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.

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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...

BioPharm companies are Inviting Price Controls By Inaction

10/29/2015 05:07 PM |

The Kaiser Family Foundation ran another health care survey that asked if people wanted price controls on drugs.  As I wrote after the last KFF price control poll the results are not suprising and consistent with surveys on the subject taken over 50 years.  

Let's set aside the fact that people have also supported price controls on hospitals, gas, oil, cable TV rates, etc.  The polls this time around are being used as part of a broader campaign to impose price controls on a state level and to get the next president to "do something" through executive order.  

Where is a poll that asks people what they think PBM and insurer cost sharing strategies.  In particular, where is the poll taken  after massive negative coverage (similar to that dumped on drug companies) showing how "PBMs to create a preference for drugs and generics that yield the greatest rebates and
profits. What is more, this arrangement actually incentivizes PBMs to promote the drugs for which they receive the largest per-prescription rebate,
rather than the cheapest or best-value prescription."  Or that insurers will pocket rebates and then force consumers to pay up to 40 percent of the cost of the rebated drug.  Or that insurers will create step therapy programs that reinforce their profit margin.

Don't you think Phrma should conduct it's own poll about price controls?  Guess what?  It did.  But the media ignored it as biased.  And a one and done poll will never get traction if it isn't part of a broader conversation. 

The industry will never get the media to cover this.  So it's up to them to invest time and money in a real campaign.  It had no problem forking over $150 million to the campaign to pass Obamacare.  You'd think they'd find the ability and resources to do the same to put drug prices in proper perspective.

If the industry thinks playing nice with it's opponents will work, it will be inviting price controls.   I don't  know how many times I have heard from pharma that they can't attack PBMs and insurers because they are "our" customers.   Meanwhile their customers are deeply involved in pushing price controls and running tough negative media campaigns against them.

There's a point at which concililation and civility is taken too far.  Past that point, it becomes defeat by default.

The collection of companies that comprise the biotech and pharma industry has developed and commercialized more important products than any known to humankind.  It -- and the hundreds of thousands of scientists working for them -- deserve better than to be treated like predators.  



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Are We Still Thrilled? Express Scripts Seeking to Limit Access to Cancer Rx the Same Way It Did With Hep C

10/23/2015 04:56 PM |

Ben Levisohn from Barron's discusses an analyst's report regarding the FDA's warning about Viekira Pak, one of the newer Hep C drug's.  

"Yesterday the FDA warned that Hep C treatments with Viekira Pak can – in some cases – cause serious liver injury mostly in patients with underlying, advanced liver disease. We believe this disclosure will impact some physician prescribing and drive incremental share shift to Gilead’s Hep C drugs. At the beginning of the year, Express Scripts positioned Viekira Pak as the exclusive option on its National Preferred Formulary (NFP) for patients with genotype 1, and we view this announcement as an incremental negative for Express Scripts. While Express Scripts also has access to Gilead’s (GILD) drugs (e.g., Sovaldi and Harvoni), we estimate that Express Scripts generates higher rebate dollars and profitability from Viekira Pak."

Note:  the reason for forcing patients to fail first on Viekira Pak before being 'allowed' to pay for another drug is to maximize profits.  

Two questions:

First, how many other step therapy or fail first protocols -- structured to maximize rebates and profits --  are exposing patients to drugs that could injure or kill them?  CMPI will be looking into this issue.  In depth. 

Second, I wonder what the ASCOs and oncologists posing as economists will do since they have essentially rallied around 'value' frameworks that extend the Express Scripts Hep C approach to cancer patients.  A few months ago,  these 'experts' were more than happy not only to put the seal of approval on fail first but also help design them.  

As in Peter Bach tweeting Thrilled @ExpressScripts to operationalize my Indication specific pricing model for cancer drugs    As in "clinical trial data and input from experts like Dr. Peter Bach, director of the Center for Health Policy and Outcomes at Memorial Sloan Kettering Cancer Center, will shape Express Scripts' further strategy."

If you are going to be thrilled about the operationalization, you should be willing to accept responsbility for the harm done when adopted.  

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Don Wright: Outrunning Cancer

10/23/2015 04:29 PM |

The Marine Corps Marathon is being run this weekend in Washington, DC.   The whole idea of a marathon never made sense to me.. why run that far when we have cars to get you there faster?   

But that was before I met Don Wright.  Don is 74 and running in his 90th -- as in just 10 less than a 100 -- marathon.  

Oh, and Don has had multiple myeloma since 2004.  

I met Don about 40 marathons ago in 2012.   He had just finished running his 50th marathon in Hawaii, which was the 50th state in which Don has completed such a race. 

He was diagnosed with the disease 12 years ago when his doctor told him that he had about 4 years to live, max.  Back then, all he wanted to do was run one marathon. He was fortunate enough to be put on an experimental medicine (now approved) that knocked the disease into remission.   As he has said more than once (but never enough)" It's just a little.. pill that I take every night."  Earlier this year Don was worried that he wouldn't be well enough to run the Marine Corps event.  It wasn't cancer.  He had a pulled hamstring from overtraining!

I have had the deep privilege of spending time with Don.  He is a powerful voice for medical innovation, a loving and dedicated husband and father and a source of comfort and support for everyone reeling from the diagnosis of a deadly disease.  

The greater marathon runner Bill Rogers once said, "the marathon can humble you."   Don Wright has shown that it can inspire us as well. 



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Opioid Conflicts at the CDC Drawing Heat on the Hill

10/21/2015 09:10 PM |

The American Academy of Pain Management sent this letter to the chair and key members of the House Energy and Commerce Committee, regarding the flawed process used by CDC in developing its opioid prescribing guidelines. The letter was also sent this letter to the chair and ranking member of the Senate HELP Committee.

The letter details the most important methodological shortcomings in CDC’s process, and asks that the committees investigate to determine why a more robust and appropriate procedure was not followed. Finally, the Academy asks the committees to suggest to CDC that they scrap this guideline and start over, using a more inclusive process.

This issue is not going away.

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It's the Cost of R and D Stupid

10/21/2015 11:46 AM |

An excellent piece by Razelle Kurzrock and Hagop Kantarjian among others, that puts the price of drugs in proper perspective.  The faster we bring medicines to market using new tools that promote precision medicine, the less expensive it will be to develop these medicines.  And the less expensive it is, because of greater certainty of benefit, the more investment (and competition) there will be. 

See what happens when regulation doesn't get in the way of producing valuable technologies?

Let's hope that this important article can be the foundation for a broader effort to promote faster, less expensive access to new medicines. 

The Urgent Need for Clinical Research Reform to Permit Faster, Less Expensive Access to New Therapies for Lethal Diseases
October 19, 2015

Written by David J Stewart, Gerald Batist, Hagop M. Kantarjian, Joan Schiller, John-Peter Bradford, Razelle Kurzrock

Technical Abstract

High costs of complying with drug development regulations slow progress and contribute to high drug prices and, hence, mounting health care costs.  If it is exorbitantly expensive to bring new therapies to approval, fewer agents can be developed with available resources, impeding the emergence of urgently needed treatments and escalating prices by limiting competition.  Excessive regulation produces numerous speed bumps on the road to drug authorization.  Although an explosion of knowledge could fuel rapid advances, progress has been slowed worldwide by inefficient regulatory and clinical research systems that limit access to therapies that prolong life and relieve suffering.  We must replace current compliance-centered regulation (appropriate for nonlethal diseases like acne) with “progress-centered regulation” in lethal diseases, where the overarching objective must be rapid, inexpensive development of effective new therapies.  We need to (i) reduce expensive, time-consuming preclinical toxicology and pharmacology assessments, which add little value; (ii) revamp the clinical trial approval process to make it fast and efficient; (iii) permit immediate multiple-site trial activation when an eligible patient is identified (“just-in-time” activation); (iv) reduce the requirement for excessive, low-value documentation; (v) replace this excessive documentation with sensible postmarketing surveillance; (vi) develop pragmatic investigator accreditation; (vii) where it is to the benefit of the patient, permit investigators latitude in deviating from protocols, without requiring approved amendments; (viii) confirm the value of predictive biomarkers before requiring the high costs of IDE/CLIA compliance; and (ix) approve agents based on high phase I–II response rates in defined subpopulations, rather than mandating expensive, time-consuming phase III trials.  Clin Cancer Res; 21(20); 4561–8.
2015 AACR.  Read More & Comment...

Towards an Intramural Approach to Biomarkers

10/21/2015 09:19 AM |

Towards a More Intramural Approach to Biomarker Development

A recent article by Shashi Amur and FDA colleagues on the future of biomarker development (Biomarker Qualification: Toward a Multiple Stakeholder Framework for Biomarker Development, Regulatory Acceptance, and Utilization) provides a solid foundation for ongoing development and review process for biomarker qualification. FDA should be applauded for their progress in agency collaboration with the Critical Path Institute (in biomarker consortia development), the recent total kidney volume and plasma fibrinogen prognostic marker approvals, and sponsorship of interactive sessions such as the recent CERSI meeting at University of Maryland, as well as their EMA partnership to facilitate collaborative review of drug development tool qualification.

We would encourage additional measures to hasten biomarker development, including:

• Maximizing Expert Resources: FDA needs adequate resources to provide advice and oversee review and decision-making. One solution is to partner with an external entity (an Intramural Biomarker Consortium-IBC) to develop early advice and serve as an expert sounding board for nascent biomarker efforts.  The IBC could be a required or voluntary resource in the review process, especially for initial data package reviews. This approach would allow FDA staff to focus on their primary role of product review and regulatory oversight.

• Refined Evidentiary Considerations: The product development and research community should collaborate to support FDA in developing a framework for the proper level of evidentiary substantiation required for qualification and the criteria used to evaluate them – such that FDA can issue guidance -standards which do not exist today.  The IBC could be charged with overseeing relevant workshops and the drafting of initial guidance documents (consistent with FDA’s Good Guidance Practice recommendations and provided FDA is actively participating and has final approval).

• Qualification Plan: FDA should clarify the components of individual qualification plans and judge submitted data packages against them.  Decisions not to qualify a proposed biomarker for a particular context should be accompanied by an explanation of the evidentiary gaps between the agreed plan and the submitted qualification package. IBC could work with biomarker developers to build these plans and perform initial data package reviews.

• Enhance Learning:  Give FDA the authority to share information about biomarker qualification programs that are being advanced through collaborative efforts. Much can be learned by reviewing successes and failures across ongoing biomarker programs, and would inform the broader research community to enable refined evidentiary standards.

• Timeliness: FDA must clarify and communicate timelines for the qualification process in order to foster predictability and encourage participation.  Such resources could be provided via PDUFA VI.

FDA can further solidify its place squarely in the center of the innovation ecosystem by fostering collaborative alliances with all stakeholders, enhancing qualification planning, sharing developmental endeavors, and clarifying standards.

Peter J. Pitts
President, Center for Medicines in the Public Interest
Former FDA Associate Commissioner

Timothy R. Franson, M.D.
Chief Medical Officer – YourEncore
Immediate Past President- US Pharmacopeial Convention

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Insurers Rack Up Profits Raising Consumer Drug Costs

10/20/2015 11:18 AM |

Health plans and pharmacy benefit management firms are hiking co-insurance rates for new medicines.  As Adam Fein points out in his Drug Channels blog,  they are daring biopharma companies to NOT provide copay assistance which can amount up to 25 percent of the total cost of a drug.  (Federal programs like Medicare Part D don't allow patients to get such support directly.)  

Moreover, rebates cost biopharma about $40 billion each year.  Nearly 90 percent of that $40 billion ($36 billion) is passed on to health plans by PBMs.   In 2014,  health insurers generated  $663 billion in revenue, which means drug rebates are about 5 percent of total revenues.  

I now see another layer to the strategy of make drug prices, which are effectively set by PBMs and insurers with higher coinsurance, the issue and blaming drug companies.  It's all about making money coming and going.   PBMs and insurers can extract deeper discounts from companies whose products they carry AND get the innovator firms to pay for the chunk that consumers have to cover:

Adam discussed an IMS study " Emergence and Impact of Pharmacy Deductibles: Implications for Patients in Commercial Health Plans"  As he notes: "The report’s overarching theme is unsurprising: Higher out-of-pocket costs reduce patients’ adherence to drug therapy and increase prescription abandonment rates.

The report’s major contribution, however, links the growth in pharmacy deductibles to manufacturers’ copayment offset programs, which cover a beneficiary’s out-of-pocket costs for a brand-name drug. High deductible plans are shifting costs from payers to consumers and—in many cases—back to manufacturers.

The findings echo what payers have been doing by adding coinsurance rates to higher-tier products, per Employers Get Tougher About Pharmacy Benefits and Specialty Drug Management. Most people can’t afford to pay hundreds or thousands of dollars every month. Payers are therefore essentially daring pharmaceutical manufacturers not to pick up the patient’s coinsurance with a copayment offset program. This is the same dynamic that links the growth in four-tier benefit plans with copay offset program. See How the Fourth Tier Coinsurance Boom Drives Copay Offset Programs.

I’m not sure how many manufacturers have analyzed the codependent relationship between benefit design and their consumer-directed programs. This report suggests that such analysis would be truly therapeutic."

To which I would add.. maybe the innovators should show consumers how their drugs are priced by insurers to force them to cover the difference and suggest how patient hostile such an approach is.   This co-dependency undermines the doctor patient relationship and moves medicine away from the kind of personalized treatment selection medical innovation is making possible.    Read More & Comment...