From the desk of Mitchell Stein
Good morning. After last week’s events around the AHCA, you might think that Washington would take this week to regroup. You’d be wrong. Lots of news to talk about both inside and outside the beltway. Today we’ll look at:
ICER in the news – Dupixent’s approval and pricing, ICER draft report on atopic dermatitis, New England CEPAC meeting on rheumatoid arthritis, approval of drug for MS, and April’s New & Noteworthy
Inside the Beltway – AHCA failure and drug reform efforts, Trump and drug pricing, NIH cuts, Price defends cuts to Congress
Industry Trends – New pricing strategy: charging less, manufacturers/PBMs blame each other, UK continuing budget issues, priority review drugs more likely to have issues, compassionate use and the FDA, Express Scripts/Anthem lawsuit continues, and a deep dive into Alzheimer’s treatments
Company/Drug Specific – Novartis and CAR-T, Vertex’s CF drug, Tesaro’s ovarian cancer drug and more problems for Dr. Soon-Shiong
Weekly View In-depth – Democrats’ New Drug Bill and Gottlieb’s Upcoming Confirmation Hearing
Remember, I’d love to hear from you with comments and suggestions. Now, on to the news.
ICER In The News
Dupixent was approved by the FDA for the treatment of atopic dermatitis patients who do not respond to or should not use topical prescription therapies. This story could have played out like many others, another breakthrough drug introduced at a sky-high price. But this one is different. From the Forbes report:
“Regeneron reached out not only to benefit managers like Express Scripts, but also to the Institute for Clinical and Economic Review (ICER), a nonprofit partly funded by insurers that releases independent judgements over whether drugs are worth the money. A draft report released Friday says that Dupixent is cost-effective, and may be underpriced.”
The price announced may still be considered too low by shareholders, and too high by payers – but to me, that sounds like a sweet spot for the drug’s price.
It was a symbolic act that spoke volumes. On stage with other pharmaceutical executives to talk about drug pricing at the Forbes Healthcare Summit last December, Regeneron cofounder, chairman and chief executive Leonard Schleifer physically moved his chair away from those of his peers.
The report referenced in some of the coverage was released by ICER on Friday (3/24/17). The Draft Evidence Report assesses the comparative clinical effectiveness of crisaborole for the treatment of mild-to-moderate atopic dermatitis in adults and children, and the clinical effectiveness and value of and dupilumab for the treatment of moderate-to-severe atopic dermatitis in adults. The Evidence Report will be the subject of a public meeting of the Midwest Comparative Effectiveness Public Advisory Council on May 25, 2017, in Chicago, IL.
March 24th was a busy day for ICER – in addition to the draft report mentioned above, New England CEPAC held a meeting to discuss ICER’s report on rheumatoid arthritis. There was extensive coverage in Medpage Today including this quote from ICER President Steve Pearson:
“The meeting concluded with a statement from ICER’s president Steven Pearson, MD, who said, “I come out of this meeting with a strange mixture of optimism and despair. The despair is based on just how awful the system can be, but in some weird way, ICER has looked at some other topics where patients have been waiting for the first treatment and are just desperate to get something. Here we have had wonderful innovation over time but we’re still stuck in the swamp somehow and that’s the disheartening part. It feels like the science, the innovation, has been brilliant — the ability to transform patients’ lives has been brilliant, and yet our system has put patients still in a bad situation and has left people who have very good intentions trying to figure out how to change the grinding gears in a way that makes sense.”
On Friday March 24, the nonprofit Institute for Clinical and Economic Review (ICER) presented its report on the cost and value of targeted immune modulators (TIMs) for rheumatoid arthritis (RA) in a meeting in Boston that included clinical experts, manufacturers, payers, and patients.
Another drug approval, this one the first for severe MS, mentions ICER’s work on the topic: “In the multiple sclerosis space specifically, the price watchdog Institute for Clinical and Economic Review (ICER) recently concluded that nearly every drug on the market is too costly relative to the value they provide for patients. Only one was reasonably priced, according to ICER: French pharma giant Sanofi’s Lemtrada.” The Fortune story mentioning ICER is below; you can find additional information in this NY Times story.
Late Tuesday, the Food and Drug Administration (FDA) approved a groundbreaking multiple sclerosis treatment from Roche/Genentech-the first one ever approved to treat a severe form of MS called primary progressive multiple sclerosis (PPMS).
The April edition of ICER’s New and Noteworthy is now available.
Inside the Beltway
In addition to the stories in this section, take a look below at Weekly View In-depth for closer looks at Scott Gottlieb (in advance of his Senate hearing next week) and the new prescription drug bill introduced by Democrats.
How will last week’s AHCA debacle impact drug cost reform efforts?
The Push Me Pull Me may be the animal totem to represent the relationship between Trump and the Democrats when it comes to moving forward on prescription drug pricing issues.
“I think with the defeat of repeal, the pressure will be on to do something about all the concerns about high prices. Drugs seem like the obvious place to go,” said John Rother, who heads the Campaign for Sustainable Rx Pricing. “They need a win on health care, and they need to resume their standing with consumers and constituents.
The President’s budget calls for drastic cuts to NIH and the FDA.
The Trump administration is proposing that Congress cut the current fiscal year’s funding for NIH, FDA and other public health agencies. The proposal, which has been rejected by Democrats and has not received public support from Republicans, is unlikely to be enacted without major modifications.
Price defended those cuts before Congress this week.
The chaos dimension has created far more chaos than anticipated. Come nightfall, Trump is often on the phone with billionaire, decades-long friends, commiserating and critiquing his own staff. His most important advisers are often working the phone themselves, trashing colleagues and either spreading or beating down rumors of turmoil and imminent changes.
This story takes the approval of Dupixent and the new MS treatment (discussed separately above) and examines what their pricing says about how the industry’s approach to pricing has changed.
Regeneron CEO calls decision ‘setting a new paradigm’ Political pressure is rising in U.S. over cost of medicine Two of the biggest pharmaceutical companies in the world did something unusual this week when they announced the introduction of breakthrough drugs: they charged less. If that sounds odd, it is.
Meanwhile, manufacturers and PBMs continue to point fingers at each other when it comes to responsibility for high drug prices.
As anger over rising drug prices continues to simmer in legislative chambers across the U.S., major industry trade group PhRMA is continuing its efforts to shift blame onto the payers and middlemen, which it claims fail to fully pass on the discounts given by drugmakers to secure coverage.
The UK (and NICE) continue to grapple with budget pressures.
Can society afford modern medicines? It’s a question facing governments worldwide and nowhere more so than in Britain, where a new budget test due to take effect on April 1 threatens to throw up another hurdle to patients getting the drugs.
As talk continues regarding speeding up drug approvals, it’s worth noting the experience to-date.
Discussing compassionate use and how the FDA needs to maintain its role.
‘I’m Willing To Try Anything’: Compassionate Use Access To Experimental Drugs And The Misguided Mission Of Right-To-Try Laws
March 27, 2017 Some patients facing death take drastic, or even desperate measures in order to prolong their lives. Such actions often include taking unapproved, investigational drugs.
An update as the Express Scripts/Anthem lawsuit continues.
A federal judge has dismissed two counterclaims by Express Scripts Holding Co. in a multibillion dollar lawsuit brought on by insurer Anthem over its drug charging policies. In the decision made public on Friday, U.S. District Judge Edgardo Ramos of the Southern District of New York tossed two claims brought by Express Scripts’ for being too similar to other counter-claims by the pharmacy benefits manager.
Endpoints News takes a deep dive into the state of Alzheimer’s treatments (free registration required).
by SHEHLA SHAKOOR, JOHN CARROLL, ARSALAN ARIF, and the ENDPOINTS NEWS TEAM In biopharma R&D, the biggest and best drug targets always involve unmet medical need-and Alzheimer’s fits that definition better than any other major disease. There’s good reason for that, and it’s inside our heads. The circuitry of the
Novartis gains the lead in the race for the first CAR-T approval.
Novartis $NVS has pulled into the inside track at the FDA with its CAR-T CTL019, picking up a priority review from the agency that will land a marketing decision for relapsed and refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia. The pioneering application comes as we
Positive results for a CF combo, but are they positive enough to justify the price?
Shares in Boston’s Vertex Pharmaceuticals are up 22% to $109 after the biotechnology company released data on an experimental combination pill to treat cystic fibrosis, a genetic disease that shortens patients’ lives by affecting their lungs and digestive systems. That gives the biotech a market capitalization of $27 billion.
Broad label approval for Tesaro’s ovarian cancer drug.
The FDA has wasted no time in giving Tesaro $TSRO a broad approval for the use of its PARP drug niraparib, which will now be marketed as Zejula as a maintenance therapy for recurrent ovarian cancer. The OK and the label that came with it represents a setback for Myriad
Weekly View In-depth
Democrats’ New Drug Bill
Improving Access to Affordable Prescription Drugs Act was introduced this week. You can read the summary of the bill here. Rachel Sachs takes a deep dive into the significance of the different provisions here. Below is RAPS account of the bill:
Posted 30 March 2017 ByZachary Brennan More than a dozen House and Senate Democrats late Wednesday unveiled a host of new provisions to bring down the rising cost of pharmaceuticals, offering up many ideas that have been publicly opposed by the pharmaceutical industry.
Gottlieb’s Upcoming Confirmation Hearing
Scott Gottlieb’s Senate confirmation hearing is next week (Wednesday, April 5). Considering some of the alternative nominees mentioned to head the FDA, industry is supportive of his nomination. Here is a look at his relationships with the industry. He has said he will recuse himself as needed.
Looking forward, part of his focus will be on approving “complex generics” faster (loosely defined as involving a drug and delivery device). And finally, Amitabh Chandra, Ph.D., and Rachel E. Sachs, J.D., M.P.H. look at the challenges he will face: An FDA Commissioner for the 21st Century