ICER Weekly View 01-12-18


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From the desk of Mitchell Stein

Good morning. This week J.P Morgan took over San Francisco with their annual Healthcare Conference. I’ll devote Weekly View In-depth to coverage of the event. Congress also returned to DC resulting in lots of Beltway news. Here’s today’s full rundown:

ICER in the news – Draft scoping document posted for Elagolix (for endometriosis), 2 stories on Humira pricing, Pharma Exec’s look ahead, and drug pricing for communicators

Inside the Beltway – 2 stories on the HHS nominee, new tax law reactions, 340B developments, FDA’s 2018 priorities, a national “right to try” law campaign, and Maryland fighting pharmacy “gag rules”

Industry Trends – Generic manufacturing issues, CRISPR snag, clinicaltrials.gov improvement, Spark’s pricing models, 2 stories on patient charity developments, crisis vaccine development problems, and swallowing diagnostic tests

Company/Drug Specific – Roche’s Rituxan, Pfizer’s changes, GlaxoSmithKline reshuffling, another Alzheimer’s failure, and Teva update

Weekly View In-depth – 2018 J.P. Morgan Healthcare Conference

Now, on to the news.


ICER In The News

ICER has posted a Draft Scoping Document outlining a planned review of elagolix (AbbVie/Neurocrine Biosciences) for the treatment of endometriosis and associated pain. The scoping document will be open to public comment for three weeks until February 1, 2018. Elagolix is currently under review by the FDA, with an approval decision expected by mid-2018. The report will be subject to deliberation during a public meeting of the New England Comparative Effectiveness Public Advisory Council in July of 2018.

The New York Times took an in-depth look at Humira and its pricing includes comments from ICER’s President Dr. Steven Pearson.

Humira’s Best-Selling Drug Formula: Start at a High Price. Go Higher.

“People are skipping doses, people are rationing, people are going into bankruptcy because of this drug,” he said in an interview, arguing that Humira is both more expensive per dose and has a far higher volume than Daraprim. AbbVie, which was spun off from Abbott Laboratories in 2013, declined to comment.

More on Humira includes mention of ICER’s work on the drug.

Pharm Exec’s Washington Correspondent Jill Wechsler’s look ahead includes mention of ICER.

Drug pricing brief for communicators includes mention of ICER’s work.

Drug Pricing For Healthcare Communicators

Biopharmaceutical companies and their research partners are pursuing, discovering and developing therapies for some of the most intractable medical conditions including aggressive cancers, rare genetic diseases and progressive neurological and autoimmune disorders, among others. In many respects, they’re making strides at a faster pace than ever before and achieving outcomes previously not thought possible.

ICER is growing! Interested in joining the ICER team? Check out our open positions in research, health economics, program management, and more on ICER’s Careers Page

This week’s job spotlight:  Senior Visiting Research Fellow (NOTE: The deadline for applications is January 15th!)

Applications are being accepted from distinguished senior clinicians, policymakers, members of the patient community, academic research experts, public or private insurers, purchasers, and representatives from the life sciences industry to participate in ICER’s new Visiting Research Fellowship program. Selected fellows will receive financial support for six- to 12-month visits to work at the ICER headquarters in Boston. Read more about the fellowship here.


Pharmaceutical News

Inside the Beltway/State Regulatory Developments

Alex Azar, nominee to be Secretary of HHS, testified before the Senate this week. Below are two stories covering the nomination. First, a look at his testimony and background, then questions about Eli Lilly’s activities during Azar’s tenure there arise. Despite the questions and controversy, the consensus is that he is on a path to swift confirmation.

HHS Nominee Vows To Tackle High Drug Costs, Despite His Ties To Industry

Senate Democrats on Tuesday pressed President Donald Trump’s nominee for the top health post to explain how he would fight skyrocketing drug prices – demanding to know why they should trust him to lower costs since he did not do so while running a major pharmaceutical company. Alex M.

How Trump’s HHS nominee’s drug company ‘gamed’ a patent

When Donald Trump’s nominee for HHS secretary was a top executive at Eli Lilly, the patent on its blockbuster Cialis was soon to expire. So Lilly tested it on kids. Story Continued Below The drugmaker believed the erectile dysfunction drug might help a rare and deadly muscle-wasting disease that afflicts boys.

Reaction and analysis of the new tax law’s impact on pharma continue. Expectations are that “bringing back cash” from overseas will spur acquisitions resulting in further consolidation.

U.S. pharma executives expect deals to pick up after tax overhaul

SAN FRANCISCO (Reuters) – The Trump administration’s tax overhaul should accelerate major acquisitions by drugmakers in 2018 after a slow year for deals in 2017, according to senior executives from some of the largest pharmaceutical manufacturers. The $1.5-trillion bill, which U.S. President Donald Trump signed into law in December, lowers the income tax rate for U.S.

Two developments this week for the 340B program; organizations have appealed the dismissal of the lawsuit regarding program changes, and Republican congressmen issue a report suggesting more oversight of the program.

AHA, health systems appeal 340B decision

As promised , the American Hospital Association (AHA), Association of American Medical Colleges (AAMC), America’s Essential Hospitals, Eastern Maine Healthcare Systems, Henry Ford Health System and Fletcher Hospital, Inc., on Tuesday when a judge dismissed their 340B drug prescription payment lawsuit appealed the decision.

The FDA announces its priorities for 2018.

FDA Plots Policy Priorities for 2018

The US Food and Drug Administration (FDA) on Thursday announced its priorities for policy activity in 2018, which will include reducing the misuse and abuse of opioids, promoting generic drug and biosimilar competition, creating a total product lifecycle office for medical devices, advancing digital health technologies and strengthening the agency’s workforce.

Push for “right to try” law heading from statehouses to Congress.

Koch-backed groups launch ‘Right to Try’ campaign

Koch brothers-backed groups are launching a campaign urging Congress to pass legislation allowing terminally ill patients to request access to experimental drugs the Food and Drug Administration (FDA) hasn’t approved. Nearly 40 states have this law, known as “Right to Try,” already on their books.

Maryland may become the next state to prohibit pharmacist gag rule in PBM contracts.

Maryland to fight pharmacist ‘gag rule’ on drug costs

Maryland wants to eliminate a “gag rule” that prevents pharmacists from telling consumers that they can pay less for their prescription drugs, joining a nationwide movement to rein in soaring costs. If Maryland succeeds, it would mean that pharmacies would be allowed to inform customers they can pay less for a drug out of pocket than through their insurance.

Industry Trends

The FDA may be approving a record number of generic drugs, but getting those drugs manufactured is becoming problematic.

Plans to Slash Medical Costs in America Run Into Generic Drug Concerns

FDA generics push comes amid quality enforcement woes in India Agency struggles to keep up with lapses as records destroyed As U.S. regulators push hundreds of new generic drugs to market in an effort to drive down prices, the industry is facing a continuing problem in India, where many copycat medicines are manufactured.

There may be a problem with some forms of CRISPR being effective in people. The paper received a great deal of attention, but the full implications (and how much the most recent techniques are at risk) is unclear.

CRISPR hits a snag: Our immune systems may attack the treatment

new paper points to a previously unknown hurdle for scientists racing to develop therapies using the revolutionary genome-editing tool CRISPR-Cas9: the human immune system. In a study posted Friday on the preprint site bioRxiv, researchers reported that many people have existing immune proteins and cells primed to target the Cas9 proteins included in CRISPR complexes.

Reporting to clinicaltrials.gov has vastly improved.

Facing public pressure, research institutions step up clinical trial reporting

he reporting of clinical trial results to a public database – mandated by a 10-year-old federal law – has improved sharply in the last two years, with universities and other nonprofit research centers leading the way, according to a new STAT analysis of government data.

Spark’s is trying new pricing  models, if successful they could set the standard for gene therapies.

Drugmakers see a pricing blueprint in an $850,000 gene therapy

SAN FRANCISCO (Reuters) – Global drugmakers are looking to a tiny biotech’s $850,000 therapy for a rare type of blindness as a model for getting paid for highly expensive – and effective – new medicines. Spark Therapeutics Inc plans to launch its recently approved Luxturna treatment for an inherited genetic mutation that causes blindness in March.

Patient charity funded by industry is fighting back against increased oversight.

A Big Pharma-funded charity that helps patients pay for drugs just sued the government

A charity that helps patients pay for expensive prescription drugs is suing the federal government over what it alleges are unlawful federal restrictions that put a “stranglehold” on its ability to communicate with donors – which happen to be pharmaceutical companies that sell expensive drugs.

The Office of the Inspector General seeks to minimize the fallout from the increased scrutiny of patient charities.

HHS’ OIG to PhRMA: Free Pharmaceuticals Needed in 2018

With a nonprofit that paid almost $100 million in 2015 financial grants to patients now saying that it will not offer such payments in 2018, Gregory Demske, chief counsel to the Department of Health and Human Services’ Office of the Inspector General (OIG), sent a letter on Thursday seeking help from the pharmaceutical industry group PhRMA in providing free drugs.

The current vaccine emergency response system seems unsustainable – a new alternative is desperately needed.

Drug makers signal interest in exiting vaccine development during crises

very few years an alarming disease launches a furious, out-of-the-blue attack on people, triggering a high-level emergency response. SARS. The H1N1 flu pandemic. West Nile and Zika. The nightmarish West African Ebola epidemic. In nearly each case, major vaccine producers have risen to the challenge, setting aside their day-to-day profit-making activities to try to meet a pressing societal need.

Until you’re ready to talk about swallowing miniaturized submarines, I’m not interested.

Gut Check: Gas-Sniffing Capsule Charts The Digestive Tract

To study the human gut and the microbes that live within it, scientists have a couple of options. They can grab a small piece of tissue from the gastrointestinal tract or collect a sample of fecal matter. Neither way is ideal, says Jack Gilbert, a microbiologist and director of the Microbiome Center at the University of Chicago.

Company/Drug Specific

Interesting news regarding MS drugs and Roche’s Rituxan (not approved for MS).

Rituxan Outperforms A Half-Dozen Multiple Sclerosis Therapies In Study

In a study of how people newly diagnosed with multiple sclerosis fared on the first therapy they tried, a drug that isn’t even approved for the disease came out on top. Rituximab, sold by Roche as Rituxan to treat non-Hodgkin’s lymphoma, chronic lymphocytic leukemia, and rheumatoid arthritis, outperformed newer MS drugs from Biogen and Novartis.

Pfizer going through big changes.

Pfizer is axing its neurosciences division, laying off 300 and discarding new drugs

Pfizer is starting out 2018 by ripping up its early-, mid-stage and preclinical R&D operations focused on neurosciences and laying off hundreds of workers engaged in the development work. In a statement, Pfizer $PFE says it is is axing about 300 workers in Connecticut and Massachusetts – about 100 each

The changes are GlaxoSmithKline deeper and wider than previously thought.

GlaxoSmithKline CEO reshuffles 40% of management team in bid to bring in new ideas

GlaxoSmithKline CEO Emma Walmsley has grabbed headlines with some of the top management changes she’s made since taking the helm in March, namely poaching Luke Miels from rival AstraZeneca to head up pharmaceuticals, and appointing Roche and Calico veteran Hal Barron as CSO. But behind the scenes, her scalpel has actually cut much deeper.

The latest Alzheimer’s failure.

One of the most promising drugs for Alzheimer’s disease fails in clinical trials

To the roughly 400 clinical trials that have tested some experimental treatment for Alzheimer’s disease and come up short, we can now add three more. An experimental drug called idalopirdine failed to help people with mild to moderate Alzheimer’s disease in a trio of trials that involved 2,525 patients in 34 countries.

This week’s Teva update.

Teva to cut up to 40 manufacturing sites, with 25 slated to go in next 2 years

Teva has worked out agreements with workers to close another plant in Israel. That is two down and about 40 to go.

Weekly View In-depth

2018 J.P. Morgan Healthcare Conference

JP Morgan held its annual health care conference in San Francisco this week. As usual, lots of news came out of the event. 

First, an overview of the conference from Kaiser.

Must-Attend Health Conference All About The Deals Amid Uncertainty For Millions

SAN FRANCISCO – There’s so much money floating around here this week, you can almost see it wafting through the air. About 10,000 attendees, mostly confident men in well-cut suits and even nicer watches, are packing the elegant Westin St. Francis Hotel for the invite-only J.P. Morgan Healthcare Conference, which ends Thursday.

Here, courtesy of Biopharma Dive, are daily recaps of the proceedings.

JPM18 Day 1: Celgene bullish, Alnylam tempers hopes, Axovant shocks

Another bout of rain grayed skies over J.P. Morgan Healthcare Conference in San Francisco this year. Unlike last year, however, there was more excitement and a bit of buzz as the industry expects deals to pick up now that a tax overhaul is complete.

JPM18 Day 2: Roche on gene therapy, Aimmune nears key readout and outpatient CAR-T

Investors came out in droves Monday night to attend the parties in San Francisco hosted by nearly every law firm, investment house and pharma company. Evercore ISI’s Burgers and Beers featured black suited men spilling into the bedding section of Macy’s, while the famed Tweetup saw communications folks waiting in the rain with the venue at capacity.

JPM18 Day 3: Valeant and Acorda make plans, promises of M&A

The rain has slowed down in San Francisco and biotech stocks have recovered somewhat from earlier in the week, but as day three of the J.P. Morgan Healthcare Conference wraps up, it’s become clear that this year isn’t going to shake up the industry.

Here is the candidate for firm having the worst conference ever.

Hung out to dry? Axovant’s terrible JPM week

David Hung is the CEO who sold his company, Medivation, to Pfizer for $14 billion. He got the plaudits, a big payday and seemingly a free pass to almost any biopharma job he wanted. Now his decision to join Axovant looks more like a gamble that isn’t paying off.

A candidate for the most ambitious announcements of the conference.

Bluebird hopes for 3 drug approvals by 2019, including CAR-T and gene therapies

Bluebird bio is hoping for an FDA OK for three of its experimental drugs by 2019, including the CAR-T bb2121 partnered with Celgene hat caused a buzz at ASH. That’s according to the company’s CEO Nick Leschly, who announced bluebird’s ambitious plans during his presentation at JPM. The CAR-T, meant

Perhaps the most frustrated firm at the conference.

Novo Nordisk tries to tackle Ablynx and bring it into the fold with a $3B buyout offer, but the biotech keeps saying no

Playing a hot hand with a promising late-stage asset and a newly completed $200 million IPO, Ablynx has waved away two bids by Novo Nordisk to acquire the company for a hefty premium with a bid of up to $3.1 billion. But Novo isn’t taking no for an answer. The

Forbes Matthew Herper with some closing thoughts from the conference.

Wait, It Costs How Much? Biotech Balances Optimism, Uncertainty For 2018

I cover science and medicine, and believe this is biology’s century. Inventors, investors and fundraisers from the pharmaceutical industry arrived at the annual J.P. Morgan Healthcare Conference, their annual kickoff party, with lots of questions. They’re probably leaving with them, too.

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