ICER Weekly View 10-05-18

From the desk of Mitchell Stein


Good morning. Nobel prizes were awarded this week with the winners in two categories touching on our area of interest. We’ll have full coverage below, but before we get to this week’s news, a bit of reflection. The winners of the awards are examples of the heights we can climb to — and by comparison with other stories sharing the front page, a reminder of the depths we are capable of. The same week as new winners were announced, a previous winner in physics (Leon Lederman) passed away. His obituary included the sad revelation that his Nobel prize had been sold years earlier to help pay for his medical expenses. As I said, heights and depths. Other news we’ll cover this week includes: 

ICER in the News – Final Evidence reports on antiandrogen therapies for prostate cancer and treatments for hereditary transthyretin amyloidosis, op-ed discusses ICER’s positive contribution, new pro-pharma group a bit of a mystery, and more migraine coverage

Inside the Beltway – NAFTA replacement has drug price implications, more efforts to roll back donut hole changes, citizen petition changes coming, and pharma fears a Democratic House

Industry Trends – Nobel coverage (multiple articles), high cost of cancer drugs, march-in rights, patent games, comprehensive tumor profiling, evaluating progression-free survival as surrogate outcome measure, another PD-1 inhibitor approved, risks of alternative treatments, patients like being prescribed antibiotics (even if they don’t need them), two new antibiotics approved, new flu drug we may not need, and discussing the fish oil trial coverage

Now, on to the news.

ICER in the News

ICER released a Final Evidence Report and Report-at-a-Glance on three antiandrogen therapies for the treatment of nonmetastatic castration-resistant prostate cancer (nmCRPC): abiraterone acetate (Zytiga, Janssen Biotech, Inc; a separate formulation [Yonsa, Sun Pharma] was not evaluated), enzalutamide (Xtandi, Astellas Pharma, Inc.), and apalutamide (Erleada, Janssen Biotech, Inc.). ICER’s report was reviewed at a September 2018 public meeting of the Midwest Comparative Effectiveness Public Advisory Council. During the meeting, the Council found the evidence sufficient to show a net health benefit of treating nmCRPC with either apalutamide or enzalumatide, compared to androgen deprivation therapy (ADT) alone. However, the Council found the evidence insufficient to show a net health benefit for treating with abiraterone acetate.

Institute for Clinical and Economic Review Highlights the Clinical Benefits of Using Antiandrogen Therapies to Treat Earlier Stage Prostate Cancer

ICER released a Final Evidence Report and Report-at-a-Glance on inotersen (Akcea Therapeutics) and patisiran (Onpattro, Alnylam Pharmaceuticals) for the treatment of hereditary transthyretin amyloidosis (hATTR). ICER’s report was reviewed at a September 2018 public meeting of the Midwest Comparative Effectiveness Public Advisory Council. During the meeting, the Council found that both inotersen and patisiran provide a substantial net health benefit when compared to best supportive care alone, but the evidence is insufficient to distinguish between the two treatments. However, current pricing far exceeds commonly cited thresholds for cost-effectiveness.

Institute for Clinical and Economic Review Final Report Highlights Uncertainty in Long-Term Safety and Effectiveness of New Treatments for Hereditary Transthyretin Amyloidosis, Discusses Options for Insurance Coverage Criteria

Miranda Motter, President, and CEO of the Ohio Association of Health Plans, writes an op-ed in The Columbus Dispatch. She discusses ICER’s work and the need for drug prices based on value.

Column: Drug prices should be based on their value

Pharmaceutical companies produce a pipeline of essential medicines that can make people’s lives longer, happier and more comfortable. Their innovations are respected and appreciated by all of us.Unfortunately, some of the big drug companies price their drugs at unreasonable and unaffordable levels that disproportionately impact those of low-to-moderate incomes and with chronic health challenges.Prescription drugs represent 23 cents of every health-care dollar.

I’ll let this article from STAT speak for itself: “It’s less than a week old, but a shadowy new pharma advocacy group is already launching diatribes against advocates for lower drug prices, blasting pharmacy middlemen and defending even sky-high list prices for prescription drugs.” After the article below was published, more information on the set-up of the new group came to light: A Republican lobbying firm is behind Washington’s newest pro-pharma advocacy group.

This new advocacy group is an unapologetic defender of high drug prices – STAT

W ASHINGTON – It’s less than a week old, but a shadowy new pharma advocacy group is already launching diatribes against advocates for lower drug prices, blasting pharmacy middlemen and defending even sky-high list prices for prescription drugs.

More coverage of Lilly’s migraine drug approval includes discussion of ICER’s work on the topic (Xconomy item below). While not mentioning ICER, a Bloomberg piece looks at some of the unique pricing strategies Lilly and its competitors in the new migraine space will be using (Eli Lilly Migraine Drug Approved and Will Be Free to Patients).

Xconomy: Eli Lilly Migraine Drug Approved, the Third in New Class of Medicines

Xconomy Indiana – [ Updated, 9/28/18, 12:54 p.m. See below.] An Eli Lilly drug developed to prevent migraine headache pain before it starts has won FDA approval. The FDA decision announced late Thursday marks the third drug in this new class of medicines to receive the regulatory nod this year.

Careers at ICER

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:

Research Lead

The Research Lead, Evidence Synthesis will participate in multiple scientific activities, including developing strategy for and performing literature searches, critically evaluating the research methods and statistical findings of health outcome studies from the medical literature, and conducting qualitative and quantitative synthesis of evidence from literature and other sources.

Pharmaceutical News

Inside the Beltway/State Regulatory Developments

Pharma may have lost out concerning donut hole changes in the funding bill, but they made up for it with the new trade agreement. As part of the revisions to NAFTA, the minimum period of patent protection for biologics is set at ten years. This will necessitate a change in Mexican (currently five years) and Canadian (currently eight years) law and prevent much change in current US law (currently 12 years). (Report from Donna Young, S&P Global)

Trump tosses drug price pledges aside in NAFTA replacement

Politico looks at industry’s continuing efforts to reverse donut hole changes.

Why Congress is poised to give the drug industry a $4B windfall

President Donald Trump may rail against drug companies “getting away with murder,” but Congress appears to be moving in the opposite direction – helping to boost industry profits.

The FDA issued revised draft guidance to allow them to reject citizen petitions being used to block generic drugs (RAPS).

Citizen Petitions: FDA Revises Guidance to Block Drugmakers From Abusing the System

The US Food and Drug Administration (FDA) on Tuesday issued a revised draft guidance designed to allow for FDA to reject 505(q) petitions if the agency determines the primary purpose of the petition is to delay the approval of an abbreviated new drug application (ANDA).

It remains unclear how the elections in November will change Congress, but The Hill reports that pharmaceutical firms are concerned about one possible outcome: that if the Democrats regain the majority in the House, it will cause them problems.

Drug companies fear Democratic Congress

Drug companies are gearing up for a fight if Democrats take over the House. Democratic lawmakers say Republicans have gone too easy on the industry and are vowing that will change if they take power in November’s midterm elections.

Industry Trends

As noted in this week’s introduction above, Nobel Prizes were awarded this week. The winners in both medicine and chemistry contributed to pharmaceutical innovation.

The winners for Physiology or Medicine did groundbreaking work in immunotherapies (Reuters).

Scientists behind game-changing cancer immunotherapies win Nobel…

STOCKHOLM/LONDON (Reuters) – American James Allison and Japanese Tasuku Honjo won the 2018 Nobel Prize for Physiology or Medicine on Monday for game-changing discoveries about how to harness and manipulate the immune system to fight cancer.

Of course, like everything else in health care, who discovers something is complicated. Some felt there were crucial contributors to immunotherapy overlooked by the prize committee (STAT).

Crucial contributors to cancer immunotherapy were excluded from the Nobel – STAT

T he Nobel committee’s account of discoveries critical to harnessing the immune system to treat cancer lists just under 100 papers authored by hundreds of scientists.

The winners in chemistry used sped-up evolution to create new proteins, leading to new drugs (Washington Post).

Chemistry Nobel for using evolution to create new proteins

STOCKHOLM – Three scientists won the Nobel Prize in chemistry Wednesday for using a sped-up version of evolution to create new proteins that have led to a best-selling drug and other products. The Royal Swedish Academy of Science said their work has led to the development of medications, biofuels and a reduced environmental impact from some industrial processes.

Vox uses the awarding of a Nobel Prize for immunotherapy work (coverage above) to discuss the high cost of cancer drugs.

The Nobel Prize is a reminder of the outrageous cost of curing cancer

For the first time ever, we’re living in a moment when many of our most promising medical advances are far out of reach for the vast majority of people who could benefit from them. And nowhere is that truer than for cancer immunotherapy, the fast-moving field of cancer treatment research that was honored on Monday with the Nobel Prize in physiology or medicine.

Kaiser Health News with two in-depth pieces this week on drug pricing issues.

First, Shefali Luthra looks at march-in rights and the strange alliances forming in support of the action.

In The Battle To Control Drug Costs, Old Patent Laws Get New Life

In the drug pricing battle, progressive lawmakers such as Sen. Bernie Sanders (I-Vt.) and patients’ rights activists rarely find themselves in step with the health industry’s big players. But in a twist, these usually at-odds actors are championing similar tactics to tame prescription drug prices.


Second, Sarah Jane Tribble looks at the patent games manufacturers play.

Drugmakers Play The Patent Game To Lock In Prices, Block Competitors

This story also ran on This story can be republished for free ( NPR. details). David Herzberg was alarmed when he heard that Richard Sackler, former chairman of opioid giant Purdue Pharma, was listed as an inventor on a new patent for an opioid addiction treatment. Patent No.

Comprehensive tumor profiling — should it be the new standard (now that Medicare is covering the cost)? The Washington Post takes a look. And before you say of course it should be, make sure you read this part of the article: “Only one randomized clinical trial has investigated whether treatment decisions influenced by genetic analyses were better for patients than an oncologist’s best-educated guess. The study found no difference in progression-free survival — the length of time that a patient’s disease does not worsen — between the two strategies. “

Conquering cancer by attacking the disease’s genetic abnormalities

When Teresa McKeown was diagnosed with breast cancer in 2006, her disease was easily treated with standard therapies. But 11 years later, the cancer returned. This time, it morphed into what’s called triple-negative disease, an aggressive and difficult-to-treat form. “I had one therapy after another,” she said, “and failed them all.”

Of course, there is also the question, is progression-free survival the right measure? A new systematic review published this week in JAMA found no correlation between progression-free survival and health-related quality of life.

Evaluating Progression-Free Survival as a Surrogate Outcome for Health-Related Quality of Life in Oncology

What is the cost of a clinical trial? Endpoints News reports on a new JAMA paper that tries to answer that question.

Are drug R&D costs exaggerated for effect? Researchers peg the median price of pivotal success at an economical $19M

One of the most frequently cited stats in the biotech biz revolves around the claim that it can take more than a decade and $1 billion-plus to get a drug to market. For lobbyists, it’s a chance to underscore the high cost of R&D that goes into a new treatment

Biopharma Dive reports on the FDA approval of Sanofi and Regeneron’s immunotherapy entrant (a PD-1 inhibitor).

Regeneron wins early approval for cancer immunotherapy

Regeneron Pharmaceuticals on Friday secured an early approval from the Food and Drug Administration for a cancer immunotherapy that works in the same fashion as Merck & Co’s Keytruda, adding yet another contender to the fast-growing immuno-oncology field. Regeneron’s drug, called Libtayo, is a latecomer, arriving years behind Keytruda and Bristol-Myers Squibb’s Opdivo, both of which target the same PD-1 protein.

As we’ve noted here in the past, modern medicine is not perfect. However, in most cases, it is the best option available. The New York Times reports on a recent study looked at those who tried alternative treatments and experienced vastly higher mortality rates.

The Risk of Alternative Cancer Treatments

Personal Health Avoiding evidence-based treatments in favor of untested ones can contribute to higher death rates, a Yale study found. A diagnosis of cancer, even an early-stage, highly curable cancer, can prompt some people to feel as if they’ve suddenly lost control of their future and that they must do whatever they can to regain it.

People want drugs — it makes them feel taken care of — at least that’s one explanation for why physician ratings are higher when an antibiotic is prescribed. NPR looks at what to me is a dangerous correlation (since we know we have an antibiotic resistance problem, less is more when it comes to using them).

Patients Give Doctors High Marks For Prescribing Antibiotics For Common Sniffles

When they’re sick, Americans seem to know what they want: antibiotics. And if they don’t get them, their doctors’ reputations may suffer. A study published Monday finds that patients rated themselves happiest with their doctor’s visit when they got an antibiotic after seeking care for a respiratory tract infection, such as a common cold, whether they needed the drug or not.

And speaking of antibiotic resistance, two new antibiotics were approved this week. Since many existing drugs are losing their effectiveness, new entrants are desperately needed (FiercePharma).

2 Paratek antibiotics win FDA nods, with 1 slated for $500M in peak sales

Paratek Pharmaceuticals felt double the love from the FDA this week, as two of its antibiotics won back-to-back approvals-one for Paratek itself, and the other for deal partner Allergan’s dermatology buyer Almirall. Tuesday, the FDA greenlighted Nuzyra, or omadacycline, for community-acquired bacterial pneumonia (CABP) and acute skin and skin structure infections (ABSSSI).

Sometimes new drugs may not have a niche to fill. EndPoints looks at one new entrant we may not need, a new flu drug that was no better than the generically available Tamiflu (in a Phase 3 study).

Roche just heralded a great set of placebo comparisons for their new flu drug – too bad it flopped against generic Tamiflu

Genentech just rolled out some of the hard data around its Phase III CAPSTONE-2 trial for their new flu medicine baloxavir marboxil, and it doesn’t look good. Once you dig through the bonny remarks and keep going on to the statistically significant placebo comparisons, the pharma giant throws in a

Including this story for two reasons. First and most importantly, it asks important questions about the topline results released last week for the Vascepa trial. The second reason should be obvious – how could I resist that headline?

Without full data, Vascepa trial results still a little bit fishy – Lown Institute

You may have heard the conventional wisdom that fish oil is healthy for the heart (or that it gives your hair more shine, as teen magazines used to tout). But does taking omega-3 fatty acid supplements help prevent cardiovascular events in patients at high risk of heart attack or stroke? 
@mhstein  |

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