From the desk of Mitchell Stein
Good morning. As always lots to cover this week. I hope you find all the information I include useful and some of it fun. Today, if you are looking for fun, I point you to the profile of virus hunter C.J. Peters (last item in Industry Trends). After reading the piece, I wondered why he wasn’t wearing a fedora and carrying a whip in his picture. Here’s the full rundown for this week:
ICER in the news – ICER to review treatments for amyloidosis and prostate cancer, coverage of CAR-T report, reports on health care values and opioids include reference to ICER’s work
Inside the Beltway – Azar on drug prices, White House says Medicare & Medicaid driving up drug prices, CAP introduces health plan that includes negotiation on drug prices, Trish Riley on state flexibility, KFF on state initiatives, and Maryland weighs drug cost controls
Industry Trends – How drug firms are using tax cut money, manufacturing quality issues have an impact, biosimilar delays (2 articles), China & CRISPR, PBMs as gatekeepers, finding enrollees for sickle cell trials, immunotherapy doesn’t work when it should and works when it shouldn’t, virus hunter extraordinaire, and organs on a chip
Company/Drug Specific – Flatiron, Novartis, AstraZeneca, and Gilead
Weekly View In-depth – Deals and Flu
Now, on to the news.
ICER In The News
ICER will assess the comparative clinical effectiveness and value of therapies for high-risk prostate cancer and hereditary amyloidosis in upcoming reports. Both reports are set to be reviewed during a public meeting of the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC) in September of 2018.
The review of prostate cancer treatments is expected to assess the clinical effectiveness and value of three anti-androgen drugs: enzalutamide (Xtandi®, Astellas and Medivation), approved by the FDA in 2012, abiraterone acetate (Zytiga®, Janssen), approved in 2011, and apalutamide (Erleada™, Janssen), approved last week. The second report is expected to review inotersen (Ionis Pharmaceuticals, Inc.) and patisiran (Alnylam Pharmaceuticals) for hereditary transthyretin-related (hATTR) amyloidosis, a rare genetic condition characterized by nerve, heart, and eye damage.
Institute for Clinical and Economic Review to Assess Treatments for Amyloidosis, Prostate Cancer in Upcoming Reports
AJMC and the Journal of Clinical Pathways report on ICER’s CAR-T report.
US Food and Drug Administration (FDA)-approved chimeric antigen receptor (CAR) T-cell therapies may be associated with high costs, but the therapies are priced in alignment with their clinical value, according to an Institute for Clinical and Economic Review (ICER) evidence report (February 15, 2018).
Discussion of Choosing Wisely, value and QALYs includes ICER’s work.
When doctors diagnosed Amy Berman with incurable stage 4 inflammatory breast cancer, they told her that the most life-maximizing treatment regimen was probably mastectomy follwed by radiation, all sandwiched between rounds of aggressive chemotherapy. But another oncologist suggested an alternative: a mild hormonal drug that is taken as a pill.
A discussion of opioid issues includes ICER’s work on abuse-deterrent formulations.
NEW YORK – While listening to panelists during the BIO CEO & Investor conference discuss the current state of the opioid epidemic, it occurred to me that there was something about this narrative that seems familiar. I feel like I’ve written this story before.
Inside the Beltway/State Regulatory Developments
Inside the Beltway
HHS Secretary hints at more action on drug prices. (On a related note, the President comments on his work yesterday: Trump: New HHS chief has already lowered drug prices.)
Health and Human Services (HHS) Secretary Alex Azar says his department is working on additional proposals aimed at high drug prices, including some that could be enacted without congressional action.
In a new report released this week the White House states “Government policies and programs like Medicare and Medicaid are dampening competition and artificially driving up the prices of prescription medicines….”
The Center for American Progress (CAP) released a new health care proposal that includes the provision below on drug prices. While no one expects this full proposal to be enacted if the Democrats retake Congress and the White House, it does reflect the priorities of many Democrats.
“Medicare Extra would negotiate prices for prescription drugs, medical devices, and durable medical equipment. To aid the negotiations, multiple nonprofit, independent evaluators would vet data submitted by manufacturers, conduct studies, and make periodic value assessments. If negotiated prices are within the range of prices recommended by all evaluators, Medicare Extra would include the product on a preferred tier with limited cost sharing. If prices for existing products rise faster than inflation, manufacturers would pay rebates on products covered under Medicare Extra—just as they do under the current Medicaid program.”
Health care is a right: No American should be left to suffer without the health care they need. The United States is alone among developed countries in not guaranteeing universal health coverage. Over the past half century, there have been several expansions of health coverage in the United States; today, it is past time to ensure that all Americans have coverage they can rely on at all times.
Trish Riley (Executive Director of the National Academy for State Health Policy) talks about potential state activity on drug costs.
From the Kaiser Family Foundation.
Rising #Medicaid spending on #prescriptiondrugs has prompted many states to look for new ways to control such costs. What initiatives are states considering? After several years of spending growth below five percent, Medicaid spending on outpatient drugs increased 25 percent from $22.4 billion in 2013 to $28 billion in 2014 and another 13 percent in 2015 to $31.7 billion (Figure 1).
From the state that brought you hospital price setting, is drug price setting next?
More details on how drug firms are using money from tax changes (hint, not much going to R&D).
The money is enriching investors and drug company executives, but it isn’t necessarily helping patients.
We talk a lot about innovation and pricing, but the day-to-day details of quality manufacturing are important as well.
Warning letters from the Food and Drug Administration’s Center for Drug Evaluation and Research spiked last year as drugmakers struggled to maintain quality control at their manufacturing sites, according to a new report from PwC. Though the FDA actually issued 22% fewer warning letters overall in 2017 versus 2016, the number coming from its CDER unit rose 35% for a total of 85.
Wow – looks like FDA approval is not the impediment to biosimilars some think it is. Five of the top 10 selling drugs have biosimilars approved, but most are not on the market due to litigation.
Posted 15 February 2018 | By Zachary Brennan Critics of the US Food and Drug Administration (FDA) have long pointed fingers at the agency’s complex and arcane approval process as one of the main reasons for a lack of competition, and consequently, higher drug prices and costs.
This Health Affairs piece discusses the reasons for biosimilar delays and offers possible solutions.
China may already have eight CRISPR clinical studies ongoing (the US has one).
Shaorong Deng is sitting up in bed at the Hangzhou Cancer Hospital waiting for his doctor. Thin and frail, the 53-year-old construction worker’s coat drapes around his shoulders to protect against the chilly air. Deng has advanced cancer of the esophagus, a common form of cancer in China.
U.S. insurers, drug plans more agressive at getting discounts Upcoming drugs for migraine, NASH are a ‘watch out:’ insurer It’s a tough time to be a middleman in the U.S. pharmacy business. Jeff Bezos is teaming up with two fellow billionaires in what could be a prelude to bigger plans to undercut the dominance of intermediaries in the country’s complex health-care system.
Given historical treatment of the impacted population, getting patients to participate in this trial will be no easy task.
he first attempts to use a groundbreaking gene-editing technology in people will likely target patients with sickle cell disease, a crippling inherited disorder that in the U.S. predominantly strikes African-Americans. That should be welcome news, after decades of sickle cell patients being neglected by the health care system, scientists, and drug companies.
We know that immunotherapy does not help a lot of people who we expect it to help – now it seems it does help some people we don’t expect it to help. Still so much to learn.
“We need to study the people who have a biology that goes against the conventional generalizations.” Four women hardly constitutes a clinical trial. Still, “it is the exceptions that give you the best insights,” said Dr. Drew Pardoll, who directs the Bloomberg-Kimmel Institute for Cancer Immunotherapy at Johns Hopkins Medicine in Baltimore.
This week’s episode of science or science fiction – organs on a chip hit the mainstream.
Two pharma giants – Roche and Takeda – are buying into a drug testing technology that wants to be the next generation’s “lab rat,” inking partnerships with the tech’s maker to introduce the systems to their R&D labs. The tech, made by Wyss Institute spinoff Emulate, includes small chips designed to hold living cells in chambers.
A fascinating read – for good or ill, I fear we won’t see virus hunters like C.J. again.
ALVESTON, Texas – Drive a few minutes from the seawall here, where the Gulf of Mexico crashes into this island city, and follow some meandering streets through a subdivision of graceful, low-slung homes. Look for the one with the Mardi Gras doll hanging beside the front door.
As a follow-up to Roche’s purchase of Flatiron, here is a detailed look at their business model. Ironic that what is touted as a tech play relies on human intervention to make the process work.
Flatiron’s key insight was that, in cancer, at least half of the most important data in health records isn’t in structured data fields but rather in unstructured data, the free text fields of pathology reports and clinical notes, and extracting this information (at least today) requires people.
Novartis prepares to shed its generic drug business.
(Reuters) – Switzerland’s Novartis AG () is preparing to auction its U.S. generic pill business, looking to shed a unit that has struggled amid fierce price competition, people familiar with the matter said on Friday. The move illustrates how the unit has diverged from the fortunes of the rest of Novartis’ $10 billion Sandoz generics and biosimilars division.
Only months after positive clinical data, the FDA greatly expanded the approved use of Imfinzi.
LONDON (Reuters) – AstraZeneca’s immunotherapy drug Imfinzi has won crucial approval from U.S. regulators for use in lung cancer, opening up a multibillion-dollar market for a medicine that has so far lagged behind competitors. The U.S.
Gilead gets good news on patent verdict.
The latest victory in a years-long hepatitis C patent battle between Gilead Sciences and Merck goes to Gilead. On Friday, a federal judge overturned a $2.54 billion verdict against the biotech on grounds that a key Merck patent used to support the infringement lawsuit is invalid.
Weekly View In-depth
Albertsons to buy remaining Rite Aid stores.
CLOSE Corrections & Clarifications: An earlier version of this story misidentified the $3.7 billion in earnings before interest, taxes, depreciation and amortization. Supermarket giant Albertsons is buying part of Rite Aid, the nation’s third-largest pharmacy chain, as the companies try to navigate sweeping shifts in the food and health care industries.
Merck purchases Viralytics.
Merck & Co. plans to grow its immuno-oncology pipeline through a nearly $400 million acquisition of Australian drugmaker Viralytics Ltd. Viralytics uses oncolytic viruses to create targeted cancer therapies. Its main asset, Cavatak, is a unique formulation of a common cold virus in early- and mid-stage clinical investigations for a variety of cancer types, including lung, prostate and skin.
Gilead enters deal with Sangamo.
U.S. drugmaker Gilead Sciences Inc will use Sangamo Therapeutics Inc’s gene-editing technology to develop cancer treatments in a deal potentially worth about $3 billion to Sangamo, the companies said on Thursday.
In what can only be described as a lukewarm endorsement, FluMist will once again be recommended (after two years in the wilderness).
luMist, the only non-injected flu vaccine licensed in the United States, will be recommended for use here next winter, ending what was effectively a two-year suspension of the popular vaccine.
While we’re hopefully past the peak of a tough flu season, this is a useful reminder: “A 2014 Cochrane review based on a review of data of trials in more than 24,000 people challenged the assumption that antivirals Tamiflu (oseltamivir) and Relenza (zanamivir) are effective at fighting the flu.”
A number of news stories on Monday linked the flu-related death of a Texas schoolteacher to her reluctance to fill a prescription for a generic version of antiviral drug Tamiflu. They were misleading because there’s no evidence that antiviral medications can prevent an otherwise healthy person from dying of the flu.