From the desk of Mitchell Stein
Good morning. Did you miss me? These days, two weeks in the news cycle feels like an eternity. While I’ve focused on the more recent items, today’s issue includes items from the two weeks since we last met. As always, lots going on in the drug world. We’ll talk prices, biosimilars, generics, lions, tigers, and bears (oh my). Here’s the full line-up:
ICER in the News – Two Evidence Reports released: 1) hereditary transthyretin amyloidosis and antiandrogen therapies for the treatment of nonmetastatic castration-resistant prostate cancer, John Arnold on PBMs, Interview with CVS VP, and approval of lanadelumab
Inside the Beltway – Biosimilar competition hearing, BioPharma Dive looks at the changing FDA, Frakt on 340B, Azar goes to the Hill, Bach named chair of MEDCAC Advisory Committee, movement between FDA and industry, Oklahoma gets go-ahead on Medicaid value-based pricing, and lawsuit against CA drug price law hits a snag
Industry Trends – Hospital sponsored generic firm launches, blocking generics, study on the percent of medical costs going to drugs, August drug price changes, lower drug costs, new PhRMA report on prices, Cutler on user fees, NICE on CAR-T therapies, patient group & pharma collide, China’s drug market, predatory bacteria filling an antibiotic gap, and microbes to break down toxins
Now, on to the news.
ICER In The News
ICER released an Evidence Report assessing the comparative clinical effectiveness and value of therapies to treat hereditary transthyretin amyloidosis. The report includes inotersen (Akcea Therapeutics) and patisiran (Onpattro, Alnylam Pharmaceuticals). Patisiran was recently approved by the FDA. An approval decision on inotersen is expected in October of 2018. The report also evaluates evidence on inotersen. During a public meeting of Midwest CEPAC on September 13th, a policy roundtable will discuss possible contextual factors that may affect decision-making for ultra-rare conditions such as amyloidosis.
This week Onpattro was approved for sale in the EU. Two stories on the approval discuss ICER’s report: Alnylam’s Onpattro cleared in Europe as ICER sets pricing battle (BioPharmaDive) and Alnylam gets EU nod for Onpattro amid pricing backlash (PMLive).
Institute for Clinical and Economic Review Finds Current List Price of Patisiran for Amyloidosis Far Exceeds Standard Cost-Effectiveness Levels
ICER released an Evidence Report assessing the comparative clinical effectiveness and value of antiandrogen therapies for the treatment of nonmetastatic castration-resistant prostate cancer. The report focuses on three antiandrogen therapies: 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.). Added costs of treating earlier stage disease with all therapies are aligned with their added clinical benefits for patients. This report will also be discussed at the September 13th public meeting of Midwest CEPAC.
Institute for Clinical and Economic Review Report Finds Evidence Inadequate to Distinguish the Clinical Benefits of Different Antiandrogen Therapies for Men with Nonmetastatic Castration-Resistant Prostate Cancer
John Arnold co-founder, with his wife, Laura, of the Laura and John Arnold Foundation (LJAF), writes an opinion piece in STAT about PBMs. The piece is worth a read as he reviews that while some PBM behavior has been problematic, the model still holds promise. (Disclosure – ICER receives some funding from LJAF.)
n the ongoing debate over drug prices, the pharmaceutical industry has been highly effective in shifting the blame to the middlemen – in particular to pharmacy benefit managers. As they currently operate, pharmacy benefit managers are part of the problem.
Ed Silverman (Pharmalot) of STAT interviewed Dr. Troyen Brennan, an executive vice president and chief medical officer at CVS Health about his company’s new use of ICER analyses. The interview (subscription required) included this exchange:
“Pharmalot: Why rely on analyses run by ICER (the Institute for Clinical and Economic Review, a non-profit that gauges cost effectiveness for medicines)?
Brennan: We like ICER because it’s very transparent. They are very clear about the studies they rely on to generate data and their methodology. And they allow public comment. So it’s a public process. And I think the results they come up with are usually quite similar to others that are doing comparative effectiveness. They undertake this activity voluntarily, they’re supported largely by philanthropy, and when combined with market pressure, their analysis is every bit as powerful as government regulation might be.”
CVS exec: Our new reliance on cost effectiveness should make drug makers ‘think about launch prices’ – STAT
In a bid to dampen rising drug costs, CVS Caremark plans to allow its clients – such as health plans and employers – to exclude from their formularies any new medicine that has a higher price than a particular benchmark for determining value.
Article on the approval of lanadelumab discusses ICER’s work on the drug.
Shire’s much-anticipated prophylaxis treatment for a rare disease that causes severe and sometimes life-threatening swelling has received approval from the Food and Drug Administration. Lanadelumab, now branded as Takhzyro, inhibits an enzyme thought to be one of the root causes of the disease, known as hereditary angioedema or HAE.
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: 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.
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:
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.
Inside the Beltway/State Regulatory Developments
The FDA held a hearing on biosimilar competition. S&P Global’s Donna Young reports on what lead to the hearing and what was said.
With a series of five articles, “BioPharma Dive takes an in-depth look at how the regulatory landscape at the FDA has changed in recent years — and where it might be headed in the future.”
BioPharma Dive takes an in-depth look at how the FDA has changed in recent years – and where it might be headed.
New England CEPAC member Austin Frakt, writing in the NY Times, looks at the past, present and future the 340B drug discount program.
The New Health Care A program meant to help the poor has grown beyond its original intent. Most hospitals are nonprofit and justify their exemption from taxation with community service and charity care.
Given the attention paid to a certain SCOTUS nominee hearing this week, you might think nothing else was happening on Capitol Hill. You’d be wrong as Azar met with Republican lawmakers regarding the Administration’s drug price initiatives.
Secretary of Health and Human Services Alex Azar on Thursday met with Republican lawmakers on the House Ways and Means Committee to discuss ways to lower drug prices.
Dr. Peter Bach was named chair of the Medicare Evidence Development & Coverage Advisory Committee (BioCentury).
CMS has named Peter Bach, an advocate for new pricing mechanisms to combat high drug costs, as chair of its Medicare Evidence Development & Coverage Advisory Committee.
More movement between the FDA and industry.
It’s well known that leaving the US Food and Drug Administration (FDA) for industry can bring a major salary bump, so it should come as no surprise that the number of FDA employees making the leap in 2018 continues to increase.
Forbes reports on Oklahoma being the first state Medicaid program to receive a CMS waiver allowing it to pursue value-based pricing in addition to existing Medicaid rebates.
States have often been an incubator for innovative ideas in health technology pricing and reimbursement. the Medicaid drug pricing initiatives put forward by Oklahoma and Massachusetts may give impetus to an uptick in value-based pricing agreements throughout the healthcare system.
The California drug price law requiring advance notice of price increases moved a step closer to implementation as a judge threw out PhRMA’s suit against the law. However, the judge gave them 30 days to refile (ABC News).
A federal judge has dismissed a lawsuit seeking to block a California law requiring pharmaceutical companies to give advance notice before big price increases. U.S. District Judge Morrison England Jr., ruled Thursday in Sacramento that the Pharmaceutical Research and Manufacturers of…
The Washington Post reports as Civica Rx launches. It’s the new not-for-profit generic drug manufacturer announced in January by a coalition of hospitals and foundations.
A group of major American hospitals, battered by price spikes on old drugs and long-lasting shortages of critical medicines, has launched a mission-driven, not-for-profit generic drug company, Civica Rx, to take some control over the drug supply. Backed by seven large health systems and three philanthropic groups, the new venture will be led by an industry insider who refuses to draw a salary.
The above story is an attempt to deal with generic shortages through additional approvals and manufacturing. But getting approvals may not be so easy, according to a new study that highlights the measures some brand manufacturers take to slow down generic approvals (BioPharma Dive).
A study commissioned by the nation’s largest generics trade group found the U.S. healthcare system could be saving billions of dollars if branded drug manufacturers weren’t abusing federal programs meant to make higher-risk pharmaceuticals safer. The main program cited throughout the study is known as REMS for short.
Remember when people used to say that drugs made up 10% of medical costs? Hard to say that anymore. Harvard Pilgrim opened up their books to researchers who estimate that 20-25% of the health plan’s total health care spending is devoted to prescription drugs (BioPharma Dive).
In the six years between January 2011 and December 2016, the share of total healthcare spending by regional insurer Harvard Pilgrim Health Care devoted to prescription drugs increased to 25% from 20%, according to an analysis published in Health Affairs last week.
A new report from Wells Fargo on August drug price changes (Axios).
A data source reports a lowering of net drug prices. Reuters reports that some feel copay accumulator programs are responsible for the decline.
NEW YORK (Reuters) – U.S. drug prices fell again in the second quarter, likely due to a new tactic insurers are using to limit financial assistance drugmakers provide directly to consumers, according to research firm Sector and Sovereign Research (SSR). Real U.S.
A report from PhRMA talks about list prices – but I thought we weren’t supposed to pay attention to list prices. I find the whole situation very confusing (Axios).
One in six hospitals set prices that are at least seven times more than what the hospital paid.
Economist David Cutler, writing in JAMA Forum, argues for increased user fees for pharmaceutical companies to help fund government services that the industry disproportionately receives.
A central theme in economics is that people who benefit the most from a good or service should pay more for it. If a government decides to build a highway connecting 2 cities, charging a toll to users of the highway is preferred to financing the road through general taxation.
I include this story for multiple reasons – while it’s noteworthy in and of itself that Kymriah was approved by NICE, it’s a case study in what happens when negotiations take place. While Kymriah and Yescarta (both are CAR-T therapies) are approved in the US and the EU, NICE rejected Yescarta because they could not negotiate an acceptable price. A good reminder that when you negotiate, you have to be prepared to say no (FiercePharma).
Little more than a week after rejecting Gilead’s CAR-T cell therapy, Yescarta, for adults with relapsed large B-cell lymphoma, England’s cost-effectiveness watchdogs gave Novartis the go-ahead for its CAR-T drug, Kymriah, to treat children and young adults with one form of leukemia.
Kaiser Health News takes an in-depth look at a case where a patient and pharma parallel interests eventually collided.
Desperate for help in finding a lifesaving drug for a fatal genetic disease, families banded together to fund early research and then worked with drug companies on clinical trials and marketing. Yet, this small patient advocacy group is stunned by pharma’s pricing.
The Economist has a feature on how the Chinese drug market, comprised of 1.4 billion customers, is evolving.
CHINA is home to 1.4bn people. The population is ageing, and thus more vulnerable to ailments. Sustained economic growth is making the country richer, and more able to afford remedies. To foreign pharmaceutical firms, this looks like a winning combination.
We’ll end this week with a few stories for our “science or science fiction” file from NPR and the New York Times.
Here’s a bold idea to fight back against bacteria that can’t be stopped by antibiotics: Go after them with germ-eating microbes. That reasoning lies behind an intriguing line of research that might also be put to use in the event of a germ-warfare attack.
matter By manipulating DNA, researchers are trying to create microbes that, once ingested, work to treat a rare genetic condition – a milestone in synthetic biology. In a study carried out over the summer, a group of volunteers drank a white, peppermint-ish concoction laced with billions of bacteria.