"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 | Robert Goldberg

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 | Robert Goldberg

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 | Robert Goldberg

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 | Robert Goldberg

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 | Robert Goldberg

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.  
   Read More & Comment...

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.
   Read More & Comment...

Drug Importation: Stupidity Redux

06/03/2019 08:10 AM | Peter Pitts

From the pages of Politico ...

Vermont considers insulin, HIV drugs for importation
 
By Rachana Pradhan
05/31/2019
 
Vermont is eyeing birth control, insulin and pricey medications for HIV and multiple sclerosis patients as possible candidates for the state’s landmark program to import cheaper drugs from Canada, according to documents obtained by POLITICO.
 
The drugs are among the 17 most expensive that two private insurers identified for state officials and are potentially eligible for importation, show the documents, which were obtained through a Vermont public records request. Vermont officials determined the importation program could save insurers up to $5 million annually, based on that list of drugs.
 
The medications include Gilead’s Truvada, a $20,000-a-year HIV prevention pill whose price tag drew scorn from congressional Democrats during a hearing this month; Harvoni, among the recent class of drugs effectively curing hepatitis C; Merck’s Nuvaring, a contraceptive costing up to $200 per month without health insurance; and Zytiga, a blockbuster prostate cancer drug manufactured by Johnson & Johnson.
 
The list, which is from last year, isn’t a final catalogue of drugs Vermont would seek to obtain from the United States’ northern neighbor, but it provides a glimpse into the state’s deliberations more than a year after state lawmakers authorized the nation's first wholesale drug importation program.
 
“The 17 is our first cut, or our first time analyzing what would be candidates for importation that would generate savings,” said Ena Backus, the state's director of health care reform. “I think you do it a different quarter of the year you’re going to get a different list.”

Florida and Colorado approved similar importation programs this year. The idea has support from President Donald Trump but has encountered resistance from his top health officials.

Vermont still has several hurdles to overcome before its program could get off the ground. It still must finalize a plan and submit it for the Trump administration’s approval. Vermont is also delaying a statutory deadline to send its plan to HHS by a year — giving itself until July 2020 — because it wants to huddle with other larger states trying to build importation programs.

Because the process is breaking new ground, Backus said state officials were essentially "flying blind" on how to best approach federal officials.
 
“That’s why we felt like it was a great opportunity to slow down on submitting our application and to understand how more people are thinking about this," she said, adding that the state has not yet discussed its plan with HHS.
 
Vermont and other states seeking HHS approval must prove the importation program wouldn’t pose additional risks to patient safety and that consumers will pay less for drugs under the new model.

HHS set up a working group last summer to study importation, but its work — and even who’s in the group — has been kept secret.
 
The pharmaceutical industry and other importation opponents, including many Republicans, have argued that it will open up a dangerous pipeline into the United States for drugs whose safety cannot be accounted for. Further, they say it won’t produce much savings because importation could drain Canadian drug supplies, creating shortages and driving up prices, resulting in minimal savings for U.S. customers.
 
“We simply can’t fathom a duration or a scheme or a structure in which this plan will drive significant savings,” said Tom Rutkowski with CBPartners, a consulting firm focused on pharmaceutical and device issues which has analyzed the Vermont program.
 
The company said Vermont was unlikely to save much because discounts Canadian provinces negotiate with drugmakers would not be passed down to U.S. customers, and some especially expensive drugs that could drive greater savings don't qualify for importation.
 
“It’s a dumb idea now and a dumb idea in 2020,” Peter Pitts, president of the Center for Medicine in the Public Interest and former associate FDA commissioner, said of importation. “It’s a political ploy.”
 
The idea of importing drugs from Canada is gaining steam in states as officials grapple with rising costs and see a steady stream of new drug approvals with eye-popping prices. Maine and Connecticut are among the other states also considering creating their own programs, although legislation is unlikely to pass this year.
 
Trump has recently heaped praise on the idea, and he’s frequently complained that U.S. patients often pay much higher prices for drugs. He’s asked HHS Secretary Alex Azar — who has called importation a “gimmick” — to work with Republican Florida Gov. Ron DeSantis, a close ally, on his state’s importation plan.
 
“We may allow states to buy drugs in other countries if we can buy them for a lesser price — substantially less price,” Trump said at a White House event on health care earlier this month.

A plan won’t come easy, though. Vermont officials are still figuring out how much operating an importation program would cost and how it would pass cost-savings on to consumers. Backus, the health reform director, said the list of import candidates is a moving target because of changing prices and drug usage.
 
Further, the state doesn’t have the full picture of which drugs cost health insurers the most. Blue Cross Blue Shield of Vermont and MVP Health Care provided information for the state’s analysis but Cigna declined, Backus said.
 
Other regulatory issues could alter Vermont’s plans. For example, the FDA next year will regulate insulin as a biologic rather than a drug, which could make it ineligible for importation.

Backus and others said should Vermont manage to get its program off the ground, the state would likely update its list of importation candidates every quarter.
 
“This is all new ground,” said Trish Riley, executive director of the National Academy for State Health Policy, which is working with Vermont, Florida and Colorado on their importation plans.

“They want to be consistent and they want to think it through together,” she said.
 
Sarah Owermohle contributed to this report.  Read More & Comment...

Vinay Prasad: Precision oncology is not about the patient but all about the Benjamin

05/08/2019 01:22 PM | Robert Goldberg

s
 
Vinay Prasad is an associate professor of medicine at the Oregon Health & Science University School of Medicine. As his Wikipedia page states: “Prasad is a noted critic on the direction of cancer research. He has developed a following using Twitter as his platform.”  More to the point, Prasad has is adored by a group of journalists, policy types and cranks who believe that drug companies use shoddy clinical studies to market expensive medicines that are marginally effective.  
 
In particular, Prasad has singled out targeted cancer drugs as a waste of time and money, a position for which he has been praised and quoted by health care journalists who, like Prasad, publish this perspective with funding from Arnold Ventures.
 
In a recent episode (1.57) of his podcast called Plenary Session (which deserves all the neglect and inattention it receives) Prasad personally attacked University of California at San Diego cancer researchers Razelle Kurzrock and Jason Sicklick, the authors of a study published in Nature showing that attacking multiple genetic and non-genetic drives of tumor growth with combinations of targeted cancer drugs improve survival and reduces toxic side effects.
 
Before explaining why he believes precision oncology -- and the Nature study -- is largely a fraud, Prasad accused Kurzrock and Sicklick of grabbing $200 million for producing what he calls negative results about targeted cancer treatment.  (Around 3:28 into the podcast)
 
He then goes on to claim that Kurzrock and Sicklick effectively silence critics of their methods with “retribution” in the form of denying them funding for their own studies or the opportunity to publish responses.  He implies that he has experienced such retribution from Kurzrock in particular. (Around 4 minutes into the podcast.)
 
Prasad concludes that the researchers can only demonstrate clinical benefit by treating only people they know they can respond and denying others that don’t access to standard of care or by delaying the process of sequencing tumors that people die waiting. (21 minutes into the podcast)
 
Both Kurzrock and Sicklick are Jewish and both live near the Chabad synagogue in California recently targeted by an anti-Semitic animal who killed and maimed congregants during Passover services. Prasad’s personal attack intentionally or not invoked the trope about Jewish control over banking, media, manufacturing, etc. Indeed, by asserting that the researchers let people die in order to make more money through the expansion of personalized combination cancer therapy, I believe Prasad implicitly invokes the blood libel.  
 
Prasad has crossed two red lines. He has alleged, without any evidence, that the principal investigators have grabbed hundreds of millions of dollars for negative results, threaten those who dare to challenge them, covered up the deficiencies of targeted oncology and designed trials that harmed the sickest in order to enrich themselves. And he tells whoever is listening or following him on Twitter that Kurzrock and Sicklick that they did so to make as much money as possible by escalating the use of their junk science.  In other words, Vinay Prasad claims that precision oncology is not about the patient but all about the Benjamins.
 
 
   Read More & Comment...

Restoring the Balance Between Innovation and Generic Manufacturing of Medicines

05/07/2019 03:22 PM | Robert Goldberg

Restoring the Balance Between Medical Innovation and Generic Manufacturing of Medicines.    Read More & Comment...

Op-Ed from Real Clear Health: FDA Attack on Underage Vaping Could Backfire

05/06/2019 06:05 PM | Robert Goldberg

FDA Attack on Underage Vaping Could Backfire  
By Robert Goldberg
May 06, 2019
REAL CLEAR HEALTH

Scott Gottlieb’s extraordinarily productive tenure as Commissioner of the Food and Drug Administration (FDA) will be remembered for his crusading against underage use of E-cigarettes and the companies that produce them.  But his legacy is not to be found in his jawboning against the retail distribution of vaping products but in the guidance the FDA issued on developing a nicotine replacement therapy (NRT) product that reduces tobacco use.

Gottlieb announced the guidance in February, but it received little, if any, media coverage. That’s too bad because the document lays out a clear path to fully realize the contribution of vaping to the public health.  Specifically, he noted:


“Novel products with different characteristics or routes of nicotine delivery have the potential to offer additional opportunities for health-concerned smokers interested in quitting. This could also include products such as electronic nicotine delivery systems like electronic cigarettes, but which would need to be proven safe and effective for smoking cessation and regulated as a drug product. This would allow them to be marketed as a prescription or over-the-counter drug products with medical claims for smoking cessation or related indications – ultimately reducing the likelihood of someone continuing to suffer the clinical consequences of smoking. This is different from our regulation of e-cigarettes as tobacco products.”

And it is different from the public statements Gottlieb was making about e-cigarettes as tobacco products as well.  Last September Gottlieb announced the FDA was taking "historic action" by threatening e-cigarette manufacturers with criminal prosecution and sales bans unless they developed plans to reduce the "epidemic" of teen use. 

Juul, the leading manufacturer of vaping devices had anticipated the FDA move and had already begun implementation a "Youth Prevention Plan" that eliminated pod flavors, such as cucumber and mango, from the 90,000 or so retail outlets across the country. Flavored pods are still available online, but Juul also had stopped using social media, instituted age verification tools to ensure that purchasers are 21 years of age or older introduced new purchase limits and product serialization.

These actions did not mollify Dr. Gottlieb.  Instead, he stepped up his attack.  He told Vox: “The dramatic spike of youth [vaping] — that was driven in part at the very least if not largely by Juul.  I hope they recognize the problem that’s been created has been created largely by their product.”

While Gottlieb “acknowledged there’s, no definitive data showing the teens now experimenting with vaping are using Juul” he still holds the company responsible for triggering what the regards as a spike in teen addiction to nicotine that could lead to cigarette use.

Meanwhile, the guidance discusses how companies should test the effectiveness of E-cigarettes in reducing tobacco use in the pediatric population: “Given that use of tobacco products frequently starts in early adolescence, drug products approved for smoking cessation have the potential to benefit and be used in the pediatric population. Based on the current prevalence of smoking in younger children, the Agency has waived…requirements for clinical studies of NRT drug products in patients younger than 12 years of age because clinical studies would be highly impracticable in that age group.”

So, the opportunity is there to turn e-cigarettes into medical products that could be available to anyone who needs to quit smoking at any age.  Conceivably, vaping products could be available behind the counter along with other heath products that have taken the reverse course from by prescription only to OTC.  

Hence, Gottlieb was trying to prod e-cigarette companies to re-introduce themselves as an OTC medical device. The guidance notes to receive OTC approval an NRT product must demonstrate effectiveness in reducing smoking and evidence that the marketing of e-cigarettes increases the safe and effective use of products.  This is something companies such as JUUL have shown they can do.

The question is whether his efforts have made it impossible to get an e-cigarette approved as an NRT.  In stressing the risk of underage vaping the FDA has contributed to the fact that two-thirds of American adults mistakenly believe that e-cigarettes are just as harmful as smoking.  The media’s fearmongering fueled by the FDA attack and spread by so-called consumer groups has incited a movement to ban e-cigarettes altogether. Rather than being able to use vaping to reduce the use of nicotine, people will pick up a pack of cigarettes instead. 

Robert Goldberg is Vice President at the Center for Medicine in the Public Interest.  Read More & Comment...

ICER Cites Date Rape Drug as Being Cost Effective Treatment for Depression

04/23/2019 06:02 PM | Robert Goldberg

 
My colleague Peter Pitts has written an article on a Democrat proposal to submit the price and use of a new drug for binding arbitration before Medicare or another government program pays for it.   Arbitration is supposed to be a fair way to impose drug prices, but it is price controls all the same. 
 
As with everything, proof in practice is more instructive than proof in principle.  So, let me provide you with a real-world example of how such arbitration would proceed.  Proponents use the German approach to pricing arbitration as a wonderful example of how spectacular arbitration is.  There, the process comes after a company and the committee set up to determine prices can’t agree on reimbursement levels for the medicine in question. 
 
Throughout, the goal is to set a price for a drug based upon the additional value it generates and converting that into a price premium compared to the average price of an existing drug for a condition selected by the committee.
 
The selection of comparator (as well as the reference price) is one critical element.  The other is whose value is being measured.  Interestingly, the German approach does not use quality-adjusted life years to make value determinations.  The Germany process defers to an independent group to determine what comparator to use and whose value should be preferred.
 
In the US, proponents of pricing arbitration envision the Arnold Ventures funded group ICER serving as the arbitration organization, or something else that John Arnold would pay for by sponsoring its formation or operation.   The Center for American Progress, in particular, loves the idea of the ICER driven pricing deals.
 
ICER puts limits on how many people could use a drug using a price that is sensitive to insurer and PBM profits.  And that price is also based on a comparator that ICER also selects.  The cheaper the comparator, the greater the hurdle for a new drug regardless of benefits, particularly since the goal is to save insurers money and maintain the PBM spread between net and list pricing.
 
Here’s an example of how the binding arbitration proposal in the US would work using a real-world example and ICER’s most recent analysis of a new drug:
 
Spravato, a new drug for major depression was recently approved, a form of ketamine that quickly improves mood without resort to ketamine’s hallucinogenic effects.
 
ICER’s initial review of the drug determined Spravato was effective but said that at the list price it was not cost effective and could only be used by a small percentage of people. 
 
ICER’s comparator?  Injectable Ketamine which is now used to knock out animals and some patients.  The ketamine that, in the ICER preferred formulation, has the same mechanism of action at PCP.  What’s more, ICER’s comparator is not even approved for treating depression.  Untested, off-label use of ketamine.  You might know it as Cat Tranquilizer, Cat Valium, Jet K, Kit Kat, Purple, Special La Coke, Super Acid, Super K, and of course, Special K, the medicine preferred by Bill Cosby.
 
You read that right.  ICER used a form of a date rape drug as the comparator for determining price and is recommending the use of a date rate drug as a more cost-effective treatment. 
 
That might sound extreme, but in fact, the capricious and one size fits all selection of comparators is a hallmark of every price negotiation.  Standard of care is chosen to make price decisions.  Benchmarks are adjusted, goal posts are moved to maximize rebates, not well-being.  Drugs that require price cuts based on comparators are often not sold in a particular market because they lose money.   Advocates of arbitration claim that is just an example of value-based care.  Yet in Germany, 82 percent of drugs that were withdrawn because they didn’t meet the comparator price-value threshold were recommended by at least one guideline.  Hence, drugs recommended because of their clinical benefit were no longer available.   
 
Early assessment of new medicines is fraught with danger because prediction is not just political, it is unscientific and uncertain.   ICER’s involvement assures that such arbitration related assessments and comparisons will be downright immoral.   Read More & Comment...

Virginia's Northam Leaves PBMs Red-Faced

03/28/2019 07:38 AM | Peter Pitts

From the pages of the Roanoke Times …
 
Med Beat: Drug makers' coupons to count toward deductibles

Gov. Ralph Northam signed a bill that will require insurers to count toward deductibles and co-pays any payments made on behalf of a patient, including assistance by pharmaceutical makers.
 
The practice known as copay accumulator adjustments excluded from patients’ deductibles and maximum out-of-pocket costs the coupons and assistance programs that lower the price of medication.
 
Fair Health Care VA Coalition lauded passage of the bill.
 
“As out-of-pocket costs continue to rise, Virginia patients already face enough barriers to accessing the health care coverage that they need. Copay assistance programs are a critical resource, particularly for patients whose health care costs could bankrupt their families or force them to live without the care they need,” Dr. Bruce A. Silverman, a Richmond nephrologist and advocate for the collation, said in a news release. “Patients should not be denied one of the key benefits of copay assistance programs, particularly since insurers are already getting the value of negotiated drug price discounts while withholding these benefits from patients.”
 
When the change goes into effect July 1, all payments made by patients or on their behalf will count toward maximum out-of-patient payments and deductibles.  Read More & Comment...

Drug Rebate Head Fake

03/12/2019 03:30 PM | Robert Goldberg

Today United HealthCare announced that they will be passing rebates directly to patients.  But the impact is not as impactful as may seem. 

United noted that “all new employer-sponsored health plan customers that use UnitedHealthcare must give discounts they get for including certain drugs in their lists of covered medications directly to consumers at the point of sale… UnitedHealthcare said Tuesday that its expanded requirement does not apply to existing employer customers that do not already give rebates directly to the consumer.”

As Drug Channels notes: “PBMI found that only 4% of employers reported that rebates were used to reduce member out-of-pocket costs at the point of sale.”

Indeed, despite the announcement of these offering, Drug Channels demonstrates (see chart below) that employers really don’t want to use rebates to cut out of pocket drug costs.

 

So much for sharing the savings.  

But even passing rebates on to patients is not enough.  In fact, it could make things worse in a perverse fashion.  For example, a pass-through model would reward the most rebated drugs but lead to even more step therapy or greater formulary restrictions in an effort by PBMs to make money on contracts that still reward them for lowest net cost of medicines.   Even drugs that offer zero copay but do little to affect overall net cost of medicines could be excluded.  

Another question:  Will insurers and PBMs increase the use of (illegal) co-pay accumulators – programs that intercept money designated by drug companies to reduce out of pocket costs is confiscated by insurers and NOT used to reduce cost sharing -- and then pass that cash to employers.  Can we say racketeering?  (You can read my colleague Peter Pitts article on the wholesale thievery called copay accumulators here. 

To be sure, many business health groups claim they want to optimize the use of medicines.  However, they lack the data and bandwidth to do so.  Some individual companies do make an effort to support value-based design, but most of these offerings are designed to drive people to more generic drug use.  

Absent these insights, the well-intentioned effort to reduce out of pocket costs by shifting rebates will help some but likely hurt others. Which is why at some point, large employers will be asked to explain why they aren’t doing more through smarter changes to benefit design to help their employees stay health – and alive. 

   Read More & Comment...