Latest Drugwonks' Blog
Megan and John Crowley (Courtesy of the Crowley Family)
Last night President Trump was attacked for speaking about Megan Crowley a 20-year-old freshman at Notre Dame who was diagnosed at birth with an orphan illness called Pompe Disease.
The reason? The President used the opportunity to call for more medicines to keep young women like Megan alive.
Here’s what the President said:
"Megan was diagnosed with Pompe Disease, a rare and serious illness when she was 15 months old. She was not expected to live past 5.
On receiving this news, Megan's dad, John, fought with everything he had to save the life of his precious child. He founded a company to look for a cure, and helped develop the drug that saved Megan's life. Today she is 20 years old -- and a sophomore at Notre Dame.
Megan's story is about the unbounded power of a father's love for a daughter.
But our slow and burdensome approval process at the Food and Drug Administration keeps too many advances, like the one that saved Megan's life, from reaching those in need.
If we slash the restraints, not just at the FDA but across our Government, then we will be blessed with far more miracles like Megan."
He had not even finished his speech when cynics on Twitter and in the media, belittled the Crowley family’s courage because, in their view, they were tools for what they hysterically call Trump’s reckless gutting of the FDA. (Note that they never specify what would be gutted but assure us that if Trump got his way, the FDA would turn America into The Walking Dead.
Of all the sniping and snide observations, Former FDA commissioner David Kessler and Washington Post reporter Carolyn John stand out as the most obnoxious and condescending.
For his part, David Kessler posted on Twitter @DavidAKesslerMD
Trump mischaracterizes Pompe drug approval process. Approved in 9 months based on 39 patients. Not "slow and burdensome."
First, Trump never even mentioned the Pompe drug (Myozyme, the drug Crowley’s company developed) approval.
Second, “approval” refers to the FDA’s review of all the other studies and data the agency requested. The FDA is required by law to review such completed applications in 180 days. Not. Nine. Months.
Meanwhile, it took about 6 years to complete the studies used to establish the drug’s safety and efficacy. That’s a lot less than the 10 years it takes to evaluate drugs for larger groups of patients.
But third, and most important, Crowley was in the audience because if the FDA was using its current regulatory approach to orphan drugs in 2001, the drug still wouldn’t be approved. Nor is Crowley is calling for gutting the FDA as critics hysterically claim. As Crowley points out in an op-ed in the New York Observer: “the FDA must always put patient safety first.”
The problem is that despite having a decade of experience in dealing with rare pediatric conditions like Pompe or Fabry, the FDA now often requires more information than ever before to approve orphan drugs. As a result, they take as much time and money to get through the FDA as do medicines more common conditions,
But the award for the most sneering and fact-free hit piece goes to the Washington Post’s Carolyn Johnson:
“The actual drug, that saved Megan’s life is manufactured in Belgium and was developed by a biotech company founded by a Dutch immigrant — a company that is today owned by a French firm. The drug was invented through a scientific experiment that couldn’t have happened without international collaboration. And a president who has said he wants to bring down drug prices just held up as a shining example of innovation a drug that costs an average of $298,000 a year, per patient.”
The company was Genzyme, a US biotech company founded in Boston – not Europe -- to bring orphan drugs to market. Genzyme was sold to Sanofi, which invests most of its R&D in the United States.
Moreover, the cheap shot about drug prices (“a shining example of innovation” Johnson snarks) ignores the fact that the average price of hard to manufacture medicines for very small groups of patients has declined since Genzyme developed Ceredase for Gaucher’s disease in 1990 for a list price of $360000. (That’s $558000 in 2016 dollars.)
The sneering continues:
"It’s also unclear whether the Crowley’s inspiring story and the development of the drug Myozyme (also called Lumizyme) is really an example of how “our slow and burdensome approval process at the Food and Drug Administration keeps too many advances, like the one that saved Megan’s life, from reaching those in need,” as Trump described.
Other companies have pointed to Myozyme as an example of the agency’s flexibility in getting drugs approved quickly, based on small amounts of data.
Briefing materials prepared by the drug company Sarepta Therapeutics to support the approval of their drug last year cited Myozyme as an “approval precedent” that they hoped to follow.
Last April, at a hearing in support of Sarepta’s drug, one of the researchers involved in Myozyme’s approval testified to the advisory committee that its passage through the regulatory process as a helpful parallel to consider.
Against the recommendation of its advisory committee, which wanted to see more evidence, regulators approved that drug."
Omission: In fact, FDA reviewers virtually told the FDA advisory committee not to approve the Saretpa drug because it followed the Myozyme approach.
No one is suggesting that Myozyme was delayed by the FDA or that the agency in the last couple of years has been quicker in reviewing applications for drug approval.
The issue is whether the FDA could move more quickly by requiring data that has less to do with establishing the safety and benefit of medicines and more to do with employees demanding an increasingly higher degree of statistical certainty that has nothing to do with those goals
The issue is whether the Amicus drug for Fabry Disease – approved in Germany, Japan, and Great Britain – requires another randomized study and two more years of data to determine whether patients are more likely to have diarrhea.
The issue is whether patients and their families should have as much to say about the drugs that shape whether they live or die as well how they live, as the naysayers who claim that the experts know better.
The smug elite – like Kessler and Johnson -- realize their power over the FDA review process is slipping away and they are beside themselves that Donald Trump is determined to wrest their control away. How can you tell? Because they turned a courageous young woman and her father’s decade-long struggle to save her life into objects of their rage against the new President.
U.N.'s errant prescription for drug access
What's the best way to improve health care for billions of people in the developing world? If you answered, “Attack health care firms,” you qualify for a job with the United Nations.
You'd also be dead wrong.
The U.N. recently released a plan to severely weaken patent protections and other forms of intellectual property (IP). U.N. officials believe that this will bring down the price of medicine in the developing world, thereby improving people's access to drugs.
Their recommendations would do the exact opposite. Dissolving IP protections would disincentivize drug research and slow the discovery of new treatments. Patients in both the developing and developed worlds would be worse off.
Drug development is an enormously expensive endeavor. It costs, on average, $2.6 billion to bring a new medicine to market. Just one out of every 5,000 promising compounds makes it from the lab through clinical trials to the pharmacy shelf. And just two out of every 10 approved drugs ever turn a profit.
IP rights are what make risky drug research a worthwhile bet for investors. By temporarily preventing the production of generics and creating a market monopoly, they ensure that the original innovator has a chance to profit from successful new products.
America has the strongest IP protections in the world, and they've driven a spectacular rate of innovation.
We've created more than 500 new medications in the past 15 years alone. That's more than the rest of the world combined. And we account for 70 percent of the 7,000 new drugs now under development globally. We're also home to 12 of the top 20 medical-device companies in the world.
These research efforts are aimed at the most devastating diseases. Today, American scientists are working on 74 new asthma medicines, 92 new arthritis treatments, and 93 therapies for Alzheimer's disease. They're also developing over 800 cancer treatments.
These products lead to longer lives for people all over the world. Consider HIV/AIDS: Back in the 1980s, a diagnosis was a death sentence. Today, antiretroviral cocktails keep patients living for decades. There's no way firms would have plowed billions into researching and developing these therapies without strong patent protections.
Patent protections are not the reason poor patients in the developing world can't access breakthrough medications. Among the 375 drugs the World Health Organization has deemed essential, just 25 are still patented.
Cost isn't a problem either. Poor patients pay just 6 percent of the U.S. retail price for 350 off-patent drugs.
The U.N.'s war on IP rights ignores the real barrier to drug access: insufficient medical infrastructure.
For instance, many developing countries suffer from a severe shortage of health care professionals, particularly in rural areas. In Nicaragua, 50 percent of health personnel work in the country's capital, yet only 20 percent of the country's population lives there.
The U.N. needs to ditch its fixation on intellectual property rights and instead focus on the real problems of the developing world. Undermining IP protections would deter drug development and ultimately deprive needy patients of lifesaving treatments.
Peter J. Pitts, a former FDA associate commissioner, is president of the Center for Medicine in the Public Interest.
Instead, the rare disease community finds itself under assault as little more than front groups for pharma companies who – critics claim – want the freedom to bankrupt our health care system by charge hundreds of thousands of dollars for medicines that aren’t that safe or effective.
The critics are attacking patient groups for strategic reasons: shut down patient groups or at least neutralize their influence and their organizations – anointed as objective and expert – will consolidate their power over the development, pricing and use of new medicines.
One of the leaders of this movement is Steven Pearson, the founder of the Institute for Clinical and Economic Review – an organization that claims it’s “a trusted non-profit organization that evaluates evidence on the value of medical tests, treatments, etc.” Pearson claims the influence of patient groups will drive our health care system and economy into bankruptcy. As the number drugs for rare diseases for small groups of patients increase, their higher prices (relative to medicines for common medical conditions) will become unaffordable. And the driving force is the ability of rare disease groups to advocate for new medicines.
Or as he puts in an article entitled, “Which Orphans Will Find a Home? The Rule of Rescue in Resource Allocation for Rare Diseases,” publicity can be a powerful and important tool for advocacy groups, “but it is not an appropriate ethical justification for coverage of particular orphan drugs over others.” Pearson writes, our nation needs a framework that will restrain “society’s desire to help those weakest among us, especially when their small numbers allow us to see them as unique individuals.”
Armed with this noble sentiment (and about $5 million from health insurers and the Laura and John Arnold Foundation) Pearson is positioning ICER to develop this ‘value framework.’
In May, ICER will be meeting with patient groups and others to recommend “fair prices that reflect the value of orphan drugs to patients and the health system to allow for broader insurance coverage for innovative new treatments.” (It is targeting Spinraza™ (a new drug for spinal muscular atrophy and Exondys-51™ for Duchenne muscular dystrophy (DMD))
Pearson believes that a “bright line between what constitutes a fair claim on health benefits and what does not will be difficult to draw.”
To ICER and Pearson, that bright line is $150000 for an additional year of life. Most new medicines for rare diseases are expensive and don’t save health insurers money. And ICER measures value from the insurer or government health program perspective. So most new orphan drugs would have been discounted more than 90 percent of the rebated price a medicine just to stay behind Pearson’s bright line.
Next, ICER sets a limit of $915 million on what should be spent on each new drug. It multiplies the price of the drug by the number of people who could benefit. Going over the cap mean that many people with rare diseases will be denied access to a growing array of new medicines. ICER uses these bright lines to “improve affordability” with changes to pricing, payment, or patient eligibility
ICER justifies such limits because beyond that cap it “…we’re siphoning off resources for other things we need like better schools and more resources for local police, roads, and bridges. “
These claims of budgetary Armageddon are overhyped. Between 2007 and 2014, orphan drugs have increased as a percentage of spending on drugs (from about 2 percent to 4 percent in Europe and 5 percent to 8 percent in the United States) even as the percentage spent on drugs has remained the same. And a study by Dr. Frank Lichtenberg shows new medicines for rare diseases are reducing the number of life years lost by about five percent a year.
Conversely, ICER’s limits on access will hurt people and rob them of their lives. Lichtenberg’s study found in France, which took longer to pay for fewer orphan drugs relative to the US, the number of deaths declined by 1.8 percent.
It is precisely our moral sense to save lives in immediate danger and at any expense that sustains humanity and economic progress. We need more orphan drugs not just because more people will be able to enjoy life and live longer. It’s because civilization is enriched when we provide people who are marginalized because of their medical condition the opportunity to contribute to our well-being and happiness.
Pearson and ICER are a threat to that moral vision. They threaten the remarkable advances in medicine that Rare Disease Day celebrates, made possible in large part by the patient advocacy groups that ICER seeks to replace.
Specifically, Rockoff and Loftus report: most of the increases in list prices are not paid by insurers. PBMs and insurers have their drug costs reduced by rebates and discounts, while consumers (patients) pay the full price. Here's the chart that demonstrates this fact.
Source IMS Health
In addition to pocketing rebates, PBMs and insurers make money by charging consumers of the newest drugs up to 50 percent of the list – not rebated price of the drug. Indeed, Rockoff and Loftus try to skate past this ripoff by blandly noting: "regardless of discounts to middlemen, patients who have high-deductible health plans may have to pay close to full price for at least part of the year. Indeed, patients who have high deductible plans are not paying anywhere near full price for any other medical service except prescription drugs.
The article notes: "The discounts mean that manufacturers must share the increased revenue with others, but they can still leave buyers such as insurers paying the higher price, or most of it. And regardless of discounts to middlemen, patients who have high-deductible health plans may have to pay close to full price for at least part of the year."
As if on cue the American Cancer Society Cancer Action Network released its most recent review of drug cost sharing for cancer patients on Obamacare plans and concluded:
"Unfortunately, most plans place many, or even all, covered cancer drugs on the highest cost-sharing tier. Among the formularies we studied, even generic cancer drugs appeared on the most expensive tier with regularity (41 percent of the time in the case of Etopside, and 61 percent for Imatinib Mesylate). Most of the time, the highest cost-sharing tier requires coinsurance rather than a flat copayment; but it is very difficult for consumers to manually estimate their coinsurance costs because the negotiated drug price on which coinsurance is based is not shown.”
So if drug companies are keeping the lid on drug prices, why aren't cancer patients seeing any difference at the pharmacy?
As the folks at OhMD quip, “As a point of reference, 7% of the American population also believes the moon landing was faked, if that helps give you some perspective.”
Why? Consumer technology (the apps we all use in our daily lives) typically solve a problem in a very simple way.
As I’ve said before – healthcare app-ens.
ProPublica, although calling itself "journalism in the public interest,” remains silent about its own funding and conflicts of interest while it brazenly challenges others.
A recent article, " Big Pharma Quietly Enlists Leading Professors to Justify $1,000-a-Day Drugs," questions the credibility of respected academic experts who explain and defend the high cost of developing new treatments and cures, simply because they get funding from the pharmaceutical industry.
Yet, ProPublica receives funding from Arnold Foundation, dedicated to attacking drug pricing, drug spending and by extension the pharmaceutical industry.
Since 2013 ProPublica has received $4 million from the Arnold Foundation. The support is part of nearly $20 million in multiyear grants to organizations that are being paid by the foundation to develop new policies to attack drug prices in a way that will reduce the development of new treatments and cures.
In addition, the foundation is funding news outlets like ProPublica to report on the organizations it is funding, and to support a group called Patients for Affordable Drugs who advocate for policies the other Arnold entities are creating and publicizing. So ProPublica is part of what the foundation calls a 'portfolio of investments' in attacking drug pricing and drug spending. That's journalism in ProPublica's financial interest, not in the public interest.
The piece claims that the scholars (who have a firm called Precision Health Economics of PHE) enlisted by drug companies to defend prices didn’t regularly disclose funding, In fact, the economists who such as Tom Philipson, Dana Goldman and Darius Lakdawalla have been conducting such research for nearly 20 years and they have been disclosing their funding when required or relevant. In any event their relationship with companies was well known to everyone in the field.
Ironically, ProPublica alleges monkey business because of PHE’s failure to disclose in an article But Propublica has also has conflicts which,unlike PSE, it doesn’t disclose at all.
That’s not just being “quietly enlisted: That’s keeping quiet to avoid being criticized for hypocrisy.
Indeed, Annie Waldman, the author of the article, interviewed several individuals and discusses alternative value frameworks who disagree with the PHE methodology and belittle their assertions about prices reflecting values.
These sources are funded by the Arnold Foundation as well.
To discredit the claim that new drugs cost a lot to develop Waldman cites Dr. Aaron Kesselheim who states: “There is substantial evidence that the sources of transformative drug innovation arise from publicly funded research in government and academic labs.” Kesselheim is “an associate professor at Harvard Medical School whose research looks at the cost of pharmaceuticals. Pharmaceutical pricing, he says, is primarily based on what the market can bear.”
And Kesselheim also gets funding from the Arnold Foundation.
Waldman also discusses the role of ICER in setting drug prices based upon its opinion of value. She describes ICER as an organization vigorously attacking US drug prices. Waldman states that: “Some patient groups have contended that ICER emphasizes cost savings because it receives funding from health insurers. However, foundations are ICER’s biggest source of funding, and it is also supported by the pharmaceutical industry and government grants. “
ICER does NOT get money from the drug industry. Waldman fails to mention funding from California Blue Cross Blue Shield Foundation or that ICER receives most of its funding -- $4.6 million from the unmentioned Arnold Foundation.
It is perfectly acceptable to criticize PHE approach on substance. But as a colleague of mine observed: “foundation money is no different than pharma money if the purpose is the same: to support advocacy-driven research.” I applaud the Arnold Foundation for supporting groups that advance its drug pricing agenda and I am grateful to receive funding to advance other ideas about how to make medical innovation accessible and affordable.
However, if you are going to make funding sources an issue, it should apply to thee and me. And more to the point, if you are a media outlet receiving money from an organization that also funds the groups you cite in your article and use to research your piece, you should at least tell the public that. That’s not just nondisclosure. That’s misleading. It certainly isn’t journalism.
"A new patient advocacy group launches Wednesday that distinguishes itself by focusing only on drug prices and eschewing money from the pharmaceutical industry at a time when drug makers are pouring millions into a campaign fighting efforts to regulate them."
In otherwords, groups that get money from biopharma companies are not legitimate. O' Donnell claims the new group -- Patients For Affordable Drugs -- is the only organization tackling policies to bring down drug prices because they are pharma free. That is untrue. What is true is that most patients groups -- and not PAFD focusing on the rigged system wherein PBMs, insurers and government health programs that set drug prices to maximize rebate revenue. Those are the prices that matter. If you want to reduce launch prices and price increases of drug companies, change the way drugs are paid for and the cost of drug development. PFAD ignores both. Why?
They focus on the immoral practice of getting $100 billion in rebates (that reduce drug prices) and then forcing the sickest patients to pay up to 50 percent of the retail price of the same drugs. (That's another $30 billion from less than 3 percent of all patients) But PFAD is perfect because it doesn't take 'drug' money. So what if it ignores the rebate games, the forced drug switching, fail first step therapy, etc. So what if it ignores the fact that by passing through rebate dollars the patient share of any drug cost could be zero, without raising premiums.
PFAD is perfect because it doesn't get drug money and supports government negotiated drug pricing for Medicare without acknowledging that such practices have hurt patients in Medicaid, the VA and everywhere price controls are used around the world.
Guess what other organizations share the same approach or seek to promote it? ICER, the drug pricing group at the Oregon Health & Science University, and several others. And they all get money from the John and Laura Arnold Foundation which has publicly stated it wants to build a network of groups attacking drug prices and the 'grass roots' entities to lobby for the policies and approaches the other entities produce.
So the real debate is how to best increase the pace of medical innovation and ensure that they are accessible. The Arnold-funded family of groups pursue administrative approaches and regulations to limit price and the pace of drug development. No mention of PBMs, insurers, etc. Patient groups are focusing on the system as a whole. And patient groups are less likely to support more government control over prices and access. They want a patient-centered drug development process. Arnold-funded 'experts' want more randomized trials where patients are exposed to placebos half the time.
To assert that Arnold foundation money is less tainted than money from a biotech company is ridiculous. If patient groups got money from the Merck Foundation instead of Merck for instance, would it pass O'Donnell's purity test?
(Many companies fought against eugenics in the early part of the 20th century. Foundations supported eugenics, accusing corporations of simply wanting more immigrants to work in their factories. )
It is time to stop branding patient groups as tainted because of their funding sources. Let's focus on the issues. The Arnold Foundation is seeking to change policy. So are patient groups.
In other words, it’s not just fewer opioids for patients with less severe pain or with conditions for which there are non-opioid alternatives (such as fibromyalgia and diabetic neuropathy). It means we need better ways of tracking the patient experience. One solution to narrowing the gap between prescription and outcomes measurement are mobile apps. The gathering and appraisal of real world evidence can expedite identification of problems before they become deadly. If we can identify misuse earlier, we can help eradicate abuse and addiction.
Apps present us with just that opportunity – a virtual ounce of prevention.
ICER CEO Steve Pearson has tried to salvage what's left of the organization's shredded credibility by claiming ICER's mission is to help patients. Specifically, ICER argues that it all it wants to do is "spur discussion among stakeholders to ensure that patients have access to the medications at prices that are aligned with the value they bring to patients.”
In fact, ICER was established to evaluate whether the price of drugs reflected value from the perspective of PBMs and health insurers. As ICER notes, it uses a "US health system perspective (i.e., focus on direct medical care costs)."
So when Pearson told MS patients today at an ICER meeting that the institute cannot quantify the benefits of new drugs to MS, it was just another evasion. In fact, ICER excludes the patient perspective because its mission to maximize the benefits to insurers and PBMs. Indeed, if spending exceeds that cap and therefore hurts the health system, ICER recommends changes in which MS patients would get medicines and how many would benefit. Of course none of these components of the ICER analysis were discussed. The same goes for the impact of limiting spending on each new drug to $915 million. The goal is to hide ICER's real face, which it shows to a fawning media and its PBM and insurer constituency.
Similarly, every time discussion turned to step therapy, how patients pay a share of the list price of a drug even as PBMs and insurers grab more rebates through price increases, Pearson steered the conversation in a different and self-serving direction. Instead, Pearson reminded everyone how drug prices rose and asserted that if MS drug prices had remained the same, then all of the medicines would be cost-effective.
Let's deal with this claim before turning to how ICERs value framework affects MS patients
To be sure, since 2011 the list price for Copaxone, Betaseron, Avonex, and Rebif have risen substantially to keep pace with the launch price of newer MS drugs in an apparent effort to maximize revenue as these injectable products lost market share. It also turns out that since 2011, rebates and discounts (which go to PBMs and insurers) were 60 percent of the price increase of these older products.
Further, since 2011 the primary driver in MS drug spending was the introduction of new medicines and greater use of oral MS medications. As the IMS study of drugs use notes: "Oral medicines now account for half of new treatment starts in 2015, steadily increasing since the introduction of these new treatment options six years ago and up from 26% in 2011."
But here too, rebates and discounts whittled down the actual increase in spending by about 30 percent according to my estimates based on Credit Suisse rebate data.
Further, even though ICER now attempts to calculate drug prices net of rebates, it is silent about the fact that these savings are pocketed by PBMs and insurers. (Indeed, Pearson is afraid to raise the issue.) And ICER is quiet about the fact even as payors rake in cash rebates that reduce the cost of medicines; they continue to charge patients up to 50 percent of the list price of medicines.
And price increases don't change the fact that If ICER had been in place 15 years ago, not one of the medicines used today would be considered cost effective at $150K per QALY
Between 2000-2015 the combined deficit in life years lost (those that would not be saved and the additional years lost) would have been 59000 with a loss of $17 billion in value.
Similarly, ICER claims not one MS drug developed since 2015 is cost effective. I estimate that between 2017-2022 limited use of these medicines would cost patients 11300 life years and $3.9 billion a year.
ICER’s public relations campaign to portray itself as the voice of the patient is deadly deception. It cannot be trusted to protect patients or fully include the value of new medicines to the people who are most in need of medical innovation.
In short, ICER’s policy prescriptions will cripple MS patients.
President Trump will ask prominent vaccine safety skeptic Robert F. Kennedy Jr. to lead a planned commission to study vaccine safety, Kennedy said Wednesday. Commission members will include "household names" who "have not taken a position on the issue" of vaccine safety, he said.
Kennedy said that in a Jan. 10 meeting, Trump told him he expected an "uproar" from the pharmaceutical industry concerning vaccines, and that the industry "would try to make him back down and he wouldn’t back down.” Immediately after the meeting, Kennedy told reporters that Trump had asked him to lead a vaccine safety commission, a claim Trump staff quickly denied. At a press conference Wednesday, Kennedy said he has since spoken with Trump staffers twice.
"They say they are still going forward with” a commission, he said. Kennedy spent much of the press conference repeating widely discredited theories about links between thimerosal in vaccines and neurological disorders in children. In fact, almost no pediatric vaccines contain the preservative, according to an FDA document.
Kennedy also vilified the CDC as a “cesspool of corruption,” and accused FDA, the drug industry, and the scientific and medical establishment of colluding to poison American children.
Actor Robert De Niro, who was also in attendance, said he "agreed 100%" with Kennedy’s comments. Kennedy and De Niro said they are not "anti-vaxxers," but they remained silent when Tony Muhammad, a representative of the Nation of Islam, told reporters that polio vaccines had caused 95 million cases of cancer in America.
Considering Mr. De Niro’s macho man video aimed at the President during the election, he’s come a long way – except that he hasn’t.
Shame. Shame. Shame.
How about a Presidential commission on how to educate the American public (and particularly the parents of young children) on the safety and urgency of vaccinations?