Latest Drugwonks' Blog

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.

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."

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.

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.  
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

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.


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!

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.

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
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.  

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.  


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

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