Latest Drugwonks' Blog

Abusing the Privilege?

  • 11.02.2016
  • Peter Pitts
From Medpage Today ...

FDA, Industry Focus on Abuse-Deterrent Opioids

No consensus immediately apparent after 2-day meeting

FDA officials and representatives from both the generic and branded drug industries spent two days hashing out next steps for development of abuse-deterrent opioids.

The two topics on the table: reviewing a draft guidance for the development of generic versions of abuse-deterrent opioids, and developing standard in vitro testing methods to characterize a drug's abuse-deterrent properties.

The meeting was in line with FDA's strategic plan for mitigating the opioid epidemic, which it released in February 2016, said Douglas Throckmorton, MD, deputy director of regulatory programs at the FDA's Center for Drug Evaluation and Research. That includes incentivizing the development of "progressively better" abuse-deterrent opioids and supporting a transition to these products, he said.

Throckmorton acknowledged the tension between generic and branded developers, but advocated working together to come up with a solution that benefits all parties.

"If you all came up with a single approach that suits the best product development, we would be delighted and take it very seriously," Throckmorton said. "It would move the field a great deal. It would require careful collaboration, but it's been successful in other fields, like drug-eluting stents, which had a similar challenge but the industry pulled together and made suggestions we all make use of."

FDA released its draft guidance for evaluating generic abuse-deterrent formulations last March, with the goal of ensuring that a generic is no less abuse-deterrent than the brand-name opioid. No generic abuse-deterrent opioids are currently approved.

In general, an ANDA applicant isn't required to provide independent evidence of the safety and effectiveness of the generic drug; instead it relies on the FDA finding that the previously approved product is safe and effective and must demonstrate that the generic is "bioequivalent" to the reference drug, primarily on the basis of pharmacokinetic studies.

Robert Lionberger, PhD, director of the office of research and standards in the FDA CDER's office of generic drugs, noted that the ANDA for any generic abuse-deterrent opioid would cover all routes of abuse: oral, nasal, injected, and smoked. It would also evaluate the drug in comparative in vitro studies and, in some cases, in relevant pharmacokinetic or other studies to show it is no less abuse-deterrent.

But some industry voices -- particularly from the branded industry -- said these assessments did not go far enough.

"I am concerned that this draft guidance is not sufficient to ensure that generic versions will be no less abusable than the reference drugs," said Alexander Kraus, PhD, of Grunenthal USA in Morristown, N.J. "It does not do enough to ensure that the generic meets therapeutic equivalence. It can't be based solely on in vitro testing. I encourage the FDA to require not only Category I in vitro tests, but Category II pharmacokinetic and Category III human reference studies as well."

Those Category I in vitro tests for abuse-deterrent opioids have their own issues to be worked out, which was why they were also a focus of the meeting.

Xiaoming Xu, PhD, a senior staff fellow at the FDA's division of product quality, said in vitro testing for abuse-deterrent opioids has not been standardized, making both comparisons with other drugs and overall assessments challenging.

Ideally, that testing should assess each potential route of abuse -- oral, injectable, nasal, and smoking -- starting with simple and gentle mechanical and chemical manipulations, progressing to complex and more destructive manipulations. It should also address mechanisms by which abusers can be expected to attempt to overcome abuse-deterrent properties as well as the ways that patients may alter the formulation to change the amount of of drug that gets released, Xu said.

The challenge is that the design of these experiments is complex: any single product can have more than a dozen possible methods to achieve a desired manipulation; scores of different solvents; different reactions to various temperature conditions; different volumes released, and so on. Indeed, the number of experiments can run into the thousands, Xu added.

The problem garnered unique observations about the automobile industry and coffee grinders. Richard Lostritto, PhD, acting associate director in the office of policy for pharmaceutical quality at FDA's CDER, noted that the auto industry standardized the type of hammer it would use in safety tests; Karsten Lindhardt, PhD, of Egalet Corporation, noted that he's had to buy several different types of coffee grinders to do proper in vitro testing.
"You end up with each material behaving differently," he said. "One grinder may be optimal for one drug but not for another."

Throckmorton acknowledged the tension between developing standardized versus individual tests for these types of analyses: "We've seen that even small changes in some formulations can have a large effect on product performance."

Although it was not a point of the meeting, critics at past FDA advisory committee meetings for abuse-deterrent opioids have called for epidemiologic data on whether or not these formulations can actually reduce abuse and the more serious consequences of addiction, overdose, and death.

Also, the meeting did not address patent issues, which generally fall outside FDA's jurisdiction. Although the major opioid drug compounds are now off-patent, it may be many years before patents expire on the specific abuse-deterrence technologies now in use.
The November issue of Lancet Oncology focuses on pharmacovigilance and drug safety. The lead editorial drives home some key points:

At a time when the number of biological agents due to come off patent is increasing, and in the face of a market fuelled by escalating drug prices and pressure from pharmaceutical companies and patient groups alike for expedited drug approval, issues surrounding the safety and efficacy of agents such as biosimilars and generics are paramount.

Substantial variation exists between high-income and low-to-middle-income countries with regards to manufacturing and supply chain regulation of generic drugs, despite countries such as India providing a large market share of generic drugs worldwide. Moreover, bioequivalence—a key consideration when comparing generic formulations with their trademarked counterparts—can vary substantially, making appropriate regulation particularly important. Issues of safety and regulation are further compounded when approving biosimilars: despite nearly 10 years’ of experience in dealing with biosimilar agents, regulators still need to streamline and expedite approval processes, and improve ways of reducing cost. Thus, taken together, the importance of pharmacovigilance has never been greater.

Given that multiple health-care systems encourage or enforce generic and biosimilar prescribing, sometimes without physician knowledge or consent, coupled with further potential complications created by generic or biosimilar switching during a course of treatment, pharmacovigilance needs to evolve beyond merely the uncovering, monitoring, and reporting of adverse events, to continual pre-marketing and post-marketing surveillance.
Although a focus on regulatory and policy issues is key to monitoring safety and     efficacy, especially for newer agents, there are other ways in which drug safety can be improved.

The November issue also includes my new paper, 21st century pharmacovigilance: efforts, roles, and responsibilities.

Here’s the abstract:

In an era when the number of expedited and conditional review pathways for newly available brand-name drugs and biosimilar medicines to treat serious and life-threatening diseases is increasing, defining pharmacovigilance has never been more crucial. 21st century pharmacovigilance is not merely about uncovering, reporting, and addressing adverse events associated with already approved and marketed agents, but can be described as the systematic monitoring of the process of pre-market review and post-market surveillance, which includes the use of medicines in everyday practice. Pharmacovigilance identifies previously unrecognised adverse events or changes in the patterns of these effects, the quality and adequacy of drug supply, and should ensure effective communication with the public, health-care professionals, and patients about the optimum safety and effective use of medicines. In this paper, the first in a Series of three about drug safety in oncology, we discuss evolving challenges in the purview, roles, and responsibilities of the US Food and Drug Administration and the European Medicines Agency with respect to pharmacovigilance efforts, with a special emphasis on oncology treatment.

If you’d like a copy of this article, please contact me at
ICER released a report claiming that at retail prices,  immunotherapies (Tecentrig, Keytruda and Opdivo)  for non small cell lung cancer (after a first line of treatment) are not worth using compared to a 20 year old drug (docetaxel) that is 3-4 times less likely to help patients and when it does work, is half as effective in extending life and generating a treatment response.   The table below shows the significant clinical benefits of new drugs that block PDL-1 and PD-1 protein expression, 
Drug Ratio of Patients Likely to    Benefit from Immunotherapy Compared to Docetaxel Expected Survival in Months Differnce in Duration of Response in Months
Docetaxel   8.5  
Acentriq 3.4 14.8 7.2
Opdivo 4.0 11.1 11.6
Keytruda 3.8 19.4 Not reached
  Source: Institute for Clinical and Economic Review, Evidence Report - Non-Small Cell Lung        Cancer, Table 6 page 36 and Table 10 page 45

ICER determined that the maximum amount of money to be spent on an additional year of life in perfect health -- a quality adjusted life year (QALY) is $150K.   Which means that most of the new drugs developed and to extend the life of lung cancer patients over the past ten years would not be used. 

Applying the ICER QALY calculations to lung cancer patients over the past ten years, I found that if ICER had its way 10 years ago, and denied access to new lung cancer drugs, today the death rate from lung cancer would be 23 percent higher.  And the cost of care will be more expensive, increasing hospital related costs by $3 billion a year.

ICER ignores indirect costs, such as lost productivity and caregiver salaries or the productive possibilities of being able to make plans to marry, raise kids, compose music, travel, go to school. ICER ignores the value of two additional years of life to a patient who has run out of options.

But the worst element of the ICER framework is how it devalues the additional months and years of life people with advanced lung cancer can get from newer medicines.  

At face value ICER’s QALY standard of value is arbitrary. Patients who live twice as long as those taking a generic drug should be credited with the total years of extended life.  ICER believes that an additional year of life for a lung cancer patient is really worth two-thirds of an additional year of life for a healthy person.  


Where has this metric been used before?    Article 1, Section 2, Paragraph 3 of the United States Constitution, which allowed slave states and their collective slave drivers to count each of their slaves as 3/5 of a white man.  

To mind the use of a QALY in determining access and drug prices violates the spirit, if not the letter of the Equal Protection Clause.

Worse (perhaps)ICER uses that same discriminatory rationale by assigning a lower value to patients and a higher value to insurers.   When it comes to patients, ICER uses the retail price of the drug to determine a QALY   When it comes to payors,  ICER uses the price or cost of the drug LESS any cost savings generated. 
Immunotherapy  Payer Cost per patient 
net of drug generated savings 
Cost used for patient QALY  
Tencentriq       39000   102519    
Keytruda 73900 108841  
Opdivo  44500 180489    

ICER is the worst example of how economic analysis has been applied to health care.  In the end, it is not about numbers. It is about people and the additional years and vitality medical innovation generates.  ICER is not alone in failing to factor in the economic value of hope or the virtue of forward looking, of having a project.

Modern medicines are extending lives, improving lives and saving lives. Setting an arbitrary limit on what that is worth is simply closing the door on medical progress, increasing the death rate and driving up costs at the same time.  And it also forecloses the possibilities of life that, while intangible or uncounted, adds more to our well-being than consumption.  As the great economist Irving Fisher noted: The true “wealth of nations” is the health of its individuals. And greater health through the diffusion of medical innovation is the most important way to eliminate inequality and eliminate barriers to people battling disease. ICER's value framework diminishes the value of longer life as part of an effort to show most new drugs are NOT cost effective.   In doing so, it is violating the civil rights of people because they have lung cancer.   

Bernie's Terrible Tweet

  • 10.23.2016
  • Peter Pitts
In a recent tweet, Senator Bernie Sanders made it clear he’s upset about high drug prices. Less clear is that his righteous wrath will put patients in harm’s way.  He proved yet again he’s uninformed about the facts and unconcerned about the unintended consequences of his actions.

The target of Senator Sanders terrible tweet was a small innovative pharmaceutical company (Ariad Pharmaceuticals) and its innovative drug Iclusig (for chronic myeloid leukemia treatment). The company raised the price – and Senator Sanders can’t see why. He should open his eyes.

A few facts that are worth sharing. The first is that Iclusig serves a population of approximately 1,000 to 2,000 patients. And these patients have limited options. Ariad Pharmaceuticals, works to ensures that no patient is prevented from treatment due to price. (They provide a robust support program for patients who have accessibility and affordability concerns.)

Second, these types of ultra-orphan disease cancer patient population programs require large investments and face tremendous odds.  Ariad sure doesn’t look profitable. They’ve invested more than $1.3 billion in R&D and accumulated losses of approximately $1.4 billion since the company was founded. But they’re betting on innovation. In 2015, Ariad generated $119 million in total revenue and invested $171 million, or 143% of revenue, in R&D alone.

Third and most importantly, Iclusig works.  It significantly increases 10-year survival rates for patients with no other hope or option except for expensive stem cell transplants and draconian radiation treatments.

Attention Senator Sanders. It’s not just a question of an ecosystem-driven price. It’s also about investment and value – precisely the rationale behind the Orphan Drug Act -- to encourage investment in treatments for small and in this case, tiny, populations. “Facts,” as John Adams quipped, “are pesky things.

Fact Checking Hillary on Drug Pricing

  • 10.23.2016
  • Peter Pitts
As seen in the Daily Caller

Fact Checking Hillary Clinton On Drug Pricing

Both Donald Trump and Hillary Clinton get a lot of flak for lying. But in Secretary Clinton’s latest speech on healthcare reform, she didn’t lie — she just got all her facts wrong.

Clinton used her speech to demonize pharmaceutical companies. She argued that greedy firms are gouging consumers and that government-imposed price controls are needed to protect us from them. Her rhetoric ignores reality and her proposals would harm the patients she wants to help.

According to Mrs. Clinton, Americans are “paying the highest price” for medicines, compared to citizens of other developed nations.  She implied that drugcompanies are overcharging Americans just because they can. It’s not so simple.

The real reason medicines are more expensive in the United States is that the socialized medicine systems in other countries cap prices on innovator drugs, while also rationing their use. Many foreign governments threaten to break ‘drug patents if firms don’t agree to sell their products at below-market rates. As a result, American consumers shoulder a disproportionate share of the world’s research and development burden. That’s “free-riderism” and it’s not fair. It’s also important to note that generic drugs, which account for 85 percent by volume of all the medicines used in the United States, are cheaper here than in Europe or Canada.

 But the answer isn’t to impose our own price caps. That would only discourage research into new medicines. Here’s a fact that Clinton didn’t mention — America invents more than half of new medicines in the world.  Stifling U.S. research would lead to vastly fewer medicines here and across the globe. It’d be smarter economically, and better for patients, to negotiate stronger trade protections to prevent other nations from freeloading off American investments.

Secretary Clinton told her audience that their tax dollars fund drug safety evaluations. They do not. All of the complex and costly clinical trials that must be done to bring a new medicine to market are fielded and funded — 100 percent — by the pharmaceutical industry. They are then reviewed by the Food and Drug Administration. And industry pays for that privilege through “user fees” the FDA collects from pharmaceutical companies.

She also slammed the Medicare Part D drug benefit, touting a doctor’s claim that he “can’t prescribe certain drugs that [his] patients need” because government health programs won’t pay for them. But when it comes to Medicare, that assertion is simply false. Medicare’s prescription drug plans cover, on average, 191 of the 200 medicines most used by seniors.  That’s more than most Obamacare exchange options.

In her assault on capitalism and private enterprise, Secretary Clinton singled out the price of hepatitis C drugs. She claimed that makers of these cures — which are vastly more effective than previous therapies — are gouging Americans.

The truth is radically different – and highly documented. Hepatitis C drugs are now cheaper in the United States than in Western Europe, thanks to a price war between competing manufacturers.  Clinton inadvertently picked an example that proves the free market yields better, cheaper medicines than socialist systems and fix prices and ration care.

Bashing the companies that research and produce the world’s most groundbreaking medicines might give Clinton a bump in the polls. But her reality-free rhetoric has dangerous consequences.

Clinton’s attacks on drug makers have prompted a sell-off of biotech stocks multiple times over the past year.  If companies can’t raise funding from investors, they’ll have to limit new research projects. That means fewer drugs down the road. What’s political expediency worth?

And if Clinton reaches the White House and actually implements price controls, it’s statistically certain Americans will lose out on lifesaving drugs. Price controls in other countries depress research spending by up to $8 billion each year — the equivalent of three or four new drugs, according to a Department of Commerce study.

For a super policy wonk, Secretary Clinton got an awful lot wrong in her recent speech. If she really wants to help patients, perhaps she should pay a little less attention to her focus groups and a little more attention to the facts.

Peter J. Pitts, a former FDA Associate Commissioner, is president of the Center for Medicine in the Public Interest. 
The Wikileaks disclosure of John Podesta's emails reveals that the Clinton campaign considered attacking FDA Commissioner Robert Califf for ties to drug companies as part of a broader effort to divert attention from Secretary Clinton's email scandal.

FDA Commissioner and Clinton Campaign Target Robert Califf, MD

It start with an email from long-time Clinton adviser Mandy Grunwald to Clinton policy adviser Ann O Leary.  The email contains a link to a NY Times article on acting FDA Commissioner Rob Califf who had been nominated by President Obama to become the full time commish.  

On Sep 21, 2015, at 10:33 PM, Mandy Grunwald sent an email to Clintion policy advierse Jake Sullivan and Ann O'Leary,  Joel Benenson a strategist and pollster for the presidential campaign., press secretary Brian Fallon and Communications Director Jennifer Palmieri and asks: Do we want to weigh in on this?

Jake Sullivan responds:   What do you think?

On Mon, Sep 21, 2015 Mandy Grunwald replies: I don't know anything about the guy. If we weren't hitting the Administration with Keystone this week, I might be tempted, but I think that probably makes it a bad idea. Lets keep an eye on it and see about those Pharma ties. 

Then Anne O Leary:  Interesting. I'll do some asking around to see what folks in the public health world think.

On Sat, Sep 26, 2015 at 12:53 PM, Brian Fallon :  Any update on this? As we consider fights that fit into the larger themes we are trying to promote, this seems like a good fight to have. Plus, the VP would be in a box of having to support this nominee.

On Sat, Sep 26, 2015 at 12:59 PM, Jake Sullivan wrote: This is ultimately a political call, not a policy call. I don't really like the idea of bashing this White House's nominees, knowing their vetting process and standards.  But if you guys want to do it, I cannot identify a policy reason not to -- you all know the facts. Ann, any other intel?

On September 26, 2015 at 1:13 pm  Anne O'Leary wrote:

Califf the Obama nominee does have real ties to the drug industry - Chris Jennings is calling a few people for me to learn more so we don't tip our hand directly. We are clean on Clinton Admin FDA Commissioner - it was David Kessler, an academic who had run a teaching hospital - and best known for taking on big tobacco. We could certainly signal that we want someone willing to stand up to Pharma (in the same way Kessler stood up to Tobacco). BUT - I want to do a little more digging and due diligence before we hit this guy. Having been through a nomination fight with my husband (in which he lost), this is personal and messy and horrible on the person nominated and their families - so I don't take attacking this guy lightly. Do you want to do it on Meet the Press? 

That was follwed by an email from Podesta on Sep 26, 2015, at 2:27 PM,  who wrote: " I think we will pay a huge price with the WH on this one. Worries me."

Nothing transpired.   But the back and forth about whether to trash Califf came down to politics, not policy differences.  And it had NOTHING to do with Califf as a person, a physician, a researcher and public servant.  It was clear that Team Clinton was interested only in dirt that was newsworthy.  It was simply a matter of whether attacking Califf on Meet The Press would be worth the headache of taking on an Obama nominee.   How lovely. 

Rob Califf should remain FDA commissioner regardless of who is elected president because of his qualifications and commitment to accelerating access to safe and effective medicines.   I am concerned that the Clinton team -- which exults about going to war against Pharma -- will seek to replace Dr. Califf.  That will trigger a prolonged political war that will undermine the FDA.    

The emails reveal a Team Clinton eager to find ways to smash up people for short term political gain without tipping their hand that they are behind the hit job.   Like it's war on pharma, it has less to do with policies and more to do about the benefits of creating and attacking enemies. 


Without commercial interruption please read this important blog from Drug Channels about how insurers and PBMs are rigging drug prices to maximize profits and discourage access.  

Insurance is -- to paraphrase the song Me and Bobby McGee -- has become another word for nothing left to lose, especially if you are one of the 4-5 percent of patients fighting cancer,  automimmune diseases and orphan conditions.   As Adam points out, the system is rigged against a minority of people.  That's discrimination.  

There is plenty of revenue from PBMSs, insurers and pharma that could be used to eliminate out of pocket spending on drugs.   that mean drug companies can charge whatever they want, as critics claim?  Hardly.   Rather it means that drug spending will have to be part of a solution that are offered to consumers based on what they value.   New medicines do not drive up health care costs long term, rather they make staying healthy more affordable and convenient.    Every economic analysis shows that.  

Instead of finger pointing about who is to blame, it's time to ramp up the shift to figuring out what value-based purchasing is and making it work.  And it's time to find more ways to reduce the cost, time and uncertainty of drug development.   Both challenges can be overcome by applying precision health tools to improve population well-being and leveraging new medicines to deliver even more value directly to people.    


Written by Rafael Fonseca MD and Robert Goldberg PhD

Two recent articles in the NEJM (1) e) and Annals of Internal Medicine (2) propose that restrictions or eliminations of co-pay assistance programs by pharmaceutical companies are needed since they distort the market and therefore, while few benefit, many suffer in the form of higher healthcare expenditures. They are factually correct but the conclusions point to a larger problem; the third party payer system in health care. When consumers (in this case patients) are detached from the price consequence of transactions they will be less careful in their selections. However, the proposal presented in these articles are, at least in the short-term, patient unfriendly and immediate implementation of their recommendations would limit, severely, access; particularly for patients with serious or life-threatening conditions. The Annals article is notorious in that it fails to mention the increased cost-sharing pressure that patients are facing from the payers.

So what are the solutions?  Ideally, healthcare should be divided between the routine care and the catastrophic care.  Routine care should involve much more “skin in the game.”  In my view, regular care should not be covered under traditional heath insurance but be bought directly, with compassionate support tools for those in need.  In this type of care I could envision more direct pressure in the selection of older medications for which many alternative exist. There are many options for anti-hypertensive medications, statins, etc.  In the absence of a third party payer system the Epipen would never cost $600.  This is where consumer pressure, with co-pay requirement as a discriminator, should be employed; vigorously, if you may.  I would not disagree that if an equivalent generic exists, a true equivalent, then having the patient paying more for a branded product is fine.  This is not to say that branded products and generics are always the same.  Sometimes production quality may suffer or sometimes an alternative may not be the same as the original, similar and yet not the same product.  But if you want the branded statin maybe you should pay a bit more.  Who could argue with that?

However, for more expensive medications, medications used for serious, chronic severe or catastrophic illnesses the pressure seems misguided.  For instance preventing co-pay assistance for patients with cancer seems inhumane.  Dusetzina and colleagues have documented that lack of supplemental insurance delays initiation of treatment for CML (3). Should we disincentivize the use of medications that can be lifesaving for a cancer patient so they consider inferior treatments?  Should we place additional burdens in someone who is at high risk for bankruptcy? Someone who cannot work or whose caregiver cannot work?  That seems inappropriate.  Furthermore, for many of these newer and expensive medications there are no suitable alternatives (4).  Dr. Bach and colleagues were able to reduce the price of a competing monoclonal antibody, but that is a rarity in oncology and more of an exception. Should myeloma patients forgo lenalidomide in favor or thalidomide and face certain peripheral neuropathy?  Or should a diabetic myeloma patient be forced to be treated with bortezomib instead of carfilzomib and again face neuropathy? To create a disincentive as this for people facing serious medical illness appears to me as a fundamental violation of medicine’s stance of benevolence. Rather this has the appeal of altruism, sacrifice the few for the benefit of the many.

Lastly, government regulations that eliminate this co-pay assistance are demonstrably patient unfriendly. Commercially insured patients can receive this directly and in another study by Dusetzina she shows that the average co-pay for specialty medications is only $35 (5). In another study it was shown that 90% of myeloma patients pay less than $100 per month for lenalidomide, a backbone for the treatment of this disease. In the meantime, Medicare beneficiaries struggle to find indirect support with funds provided by the pharmaceutical companies to third parties. I’ve written about this topic before, but again is a built-in disincentive to lessen the use of medications (6).  What if these medications save dollars by preventing hospitalizations and decreasing expenses elsewhere?  Once again it is pound foolish to only concentrate on the price of drugs.

1.    Dafny LS, Ody CJ, Schmitt MA. Undermining Value-Based Purchasing - Lessons from the Pharmaceutical Industry. The New England journal of medicine. 2016. Epub 2016/10/13. doi: 10.1056/NEJMp1607378. PubMed PMID: 27732125.
2.    Ubel PA, Bach PB. Copay Assistance for Expensive Drugs: A Helping Hand That Raises Costs. Annals of internal medicine. 2016. Epub 2016/10/11. doi: 10.7326/M16-1334. PubMed PMID: 27723893.
3.    Winn A, Keating N, Dusetzina S. Factors Associated With Tyrosine Kinase Inhibitor Initiation and Adherence Among Medicare Beneficiaries With Chronic Myeloid Leukemia. Journal of Clinical Oncology. 2016;10.1200/JCO.2016.67.4184.
4.    Fonseca R. 2016. Available from:
5.    Dusetzina SB. Share Of Specialty Drugs In Commercial Plans Nearly Quadrupled, 2003-14. Health Aff (Millwood). 2016;35(7):1241-6. Epub 2016/07/08. doi: 10.1377/hlthaff.2015.1657. PubMed PMID: 27385240.
6.    Fonseca R. 2016. Available from:
From the pages of today's edition of the Boston Herald:

Insurers not covering new cancer treatments

Poor insurance coverage is causing cancer patients to miss out on cutting-edge technologies that use gene analysis to determine the best treatments — a fact a former commissioner of the Food and Drug Administration is calling another example of Obamacare’s failure to provide Americans with high-quality health care.

“There’s a degree of dishonesty about what the Affordable Care Act provides, and that is starkly clear when a patient has a serious type of cancer,” said former FDA Associate Commissioner Peter J. Pitts, who now serves as president of the Center for Medicine in the Public Interest. “The soundbite of the ACA is, many more Americans now have health insurance. But the health insurance isn’t worth the paper it’s written on.”

Pitts said companies are covering the old-fashioned, less-effective chemotherapy regimens rather than more sophisticated approaches and prescription benefit managers are opting not to reimburse the more innovative, expensive treatments. Instead, they are negotiating rebates with pharmaceutical companies that they pocket, rather than passing the savings along to the patient.

“It’s time for insurance companies and prescription benefit managers to step up and do the right thing by putting patients first,” Pitts said. “When they choose not to reimburse for a product because it doesn’t earn them enough money, even though it could save a patient’s life, I think it’s despicable.”

And coverage for the treatments tends to vary by tens of thousands of dollars nationwide, depending on the company and geographic region, according to a University of Texas MD Anderson Cancer Center study published Monday in the journal Cancer.

The study found that insurance costs varied by as much as $47,000 for women on a chemotherapy plan that included the drug Herceptin, which is used to treat breast and stomach cancers by keeping cell growth at bay — and out-of-pocket costs range from $2,700 to $3,400.

The extra costs caused by these variations leads to an additional $1 billion spent to treat breast cancer in the U.S. each year, the study found. But cancer patients will often be covered for a treatment if it has been proven effective by the FDA, according to Dr. Harold Burstein, a breast cancer specialist at Dana-Farber Cancer Institute, who said: “The business of cures not being covered is extraordinarily uncommon.”

Many of the additional costs for the patient, he said, come in the form of lost income and child care.

“Those things are harder to measure than direct hospital bills,” he said, “and they generally have more of an impact.”
Anthem Health has issued a decision deny coverage of Exondys 51, the first drug to treat a form of Duchenne’s muscular dystrophy.  As STAT’s Ed Silverman reports : Anthem “claims that the drug is “not medically necessary” and that “the clinical benefit … has not been demonstrated.” Duchenne is a rare disease that confines boys to wheelchairs and condemns them to an early death.

The decision was followed by this: "Exondys 51 failed to show it improves health outcomes, and therefore it is not a covered benefit for our members," Anthem spokeswoman Leslie Porras said in an emailed statement on Friday.

Not exactly.   Anthem conveniently ignored the fact that the FDA does not approve drugs that have no medical benefit or more precisely,  the FDA approves drugs that are likely to promote treatment of specific conditions.  Anthem claims it is denying coverage because a multitude of limitations cast further doubt on the reliability of dystrophin levels as a surrogate endpoint for clinical efficacy in DMD (FDA, 2016).   But these limitations were considered and approval was given.  In fact, in ignoring the FDA’s decision Anthem is taking refuge in the statements of FDA reviewers who raised concerns about clinical trials but were overruled by Janet Woodcock and Robert Califf, the FDA commissioner.  

I wonder if Anthem would have a problem with doctors and its utilization review munchkins ignoring the company’s medical policy on any treatment based on evidence ultimately rejected by Anthem.   And I wonder what a disability rights attorney will do with the fact that Anthem is willfully misapplying the term medically necessary.  

In fact, Anthem has no problem covering acupuncture, massage therapy, spine manipulations and naturopathy.   

Compare Anthem’s demand for more evidence before covering Exondys with the statement accompanying it’s decision to cover acupuncture:

"Acupuncture as a therapeutic intervention is widely practiced in the United States. There have been many studies of its potential usefulness. However, many of these studies provide equivocal results because of design, sample size, and other factors. The issue is further complicated by inherent difficulties in the use of appropriate controls, such as placebo and sham acupuncture groups…Further research is likely to uncover additional areas where acupuncture interventions will be useful.”

Anthem covers treatments that have less medical evidence of benefit for less severe ailments than DMD.  And it covers treatments based on the it’s belief that research will find acupuncture useful” (though not clinically effective.)   That’s because they attract millions of fairly healthy consumers.

There are about 17000 boys with DMD.    Providing the drug also means paying for all the other supportive services, emergency care, hospitalizations that such kids may require.  To Anthem, spending money to help boys with DMD stay independent and alive is a money loser. 

That’s the only evidence Anthem cares about.  

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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