Latest Drugwonks' Blog

When it comes to medicine, who should make decisions about safety and efficacy – and on what evidence should those choices be made? At present, the Food & Drug Administration has the responsibility to determine approvals and labeling language based on a scientific review of the evidence. Should this authority be ceded to the tort bar?

Consider the recent spate of litigation against the manufacturers of opioid pain medications. One example is the City of Chicago’s lawsuit against multiple manufacturers of opioid pain treatments. In the United States District Court for the Northern District of Illinois (Eastern Division), the City of Chicago’s First Amended Complaint (“FAC”) seeks to limit the ability of Chicago doctors to treat the chronic, non-cancer pain of patients in the manner doctors deem most appropriate. Although the Food and Drug Administration has approved certain opioid pain medications for the treatment of chronic non-cancer pain, the FAC seeks to deprive patients and doctors of that treatment choice by having six lay jurors determine that “the use of opioids to treat chronic pain is not ‘medically necessary’ or ‘reasonably required’ in that their risks do not exceed their benefits.”

The FDA has determined that opioids serve an important public health role: “When prescribed and used properly, opioids can effectively manage pain and alleviate suffering—clearly a public health priority. Chronic pain is a serious and growing health problem: it affects millions of Americans; contributes greatly to national rates of morbidity, mortality, and disability; and is rising in prevalence. At the same time, there is no dispute that opioids pose significant public health risks: Opioids also have grave risks, the most well-known of which include addiction, overdose, even death. The labeling for these products contains prominent warnings about these risks. Moreover, the boxed warning states that all patients should be routinely monitored for signs of misuse, abuse, and addiction.

In September 2013, the FDA ruled on a citizen’s petition filed by a group of clinicians, researchers, and health officials called Physicians for Responsible Opioid Prescribing (“PROP”). Like the Chicago FAC, the Petition directly challenged the use of opioids for “chronic non-cancer pain.” PROP contended that the “long-term safety and effectiveness of managing [chronic non-cancer pain] with opioids has not been established,” and requested that the FDA, inter alia, impose a “maximum duration of 90-days for continuous (daily) use for non-cancer pain.” The FDA carefully reviewed the Petition and more than 1900 related comments. The agency assessed the relevant literature. It held a two-day public hearing at which it received “over 600 comments” and dozens of experts and concerned citizens testified. The FDA noted that “the majority of comments” “opposed PROP’s requests” and that “many professional societies,” including the American Medical Association, “did not support the Petition and stated that the data cited by PROP did not support PROP’s requests.” After completing a 14-month review, the FDA determined that opioids should continue to be available for the treatment of chronic pain, while also directing further study and certain labeling changes for some opioid drugs. Significantly, after being presented with the same assertions as those now alleged in the Amended Complaint, the FDA made two findings directly at odds with the underlying premises that form the cornerstones of the FAC.

But the lawsuits keep coming. The FDA has the authority, the ability, the means, the mission, and the mandate to manage the health care risks and benefits inherent in the products it regulates on behalf of the American public.

A more balanced legal system will occur only when elected officials determine the time has come for real tort reform, as it affects pharmaceutical companies.  But that day is likely very far off. Healthcare leaders must devote their most aggressive efforts toward reform.

Maybe when our elected officials understand that it's the health of their constituents versus the pocketbooks of lawyers, our public servants will finally get serious on tort reform.

Bach to the Future

  • 01.20.2015

Time and again, the dangerous idiots of healthcare reform claim that there's no difference between so-called "me-too" drugs, and that they're simply a way for drug companies to turn a quick profit. The New York Times,in an editorial, claimed that "the nation is wasting billions of dollars on heavily marketed drugs that have never proved themselves in head-to-head competition against cheaper competitors." Wrong.

A recent op-ed in the New York Times, (Why Drugs Cost So Much, NYT, January 14, 2015) by Peter Bach is only the latest in a series of such bandwagon drivel – and he should know better.

The truth is that despite the assertions in and by the New York Times, different drugs are indeed different, even if you describe them as "me-too" medications. Likewise, even though every brand of store-bought peanut butter contains peanuts, oil, sugar, and salt, no one would argue that Skippy and JIF taste exactly the same.

Time and again, different medicines have proved themselves where it counts - in the bodies and biochemistries of patients. No two patients are alike, and the more options available, the more likely doctors are to find what works best for each patient. A few points to consider:

* When patients have access to more effective medications, their overall health improves, even as their overall medical expenses go down. That, in turn, reduces national health-care spending and boosts the economy. Value must be measured in patient outcomes.

* Healthcare innovation saves lives, saves money, promotes economic growth, and provides hope for hundreds of millions of people (both patients and care-givers) in the United States and around the world.

* If we do not support the development of new medicines through timely licensing and fair pricing, innovation will be stopped in its tracks – and that is not an acceptable public health outcome.

* Regulators can be partners in innovation three ways: Through robust oversight, through active collaboration, and, most importantly, by being an innovation enabler.

 The deviation between value and pricing in a distinction with a difference – and while both are important, it is value that’s the higher priority since driving patient outcomes is the higher calling (and better long-term economic investment).

After all, as Yale economist William Nordhaus has written, "The social productivity of health care spending might be many times that of other spending.”

Luckily, America's doctors, drug researchers and patients are smarter than that. Just like there aren't any "me-too" patients, there aren't any "me-too" drugs. And if cars and peanut butter are going to be personalized, then medicine is too important not to be.

Peter Bach yet again tries to make the case that new drugs are too expensive.

He writes: "The price of drugs is so unfair and mystifying that publlic often pays a high price when the identical drug is available at a fraction of the cost."

Actually, he didn't write that.  It's from another New York Times article: " Drug Prices Here Held Inequitable. "   It was written 40 years ago.  Burks, E. (1965) Drug Prices Here Held Inequitable. The New York Times Retrieved from

What he did write is another variation on this very old theme: "We can free insurers and government programs from the requirement to include all expensive drugs in their plans as we explain to the public that some drugs are not effective enough to justify their price. If we do this, we can be confident that manufacturers will lower their prices to ensure their ability to sell their products. Or we can piggyback on the gumption of bolder countries, and demand that policy makers set drug prices in the United States equal to those of Western Europe. Either approach would be vastly superior to the situation we have today."

First the facts.  

1. Nearly 90 percent of all brand drugs are discounted.  Companies pay rebates to Medicare, Medicaid, and the Veterans health system.    In the private sector (including Obamacare plans) drug firms pay rebates to pharmacy benefit management companies to get preferred listing.  

2. Bach is incorrect in claiming insurers have to cover every ‘expensive’ drug.  Express Scripts has 66 products on its 2015 formulary exclusion list, compared with 48 in 2014. CVS Caremark’s 2015 list has 95 products, including 72 carryovers from the 2014 edition.  

He loves this idea.  But what about patients?

From Managed Care Magazine:  

"The problem with this approach is that by adding more tiers health plans have confused employees and made some medications more expensive — the antithesis of a value-based strategy. They also have retained cost-sharing mechanisms that make high-priced drugs unaffordable for many patients, Vogenberg says. A 25% copayment on a drug priced at $1,000 a day costs a patient $1,750 each week or more than $7,000 per month. Many patients simply cannot afford their medications, even though commercial plan members often use copayment assistance programs from pharmaceutical manufacturers and can avoid such high cost-sharing amounts, says Brenda Motheral, RPh, MBA, PhD, president of Artemetrx, a specialty drug management consultant.

Bach is silent as PBMs pocket rebates while at the same time sticking patients with the higher costs of meds.

3.  Compared to Europe and Canada, on average Americans then pay slightly more for new medicines  in exchange for faster and broader access.   On average we get twice the new medicines about a year and a half earlier with fewer restrictions.  When all is said and done, using Euro-prices would save a grant total of 8.6 billion.. less than one third of one percent of total health care spending."

4.  Contrary to Bach, Americans also pay less out of pocket for medicines than in Europe. On average, out of pocket costs are about $800.  

4.   European speand more on cancer care as a percentage of health care and less on cancer drugs.  

Nowhere in Bach's article does he mention the value of broader, faster access to new medicines.   Let's stick with the European comparision.

Cancer mortality rates in the US are lower than most anywhere in Europe for every major cancer.  And five year survival rates (adjusted for lag time and incidence) are much higher and increase faster.  That doesn't include the life years lost when people with advanced forms of cancer die waiting a year and half for a new medicine or have strict limits place on access.    

American get broader access to better drugs that save money at about the same price as medicines elsewhere.  Price controls and PBM restrictions on access to new drugs actually increases cost and makes people sicker.  And probably let a lot of people die waiting for new medcines.  If a new medicine adds 3 months on average to the life of a cancer patient it would mean that each year (based on 590,000 cancer deaths a year) someone is denied access robs people with cancer of about 145000 life years annually. 

Back claims other systems are vastly superior.  The evidence suggests otherwise.  Cutting drug prices does kill people. 

The gain in pain is plainly in the main.

Abuse of opiate-based prescription painkillers such as oxycodone and morphine peaked around 2010-2011 and now may be on the decline in the United States, according to an analysis of databases designed to track illicit use of the drugs.

New laws, programs and policies, such as prescription tracking systems and the reformulation of oxycodone to make it harder to abuse, may be combining to reverse the once-growing trend, researchers said.

"I think we're at an inflection point and we're starting to turn this steamship around," said Dr. G. Caleb Alexander, co-director of the Johns Hopkins’ Center for Drug Safety and Effectiveness, who was not involved in the research.

The new study, published in the New England Journal of Medicine, looked at data collected between 2002 and 2013 from substance-abuse treatment centers, poison centers, college students and drug-diversion investigators.

There were "large increases in the rates of opioid diversion and abuse from 2002 to 2010, but then the rates flattened or decreased from 2011 through 2013. The rate of opioid-related deaths rose and fell in a similar pattern," write the study authors, led by Dr. Richard Dart of the Rocky Mountain Poison and Drug Center at the Denver Health and Hospital Authority in Colorado.

The full NEJM article can be found here.

Now we need to continue to move (and act) beyond the rhetoric and pursue additional solutions. And at the top of the list is the foundation of the Hamburg Manifesto -- prescriber education.

E&C Show and Tell

  • 01.14.2015

Per BioCentury, Reps. Fred Upton (R-Mich.) and Diana DeGette (D-Colo.) previewed on Tuesday the goals of legislation they plan to release this month as part of their 21st Century Cures initiative.

In commentary published by CNN, the legislators wrote that their bill will seek to “modernize clinical trials to streamline the approval of drugs and devices,” in part by reducing paperwork and promoting adaptive trials. It will also help FDA “better integrate the patient perspective into the regulatory process,” including using public-private partnerships to strengthen science around biomarkers and patient-reported outcomes.

The bill's sponsors also aim to promote “better access to and sharing of information such as genomic and other clinical data to foster more collaboration among researchers," and to invest in programs for young scientists.
Upton -- the chair of the U.S. House's Energy & Commerce Committee -- and DeGette also plan to “incentivize new drugs and devices for unmet medical needs” by “streamlining the premarket process while establishing mechanisms to better capture real world evidence post-market." They also said they will examine incentives, including “exclusivity or simplifying the reimbursement process,” to stimulate the development of new drugs and devices for unmet medical needs.

More as more develops.

Brill not so bril

  • 01.13.2015

The review of Steven Brill’s “What Ails Us” (NYT Book Review, January 11, 2015), refers to the pharmaceutical industry’s back-room negotiations to "gut" comparative effectiveness under Obamacare. The truth is that the Recovery Act of 2009 provided $1.1 billion for patient-centered health research (also known as comparative effectiveness research) – and that’s only part of the story.

What Mr. Brill is actually referring to is the administration’s “deal” with the pharmaceutical industry to leave in place what is known as “the Non-Interference Clause,” which prohibits the Federal government from negotiating drug prices for (among other things) the highly successful Medicare Part D drug program. It’s important to note that the Non-Interference Clause was the brainchild (during the Clinton years) of Senators Tom Daschle and Ted Kennedy – hardly candidates for the Tea Party Hall of Fame.

According to the Congressional Budget Office (in 2004), revoking the Kennedy/Daschle Non-Interference Clause, “would have a negligible effect on federal spending because CBO estimates that substantial savings will be obtained by the private plans and that the Secretary would not be able to negotiate prices that further reduce federal spending to a significant degree. Because they will be at substantial financial risk, private plans will have strong incentives to negotiate price discounts, both to control their own costs in providing the drug benefit and to attract enrollees with low premiums and cost-sharing requirements.”

In 2009 the CBO reiterated its previous views, stating that they, “still believe that granting the Secretary of HHS additional authority to negotiate for lower drug prices would have little, if any, effect on prices for the same reason that my predecessors have explained, which is that…private drug plans are already negotiating drug prices.” Unlike other healthcare benefits, Part D is also cost-effective for taxpayers. In 2014 the CBO reported Part D drug spending was 45 percent below original cost projections. 

Importantly, the CBO says that no further savings are possible unless the government restricts beneficiary access to medicines or establishes market-distorting price interventions. In other words, price controls lead to choice controls.

Fil in the blanks

  • 01.12.2015

In October 2013, California Governor Jerry Brown vetoed a bill, overwhelmingly passed by state lawmakers that would limit automatic biosimilar substitution to those the FDA deems “interchangeable.” SB 598 also would have required pharmacists to notify physicians when an interchangeable is substituted for a prescribed biologic.

One of the Governor’s stated reasons for his veto was, “The FDA, which has jurisdiction for approving all drugs, has not yet determined what standards will be required for biosimilars to meet the higher threshold of ‘interchangeability.’ Given this fact, to require physician notification at this point strikes me as premature.”

Much has changed since that day – especially per physician notification. Today even the GPhA supports this public health imperative. Perhaps the biggest change in the landscape was yesterday’s ODAC meeting on a biosimilar filgrastim – and not just the overwhelming 14-0 vote in favor, but also the FDA’s strong support of the Sandoz application.

All of a sudden the issues addressed in SB 598 don’t seem so “premature.”

But the filgrastim adcomm isn’t the end of the debate. There are still many issues yet to be determined via FDA guidance  (such as nomenclature) and corporate strategy (read, “pricing”). As BIO’s Jim Greenwood said, “This week’s advisory committee meetings facilitated important discussion of the scientific approach of reviewing biosimilar applications and we encourage this positive momentum, however, we believe the appropriate way to develop policy on such a significant new approval pathway is through published guidance documents with the opportunity for public comment, rather than through single-application advisory committee meetings.”

One issue that’s taking a back seat to the approval of biosimilars is the requirement for new thinking on their post-approval safety and surveillance. Biologics aren’t the new kid on the block anymore. While it’s important to pursue ways to expedite 21st century cures (as well as the eponymous legislation), it’s equally important to focus on the details of 21st century follow-on products (both biosimilars and non-biologic complex drugs). Just as the FDA has been diligently pursuing patient-centered drug development, so too must it develop new strategies and tactics (“guidelines”) for patient-centered pharmacovigilance of biosimilars and NBCDs (such as Copaxone).

It’s also time for NORD and all the other patient and disease organizations who were so wonderfully outspoken on the urgency of expediting the FDA review process for new therapies and cures to hoist the banner of follow-on safety.

On pages 21-22 of the FDA briefing documents, an agency analysis found that, statistically, the commercial variety of EP2006 (Sandoz’ biosimilar) was lower in protein content than the comparator product (Neupogen) and was nonequivalent.  FDA dismissed this as something that could be worked out with better manufacturing controls and asked Sandoz to correct it. Okay, but isn’t this exactly the kind of thing that can cause “poor responders” to filgrastim to have suboptimal responses?  An important fact to consider when debating the value of differential nomenclature for biosimilars.

And then there’s the issue of cost. Many members of Congress have been leaning heavily on the FDA to expedite biosimilar guidances so that payers can realize cost savings.

But what will those savings be?

When ODAC member James Liebmann (assistant professor at the University of Massachusetts Department of Medicine) asked what the price of the Sandoz biosimilar would be, the answer wasn’t a resounding success for those counting their savings before they’re hatched. According to Mark McCamish (Sandoz global head of biopharmaceuticals and oncology injectables development), “We can’t say that the price would be less because in some situations the price will be at parity because of other relative terms that will come into existence that’s there. Price is a relatively complex situation.”

Indeed – as is regulatory science, which is why attention must be paid to creating ever-greater clarity for both biosimilar and NBCD pathways – and in post-approval surveillance.

It’s not just about price – it’s about safety. And it’s not about getting it done fast – it’s about getting it done right. Members of Congress should be focused on greater clarity through guidances because of patient safety issues rather than vague promises of cost-savings. As Brian Harvey (Pfizer’s VP for Regulatory Strategy -- and my former agency colleague) commented, “In lieu of a finalized guidance,” the agency has been making “very good efforts” to provide case-by-case feedback about biosimilars as companies move forward.”

As Brian Harvey (Pfizer’s VP for Regulatory Strategy -- and my former agency colleague) commented, Pfizer has been “very pleased” with the frequency of its interactions with FDA regarding biosimilars and “the granularity of the feedback” the company is receiving.”

For those in the know, that’s great, but it leaves the rest of us guessing. Official guidance would be even better.

This past summer Qasim Rashid  wrote an article entitled: "When Will the Muslim Honor It's First Nobel Laureate?"  It's a moving piece  about the failure of Islam to honor science and indeed how scientists who's research deviates from or challenges Islamic dogma are treated as infidels.  

Rashid writes: "Muslims today boast, rightfully, about Islam's Golden Age and its unprecedented contributions to the sciences. Muslim leaders worldwide implore Muslims to rise up to that greatness once more. But in doing so, too many ignore the 20th century's most prominent Muslim scientist--one who once again rekindled the brilliance of the countless Muslim scientists who created the Golden Age of Islam."

The source of extremistism is the insistence upon doctrinal certainty and the enforcement of that world view through defilment, descration and death.   The refusal to engage in fact-based discussions about the origins of the Universe and the evolution of man means that much of physics and biology is off limits.   

In his book "The Ascent of Man"  Jacob Bronowski wrote: “There is no absolute knowledge. And those who claim it, whether they are scientists or dogmatists, open the door to tragedy.”

The massacre of this week, the killing of 122 children in Pakistan, the rise in attacks on Jews in Europe are the result of this monstraous centainty.  Islamic leaders will be hard put to undermine this aspect of it's epistemoilogy but they must do so to save their religion.  

We have not faced a similar assault on freedom since Nazi Germany.  And no surpise, the Third Reich rose to power by subsuming science to it's Aryan vision. 

Back then the West rose up to defend freedom in the "hour of maximum danger."  It must do so now. without apology or resort to the usual relativistic excuses that hamstring the actions and intellectual honesty necessary to that defense.  And it can begin by pointing that the gap betweeen Islam and the rest of the world that opens wider with every savage act of violence can be bridged only when the scientific impulse -- the challenge of past dogmas and theories and the dedication to critical thinking -- is wovem into the fabric of education and institutions.  Honoring the scientists of today is a good way to demonstrate that necessary shift in outlook. 

You can read his article by going to this link: When Will The Muslim World Honor Its First Muslim Scientist Nobel Laureate?

Today, as the FDA’s Oncologic Drug Advisory Committee (ODAC) adcomm debates and discusses whether to recommend approval of a Sandoz filgrastim biosimilar, (supported by the agency in their meeting materials), a few interesting items of note.

On pages 21-22 of the FDA briefing documents, an agency analysis found that, statistically, the commercial variety of EP2006 (Sandoz’ biosimilar) was lower in protein content than the comparator product (Neupogen) and was nonequivalent.  FDA dismissed this as something that could be worked out with better manufacturing controls and asked Sandoz to correct it. Okay, but isn’t this  exactly the kind of thing that can cause “poor responders” to filgrastim to have suboptimal responses?  An important fact to consider when debating the value of differential nomenclature for biosimilars.

Here’s what the FDA had to say …

 More concerning was that Sandoz was apparently confused in providing multiple lots of the biosimilar to the FDA for the protein testing.  As a result, they thought they had provided 6 lots for testing that turned out to be only 4.  Page 4 of the Addendum states:

“On November 25, 2014, Sandoz responded to the Agency request. In the response, Sandoz clarified that the actual number of EP2006 commercial drug product lots used in the statistical analysis referred to above is four lots instead of six lots. The six EP2006 commercial drug product lots initially considered were determined to be not independent because four of those six EP2006 commercial drug product lots were split-fill lots from two EP2006 bulk drug product batches, resulting in only four independent EP2006 commercial drug product lots.”

If Sandoz, a world-class company with a stellar record for cGMPs, in their highly reviewed and internally scrutinized licensing application confuses batches of their biosimilar, what can we expect in the real world after marketing?  If Sandoz can’t track different lots of their experimental drug, what will the reality be in the real world? Another cry for sanity in the debate over nomenclature.

Biosimilars are here to stay -- and we need a nomenclature safety net.


A recent article in Biocentury by Roger Longman and Jane Borne of Real Endpoints has some undiluted advice about the challenge drug companies will face getting customers for the record number of new medicines approved in 2014

"HCV has taught payers they can in fact limit access to valuable drugs even when there’s no competition at all. Five years ago, it would 
have been unthinkable to deny a relatively healthy but HCV-infected patient a curative drug. Today it’s routine."

Ditto for HIV, MS, psoriasis, various cancers.  

"They are willing to challenge head-on, as far as we can see for the first time in a major, potentially fatal disease, the resistance of physicians and patients in order to provide their customers with a mechanism for bending the cost curve in specialty drugs. And that means that we will see similar deals in all significant competitive specialty categories — including in some areas of cancer."

RIght now companies are responding by offering the big pharmacy benefit networks discounts in exchange for being the only drug of it's kind covered.  And since the first movers are likely to cut such deals to limit competition,  what will happen to other medicines that may have significant benefits for patients or to other medicines that need to be used in combination.  When price is the only thing that matters, outcomes take a backseat.  And pharmacy plans are doing because, under Obamacare, they can.  

Isn't that a restrictive formulary?  Won't that kill the other speciality pharmacies and small drug stores that sell on service and not just margin?  Isn't that bad for patients?

There's only one right answer: Yes.

There are three things companies must do to get their products to patients.  I am not sanguine about the ability or desire of  most companies to take these steps.

1.  Identify groups of patients that benefit most from a new medicine, especially groups that will benefit and require a combination of treatments to improve health.

2.  Demonstrate the benefit of such treatments to patients and their families in terms of a return to or continuation of life free for disease.   Estimate the value of this state of wellness and estimate the cost -- in terms of out of pocket spending, lost productivity and even death -- of forcing people to use a drug that's cheaper for a health plan but not best for a patient.  

3.  Demonstrate the benefit of such treatments to employers, universities, retirement funds, life and disability insurers, Medicare, Social Security.   And share this information widely and with Congress.  Otherwise valuable Obamacare reforms to eliminate discrimination against chronically ill people through restrictive formularies will fail.  

As Longman and Borne note:  It’s crucial that biopharma provide (these other stakeholders) the appropriate measurement tools. If they don’t, buyers will settle on the easiest point of comparison: price.


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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