Playing the Spread

03/13/2020 08:40 AM | Peter Pitts

When it comes to drug pricing, some questions are easier to answer than others. Here’s an easy one – why do physicians prefer innovator biologics to biosimilars? A big part of the answer is --  because the more expensive the product, the more money they make. (Although Medicare pays the provider the same administration fee for a biosimilar and the originator biologic – that is not the case with commercial plans.) That’s Econ 101. As Adam Smith reminds us, “It is not from the benevolence of the butcher, the brewer, or the baker that we expect our dinner, but from their regard to their own self-interest.”

How can we make that “self-interest” work in the interests of lowering costs via a more robust use of biosimilars? (Hint: It’s not by making them more expensive.)
 
That’s the theory of the Increasing Access to Biosimilars Act. Its main idea is to develop a pilot program (run by the Department of Health and Human Services) to explore ways to encourage physicians to prescribe biosimilars within Medicare Part B. Since Medicare is the largest insurer in the country, any changes in its reimbursement policy will ultimately change behavior in the private insurer sector.

Better physician education as to the safety and efficacy of biosimilars? It’s in there. More education (properly scientific, balanced and vetted) is important. (This is also a legitimate focus of the joint FDA/FTC program to address the same issue.) More important, however, is the physician pocketbook strategy. Per this legislation, physicians would share in the savings. What does that mean? Simply put, it means doctors will receive a percentage of the spread between the cost of the innovator product and the biosimilar. De minimus, this will remove the perverse incentive for physicians to prescribe a more expensive product over its biosimilar cousin. Obviously, cost isn’t the only parameter in product choice – but it’s a potent counterweight to “their own self-interest.”

The proposed legislation also eradicates the need to pursue foolish policies such as the Administration’s concept of an International Pricing Index. Also, the Increasing Access to Biosimilars Act is a shot across the bow to payers who will swiftly recognize the incentives to stop using the tools of exclusionary contracting for non-Medicare Part B patients. Why? Because it will be in “their own self-interest” to do so.

More as more develops.
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Vaping Has Its Wakefield Moment

03/12/2020 03:37 PM |

Last July, the Journal of the American Heart Association (JAHA) published an article that claimed vaping doubled the risk of heart attacks.  The research, conducted by Stanton Glantz, director of the University of California, San Francisco’s Center for Tobacco Control Research and Education was unveiled right in the middle of a wave of articles, hearings and statements by public health officials asserting e-cigarettes were dangerous.  

The Glantz article fueled fear that vaping was deadly just as Andrew Wakefield’s 1998 Lancet study linking autism to the measles-mumps-rubella vaccine triggered anti-vaccine panic that in turn led to a decline immunization rates.  We now face the possibility of millions rejecting flu shots or a coronavirus vaccine because of the fears sowed by Wakefield. 

There’s something else both articles have in common.  They were both retracted by the journals that published them. The Lancet retracted the Wakefield paper in 2010 for being untrustworthy and fraudulent.  The Glantz article was yanked for similar reasons.

However, there is a big difference.  Wakefield was swiftly condemned after the retraction by the Food and Drug Administration (FDA), the National Institutes of Health (NIH), WHO and the Centers for Disease Control and Prevention. Today these same agencies are funding Glantz and relying upon his publications to shape public perceptions and regulation of e-cigarettes. In fact, Glantz  has received nearly $50 million from the Food and Drug Administration and the National Institute of Health to “inform the FDA’s regulation of the manufacture, distribution, and marketing of tobacco products to protect public health.”   

The JAHA article claimed that independent of other factors, e-cigarettes were as likely to increase the risk of heart attacks as smoking. When other public health researchers, including Dr. Brad Rodu, who chairs the Center for Tobacco Harm Reduction Research at University of Louisville analyzed the data Glantz used they found the majority of patients in the study who had heart attacks had them before they started vaping — by an average of 10 years earlier. 

In fact, one JAHA reviewer raised this issue with Glantz and asked him to redo the analysis by excluding people who had previously smoked or had a heart attack.  Glantz failed to do this simple calculation. Instead, he told JAHA editors that he controlled for previous heart attacks by analyzing only those people who had one five or fewer years ago. JAHA published the study and to its credit, pulled the paper after Rodu and other researchers continued to press the issue. 

This was not the first time Glantz used statistical gerrymandering to produce misleading results that aligned with aggressive anti-vaping forces.  In 2018 he published another study about e-cigarettes causing heart attacks in the American Journal of Preventive Medicine (AJPM) that has not been retracted.  Nor was it the last. In December of 2019  AJPM published a Glantz study that was hailed as the first study on the long-term health effects of electronic cigarettes finds that the devices are linked to an increased risk of chronic lung diseases

Indeed, Glantz doubled down on the link, telling the New York Post: "Everybody, including me, used to think e-cigarettes are like cigarettes but not as bad. …It turns out you're worse off. E-cigarettes pose unique risks in terms of lung disease." 

To reach this conclusion, Glantz again counted people who smoked as e-cigarette users.  Also, he didn’t control for the fact adults with asthma are 11 times more likely to develop other types of COPD (independent of smoking).   

Moreover, Glantz said it only took 3 years for e-cigarettes to trigger lung disease. That’s a remarkable finding for two reasons. First, it takes at least a decade or longer of consistent smoking for COPD to develop. Second, most clinical trials show that biomarkers of tobacco use associated with lung and heart disease decline when people switch to e-cigarettes.

The Glantz retraction raises questions not just about his other research, but the use of his research methods to influence public opinion and regulation of e-cigarettes.  As Aaron Wildavsky wrote, “when noble lies" are told in the belief that the system is so bad that any argument against it can only counteract a small part of its falsehoods, the task of the citizen is made much more difficult.”  In this case, it is now harder to determine whether restricting or eliminating e-cigarettes – which means leaving combustible tobacco products on sale, increase or reduce the 500,000 deaths a year attributed to smoking?  

After Wakefield’s fraud was exposed, the British Medical Journal wrote that “the damage to public health continues, fueled by unbalanced media reporting and an ineffective response from government, researchers, journals and the medical profession.”  Today, Glantz is still being published by mainstream medical journals, championed by major media outlets, given legitimacy by other scientists and politicians. Can we even imagine having the response to the coronavirus based on such fraudulent research?

The JAHA retraction was a Wakefield moment. Jacob Bronowski, the great science historian and mathematician observed that “no science is immune to the infection of politics and the corruption of power.”  The way to prevent such distortion is more people engaging in better science.  But when government funding supports one point of view, then it becomes easy to overrun scientific discourse, bully those with different points of view and control public policy.  The JAHA retraction can be a step back from that abyss. 

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Occam's Co-Pay

03/12/2020 01:55 PM | Peter Pitts

Here’s the headline:

“OptumRx and UnitedHealthcare are expanding their innovative consumer point-of-sale prescription drug discount programs to apply to all new employer-sponsored plans, making medications more affordable and improving health outcomes.”

Translation: Lower out-of-pocket costs result in higher medication adherence rates.  And this equals many good things – not the least of which is – better patient outcomes!

According to a new paper from the Progressive Policy Institute, Progressive Policy Institute on point of sale (POS) rebates:

* POS clarifies the true cost of prescription medications and allows consumers and physicians to make better cost-benefit trade-offs.

* POS reduces the incentive for companies to raise their list prices while offering bigger rebates to insurance companies and pharmacy benefit managers (PBMs).

* And, perhaps most important,  passing the manufacturer rebates through to consumers helps the high spenders, who would be able to take better advantage of discounts and rebates.

Per the OptumRx/UnitedHealthcare press release, “UnitedHealthcare’s fully insured members at the point-of-sale; consumers already seeing average savings of $130 per eligible prescription in 2019” and “Programs strengthen prescription drug adherence by up to 16%, lead to improved patient health.”

Bravo. But this welcome and appropriate bragging begs the question – why are they still pushing patient-punitive measures such as co-pay accumulators – the bleeding edge of the co-pay razor?
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High Science

03/07/2020 03:05 PM | Peter Pitts

Last week the FDA issued its most recent thinking on CBD.

Under the title, “FDA Advances Work Related to Cannabidiol Products with Focus on Protecting Public Health, Providing Market Clarity,” it was, to anyone paying attention, a regulatory finger-wag to a still-nascent industry that still just doesn’t get it. Alas, a lot of bad science doesn’t equal even a little good science. Sometimes the view from the front of the black helicopter gets boring.

The FDA statement begins, “Over the past year, the U.S. Food and Drug Administration has embarked on a comprehensive evaluation of cannabidiol (CBD) products, with a focus on educating the public about the risks and unknowns of these products, gathering the science needed to better understand both these safety concerns and potential benefits to inform our regulatory approach, as well as taking steps when necessary to address products that violate the law in ways that raise a variety of public health concerns.”

Translation: There’s no good science and we’re going to start writing enforcement letters.

The agency continues, “Today, we are providing updates on our efforts in this area, including several new steps in areas of education, research and enforcement with the ultimate goal of continuing to protect the public health and working to provide market clarity."

Translation: It’s going to be a slow process. We’re obviously going to have to help you. Ignore the need for real science at your own peril. Oh, about those claims, “… we are concerned that some people wrongly think that the myriad of CBD products on the market have been evaluated by the FDA and determined to be safe, or that using CBD ‘can’t hurt.’ Aside from one prescription drug approved to treat two rare, severe pediatric epilepsy disorders, no other CBD products have been evaluated or approved by the FDA.” Better lawyer up, bro.

Safety issues? “There may be risks that need to be considered before using CBD products outside of the monitored setting of a prescription from your health care provider.”

Translation: We think there may very well be safety issues such as:

“… potential liver injury, interactions with other drugs and male reproductive toxicity, as well as side effects such as drowsiness. In addition, there is still much we do not know about other potential risks. For example, other than the approved prescription drug, we know little about the potential effects of sustained and/or cumulative use of CBD, co-administration with other medicines, or the risks to vulnerable populations like children, pregnant and lactating women, the elderly, unborn children and certain animal populations. This does not mean that we know CBD is unsafe …”

Translation: … or if CBD is an antidote to COVID-19. But we have our suspicions.

“To address the questions and concerns we’ve already raised, we’re seeking reliable and high-quality data.”

Translation: We don’t have it because it doesn’t exist.

“This includes data on, among other things: the sedative effects of CBD; the impacts of long-term sustained or cumulative exposure to CBD; transdermal penetration and pharmacokinetics of CBD; the effect of different routes of CBD administration (e.g., oral, topical, inhaled) on its safety profile; the safety of CBD for use in pets and food-producing animals; and the processes by which “full spectrum” and “broad spectrum” hemp extracts are derived, what the content of such extracts is, and how these products may compare to CBD isolate products.”

Translation: You guys had better hire some pharmacologists.

“Given the importance of answering these questions, we’re exploring a number of ways to address the data gaps as quickly as possible. This includes encouraging, facilitating and initiating more research on CBD, providing venues for industry and researchers to share new data with the agency and identifying opportunities to further collaborate with our federal partners at Centers for Disease Control and Prevention, Substance Abuse and Mental Health Services Administration and National Institute on Drug Abuse on this important issue.”

Translation: We’re going to hold meetings!

But there is also good news:

“Importantly, the Agriculture Improvement Act of 2018 … has opened significant new opportunities for research, and as that body of research develops and grows, there will be considerably more information available. In particular, there’s been an increased interest in drug development from CBD and other compounds found in cannabis and we are working to support drug development as much as possible.”

Translation: Goodbye IPO dreams and hello CRO expenses and academic research grants that don’t care a brass farthing about marketplace needs and schedules.

Comrades, it’s going to be a long and winding road.

“In the coming days we are re-opening the public docket we established for our May 2019 public hearing. The docket provides a valuable conduit for submission of scientific data on CBD to the agency, so we have decided to extend the comment period indefinitely to allow the public to comment and to share relevant data with the agency. As the agency continues to move forward to explore viable pathways for CBD products outside the drug context, this extension will allow stakeholders to continue to provide relevant data as research in this area evolves.”

Translation: Key word, “indefinitely.”

And in conclusion:

“We recognize the significant public interest in CBD and we must work together with stakeholders and industry to develop high-quality data to close the substantial knowledge gaps about the science, safety and quality of many of these products. We are committed to working efficiently to further clarify our regulatory approach to these products – as always, using science as our guide and upholding our rigorous public health standards.”

Translation: Stop moaning and groaning, take off the tin foil hats and science-up.
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World Health Organization Worries More About Vaping Than the Coronavirus

02/09/2020 06:47 PM |

 

 
At the same time the World Health Organization (WHO) was dithering about the dangers of the coronavirus, it was perpetuating another public health threat of greater magnitude. 

Specifically, WHO took to Twitter to claim, in the middle of the epidemic that it was slow to recognize, that “e-cigarettes increase the risk of heart disease and lung disorders and pose significant risks as they can damage the growing fetus”.

These claims are false. People using non-combustible sources of nicotine have better lung function and fewer attacks. Quitting reduces death from lung and heart disease. In fact, "Quitting smoking before age 40, and preferably well before 40, gives back almost all of the decade of lost life from continued smoking."

As for damaging the growing fetus, pregnant women use to nicotine patches to stop smoking. In fact, compared to infants whose mother smoked infants born to women who used nicotine patches had higher rates of 'survival without developmental impairment’. Additionally, "nicotine patch replacement therapy also decreased the risk of prematurity and small for gestational age."

Indeed, while the WHO has bungled its response to the coronavirus, SARs, Avian Flu, Ebola, and tuberculosis outbreaks, it has waged an aggressive, unscientific campaign against e-cigarettes. It regards e-cigarettes not as less lethal alternative to smoking, but as a Trojan Horse built by tobacco companies to enslave billions more to nicotine. 

The WHO's latest act of fearmongering was prompted by the Phillip Morris Inc., UnSmoke Your Mind"campaign. The effort is designed to get people to stop smoking and providing people who can't quit a technology-based solution to reduce the harms of cigarettes. 

PMI developed and markets a smoke free product called IQOS which heats the tobacco without combusting, In application to the Food and Drug Administration, (which designated the product as a modified risk tobacco product) PMI demonstrated that lower temperature and lack of combustion reduces the levels of chemicals released compared to those released in cigarette smoke. 

As part of the Unsmoke initiative, PMI wants to get 40 million people who use cigarettes but are unlikely to quit smoking to switch to a smoke-free product. In fact, the IQOS is rapidly reducing cigarette sales in Japan, at the expense of PMI cigarette brands such as Marlboro. That's consistent with US data showing that a decline in smoking was 2-4 times fast after 2014 as vaping became more prevalent.  As for claims that people are trading one addiction for another, a recent randomized trial found that 18% of those assigned to use e-cigarettes were smoke-free after a year, compared with 9.9% of using other nicotine replacement therapies such as patches and gum. 

Yet the WHO claims the Unsmoke effort, along with the e-cigarette generated decline in cigarette smoking, is part of a well-funded covert Big Tobacco strategy that has the “goal of weakening tobacco control." In addition to peddling falsehoods about smoke free products, the WHO is also planning to push for vaping bans around the world this coming year.  To help lead the fight, the WHO’s agency for developing such proposals – the Framework Convention on Tobacco Control (FCTC) -- elected Iran (who the WHO may not realize is a state sponsor of terror) as its chair. Indeed, Iran and WHO appear to take the same approach to truth in their crusade against smoking: The Iranian Anti-Tobacco Association claimed that PMI is a Zionist company smuggled Marlboro cigarettes into Iran laced with pig blood and nuclear material. 

The WHO's sluggish response to epidemics and its fearmongering against e-cigarettes reflect the values and politics of the agency as well as the fact that it functions mainly as a launching pad for the pet agendas of donors and public health technocrats.  As an article in the Independent notes: "WHO has moved from being a global health ambassador to a pernickety lifestyle watcher, campaigning on subjects like sugar taxes, obesity and (via the Framework Convention on Tobacco Control) for a global ban on e-cigarettes, for plain cigarette packaging, and against the effects of smoking in films. At an anti-tobacco conference last year, it left itself open to accusations of censorship after banning the press."

Pandemics cause hundreds of thousands of deaths each year.  Nearly 8 million people die from smoking-related diseases in same time period. Telling people e-cigarettes are as lethal as smoking is like saying that vaccines being developed to prevent the coronavirus are as dangerous as the disease itself.  The WHO war of misinformation against vaping, much like its tardy response to the coronavirus, can be deadly.

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AARP Can Stop Rx Greed By Not Screwing Seniors

02/06/2020 12:20 PM |

In advance of the 2020 election, AARP has launched a Stop Rx Greed campaign that is demanding elected officials and candidates “crackdown on price gouging and the greedy practices that keep prices artificially high.”

That’s a noble objective.  But if AARP wants to crack down on such behavior, it should start with its own profiteering. 

AARP is a for-profit financial juggernaut with $4 billion in assets (including $365 million in cash) that generates nearly $1 billion a year in fees and royalties from marketing Medicare Part D prescription drug and health plans for United Healthcare.
  
 AARP- United Healthcare Medigap plan (insurance that covers certain drugs and other expenses not covered by the traditional Medicare program) currently serves 4.9 million seniors nationwide through various Medicare Supplement products in association with AARP. With 34 percent of the market, AARP is the largest Medigap insurer in the country. That’s three times the share of its closest competitor Mutual of Omaha.

Anyone who buys Medigap insurance knows,  premiums keep climbing.  Yet AARP doesn’t use its buying power to help offset such increases. Instead, nearly 60 percent of AARP’s total revenues— $940 million - comes from a 4.95% rebate paid to them by United Health Care and other insurance companies who license the AARP name to sell Medicare supplemental plans and other insurance products.  In fact, the 4.9% rebate is the single largest expense of United’s Medigap program and more than double the 1.85% profit that UnitedHealthcare makes on the insurance product.

Next, AARP also collects premium payments for United Healthcare.  It holds the payments for 31 days. During that time, AARP invests the money in real estate, hedge funds, bonds and stocks before transferring the money to United. All told, AARP collected $11.8 billion in premium dollars in 2018. It has used that money to build a portfolio of about $4 billion in net assets that generate about $300 million a year in cash, tax-free.

AARP also gets a flat fee for sponsoring United HealthCare  Medicare part D  and Medicare Advantage plans.  Both forms of insurance have the largest share of their respective markets. Such plans get cash rebates from drug companies in exchange for covering specific medicines. Those rebates don’t reduce the out of pocket cost of drugs patients take.  Indeed the biggest portion of rebates come from expensive new drugs used by people with life-threatening conditions such as cancer, autoimmune diseases, and HIV.   Patients taking such medicines don’t get to use rebates to reduce their spending.  Indeed, AAPR plans require them to pay up to 40 percent of the retail cost of such medicines.   

And the rebates don't reduce premiums either: While the Stop Rx Greed campaign claims drug prices are skyrocketing rebates reduced net brand drug prices by 52 percent between 2014-2019   Meanwhile, AARP Medicare Part D premiums jumped by 68 percent during the same time period. 

The campaign criticizes the rising price of insulin medications.  But AARP plans do not cover Basaglar a less expensive biosimilar version of brand-name insulin’s, Lantus.  The retail price of Lantus has increased, but the net price has fallen due to rebates, which the AARP part D plans pocket.  It covered Neulasta, a drug that boosts the immune system of cancer patients, that retails for $6100 a month but not biosimilars  Udenyca and Fulphila which retail for $4100. 

Even when they cover generic versions of high-priced brand drugs,  AARP plans still have seniors pay up to a third of the retail price of medicines. AARP plans cover a brand drug for  versions hepatitis C called Epclusa as well as its generic formulation. It turns out that the post-rebate prices of these drugs are about the same.  As a result, AARP plans will maximize the spread between list and net prices even when generics are available.

You would think that AARP and its campaign support passing rebates dollars to patients.  Think again.  AARP aggressively lobbied against government regulations that would have required part D plans to use rebates to reduce out of pocket costs.  Then again, AARP makes billions from plans that profit by forcing the sickest patients to pay the most. Perhaps before campaigning against price gouging, AARP should first stop screwing the consumers they claim to represent.   



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Follow the Money

01/14/2020 07:50 PM | Peter Pitts

Nearly 50% of Brand Medicine Spending Goes to the Supply Chain and Others

Nearly half of total spending on brand medicines – the sum of all payments made at the pharmacy or paid on a claim to a health care provider – went to the supply chain and other entities in 2018, according to a new analysis from the Berkeley Research Group (BRG). This transformative research shines a spotlight on the misaligned incentives in the supply chain and underscores the need to fix the rebate system.

BRG found that hospitals, health insurers, pharmacy benefit managers, the government and others got nearly 50% of what was spent on brand medicines in 2018, up from 33% five years prior. By contrast, innovative biopharmaceutical companies that research, develop and manufacture medicines retained just 54% of total point-of-sale spending on brand medicines.

According to the analysis, the share of total spending on brand medicines that biopharmaceutical companies retain has been steadily declining as rebates and discounts have increased. Between 2015 and 2018, the amount innovative biopharmaceutical companies retained from the sale of brand medicines increased, on average, 2.6% annually, in line with inflation. In this same timeframe, companies brought nearly 200 new innovative treatments and cures to patients.

Meanwhile, nearly half of the increase in the total amount spent on brand medicines went back to payers during this same time period. And 20% of the overall increase went to hospitals, pharmacies and other health care providers, which is the same amount that went to biopharmaceutical companies that research, develop and manufacture medicines.
The amount hospitals, pharmacies and other health care providers retained on the sale of brand medicines nearly doubled between 2013 and 2018, increasing from $24.7 billion to $48.6 billion. This trend was primarily driven by unprecedented expansion in the 340B drug pricing program. In fact, the amount hospitals and other 340B entities retained from the sale of brand medicines purchased through the 340B program was 9 times larger in 2018 than in 2013.
We are committed to ensuring patients benefit from significant discounts and rebates at the pharmacy counter, and this analysis reaffirms the need to look at the entire supply chain to fix misaligned incentives. We must work to fix the broken rebate system, as well as programs like 340B, to lower out-of-pocket costs and solve patient affordability challenges.

The full study from BRG can be viewed here.
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"Fixing" the FDA

01/13/2020 09:36 AM | Peter Pitts

Per, “How to Fix the Troubled FDA” (NYT, January 12, 2020)

Is the FDA approving drugs too fast or not fast enough? Are they demanding too much data or not enough? There isn’t any dearth of commentary supporting either proposition. There is, however, no evidence to support the sound bite that the FDA is approving “everything,” or that every product that requests an expedited pathway receives it, or that “all” those that do receive an expedited pathway designation get approved, or that every product that does reach the market via an expedited approval is in some way more dangerous than other medicines.

· An analysis of every product (364) requesting a Breakthrough Therapy designation (from July 2012 – June 2016) shows that CDER granted 133 (37%) of those requests, denied 182 (50%), and the sponsor withdrew their request 49 times (13%) before the agency made a decision.[i] Hardly a regulatory carte blanche.

·  In 2013, the first full year of the Breakthrough Designation, the FDA approved 3 new drugs, 14 in 2014, and 9 in 2015.[ii] Hardly an onslaught of new medicines.

· Among 22 drugs with 24 indications granted accelerated approval by the FDA in 2009-2013, efficacy was often confirmed in post-approval trials a minimum of 3 years after approval, although confirmatory trials and preapproval trials had similar design elements, including reliance on surrogate measures as outcomes.[iii]

New science and the strategies and tactics to incorporate them into regulatory thinking does not mean a free pass for bad science. The FDA must be an innovation accelerator with a recalibrated sense of regulatory velocity (Speed + Accuracy + Public Health Need). It’s the agency’s next step toward a more appropriate and entrepreneurial regulatory attitude
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Newsom's idea on generic drugs = poor staff work

01/10/2020 09:44 AM | Peter Pitts

Governor Newsom’s idea to have California “white label” its own generic drugs is deeply flawed. It’s not as easy as simply finding a factory. Anyone manufacturing and selling a specific generic drug must first have approval from the FDA to do so. That’s not an inexpensive, swift or easy proposition. Also, generic prices in the United States are the lowest in the western world. Third, prices are based on volume. If the Governor of our nation’s most populous state wants to lower prices, he should follow the money. The middlemen of the pharmaceutical industry – Prescription Benefit Managers (PBMs) add tremendous costs to the system while providing little actual benefit. If the Governor can’t  disintermediate the middleman and offer generics at lower costs, he should demand that the PBMs hired by the state cut their fees so that the people of California can enjoy lower prices.
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Glantz In, Garbage Out

12/23/2019 01:46 PM |

Stanton Glantz is the Distinguished Professor of Tobacco Control, and director of the Center for Tobacco Control Research and Education at the University of California, San Francisco (UCSF) School of Medicine.  Glantz is also the Andrew Wakefield of the anti-vaping movement.   In keeping with that awesome responsibility, the Distinguished Professor has come out with another retraction worthy analysis claiming non-combustible nicotine devices are more dangerous than cigarettes.  

This time his research paper in the  American Journal of Preventive Medicine is, as NBC News puts it: is "the first study on the long-term health effects of electronic cigarettes finds that the devices are linked to an increased risk of chronic lung diseases." (Chronic obstructive pulmonary disorder or COPD, emphysema, asthma, and chronic bronchitis.)

As NBC reports: "The study included 32,000 adults in the U.S. None had any signs of lung disease when the study began in 2013. By 2016, investigators found people who used e-cigarettes were 30 percent more likely to have developed chronic lung disease, including asthma, bronchitis and emphysema, than nonusers."

Having some who his healthy getting lung disease less than three years after vaping is pretty remarkable. But Glantz told the New York Post:

"E-cigarette use predicted the development of lung disease over a very short period of time. It only took three years."

In fact, Glantz asserts: "Everybody, including me, used to think e-cigarettes are like cigarettes but not as bad. If you substitute a few e-cigarettes for cigarettes, you're probably better off…It turns out you're worse off. E-cigarettes pose unique risks in terms of lung disease."

The conclusion that short term vaping causes lung disease is biologically implausible. 
COPD and other severe lung diseases often take years to develop and often escape diagnosis until they are relatively advanced.
As many as 1 out of 4 Americans with COPD never smoked cigarettes. In fact, "never smokers account for 23% of the total COPD burden. Among these obstructed never-smokers, 19% reported a prior diagnosis of asthma alone, and 12.5% reported COPD (solely or with asthma), leaving 68.5% with no prior respiratory diagnosis. "
 
And even among smokers, it takes at least a decade or longer of consistent smoking for COPD to develop. Most studies (ignored by Glantz) conclude that "prolonged tobacco use is associated with respiratory symptoms and COPD after controlling for current smoking behavior."

For example a longitudinal study examining the risk of developing COPD in a general population found that after 25 years of smoking, at least 25% of smokers without initial disease will have clinically significant COPD and 30–40% will have any COPD.

Moreover, there is no clinical evidence that COPD rapidly emerges. On the contrary, the most recent research suggests that disease severity (degree of impairment) should be distinguished from disease activity (rate of progression) since there is no one factor that triggers either. So unless Glantz has discovered a novel biological mechanism triggered by vaping, his claims are absurd.

The same goes for asthma, chronic bronchitis and emphysema. A large percentage of people have conditions and often go undiagnosed for years. Indeed, Glantz when he claims that the population studied didn't have lung disease at the outset, he is being deceptive. They didn't have a diagnosis in the past 12 months. That's different than not having it all. 

In fact, Glantz fails to compare the prevalence of these diseases in vapers vs the rest of the population. That's because adults who had asthma have an 11 times greater risk of COPD (independent of smoking) than those that don't. Glantz doesn't bother controlling for this important factor.
  
 
   
Age-specific and age-adjusted* percentage of adults aged ≥18 years with COPD,  Behavioral Risk Factor Surveillance System, 2017
Ever had asthma Current Smokers      Former Smokers
                                           Yes  19.5                             11.2
                                          No    4.1                                 1.6
 Source: Wheaton AG, Liu Y, Croft JB, et al. Chronic Obstructive Pulmonary Disease and Smoking Status — United States, 2017. MMWR Morb Mortal Wkly Rep 2019;68:533–538. http://dx.doi.org/10.15585/mmwr.mm6824a

                            
 There's more. Glantz did not control for other smoking characteristics that matter (dual users are heavier smokers). As Peter Hajek, a professor of clinical psychology and the director of the Wolfson Institute of Preventive Medicine's Tobacco Dependence Research Unit at Queen Mary University of London pointed out in a email to me: “He didn't compare the relationship of vaping with other products and approaches for reducing smoking (such as counseling, patches, and medications) on lung disease. That data is available too.”

In addition to these flawed assumptions and approaches, Glantz still refuses to show whether other variables or factors that he doesn’t analyze can explain the relationship he claims to show.  Anyone making a claim about causality has a duty to find and disclose all the relevant factors just as a district attorney is required not to hide evidence to convict a defendant.  As Chelsea Boyd at R Street wrote to me: (Glantz) “does not show a full interaction analysis, likely because it would remove the association between e-cigarettes and his disease du jour. It's also telling that some of his associations don't make any sense if you consider the larger context.” 

Indeed, his effort to discount the possibility of having lung disease might cause someone to try vaping backfires. Glantz notes, "this study assessed the possibility of reverse causality by estimating the odds of initiating e-cigarette use... combined as a function of having respiratory disease among people who had never used e-cigarettes (previously).” In fact, that analysis shows, as the article states, that "having respiratory disease at significantly predicted future e-cigarette use (p<0.001),”  Incredibly Glantz claims the opposite is true.

Finally, this is NOT the first study to look at the long-term effect of vaping on lung disease. There are others, and unlike Glantz, they actually study real people over time. 

One recent study found that e-cigarette (ECs) "use may aid smokers with COPD reduce their cigarette consumption or remain abstinent, which results in marked improvements in annual exacerbation rate as well as subjective and objective COPD outcomes." That was followed by another analysis in 2018 that concluded, "EC use may ameliorate objective and subjective COPD outcomes and that the benefits gained may persist long-term. EC use may reverse some of the harm resulting from tobacco smoking in COPD patients.

Meanwhile, as they did in using Wakefield’s bogus studies claiming the measles vaccine caused autism, the incurious media now claim that Glantz’ research "adds to a growing body of evidence that vaping can cause physical harm, whether it's chemical burns to lung tissue, toxic metals that leave lasting scars on lungs, vitamin E oil that clogs lungs or even overheated batteries that explode.

Whether any of that evidence can be used for something other than fearmongering is a different question. As the saying goes: Glantz in, garbage out. Or something like that. 

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The Twelve days of Rxmas

12/21/2019 01:41 PM | Peter Pitts

The Twelve Days of Rxmas

On the first day of Rxmas
The folks at Drugwonks gave to me
An Adcomm adjourning at 3:00.

On the second day of Rxmas
The folks at Drugwonks gave to me
Two Warning Letters
And an AdComm adjourning at 3:00.

On the third Day of Rxmas
The folks at Drugwonks gave to me
Three Draft Guidances
Two Warning Letters
And an Adcomm adjourning at 3:00.

On the fourth day of Rxmas
The folks at Drugwonks gave to me
Four Calling Sponsors
Three Draft Guidances
Two Warning Letters
And an AdComm adjourning at 3:00.

On the fifth day of Rxmas
The folks at Drugwonks gave to me
Five Generic Lawsuits
Four Calling Sponsors
Three Draft Guidances
Two Warning Letters
And an AdComm adjourning at 3:00.

On the sixth day of Rxmas
The folks at Drugwonks gave to me
Six Hemp States Baying
Five Generic Lawsuits
Four Calling Sponsors
Three Draft Guidances
Two Warning Letters
And an AdComm adjourning at 3:00.

On the seventh day of Rxmas
The folks at Drugwonks gave to me
Seven Gottliebs Tweeting
Six Hemp States Baying
Five Generic Lawsuits
Four Calling Sponsors
Three Draft Guidances
Two Warning Letters
And an AdComm adjourning at 3:00.

On the eighth day of Rxmas
The folks at Drugwonks gave to me
Eight Divisions Dividing
Seven Gottliebs Tweeting
Six Hemp States Baying
Five Generic Lawsuits
Four Calling Sponsors
Three Draft Guidances
Two Warning Letters
And an AdComm adjourning at 3:00.

On the ninth day of Rxmas
The folks at Drugwonks gave to me
Nine Pharmas Dancing
Eight Divisions Dividing
Seven Gottliebs Tweeting
Six Hemp States Baying
Five Generic Lawsuits
Four Calling Sponsors
Three Draft Guidances
Two Warning Letters
And an AdComm adjourning at 3:00.

On the tenth day of Rxmas
The folks at Drugwonks gave to me
Ten reviewers bleeping
Nine Pharmas Dancing
Eight Divisions Dividing
Seven Gottliebs Tweeting
Six Hemp States Baying
Five Generic Lawsuits
Four Calling Sponsors
Three Draft Guidances
Two Warning Letters
And an AdComm adjourning at 3:00.

On the eleventh day of Rxmas
The folks at Drugwonks gave to me
Eleven Vapers Vaping
Ten reviewers bleeping
Nine Pharmas Dancing
Eight Divisions Dividing
Seven Gottliebs Tweeting
Six Hemp States Baying
Five Generic Lawsuits
Four Calling Sponsors
Three Draft Guidances
Two Warning Letters
And an AdComm adjourning at 3:00.

On the twelfth day of Rxmas
The folks at Drugwonks gave to me
Twelve hires pending
Eleven Vapers Vaping
Ten reviewers bleeping
Nine Pharmas Dancing
Eight Divisions Dividing
Seven Gottliebs Tweeting
Six Hemp States Baying
Five Generic Lawsuits
Four Calling Sponsors
Three Draft Guidances
Two Warning Letters

And an AdComm adjourning at 3:00.


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Are Profits Compounding the Public Health Danger of Compounded Drugs?

10/08/2019 11:35 AM |

Last week, federal prosecutors in Los Angeles arrested 53 people for raking in $150 million in Medicare and Medicaid payments for medically unnecessary compounded drugs. In cooperation with a large L.A. based compounding pharmacy --Fusion Rx  -- the doctors to bill health care providers for those compounded drugs, many of which were reimbursed at rates much higher than common medications. 
 
Doing this enabled the pharmacies to boost the reimbursement rates for the prescriptions and routinely waive patient copayment obligations.  FusionRx is one of many compounding pharmacies that are endangering patient lives by driving up the cost of prescription drugs.
 
It's no surprise that few people are familiar with compounding pharmacies, purely because the altered medications account for less than one percent of prescriptions. However, as the DOJ suit notes, the price of compounded drugs has surged by as much as 3,400 percent since 2006.  Despite legislative efforts to strengthen oversight of compounding pharmacies, their production and distribution are still mostly unregulated. This is not the time to ease up on federal oversight of compounding drugs. 
 
Compounding – the combining of two or more ingredients to produce a medicine for a patient – has been around for centuries, evolving from age-old concoctions into a service that increasingly customizes lifesaving medicines. Most drugs were compounded until large-scale development and manufacturing emerged in the 1950s, allowing companies to bolster their research and development to provide tailored treatments for patients.   
 
Over the past decade, however, there has been a substantial increase in compounding medications to address the need to customize dosing for patients who would otherwise not benefit from specific treatments. Small compounding pharmacies don't have to get Food and Drug Administration (FDA) approval for their medicines. This is because Congress exempted compounding pharmacies from FDA oversight in 1998 and handed the responsibility over to state pharmacy boards. 
 
Traditionally, the bespoke nature of compounding has allowed pharmacies to charge health plans a premium for their products. While no public estimates are available, industry experts believe that the gross margins from compounding products is between 70-87 percent. As the pharmacy benefit management market consolidated around large companies such as Express Scripts and CVS, small specialty drug firms saw their margins squeezed. Compounding provided the opportunity to make more money due to the markups involved, and as a small portion of drug spending, most PBMs and health plans paid for compounded medicines without question. 
 
Soon it became clear that in the absence of FDA regulation, a compounding pharmacy could produce large commercial quantities of medicines and ship them anywhere in the United States. Around 2010, private equity groups invested in several compounding pharmacies to increase capacity. 
 
It didn't take long for small compounding companies to become large companies, pumping out mass quantities of medicines originally meant for single patients. As noted, Medicare Part D spending skyrocketed for topical compounded drugs-such as creams, gels, and ointments to relieve pain. Most of the increase in spending is a result of expenditures for pain medications, including opioid-based medicines and steroids. 
In 2004, TRICARE – the Department of Defense's health system – spent approximately $5 million for compounded prescriptions. By 2010, the cost had risen to $23 million. In the first nine months of 2015, TRICARE paid $1.7 billion for compounded drugs. Shockingly, this is over 20 percent of TRICARE's total prescription budget. The average cost of a compounded drug is now $2,595, with some drugs costing as much as $40,000 per prescription.
 
As noted, FusionRx is just one of several criminal cases the DOJ is pursuing against compounder.  For example,  the owners of Parks and Lee compounding pharmacy are accused of paying kickbacks and bribes to physicians to get them to write prescriptions to their pharmacies. After the prescriptions were written and carried out, the defendants allegedly submitted huge claims for payment to federal health care programs – dividing the profits.
  
In 2013, The Drug Quality and Security Act (H.R. 3204) was passed by the Senate and signed into law by President Obama on November 27, 2013. Since that time, the FDA has made significant steps for heightened enforcement and site inspections of compounders, shutting some facilities down and helping others correct public health threats. The 2013 Act gave the FDA control over the quantity of compounded drugs that could be shipped or sold across state lines.  Proposed regulations for doing so were not finalized until May of this year. 
 
The proposed regulations continue to exempt compounded drug products from demonstrating to the FDA that they are safely and effectively manufactured.  However, the compounder must agree to limit intrastate distribution to 50 percent of total prescriptions or less. If not, then the amount of out of state sales drops to 5 percent. 
 
The compounding industry opposes these generous limits and declares them as anti-patient. But, as the FDA notes, Congress did not intend for compounding pharmacies to grow into conventional manufacturing operations that can make and sell large quantities of unregulated medicines. 
 
Though steps have been taken to instigate proper oversight, compounded drugs are still laced with containments and corruption. The FDA's proposed regulations increase transparency and accountability. The failure to adopt these modest requirements suggests that compounding pharmacies are more interested in their profits than in public health.  Read More & Comment...

New House Drug Pricing Bill is a Joke

09/10/2019 08:18 AM | Peter Pitts

In my new book, “Common Sense Health Policy for Common Sense Americans (and Presidential Candidates),"  I plead for sanity and stress that it’s time to put away unworkable soundbite solutions. I sent copies to every member of Congress. Reviewing H.R. 3 (the new price negotiation bill), it’s clear that no one (at least on the majority side) has read it.

The bill is a hit parade of bad ideas.  For example:

An International Pricing Index. Patients often lose access to the best medicines when their government adopts price controls. Of the drugs launched in the last seven years, only 60% were available in Sweden. And only half made it to patients in Canada. In the United States, meanwhile, nearly 90% of those medicines were available. Americans will no longer enjoy generous access to the newest drugs if we embrace price controls. Importing the socialist pricing tactics of foreign governments is no way to stand up for Medicare patients. Bad idea when it comes from the White House, Bad idea when it comes from the People’s House.

Direct Government Negotiation. Is the direct federal negotiation of drug prices a good idea? Consider the “non-interference clause” that currently prohibits such actions in Medicare Part D — the federal program that subsidizes prescription drugs for seniors. A repeal of the non-interference clause would result in a sharp increase in Medicare drug prices and a substantial decline in patient choice. The Congressional Budget Office observed that Part D plans have “secured rebates somewhat larger than the average rebates observed in commercial health plans.” According to the CBO, to achieve any significant savings, the government would have to follow through on its threats of “not allowing [certain] drug[s] to be prescribed.” In other words, the government would drop some drugs from Medicare’s coverage to save money. That would be a raw deal for patients. The average Part D plan provides access to more than 95 percent of the top 200 Medicare Part D Drugs. (PS/ The Non-Interference Clause was written by Senators Ted Kennedy and Tom Daschle.)

Rebates to Off-Set Price Hikes. When Americans say, “My drugs are too expensive,” what they generally mean is that their co-pays at the pharmacy are too expensive. And they’re right. But co-pays aren’t tied to list prices. Consider this: payers negotiate discounts of between 30-50% of the list price – and then base the co-pay off of the list price. What happens to the discount? They pocket the difference. When payers say that higher co-pays are a result of higher list prices they are lying. Surprisingly absent from H.R. 3 is any call for pricing transparency. Shameful.

H.R. 3 is a cruel  joke. It's time to put down the political talking points and pick up Common Sense Healthcare Policy.
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"Disrespect" is in th eyes of the CRL beholder

08/28/2019 01:31 PM | Peter Pitts

Some snippets from the Pink Sheet …

Sarepta, US FDA Offer Sparring Positions Over Release of Complete Response Letters

Following Vyondys 53 rebuff, Sarepta CEO contends it would be "disrespectful" to release the complete response letter, while FDA contends that there is nothing stopping companies from publishing the letters.

During a webinar conversation between Sarepta and Parent Project Muscular Dystrophy (PPMD) released on 22 August, company CEO Doug Ingram said multiple times that it might be "disrespectful" for Sarepta to publish the CRL.

"These complete response letters are not public," Ingram said. "They're non-public letters between the agency and sponsors. I am not comfortable making the complete response letter public for the simple reason that I think it might look disrespectful to the agency as we are working through these issues."

An FDA spokesperson, however, told the Pink Sheet that there is nothing stopping companies from making their CRLs public. "Applicants can release the CR letter it receives from FDA (or any other correspondence it gets from us)," the spokesperson said.

A former FDAer chided the company's assertion that it would be disrespectful to the FDA by releasing the CRL. "Any company that is doing business with the FDA understands what is and what is not commercial confidential," Peter Pitts, president of the Center for Medicine in the Public Interest and a former FDA associate commissioner, told the Pink Sheet. "Whether the company chooses to release a CRL is their decision. But to say it is disrespectful is just living in fantasy land. It's just another way to blame the FDA."

The full Pink Sheet article can be found here.
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A Serious Proposal

08/01/2019 03:27 PM | Peter Pitts

Kudos to President Trump, HHS Secretary Alex Azar and Acting FDA Commissioner Ned Sharpless for taking the idea of drug importation from the absurd to the serious. Moving away from empty political talking points to a real regulatory agenda takes guts – especially since it has the very real possible outcome of demonstrating once and for all that this idea has no merit.

The “Safe Importation Action Plan” posits two pathways. Under the first, states, wholesalers, or pharmacists could submit plans for demonstration projects for HHS to review outlining how they would import Health-Canada approved drugs that are in compliance with section 505 of the FD&C Act. The importation would occur in a manner that adequately assures the drug is what it purports to be and that meets the cost requirements of the rulemaking. The demonstration projects would be time-limited and require regular reporting to ensure safety and cost conditions are being met. Controlled substances, biological products, infused drugs, intravenously injected drugs, drugs inhaled during surgery, and certain parenteral drugs would be excluded from this pathway. (Yes, that means no Canadian insulin.)

The NPRM would explain the requirement for demonstrating that drugs imported under this pathway must result in a significant reduction in the cost of covered drug products to the American consumer. As such, the NPRM would seek  feedback on the best way to identify the expected acquisition cost of the imported drug, the cost of assuring the drug is safely imported, and the mechanism for delivering those savings to the consumer (as opposed to the savings being absorbed by the supply chain). That’s a tough assignment. Will such a plan lower co-pays for a single patient with health insurance?

Pathway 2 would allow manufacturers of FDA-approved drug products to import versions of these FDA-approved drugs that they sell in foreign countries into the US. To use this pathway, the manufacturer or person authorized by the manufacturer would need to establish with FDA that the foreign version is the same as the U.S. version (such as through manufacturing records). If this condition is met, FDA would allow the drug to be labeled for sale in the US (potentially with labeling that identifies the product as originally manufactured for sale abroad) and imported pursuant to section 801(d) of the FD&C Act under the existing approval for the US approved version.

What’s missing from the Administration’s action plan is a bilateral meeting to discuss drug importation from Canada – with the Canadian government. “Canada does not support actions that could adversely affect the supply of prescription drugs in Canada and potentially raise costs of prescription drugs for Canadians,” reads an April briefing for Canadian officials obtained under freedom of information laws.  No plan can ever be taken seriously without this essential cross-border conversation.

Pathways 1 and 2 will both be driven by a Notice of Proposed Rulemaking (“NPRM”). It’s a detailed and lengthy process –precisely the opposite of loose political rhetoric. It’s about time we get serious and base our future discussions about drug importation on facts rather than fiction.
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American Hospital Association and AHIP: Price Transparency for Thee Not Me

07/30/2019 01:56 PM |

It appears that the American Hospital Association  (AHA) and the health plan lobby, America's Health Insurance Plans (AHIP) are not fans of the Trump administration's executive order to require hospitals to " publish prices that reflect what people pay for services."  According to HHS Secretary Alex Azar, the rule would "require hospitals to disclose the prices that patients and insurers actually pay in "an easy-to-read, patient-friendly format"  and "require health care providers and insurers to provide patients with information about the out-of-pocket costs they'll face before they receive health care services." 

AHA responded: (Consumers) "don’t look at price alone when it comes to seeking the highest quality care for themselves or loved ones. Moreover, consumers say they are most interested in what their out-of-pocket costs for care will be, what is covered by their health plan, which providers are in their networks and what their health plan’s cost-sharing obligations are in terms of their deductible and coinsurance. 
 Meanwhile, economists and analysts have suggested that publicly posting certain information, such as privately negotiated rates, could, in fact, undermine the competitive forces of private market dynamics with unintended consequences such as insurers coordinating to disadvantage providers and consumers."

AHIP   CEO Matt Eyles said the same thing, claiming that  "Publicly disclosing competitively negotiated, proprietary rates will reduce competition and push prices higher — not lower — for consumers, patients, and taxpayers," said in a statement. He says it will perpetuate "the old days of the American health care system paying for volume over value. We know that is a formula for higher costs and worse care for everyone."

Wow, it sounds like the Trump proposal would violate the economics equivalent of the third law of thermodynamics.  So it must apply across the board, to any industry and therefore AHIP and AHA would take a principled stand against any proposal to reveal negotiated prices.  Or maybe not:

AHIP and USA Today Agree: It’s Time for Open and Honest Drug Pricing

When it comes to out-of-control drug prices, the USA Today Editorial Board gets it right. (“How the Trump prescription for drug prices transparency could make health care well again,” May 16). Disclosing prescription drug prices in direct-to-consumer (DTC) advertising is an effective way to help patients make informed decisions about their health.
Lifesaving drugs and treatments are placed out of reach for too many Americans because of the outrageously high list prices set exclusively by Big Pharma. The Trump administration’s proposal will help consumers learn more about what their prescription costs before they access it, and will empower them to discuss cheaper alternatives with their doctor, including generics.
Americans deserve to know how high prices are fueling Big Pharma’s marketing machines and bottom lines at our expense. Direct-to-consumer advertising price disclosure is an important first step in helping us get there.

Matt Eyles
President and CEO
America’s Health Insurance Plans

Well, okay.  But maybe the AHA has a more principled position:

Committee approves AHA-supported drug price transparency bill

The House Ways and Means Committee on Tuesday approved the Prescription Drug Sunshine, Transparency, Accountability and Reporting Act (H.R. 2113), AHA-supported legislation that would increase transparency with regard to prescription drug pricing. 
  
"As it considers this legislation, we want to commend the Committee for including several policies that will better hold drug manufacturers accountable," AHA said in a letter of support for the bill. 
  
Specifically, AHA applauded the inclusion of the Reporting Accurate Drug Prices Act, which would require all manufacturers to submit pricing data to the Department of Health and Human Services.

Sp do AHA and AHIP agree with PhRma that publishing sticker prices "would potentially confuse patients who might be misled into believing the list price is the price they would pay and would potentially deter them from seeking needed medical care?"

Or more to the point: hey support it when it benefits them and oppose transparency when it hurts their industries.

The line between self-interest and hypocrisy in politics is thin.  For AHIP and AHA it is non-existent. 


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ICER Recommending Eugenics for Kids With Duchenne Muscular Dystrophy

07/25/2019 12:59 PM |

ICER claims :” Unfortunately, there is no persuasive evidence that [ Exondys and other treatments for Duchenne Muscular Dystrophy (DMD) ] improve outcomes that matter to patients, including functional status, quality of life, or length of life. Eteplirsen has been on the market for three years and yet we still found notably inadequate data on patient outcomes.”

But under the ICER model, no treatment for Duchenne Muscular Dystrophy would be considered valuable under that standard because none of the existing treatments had clinical trial data to support use.  ICER is engaged in hypocritical data manipulation:


1.    ICER claims there is no evidence that compared to ventilation that Exondys is improving outcomes.  Yet, there was never any prospective study of ventilation. So according to ICER’s methods, there is no persuasive evidence that ventilation improves outcomes that matter to patients. 
2.    Further, not one study demonstrated that ventilation meets ICER’s 100K per QALY threshold.  Indeed, one study found that mechanical ventilation users had 16.4 hrs per day of licensed practical nurse/ registered nurse care costing $269,370 per year.   Yet non-invasive ventilation, which has replaced mechanical or conventional non-variable ventilation (CNVS) which is less expensive would have been denied by ICER. 
3.    If the ICER standard has been applied to non-invasive ventilation, then tracheotomy would still be the preferred treatment.  Yet, without CNVS these patients would either still be in iron lungs around-the-clock or, more likely, would have undergone tracheotomies and by now be dead from complications related to the tube since four out of five patients with some NMDs using tracheostomy
4.    In fact, current treatments are also not cost-effective because they prolong life and add to the total cost of care.  Indeed, a recent study concludes that “DMD should be now considered an adulthood disease as well, and as a consequence, more public health interventions are needed to support these patients and their families as they pass from childhood into adult age.”
5.    That is, death is the preferred outcome if you only care about saving money:“(E)xtending life for a patient with DMD by 1 year would be associated with a QALY gain of 0.18 at a cost of $54,000 (not accounting for the cost of the evaluated intervention), resulting in an incremental cost-effectiveness ratio of $300,000, well above “ the ICER cut-off. 
6.    Further, the main cause of death in DMD in our population remains cardio-respiratory failure.  People with DMD are more likely to die if they have poor respiratory profiles and elevated cardiac biomarkers. Collectively, these factors highlight a high-risk cardiovascular population with a worse prognosis.  
7.    However, other technologies that would extend life that ICER compares to Exondys are also NOT cost-effective. For example, the incremental cost-effectiveness ratio (ICER) for destination ventricular assist device therapy (DT-VAD) was $179,086 per quality-adjusted life-year (QALY).


The documented delay in loss of lung and heart function from Exondys exceeds that of ventilation.  Moreover, the use of Exondys is likely to help reverse weight loss, also a risk factor for death, by allowing people to swallow food safely.  

Hence, every treatment for DMD between the 1980s and today has increased life expectancy and improve quality of life.  Yet NONE meet ICER thresholds.  ICER would have deprived tens of thousands of patients longer, better lives.  

This assumption, that people who, because of genetic conditions, are likely to be sicker and more likely to require medical care as they live longer is shaped by the same impulse justifying eugenics at the turn of the century. 

Here is the statement of ICER president Steve Pearson “In practice, however, a sickest-first principle might require allocation of resources even when only minor gains can be achieved and the cost is very high, which is obviously inefficient…coverage decisions must not only incorporate consideration of the benefits gained but the opportunity costs incurred when covering expensive orphan drugs.”

And here is the statement of Margaret Sanger, a eugenics proponent: 

"Every single case of inherited defect, every malformed child, every congenitally tainted human being brought into this world is of infinite importance to that poor individual; but it is of scarcely less importance to the rest of us and to all of our children who must pay in one way or another for these biological and racial mistakes."

Limiting the use of new medicines that prolong and improve the lives of people with DMD to save money is eugenics by another name.  And that name is ICER. 
   Read More & Comment...

How the Media Enables ICER's Evil Agenda

07/25/2019 11:34 AM |

Today ICER is holding a public discussion about its assertion that “there is no persuasive evidence that... “Exondys 51, a treatment from Duchenne Muscular Dystrophy exon-skipping  improve outcomes 
that matter to patients, including functional status, quality of life, or length of life.”

The media coverage of this event will, if past reporting is any indication, ignore the science, ignore the perspectives and courage of the patients who have benefited from Exondys, who have found the medicine to be a source of freedom and hope.  Instead, it will focus on the fact that patient groups receive money from companies who have developed such medicines. 

In doing so, such journalists are seeking to marginalize the plight of people with rare, degenerative conditions, tarring them as mere tools of the evil Big Pharma while exalting groups such as ICER, ally the while ignoring the massive amount of funding ICER receives from former Enron trader, John Arnold.  Indeed, some journalists are being paid from money John Arnold has given their publications.  

The demonization of small patient groups as somehow being mere mouthpieces for drug companies is consistent with the narrative that pharma funding of organizations who would otherwise have no voice is illegitimate.  And it also sidelines a more serious discussion of whether patients believe  Exondys work.

The fact is, ICER ignores the value of orphan drugs and media coverage of funding enables such willful ignorance.   ICER claims that though Exondys  has “ been on the market for three years and yet we still found notably inadequate data on patient outcomes.”

Bet on journalists such as Kate Sheridan from STAT, Peter Loftus from the Wall Street Journal and Jonathan Saltzman from the Boston Globe accepting ICER’s assessment at face value and devoting more time attacking the patients who are justified in taking exception to such smears. 
 Indeed, these same reporters will side with ICER.  By demonizing patient groups for receiving pharma funds, these journalists legitimize ICER’s refusal to take into account the impact of such medicines on patients and their families. 

While ICER is welcome to conduct its assessment of our product or any other orphan medicine using its own set of value and methods, I know for a fact that ICER excludes many, if not most, of the information 
and considerations that insurers, doctors and patients took into account in determining how Exondys should be reimbursed. 

ICER assumes that everyone with DMD lives with the disease in the same way. Indeed, ICER favors those with more treatable conditions and those with greater potentials for health—be it in terms of functioning or longevity.  Its founder noted in an article entitled “Which Orphans Will Find a Home: The Rule of Rescue in Resource Allocation for Rare Diseases” that there is no apparent obligation to rescue identifiable rare  disease patients based on a duty of rescue within personal morality.”   

In evaluating the value of Exondys and other medicines for rare diseases, ICER claims to take into account that to people with the most serious rare diseases a small improvement in well-being is important. Yet in determining this value – on behalf such patients – ICER states: “The opportunity cost of supporting the use of ultra-orphan drugs necessitates that patients with a more common disease, for which a cost-effective treatment is available, are denied treatment.” 

In other words, ICER’s assessment of orphan drugs is shaped by its desire as Pearson writes, to “ensure that an undue burden is not placed on others for the sake of a few.” More specifically, ICER claims that paying for orphan drugs means “…we’re siphoning off resources for other things we need like better schools and more resources for local police, roads and bridges. “


These assertions are morally repugnant and factually misleading. Why should the cost of orphan drugs be pitted against spending on other forms of care for most other patients? Why not pit the high cost of hospitalization relative to medicines for so-called common diseases? What about the nearly $400 billion that is spent each year on ineffective or needless care? Indeed, to the extent that ICER does not  evaluate whether each additional dollar spent on police, roads and bridges generate more benefit using 
the same methods it applies to people with rare diseases, such trade-offs are both arbitrary and ideological.

Ultimately, what ICER fails to measure is what better health provides people living with orphan diseases: human dignity that is made possible when every individual, no matter how rare their condition has the same opportunity to live a full and long life. The price of Exondys reflects the size of the community with DMD and an indication of the importance of that social contract. But it also measures what ICER  does not: The effect of increasing the freedoms – the capabilities – to choose to do and be more of what people value. 

As Nobel Prize-winning economist Amartya Sen points out: groups that weigh the cost and benefit of helping people with serious illnesses against the total cost of health care, do so “without taking any direct interest in freedom, rights, creativity or actual living conditions. To insist on the 
mechanical comfort of having just one homogeneous “good thing” would be to deny our humanity as reasoning creatures.”

Instead of focusing on the freedoms generated by medical innovation, ICER and its journalistic allies measure medical innovation in terms of how much money it “siphons off” from governmental and insurance budgets. 

It does not measure “What is each person able to do and to be?" In other words, ICER measures an average health benefit (in QALY units) that has nothing to with increasing the capabilities and
opportunities available to each person. 

A measure of what advances in medicine contribute to increasing our capabilities is focused on choice or freedom. Rather than budget impact, the value of medicines should be measured by how they contribute to each individual’s ability to do or be what they hope for. And unlike ICER, both the approach and methods of measuring value must be inclusive, taking into account that the capability achievements that are central for people are different in quality, not just in quantity.

Rejecting a price or price increase using the ICER standard is tantamount devaluing the lives of people such institutions are responsible for. ICER’s use of standard cost-benefit analysis does not capture the tragic choices people with rare disease must live each day. It does not capture the daily and accumulated costs of living with a fatal or degenerative disease that people with good health don’t have to bear. Nor does it capture what living a life with more capabilities means to individuals or our nation.  The media is complicit in ICER’s implementation of soft eugenics.

 Finally, ICER suggests limiting spending on any medicines if the total amount goes over a certain percentage of our GDP. This is not the first time some have made the GDP a measure of what’s important and ultimately valuing individuals in terms of whether they add or subtract to that total.  Before the Nazis did so to justify sterilizing and killing people with hemophilia, depression, spina bifida or DMD, private foundations in the United States spent millions to support a similar program of extermination to save money and ensure that a small group of patients doesn’t impose a drain on society. 

In 1968 Robert F. Kennedy discussed the danger of this approach. His words are as important today as they were over a half-century ago:

“Too much and for too long, we seemed to have surrendered personal excellence and community values in the mere accumulation of material things. Our Gross National Product, now, is over $800 billion dollars a year, but that Gross National Product - if we judge the United States of America by that - that Gross National Product counts air pollution and cigarette advertising, and ambulances to clear our highways of carnage. It counts special locks for our doors and the jails for the people who break them. It counts the destruction of the redwood and the loss of our natural wonder in chaotic sprawl. It counts napalm and counts nuclear warheads and armored cars for the police to fight the riots in our cities….

Yet the gross national product does not allow for the health of our children, the quality of their education or the joy of their play. It does not include the beauty of our poetry or the strength of our marriages, the intelligence of our public debate or the integrity of our public officials. It measures neither our wit nor our courage, neither our wisdom nor our learning, neither our compassion nor our devotion to our country, it measures everything in short, except that which makes life worthwhile. And 
it can tell us everything about America except why we are proud that we are Americans.”

Ultimately medicines for rare diseases should be measured by the latter set of standards. And journalists who savage rare disease groups should care how they stack up against that moral measure.  My guess is they won’t and will continue to give ICER the legitimacy it requires to advance an inherently evil agenda.  
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Pharma Faces a Penalty Kick

07/24/2019 11:23 AM | Peter Pitts

The US Senate is debating whether to implement a Consumer Price Index (CPI) penalty on drug price increases. In other words, if any given product price increase is greater than general inflation, a penalty will be assessed by the government with monies then returned to Uncle Sam in the  form of a rebate. (This mechanism already exists in Medicaid.)  Sound good? Not so fast.
 
Drug manufacturers are already subject to such penalties today in the  form of “price protection rebates” negotiated by Pharmacy Benefit Managers (PBMs) and insurers.  These rebates effectively establish a private sector ceiling or cap on the amount by which medication prices can increase. Almost 100% of contracted medicines already have price protection built into their contracts Now that the HHS rebate rule is dead, a better and more timely question is: how much of those rebates/fees collected by PBMs and insurers are going back to the government? Without complete supply chain transparency we will never know.

And there’s more than one CPI. Which is the right one? General inflation or medical inflation? Medical inflation rates are at least 1-2% above general inflation. According to the U.S. Bureau of Labor Statistics, prices for medical care were 88.70% higher in 2019 versus 2000 (a $887.03 difference in value). Details count.

Between 2000 and 2019: Medical care experienced an average inflation rate of 3.40% per year. This rate of change indicates significant inflation. In other words, medical care costing $1,000 in the year 2000 would cost $1,887.03 in 2019 for an equivalent purchase. Compared to the overall inflation rate of 2.08% during this same period, inflation for medical care was higher.

Another unaddressed detail is, which price will be used for the inflation penalty? Will it be the retail price increase or the net price increase after all of the systemic cross-trading rebates and fees have been accounted for? Per IQVIA:



Our national policy makers are laser-focused on drug costs. But what about hospitals or physician expenditures, shouldn’t they peg the inflation rate to where the real inflation is -- the growth in hospital and physician per capita spending? Per Axios:



As always, the devil is in the details but, as Admiral Hyman Rickover reminds us “so is salvation.”  Read More & Comment...

Contracting Biosimilars

06/24/2019 11:19 AM | Peter Pitts

Per Denny Lanfear ‘s op-ed, “How Big Pharma Suppresses Biosimilars” (WSJ, June 24, 2019), it’s nice to see that the group Patients for Affordable Drugs has finally recognized that Prescription Benefit Managers (PBMs) are  a major roadblock to affordability and access to medicines. It just goes to show you that even David Mitchell can't be wrong 100% of the time. Shenanigans such as exclusionary contracting that prioritize PBMs profits over patient access to FDA-approved, safe and effective biosimilars must end. Just because such actions aren’t illegal doesn’t make them right. It’s past time to prioritize what’s best for patients.
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