ICER Weekly View 04-13-18

From the desk of Mitchell Stein


Good morning. I couldn’t decide how to lead off this morning… Do I write about today’s date? Do I write about how cold it still is? Do I pick out a favorite story to highlight? Since I can’t decide, let’s jump into the news. This week we’ll look at:

ICER in the news – Draft evidence report on migraine prevention, draft scoping document on therapies to prevent hereditary angioedema attacks, release of two white papers on use of real world evidence, Pearson on cost, coverage of opioid use disorder topic announcement, and coverage of PCSK9s

Inside the Beltway – Rare pharma loss, patient groups march to Sarepta, questions about former CVS exec overseeing drug pricing efforts, Gottlieb’s popularity, and NASHP on state transparency laws

Industry Trends – new oncology drug prices rising faster than their value, some generics may be priced too low, CVS and pharma don’t agree on who’s responsible, stock analyst isn’t buying the prices are lower argument, CVS to flag therapeutic equivalents, abortion pill turns into orphan drug, company payments influence oncologists’ choices, diagnostic tests don’t always agree, animal studies not always reliable, and CRISPR comes to a movie theatre near you

Company/Drug Specific – Novartis, GSK, Merck (Keytruda), AbbVie, Roche, Incyte, and Nuplazid

Weekly View In-depth – Alzheimer’s and New Factories

Now, on to the news.

ICER In The News

ICER released a Draft Evidence Report assessing the comparative clinical effectiveness and value of calcitonin gene-related peptide (CGRP) inhibitors as preventive treatments for chronic or episodic migraine. Therapies reviewed include erenumab (Amgen/Novartis), fremanezumab (Teva), and galcanezumab (Eli Lilly). Findings contained in this report are preliminary and subject to change based on additional stakeholder input and further analysis of data. STAT Plus (subscription required) took a look at the introduction of these drugs: New migraine drugs may give insurers a headache.

Institute for Clinical and Economic Review Releases Draft Evidence Report on CGRP Inhibitors for Migraine Prevention


ICER has posted a Draft Scoping Document outlining a planned review of the comparative clinical effectiveness and value of therapies for hereditary angioedema, a rare condition characterized by attacks of deep tissue swelling within the skin and/or mucosa. ICER’s report is expected to focus on four therapies for the prevention of hereditary angioedema attacks: lanadelumab (Shire), which is currently under review by the FDA with an approval decision expected in August 2018, and three C1 inhibitors (Haegarda®, CSL Behring; Cinryze®, Shire; and Ruconest®, Pharming). The report will be subject to deliberation during a public meeting of the California Technology Assessment Forum in October of 2018. 

Institute for Clinical and Economic Review Posts Draft Scoping Document on Therapies for Prevention of Hereditary Angioedema Attacks


ICER, in collaboration with the Office of Health Economics (OHE), has released two new white papers to provide guidance to payers and manufacturers on the development and use of real world evidence for drug coverage and formulary decisions. The papers were generated as part of the annual ICER Policy Summit, where life science and payer organizations meet to develop collaborative approaches to addressing key policy issues related to the generation and application of evidence to improve value in the US health care system.

Institute for Clinical and Economic Review Publishes Guidance on Improving the Development and Use of Real World Evidence for Drug Coverage and Formulary Decisions


ICER’s President Steve Pearson was part of a panel on health care costs. This article discusses the panels including quotes from Dr. Pearson.

How to reduce health care costs? Experts say lots of ideas, few successes

OSTON – Whenever experts get together to talk health care, the issue of ballooning costs is never far behind. And an event in Boston hosted by STAT and The Atlantic was no exception, as experts wrestled with the multitude of challenges facing patients, clinicians, payers, and policymakers in the U.S.

ICER’s topic announcement on opioid use disorder was covered by The PharmaLetter (below) and Inside Health Policy (subscription required).

ICER eyes on treatments for opioid use disorder

ICER eyes on treatments for opioid use disorder

Look at reimbursement for PCSK9s cites ICER’s work on the topic but does not discuss the recent Evidence Update.

PCSK9s appear to be meeting less payer resistance

Major U.S. insurers appear to be opening up to a relatively new and expensive class of cholesterol-lowering drugs. They accepted nearly three-fourths of claims for the drugs in 2017, according to a new report – a marked improvement from prior years.

Pharmaceutical News

Inside the Beltway/State Regulatory Developments

Bloomberg takes a look at a rare loss for Pharma – changes in the budget bill to the discount required for Medicare recipients in the donut hole.

A Rare Loss for U.S. Pharma Lobby Will Cost the Industry Billions

Medicare-rule defeat raises questions about battles ahead ‘They’re in a defensive position’ as Trump ponders drug plan Deep in a budget deal Congress passed earlier this year — just 118 words in Section 53116, a little before passages on prison reporting data and payment yields for seed cotton — was a hit to pharmaceutical companies that will cost them billions, and could signal more losses to come.

Will patient groups convince the government to march in and reclaim several patents? Not likely, but this represents a new front in the drug pricing battles.

Should Sarepta’s patents be seized by the government? Patient advocates pitch controversial drug pricing proposal

Six advocacy groups are sending a big ask to the federal government to lower the price of one rather expensive drug for Duchenne muscular dystrophy, petitioning health regulators to flex power it’s never exercised before. Amidst a years-long debate over drug pricing, Sarepta has hit a sensitive chord with the

“Rep. Keith Ellison is questioning the hiring of a former CVS executive to oversee drug pricing efforts at the Department of Health and Human Services.

Dem questions hiring of former CVS executive to lead drug pricing reform

Rep. Keith Ellison Keith Maurice Ellison Dem says frosted glass indicates lack of ‘transparency’ at CFPB Overnight Health Care: Trump officials create new mandate exemptions | Insurance official warns of premium spikes | Dem questions hiring of drug pricing official Dem questions hiring of former CVS executive to lead drug pricing reform MORE (D-Minn.)

Why is Gottlieb so popular?

Why Scott Gottlieb is the one Trump official everybody seems to like

When Scott Gottlieb was appointed commissioner of the Food and Drug Administration last May, some were concerned he’d be a shill for the pharmaceutical industry. A major sticking point at his confirmation was what Sen. Patty Murray (D-WA) called “unprecedented financial entanglements” – including previous posts on five pharmaceutical company boards.

NASHP reviews state transparency laws.

States’ Prescription Drug Transparency Laws Open the Black Box of Drug Pricing – NASHP

Several states have passed drug price transparency laws that require drug makers to report the reasons behind dramatic price increases. These laws are an important first step to shine a light on why drug prices are rapidly climbing. To address a problem, state health policymakers need to understand it, but transparency laws don’t yet give …

Industry Trends

Study shows that as oncology drug prices rise, the value of those drugs is not keeping pace.

As cancer drug prices climb, value not keeping pace

By Anne Harding(Reuters Health) – The cost of new anti-cancer drugs increased more than five-fold from 2006 to 2015, but a new analysis suggests that cancer patients and insurers may be getting less for their money.Anticancer medications account for the lion’s share of global drug spending, and the average price per month of these drugs is known to have more than doubled in recent years, Dr. Kelvin Chan of Sunnybrook Health Sciences Centre in Toronto and colleagues note in the Journal of Oncology Practice.

We know some drugs are priced too high – it’s also possible some are priced too low.

Why We May Lose Generic Drugs

The mood at the annual generic drug industry confab in Orlando in February was especially somber. The discussion during one panel was all about plunging drug prices, consolidation among drug-buying groups, and the increasingly cutthroat nature of the business.

It’s your fault. No, it’s your fault. Narrator: It was both their faults.

CVS points finger back at pharma for drug prices

First Published on In the latest tussle between pharma and pharmacy benefit managers, a CVS Health report finds almost flat drug pricing growth for its pharmacy benefit management clients at 0.2%, against a manufacturer price growth of 8.3% for specialty brands and 9.2% for “traditional” brands.

Stock analyst takes a critical look at industry’s claims of lower prices.

Despite reports from Merck and Johnson & Johnson, pharma’s net prices ‘likely not coming down’: analyst

Are drug prices coming down? Two leading drugmakers presented numbers suggesting the industry is facing increased pricing pressure, but Bernstein analyst Ronny Gal remains skeptical. Johnson & Johnson recently reported that net prices fell 4.6% in 2017 after the company paid out $15 billion in rebates and discounts.

A new tool will flag cheaper therapeutic equivalents so that CVS pharmacists can offer to assist patients with a switch.

CVS pharmacists will have new tools to help patients save money on drugs

A review of where things stand with insurers providing rebates to consumers at the point-of-sale.

Insurers Look to Pass Drug Price Breaks Straight to Consumer

WILL YOU START RECEIVING THEM SOON? That’s unlikely. Aetna estimates that 3 million customers could receive rebates when it starts offering them next year, while UnitedHelathcare’s plan will initially apply to over 7 million people. Those are big numbers but small slices of the more than 67 million U.S.

In this case, they’ve figured out how to do indication specific pricing.

How A Drugmaker Turned The Abortion Pill Into A Rare-Disease Profit Machine

Even though the $550 yellow pills sold as Korlym have a controversial origin as the abortion pill, Leslie Edwin says they “gave me life.” The 40-year-old Georgia resident lives with Cushing’s syndrome, a potentially deadly condition that causes high levels of the hormone cortisol to wreak havoc on a body.

Choices oncologists make correlate with payments from drug manufacturers. You can read the JAMA Internal Medicine article here.

Cancer drug choices tied to drugmaker payouts to doctors

By Lisa Rapaport(Reuters Health) – Some oncologists may be more likely to prescribe certain cancer medicines when they receive payments from the companies that make these drugs, a U.S. study suggests. Researchers examined data on payments drug companies made to doctors in 2013 for research funding as well general payments such as gifts, fees for speaking or consulting work, meals or travel.

Great that we know Keytruda works on a certain type of tumor – not so great that we can’t reliably identify who has those tumors.

Cutting-edge cancer drug hobbled by diagnostic test confusion

A landmark cancer drug approved last year seemed to herald a long-anticipated change in the treatment of some tumours: with medicines selected on the basis of molecular markers, rather than the tissue in which the cancer first took root. But clinicians and researchers are struggling to put that theory into practice.

Half of the studies used to justify the start of human testing included no more than eight animals.

Medical studies in humans often launched on faulty evidence base

By Anne Harding(Reuters Health) – New research reveals serious flaws in the animal studies that regulators and ethicists use to decide if an experimental drug should be tested in humans.Dr. Daniel Strech, a bioethicist and professor at Hannover Medical School in Germany and his colleagues are the first to take an independent look at so-called investigator brochures (IBs), which regulatory agencies review to weigh the risks and benefits of the experimental treatment and determine whether trials in humans should move forward.

On a related topic, here’s a review of what monkeys have (and have not) taught us about CRISPR.

CRISPR trials are about to begin in people-but we still don’t know how well it works in monkeys

VCG | Getty Sometime this year, people in the US and Europe will start getting treated for diseases using the gene-editing tool CRISPR, but a big question remains-will it actually work? Our primate cousins may hold the answer. The first use of CRISPR to edit human cells in a dish was reported in 2013.

Speaking of monkeys (apes) and CRISPR, we’ll close this section with some fun. STAT reviews a new movie starring The Rock where CRISPR wreaks havoc. (And yes, if you are of a certain age and remember a video game by the same name, the movie is “based” on that game.)

Could CRISPR create monster animals? STAT reviews ‘Rampage’

e here at STAT cover CRISPR a lot. But it’s not every day we get to cover Dwayne “The Rock” Johnson. The Rock and the genome-editing technology meet in a new movie, ” Rampage,” coming out Friday. Through a freak accident, a gorilla, a wolf, and a crocodile ingest some CRISPR complexes.

Company/Drug Specific

Novartis makes $9 billion purchase as their strategic shift continues.

Why Did Novartis Spend $9 Billion On Gene Therapy? CEO Says: ‘A Journey To Focus On Data And Digital’

“We’re in a journey to focus Novartis as a medicines company powered by data and digital,” chief executive Vas Narasimhan said on a conference call explaining the deal to analysts. “And already this year, we’re off to a strong start to realize that goal.”

GSK sheds rare disease unit, but not the way it wanted to.

Continuing an R&D revamp, GlaxoSmithKline hands off its rare disease unit to Orchard Therapeutics

GlaxoSmithKline couldn’t find a buyer for their gene therapy unit, but they did find a taker. GSK said this morning that it has lateraled off the group to a venture-backed startup called Orchard Therapeutics, which includes co-founder Andrea Spezzi in a group of ex-GSK staffers with close ties to Strimvelis,

Good news for lung cancer patients, and for Merck.

Merck’s Keytruda upends lung cancer market with latest survival win

Merck’s Keytruda already has the immuno-oncology lead in non-small cell lung cancer, and it’s making sure it stays that way. On Monday, it rolled out new monotherapy data that could significantly expand its patient pool. In a closely watched trial, the blockbuster topped chemo at improving overall survival among first-line patients with the disease.

AbbVie beat its own Humira for RA with new phase 3 data.

With study success, AbbVie’s arthritis hopeful looks to be a safer bet

AbbVie Inc.’s experimental arthritis drug upadacitinib succeeded in its fourth and largest-to-date Phase 3 study, beating out both placebo and the drugmaker’s top-selling Humira in treating rheumatoid arthritis. Treatment with AbbVie’s new drug, a JAK1 inhibitor, helped more patients achieve a 20% improvement in joint tenderness through 12 weeks and led to a higher proportion of clinical remission than either Humira or placebo, according to data released April 9 by AbbVie.

Roche pauses phase 2 study after patient deaths.

Roche pauses Tecentriq/Cotellic cancer trial after 4 patient deaths

Following the deaths of four patients, Roche said in a statement sent to BioPharma Dive that it has “made a decision to temporarily halt recruitment in the MODUL Phase 2 signal seeking study for metastatic colorectal cancer” following a recommendation from an independent data monitoring committee.

Bad news for Incyte and perhaps IDO inhibitors in general.

Incyte’s cancer drug fails trial, a major blow for hopes for new treatment

he first real clinical test of the cancer immunotherapy combination thesis has come back negative. Incyte said Friday that its experimental drug epacadostat failed to improve the efficacy of Merck’s checkpoint inhibitor Keytruda when the two drugs were used together to treat patients with newly diagnosed melanoma.

In hindsight, it’s hard not to ask the question if Nuplazid should have been approved.

Reports of deaths spark concern over new drug

Still, to recommend approval, the advisory committee would have to find that the drug’s potential benefits outweighed its risks for its intended patients. Some FDA officials concluded that Nuplazid’s public health benefit was enough to merit approval of the drug.

Weekly View In-depth


New definition proposed for identifying the disease; it bases the definition on biological changes instead of diagnostic symptoms. (Two stories on the proposal, each highlighting different information.)

Scientists Push Plan To Change How Researchers Define Alzheimer’s

An international coalition of brain researchers is suggesting a new way of looking at Alzheimer’s. Instead of defining the disease through symptoms like memory problems or fuzzy thinking, the scientists want to focus on biological changes in the brain associated with Alzheimer’s.

After yet another PhIII Alzheimer’s failure, experts try to map a path out of the wrecking field

Pushed by a 15-year record of clinical failures and pulled by an FDA searching for a practical new path forward for Alzheimer’s drug research, a joint committee organized by the NIH’s National Institute of Aging and the Alzheimer’s Association is suggesting a biomarker-based approach to defining the illness that can

This proposal comes in the wake of yet another Phase 3 failure.

Latest Phase 3 failure underscores Alzheimer’s indomitable nature

The Alzheimer’s disease space saw another late-stage drug failure on Monday as vTv Therapeutics Inc. announced its lead candidate didn’t outperform placebo in Part A of the biotech’s STEADFAST program. STEADFAST consists of a pair of identical Phase 3 studies, each enrolling 400 participants and evaluating the efficacy of vTv’s azeliragon through two cognitive tests.

New factories

Lorenzo is building in Texas.

Lonza’s virus factory shows gene therapy’s Texas-sized promise

ZURICH (Reuters) – Swiss drugmaker-for-hire Lonza () is betting that trillions of customized viruses made at a giant factory in Texas will be the lucrative raw materials of a medical revolution. The 121-year-old Basel-based contract manufacturer (CMO) has a long history supplying ingredients for other companies’ drugs.

Amgen is building in Rhode Island.

Amgen goes back to Rhode Island to build its first next-gen manufacturing site in the US

In a bid to lead the way in developing a new generation of more efficient biologics manufacturing facilities, Amgen $AMGN is investing $160 million in a new plant to be constructed on its Rhode Island base. The pharma giant $AMGN – headquartered in Thousand Oaks, CA – has had a 
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