From the desk of Mitchell Stein
Good morning. In the week since we’ve talked, I’ve been buried both literally and figuratively. Here in Portland ME, we’ve had over 30 inches of snow this week, while nationally, the “news” just keeps on coming. To help with the volume, today I’ve divided our stories into four categories:
Inside the Beltway – Investigating potential abuses of the Orphan Drug Act, Seema Verma’s hearing, allowing Canadian drug imports, and thoughts on potential changes at the FDA
Industry Trends – New national spending projections, right-to-try laws, step-therapy, subgroup analysis, and another rebranding
Company/Drug Specific – Dr. Soon-Shiong’s cancer moonshot, Allergan makes another deal, and a new psoriasis drug is approved but with a black-box warning
Weekly View In-depth – In this new section we’ll take a deeper dive into some of the top stories of the week with several articles on each topic. This week we look at CRISPR developments, Marathon’s DMD drug, and Alzheimer’s
Thanks to those of you who provided feedback last week. Remember, I’d love to hear from you about how this is working. Now, on to the news.
Inside the Beltway
As a result of investigative reporting by Kaiser Health News, the Senate is launching an investigation into potential abuses of the Orphan Drug Act.
This investigation examines the booming orphan drug business and how drugmakers have rushed into the marketplace with hundreds of drugs for rare diseases, helping patients while landing lucrative federal incentives and monopoly control for every drug that gets approved. Republican Sen.
On Thursday, Seema Verma, the nominee to head CMS, appeared before the Senate Finance Committee. Of particular note were her comments on PBMs: “When asked whether Medicare should negotiate drug prices, she gave a shout-out to the increasingly villainized pharmacy benefit managers. “I think we need to do everything we can do to make drugs more affordable for seniors. I’m thankful we have the PBMs and Part D program.””
Said she couldn’t comment on the new Obamacare regulation CMS released yesterday to tighten the enrollment rules: “I have not been involved in the development of that rule.”
Several prominent Senators push for canadian drug importation.
Industry execs are not fans of many of the prospective changes at the FDA being discussed by the Administration.
Nobody wants radical changes at the FDA, except perhaps President Trump and a few close associates. We’ve been tracking the industry’s clear reluctance to discard the FDA gold standard in drug development.
Dr. Robert Califf is also concerned with prospective changes at the FDA.
The just-departed commissioner of the Food and Drug Administration has concerns about plans to speed up drug approvals and dramatically reduce regulations at the agency, as advocated recently by President Donald Trump. Dr. Robert Califf, who stepped down last month, shared his thoughts about keeping Americans safe – and making sure drugs actually work – after about a year overseeing the federal agency.
New health care spending projections, including this on prescription drugs: “When the final numbers are in, the growth in prescription drug spending for 2016 is expected to have slowed to 5 percent from 9 percent in 2015. However, CMS has forecast growth of 6.4 percent per year between 2017 and 2025, in part because of spending on expensive newer specialty drugs, such as for cancer and multiple sclerosis.”
The cost of medical care in the United States is expected to grow at a faster clip over the next decade and overall health spending growth will outpace that of the gross domestic product, a U.S. government health agency said on Wednesday.
Will additional right-to-try laws do more harm than good?
The Trump administration has thrown its weight behind giving patients the “right to try” experimental drugs outside clinical trials. While such laws purport to offer hope for the dying, they will actually slow medical progress. “Right-to-try” laws have already passed in 33 states at the urging of the Phoenix-based Goldwater Institute.
Step-therapy, some love it, some hate it – this piece takes an in-depth look and makes some recommendations.
Increasing prescription drug costs impose economic burdens for patients and payers and are reflected in substantial increases in insurance premiums for individuals and employers and budget stress for public programs. One widely used approach to control prescription costs is “step therapy”: requiring patients to try a less expensive drug for a given condition before a more expensive option can be approved.
Subgroup claims in clinical trials are often not reproducible.
Evaluation of Evidence of Statistical Support and Corroboration of Subgroup Claims in Randomized Clinical Trials
Not wanting PhRMA to have all the fun with their “Go Boldly” campaign, the Generic trade group has rebranded itself as the Association for Accessible Medicines.
The Generic Pharmaceutical Association has rebranded to become the Association for Accessible Medicines (AAM), covering both generics and biosimilars. The new name and accompanying national marketing campaign were announced Feb. 14 during the group’s annual meeting. Rebranding will allow AAM to “communicate its mission to make more medicines more accessible to more people who need them,” the organization said.
As always, beware the hype.
he world’s richest doctor had a very bold plan. He’d assemble an unprecedented collaboration of companies, researchers, and doctors. Their mission: to vanquish cancer. By the year 2020, they’d build a working vaccine and test therapies in 20,000 patients. They would forever transform medicine.
Allergan makes another deal, this time $2.48 billion for a fat treatment that is a “cash business” (often not paid for by insurance).
Botox maker Allergan Plc agreed to pay $2.48 billion in cash for Zeltiq Aesthetics Inc, adding a system that it says helps people slim down by freezing fat away to the company’s line-up of aesthetic products.
A new psoriasis drug is approved, but with a black box safety warning that could suppress interest.
The FDA has come through with an approval for Valeant’s psoriasis drug brodalumab. But they’ve hobbled it with a black box safety warning on suicidal ideation and a risk mitigation marketing program that will likely hobble its revenue in an intensely competitive market. An FDA panel already flagged through the
Weekly View In-depth (a deep dive on significant stories of the week)
This week we’ll take a close look at three of this week’s major stories
Clustered Regularly Interspaced Short Palindromic Repeats – OK, how many of you knew that is what CRISPR means? This was a big week for the acronym as a group said it was OK to manipulate the genes of human embryos, and a court reached a decision in the patent battle on the technology. A group formed by the National Academy of Sciences and the National Academy of Medicine endorsed alterations designed to prevent babies from acquiring genes known to cause “serious diseases and disability.” This type of work is already going on in China and other countries. The following day, a patent court seemed to rule for the Broad Institute over UC Berkeley in the ongoing battle over who owns the patents on the technology. However, upon further analysis, the impact of the decision are far from clear. Additionally, there is concern that the licenses granted to-date will bottleneck the path to new discoveries.
I’ll turn to Forbes’ Matthew Herper for the last word as he provides some perspective.
It’s been a huge two days for CRISPR, the biotechnology innovation that makes it far easier for scientists to edit DNA in living cells. Yesterday, a report from the National Academies of Science didn’t rule out eventually using the technology to rewrite the genes of babies shortly after conception.
When we published last week, word had just come out regarding Marathon’s price for deflazacort. Needless to say, the reaction was not positive. First, we had lawmakers call the price “unconscionable.” Then, in reaction to the uproar, Marathon announced they were pausing their launch plans. While the drug has been around for a long time, it had not been approved by the FDA. Marathon touted all work that went into the research to assure approval, but some question exactly how much that research cost. PhRMA, who has recently launched a PR campaign to improve their image, announced that Marathon’s “recent actions are not consistent with the mission of our organization.” Kaiser Health News sums it up for us:
The latest flashpoint in the ongoing debate over high drug prices is Emflaza, an $89,000-a-year drug that treats Duchenne muscular dystrophy. People who have been watching the drug price issue closely, however, can reasonably ask why there is so much heat at that price tag?
Another day, another failed Alzheimer’s drug study. This time Merck halted a Phase 3 trial mid-way when it was concluded there was no hope of a positive result. Based on this latest failure, some are asking if it’s time to give up on the amyloid hypothesis. Guess How This Latest Alzheimer’s Trial Came Out, Newsweek’s new cover story out this morning, looks at moving even earlier in the process to find effective treatment (prevention).