Drug Importation and John McCain's Flying Pig

12/03/2015 07:33 AM |

John McCain has always supported drug importation “from Canada.” And he has been wrong every time.

He has resurrected his effort (via Amendment SA 2884) and cites the Turing Pharmaceuticals example, “"Don't you feel when people are paying $750 for a pill that maybe they ought to be able to go to Canada and buy one that's reasonably [priced]?”

Note to Senator McCain –the Turing product is an off-patent drug but there isn’t any generic competition. And that’s important because generic drugs are less expensive in the US than in Canada. The way to drive down the prices of single-manufacturer off-patent drugs isn’t to import foreign price controls -- it’s to inject healthy competition into the equation by making it worthwhile for multiple manufacturers to offer products like Turing’s infamous Daraprim.

As to the broader arguments against drug importation – here we go again.

Importing drugs from Canada is exceedingly dangerous for a number of reasons. For starters, many Internet pharmacies based up north are stocked with drugs from the European Union. And while many wouldn’t hesitate to take medicines purchased from countries like France and Great Britain, there’s plenty of risk involved.

The EU currently operates under a system of “parallel trade,” which allows products to be freely imported between member countries. This means that any drugs exported from the U.K. to Canada could have originated in an EU country with significantly less rigorous safety regulations like Greece, Portugal, Latvia or Malta.

Just last year, EU officials seized over 34 million fake pills in just two months. And in May, Irish drug enforcers confiscated over 1.7 million pounds of counterfeit and illegal drug packages. So if American customers start buying drugs over the Internet from Canadian pharmacies, they could easily wind up with tainted medicines of unknown European origin.

It’s also important to note that drugs from anywhere in Europe aren’t even legal for sale in Canada. So when politicians say we can get “the same drugs” that Canadians get, they’re just plain wrong.

Even more worrisome is outright fraud — many “Canadian” pharmacies are actually headquartered somewhere else.

A 2005 investigation by the Food and Drug Administration looked at 4,000 drug shipments coming into the U.S. Almost half of them claimed to be from Canada. Of those, a full 85 percent were actually from countries such as India, Vanuatu and Costa Rica.

As part of another investigation, FDA officials bought three popular drugs from two Internet pharmacies claiming to be “located in, and operated out of, Canada.” Both websites had Canadian flags on their websites. Yet neither the pharmacies nor the drugs were actually from Canada.

As an FDA official told Congress, “We determined there is no evidence that the dispensers of the drugs or the drugs themselves are Canadian. The registrants, technical contacts, and billing contacts for both web sites have addresses in China. The reordering website for both purchases and its registrant, technical contact, and billing contact have addresses in Belize. The drugs were shipped from Texas, with a customer service and return address in Florida.” And in laboratory analysis, every pill failed basic purity and potency tests.

The on-the-ground reality of state and local importation schemes has been dismal and politically embarrassing. Remember Illinois’ high profile “I-Save-RX” program? Over 19 months, only 3,689 Illinois residents used the program—that’s .02 percent of the population.

And what of Minnesota’s RxConnect? According to its latest statistics, Minnesota RxConnect fills about 138 prescriptions a month. That’s in a state with a population of 5,167,101.

Remember Springfield, Massachusetts and “the New Boston Tea Party?” Well, the city of Springfield has been out of the “drugs from Canada business” since August 2006.

And speaking of tea parties, according to a story in the Boston Globe, “Four years after Mayor Thomas M. Menino bucked federal regulators and made Boston the biggest city in the nation to offer low-cost Canadian prescription drugs to employees and retirees, the program has fizzled, never having attracted more than a few dozen participants.”

The Canadian supplier for the program was Winnipeg-based Total Care Pharmacy. When Total Care decided to end its relationship with the city, only 16 Boston retirees were still participating.

Programs like this wouldn’t do any better on a national basis. A study by the non-partisan federal Congressional Budget Office showed that importation would reduce our nation’s spending on prescription medicines a whopping 0.1 percent—and that’s not including the tens of millions of dollars the FDA would need to oversee drug safety for the dozen or so nations generally involved in foreign drug importation schemes.

Calling foreign drug importation “re-importation” is a clever way to sell the idea to the American people. But the term simply doesn’t fit with the facts. In reality, in addition to importing foreign price controls, Americans would end up jeopardizing their health by purchasing unsafe drugs while not saving money.

McCain acknowledged that similar efforts to legislate drug importation had fallen short many times in the past, but said, "sooner or later, even pigs fly.

Think again, Senator McCain.

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Doubling Down on Health Care Discrimination

12/01/2015 05:56 PM |

In proposing steps towards value-based drug pricing Peter Bach proceeds from several false assumptions to justify proposals that would make drugs less affordable and give health insurers even more control of the drug prices Bach seeks to regulate. 

He claims that drugs are the “only major category of health care services for which the producer is able to exercise relatively unrestrained pricing power.”  He states that “by law, drug manufacturers can set the price that Medicare and Medicaid programs pay for new drugs, and they also benefit from significant negotiating advantages over private insurers, who are required to cover most new drugs and are unable to obtain significant price concessions from manufacturers, particularly for drugs that offer some clinical advantage or use alternative mechanisms of action compared with available treatment options. As a result, drug prices in the United States are generally 2 to 6 times higher than prices for the same drugs in other major industrialized nations.

These statements are incorrect and the assertion that free market pricing leads to high drug prices in the United States compared to “other major industrialized nations” is largely untrue.

Like every other health care good and service, drug prices are negotiated by private insurers and government health programs.   Bach’s description of Medicare and Medicaid’s approach to drug prices also characterizes how wheelchair and hip replacement prices are established. 

The average discount of off the Average Wholesale Price (a sticker price if you will) is anywhere from 10-50 percent on new medicines.  Those discounted prices are about the same as the prices listed by health systems in Germany, UK, Canada, etc.   The negotiated price for Solvadi in Germany is $46625.  In France, it’s $51000. 

In the United States?  The government programs got a 65% discount. That’s $30000. Private health plans received a 40 percent discount.  That’s $50000.

Bach usually fails to ask if these discounts ever make it to the patients that will be using the drugs.   This, from someone who raised the same question about hospitals marking up drugs discounted for poor patients and re-selling them at a huge markup.  

Some of the biggest health plans take the discounted drugs and then require patients to pay up to 50 percent of the cost of the drug.   The drug price we see is therefore set by PBMs and insurers.   And it is never the price they get the drug for in the first place.

Bach never questions the morality of such business practices.  Indeed, he has successfully avoided answering an inconvenient question:  If new drugs for cancer, heart failure, Hepatitis C etc. are en route to bankrupting America, why are they only 2 percent of the total insurers spend on health care each year.   Bach’s false assumptions are designed to divert attention from this fact.  And to justify a proposal that would give PBMs and insurers even MORE control over access to new medicines and their prices.

Bach and his co-author Steven Pearson have produced an interesting variation of value-based health insurance design.  The would reduce out of pocket cost sharing for drugs that work or benefit patients, extend patent protection to those that demonstrate value in new patients with other illnesses and discourage use of medicines that don’t work.

That sounds sensible.  However, Bach and Pearson would impose this value-based design on patients.  And who would define value?  The payors and PBMs who pocket rebates, re-sell discounted drugs to patients for a profit, force patients to pay a share of the marked up price for drugs they need and use cost sharing and fail first programs that make people sicker and ultimately discourage patients from enrolling in their plans in the first place

In fact, Bach and Pearson’s approach – a one size fits all value that gives PBMs and insurers to cut deals and design drug plans that maximize profits at the expense of the sickest patients -- would legitimize this questionable and counterproductive practices. 

In the absence of dramatic reductions in the time, cost and uncertainty associated with developing new medicines for smaller groups of patients, prices will be what they are.  

The best way to reduce the cost of health care is to insure that more people get the treatment best for them the first time.   We have the ability to customize treatments to patients and replace large upfront costs with long term financing for many medicines.  We are barely using precision medicine tools that would enable such a patient-driven approach.  And what we learn from these new approaches can be used to improve medical care and make it more predictive, prospective, personalized and participatory.   But Bach and Pearson ignore these tools. 

Hence, the value based approach to drug pricing as proposed by Bach and Pearson institutionalizes the marginalization that sustains many (but not all) PBMs and insurers.  Value-based drug pricing is nothing more than the health care policy way to pretend that separate and equal is not discrimination. 

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A Pharma Pile-On

11/30/2015 07:01 AM |

An interesting look at the current contretemps facing the pharmaceutical industry, the FDA, American healthcare, and some of our candidates for the Presidency -- from the pages of Medical Marketing & Media.

The article is titled, "A Pharma Pile-On" and can be found here.

An important and enjoyable read.  Read More & Comment...

Why I Am Very Thankful For Pharma

11/25/2015 02:42 PM |

The last few weeks have been unsettling. There is a foreboding and unease about radical Islam, the fear, to paraphrase Winston Churchill that “the whole world, including the United States, including all that we have known and cared for, will sink into the abyss of a new Dark Age made more sinister, and perhaps more protracted, by the lights of perverted science.”

Churchill inserted this last sentence of his 23 page typed speech with a red pen.  Many historians have speculated about why he decided to close with such a warning and what he meant by perverted science.

In an article in The New Atlantis, Justin Lyons observes that Sir Winston was referring to the Nazi’s use of technology to wipe out Western civilization.  In one of his many books, Churchill wrote: The material progress that science offers is “really only valuable in so far as it liberates the innate goodness of the human heart. It would not be a blessing but a curse if it rolled forward uncontrolled by the moral principles of simple decent men and women. It can never be our salvation. It may be our doom.” 

The Nazis – as do the radical Islamist states of today – seek to use technology to obliterate, imprison and enslave humanity, to pave over the moral principles of most decent men and women and rebuild society according their worldview.  These two evil forces have in common a belief that have absolute certainty.

As Jacob Bronowski, biologist and philosopher wrote in “The Ascent of Man”:

The Principle of Uncertainty fixed once for all the realisation that all knowledge is limited. It is an irony of history that at the very time when this was being worked out there should rise, under Hitler in Germany and other tyrants elsewhere, a counter-conception: a principle of monstrous certainty. When the future looks back on the 1930s it will think of them as a crucial confrontation of culture as I have been expounding it, the ascent of man, against the throwback to the despots' belief that they have absolute certainty.

We must guard against this mindset ourselves.  Bronowski assert that Heisenberg’s Uncertainty Principle should be called the tolerance principle.  As Simon Critchley notes: “The relationship between humans and nature and humans and other humans can take place only within a certain play of tolerance. Insisting on certainty, by contrast, leads ineluctably to arrogance and dogma based on ignorance. “

This lack of tolerance shows up in the discourse and debates of today.  Setting aside the campus “crybullies” who have captured our attention, the current debate about drug prices – one of many over the past 50 years – is controlled by those who believe they have absolute certainty that by rationing drugs based on their measure of what a life is worth or what institutions they control should pay, we can save money and sustain medical innovation. 

Whereas, human knowledge is personal and responsible, an unending adventure at the edge of uncertainty, there are those who wish to subvert science to their specific social and political agenda.   They justify restricting access to new medicines and reducing drug prices without reducing the risk and cost of developing treatments by claiming – with absolute certainty – that doing so is necessary to keep America from going bankrupt and that doing so would actually increase innovation.

As I have written elsewhere, if the absolutists had won the day in the times past when they proposed the same policies, we would be bereft of the many medicines for HIV, TB, heart disease and various cancers that saved lives and transformed a difficult problem into a world filled with opportunity.  We would die sooner, suffer more, love, learn and live less. 

I am thankful to pharma for it is truly – warts and all – the most fruitful, sustained investment in the most personal and responsible manifestation of human knowledge: increasing the sustainability of humankind.  It is the highest, most human use of free market capitalism and technology the world has ever seen.  Pharma has contributed more good to our planet in a shorter period of time than any other commercial enterprise, precisely because it is always pushing back against certainty, against what is already known.  And yes, at the core of this enterprise is not just a moral code, but a moral code that has shaped decent and just societies throughout history; every life – no matter who or where or how sick – has absolute value. 

To pursue or use science to replace that moral code (with QALYs based on comparative effectiveness)  is to pervert science anew.
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Healthcare Reform? Don't Give Up the Ship.

11/25/2015 07:24 AM |

Expert: Too soon to start writing Obamacare's death certificate

WASHINGTON (Sinclair Broadcast Group) — The Department of Health and Human Services (HHS) insisted Tuesday that the individual health insurance markets under the Affordable Care Act (ACA) have a strong future, despite a warning from one of the nation's biggest insurers that it may drop out of the markets due to unsustainable losses.

UnitedHealth Group (UNH) made the announcement Thursday, saying that the pool of customers that have enrolled through the marketplaces is older and sicker than anticipated. The lack of new healthy customers has led to higher costs than originally projected.

"The Company is evaluating the viability of the insurance exchange product segment and will determine during the first half of 2016 to what extent it can continue to serve the public exchange markets in 2017," UNH said in a statement.

While UNH is the country's largest insurer overall, it has had a relatively small role in the individual markets, covering about 500,000 of the 9.9 million total customers there. If UNH actually does leave and insurers that control a bigger piece of the individual market follow its lead, the viability of the system could be in question. There is no indication they intend to do that, though.

"We can't really subsidize a marketplace that doesn't appear at the moment to be sustaining itself," UNH Chief Executive Officer Stephen Hemsley reportedly told investors on a November 19 conference call.

HHS remains confident, though, noting statements from several other big insurers that their business on the health exchanges has been in line with their expectations and they are still committed to the program.

"This is further indication that statements from one issuer are not reflective of the Marketplace's overall strength going forward," HHS said in a statement.

The exchange markets, which launched in 2013, were always expected to take up to five years to stabilize, Hospital Corporation of America management told UBS analysts.

Taken with reports of co-ops failing, premiums rising, and enrollment projections being cut, critics of the ACA, also known as Obamacare, have argued these developments are proof the whole program is in a "death spiral."

Some experts say such pessimism is unwarranted at this point.

"It's important to understand that UnitedHealth Group...has never been a big player in what we call the individual or non-group market," said Sabrina Corlette, a research professor at the Georgetown University Health Policy Institute.

Before and after the ACA took effect, the company was hesitant to offer plans on the individual markets. When it did, it was sometimes one of the higher-priced options, Corlette said.

"It's never been my sense that this is a market that they've embraced with any sort of enthusiasm."

The individual marketplaces have presented "a very challenging environment for insurers," though.

According to Corlette, the ACA has required a fundamental shift in the way health insurance companies do business from avoiding risk to managing risk, since they can no longer exclude sick patients with pre-existing conditions. The individual exchanges have not been very profitable for anybody at this point and the big insurers have struggled with them.

For the companies that are patient, though, she said the markets could become an opportunity for growth if they eventually stabilize and the risk pools balance out. How long insurers are willing to wait for that to happen is an open question.

"I don't think any of these companies want to see losses year after year after year," Corlette said.

The UNH news comes after several of the non-profit co-ops established under the ACA to compete with for-profit insurers failed and some customers seeking insurance for 2016 found higher prices and deductibles than they expected. Also, the government has decreased its projection for new enrollees, in part because fewer people than expected have lost employer-provided coverage since the ACA was implemented.

Some of the challenges currently facing the ACA were predictable when it was passed five years ago, Corlette said, adding that patient advocates expressed doubts at the time about the affordability of insurance under the law.

"A number of the insurance companies are complaining that the population they are seeing is sicker than they expected," she said, and they have faced difficulties adapting to the law.

Some of the problems may also be temporary. According to Corlette, mitigating factors could make the individual markets more profitable in the coming years.

As the penalty for failure to sign up for health insurance rises, the cost of not complying with the individual mandate could motivate healthy people to seek out insurance on the exchanges. Also, a provision that allowed people to keep some pre-ACA plans is expiring. Those consumers are often healthy people who did not need comprehensive coverage, so the risk pool could widen if they then transition into the marketplace.

"I would not write anybody's death certificate yet," Corlette said.

Other health care analysts agree that UNH leaving the individual exchanges would be more of a symbolic loss than a fatal blow to the program.

"UnitedHealth isn't yet a big ACA player and came late to the party. But their concerns show why enrollment growth is so important," tweeted Larry Levitt of the Kaiser Family Foundation.

"No--emphatically no--Obamacare is nowhere close to collapsing," wrote Wendell Potter on healthinsurance.org. "In many regards, it is a big success, much to the chagrin of the law's critics."

"United pulling out of Obamacare might signal something even more troubling: that the marketplaces aren't a good business decision for other large carriers," said Sarah Kliff of Vox. "Right now, that doesn't seem to be the case."

"Yes, the Obamacare exchanges are proving to be as dysfunctional as many conservatives had anticipated," wrote Reihan Salam on Slate. "But until Republicans can make a convincing case that they will do a better job of meeting the needs of Americans with pre-existing conditions, Obamacare is here to stay."

Some liberal commentators have a more upbeat assessment of the ACA's progress.

"Obamacare has hit a few rough patches lately," wrote economist Paul Krugman in the New York Times. "But they're much less significant than a lot of the reporting, let alone the right-wing reaction, would have you believe. Health reform is still a huge success story."

Krugman characterized the 11% average increase in premiums for 2016 as a "slight disappointment," noting that increases in the two previous years were smaller than expected.

However, Peter Pitts, president and co-founder of the Center for Medicine in the Public Interest, cautioned that UNH's announcement may be "an early indicator" of things to come.

"It is an indicator that these types of insurance designs are not profitable," he said. He called the need for healthy customers to balance out the risk and reduce the cost of coverage "the cardinal flaw in the whole ACA concept."

Any insurance business is built on a model where premiums of people who buy policies and do not use them help cover the costs of services for people who do use them. If the people who need to use the insurance are all there is, the system falls apart.

"I think there's disappointment on the provider and participant levels," Pitts said of the current state of the individual exchanges.

He could not speculate about whether UNH is serious about dropping out of the marketplaces, but if the company's projection of $500 million in losses in 2016 is accurate, it would make sense for them to stop offering the individual plans.

"Public companies do not pursue programs that cost the company hundreds of millions of dollars in losses," Pitts said. "For that, rocket science is not required."

He said he anticipated many of the challenges the ACA is facing now back before it was passed in 2010. The reliance on healthy young people to buy insurance they do not want is one of the underlying problems he saw, and the effects of that are being seen in rising premiums and companies leaving the exchanges.

"It's fair to say that the foundational design flaws are becoming evident, and the question now is what you do about it," Pitts said. He argued that these problems with the law need to be fixed before the situation becomes a crisis.

Others see a bleaker future for the law.

"For anyone involved with the Affordable Care Act, yesterday was a big day," wrote Robert Laszewski, a health policy and strategy consultant, Friday. "It's the day any vestige of the notion that the Obamacare insurance exchanges have a chance of being successful in their current form lost whatever credibility was remaining."

"These problems aren't incidental to Obamacare," said Sally Pipes of the Pacific Research Institute on Forbes.com. "They're the inevitable result of the law's central premise--that lawmakers and government bureaucrats can make better decisions about small businesses' health care than millions of businesses themselves can."

"This may be the year of the health care law's collapse," wrote Rep. John Barrasso (R-WY) in an op-ed days before the UNH report, citing rising premiums and the failure of the co-ops.

The public seems skeptical of the Obama administration's position that the law is working. According to a recent Kaiser Family Foundation poll, 45% of respondents view the ACA negatively and 38% have a favorable opinion. 30% support a full repeal, 12% want to scale it back, and 26% want to expand it. The opinions largely break down along party lines.

Democratic presidential candidate Martin O'Malley unveiled his plan for health care reform Tuesday with new measures aimed at controlling costs.

Front-runner Hillary Clinton has her own plan that would make some changes to the system.

The other Democratic candidate, Bernie Sanders, has endorsed a national single-payer system, but the exact details of how that would be paid for are unclear.

 Republican candidates have proposed complete overhauls of the system that begin with the repeal of the ACA.

Pitts believes it will be hard for politicians to sell a repeal plan that takes insurance away from those the ACA has helped, but he said it is difficult to determine exactly how many people have benefited from it. At the same time, he worries about the consequences for the public if the law's apparent shortcomings are not addressed in some way.

"Nobody should go down with the ship. Least of all, patients."

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Doctors Still Shouldn't Use Cost To Decide What Drugs To Use

11/23/2015 01:56 PM |

In a recent Newsweek article I criticized the value framework applied by Daniel Goldstein and colleagues in Necitumumab in Metastatic Squamous Cell Lung CancerEstablishing a Value-Based Cost.

Goldstein had the chance to write a reponse in Newsweek to suggest I was wrong on the facts.

You decide.

Goldstein writes:  In “Should Doctors Worry About the Cost of Extending Life?” Robert Goldberg suggests that our study was endorsed by the American Society of Clinical Oncology (ASCO), which is simply incorrect.

ASCO has recently developed a method to assess the value of cancer drugs, but it uses a completely different methodology. Our study used very sophisticated economic techniques that are used by researchers in many countries worldwide to guide coverage decisions. With value-based pricing, we proposed that the price of a drug should simply be linked to the benefit that a drug provides.

My Response:  Dr. Goldstein plays a prominent role at ASCO conferences where he presented his value framework as an example of how to limit access based on price. That’s not a formal endorsement but Goldstein’s work was highlighted by ASCO media outlets, etc.    The fact that he claims to use “very sophisticated economic techniques that are used by researchers in many countries worldwide to guide coverage decisions” and measure value that ASCO has highlighted as opposed to getting ASCO’s seal of approval  is hair splitting. 

In fact, it is these techniques (which are not very sophisticated) are what I criticized.  My problem is not with the methodology but it’s use, which I believe is unethical.   

Goldstein claims: “Goldberg also does not seem able to perform basic arithmetic. He suggests that we put a price of $20 on each day of life. If this were the case, it would amount to $7,300 per year.”

Me Again:  Here's what Goldstein concluded: "These findings provide a value-based range for the cost of necitumumab from $563 to $1309 per cycle".   Patients in the SQUIRE clinical trial comparing necitumumab with standard treatment for advanced head and neck cancer received six cycles of the new drug.    Multiply 563x6 = $3378.    So I should have divided $3378 by 365 days.  That would be $9 a day.  At the upper limit of price to value established by Goldstein, (1309x6= $7854) comes out to $21 a day.

Finally, Goldstein says I am not “understanding complex policy issues.”  If stating at pricing drugs based on an arbitrary measure of value, a measure that is not scientific, but a rule of thumb is wrong is a reflection of my lack of insight I plead guilty.   Similarly,  if pointing out that the use of necitummab is the first treatment to extend the lives of people in 20 years is valuable for reasons beyond average survival in a clinical trial shows my ignorance, well I wear that dunce cap with pride.


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Bass Pfishing and Hedge Fund Hijinks

11/23/2015 07:35 AM |

From today’s edition of the New York Post …

The hedge-fund ploy that’s aborting miracle medicines

Hedge-fund investors are coming to raid your medicine cabinet.

Cutting-edge treatments for diseases affecting millions of Americans are threatened by Wall Street’s latest moneymaking scheme. The ploy: Hedge funds bet against a drug company’s stock price, launch attacks against the firm’s patents on its best-selling medicines and then reap windfall profits when investors panic and the stock price plummets.

Unless Congress protects pharmaceutical-research firms from these assaults, funding for drug discovery will dry up and many new treatments won’t ever make it into the hands of patients.

Hedge-fund manager Kyle Bass pioneered the strategy, which relies on a new legal procedure known as “inter partes review,” or IPR. This year, Bass — who previously made $590 million betting that homeowners wouldn’t be able to make their mortgage payments during the financial crisis — filed a review against Acorda Therapeutics’ patent on a drug that helps multiple-sclerosis patients walk. The challenge caused Acorda’s share price to crash by 10 percent.

Wall Street began exploiting the system after Congress created the Patent Trial and Appeal Board, a new arm of the Patent Office tasked with taking a second look at patents that some consider too vague. Since its formation in 2013, the board has proven so hungry to annihilate patents that it’s been called a patent “death squad.” Even the board’s own chief judge has started to admit “the number of patents [going down] is starting to get excessive.”

Bass quickly saw how the board was wiping out patents and jumped at the chance to make money from a self-fulfilling prophecy: challenge the patent and short the company’s stock to profit on the market’s reaction.

Other funds have copied Bass’ strategy. The Mangrove Partners Master Fund acquired a 270,000-share “short” position in VirnetX, which owns patents for a Internet-privacy technology. Within weeks of filing two reviews against VirnetX patents, Mangrove unwound its short position, profiting from an 8 percent drop in the stock price.

Although these reviews can be filed against any patent, they’re particularly threatening to medicine patents and the companies that own them. David Winwood, an executive at Louisiana State University’s Biomedical Research Center, explains that “most often, a startup company’s sole asset is a patent application or issued patent, upon which the company’s fortune lays.”

With the cost of developing a new drug estimated at nearly $2.6 billion, biotech companies need strong patent protection to attract funding and keep competitors out of the marketplace while they recoup their investment. Wall Street’s abuse of these pharmaceutical reviews weakens patents and makes medical research look increasingly like a poor investment.

And with more than 110 petitions filed against pharmaceutical patents in fiscal 2015 — roughly double 2014’s total — the threat to R&D funding is only growing.

Fortunately, Congress can protect drug-development efforts while still enabling judges to weed out bad patents.

Under the Hatch-Waxman Act, more than 80 percent of drug patents are already challenged by companies seeking to enter the market with generics — compared with only 14 percent in the 1990s. Another law, known in Washington as “BPCIA,” also encourages challenges to pharmaceutical patents.

In short, if a drug patent is actually too vague, generic manufacturers can challenge and invalidate it and enter the market. IPR challenges simply aren’t necessary.

That’s why Congress needs to exempt pharmaceutical patents approved by the FDA from these challenges. Such an exemption would ensure that Wall Street profiteers don’t deprive innovators of research funding, while still being able to take down vague tech patents.

Preserving the existing patent-challenge procedures will ensure that companies have the confidence to invest in research and development. The laws give patents adequate protection, enabling firms to recoup the decades and billions of dollars invested in drug research.

Armed with that confidence and shielded from hedge-fund attacks, research firms will continue to create the innovative, life-saving treatments patients need.

Peter J. Pitts, a former FDA associate commissioner, is president of the Center for Medicine in the Public Interest.

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Express Scripts Pulls a Putinesque Move on Linden

11/20/2015 11:16 AM |

PBMs – most particularly Express Scripts -- are using their near monopoly control over prescriptions to shakedown smaller specialty pharmacies and vulnerable patients.  Imagine if Vladmir Putin ran a PBM: that comes close to the business practices of Express Scripts.

PBMs generate according Credit Suisse about $90 billion in revenue – all from drug companies paying them rebates -- because they have been given the near monopoly power to determine who gets what medicines and how much they will pay for them.   PBMs demand rebates in exchange for giving drug companies preferred position on formularies.  (They are also rumored to demand a discount off of the already discounted price Rx companies give before rebates.) 

On top of that, PBMs then – in partnership with many health insurers – take the drugs that they have gotten at a discount and put them in the highest cost sharing tier for the sickest patients, those with cancer, autoimmune disorders, HIV, hepatitis C and chronic pain.  And at that point, drug companies – who have already pledged rebates of up to 40 percent of something near the agreed upon sale price – pick up a lot of the out pocket cost that the PBMs have dumped on patients.  

It gets even better.  To top it off, the PBMs, led by Express Scripts, have been portraying themselves as the thin blue line between profiteering specialty pharmacies and the same consumers they are screwing to fatten their margins.

The Valeant controversy prompted journalists to wonder if other specialty pharmacies were owned and operated by drug firms whose products they distributed.
Indeed, in recent weeks Express Scripts (ESI) has capitalized on the journalistic frenzy feeding over the relationship between Valeant and Philidor to shove aside many specialty pharmacies, replacing these entities with ESI’s own retail pharmacies and self-serving drug formularies without notice.

That was enough cover to permit Express Scripts and other PBMs consolidate its already substantial control over prescription drug sales and increase profit margins by tearing up contracts with any specialty pharmacy that distributed unique drugs to specific groups of patients.

The most recent example of this Putin-like business practice:  Express Scripts sudden decision to terminate a relationship with a specialty pharmacy company called Linden.  Because Linden Care manages the pain meds of many patients using drugs made by Horizon Pharma, Express Scripts smeared Linden as another Philidor.

And if anyone had taken the time to look at what Linden did, it would discover (it took me all of 10 seconds) that Linden is not a ‘captive pharmacy.’ Horizon does not own Linden’s business.  Moreover, specialty pharmacies like Linden that are focused on pain management have state-of-the-art security systems, sophisticated software to identify and weed out “doctor-shopping” patients and corrupt prescription factories.  They work closely with the Drug Enforcement Agency and local law enforcement issues to prevent fraud and abuse.  Most importantly, they ensure that patients suffering from chronic and severe pain can access the medicines they need.  Which is why Horizon and many other companies with pain management products use such niche pharmacies.

In contrast ESI just terminated the contract, forcing patients to find – without notice – a new retail pharmacy under ESI’s control. As Adam Fein recently asked:  PBMs routinely monitor their networks, why did it take a highly publicized pharmacy meltdown before PBMs finally cracked down?

Maybe it’s because ESI wanted the business.  Maybe it was because it also has its own specialty pharmacy Accredo that has a larger share of Horizon’s business than does Linden.

CMPI has dealt extensively with the safe distribution and use of pain meds.  Linden is the gold standard for doing so.  Pain management is a highly specialized medical field, with doctors, pharmaceutical companies, and pharmacies working together to provide vital care to patients in severe chronic pain while ensuring adherence to the strictest standards of compliance and regulations in the industry.

Linden is asking a federal court to put the termination on hold so, at the very least, it can negotiate a new contract with ESI and assure that in the interim patients are not endangered.  It is within the jurisdiction of the court to ask: How many patients will be deprived of – or forced to change their medicines -- because of this shift?  Read More & Comment...

Turkish Delight

11/19/2015 09:24 AM |

If the earth were a single state, Istanbul would be its capital.

                                                                        -- Napoleon Bonaparte

I’ve just returned from the World Cancer Leaders’ Summit in Istanbul. (The event was held in partnership with the International Agency for Research on Cancer, the International Atomic Energy Agency (IAEA), and the World Health Organization.) It was a Turkish delight in more ways than one.

I had the opportunity to talk about the value of quality as it pertains to access. Beyond discussing bioequivalence, biosimilarity, and the many and varied issues pertaining to 21^st century pharamcovigilance, my major points were that (1) the most expensive drug is the one that doesn’t work and, (2) broader access to poor quality medicines is not a public health victory.

A key learning for me was that, when it comes to “the market” and “society" -- the market is society. That’s important to remember whether the debate is about access in the Developing World or price/value in the First World. We are all in this together.

During my session’s Q&A a representative from India accused me of slamming generic drugs. I told him that he was hearing what he wanted to hear. I reiterated that generic drugs play a crucial role in access to healthcare around the world – but that quality is a non-negotiable variable for all drugs (both innovator and generic). I reminded him about what I’d said no more that 90 seconds earlier about the USFDA’s new Super Office of Pharmaceutical Quality – that we must approach the issue with “one quality standard” for all medicines.

After all, per the Journal of Infection (2008;56:35-9), “Nothing is more expensive that treatment failure.” In the 21^st Century, pharmacovigilance must be about ensuring predictable patient outcomes.

The Indian gentleman’s question highlights an important problem – what happens when your desires do not match the facts? The answer is that we have to listen with an open mind, carefully, and respectfully. We all have to enlarge our circle of policy colleagues. I’m pleased to report that he and I had a long and productive chat afterwards.

The theme of the event was, “Effective international collaboration … across country borders, across disease groups and, through public private partnerships.” It's a small world after all and, at least for a few days, Istanbul was indeed it’s capital.

  Read More & Comment...

Should Doctors Worry About the Cost of Extending Life?

11/17/2015 10:04 PM |

My article in Newsweek

Oncologists are supposed to treat cancer patients. But some have decided instead to set themselves up as judges of what patients' lives are worth.

This type of bean-counting ought to be abhorrent to anyone who calls himself a physician.

A recent article in JAMA Oncology is emblematic of this strain of thinking. The piece employs a "value framework" endorsed by the American Society of Clinical Oncologists to determine what a new drug "should" cost. Once approved by the FDA, the drug in question, necitumumab, will be the first of its kind to treat stage IV squamous non-small-cell lung cancer.

The lead author of the article, Daniel Goldstein, noted that adding necitumumab to the standard combination of drugs for such patients "only" adds about two months to patients' life, on average. As a result, he calculates, necitumumab should cost $600 a month.

Put another way, Goldstein believes that each extra day of life is worth, at most, $20. About the same price as a new toaster.

Goldstein's morbid conclusion demonstrates the callousness of ASCO's value framework, which seeks to derive "appropriate" drug prices from their average effect on longevity. In the process, the framework marginalizes the value of life.

Goldstein notes that "[t]here is a desperate need to find appropriate prices for new treatments while maintaining incentives to drive game-changing innovations." Yet his approach would whittle away payments for the hardest to treat cancers and for patients who have had no real advances in care for decades.

Necitumumab is not just another drug. As Nick Thatcher, the lead investigator for the necitumumab clinical trials, observed: "I think it's important to remember we're dealing with squamous cell lung cancer," which has proved notoriously difficult to treat. "It's the first time," he notes, "that we've seen benefit in this group of patients over the last 20 or 25 years." In that context, demonstrating any effectiveness at all counts as a breakthrough.

History indicates that more profound breakthroughs will follow, as researchers build on each incremental advance. The first anti-AIDS treatments added "only" two months of life in 1987. By 2006, a combination of new drugs—tailored to the particular biology of the patients—added 15 years of life. The value framework doesn't account for increasing medical progress over time.

Moreover, the value framework does not measure the value of living longer from the patient perspective. The five-year survival rate for those diagnosed with this form and stage of lung cancer is 1 percent. Isn't every extra day with loved ones—and beating cancer—worth more than $20?

Goldstein's conclusion—that many oncology drugs in clinical practice in the United States would fare poorly in cost-effectiveness analyses—could be applied to lung cancer treatment prices as a whole.

Since 1973, the average survival generated by any type of chemotherapy is 1.7 months. The average cost of treating lung cancer patients in the last year of life is $94,000. Drugs are about 20 percent of that spending. Why not let people die 1.7 months sooner and save the money?

When insurance companies try to control costs by restricting access to treatments, they are rightly lambasted for it. Doctors who seek to determine which drugs are worth paying for are no better.

We would not countenance paying more to extend the life of a rich man than a poor one, whatever the difference in their economic "value." Nor would we deem the life of a mother of five more worth saving than that of a childless woman, without a family to mourn her passing. But it is no more acceptable to average the social value of their lives in deciding whether it's worth it to treat them.

A cynic, it is said, knows the price of everything and the value of nothing. By this measure, ASCO's framework is cynical in the extreme. The only framework worth defending is one that says life is too precious to put a price on it.  Read More & Comment...

A Garden (State) Variety Bad Idea for Drug Pricing Reform

11/17/2015 10:47 AM |

Prescription drug plan is bad medicine for N.J. economy

White House contenders want to address – but don't understand – prescription drugs prices. The Center for American Progress has a plan. A bad one. The CAP plan would effectively turn the U.S. government into the world's biggest intellectual property thief, thus gutting pharmaceutical innovation and crippling New Jersey's economy.

New medications are expensive to develop. On average, it costs $2.6 billion and it takes a decade to bring a new medication successfully to market.

One of the reasons the process is so expensive is that most potentially promising compounds don't pan out. The vast majority of compounds never make it from the lab to the clinical trial stage, and the FDA approves only 12 percent of those that do.

Only a small percentage of approved medications ever recoup their development costs. Pharmaceutical companies have to set prices to cover the costs of both the occasional successes and the many, many, many failures that go along with them.

CAP's 45-page report ignores this reality and offers to lower drug prices by stripping pharmaceutical companies of their intellectual property. The federal government, CAP says, has the authority to license patents to knock-off generic manufacturers any time it deems brand-name drug prices are too high.

The authority for this heist is supposedly a 1980 federal law known as the Bayh-Dole Act, which ushered in a Golden Age of pharmaceutical innovation by shoring up intellectual property rights.

One provision of Bayh-Dole holds that the government retains so-called "march-in" rights to license a patent when its owner fails to take "effective steps to achieve practical application of the subject invention." In other words, if a patent is languishing unused, the feds can license it to encourage development.

CAP evidently believes that a high price tag constitutes a failure to take "effective steps to achieve practical application" – and is therefore grounds for the feds to seize a patent and license it to others.

Who decides what price is too high? Why, an all-powerful new government panel housed in the Department of Health and Human Services, of course. It's supposed to set a range for drug prices, and any drug priced more than 20 percent above this range is subject to patent seizure.

This interpretation of Bayh-Dole is the antithesis of what the authors sought. The law empowered innovators by protecting the work of university researchers, small businesses and nonprofits that incorporated basic concepts that had previously been discovered via federally funded research.

The bulk of biopharmaceutical innovation comes from private industry, which spends more than $51 billion annually to develop new drugs. These investments will simply stop if government has the authority to seize the patents of innovators any time they don't meet a government-set price.

Similar price-capping schemes in Europe, once an industry leader, essentially capped innovation and jobs. In the mid-1980s, Europe spent 24 percent more on research and development than firms did in the United States. As price caps took effect, by 2004 European development dropped 15 percent. Between 2001 and 2009, more than 60 percent of drug patents went to U.S.-based companies. In 2012, the U.S. biotech industry employed 100,000 people – twice as many as in all of Europe. Alas, as John Adams quipped, "Facts are pesky things."

As a major hub for the biopharmaceutical industry, New Jersey will bear the brunt of the CAP plan's job-killing regulations. Over 70,000 Garden Staters work for biopharmaceutical firms. Industry spending supports another 250,000 jobs in other sectors. All told, drug firms add $87 billion to New Jersey's economic output.

Government-sanctioned patent theft will lead to massive investment cuts and job losses in New Jersey. More worryingly, CAP's caps on drug prices would prevent the development of new medicines. Candidates embracing this approach pose a threat to our health.

Peter J. Pitts, a former FDA Associate Commissioner, is the president and co-founder of the Center for Medicine in the Public Interest.

  Read More & Comment...

Pharmaceutical Pricing -- Let's Debate the Facts

11/13/2015 03:13 PM |

Medicines aren’t priced in a vacuum. Pricing decisions are based on numerous meetings and tough negotiations between manufacturers and payers over a long period of time – often years before the FDA approves a product. In fact, many product programs are stopped or altered well before Phase III trials depending on these conversations.

So, when you hear about a new medicine and payers are complaining about the price, it’s (at best) disingenuous, but they get away with it. Why? Because manufacturers are uncomfortable calling out the insurance companies and PBMs with whom they must do business. The reverse is untrue in the extreme. And “disingenuous” is a polite way to say something else.

A new study from the IMS Institute for Healthcare Informatics puts some hard numbers behind the debate. And it’s about time.

As the report’s executive summary reports:

Price levels for pharmaceuticals in the U.S. market are often reported to the public based on list prices, and therefore do not reflect the series of adjustments that occur throughout the healthcare system and ultimately determine who pays what for medicines.

The purpose of this healthcare brief is to draw specific attention to previously published research from the IMS Institute which highlights not only the visible aspects of price increases, but also the less visible off-invoice discounts, rebates, coupons, and other price concessions to payers that often substantially offset these changes in list price. By bringing context and perspective to the complex interplay of factors that determine the level of price changes for branded medicines we hope to better inform discussions of the issues.

… Our analysis shows that branded pharmaceuticals raised invoice prices on average 13.5% in 2014, but on a net basis, after all of the concessions are adjusted for, the increase was 5.5%. This level of net price increases is notable for being the lowest of the past five years and has occurred even as invoice price increases have accelerated.

It’s time for everyone to debate the facts.

The complete report can be found here. It’s an important read at an important time.

  Read More & Comment...

PCSK9 Patient Assistance Programs: Do ask. Do tell.

11/11/2015 07:40 AM |

Much ado about PCSK9 inhibitor patient assistance programs that requires users to share rights to their personal health information. Is this a legitimate quid pro quo? That’s a tough question – but not the most important one.

The foundational question is, why do companies want this information?  Because it has valuable public health applications. Cumulative, de-identified real world data will provide the company with outcomes intelligence that can be shared with payers, physicians, and the scientific community. For payers and physicians, it will help better define what subset of patients with high cholesterol should receive Repatha as first line therapy, avoiding a costly (both in terms of dollars and cardiovascular health) “fail first” step-therapy scenario. And the information will allow researchers to better focus their efforts on expanding our understanding of the PCSK9 universe and the potential development of companion diagnostics.

All good reasons to have patients share their data. But should it be an “either/or” proposition? It needn’t be. Why not make such data sharing voluntary? Let patients know that by sharing their personal information they are helping “people like them” get diagnosed more directly and achieve better health results more rapidly. People want to do the right thing – but they have to understand why and how. Under this scenario the innovative biopharmaceutical industry can be the facilitators of better, more cost-efficient outcomes and be hailed as healthcare heroes.

Which is a lot better than what they’re currently being called.

  Read More & Comment...

The Truth About Specialty Drugs

11/10/2015 11:49 AM |

A great article by Tomas Philipson that states the truth about the cost and value of new medicines.   

Here's the knock-out blow against the lies and mis-statements regarding the 'unsustainable' cost of of new drugs"

"As costs have risen, many insurers have responded by increasing cost sharing for specialized therapies as part of their pharmaceutical insurance design.  For example, CMS allows Part D plans to create a “formulary tier” specifically for drugs costing $600 or more per month.  About 90% of plans use this tier and among these plans, more than half require patients to pay 25% or more of costs.  Co-insurance for specialty drugs, which are taken by the sickest 3% of patients, can be as high as 50%. 

This design means that the sickest patients also take the largest financial hits; a form of “double jeopardy.”  However, this has primarily led to outrage against manufacturers rather than payers.  For example, oncologists have criticized manufacturers for high prices because their patients cannot afford treatment.  This is somewhat ironic since, as the General Accounting Office noted in July, cancer centers with higher markups on cancer drugs prescribe more of them, a practice partially enabled by the federal government’s 340B drug pricing program intended to provide discounted drugs to lower income patients.  In addition, other forms of health care are equally, if not more expensive – such as ICU care – yet there appears to be comparably less concern over these costs.  Perhaps the reason is that ICU care – which often costs approximately $4,000 per day – is often fully covered (as it should be), whereas specialty treatments remain only partially covered. 

The reality is that less generous coverage of specialty drugs may punish the sickest patients and is not consistent with basic tenets of insurance, which are designed to cover rare but expensive events."  Read More & Comment...

Asking the right questions about biosimilars

11/09/2015 10:06 AM |

I just returned from Kaohsiung, where I was pleased to keynote the Taiwan Pharmacy International Collaboration Center’s Generic Equivalency and Biosimilarity conference.

Of the many excellent presentations from global experts, one of the most interesting was by Churn-Shiouh Gau, the Chief Executive Director of the Taiwanese Center for Drug Evaluation at National Taiwan University. She spoke on the current state of affairs of biosimilar regulation – and her talk generated some tough and specific questions.

For example – should Taiwan approve a biosimilar is the originator product hasn’t been approved. (For example, Remicaide isn’t an approved therapy in Taiwan – and there are already a few biosimialrs on the market in many other places – such as the EU.) Her view was “no.”

Also, should Taiwan approve a biosimilar that isn’t approved for use in its country of manufacture? Following the theory that what’s good for the goose is good for the gander, why should one country take a risk on a product (especially ones so new and complicated as biosimilars) that hasn’t been deemed safe and effective in its native land? Her view was, “no.”

Representatives of the Taiwan FDA (a First World Regulatory body) weighed in with many comments and caveats. Clearly questions such as these need to be addressed as seriously as those concerning biosimilar review pathways.

Voltaire said, “Judge a man by his questions rather than by his answers.” And the same can be said of medicines regulators. But we need to ask the right ones or we fall into the trap that Thomas Pynchon warns about when he writes, ““If they can get you asking the wrong questions, they don't have to worry about answers.”

“They” can broadly be defined as those focusing on biosimilars exclusively as a cost-saving mechanism. Safety and quality must always drive the regulatory discussion.

  Read More & Comment...

Peter Bach's Deadly Cancer Math

11/06/2015 11:05 AM |

In his latest rant against drug prices Peter Bach provides two bits of deadly disinformation.

In talking about Opdivo, the new drug for people with advanced lung cancer that can add up to 2 years of life, Bach asserts:

"Federal law prevents the maker of nivolumab (Bristol-Myers Squibb) from providing assistance to patients who cannot afford the treatment. Programs such as Genentech's for Avastin, in which beneficiaries receive the drug free once they have spent a certain amount in a calendar year, are rare."

Untrue.  Companies can't provide assistance directly but it can do so through 3rd party foundations and do so regularly.   BMS provides an incredible amount of support to patients which frankly is provided because insurers don't cover the cost of the entire drug though it saves them money and improves patient lives better than stuff like surgery (which insurers cover completely).    But in Bach's warped world, this is fine.  

Then he proclaims that the only way to measure value is to see how much a new medicine or technology adds to a health plan's budget.  Let's set aside the fact that most health care spending increases are the result of an increase in the use of other services and Bach never scrutinizes that.  More important, new medicines almost always over time reduce the use of other services and contribute to at least half the increase productivity and longevity.  

Bach claims that new medicines are no more effective than old medicines because they don't add any more average survival.   Really? If that's the case than, how has cancer survivorship and life expectancy steadily increased over the past 20 years.  Perhaps what he is say is that the additional increment of average survival (which ignores genomic variation) is not worth say $100000.  But that ignores the fact that treatments are targeted to smaller populations that have fewer options than previous generations.   Unfortunately, high prices are partially a result of investing the same amount of time and money on tinier groups of cancer patients.  Bach knows better.  After all, one of his co-authors in the paper he cites in his NEJM oral hallucination about new drugs not adding more survival despite higher prices, makes that very point in another study: " In the absence of significant pricing and total oncology outlay flexibility by payers, our analysis suggests that private sector investment in small oncology segments, and in stratified medicine generally, may not prove economically sustainable, thus endangering the translation of scientific advances into bedside medicines. Beyond increasing reimbursement, decreasing development cycle time and costs, or both, would most directly improve the economic incentives facing developers. By contrast, extending exclusivity periods, or initiating advance market commitments and awarding prizes would likely have less impact and involve greater implementation challenges." (Trusheim, Berndt "Economics of Stratified Medicine" Personalized Medicine (2012) 9(4), 413–427) 

So in otherwords, Bach wants lower prices at all costs, even if it kills innovation and people.  

Bach is channelling Andrew Wakefield, another lighly published doctor who used the media to advance an agenda that proved toxic to the public health.    Read More & Comment...

Invest in Cures To Save Medicare, Cut Taxes

11/02/2015 12:46 PM |

Two good articles on why America needs to do more to accelerate the commercialization of medicines..  Both in the Daily Caller.  One by presidential candidate Mike Huckabee and the other by the man who should be president, Jim Pinkerton.  

Why Is the GOP So Blind on Medicare, Healthcare and Cures

Actually, Curing Disease Is A Pretty Good Way To Save Uncle Sam Money  Read More & Comment...

Kansas City Royals Fans Have Another Reason To Celebrate

11/02/2015 12:31 PM |

From our friend and inspiration, Bob Tufts.   BT pitched for the Royals around the last time they won the World Series.  He  was up to see the final out of last night's game and in all the excitement, I forgot to wish him Happy Birthday while we were texting each other.   CMPI is celebrating his birthday tonight, not just for his pitching prowess but for his courage and consistent advocacy of medical innovation.


Here's the great article he wrote for the Huffington Post about shutting out cancer and how he did it.


60th and 6th is not a location that you can find on a map of Manhattan, but it is a good place to be.

November 2nd will mark my 60th birthday -- and also mark the sixth anniversary of my return home after undergoing an autologous stem cell transplant to deal with cancer. It is a good time to reflect on the past six plus years and my long journey with this deadly disease.

I was diagnosed with multiple myeloma, a cancer that affects the white blood cells in your bone marrow, on St. Patrick's Day 2009. Nothing can prepare you -- or even your doctor -- to say the dreaded words "you have cancer." It was a shock, especially since I had never missed a day of work from any illness in my life, be it playing major league baseball or working on Wall Street. I considered myself very healthy and did not ever expect to hear these words.

At the time of my diagnosis, my version of myeloma was deemed high risk. If my initial treatment did not work, I might be dead within a year. Fortunately, the pill-based regimen that I received did work extremely well, and by October of 2009 I was ready to have the stem cell transplant to further battle the disease. Five weeks in an isolation room being anemic, having a limited white blood count and suffering from 103 degree fevers and a blistered alimentary system was stressful, but my response to the treatment was excellent. Shortly after coming home I was placed on a maintenance dose of the same medication and I have taken it ever since.

From November of 2009 through today, I have not shown any perceptible sign of the cancer. At this time last year, my oncologist told me that it was time to talk about the "C" word. I nervously asked "do you mean the cancer is back?" He said no, I mean "cure" - you are as close to being cured of an incurable disease as I have seen". I realize that the odds are that I will relapse at some time in the future, but for now I will enjoy the fact that I have told cancer to get lost for at least a few years.

My excellent response to the myeloma treatment made me an outlier, as the median survival rate when I was diagnosed was only one to three years, but science and innovation have changed that. The five year survival rate for myeloma patients is now almost 50 percent, and at some hospitals it is 63 percent. Continuous innovation in the blood cancer field has made remarkable strides in the past decade, and it is poised to do more. If and when I do relapse, other drugs have already been developed in the past few years that can be used to treat me.

2015-10-30-1446221896-8069460-CancerSurvivorsinUS.png

Was I now returning home an invalid, unable to contribute to society? Hardly! After a brief period of excess caution to avoid infections, I was able to cook, clean and even assist with my mother-in-law's health care at her nursing home. I was able to be there when our daughter graduated from college. I was able to attend numerous lifecycle events, both happy ones and somber ones. In the past six years I taught approximately 1500 students at three colleges, served as a school advisor, counseled many on career and life choices. I coached hundreds of young baseball players at Major League Baseball Players Alumni clinics. And, I began to attend major medical conventions to deliver the patient's perspective on access and choice in care.

This last item is the most important. If I had lived anywhere other than the United States, systems such as QALY (Quality Adjusted Life Years) are used to evaluate whether treatments should be given to patients based on their expected survival time post-care. In my case, based on some average expected survival rate, I would have been denied the life-saving treatment that I received and would probably have died sometime in 2009.

Perhaps another treatment might have worked, but would you take that chance with your life? Insurance practices such as "fail first" exist, where patient must try the older and less expensive drug and fail to respond to it before being allowed to take the novel therapy. How many patients may have ended up prematurely dead under this scenario where the right drug at the right time is kept away from a person in need? Patients should be proactive and have DNR's, living wills and powers of attorney to make sure their wishes are honored. However, patients should also have a doctor who is ready to fight for their life, with access to as many weapons they deem necessary to battle a lethal disease. That decision should not be a theoretical and impersonal one made by an unseen administrator far removed from your bedside.

These medically harmful attempts to limit access based on an administrator's determination of value need to be debated in the public square. We patients pay the co-pays, insurance premiums, taxes and other fees that fund the entire medical system, but at conference after conference, when discussions on cost and value occur, patients are not represented on the stage. Panelists from on high -- medical administrators, Masters in Public Health and insurance executives -- lecture us about how much we should pay and how our dollars will be divided in the health care system. Bureaucrats want our dollars but do not want our opinions, even though the decisions being made affect the quality of our individual care. The reactions that I receive at conventions when I bring up this point and mention their usage of Orwellian definitions like "choosing wisely," "evidence based" and "unnecessary care" is frosty at best.

I plan to redouble my efforts in 2016 and beyond through "My Life Is Worth It", an online campaign that I co-founded to fight for fellow patients because we want, need and deserve to be at the table when discussions linking cost and value of our care occur. We believe that medical innovation can and will save lives, reduce the cost of health care and stimulate economic growth.

We will continue to push back against "fail first", restrictive insurance formularies, obscene co-pay requirements, time consuming data entry requirements that do nothing other than keep doctors from looking into the eyes of a scared patient with a chronic disease. We will also question the propriety of medical administrators and medical trade groups forming agreements with insurance companies, a blatant conflict of interest against the Hippocratic Oath. We will make sure that the doctor is allowed to practice the art and science of medicine on behalf of the patient and not at the whim of the administrator.

I will question Big Data collection and whether it truly provides value to those who are ill, or does it merely create a system of medicine in which meeting the average or a satisficed level is considered proper care. To borrow a phrase from my baseball days, Big Data may get us in the ballpark, but personal care from a trusted physician gets us to our seat.

We patients are not averages; we have different genomic responses to the initial phases of the disease, its diagnosis, treatment and maintenance protocols. One size fits all care will not advance survival rates or cures. Treating cancer patients based on an average will only yield average results. What is needed is to beat, not merely meet, the norm, to raise the bar and to make the exceptional result today the norm in the future.

I want others to also become outliers, to live longer and better with their chronic illnesses - and to be able to fill their time not merely being alive, but using the time that innovative treatments provide us to be at those lifecycle events - to do the things that make living worthwhile.

I have a lot to be thankful for this November, and celebrating this day with people who reached out to me and my family during our time of crisis is where I want to be today. I lift a glass and say thanks to friends, family and medical professionals whose actions and words helped make today's birthday happen.

This day makes living worthwhile. This is an example of real value that cannot be captured by an app or in an accountant's spreadsheet by those trying to ascertain the value of medical treatments who know the price of everything and the value of nothing.

This party today is being held at the corner 60th and 6th, and I am glad you were able to meet me here. I'm still here, dammit, and I plan to be for a long time.

To all of you who helped, I say thanks again!  Read More & Comment...

FTC just plain wrong on biosimilar naming

10/31/2015 07:54 AM |

In a comment filed in response to FDA's proposal, the FTC said distinct suffixes could lead physicians to believe biosimilars differ from their reference products in clinically meaningful ways.

This is precisely what was expected after the FTC’s February 2014 hearing on the topic – the one where the FDA wasn’t invited to testify. If there had been an FDA speaker, there might have been appropriate comments about the FDA's Pharmaceutical Science and Clinical Pharmacology Advisory Committee that debated and determined that the bioequivalence specifications should be tightened for, among other categories, generic versions of epilepsy medications – and that FDA officials presenting at that adcomm signaled strong agency support for the move.

The FTC even ignored it’s own expert commentary. In it’s 1979 report on generic drug substitution, the FTC concluded, “increased communication (as well as lower prices) may explain why most pharmacists report that product selection laws have had a positive effect on their relations with patients”

Safety and trust and exactly why differential naming is needed. As Sumant Ramachandra, Senior Vice President & Chief Scientific Officer of the biosimilar manufacturer Hospira, said at the FTC hearing, “Communications fosters confidence.”

The facts speak for themselves (even if they didn’t get a chance at the FTC event). A poster presentation from the European Crohn’s and Colitis Organisation, titled, “Biosimilar but not the same,” offers some timely and important real-world data on the differences between originator biologics and their biosimilar cousins.

The study, from Mercy University Hospital, University College Cork, Centre for Gastroenterology, Mercy University Hospital, Cork, Ireland, studied the clinical impact of both the innovator product (Remicade) and it’s EMA-approved biosimilar (Inflectra). The findings are important. Specifically, the rates of surgery in Infliximab and Inflectra groups were significantly different.

80% of the Inflectra group required hospital readmission versus 5% of the infliximab (Remicade) group. (p=0.00004). 60% of patients in the Inflectra group needed steroid augmentation of standard steroid tapering protocol with 50% requiring multiple increases in steroid dose versus 8% of patients in the Infliximab (p-value = 0.0007). Over the course of 8 weeks, 93% of patients in the Inflectra group had an increase in CRP with 7% remaining unchanged whereas 100% of patients in the infliximab group had a decrease in CRP (p=<0.001).

The conclusion is not ambiguous, “Our results suggest that biosimilars may not be as efficacious as the reference medicine. The results found reflect the ECCO statement position that the use of most biosimilars in IBD will require testing in this particular patient population and cannot be extrapolated from other disease populations."

The complete poster can be found here.

These First World data points about a product from a respected manufacturer (Hospira) cannot be ignored and must be used to inform the policy debate over nomenclature, interchangeability, label extrapolations, and overall pharmacovigilance practices.

Does the FTC believe that safety and outcomes are a constraint to competition?

That’s a comment worth repeating.

  Read More & Comment...

Confirm Califf, says NEJM

10/29/2015 09:06 PM |

An editorial in the New England Journal of Medicine endorsed Robert Califf for FDA commissioner. Written by Editor-in-Chief Jeffrey Drazen, the editorial rejected criticisms of Califf's nomination due to his industry ties, saying that "it is impossible to argue that Califf has a pro-industry bias" based on the outcomes of industry-sponsored clinical trials in which he was an investigator  Read More & Comment...