From the desk of Mitchell Stein
Good morning and happy Saint Patrick’s Day! ☘️ So much news, so little time. Today we’ll look at:
ICER in the News – ICER more transparent than NICE, update to ICER’s PCSK9 work, ICER recognizes the National MS Society with a leadership award, RA evidence report released, and new members named to ICER councils.
Inside the Beltway – Medicaid waivers and Sanders talks TV coverage (plus two big stories in In-depth)
Industry Trends – Cost of anticancer drug (2 stories), more on NICE, chemo not going away, and the growth of tiered networks
Company/Drug Specific – Additional Keytruda approval, Opana, Kisqali, Mylan, and Marathon.
Weekly View In-depth – Gottlieb at FDA and a budget proposal from the administration.
Now, on to the news.
ICER In The News
PharmExec takes a look at the budget impact approach used by England’s NICE and NHSE. In comparison, it has this to say about ICER’s approach:
“It seems a missed opportunity to have not considered a more principled and transparent approach—for example the Institute for Clinical Effectiveness Review (ICER) in the US has a budget impact threshold informed by growth in the economy and other adjustments—that could be applied instead of a retrospective and opaque calculation that NICE and NHSE have used. Even aside from trying to come up with a better way to calculate the threshold, would be a process for updating as NHS funding changes over time.”
Issues of healthcare affordability are universal. ICER’s work continues to be at the forefront of those organizations trying to grapple with these issues in a transparent and unbiased way. (There is an additional story about NICE and drug costs in the Industry Trends section.)
With important new clinical evidence now available, ICER announced this morning (3/17) that they had begun a “New Evidence Update” to their 2015 review of the comparative clinical effectiveness and value of PCSK9 inhibitors alirocumab (Praluent®, Sanofi/Regeneron) and evolocumab (Repatha®, Amgen).
Institute for Clinical and Economic Review to Produce “New Evidence Update” Including Updated Value- based Price Benchmarks for PCSK9 Inhibitors to Treat High Cholesterol
ICER has named the National Multiple Sclerosis Society as this year’s recipient of the ICER Comparative Effectiveness Research National Leadership Award. This award recognizes individuals and organizations that are innovative leaders in advancing the methods and practical applications of comparative effectiveness research in the United States.
ICER Recognizes National MS Society with Comparative Effectiveness Research National Leadership Award
ICER released an Evidence Report assessing the comparative clinical effectiveness and value of targeted immunomodulatory drugs for the treatment of rheumatoid arthritis (RA). This Evidence Report will be the subject of the upcoming public meeting of the New England Comparative Effectiveness Public Advisory Council on March 24, 2017. Read ICER’s full release here, or the account in thepharmaletter below (free registration required).
ICER announced they had appointed new members to each of its three independent evidence appraisal councils: the California Technology Assessment Forum, the Midwest Comparative Effectiveness Public Advisory Council, and the New England Comparative Effectiveness Public Advisory Council.
The Institute for Clinical and Economic Review Appoints New Members to Each of its Three Independent Evidence Appraisal Councils
Inside the Beltway
Two of the week’s biggest stories are the nomination of Scott Gottlieb to head the FDA and the release of President Trump’s budget. See the “In-depth” section below for coverage of those stories. Meanwhile…
The loosening of standards for Medicaid waivers is worth watching – will a freer hand mean changes in Medicaid prescription drug coverage?
The Trump administration told states on Monday that it was opening the door for them to make changes to their health insurance programs by using a section of the 2010 Affordable Care Act that gives them the flexibility to do so.
Senator Sanders asks why drug pricing issues aren’t being covered on TV.
As the U.S. drug pricing debate has ratcheted up in recent months, pharma has had a relative safe haven on television screens. Now, Sen. Bernie Sanders is asking six TV network execs about their “lack of coverage” on an issue so many Americans care about-particularly at a time when drugmakers have hiked their spending on consumer advertising.
A new paper titled “The high price of anticancer drugs: origins, implications, barriers, solutions” was published in Nature Reviews: Clinical Oncology this week. The paper is behind a paywall, but here is an account of the article:
If left unchecked, the rising cost of cancer drugs could have devastating implications for individuals, societies and national economies, a group of cancer physicians and researchers said. In a new paper published Tuesday in Nature Reviews: Clinical Oncology, the cancer experts excoriated the pharmaceutical industry for pricing oncology drugs at rates that make them inaccessible and are unjustifiably high given the often scant benefit some of these drugs bring patients.
Using the article and additional data, NPR took a look at the issue of financial toxicity in cancer care.
John Krahne received alarming news from his doctor last December. His brain tumors were stable, but his lung tumors had grown noticeably larger. The doctor recommended a drug called Alecensa, which sells for more than $159,000 a year. Medicare would charge Krahne a $3,200 copay in December, then another $3,200 in January, as a new year of coverage kicked in.
England asks tough questions about drug prices – for them, sometimes the answer is no.
Nation mulls raising bar on treatments for very rare diseases Medicines from Sanofi, Novartis feel pain of new resolve For patients with a very rare, inherited disease that causes excessive bleeding, fragile bones and bloated livers, French drugmaker has a pill available in countries from Germany and Greece to the U.S.
Chemo is not going away any time soon.
Chemotherapy remains one of the mainstays of cancer treatment, but these harsh drugs are slowly being edged aside in medical research, as new treatments, like immunotherapy, grab the spotlight. Still, this is not the end of the road for chemotherapy.
Use of tiered pharmacy networks poised for growth.
One of California’s largest insurers has proposed a change in the benefits of commercial plans next year that would require consumers to pay more for drugs at pharmacies outside an established network.
Keytruda approved for the first time for use in blood cancer.
Merck has racked up another FDA approval for Keytruda, this time for classical Hodgkin lymphoma, marking the PD-1 blocker’s first green-light for use in liquid tumors. Regulators handed out the OK for patients who had already failed at least three therapies. The agency based this approval on the data Merck
“A joint FDA advisory committee voted 18-to-8, with one abstention, that the benefits of Opana ER (oxymorphone hydrochloride) do not outweigh its risks to public health, following outbreaks of HIV, hepatitis C, and vascular issues related to intravenous abuse of the drug.”
A joint FDA advisory committee voted 18-to-8, with one abstention, that the benefits of Opana ER (oxymorphone hydrochloride) do not outweigh its risks to public health, following outbreaks of HIV, hepatitis C, and vascular issues related to intravenous abuse of the drug.
Kisqali approved for first-line treatment of breast cancer when used with another drug.
Novartis picked up a key regulatory win today, gaining the FDA’s quick OK for LEE011, its CDK 4/6 drug now approved as a frontline therapy for postmenopausal women with hormone-receptor positive, human epidermal growth factor receptor-2 negative advanced or metastatic breast cancer in combination with any aromatase inhibitor. The drug,
Another step forward in the launch of the Mylan’s biosimilar of Roche’s cancer drug Herceptin.
Mylan has removed a legal hurdle blocking the potential U.S. launch of its biosimilar to Roche’s blockbuster cancer drug Herceptin (trastuzumab), announcing Monday it had reached a settlement agreement with the Swiss pharma. Under the agreement, Roche granted Mylan a license for marketing its trastuzumab biosimilar in all countries except Japan, Brazil and Mexico.
Marathon decides it just wasn’t worth it and sells its DMD drug.
Marathon Pharmaceuticals’ controversial $89,000-a-year drug that has drawn outrage from patients and intense questioning from Congress is getting a new owner. After striking a deal Wednesday evening, PTC Therapeutics announced plans early Thursday to buy the Duchenne muscular dystrophy drug Emflaza from Marathon for $140 million in cash and stock.
Weekly View In-depth
Everything you wanted to know about Scott Gottlieb but were afraid to ask (a sample of this week’s coverage):
- Forbes Posts Show How Scott Gottlieb Might Run The FDA – an annotated list of some of his more significant Forbes posts
- By The Numbers: Trump’s Choice For FDA Chief Is Versatile, Entrenched In Pharma – from Kaiser Health News lots of numbers (investments, Boards, clients, etc.)
- Scott Gottlieb, Trump’s FDA pick, explained – from Vox a detailed look at his career and possible approach to leading the FDA
- Biopharma execs give Scott Gottlieb’s nomination for FDA commish (almost) a standing O in Endpoints poll – the sighs of relief from industry executives cause a gale warning to be issued
The Administration released their budget proposal. It contains extreme cuts to discretionary spending. Here are some of the observations we’ve seen:
- There is a disconnect between what the President has said regarding medical research and the realities of his proposed budget. His plan calls for a $5.8 billion cut for the National Institutes of Health, the world’s biggest funder of biomedical research — nearly a fifth of its $31.7 billion discretionary budget.
- The budget is calling for a hike in user fees from industry to make up for cuts elsewhere at the US Food and Drug Administration.
- Overall, biomedical research and public health are among the big losers in the Trump administration’s proposed budget.
- Here’s an overview of how it will hit pharma.
- This morning’s Axios Vitals newsletter has a selection of reactions from Congress – GOP to Trump on NIH cuts: Um, we’ll get back to you (item about halfway down the page). The bottom line is don’t expect this budget to have smooth sailing, even among the President’s allies.