Latest Drugwonks' Blog

Lots of headlines about the pricing practices of Turing and Valeant, which are the subject of an investigation lead by  Democrats on the House Oversight Committee .  The large retail price increases of these two companies are, we are told by mainstream reporters at Bloomberg, Wall Street Journal, etc media and Congressman Elijah Cummings, is indicative of what is going on in almost every company. 

Well sort of.   As Adam Fein has noted,  most generic drug price increases are a response to shortages, of which we have way too many.  In most cases, the price increases can be blamed on drug shortages. "For example, the NADAC per unit for doxycycline hyclate (100 mg tab) increased from 5.6 cents to $3.65 (+6,351%). The increase is most likely due to a nationwide shortage. I presume there’s also an active gray market, as in generic injectables. For context, see Drug Shortages and Gray Market Profiteering."

And the retail price, once again, is not the real price.  Again, Adam (drug)channels Mr. Spock in his logical analysis of the gap between retail and acquisition cost.

Adam's columns on pricing are more educational and authoratative than the turgid reports from some members of Congress. 

Apparently the quality of journalism – if it can be called that – about the cost of medicines has not hit bottom.

Two new lows were hit by Robert Langreth (with Rebecca Spalding) at Bloomberg and  USA Today's Jane O’Donnell. 

Langreth and Spalding wrote a predictably predictable article on the eve of Martin Shkreli’s no show congressional flogging.   Predictable and derivative since it recycled the same stuff written by other low achieving reporters.  The headline says it all: Shkreli Was Right: Everyone's Hiking Drug Prices.

So I will respond by recycling a previous blog on a ‘me-too’ pricing article in noting that Langreth fails to put drug price increases (net price or otherwise) into perspective.  Langreth states that U.S. prescription-drug spending rose 12.2% in 2014, accelerating from 2.4% growth in 2013. But “price increases for protected brands increased spending by $26.3 billion, contributing 8.2% to total market growth on an invoice price basis; estimated net price growth was substantially lower as rising off-invoice discounts and rebates offset incremental price growth and reduced net price contribution to growth to 3.1%.” 

That’s an increase in spending of about $7.1 billion.  Total US health care spending increased by $100 billion from 2013-2014.  So brand drugs were 7 percent of that amount. 

O’ Donnell takes reporting on drug prices to a new low in "Patient groups funded by drugmakers are largely mum on high drug prices"  She accuses patient groups that receive support from biotech firms from blocking efforts to impose price controls.  The headline is the tipoff.   To be more precise, she let’s Zeke Emanuel do the smearing.  If you want a job done well, hire a pro:

"It is worrisome because it is a conflict of interest even if you can’t prove it changes their position," says Ezekiel Emanuel, an oncologist and professor who chairs the University of Pennsylvania's department of medical ethics and health policy. "The patient voice carries a disproportionate amount of weight."

So if someone alleges it changes your position, that’s a conflict of interest.  

By that standard, the patient groups she cites as conflict free should also be suspected of conflict. More specifically, the Patient Voice Institute works with the Leapfrog Group which also gets money from large employers, health purchasing groups and AARP.   All three are quoted in the article.   But applying Zeke’s conflict benchmark, the fact that I can’t prove any connection means it is a conflict. 

The j’accuse of getting funding from corporations is a diversion.  O’Donnell, like many of her colleagues fail to look at the cost of new drugs relative to what insurance companies spend.  It’s about 3 percent.  And that spending makes treating illness less expensive by reducing hospitalization, saves lives and improves quality of life.  

The question O’Donnell ignores is the one the Leukemia and Lymphoma Society answered about a year ago:  How much would it really cost to pay for the drugs insurers scream are too expensive? 

They commissioned a Milliman study and found that it would cost on average about 50 cents per patient per month.  If drugs were driving up overall costs (as opposed to reducing them, which they do) why such a small increase to make people whole?

O’Donnell had the opportunity and column space to look at this issue.  Instead she went down a darker, more deceptive road.   What a shame.  Then again, it's not surprising.  

Compound Fracture

  • 02.01.2016

Mark Baum’s  self-serving op-ed in the Wall Street Journal (New Prescription for Lower Drug Prices) that compounded-drug makers can bring inexpensive, off-patent medicines to market, “if the FDA will let them” omits a key issue in the debate – public safety.

Just last week, two Alabama pharmacists agreed to plead guilty to criminal charges in connection with the 2011 deaths of nine Birmingham-area patients who allegedly received a contaminated compounded drug.

According to federal prosecutors, the drugs were contaminated from being prepared, packed or held in unsanitary conditions. The Centers for Disease Control and Prevention found the same bacteria on a water faucet in an open container of amino acid powder, and on the surface of mixing equipment that had been used to make the drug, according to federal prosecutors.

Also last week, Dr. Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, said she has ruled out the use of compounding to combat spikes in generic drug prices. 

Woodcock told the U.S. Senate Health, Education, Labor and Pensions (HELP) Committee that "there are very great risks" from FDA allowing mass production of compounded drugs to reduce the cost of a generic drug. 
Woodcock said that while recent legislation gives FDA additional power to enforce quality standards on pharmacies that compound sterile injectable drugs, FDA has limited oversight authority over compounding of tablets and pills. She said FDA has recently withdrawn compounded vitamin and hormone products after pharmacies distributed potentially fatal super-potent formulations. 

Despite Mr. Baum’s rosy commercial projections, compounded drugs may be sub- or super potent, contaminated, or otherwise adulterated. Additional health risks include the possibility that patients will use ineffective compounded drugs instead of FDA-approved drugs that have been shown to be safe and effective.

His company, Imprimis, has had challenges with its compounding facilities in New Jersey and Southern California, both of which have been issued letters from the FDA citing Common Good Manufacturing Practices (CGMP) violations that could call into question the safety and effectiveness of the drugs compounded there.

Putting price before patient safety is bad medicine and worse policy

Sleeping with the Enemy? Hardly. Eli Lilly & Co. has announced an important collaboration with an unlikely bedfellow – their Hoosier neighbor, Anthem. The goal is to help develop common ground on policy solutions related to cost and value. Finally – two important players understand that they are, in fact, on the same team.


More information can be found here.

FDA finds Indian drug maker Wockhardt hid failed tests


Indian drugmaker Wockhardt hid the results of failed tests and deleted data from its systems at a plant in western India, according to a report by the U.S. Food and Drug Administration sent to the company earlier this month and seen by Reuters.

Issues around "data integrity", maintaining accurate and consistent databases, are key to the U.S. watchdog, which regulates the world's largest market for generics producers.

Wockhardt is the latest of several major players in the $15 billion Indian drugs industry to be hit by U.S. regulatory action over the past few months.

It makes around a fifth of its $670 million in annual revenues from the United States and had said the Shendra plant, the site that prompted the FDA report, would boost its U.S. business. Shendra makes lucrative injectable medicines, which analysts say are key to Wockhardt’s U.S. plans.

Wockhardt did not return several telephone calls and emails requesting comment on the detailed report.

The FDA did not immediately respond to a request for comment on its report. It issues such reports, known as a 'Form 483', when its staff believe that conditions at a manufacturing site could lead to products that are harmful to human health.

In the report, dated Jan. 12, the FDA said that among other violations, the audit showed that the results of 22 failed tests had not been recorded. It also found multiple data files had been deleted from some machines.

The FDA did not detail whether the files or tests related to specific drugs, or whether the violations could impact the quality of medication produced at a plant which still exports to Britain and Ireland.

FDA inspectors also reported finding pharmaceutical ingredients that were not stored or labeled properly. A rejected drug batch was stored in the "approved material" area, and some batches did not carry expiry dates, the report said.

Citing his “extensive ties to the pharmaceutical industry,” Senator Bernie Sanders has placed a hold on the nomination of Rob Califf to be FDA Commissioner.

What are those “extensive ties?” Working to design and field innovative clinical trials for FDA review. You want the best and the brightest to work with industry on such matters – because industry is the one that does them. Not academia. Not NIH. Not physicians. And not the FDA. The pharmaceutical industry. And well-designed and executed clinical trials provide important insights into the benefits and risks of potential new therapies. To those reading this column this isn’t a surprise – but to many others it is.

If Senator Sanders thinks that having one of our nation’s keenest clinical trial design experts working with industry is a reason to place a hold on his nomination, then it’s time for him to step back and reconsider his position. When it comes to clinical trials that investigate safety and efficacy, we can't afford to use only the second best and almost brightest.

Yes, Bernie has other things on his mind at the moment, but facts as pesky things.

Somewhat different (but similar) from the FDA's "Filgrastim SNDZ" naming decision, the World Health Organization has posted a final version of its proposed biologics naming policy. It proposed that each biologic, including biosimilars, would be assigned a four-letter “biological qualifier” (BQ) that would make it possible to trace the compounds globally. BQs could be used for pharmacovigilance and to facilitate transferring prescriptions among countries.
WHO would generate BQs. The qualifiers would consist of random consonants, would be separate from non-proprietary names, and could be assigned retrospectively or prospectively.

It's BQ IQ.

A new report by the Pharmacy Benefit Management Institute (PBMI) makes some very interesting points about the value of PBMs to employers and employees. Some highlights include:

* PBM generic copays are in line with overall inflation, only increasing from $9.85 to $10.85 over a 15-year period in inflation-adjusted dollars.

* Preferred and non-preferred brand copays (preferred brand from $19.43 to $31.08 and non-preferred from $37.58 to $56.65) have outpaced inflation considerably.

* Plan sponsors increased use of prescription drug benefit deductibles by 157% in 2015 compared to 2014.

* In 2014, only 14% of plan sponsors reported having a deductible for prescription drugs compared to 36% in 2015.

* There is considerable opportunity for employers who are willing to implement additional strategies to control costs and utilization without shifting additional costs to members.

* Mail order in particular can save members an average of over four monthly copayments per prescription per year (annualized). For a member taking a preferred brand in a three-tier plan design, this equates to yearly savings of $138.88 for a single maintenance medication.

The complete report (sponsored by Takeda Pharmaceuticals) can be found here.

In his blog David Shaywitz comments on an editorial in the New England Journal of Medicine (NEJM), written by editor-in-chief Jeff Drazen, along with Dan Longo. "that a new class of research person will emerge—people who had nothing to do with the design and execution of the study but use another group’s data for their own ends, possibly stealing from the research productivity planned by the data gatherers, or even use the data to try to disprove what the original investigators had posited. There is concern among some front-line researchers that the system will be taken over by what some researchers have characterized as “research parasites.”

Shaywitz writes he was delighted to see the editorial:

"Not because I agreed with it–my heart is truly with the data scientists–but because I was grateful that someone had the courage to articulate a perspective I’ve come to believe is shared by the vast majority of academic researchers, but publicly voiced by no one–until now.
The result: a classic case of stated preference vs. revealed preference, where every academic researcher dutifully claims to be interested in sharing their data widely and freely, but somehow, tend not to actually do this."

David is right.  But there is even more reason to ‘cheer’ the NEJM article.  It reveals that scientist think it is “their” data when in fact it is the patient’s data.  The researcher is entrusted with that data by us to use it to advance science and promote cures. 

And the self-interested way such data is used – or shared – often abrogates the social contract in many ways.

Let me focus on one in particular, from the research article in the NEJM -- CDX2 as a Prognostic Biomarker in Stage II and Stage III Colon Cancer -- that was the subject of the editorial.  The researchers used data collected from cancer patients by the National Cancer Institute.  Drazen and Longo call this “symbiotic collaboration.”   I would call it another permutation of an approach that betrays patients. 

The article notes: “Given the exploratory and retrospective design of our study, these results will need to be further validated. We advocate for these findings to be confirmed within the framework of randomized, clinical trials, in conjunction with genomic DNA sequencing studies.”

In other words, the symbiotic collaboration first and foremost will be used to fund randomized trials and sequencing studies support by the same programs that were paid to put together the tissue bank.  As Stuart Kauffman, Colin Hill, Sui Huang and Lee Hood have noted: the methods used to generate so-called evidence-based medicine -- the basis for medical practice and reimbursement—randomized clinical trials (RCT) and comparative effectiveness research—are dangerously broken.

Data-sharing that respects the needs and hopes of patients is defined by how broadly data is shared and the degree to which researchers use data to tailor cancer treatment combinations to achieve the best outcomes possible.

So by definition, companies that use data from a variety of sources to establish biomarkers independent of the researcher who extracted tissue from patients in single person trials and based on powerful machine learning derived algorithms are supporting mutualistic relationships. 

Researchers that boldly push for the Drazen model are, in my opinion, the true data parasites.

I think lots of researchers become researchers because they want to change the world for the better.  You can check out the LabTV YouTube channel and see short videos of hundreds of researchers with such an ethos.   The Drazen model merely reinforces the control of a small elite that are both disdainful of people like Eric Topol who is paving the way for a consumer led data revolution and fearful that the transformation means they will be out of jobs.  Lee Hood’s P4 medicine vision is based on collecting and sharing data generated by patient activated social networks.  Would Drazen call Dr. Hood a parasite??

To those who say that sharing will reduce incentives for innovation, just the opposite is true.  Establishing the relationship between molecular insights and meaningful changes in disease progression depend heavily on collaborations that leverage the digitization of biology to its fullest. 

As Shaywitz points out, now that we know the true motives of researchers, we can define the parasite problem and tackle it head on.

CDER House Rules

  • 01.25.2016

The recent mega-storm allowed many of us drugwonks to curl up with a new work of action non-fiction … from the FDA:

Guidance Agenda: New & Revised Draft Guidances CDER is Planning to Publish During Calendar Year 2016

It’s worth perusing in its entirety, but here are a few items to tickle your regulatory palate:

For those of you following the debate over off-label communications

Manufacturer Communications Regarding Unapproved, Unlicensed, or Uncleared Uses of Approved, Licensed, or Cleared Human Drugs, Biologics, Animal Drugs and Medical Devices

The New Gold Standard

Adaptive Design Clinical Trials for Drugs and Biologics; Revised Draft

Meta-Analysis of Randomized Controlled Clinical Trials to Evaluate the Safety of Human Drugs or Biologic Products

Multiple Endpoints in Clinical Trials

The broader sharing of pharmacoecomonic data

Health Care Economic Information in Promotional Labeling and Advertising for Prescription Drugs Under Section 114 of the Food and Drug Administration Modernization Act

And the continuing saga of “Emerging Electronic Media” (aka: “Social Media”)

Internet/Social Media Advertising and Promotional Labeling of Prescription Drugs and Medical Devices – Use of Links to Third-Party Sites

The evolution of discussing risk information

Presenting Risk Information in Prescription Drugs and Medical Devices Promotion; Revised Draft

For the Biosimilar Brotherhood, three things to consider

Considerations in Demonstrating Interchangeability With a Reference Product

Labeling for Biosimilar Products

Statistical Approaches to Evaluation of Analytical Similarity Data to Support a Demonstration of Biosimilarity

Attention NORD Horde

Guidance for clinical Investigators and Sponsors Natural History Studies for Rare Disease Drug Development

Women and Children First?

Measuring Treatment Benefit in Pediatric Populations: Use of Clinical Outcome Assessments

Pediatric Oncology Product Development; Revised Draft

Pregnant Women in Clinical Trials – Scientific and Ethical Considerations

Pharmacokinetics During Pregnancy and the Postpartum Period – Trial Design, Data Analysis, and Impact on Dosing and Labeling; Revised Draft

Phase Fore!

Postmarketing Safety Reporting for Human Drugs and Biological Products Including Vaccines

REMS Assessment: Planning and Reporting

Waxing Generic

Postmarketing Safety Reporting for Human Drugs and Biological Products Including Vaccines

Updating ANDA Labeling After the Marketing Application for the Reference Listed Drug Has Been Withdrawn

General Principles for Evaluating Abuse - Deterrent Properties of Generic Solid Oral Opioid Drug Products

What’s on CDER’s list is interesting … as well as what is not.

2016 is going to be an interesting year.


Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

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