From the desk of Mitchell Stein
Good morning. Our thoughts are with those experiencing the ravages of Florence (and Typhoon Mangkhut in the Philippines). Earlier in the week to help prepare for the storm, HHS declared a public health emergency in North Carolina, South Carolina, and Virginia. While the event is not expected to have the impact on pharmaceutical manufacturing that Maria had, Merck, GlaxoSmithKline, Pfizer, and others have announced plant closings in preparation for the storm. Additionally, most PBMS are making allowances regarding refill policies to ensure that patients can access their prescriptions. I hope everyone stays safe. Now, on to this week’s events where we’ll look at:
ICER in the news – ICER released draft evidence report on MAT, Midwest CEPAC meeting, indication-based formularies for Part D, and supplemental FDA application for Praluent
Inside the Beltway – Azar talks to Bloomberg, Congress passes on DTC price requirement, 340B suit by hospitals, accelerated approvals, 340B comments controversy, and a closer look at MA Medicaid formulary denial
Industry Trends – PBM pricing, two surveys cover drug pricing, high drug price defense, precision medicine isn’t so precise, two articles on CRISP concerns, CRISP patent battle developemtns, lack of COI dosclosure leads to resignation, innovative vaccine delivery, antibiotic dilema, and Alzheimers infection theory
On to the news.
ICER In The News
ICER released a Draft Evidence Report assessing the comparative clinical effectiveness and value of medications for addiction treatment (MAT) used for individuals with opioid use disorder. The draft report will be open to public comment until October 4, 2018. ICER’s report assesses the effectiveness and value of a buprenorphine implant (Probuphin, Titan), an extended-release naltrexone injection (Vivitrol, Alkermes), and two extended-release buprenorphine injections: Sublocade (Indivior) and CAM2038 (Braeburn), an investigational agent currently under FDA review. The report will be subject to deliberation during a public meeting of the New England Comparative Effectiveness Public Advisory Council on November 8, 2018. The release was covered in ThePharmaLetter.
Institute for Clinical and Economic Review Releases Draft Evidence Report on Medications for Treatment of Opioid Use Disorder
The Institute for Clinical and Economic Review’s Midwest Comparative Effectiveness Public Advisory Council (CEPAC) voted unanimously in separate tallies that the long-term value for money was low for hATTR treatments from Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) and Akcea Therapeutics Inc. (NASDAQ:AKCA).
A review of the indication-based formulary design for Medicare Part D includes mention of ICER’s potential role in informing the formularies.
The FDA accepted a supplemental application for Praluent. The application was based on results from the ODYSSEY OUTCOMES trial. In their account, AJMC mentions ICER’s assessment of the drug.
Sanofi and Regeneron announced today that FDA has accepted a supplemental Biologics License Application for alirocumab, the powerful anticholesterol therapy sold as Praluent. The application was anticipated after the presentation in March of results from the ODYSSEY OUTCOMES trial of the proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor, which found a 15% overall reduction in major adverse cardiovascular (CV) events, including heart attack, stroke, CV death, and unstable angina that requires hospitalization.
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: Chief Scientific Officer The role of the CSO includes oversight of a skilled research team at ICER and the overall production of ICER reports, management of ICER’s engagement with outside academic researchers, and leadership in the evolution of ICER’s methods of value assessment and report development. The CSO will be a key member of ICER’s Senior Management Team and a central figure in ICER’s strategic planning and all other high-level activities.
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: Chief Scientific Officer
The role of the CSO includes oversight of a skilled research team at ICER and the overall production of ICER reports, management of ICER’s engagement with outside academic researchers, and leadership in the evolution of ICER’s methods of value assessment and report development. The CSO will be a key member of ICER’s Senior Management Team and a central figure in ICER’s strategic planning and all other high-level activities.
Inside the Beltway/State Regulatory Developments
HHS Secretary Azar was interviewed by Bloomberg this week. While the headline is startling, Azar revealed no specifics about the Administration’s plans. We know a rule impacting the safe harbor exemption has been submitted to the White House, but that’s about all we know.
Yesterday, in preparation for Congress passing the HHS spending bill, the House/Senate conference committee met to reconcile the versions from the two bodies – of note was what was not in the final bill. Despite the support of the President, and bipartisan support in the Senate, House Republicans objected to the provision that would require prices in DTC drug advertising keeping it out of the final bill.
The provision, which passed the Senate on a bipartisan basis last month, would require drug prices to be disclosed in television advertisements for drugs in an effort to increase transparency and bring down prices. Lawmakers and aides said that House Republicans objected to including the provision in the final version of the funding bill, which was finished by House and Senate negotiators on Thursday.
This week in how the 340B program turns… hospitals sue to require transparency (Healthcare Dive).
Four industry associations sued HHS for the second time over its delay in adding regulations to the 340B Drug Pricing Program that would require pricing transparency for drug manufacturers and penalties for overcharging hospitals covered under the program.
Everything you always wanted to know about accelerated approval and surrogate endpoints but were afraid to ask (Undark).
O n June 28, 2011, ten police cars descended upon the headquarters of the Food and Drug Administration. Scores of protesters carrying loudspeakers and donning pink shirts had attempted to enter the building, but were thwarted by the officers. One man held a sign that read: “My wife is not a statistic: Save Avastin.”
Kaiser reports on a cloud over the comments submitted regarding the 340B program.
A proposal to sharply cut a drug discount program that many hospitals rely on drew some 1,400 comments when the Trump administration announced its plan last year. Hundreds appeared to come from patients across the country – pleas from average Americans whose treatments for diseases such as cancer depend on costly medicines.
In June, CMS denied MA request for a Medicaid waiver to create a restricted formulary. Law Professors Rachel E. Sachs (also a member of Midwest CEPAC) and Nicholas Bagley observe in the New England Journal of Medicine (subscription required) that the response did not offer any reasons for the denial.
Bloomberg takes a deep dive into PBM pricing – be prepared for innovative graphics and stunning numbers.
More people than ever think drug costs are a significant contributor to health care cost issues, but few believe the Administration’s efforts will bear fruit. From this month’s Kaiser tracking poll:
“Over the past few months President Trump has maintained a tense relationship with drug companies over the price of prescription drugs. This month’s tracking poll finds an increase in the share of the public who say drug companies making too much profit is a ‘major reason’ why people’s health care costs have been rising (78 percent compared to 62 percent in 2014). However, less than half of the public – four in ten (42 percent) – say they think President Trump’s strategy of publicly shaming drug companies and asking them to cut prices will be effective in reducing prescription drug costs overall and a similar share (38 percent) are confident that the president will be able to deliver on his promise that Americans will pay less for prescription drugs under his administration.”
Kaiser Health Tracking Poll – Late Summer 2018: The Election, Pre-Existing Conditions, and Surprises on Medical Bills
The latest Kaiser Health Tracking Poll finds recent political events weighing heavy on the minds of voters when it comes to the 2018 midterm elections. Three in ten voters (33 percent of independent voters, 32 percent of Democratic voters, and 25 percent of Republican voters) say corruption in Washington is the “most important” topic for 2018 candidates to discuss.
West Health also released a survey this week (administered by NORC), this one focused exclusively on prescription drug issues.
This article by David Crow of the Financial Times (who sadly is about to leave the pharma beat) has garnered much attention, including from FDA Commissioner Scott Gottlieb. A pharma executive said that it’s his responsibility to his shareholders to charge as much as he can for his product. For voicing that opinion, he received more attention than he might have bargained for.
A pharma executive has defended his decision to raise the price of an antibiotic mixture to more than $2,000 a bottle, arguing there was a “moral requirement to sell the product at the highest price”. Last month, Nostrum Laboratories, a small Missouri-based drugmaker, more than quadrupled the price of a bottle of nitrofurantoin from $474.75 to $2,392, according to Elsevier’s Gold Standard drug database.
Writing in the NY Times, Liz Szabo (of Kaiser Health News) reminds us the precision medicine isn’t so precise.
Doctors and hospitals love to talk about the cancer patients they’ve saved, and reporters love to write about them. But deaths still vastly outnumber the rare successes. By Liz Szabo Ms. Szabo is a health reporter for Kaiser Health News.
STAT provides two cautionary articles about CRISPR this week.
The first (STAT Plus, subscription required) reviews the questions about CRISPR and cancer.
W hen papers from two independent research groups reported in June that CRISPR genome editing is more likely to succeed in cells that have lost their cancer kill switch, it raised fears that edited cells used to treat patients might initiate tumors.
The second article looks into the similarities between hedgehogs and DNA. No really, hedgehogs roll into a ball as a defense mechanism. DNA also rolls into a ball, which also ends up being a defense mechanism – against CRISPR – preventing the editor from being effective.
owbugs, armadillos, hedgehogs … and DNA? The same strategy that some animals use to avoid being attacked – roll into a ball and keep your vulnerable bits beyond predators’ reach – turns out to let genes avoid being sliced up by the genome-editing molecules of CRISPR, scientists reported on Monday.
With this victory for the Broad Institute, Donna Young of S&P Global reports the CRISPR patent battle may finally be over.
On Saturday (September 8) ProPublica and the NY Times reported on the failure of a top researcher to disclose conflicts of interest. Then, on Thursday (September 13) the researcher, Dr. José Baselg resigned from his position as Chief Medical Officer of Memorial Sloan Kettering Cancer Center.
This article was reported and written in a collaboration with ProPublica, the nonprofit investigative journalism organization. One of the world’s top breast cancer doctors failed to disclose millions of dollars in payments from drug and health care companies in recent years, omitting his financial ties from dozens of research articles in prestigious publications like The New England Journal of Medicine and The Lancet.
During a pandemic, the last thing you want to do is gather potentially infected people in large groups with potentially uninfected people, even if it’s to vaccinate them. A new prototype holds the potential of avoiding that scenario – it provides the vaccine (and a booster) through a self-administered patch. Carolyn Johnson of the Washington Post reports on this extremely significant development (extremely significant to those of us who regularly lose sleep thinking about the next pandemic).
September 12 at 2:02 PM When the next deadly pandemic flu hits, the first challenge will be to develop a vaccine. But looming behind that obstacle is another: How to get an inoculation to millions of people without inadvertently exacerbating the crisis.
As John Carroll of Endpoints News points out, this story is a cautionary tale of how hard it is to make money off of new antibiotics. It’s clear to me if changes aren’t made, companies will have no reason to develop the new antibiotics we’re going to need.
Merck heralds a rare Big Pharma success for antibiotic PhIII, but it’s also a cautionary tale about the market
When Merck $MRK bought out Cubist close to 5 years ago for $9.5 billion, the sell-side crowd stoked considerable enthusiasm for Zerbaxa, a late-stage antibiotic that was tapped for peak sales in the lofty blockbuster range of $1.2 billion to $1.5 billion or so. Today, Merck boasted that their antibiotic
I’m skeptical, but given our failure so far with Alzheimer’s, it makes sense to explore all possibilities (NPR).
Dr. Leslie Norins is willing to hand over $1 million of his own money to anyone who can clarify something: Is Alzheimer’s disease, the most common form of dementia worldwide, caused by a germ? By “germ” he means microbes like bacteria, viruses, fungi and parasites.