From the desk of Mitchell Stein
Good morning. Before we get started today, I have a vitally important public service announcement for you. It’s national donut day! Here’s a list of all the chains participating. Remember, their offers apply to donuts, not donut holes. For now, donut holes are not free and continue to cause increased out-of-pocket spending for Medicare participants. Now, on to this week’s pharmaceutical news where we’ll cover:
ICER in the news – Migraine Evidence Report released
Inside the Beltway – Right-to-try bill signed, possible lowering of drug prices in 2 weeks, new drug risk regs to ease generic approvals, look at industry lobbyist now working on drug price plan, the FDA and political pressure, and CAP reviews state action on drug prices
Industry Trends – NIH investment in drugs (2 perspectives), to get around gag rules just ask, potential industry takeover activity, Theranos (one last story), Takeda shareholders nervous, and clinical hold on sickle cell work
Now, on to the news.
ICER In The News
ICER released an Evidence Report assessing the comparative clinical effectiveness and value of three calcitonin gene-related peptide (CGRP) inhibitors for prevention of migraine attacks: erenumab (Aimovig, Amgen/Novartis), fremanezumab (Teva), and galcanezumab (Eli Lilly). Erenumab was approved earlier this month. The other two agents remain under FDA review.
ICER’s report found that the CGRP inhibitors prevented approximately one to three days of migraine per month, on average. Cost-effectiveness analyses for erenumab and fremanezumab, using an estimated annual net price of $5,000, found that the price of the therapies aligns with the value to patients for whom other preventive treatments have failed. The drugs were not found to be cost-effective, however, if used to treat patients who had not already tried existing preventive treatments, which are far less expensive.
ICER’s previously released Draft Evidence Report used a placeholder price of $8,500 per year; however, following the announcement of erenumab’s list price of $6,900 annually, ICER updated these analyses. Assuming a 27% discount reflective of typical rebates and discounts to reach a net price of $5,000, cost-effectiveness findings became substantially more favorable than in the draft report.
The release was reported on by PMLive.
Institute for Clinical and Economic Review Finds Price of Erenumab Aligns with Clinical Benefit in Patients for Whom Other Therapies Have Failed to Prevent Migraines
Inside the Beltway/State Regulatory Developments
Earlier this week, the President signed the right-to-try legislation (The Hill). During the ceremony, the President claimed that the law would save “hundreds of thousands of lives.” The NY Times fact-checked that claim and found it questionable. Additionally, Dr. Vinay Prasad did a tweettorial on the bill in general (and its overall shortcomings). In an email to her staff, CDER Director Janet Woodcock said they were working on how to implement the law and that the manufacturer should answer most questions on developmental drugs. RAPS reported on the communication and other implementation issues. Then yesterday, Senator Johnson, one of the bill’s sponsors, confirmed what many had thought – that in fact, it IS the intent of the bill to weaken the FDA’s authority and that he had thoughts on how the bill should be implemented. Two stories below, first on the signing, then on the Senator’s letter.
signed a bill Wednesday allowing terminally ill patients access to experimental medical treatments not yet approved by the Food and Drug Administration (FDA). Dubbed “right to try,” the law’s passage was a major priority of Trump and Vice President Pence, as well as congressional Republicans.
ASHINGTON – Sen. Ron Johnson, the author of the federal “right-to-try” law signed by President Trump this week, wants to make one thing clear: His new law is meant to weaken the Food and Drug Administration. “This law intends to diminish the FDA’s power over people’s lives, not increase it,” he wrote in a letter to Commissioner Scott Gottlieb Thursday.
During the signing ceremony for the right-to-try legislation (story above), the President said that drug companies would announce “massive drops” in drug prices in two weeks. Politico reports that other than the President’s remark, no details were forthcoming. CNBC reached out to several drug companies, but they either did not know anything about it or did not comment.
President Donald Trump on Wednesday said some of the nation’s biggest drug makers will voluntarily announce “massive” drug price cuts in mid-June, though he wouldn’t elaborate and some industry sources said they were unaware of any announcement.
One of the ways brand-name drug manufacturers have stalled generics is through use of the risk mitigation requirements. In working to speed approvals, the FDA announced changes to the regulations (reported on by S&P’s Donna Young).
Politico takes a look at a former industry lobbyist now steering the Administration’s drug pricing plan and asks if he is violating the Administration’s own rules.
The White House official who will shape a large part of the administration’s drug price plan worked on many of the same issues as an industry lobbyist, raising questions about whether he violated President Donald Trump’s ethics rules.
Endpoints asks the question, is the FDA making decisions based on political pressure?
FDA executes a 180 on TherapeuticsMD therapy, handing it an OK – and stoking concerns about political influence
A year after the FDA handed TherapeuticsMD $TXMD a rejection letter for its therapy preventing vaginal pain during sex, the agency has done a complete 180 – approving the treatment after waving off the safety concerns that had once warranted a CRL. The decision to drop the CRL and allow
The Center for American Progress reviews state action on high drug prices.
Download the PDF here. Over the past few years, high and rising prescription drug prices have generated significant controversy. Large, bipartisan majorities of the public support increased government action to reduce drug prices. Yet in spite of this federal inaction, several states have taken steps to address prescription drug prices over the past couple years.
The NY Times took a look at NIH’s role in funding new drugs. However, Harvard economist Amitabh Chandra took exception to the article on Twitter, discussing its shortcomings.
WASHINGTON – On Aug. 30, the Food and Drug Administration approved a radical new cancer treatment that harnesses a patient’s immune system to attack tumor cells. The drug, known as Kymriah, grew out of research conducted and supported by the National Institutes of Health. Seven weeks later the F.D.A.
Great reminder from Kaiser Health News that while everyone is talking about gag clauses in Medicare Part D contracts, there’s a simple workaround. The customer just has to ask (not the best solution, but one that patients can take today).
This story also ran on This story can be republished for free ( NPR. details). As part of President Donald Trump’s blueprint to bring down prescription costs, Medicare officials have warned insurers that “gag orders” keeping pharmacists from alerting seniors that they could save money by paying cash – rather than using their insurance – are “unacceptable and contrary” to the government’s effort to promote price transparency.
Endpoints reports on Geoffrey Porges’ thoughts on possible pharma industry takeover activity.
With $460B in borrowing capacity, will any of the Big-20 biopharmas go after one of the top-5 takeover targets?
Leerink’s always interesting Geoffrey Porges knows how to get your attention. And nothing turns the head faster than a list of major league takeout targets. Now that Takeda has lined up its $62 billion deal to buy Shire $SHPG, without working too hard to line up the money, Porges decided
Like many of you, I have been fascinated by the Theranos saga over the years, at times perhaps paying too much attention to the story. The definitive book on the subject – Carreyou’s book Bad Blood: Secrets and Lies in a Silicon Valley Startup was recently published. So let this piece by Forbes’ Matthew Herper reviewing the book serve as a coda for our coverage (at least until I review the planned movie starring Jennifer Lawrence).
Before Elizabeth Holmes was putting one over on the world with her would-be blood-testing company, Theranos, she was a child living next door to Silicon Valley venture capitalist Tim Draper, who would later help her start her company. Holmes found a friend in Draper’s daughter, Jesse.
Some Takeda shareholders ask the firm to take a deep breath before proceeding with the planned Shire deal, as reported by FiercePharma.
Takeda shareholders fear dilution, debt from Shire deal pose ‘high risks,’ demand shareholders vote on it
Takeda’s shares have lost more than 20% of their value since late March, when the company first announced its intention to buy Shire. That loss of market value is being cited by a group of 12 shareholders stepping up to oppose the deal.
Hold off on that talk about a sickle cell cure this week as the FDA placed a clinical hold on the new gene therapy. However, despite the FDA’s action, work moves forward in Europe (Endpoints).
The FDA has slapped a clinical hold on the IND filed by Vertex $VRTX and CRISPR Therapeutics $CRSP on a new gene therapy for sickle cell disease. The two biotechs announced an alliance on sickle cell disease late last year, co-developing CTX001, which was made with CRISPR Cas9 tech. The