From the desk of Mitchell Stein
Good morning. Congratulations to my DC readers on last night’s victory leading to the Capitals’ first Stanley Cup. As DC hockey fans you see vicious battles all the time, it must be nice to take a break from politics and watch peaceful ice hockey instead. This week saw two major industry events, ASCO and BIO. We’ll have some stories from each, along with the rest of the week’s news. Here’s the full rundown:
ICER in the news – Dr. Pearson on WBUR, final evidence report on CFTR modulators, Health Affairs on PCSK9 inhibitors, and coverage of ICER’s migraine report
Inside the Beltway – Impact of shifting drugs from Part B to Part D, Creates Act has new life (maybe), FDA reorg, FDA generic drug numbers, 340B rule delay, and NASHP catalogs state drug transparency laws
Industry Trends – Outcomes similar, costs greater in US vs. Canada, Medicare drug spend report, a non-profit biotech from the Gates, about putting the price in drug ads, copay accumulators, immunotherapy hype, sometimes the answer is no, and ASCO recaps
Now, on to the news.
ICER In The News
With the Bio conference in Boston, WBUR has been covering several related issues. ICER’s President Dr. Steve Pearson did an extended interview and also was included in two additional pieces.
First, Dr. Pearson is interviewed, along with a representative of PhRMA, on the movement to align prices to value.
With biomedical innovation comes high drug prices. Does pharmaceutical research and development justifies the high cost of today’s life-saving drugs?
Second, looking at the cost of gene therapy, Dr. Pearson notes: ““As exciting as a treatment like Luxturna is, and as small as the patient population is, we might think we can pay almost any price, but we can’t,” Pearson said. “Because there will be a growing number of treatments like this. And we have to create a system that is ultimately affordable and sustainable.””
Affording Miracles: As Biotech Victories In Gene Therapy Excite, Costs Spur Quest For New Ways To Pay
This spring, Dr. Jason Comander of Massachusetts Eye and Ear injected three drops of a precious fluid into each of Jack Hogan’s eyes. It contained billions of copies of a gene Jack needs for his eyes to convert light into sight.
Finally, Dr. Pearson appears in this piece looking at Vertex and their CF treatment which are the subject of an ICER report.
Along the way Vertex got a boost from a unique funding source and, as a result, developed an unusual business model.
ICER released a Final Evidence Report and Report-at-a-Glance on cystic fibrosis transmembrane conductance regulator (CFTR) modulators. The report includes combination therapies tezacaftor/ivacaftor (Symdeko, Vertex Pharmaceuticals) and lumacaftor/ivacaftor (Orkambi, Vertex Pharmaceuticals), and monotherapy ivacaftor (Kalydeco, Vertex Pharmaceuticals), for the treatment of cystic fibrosis. ICER’s analysis was reviewed at a public meeting of the Midwest Comparative Effectiveness Public Advisory Council, where a majority of the Council voted that, in their specified indications, Kalydeco, Orkambi, and Symdeko in combination with best supportive care all offer a net health benefit compared to best supportive care alone.
Institute for Clinical and Economic Review’s Final Report on CFTR Modulators for Cystic Fibrosis Calls for Manufacturer Restraint and Increased Transparency in Pricing Decisions
This Health Affairs piece looks at the history of PCSK9 inhibitors, including discussion of ICER’s work on the topic.
Amgen and the Institute for Clinical and Economic Review have often clashed over drug prices, most recently after the cost watchdog said a new class of migraine meds would be a pricey burden on U.S. healthcare. But now that ICER has Amgen’s official launch price for first-in-class Aimovig-a price several thousand dollars below expectations-it has endorsed the med’s cost-effectiveness.
Inside the Beltway/State Regulatory Developments
The NY Times looks at the impact of shifting drugs from Part B to Part D – for some, it would increase their out-of-pocket costs.
WASHINGTON – When President Trump unveiled his plan to lower prescription drug prices in a Rose Garden speech last month, he said he would inject more competition into the market by bolstering negotiating powers under Medicare. But experts analyzing the plan warn of a possible side effect: The proposal could significantly increase out-of-pocket costs for some of the sickest people on Medicare.
The Hill reports that the Creates Act is not dead yet (but note this is just a committee vote, regardless of the outcome, no indication it will make it to the floor).
The Senate Judiciary Committee announced Tuesday that is is moving ahead this week on a controversial bill aimed at lowering drug prices. The bill, known as the Creates Act, seeks to crack down on drug companies using tactics to delay the introduction of cheaper generic drugs onto the market.
RAPS reports on changes coming to the FDA’s Center for Evaluation and Research to modernize and speed up drug approvals.
The US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) Director Janet Woodcock on Monday laid out her proposal to modernize the Office of New Drugs (OND) in an effort to make drug reviews more efficient.
In a stunning report that surprised no one, the FDA announced that generics save money. OK, while not surprising, they did attempt to quantify the savings from last year’s approvals, so worth a look (RAPS).
In a first of its kind report, the US Food and Drug Administration (FDA) says that generic drugs approved in 2017 led to $8.8 billion in cost savings during the calendar year, and $11.8 billion in savings through February 2018.
Healthcare Dive covers the delay of the rule that would penalize manufacturers for 340B program violations.
After facing scrutiny from the Senate and the White House, HHS Friday pushed back a decision on the 340B Drug Pricing Program that would impose civil monetary penalties for drug manufacturers that knowingly and intentionally overcharge hospitals for outpatient drugs to July 2019.
Our friends at NASHP catalog state drug transparency efforts.
Several states have passed drug price transparency laws that require drug makers to report the reasons behind dramatic price increases. These laws are an important first step to shine a light on why drug prices are rapidly climbing. To address a problem, state health policymakers need to understand it, but transparency laws don’t yet give …
A first of its kind study compares cost, utilization, and outcomes for cancer patients in the US (Western Washington) and Canada (British Columbia). CNN reports that costs in Canadia were half those in the US but resulted in similar outcomes.
The patients had similar diagnoses, levels of education, financial situations and other demographics that commonly affect health outcomes and mortality. Some of their ages were different, but the biggest difference between them is on which side of the border they live.
Axios covers a new report on new Medicare drug spending with this topline result: “The federal government paid more money to fill fewer prescriptions in Medicare Part D between 2011 and 2015, according to a new federal report.”
An OIG report puts big pharma in the crosshairs.
At the BIO conference, a new non-profit biotech company was born. OK, not born, but the Bill and Melinda Gates Research Foundation announced their plans to develop new medicines and vaccines with an initial budget of $100 million.
The Bill & Melinda Gates Medical Research Institute, which is holding an event today to show off its plans, will aim to develop new medicines and vaccines for malaria, tuberculosis, and diarrhea, which together account for 2.6 million deaths a year globally, many of them in children.
So that “include drug prices in ads” idea… will it be implemented, and if it is what might it look like?
President Donald Trump wants to control spending on drugs. One of his big ideas: include prices in advertisements, just like warnings about side effects. That’s not as simple as it sounds. Apart from legal questions about whether the Food and Drug Administration has the authority to require pricing in ads, other uncertainties arise.
Are copay accumulator programs working? Reuters reports on recently released data which seems to indicate they are lowering drug costs.
NEW YORK (Reuters) – A recently adopted tactic by U.S. health plans to limit the financial assistance drugmakers provide directly to consumers for prescription medicines is taking a toll on drug prices, according to a new analysis released on Tuesday. Real U.S.
Sadly, the hype of immunotherapies has gotten ahead of the science. STAT reports on patients looking for treatment even when it has not been shown to be effective for their specific form of cancer.
mmunotherapy is a source of great hope in cancer care. It has rescued some patients from the brink, while giving others a reason to believe that they, too, could beat the long odds.
And speaking of getting ahead of the science, the NY Times with an example of why we still have to do clinical trials.
Drugs that activate the immune system to fight cancer have brought remarkable recoveries to many people in recent years. But one of those drugs seems to have had the opposite effect on three patients with an uncommon blood cancer who were taking part in a study.
The annual meeting of the American Society of Clinical Oncology draws 45,000 doctors, researchers, and drug company executives to Chicago every year for a deluge of data that can change medical practice and move stocks. This year is no different.