From the desk of Mitchell Stein
Good morning. We’ve had two big storms this week – one on Wall Street and one hitting land in the Florida Panhandle. Personally, I’m more worried about Florida (actually, I’m worried about Florida for a variety of reasons, but that’s for another time). With the storm coming the same week as a new dire climate report was released by the UN, it’s a reminder that if Gaia is angry with us, she has every right to be. Sending out good wishes to the people of Florida, Georgia and the Carolinas impacted by this storm – and those everywhere still recovering from storms past. On that cheery note, let’s get to this week’s pharma news where we’ll look at:
ICER in the news – Two new reviews announced, evidence report on therapies for long-term prophylaxis against hereditary angioedema, Tegsedi approval, and the value of Tisagenlecleucel
Inside the Beltway – Gag clause bill signing (3 articles), FDA-affiliated foundation, and NASHP state pharma law tracking
Industry Trends – Aetna/CVS merger gets go-ahead, biosimilars making slow progress (4 articles), price hikes responsible for growth, Bach on Medicare CAR-T options, Gardasil (2 articles), and Tom Allen on CER
Now, on to the news.
ICER In The News
ICER will assess the comparative clinical effectiveness and value of new therapies for secondary progressive multiple sclerosis (SPMS) and treatment-resistant depression 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 May of 2019. The SPMS review is expected to assess the clinical effectiveness and value of siponimod (Novartis), which is currently undergoing FDA review with an approval decision expected in March 2019. The treatment-resistant depression review is expected to assess the clinical effectiveness and value of esketamine (Janssen), which is currently undergoing FDA review with an approval decision expected in May 2019.
Institute for Clinical and Economic Review to Publish Upcoming Assessments on Treatments for Secondary Progressive Multiple Sclerosis, Treatment-Resistant Depression
ICER released an Evidence Report assessing the comparative clinical effectiveness and value of therapies for long-term prophylaxis against hereditary angioedema (HAE) attacks. HAE is an ultra-rare condition characterized by attacks of deep tissue swelling within the skin and/or mucosa. ICER’s report reviews three therapies for the prevention of HAE attacks: lanadelumab (Takhzyro, Shire Plc), and two C1 inhibitors (Haegarda, CSL Behring GmbH; and Cinryze, Shire Plc). ICER’s earlier draft report also included an additional C1 inhibitor, Ruconest (Pharming Healthcare, Inc.), which has since been removed from the assessment because the treatment is no longer under consideration for FDA approval for long-term prophylaxis. This Evidence Report will be the subject of a public meeting of the California Technology Assessment Forum on October 25, 2018.
Institute for Clinical and Economic Review Finds Treatments Prevent Hereditary Angioedema Attacks, but Cost-Effectiveness Uncertain and Sensitive to Frequency of Attacks
The FDA approved Tegsedi (inotersen) to treat hereditary transthyretin amyloidosis (hATTR). Last week, ICER released a final report reviewing the drug (and patisiran). ICER’s work was featured heavily in the coverage of the approval. In addition to the BioPharma piece below, coverage discussing ICER’s report included: FiercePharma, STAT, and MedPage Today.
Patients with a rare, hereditary disease known as transthyretin amyloidosis now have a second new treatment option, after the Food and Drug Administration on Friday cleared Akcea Therapeutics and Ionis Pharmaceuticals’ Tegsedi. Approval of Tegsedi puts Akcea in direct competition with Alnylam Pharmaceuticals, which won an FDA OK this summer for its drug Onpattro.
Despite the high cost of tisagenlecleucel, the chimeric antigen receptor (CAR) T-cell therapy to treat pediatric patients with relapsed or refractory B-cell acute lymphoblastic leukemia, the benefits of the treatment support the price, according to new research in JAMA Pediatrics .
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 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:
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
The President signed the gag clause bill this week, meaning pharmacists will be able to discuss price with all their customers (The Hill). Pharmacists have been front and center in the discussions about ending these contract provisions and concerning drug pricing issues in general. However, as noted on Twitter by Jay Hancock of the Kaiser Health News other clinicians have not been as vocal: “Pharmacists are clinical professionals like physicians. Yet pharmacists see it as part of their job to talk to patients about the price of treatment, and doctors do not.”
on Wednesday signed two bills banning “gag clauses” that keep patients in the dark about how to save money on prescription drugs. The clauses are sometimes included in the contracts insurers have with pharmacies – preventing pharmacies from telling customers they can save money on a drug if they pay with cash instead of using their health insurance.
Not as widely discussed is that one of the bills signed has a provision related to biosimilars (BioPharma Dive). For more on biosimilars, see several articles in the Industry Trends section below.
UPDATE: Oct. 10, 2018: President Donald Trump signed the bill into law at a ceremony held in the White House’s Roosevelt Room on Wednesday afternoon. President Donald Trump signed a bill into law Wednesday requiring drugmakers to send details of biosimilar deals to the Federal Trade Commission for antitrust scrutiny.
After the President’s signing of the gag clause bill, Secretary Azar told Axios’s Sam Baker that there is “more to come.”
“We’re taking on anybody that we need to take on.”
Did you know there was a federally chartered foundation created to support the FDA? No? Good, glad it wasn’t just me. As STAT reports, given the confusion over the foundation’s mission and lack of results, it’s not surprising many aren’t more aware of their work.
W ASHINGTON – A little-known nonprofit established by Congress over 10 years ago to help the Food and Drug Administration work with the private sector is still struggling with a basic question: Where is the cash?
The National Academy for State Health Policy (NASHP) is out with a new resource to track state laws aimed at PBMs.
Earlier this year, 20 states passed 31 bills to shed light on the opaque business practices of pharmacy benefit managers (PBMs). States took varied approaches to curb prescription drug costs by regulating PBMs. The chart below details the PBM practices each state tackled, and how their approaches compare with NASHP’s PBM model legislation.
To no one’s surprise, the merger between Aetna and CVS was approved by the Justice Department (in spite of the length of CVS receipts). As Reed Abelson reports in the New York Times, this marks an end to the era of independent PBMs and a return to the previous status quo when insurers were their own PBMs.
The era of giant pharmacy managers that brokered prices between drug companies and insurers and employers has been upended, now that the $69 billion merger between CVS Health and Aetna, a big health insurer, has been approved by the Justice Department.
Sometimes it seems that we’ll never see a benefit from biosimilars here in the US. However, as slowly as it’s happening, progress is being made. We’ll take a look at four articles below to see how the landscape is changing.
First, BioPharma Dive looks at an analyst’s report that reviews which manufacturers have been successful with biosimilars and how the landscape is shaping up.
While biosimilars have yet to take off in the U.S., top credit rating agency Moody’s remains optimistic of the future market opportunity for the biologic copycats, calling the competition “a rising headwind for branded pharmaceutical companies” in an Oct. 4 report.
Next, a look from Donna Young of S&P Global at this week’s approval of Celltrion’s biosimilar of Roche’s Rituxan.
Next, FiercePharma reports a small victory for Merck as the company secures a contract with the VA for its Remicade biosimilar.
Merck’s Renflexis hasn’t made much of a splash on the market since its FDA approval last April, but the company has now won business with the Department of Veterans Affairs thanks to a pricing offer that beat out its rivals.
And finally, FiercePharma reports on Europe where analysts think AbbVie needs to be more worried about Humira biosimilars.
Humira biosimilars will hit the European market next month, but AbbVie executives still soft-pedal the threat, predicting a sales drop of 20% next year, max. Analysts, however, are starting to question whether Humira really can avoid the biosimilar bloodletting that’s hit Merck’s Remicade and Pfizer’s Enbrel in the EU.
New research looks at revenue growth for certain top drugs. Spoiler alert: price increases are responsible for a lot of the growth (BioPharma Dive).
Price increases accounted for roughly 60% of recent sales growth recorded in the U.S. for many of the pharmaceutical industry’s top-selling drugs, found a new report from analysts at the investment firm Leerink. Between 2014 and 2017, U.S. sales for 45 leading products increased by 28%, or about $23 billion.
Dr. Peter Bach, writing in the New England Journal of Medicine, looks at Medicare coverage options for CAR-T therapies.
The FDA approved a label change for Gardasil 9, expanding usage up to those up to age 45 (it had previously been 26). The vaccine, which prevents HPV, has been met with mixed success globally. Let’s take a look at two articles.
First, the Washington Post discusses the expanded label while noting that less than half of the relevant population has been vaccinated.
October 5 The Food and Drug Administration expanded its approval of the HPV vaccine to include men and women between 27 and 45, an effort to protect more people from several types of cancer caused by the human papillomavirus. The vaccine, called Gardasil 9, previously was approved for people ages 9 through 26.
Second, it’s a very different story in Australia, where a combination of a school-based vaccination program and expanded screening are working together so well that cervical cancer may soon be effectively eliminated in the country.
MELBOURNE, Australia – Cervical cancer could be eliminated in Australia within the next two decades because of a government program to vaccinate children against the cancer-causing human papillomavirus, according to a new report.
Tom Allen, former Congressman from my adopted state of Maine, writes in Health Affairs about comparative effectiveness research. The historical review and look towards the future is an important reminder that we have great tools available to improve health care if we would only make use of them.
I represented Maine as a Democrat in the U.S. House of Representatives from 1997 to 2009. One of the issues I worked on was comparative effectiveness research (CER). CER has never had broad-based public understanding or support and it still carries the risk of significant special-interest opposition, yet it has undeniable potential to improve the quality and cost-effectiveness of health care in the U.S.