From the desk of Mitchell Stein
Good morning. I’m always fascinated by the early stages of a story when the narrative hasn’t quite jelled. That’s the case this week with Biogen/Eisai release of data on the study showing a benefit from their Alzheimer’s drug. Some publications were popping the champagne while some were focusing on the skepticism (guess which ones I’m featuring before you get to the story down below). Here’s the full line-up for this week:
ICER in the news – Draft Evidence Report on inotersen and patisiran for hereditary transthyretin amyloidosis, Merck cuts Hep C drug price, new ICER Board members, Regeneron and pricing, ICER reference case, and Orilissa approval
Inside the Beltway – Poll on President’s drug plan, history of price pledges, review of drug plan steps, Lilly CEO on importation, PhRMA lobbying spend, Health Affairs on out-of-pocket costs, NASHP on state drug pricing actions, and appeals court won’t review MD drug law decision
Industry Trends – Financial toxicity, sovereign immunity doesn’t fly to avoid IPR, Biogen/Eisai Alzheimer’s drug data, Kymriah manufacturing issues, 23andMe, and elephants never forget not to get cancer
Now, on to the news.
ICER In The News
ICER released a Draft Evidence Report assessing the comparative clinical effectiveness of inotersen (Akcea Therapeutics) and patisiran (Alnylam Pharmaceuticals) for hereditary transthyretin-related amyloidosis, a rare genetic condition that can affect that can affect multiple organ systems. The report, along with accompanying draft voting questions, will be open to public comment until August 17, 2018.
Institute for Clinical and Economic Review Releases Draft Evidence Report on Inotersen and Patisiran for Hereditary Transthyretin Amyloidosis
Marketwatch reported on Merck’s price cut of their Hep C drug. The piece included comments from ICER’s President Dr. Steve Pearson on Hep C drug pricing.
By Drugmakers have been lining up to tout new drug price policies lately, from deferring increases to freezing drug prices. Merck & Co. Inc. went a step further on Thursday, announcing a cap on average increases and cuts to some drug prices.
ICER announced the election of three new members to its governance board: Tanisha Carino, PhD, Executive Director of FasterCures, a center of the Milken Institute; Mark Skinner, JD, President and CEO of the Institute for Policy Advancement; and Anya Rader Wallack, PhD, Associate Director of the Center for Evidence Synthesis in Health within Brown University’s School of Public Health.
Forbes looks at Regeneron, its founder Schleifer, and its former and current pricing strategy, including working with ICER before the launch of Dupixent.
Sipping a lemon-flavored VitaminWater at a sprawling complex of laboratories and offices in Tarrytown, New York, Leonard Schleifer, the 66-year-old cofounder of Regeneron Pharmaceuticals, is, as usual, criticizing the pricing practices of other drug companies.
After the FDA’s approval on Tuesday, AbbVie announced the price of endometriosis drug Orilissa (elagolix). Several of the stories on the price included reference to ICER’s work on the drug including the story in BioPharmaDive and the Reuters story below that was picked up by several outlets.
NEW YORK (Reuters) – Drugmaker AbbVie Inc priced its new endometriosis drug Orilissa at around $10,000 a year after receiving approval from U.S. regulators for the treatment on Tuesday. Orilissa was approved by the Food and Drug Administration for the management of moderate to severe pain associated with endometriosis.
To encourage consistency in analytic approaches when modeling, ICER has defined a “reference case” specifying the approach that ICER and its collaborators follow for cost-effectiveness analyses.
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 Senior Health Economist will support the development and refinement of ICER’s methods for evaluating the cost-effectiveness, cost-benefit, and budgetary impact of drugs, devices, other technologies. The ideal candidate has doctorate-level training in health economics, decision sciences, or operations research and a compelling personal record of work in the fields of health technology assessment, comparative effectiveness research, and/or health services research.
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 Senior Health Economist will support the development and refinement of ICER’s methods for evaluating the cost-effectiveness, cost-benefit, and budgetary impact of drugs, devices, other technologies. The ideal candidate has doctorate-level training in health economics, decision sciences, or operations research and a compelling personal record of work in the fields of health technology assessment, comparative effectiveness research, and/or health services research.
Inside the Beltway/State Regulatory Developments
I am confident that everyone reading this sentence has at least heard that the President has a drug plan. Dear reader, know that you are special. In a new Politico/Harvard survey, only 27% of those polled were aware. Regarding the belief that the plan will be effective (among those who have heard of the plan) the percentage stands at 37% – I wonder if that’s representative of you, my faithful readers?
President Donald Trump was hoping for a big win with voters when he rolled out a massive blueprint to lower drug prices in May. But two months later, most Americans haven’t even heard about it, a new poll shows. And few of the Americans who are aware of his plan believe it will lower drug prices.
While we’re on the topic of being skeptical, Kaiser Health News takes a look at the history of drug price commitments and finds them lacking in impact. (As an aside, FiercePharma reported that Roche while Reuters reported that both AstraZeneca and Amgen jumped on the bandwagon this week with their pledges.)
Prescription drug prices were soaring. Angry policymakers swore they’d take action. Pharma giant Merck responded by promising to address the problem voluntarily, vowing to keep price increases under the overall rate of inflation. “We believe these moderate increases are a responsible approach, which will help to contain costs,” the Merck CEO said at the annual shareholders meeting.
That’s history, what about now? STAT takes a deep dive into some of the specifics (most of them reviewed in our edition last week). Tl:dr – some might have some impact, too early to tell.
ASHINGTON – When President Trump delivered his much-ballyhooed address on drug prices in May, even supporters conceded there were more question marks among his policy ideas than concrete proposals. But over the past week, the Trump administration has begun to put some periods at the ends of the sentences.
And finally, on this topic, two reminders that it will be tough going to implement an impactful plan.
First, Eli Lilly’s CEO speaks out against importation (The Hill).
Executives at Eli Lilly, one of the nation’s largest pharmaceutical companies, are denouncing the Trump administration’s proposal to consider ways to import prescription drugs from other countries. During the company’s second quarter earnings call Tuesday, executives said the idea of importing drugs from abroad, even in narrow circumstances, is concerning.
Second, Fiercepharma reminds us that the industry is a powerful force in Washington, and has been increasing their lobbying spend.
As the pharmaceutical industry faces potential pricing reform and continued criticism from patient advocates and members of Congress, the industry’s top trade group spent $15.5 million lobbying in the first half, an 11.5% increase compared with the same period last year.
This Health Affairs piece looks at the potential impact of the plan on Part D patients and finds good, bad, and ugly implications.
Addressing Out-Of-Pocket Specialty Drug Costs In Medicare Part D: The Good, The Bad, The Ugly, And The Ignored
But how would these recommendations directly or indirectly affect out-of-pocket costs for elderly and disabled individuals who are actually taking the most expensive medications? The combination of high drug prices and cost-sharing requirements under Medicare Part D’s current benefit structure results in a high out-of-pocket burden for patients needing specialty drugs.
NASHP reviews state action to curb drug costs. While 37 bills have been passed, their impact is still a large blinking TBD.
Twenty States Passed 37 Bills to Curb Rising Rx Drug Costs in the Short 2018 Legislative Session – NASHP
Despite a short legislative season, state lawmakers across the country introduced an unprecedented 160 bills to stem the rising cost of prescription drugs and have enacted 37 into law – with seven state legislatures still in session. Last year, state legislatures introduced 100 Rx cost control bills and passed 27.
Of course, just because a law passes doesn’t mean it gets implemented. The Washington Post reports that a federal appeals court refused to reconsider Maryland’s price-gouging law which was previously found to violate the commerce clause of the Constitution (rendering the law invalid).
A federal appeals court has denied a request from Maryland Attorney General Brian E. Frosh (D) to rehear a lawsuit challenging the state’s drug price-gouging statute. Frosh asked the U.S. Court of Appeals in April to reconsider the case after a three-judge panel of the court ruled that the law violates the commerce clause of the Constitution.
Financial toxicity – still important and still under-recognized by providers (Reuters).
(Reuters Health) – U.S. doctors must realize that many cancer patients battle “financial toxicity” along with their disease, researchers say. The costs associated with treatment, even for those with insurance, often create hardship and distress, according to a new study in the journal Cancer.
A federal appeals court puts the final nail in the coffin of the sovereign immunity scheme to avoid IPR. Endpoints News reports the novel legal maneuver – which turned into a public relations disaster – won’t be attempted again.
Federal judges ban Allergan’s sovereign immunity scheme in a slapdown that will bolster inter partes review for years to come
Allergan execs had thought they had found a clever way to dodge the threat of inter partes review of the patents for their big blockbuster Restasis. In the end, they $AGN paid a pretty price to bolster IPR, shutting and locking the door to the path they took – all
Biogen/Eisai release more details of their “promising” Alzheimer’s drug. As I noted above, the narrative on if this was indeed good news has not gelled yet leading to conflicting stories about the results. I find the Reuters story below among the most comprehensive (and appropriately skeptical). Don’t get me wrong, I would love a positive result, but we’ve been burned so many times before I’m not sure we have one here (that’s why I don’t quite get the irrational exuberance associated with these results). As of market close on Thursday, the stock market seems to be zeroing in on skepticism. Below the Reuters story, I’ve included Derek Lowe’s blog covering his review of the research issues.
CHICAGO (Reuters) – Biogen Inc and partner Eisai Co Ltd said patients with early-stage Alzheimer’s disease treated with their experimental drug BAN2401 experienced 30 percent less cognitive decline than patients who got a placebo in a highly anticipated midstage trial.
Well, the tease is over: Biogen and Eisai have released data on their Alzheimer’s antibody, BAN2401. And the situation is messy, as many had feared. The top-line results are mostly positive, but there are several confounding factors that make them a matter of argument (see below). The companies w
BioPharmaDive provides a reminder that it’s not just the discovery, implementation can be problematic as well.
Novartis is finding it harder than anticipated to commercially manufacture its cancer cell therapy Kymriah, acknowledging Wednesday that unspecified product variability has made it difficult to meet specifications in certain cases. Liz Barrett, head of Novartis Oncology, said the issues mainly involve CAR-T product for adult patients with lymphoma, rather than for children or young adults with adult lymphocytic leukemia, Kymriah’s initial approval.
When you pay 23andMe to analyze your DNA, you are helping their business model. We are reminded of that in a big way with the deal they just signed with GlaxoSmithKline to use all that DNA data for drug development (Forbes).
GlaxoSmithKline, a London-based drug giant is partnering with 23andMe, the San Francisco genetics startup, to develop new medicines. The companies will split costs and profits equally, and Glaxo will make a $300 million investment in 23andMe. The collaboration will last four years.
Among the things an elephant never forgets, is how to battle cancer. That’s because, as the Washington Post reports, they have 20 copies of a TP53, a gene that suppresses tumor cells – while we only have two copies. Has anyone seen my gene copy machine?
Elephants have 100 times as many cells as humans. But they seldom get cancer. This is surprising, because cancer is a result of cell division gone wrong, and the more cells an organism has, the higher the chances that some will mutate into tumors.