaceutical Ads and Promotions

Posted 12 March 2018 | By Zachary Brennan To better inform its regulation of biopharma advertising and promotion, the US Food and Drug Administration (FDA) has laid out plans for upcoming research.

DTC spending fell in 2017.

Here’s a DTC shocker: Pharma ad spend actually fell in 2017 for first time in years, Kantar says

​​​​​​​Pharma advertising, which typically delivers reliable growth, dropped in 2017 for the first time since the 2008 recession. Spending last year totaled $6.13 billion, a 4.6% decline, Kantar Media says, dragged down by advertising cuts at four of the top five pharma spenders-Pfizer, AbbVie, Allergan and Merck. 
@mhstein  |

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