From the desk of Mitchell Stein
Before we get started, a scheduling note – with the approaching Labor Day holiday, there won’t be an edition next week, August 31. We’ll all meet back here in two weeks Friday, September 7 – until then remember, it’s chaos, be kind. Now, on to the news where this week we’ll look at:
ICER in the News – Draft report on hereditary angioedema attacks, draft scoping document on AVXS and Nusinersen for spinal muscular atrophy, more on the CVS announcement, drug price policies to promote equity, and patient-reported outcomes
Inside the Beltway – prices in drug ads (2 articles), 100-day report on the Administration’s drug plan (2 articles), what the Administration thinks about PBMs, real-time oncology reviews, biosimilar agreement disclosures, and review of state activities on drug pricing
Industry Trends – the need for more comparative effectiveness research, when the copay is more than the cash price, primer on lowering drug prices, coordinating prescriptions, melanoma/brain developments, and a reporter talks about begining her cancer journey
Now, on to the news.
ICER In The News
ICER released a Draft Evidence Report assessing the comparative clinical effectiveness and value of therapies for long-term prophylaxis against hereditary angioedema attacks. The draft report will be open to public comment until September 20, 2018. ICER’s report includes four therapies for the prevention of hereditary angioedema attacks: lanadelumab (Shire), which is currently under review by the FDA with an approval decision expected in August 2018, and three C1 inhibitors (Haegarda, CSL Behring; Cinryze, Shire; and Ruconest, Pharming). The report will be subject to deliberation during a public meeting of the California Technology Assessment Forum, in October of 2018.
Institute for Clinical and Economic Review Releases Draft Evidence Report on Long-term Treatments for Prevention of Hereditary Angioedema Attacks
ICER has posted a Draft Scoping Document outlining a planned review of the comparative clinical effectiveness and value of AVXS-101 (AveXis) and nusinersen (Spinraza, Biogen) for the treatment of spinal muscular atrophy (SMA). The scoping document will be open to public comment until September 12, 2018. Nusinersen was approved in 2016 for treatment of SMA in both children and adults. AVXS-101 is a gene therapy currently being studied for use in infants with SMA, and an FDA decision is expected in the first quarter of 2019. ICER’s report on these therapies will be the subject of a March 2019 meeting of the New England Comparative Effectiveness Public Advisory Council.
Institute for Clinical and Economic Review Posts Draft Scoping Document on AVXS-101 and Nusinersen for Spinal Muscular Atrophy
Further analysis by STAT of last week’s CVS announcement regarding using QALY thresholds and ICER’s work.
ast week’s announcement by CVS Health, which operates one of the country’s largest pharmacy benefit managers, is a harbinger of what may lie ahead for drug pricing and access in the United States.
New England CEPAC member Austin Frakt, writing with Gilbert Benavidez in Public Health Post, discuss how drug policies can promote equity. Included in their list of suggestions is the use of value-based formularies that incorporate ICER analyses.
Viewpoint The United States has the highest drug prices in the world and it’s not even close. For millions in the country, the cost of prescription drugs is an ever-growing barrier to proper disease treatment. This is most often the case for minority groups, who have long experienced disproportionally adverse health access and outcomes.
Apparently, Patient Reported Outcomes (PRO) are only acceptable to the industry when they facilitate drug approval, not when they impact reimbursement levels. This Evaluate article discusses how the inclusion of certain PROs could impact ICER and other cost-effectiveness analyses.
If US Medicare officials follow the advice of their expert advisers, patient-reported outcomes (PROs) will be included in a national coverage plan for the costly CAR-T therapies Kymriah and Yescarta. But not if pharma has anything to do with it.
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
Last night, the Senate approved the “minibus” spending bill that among other departments included HHS. This was the second event yesterday that advanced the Administration’s plan to include prices in drug advertisements. The bill included an amendment facilitating the disclosure of prices (first article below from The Hill). Earlier in the day, CMS sent a proposed rule to OMB that would also require the disclosures (second article below from Axios). Of course, many questions remain as to how such a rule would be implemented – what prices would be included? The list price? The AWP? The coinsurance or copay for every health plan available? Stay tuned – I’m guessing we’ll be talking about this one for a while.
The Senate on Thursday passed a measure to provide funding to require drug advertisements to disclose the price of the drug after a last-minute push. The passage came as part of the massive health-care spending bill that the Senate passed on Thursday, which included the amendment from Sens.
It’s part of the administration’s drug pricing plan.
August 21 marked 100 days since the President, and his Administration began their prescription drug price lowering initiative. CMS marked the occasion with a press release, a fact sheet, and backup data. While CMS claimed significant progress on the issue, many were skeptical as reported in this BioPharma piece.
The Trump administration released a new report Monday touting the “unprecedented number of actions” it’s taken to curtail rising drug prices, seemingly pushing back against skepticism that its blueprint will result in transformative changes to the current system.
S&P Global’s Donna Young reviewed the claims and substantiations and found the evidence suspect (btw, if you’re not following her on Twitter, @DonnaYoungDC you should be):
Does the Administration love or hate PBMs? Yes. STAT looks at their mixed messages on PBMs. Personal editorial reminder – the world is made up of shades of grey – there can (and dare I say are) positives and negatives in the current PBM system.
ASHINGTON – Some days, President Trump vows to eliminate them. But then, there are days when his top health lieutenants promise to empower them. There are even days when they do both.
The FDA and “real-time” oncology reviews. This wasn’t the first (Novartis received one a few weeks ago), and it will be far from the last as reported by Endpoints.
FDA gives Merck ‘real-time’ OK for Keytruda, offering a peek at the latest extension of the agency’s data express highway
The FDA doesn’t always wait for a marketing application to come in before it begins its review these days. And that suits Merck just fine. The federal agency has stamped a full approval – following last year’s conditional OK – on Merck’s mega-blockbuster Keytruda for frontline use in a chemo
The FDA Law Blog writes about new bipartisan legislation introduced that would require agreements to limit biosimilar introductions to be publicly reported.
The NY Times looks at state measures that attempt to rein in drug costs and how manufacturers are responding. Let’s just say that so far, the state’s impact has been underwhelming.
WASHINGTON – States around the country are clamping down on pharmaceutical companies, forcing them to disclose and justify price increases, but the drug manufacturers are fighting back, challenging the state laws as a violation of their constitutional rights.
Dr. Aaron Carroll (Midwest CEPAC member) writing in the NY Times talks about comparative effectiveness research, highlighting how we need much (much) more of it before we can know if we’re spending money on the right therapies.
The New Health Care Comparative effectiveness research may be the best way to answer questions that patients and physicians face every day. We spend many billions of dollars each year on the discovery and development of new drugs, but almost none of it addresses two crucial questions: How do these new therapies compare with already known ones?
PBS Newshour with a story that can be filed under “an oldie but a goodie” as they detail why a copay can be more than the cash price of a drug. (A theme near and dear to my heart as I pay cash at the local supermarket for my generic statin instead of purchasing it through my insurer’s PBM).
Two years ago Gretchen Liu, 78, had a transient ischemic attack – which experts sometimes call a “mini stroke” – while on a trip to China. After she recovered and returned home to San Francisco, her doctor prescribed a generic medication called telmisartan to help manage her blood pressure.
Regardless of how familiar you are with drug pricing issues, this Commonwealth Fund brief is a great explainer of why they think we in the US pay so much more than other countries (Spoiler alert, they think we’re doing the whole purchasing thing wrong).
Most high-income countries spend a lot less on drugs per person than the United States does, even though their citizens use about the same amount of medications as Americans. An important reason: they pay lower prices. How do they do it?
We’re still not good at coordinating multiple prescriptions (across multiple providers). Reuters reports many still take drugs that impact each other’s effectiveness – yes, sometimes this is justified, but often it is not a conscious decision by the care team (or more likely isolated care providers).
(Reuters Health) – People who take pills to lower their blood pressure often take other drugs that reduce the pills’ effectiveness, a recent study suggests. Researchers studied data on 521,028 adults prescribed blood pressure pills for the first time and 131,764 people taking at least four different pills to lower their blood pressure.
Good news for melanoma patients whose cancer has spread to the brain – and potentially significant news for other cancers that spread to the brain, pending more research (NY Times).
A new study offers a glint of hope to people in a desperate situation: Patients with melanoma, the most serious form of skin cancer, that has spread to the brain. A combination of two drugs that activate the immune system shrank brain tumors in many melanoma patients and prolonged life in a study of 94 people at 28 medical centers in the United States.
A heartfelt piece by Politico writer Alexandra Glorioso, discussing her cancer diagnosis and initial treatment steps. Always helpful to be reminded of why we gather here each week, to help, in some small way, make their journeys easier.
It’s Wednesday morning. My boyfriend’s mom is walking around the house, humming to herself, and my puppy is waiting for her to leave, sitting patiently by the door. I’m also waiting for her to leave, just like I waited for my own mother to leave three days ago.