Fragile Fraternity

08/14/2014 11:55 AM |

So much for regulatory fraternity …

PhRMA, BIO Open Fire On FDA's Biosimilars Guidance

By Jeff Overley

Law360, New York (August 13, 2014, 7:11 PM ET) -- Pharmaceutical Research and Manufacturers of America and the Biotechnology Industry Association are leading an attack by innovator drugmakers on the U.S. Food and Drug Administration’s most recent biosimilars guidance, saying in letters released Wednesday that it could let copycat products be sold without fully demonstrating safety and effectiveness.

In correspondence provided to Law360 following a Tuesday comment deadline, the trade groups each questioned biosimilars policies the FDA proposed in May regarding so-called clinical pharmacology data — relatively early research in small groups of human subjects.

PhRMA, for example, took issue with the FDA’s introduction of four categories of similarity — not similar, similar, highly similar, and highly similar with fingerprint-like similarity — saying that the “hierarchy is beyond the scope of the draft guidance.”

If the FDA insists on preserving the categories within the guidance, it should eliminate “vague and confusing” definitions that make it unclear how regulators will decide which classification to apply, PhRMA wrote.

BIO also voiced concerns with the four categories, specifically questioning the FDA’s statement that a product merely deemed similar — and therefore not comparable enough to qualify as a biosimilar — could be deemed highly similar if additional studies help to erase uncertainties.

That additional testing could “expose human subjects to an experimental therapy that had not met the statutory analytical threshold of ‘highly similar,’” BIO wrote.

Both organizations have members that are pursuing biosimilars, but they mainly focus on brand-name products, including branded biologics that stand to lose significant market share when biosimilars arrive on the scene. Any delays in finalizing FDA policies on biosimilars therefore may work to their advantage.

PhRMA’s letter also suggested that the guidance is murky with respect to when clinical pharmacology data is sufficient to establish biosimilarity. For example, at one point in the guidance, the FDA said that such data “may be sufficient to completely assess clinically meaningful differences between products,” PhRMA noted.

But the guidance also said that clinical pharmacology studies, “if done well,” can complement other data and guide future testing.

“Based on these passages, it is unclear to PhRMA when FDA believes that clinical pharmacology studies can constitute the full clinical assessment,” the organization wrote.

PhRMA and BIO also sounded alarms over the FDA’s approach to safety, specifically the issue of immunogenicity, which can refer to immune system responses that cause allergic reactions or alter a biologic’s effects. The FDA’s guidance said that such data may need to be supplemented by findings from post-approval studies, and both groups expressed concern. 

BIO, for example, wrote that “full evaluation of safety and immunogenicity should still be necessary before approval” and that the FDA should elaborate on the role of post-approval studies in demonstrating safety.

Also, BIO asked the FDA to better explain its suggestion that biosimilarity may be established by looking at a single, scientifically acceptable biomarker — say, blood pressure or cholesterol levels — or at a composite of multiple biomarkers that are "relevant."

“While this approach may have utility, it runs the risk of merely increasing the quantity of data without necessarily improving the quality and interpretability of the results,” BIO wrote.

Swiss drugmaker Roche AG — parent of U.S.-based Genentech Inc. and a member of BIO but not PhRMA — also submitted a letter seeking more clarity on the four categories of similarity and complaining that the guidance covers more than just clinical pharmacology data.

According to a public docket, two dozen letters have been submitted on the guidance, but as of Wednesday, the FDA had published only four of them. Those letters were from Roche, the United States Pharmacopeial Convention, an independent board established by Amgen Inc., and generics maker Apotex Inc., which posed five questions but little in the way of criticism.

Sandoz Inc., the first company to request FDA approval along the Affordable Care Act's biosimilar pathway, didn’t immediately respond to a request for a copy of any comments it submitted. Hospira Inc., which is also pursuing biosimilars, directed an inquiry to the Generic Pharmaceutical Association, which did not immediately have comments to share.


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But other than that, Mrs. Lincoln, how did you like the play?

08/13/2014 11:06 AM |

As seen in The Hill.

Wanted: Better fact checkers at Consumer Reports

The national public debate over the misuse and abuse of opioid pain medicines has been in the news long enough now for state and federal lawmakers and regulators at the Food and Drug Administration to focus on the real issues rather than the tabloid headlines.

That’s why the latest story on the subject from Consumers Report is so puzzling. Not only is it a day late and a dollar short, but it reaches all the wrong conclusions – providing a distinct disservice to its readers.

The CR cover featuring the story screams, “Deadly Pain Pills!” But the only thing deadly is the reporting which is both hyperbolic and filled with obvious errors and selective omissions.

Specifically, CR has the pain medicine Zohydro ER in its crosshairs. The article calls on the FDA to reconsider its 2013 approval of Zohydro ER and to make acetaminophen standards consistent.

Strangely the piece fails to mention that one of the benefits of Zohydro ER is that it is acetaminophen-free. It also neglects to mention that this very concern was specifically addressed by FDA Commissioner Margaret Hamburg who testified before Congress that,  “We recognize that this is a powerful drug, but we also believe that if appropriately used, it serves an important and unique niche with respect to pain medication and it meets the standards for safety and efficacy.”

The investigative tigers at CR call for the FDA to approve only opioids that are “abuse deterrent.” Well, here’s what the FDA commissioner had to say on that subject (also in open public testimony that the CR story either missed or chose to ignore), “It doesn’t do any good to label something as abuse deterrent if it isn’t actually abuse deterrent, and right now, unfortunately, the technology is poor.”

Surprisingly absent from the CR story was any mention of the most promising of the FDA’s initiatives on abuse deterrence; a study (to be conducted by the National Institute for Pharmaceutical Technology and Education) to evaluate opioid product formulations and performance characteristics for solid and oral dosages. 

Unfortunately complex systems make for bad media coverage, while simplistic, dramatic demagoguing makes for sexier headlines.

Oops.

CR also believes another reason Zohydro ER should be recalled by the FDA is that it was approved, “against the recommendation of its own panel of expert advisers.” That’s true – but it’s not the whole truth.  What the CR authors left out is that, by a vote of 11-2, the experts affirmed that there was no evidence to suggest Zohydro had greater abuse or addiction potential than any other opioid.

“Facts,” as John Adams said, “are pesky things.”

What the newshounds at CR missed completely are the issues surrounding opioid misuse – at present the poor public health stepchild of abuse. In the United States, the use of opioids as first-line treatment for chronic pain conditions doesn’t follow either label indications or guideline recommendations.

In fact, 52 percent of patients diagnosed with osteoarthritis receive an opioid pain medicine from their doctors as first-line treatment as do 43 percent of patients diagnosed with Fibromyalgia and 42 percent of patients with diabetic peripheral neuropathy.

Payers in the healthcare system often impose barriers to the use of branded, on-label non-opioid pain medicines, relegating these treatments to second line options. The result is a gateway to abuse and addiction with opioids.

Another fact conveniently missed in the CR story is that the vast majority of people who use opioids do so legally and safely. A subset, approximately four percent of patients, use these medications illegally. In fact, from 2010 to 2011, the number of Americans misusing and abusing opioid medications declined from 4.6  to 4.2 percent.

How strange that Consumer Reports missed so many facts after all this time.

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Debased Silver

08/11/2014 06:41 AM |

From today’s edition of the New York Post …

From HIV to cancer to Crone’s, ObamaCare fails the sick

By Peter J. Pitts

Melanie Thompson and HIV patient Brian Albright look over his medical bills and correspondence with his insurance company in Atlanta. Photo: AP

It turns out ObamaCare didn’t solve the problem of “pre-existing conditions” after all. It made premiums more affordable for people with chronic health conditions that are expensive to treat — but at the price of sticking them with unaffordable co-payments for their medications.

The nonprofit AIDS Institute is suing four Florida health insurers for discriminating against HIV/AIDS patients. The complaint says these patients now face prohibitive out-of-pocket drug costs. Sadly, most of the plans sold via ObamaCare all across the country have similar problems — leaving those with chronic diseases without affordable access to the specialty drugs they need.

The Affordable Care Act limits the degree to which insurers can charge higher premiums for sicker patients. But ObamaCare plans found a way around these rules: impose higher out-of-pocket costs for all or most specialty drugs.

Consider the Florida suit. Carl Schmid, the AIDS Institute deputy executive director, says the plans follow a “pattern where every single [HIV/AIDS] drug for some plans was on the highest tier, including generics.” Under these policies, drug costs for AIDS patients can exceed $1,000 a month.

And Florida’s hardly unusual. A new report from consulting group Avalere Health found that a large majority of all ObamaCare exchange plans include similarly high out-of-pocket costs for patients with certain illnesses.

The breakdown of Silver plans (the most popular category) is particularly revealing. In seven classes of drugs for conditions from cancer to bipolar disorder, more than a fifth of these plans require patients to shoulder 40 percent of the medicine’s cost.

And 60 percent of Silver plans place all drugs for illnesses like multiple sclerosis and rheumatoid arthritis in the “formulary tier” with the highest level of cost-sharing.

Nearly every Silver plan across the country, in fact, puts at least one class of drug exclusively in the top cost-sharing tier. In effect, this leaves patients with a given condition — whether HIV or Crohn’s disease — without a single affordable treatment option.

Pre-ObamaCare, about half the states had a system in place for helping people with pre-existing conditions: state high-risk pool plans, which for years offered government-subsidized coverage to patients with pre-existing conditions. But the Affordable Care Act banned those pools.

So now, with exchange plans failing them, the chronically ill have nowhere left to go.

Allies of the insurance industry blame the drug companies for the high price of certain medicines. AARP policy adviser Leigh Purvis, for instance, says cost-sharing levels “wouldn’t be so high if the prices of drugs weren’t so high.” Health-policy advocate John Rother, meanwhile, claims that “reducing price is something [drug] companies could do tomorrow.”

But drug prices aren’t arbitrary. The average biopharmaceutical therapy takes $1.2 billion and anywhere from 10 to 15 years to bring to market. Firms must charge high prices for certain brand-name drugs to make back this substantial investment with enough left over to fund research into the next generation of treatments.

And while insurers like to complain that sophisticated therapies cost too much, they tend to ignore the far higher costs of denying these medicines to patients.

Increased co-pays result in “non-adherence,” failure to take prescribed medications. And that equals increased rates of hospitalization, chronic heart failure and premature death.

And this adds to health outlays: According to a recent study in the Annals of Internal Medicine, non-adherence costs the US health system from $100 billion to $289 billion a year.

In short, by making needed medications unaffordable — and by failing to cover newer, targeted therapies — insurers are jeopardizing patient health. And far from saving money, cutting off access to specialty drugs actually increases long-term health costs.

Specialty medications need to be treated as equivalent to other essential medical services — not as some luxury that only the wealthy can afford. But the ObamaCare law has only made the problem worse than ever.

Peter J. Pitts, a former Food and Drug Administration associate commissioner, is president of the Center for Medicine in the Public Interest.

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Embracing Che

08/08/2014 09:57 AM |

Here’s something you probably don’t know – once-upon-a-time I was a stand up comic. That’s why I was so intrigued when I got a call from the Daily Show asking for an interview on … opioid abuse.

Opioid abuse?

Well, the interview happened yesterday (the "correspondent" was the charming Michael Che) and it’s still a mystery to me where the humor is in this topic. So why did I choose to participate? (1) It’s a widely watched program that acts as the main source of news for a lot of people. Sad, but true. And (2) The segment will air with or without my comments. I’d rather have the opportunity to offer some truth even in the face of … comedy?

Or in the words of Molière, The duty of comedy is to correct men by amusing them.

The segment will run either just before or just after Labor Day. Stand by for more details.

PS/ Whatever the outcome, I will certainly not be as bad as Kathleen Sebelius!
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Channeling the Passion of the Patient Voice

08/06/2014 02:00 PM |

One of the hallmark pieces of FDASIA enshrines the concept of patient-focused drug development. Per the FDA’s own website:

Patients who live with a disease have a direct stake in the outcomes of the drug review process and are in a unique position to contribute to the entire medical product development enterprise.  Under FDASIA, the FDA will increase patient participation in medical product regulation.

• Patient Participation in Medical Product Discussions under FDASIA. Sec. 1137 of the new law will assist the agency in developing and implementing strategies to solicit the views of patients during the medical product development process and consider their perspectives during regulatory discussions.  This will include:
  - Fostering participation of FDA Patient Representatives as Special Government Employees in appropriate agency meetings with medical product sponsors and investigators; and,
  - Exploring means to provide for identification of potential FDA Patient Representatives who do not have any, or have minimal, financial interest in the medical products industry.
• Patient-Focused Drug Development under PDUFA V. The PDUFA V agreement provides for a new process enhancement under a commitment that will provide a more systematic and expansive approach to obtaining the patient perspective on disease severity or the unmet medical need in a therapeutic area to benefit the drug review process.  In other words, the patient perspective will provide context in which regulatory decision-making is made, specifically the analysis of the severity of the condition treatment and the current state of the treatment armamentarium for a given disease.

But patient input needs to be more than anecdotal – it needs to be data-driven so that divisional decisions can be more scientifically-driven by the patient community. And nowhere is that more urgently needed than in discussions over risk/benefit calculations.

The Center for Medicine in the Public Interest (www.cmpi.org) weighs in on this issue with a new paper by senior fellow, Dr. John Bridges (of the Johns Hopkins Bloomberg School of Public Health). His new paper, Identifying the Benefits and Risks of Emerging Treatments for Idiopathic Pulmonary Fibrosis: A Qualitative Study (The Patient, DOI 10.1007/s-40271-014-0081-0) provides important qualitative evidence on stakeholders’ views as to important issues associated with emerging therapies for idiopathic pulmonary fibrosis.

Bridges, et al., identifies multiple issues were identified spanning the impact of emerging therapies, including the need to document the patient experience with treatment, and factors associated with disease progression.

The paper the value of qualitative research both in understanding the benefits and risks of emerging therapies and in promoting patient-centered drug development.

Patient passion is important to share. Bridges and his colleagues go one step further and provide a pathway towards capturing that passion and channeling it into usable data that can be used to impact regulatory decision-making.   Read More & Comment...

Ian Read: LinkedIn and Locked on

08/05/2014 08:04 AM |

Ian Read (Chairman and CEO of Pfizer and the current Chairman of PhRMA) recently published a piece on LinkedIn under the title, Why Society Needs a Vibrant Pharmaceutical Industry: Improving Patients' Lives.

Towards the end, Read writes:

I recognize that there are differing views when it comes to society’s perception of the pharmaceutical industry. Many believe we are more focused on making profits rather than finding cures for patients, even though the industry has a long-standing commitment to providing patients access to needed medicines through many different programs globally. There is also a perception that we do not operate in an open and transparent manner when it comes to our clinical data and financial relationships with healthcare providers. This view lingers despite the significant steps that have been taken to increase transparency, even in the face of the current debate that rages over an individual’s right to privacy.

As an industry we are working diligently to improve our standing in society. We understand that we have a great responsibility. We are at the center of society’s desire and expectation for delivering potential cures and new lifesaving treatments. We will continue to fulfill that vital purpose.

Patients are waiting and we are working hard every day to earn their trust.

Fine sentiments and well-crafted words – but working hard alone isn’t enough to earn trust. Pharma must work hard to do the right thing. What does that mean?

Mr. Read offers the following:

Over the course of the past 50 years, this industry has tackled some of the leading causes of disease and life-threatening illnesses.

For example, today the number of people who have died from heart attacks and strokes has declined. In 2008 around 16 percent of the U.S. adult population was taking a statin to reduce cholesterol. This translated into 60,000 fewer heart attacks, 22,000 fewer strokes and 40,000 fewer deaths.

An article published in 2010 by the Journal of Health Economics found that from 1988 to 2000, improvements in cancer survival created an estimated 23 million additional life-years over this period.

And according to the World Health Organization, immunizations save an estimated 2.5 million lives every year. For every $1 the U.S. spends on childhood vaccinations, we save $10.20 in disease treatment costs.

Consider that pharmaceutical innovation has accounted for 73 percent of the total increase in life expectancy between 2000 and 2009 across 30 developing and high-income countries.

Those are, by any measure, extraordinary accomplishments. Why then is the biopharmaceutical industry so roundly pilloried in the press and so low in the general view of public opinion? Working hard, it seems, is not enough.

At  PhRMA’s 14^th annual meeting in Washington DC. this past April, Read said that industry needs to “fix the misperception gap.”

Pharma must embrace a new paradigm. Rather than focusing on traditional ROI (Return on Investment), they must now also consider Return on Integrity.

Integrity comes in many forms. Honesty. Virtue. Morality. It means not waiting to be told to do it or waiting to see what others do first. Integrity means being principled and, as my father used to say, “A principle doesn’t count until it hurts.”

Mr. Read’s article on LinkedIn is a good start – but there are many other issues that need to be addressed (such as comparative effectiveness, drug pricing, and off-label communications). Trumpeting accomplishments is both important and cathartic – but now its time for the America’s top drug honcho (and his counterparts at other companies) to move on to more contentious and complicated topics.

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Karen Ignagni: Bending the curve -- and the truth -- about the $1000 Pill

08/04/2014 11:15 AM |

Karen Ignagni's WSJ oped claims that spending on the new Hepatitis C (HCV) drug will hike health premiums and bring insurers to ruin. 

In fact, new drugs like Sovaldi will not only rescue patients, but also reduce the cost of health care.  The reason is simple. Cure are always less expensive than treating the same disease using no or halfway measures.  Would Ignagni wail about a cure for Ebola that cost $1000 a pill?

Before HIV medicines came to market, there were nearly 100000 people with HIV were hospitalized.   Today, about 38000 HIV patients are hospitalized each year.  Hospitalization rates plummeted thanks to HIV drugs. Without HIV medicines, 111000 HIV patients would be hospitalized each year.    Fewer people with HIV would be alive.

Sovaldi will produce similar savings.  Millman (a health actuarial firm) estimates 984510 with HCV will be treated between 2014 and 2020.  Assume everyone treated gets Sovaldi at $80000.  That would cost $78 billion or more accurately about $13 billion a year for six years.

Sounds dire right?  But Ignagni ignores how new HCV drugs eliminates a 48-week course of treatment for interferon (that is only effective half the time and has flu like side effects) costs about $6.12 billion year.   If hospitalization rates drop by half, it will save another $1 billion per year.  ($7 billion total.)   Over six years insurers will save $252 billion in the process.  Insurers will net savings of $174 billion.   

None of this takes into account that new HCV drugs increase life expectancy and well being so that people can work, pay taxes and cover the cost of health premiums.   Ignagni’s alarms about Sovaldi are intended to divert attention to these facts and to justify sticking more consumers with the cost of a treatment that saves lives and money. 
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Biosimilars: Like a Deer in the Headlines

08/04/2014 10:25 AM |

Sandoz’s Filgrastim Biosimilar Relies On Data Extrapolation, “The Pink Sheet” July 24, 2014

Biosimilars’ Next Hurdle In EU Is Physician Opposition To Extrapolation, “The Pink Sheet” August 4, 2014

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"Well" Fare Vouchers or Trading in FDA Futures?

08/01/2014 08:05 AM |

The Wall Street Journal reports:

Drug Firms Buy $67.5 Million Voucher to Speed FDA Review

Regeneron Pharmaceuticals Inc. REGN +5.80% and Sanofi SA SAN.FR +3.52% are spending $67.5 million on a novel bet they hope will help them outflank Amgen Inc. AMGN +5.43% in the race to get a new class of cholesterol drugs to the market.

The companies are paying the money to acquire a special voucher held by BioMarin Pharmaceuticals Inc. in a bid to hasten regulatory review of their drug alirocumab, one of an emerging group of medicines that lower cholesterol by targeting a gene known as PCSK9.

BioMarin was awarded the voucher early this year as part of an incentive program established by the U.S. Food and Drug Administration to encourage development of drugs for rare pediatric diseases. The voucher entitles the holder to ask the FDA for priority review of a drug application that would otherwise get a standard review. That could shorten the review process to six months from the standard 10 months.

BioMarin received the voucher in conjunction with FDA approval of Vimizim, a treatment for a rare pediatric condition called Morquio A syndrome that afflicts about 800 patients in the U.S. While BioMarin could have used the voucher itself, the program also allows companies to sell a voucher to another company. The voucher doesn't need to be used on a drug for a rare pediatric condition.

The voucher was the first to be issued under the pediatric incentive program, and also the first to change hands.

The complete Wall Street Journal story can be found here.

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Get Rid of PCORI Once and For All

07/31/2014 01:28 PM |

Scott Gottlieb has a good piece in The Morning Consult about how PCORI has morphed into a bloated grant giving agency with no function or purpose.

Scott commends the leadership of PCORI in trying to make the agency functional and focused and points out that, like much of Obamacare,  PCORI's lofty mission to be a key part of his  (the president's )purported goal of lowering healthcare costs was never to be realized.  PCORI was, like much of Obamacare, a poorly conceived idea developed by social scientists who believe the government can micromanage every aspect of healthcare.  And once it can't, well at least the same social scientists can get about $3 billion of money to support some more bland and useless research that will serve as the basis for yet another round of misguided social engineering.  

In fact,  PCORI was supposed to be like the UK's NICE.. An agency that used CER to bring health care costs to heel by rationing care.   To survive politically, the PCORI leadership quickly abandoned that objective and is focusing on grants that study how to include patients in patient centered research.  The real CER -- personalized medicine -- is not even funded for a variety of reasons not the least of which is that the PCORI staff deeply believe that central planners armed with patient-centered research on how to study patient-centered outcomes in a patient centered way using patient centered endpoints will do a much better job than point of care molecular diagnostics that capture and share gene expression data.. 


Gottlieb concludes: "will PCORI fulfill its lofty mission? Truth be told; it was never meant to. The gauzy rhetoric about an agency that would take on the tough questions was just a sales pitch to get PCORI through Congress. The agency’s undersized grants, its bureaucratic structure, and its pious mission were always going to frustrate its ambitions. Feel stressed by all this? Don’t worry. PCORI is studying that." 

You could fund a lot of great research on cures for $3 billion.  Here's for getting rid of PCORI and using the money for real medical science. 

PCORI’s Efforts Could Leave Obamacare Boosters Stressed Out  Read More & Comment...

Biosimilar Nomenclature's Geneva Convention

07/31/2014 07:29 AM |

Per a notable  squib in BioCentury, the World Health Organization published a draft proposal for a global system to assign "biological qualifiers" to biologics and biosimilars. A biological qualifier is a random four-letter code assigned to a biologic manufactured at a specified site. WHO said the scheme would be voluntary for each regulatory authority and applicable retroactively. The qualifier would not be part of a biologic's international non-proprietary name (INN), although WHO's INN expert group would oversee the scheme. WHO said the proposed scheme is intended to avoid separate national qualifier systems. Comments on the draft are due September 19.

The ill-considered alliance hyping non-differentiated nomenclature for biosimilars gets smaller and more marginalized every day.

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Multilingual Labels Lost in Translation

07/31/2014 07:13 AM |

According to an article in Pharmacy Times, “In California, pharmacists and patients face a catch-22: patients who cannot understand English say they cannot read the labels on their medications, and that translating the labels would help them. The act of translation, however, would create a situation in which pharmacists are dispensing medications that they cannot verify because they do not know the language in which the labels are written.”
 
The California Board of Pharmacy will consider whether drug labels should be translated into a language the patient understands at its meeting today.
 
California pharmacists would be legally liable for any mistake on the translated label, since they are not FDA approved.

There is only one FDA-approved label for any given product, and that is the label that is approved for use by the FDA in English. This appears to be a clear-cut case of Federal Preemption.
 
The full Pharmacy Times story can be found here.

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The New Pharma ROI -- Return on Integrity

07/30/2014 08:50 AM |

This past April, PhRMA held it’s 14^th annual meeting in Washington DC.

During his inaugural remarks, incoming PhRMA board chair Ian Read shared his concern about the industry’s failure in getting the message out about “the value we generate.” His key message, “We need to fix the misperception gap.”

Specifically he talked about the industry’s need to broaden the conversation from the economic performance of biopharmaceutical companies to the value that accrues to society and called for a “dialogue with society.” Bravo.

He asked, “Where are the headlines?” They’re not about societal value – and they need to be. There’s a strong story to tell. It’s not happening. And it needs to, because minus that narrative, nothing the industry wants to make happen (with government being a focus since the meeting was in Washington, DC) will be possible.

Read called for “industry speaking for itself.” After all, if you can’t be your own best advocate, you’re suspect in the minds of many – and rightfully so. He spoke to “better ideas and clarity” versus “more tactics.”

They were the right words – but what’s happened since that fine oration? One thing that comes to mind is the debate over the price of Sovaldi. Another is ASCO’s decision to get into the comparative effectiveness game. Both of these issues are tailor-made for a Read-led discussion on price vs. value. And neither has generated a regular and robust response from either industry or it’s trade association.

That’s not to say there hasn’t been a debate. The Center for Medicine in the Public Interest (www.cmpi.org) has been writing and speaking with both force and frequency on these issues as have other public policy institutes (aka, “think tanks”) and thought leaders across the healthcare policy spectrum.

But there has been precious little in terms of by-lined commentary from pharmaceutical executives – especially of the C-suite variety.

To achieve Ian Read’s noble goal of “dialogue with society,” there needs to be a … dialogue. And it can’t only be via third party groups – as worthy and invested in the debate as they are. Pharma must speak for itself. Can you quote any useful answers from the folks at Gilead relative to Sovaldi pricing?

Pharma must embrace a new paradigm. Rather than focusing on traditional ROI (Return on Investment), they must now also consider Return on Integrity.

Integrity comes in many forms. Honesty. Virtue. Morality. But it also means (in more common parlance) “doing the right thing.” It means not waiting to be told to do it or waiting to see what others do first. Integrity means being principled and, as my father used to say, “A principle doesn’t count until it hurts.”

The current risk-averse position of many in pharma does nothing if not reinforce the general perception that the industry only cares about profit. Mr. Read’s words hit the nail on the head – change is required and we must drive it! But the gearbox has remained firmly in neutral.

For there to be Return on Integrity, integrity must first be demonstrated – publically demonstrated with names attached. This is especially true in the age of social media where the public is watching and commenting. And nature abhors a vacuum.

In June the FDA issued “Guidance for Industry Internet/Social Media Platforms: Correcting Independent Third-Party Misinformation About Prescription Drugs and Medical Devices.” Per the FDA:

If a firm voluntarily corrects misinformation in a truthful and non-misleading manner and as described in this draft guidance, FDA does not intend to object if the corrective information voluntarily provided by the firm does not satisfy otherwise applicable regulatory requirements regarding labeling or advertising, if any.

From a regulatory perspective, that’s a lot of wiggle room and should provide significant food for thought in erring on the side of more rather than fewer voluntary corrective actions.

This provides industry with a tailor-made opportunity to demonstrate integrity at little or no risk – by correcting the mistakes of others about their products in a transparent and appropriate manner.

Who will step up to the plate? Who will be first? Who will earn the return on integrity?

Integrity without knowledge is weak and useless, and knowledge without integrity is dangerous and dreadful.
–Samuel Johnson

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First Among Unequals

07/29/2014 07:51 AM |

According to a new article in BioCentury :

Sandoz picked a relatively simple molecule for its first FDA biosimilars application, but the Novartis AG unit is presenting the agency with three tough regulatory challenges. Sandoz is asking FDA to allow it to share a non-proprietary name with the reference product, Neupogen filgrastim from Amgen Inc.; to extrapolate clinical data from a single indication to all five indications on the Neupogen label; and, sooner or later, to designate the Sandoz product as interchangeable with Neupogen.

The agency will face a more difficult scientific challenge when it reviews an application from Celltrion Inc. for its biosimilar version of Remicade infliximab, a mAb marketed by Johnson & Johnson and Merck & Co. Inc. Celltrion has publicly said it will submit an application in 2H14, and may have already done so.

As the first publicly disclosed biosimilars application in the U.S., the Sandoz Neupogen biosimilar application will force FDA to address unresolved policy issues and will demonstrate whether the agency has crafted a viable, efficient biosimilars review pathway.

One particular quote from Mark McCamish, Sandoz’s global head of biopharmaceuticals & oncology injectibles development for those of you following the nomenclature debate, “Biosimilar is a lousy term; it suggests to most physicians they are biodifferent.”

That’s one man’s opinion. But shouldn’t accuracy be applauded?

Much grist for the mill there.

Authored by BioCentury’s regulatory policy scribe, Steve Usdin, Putting FDA to Biosimilars Test is a story worth reading – and an issue worth following.

  Read More & Comment...

Off-Label Over There

07/28/2014 08:08 AM |

According to a story in the  Wall Street Journal, pharmaceutical companies are pushing back against decisions by cash-strapped European governments to reimburse patients for drugs that haven't been approved to treat their conditions.

In June, Italy became the first EU country to allow its national health system to pay for cancer drug Avastin when it is prescribed to treat age-related macular degeneration, or AMD, an eye sickness that can cause blindness. The European Medicines Agency hasn't approved Avastin for treating AMD.

On Wednesday, France's National Assembly passed a law allowing for the reimbursement of off-label medicine as an attached amendment to its social-security budget. It specifically mentions Avastin as an example of a drug that falls under this category.

A spokesman for the European Commission, the EU executive in charge of enforcing the bloc's laws, declined to comment on the French and Italian laws. However, he said the commission will launch a study on off-label prescriptions later this year to evaluate legal and scientific aspects.

But what about off-label communications? Can you have one without the other? And can only one side (meaning the government) share such information? That's not "academic detailing" -- it's more like "Euro-detailing."

In May, Richard Bergstrom, the director of European Federation of Pharmaceutical Industries and Associations, wrote a letter to Paola Testori Coggi, director general for health and consumers at the European Commission, expressing concern over what he called the promotion of off-label use by European health-care bodies. Mr. Bergstrom asked the commission to meet with an EFPIA delegation, which Ms. Testori Coggi accepted. A specific date for the meeting hasn't yet been set.

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Biosimilar Nomenclature and Some FDA Tea Leaves

07/25/2014 04:10 PM |

Per the debate over biosimilar nomenclature --Did somebody say distinct drug names assist in the reduction in medication errors?

Yes – and that somebody is the FDA.

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2014-N-1008]

Exploring the Possibility of Proprietary Name Reservation for Drug Products;

Establishment of a Public Docket

SUMMARY: The Food and Drug Administration (FDA or Agency) is establishing a public docket to discuss issues related to reserving proprietary names for drug products. During the negotiations for the 2007 reauthorization of the Prescription Drug User Fee Amendments Act (PDUFA IV), FDA agreed to several performance goals related to the review of drug and biological product proprietary names to reduce medication error. Among those goals, FDA and industry expressed an interest in exploring the possibility of “reserving” proprietary names for companies once the names have been tentatively accepted by the Agency. Accordingly, FDA is initiating a public process to discuss issues around reserving proprietary names.

Link to Federal Register Notice: https://s3.amazonaws.com/public-inspection.federalregister.gov/2014-17691.pdf

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Jan Quioxte?

07/24/2014 02:05 PM |

From the pen of our friend and colleague Robert Popovian, Senior Director of Pfizer US Government Relations.

Paying for Value NOT Volume

While the US spends more on healthcare per capita than any other developed country, it also drives innovation in healthcare. Most of the R&D in medicine is done in the US, physicians from around the world are trained in our universities, the NIH invests in transformative research and premier academic medical centers are within our borders. However, in terms of payment and delivery we are still using an outdated model, spending our valuable healthcare resources on administrative red tape; non-adherence to medicines; fraud and abuse; and unnecessary services.

It is time that US takes a leadership role in innovating how to pay for and deliver healthcare by paying for value rather than volume.  Our current fee-for-service payment model encourages quantity not quality. Payments for outpatient services, hospital admissions, pharmaceuticals and provision of other healthcare services are based on individual budgets which only take into account cost rather than value and quality.  This approach promotes an environment where decision-makers in one silo have little if any regard for the consequences of their choices on other aspects of healthcare consumption, including outcomes and impact on patient quality of care.  Shifting healthcare spending is like squeezing a balloon – you squeeze one end and the other side pops up. If you inappropriately curtail one healthcare service, inevitably you will cause unintended consequences in others.  For example, policies implemented to reduce biopharmaceutical expenditures have oftentimes led to increasing overall healthcare utilization and costs through increased hospitalizations and outpatient services. In addition, most published research supports the fact that curtailing pharmaceutical access through policies such as inordinately high cost sharing or administrative hurdles increases overall healthcare costs (including a paper that I researched and published investigating the impact of pharmaceutical capitation payments by a national insurer on patient outcomes and healthcare expenditures as a research fellow at University of Southern California).

As policymakers consider payment and delivery reform, a plethora of words, phrases and acronyms such as capitation, bundled payment, pay for performance, ACO or IPU are being thrown about as potential solutions. Each of these is simply an approach that aligns incentives to pay for value rather than volume. But determining the best payment reform approach requires the inclusion of some core principles.

So what are the principles of successful payment reform?  Prioritize patient needs; support and promote sustainable high value care; examine health outcomes over a reasonably long term horizon; provide consumers and providers access to information and interconnected data; and encourage coordination of care. One example of a payment policy reform that did not incorporate these principles is the decision by some national Pharmacy Benefit Managers to remove certain medicines from their formularies without examining how such a decision would impact overall healthcare costs, or the burden on physicians or patient quality of life.  Ideally, payers would instead follow the decision of United Healthcare which recently instituted a bundled payment model for cancer therapy and found out that payments tied to outcomes and physician flexibility to choose the right therapy for their patients resulted in reduction in overall healthcare costs.

Changing the payment scheme towards value-based reimbursement will align incentives and spur growth of new delivery models such as telemedicine, retail clinics and even change when and how physician practices are operating.  Remember when a bank in the 1980’s operated on a Monday through Friday timeline with limited hours? Banks evolved to meet their customers’ needs through automation, changes in location of where customers could do their banking and most recently through technological advances that promote using online services.  Those banks that did not evolve eventually perished. Currently, our healthcare delivery system operates much the same way the banks did in the 1980’s.  In fact, healthcare is the only industry that keeps the fax machine industry alive!

Finally, we must implement payment reform with a “do-no-harm” priority towards innovation, managing the needs of patients who are very sick with hard to treat conditions, and guarding against decisions that are primarily based on short term financial gains at the expense of long term health.  By instituting appropriate quality measures and ensuring availability of interconnected healthcare data we can ensure that we avoid such dilemmas for delivery of healthcare services.

There is risk for the biopharmaceutical industry in moving from a fee-for-service model towards payment reform which rewards the most efficient intervention. However, economic principles also establish that opportunities exist for interventions that produce efficiency in systems.  Innovative biopharmaceuticals over time have proven to be the most efficient intervention in healthcare as their use commonly reduces overall healthcare costs and improves patients’ quality of care and life.

If payment reform fails as it has in the past, the consequences are dire. The only levers left to pull for policy makers to control the increase in healthcare costs will be draconian measures such as price and utilization management through mechanisms like the Independent Payment Advisory Board (IPAB). This approach won’t promote efficiency, value or a reduction in overall healthcare costs. Implementation of effective payment reform that results in new delivery model transformation will.

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Lies, Damn Lies, and the Dangerous Myths behind the Biosimilar Naming Issue

07/23/2014 10:17 AM |

As John Adams said, “Facts are pesky things.” And facts that don't reinforce your cognitive mapping are pesky things. But that does not change the fact that nothing deserves truth and accuracy more than the public health.

According to Inside Health Policy:

“While some say an easy solution is to simply add a suffix to distinguish a biosimilar from the innovator product, the compendia group told FDA any change is a change, and regardless of the simplicity of the change, the associated coding would also have to shift. First data bank and two other databanks are sold to stakeholders who assemble them and put overlays on them and then the data are used for different purposes, including for reimbursement.”

But pay heed. As Deming warned, “Change is not required. Survival is not mandatory.”

Giving credit where credit is due, the good people at the USP understand that interoperability is important, but that naming is more of a philosohpical issue. That's why they support differentiation via discrete numerical suffix.

Per Inside Health Policy:

The drug compendia stakeholders told a group of 13 FDA drug policy experts, including drug center chief Janet Woodcock, that changing the traditional naming process would require that each piece of the compendia process be individually rebuilt in order to ensure patient safety and restore functionality to the system. Doing so, while possible, would be difficult and could lead to confusion, errors and misunderstanding, creating a "very real risk to patients," according to slides presented at the May 2 meeting and a June 6 follow-up letter, obtained by Inside Health Policy.

Here are some of the more egregious myths shared by the aforementioned group of compendia stakeholders along with the facts to debunk them.

MYTH

We are concerned that distinguishable naming for every biologic, biosimilar and interchangeable biologic could confuse both providers and patients, and have the unintended effect of slowing the uptake of these cost saving drugs.

FACT

The patient and safety communities, as well as many physician organizations have weighed in on the same issue, and have come out on the opposite side – in favor of transparency, so that patients can know which medication is being put into their bodies and the whole system can quickly connect the dots to stem negative impact when an adverse event occurs. The scope of patient-focused organizations that have come out in support of distinguishable naming is so broad, it covers virtually every single American family.

MYTH

While we agree that it is important to gather data that allows providers to better understand how biologics and biosimilars are performing among various patient groups and to assist in the tracking of adverse events, as we mention above, we believe that the current mechanisms in place (e.g., NDC code, lot number, brand name, manufacturer, etc.) are sufficient.

Traditional naming structures do a good job of tracking post market issues (NDC, lot number, brand name, manufacturer, etc.).

FACT

Other, complementary tracking systems do and should exist, however non-proprietary names are the backbone of pharmaceutical tracking within payment systems, not necessarily for tracking and tracing AEs. The National Drug Code, or NDC, system provides a unique 10- or 11-digit set of numbers for each medication, however payers do not universally use NDC codes.

A lot number alone is not sufficient enough to identify a product and its manufacturer. It is only useful when it is accompanied by an identifier that is linked to the manufacturer.  While having brand names is useful for tracking and tracing AEs, they are not always used when prescribing or reporting AEs.

Additionally, there is a lack of standard use in medical benefit setting where the majority of biologics are administered and NDC are not necessarily present in patient records. When are entered there are many instances in patient care where NDC codes have been inaccurately entered.

Current approaches and systems do not allow for adequate collection of data relating to patient subpopulations, such as women, minorities and people with specific genetic problems; depending on the level of additional clinical research that will be required to bring a biosimilar to market, it is very likely there is much we won’t know about how a biosimilar might uniquely impact these subpopulations. Distinguishable names for biosimilars support the patient advocate and medical community’s vital post-approval learning curve to determine which medicines are best for patient subpopulations. 

The clarity from distinguishable non-proprietary names will:

·    Enable better safety monitoring.

·    Promote timeliness in managing adverse events if they occur.

Provide physicians with more information to understand which products are likely to be more effective in specific patient subpopulations.

MYTH

Requiring distinguishable names would segregate the safety data for brand and biosimilar products, making it more difficult to detect rare AEs across classes of products. (Study referenced:  2013 EMA report entitled “Traceability of Biopharmaceuticals in Spontaneous Reporting Systems: A Cross-Sectional Study in the FDA Adverse Event Reporting System (FAERS) and EudraVigilance Databases”)

FACT

The 2013 study referenced in this assertion did in fact find that 96.2% of adverse events could be traced back to biosimilars if either the brand name OR INN or company name were available. However, “…products for which only the INN was available were considered non-identifiable, except for epoetin zeta, for which product the INN differs from the innovator (epoetin alfa),” (pgs. 619-620). This is precisely why there needs to be distinguishable INN/ USAN names: so that products that can only be identified by INN/USAN can still be traced. Furthermore, the 2013 study cited actually found that 1 out of every 10 (90.4%) adverse events related to biosimilars can’t be traced back to a specific product if the biosimilar was given concomitantly or interacted with another medication.

MYTH

WHO has already established a global naming convention, known as the International Non-proprietary Names (INN) system.

FACT

The INN system was established in the 1950’s to identify active ingredients in small molecule chemical compounds, well before highly complex biologics were developed.

Unlike traditional pharmaceutical medicines (small molecule, chemical entities), where the active ingredient of a generic and the originator compound are identical, the active ingredient for biologics (large molecule) is complex, and a biosimilar will not be an exact replica.

WHO has not announced how it may alter its existing INN program to effectively serve biologics including biosimilars, however the organization’s published deliberations on the issue indicate change is likely:

“Compared to a small chemical entity, biotherapeutic proteins are large and complex, with four levels of structure (primary, secondary, tertiary and quaternary). The complexity of their structure is often further augmented by glycosylation and other molecular modifications, whose variability can impact on bio-activity. There are already several different naming policies for SBPs amongst individual regulatory authorities and in some cases, alternative interpretation of INN policy has led different authorities to assign different non-proprietary names for the same product. If prescribers rely on regulatory authority names, this will lack global consistency and could lead to different SBPs having the same name in different countries.

Four approaches are suggested on how to deal with this situation:

·      Continue with the status quo

·      Treat all SBPs as unique products and provide them with a unique INN

·      Create a biosimilar ‘identifier’ to be used for all SBPs (and not just glycosylated ones), e.g. use the original INN and add a fantasy code suffix

·      Encourage regulatory authorities to provide an ‘identifier’ under the guidance of WHO”

“The last two approaches fulfill the need for a unique identifier of a biosimilar and it would be preferable for the WHO to perform this (i.e. the third option); if regulatory authorities are involved (fourth option), there is no guarantee that a name will be accepted and adopted globally. The naming of SBPs needs to be addressed globally and soon while the number of registered SBPs remains relatively small and with the INN programme being the best forum to achieve this.” (http://www.who.int/medicines/services/inn/55th_Executive_Summary.pdf)

MYTH

Changing the traditional naming process would require that each piece of the compendia process be individually rebuilt in order to ensure patient safety and restore functionality to the system.

FACT

Comparing biologics to small molecule products that share identical active ingredients misses the crucial point that biosimilars are not generics.  If the proper use of biosimilars requires modernizing the existing system of safety alerts, then it is imperative to do so.

Existing INN’s for small molecule medicines and their generic counterparts do not need new naming conventions, only biologics. This significantly reduces the alleged burden on the system.

As medicine and technology evolves, so should our naming and coding process. Patient therapies should be precise and traceable every step of the way; the cost of implementing computer upgrades should never be presented as an obstacle to ensuring the safety of patients.

MYTH

There is already a precedent for shared names that has not resulted in any known issues and are used effectively in EU, Canada, Australia and Japan.

FACT

The non-proprietary naming system in Europe (INN) has mainly been used for first-generation biosimilars, as second-generation products have only recently been approved for marketing in the EU. Historical data that is collected from first generation biologics will be largely irrelevant and will not provide an accurate picture of potential pitfalls as complex second generation biologics enter the marketplace.

Canada has one biosmilar on the market and has stated that it will likely follow guidance issued by WHO as it establishes its naming nomenclature system for biosimilars. Australia and Japan have established their own systems to biosimilars naming, both of which take a distinguishable naming approach. Australia system includes a shared INN and suffix and early evidence indicates successful entry and uptake.

Not mentioned by the cost-centric/anti-safety crowd are the well-documented situation in Thailand, which resulted from the use of shared non-proprietary names. From the Citizen Petition filed January 7, 2014 by Johnson & Johnson with the FDA:

“Between 2004 and 2007, despite our switch to coated stoppers, adverse event reporters worldwide reported 15 cases of erythropoietin antibody-mediated PRCA in patients with chronic kidney disease who had been administered subcutaneous epoetin alfa. Of these 15 cases, 11 occurred in Thailand. The Thai market included multiple epoetin alfa products and hospitals and pharmacists frequently switched patients among them, often with incomplete documentation. Despite an extensive investigation, we were unable to determine which product(s) were responsible for the PRCA in the Thai patients because we could not determine which epoetin alfa product(s) a patient had received or which of several products that a patient had received had caused the problem.

 In Thailand, several different erythropoietins were used, some of which shared the same nonproprietary name, and records did not reliably identify which specific product a patient had received. These factors confounded our ability to identify the product(s) responsible for the safety signal. To our knowledge, the product(s) responsible for the increased rate of PRCA in Thailand has never been identified.”

In the early 2000s, Thailand used non-distinguishable names for biological treatment of treat certain diseases, which lead to a dramatic increase in incidences of blood-related adverse events. Some of the products shared the same non-distinguishable name and records did not reliably identify which specific biosimilar a patient had received. These factors confounded the ability to identify the product responsible for the safety signal…When patients are switched between or among products, it can be difficult or impossible to identify the product responsible for an adverse event…contributing to the inability to identify the responsible product for cases of PRCA in the Thailand situation was the common practice of switching patients among the multiple epoetin alfa products available. And even where records identified which product(s) a patient received, it was often impossible to determine the particular product responsible for the PRCA because many patients had received more than one product.”

In order to prevent another life-threatening adverse event, more expensive registries were ultimately required in Thailand to better track these products and related outcomes.

MYTH

Distinguishable names would be “Contrary to Sound Economic Healthcare Policy and Congressional intent in BPCIA.”

FACT

The patient and safety communities, as well as many physician organizations have weighed in on the same issue, and have come out on the opposite side – in favor of transparency, so that patients can know which medication is being put into their bodies and the whole system can quickly connect the dots to stem negative impact when an adverse event occurs. In the views of these stakeholders:

·     “If untraceable biosimilars become the norm and should the hypothesis of biotherapeutic equivalency not prove to be correct it would likely cause significant morbidity and mortality and thus irreparably damage this market and consumer and practitioner confidence in this class of products.” – From January 30, 2014 comments submitted the FTC by Salvatore J. Giorgianni, Jr., PharmD, BSc, CMHE and chair of the American Public Health Association Caucus on Men’s Health and president of the Griffon Consulting Group, Inc.

·    Distinguishable names can prevent delays in determining the cause of an AE by creating a more expeditious route back to the origin of the problem and may avoid the recall of an entire class of biologics (which would be necessary if the specific medicine causing the adverse effect could not be readily identified.)”  - From a January 7, 2014 letter to the FDA signed by the Maryland State Medical Society.

Additionally, Australia opted for distinguishable codes for all biologics, and they appear to be experiencing successful rollout and uptake of biosimilars.

MYTH

The incident surrounding ado-trastuzumab shows the risk of confusion with prefixes and can cause patient safety concerns.

FACT

Safety and quality experts concluded that the lesson learned by the ado-trastuzumab incident is not that prefixes present a patient safety risk but instead that strong coordination is needed between FDA, USAN, National Library of Medicine (NLM), USP, Compendia, and HIT stakeholders regarding naming for all biologics, including biosimilars.

MYTH

Applying different names for the same biological drug ingredients:

•   Introduces confusion and unnecessary complexity

•   Is contrary to historical FDA practice and policy

•   Is opposed by virtually all pharmacy association stakeholders because it conflicts with normal pharmacy practice - employing an electronic database to recognize products by identifiers

•   Is unnecessary for product recall or other patient safety considerations

•   Undervalues the ability of existing systems (NDC- and Lot- based recalls) and new regulatory structures (track and trace) to provide adequate safeguards

FACT

The patient and safety communities, as well as many physician organizations have weighed in on the same issue, and do not believe distinguishable naming would introduce confusion and complexity, nor do they believe distinguishable names would hinder product recalls. They have come out on the opposite side – in favor of transparency, so that patients can know which medication is being put into their bodies and the whole system can quickly connect the dots to stem negative impact when an adverse event occurs.

Due to the fact that the FDA will characterize its assessment of biosimilarity (based on comparative analytical data) into one of four levels -- not similar, similar, highly similar or highly similar with a fingerprint-like similarity – it will become even more critical to provide transparency as to which biologic or biosimilar is being prescribed. Additional pharmacologic studies would be required to show that the identified difference is "within an acceptable range to consider the proposed biosimilar product to be highly similar to the reference product." FDA said only products in the top two tiers would meet the statutory requirement for analytical similarity under the Biologics Price Competition and Innovation Act of 2009.

The American Society of Health-System Pharmacists in a February 27 letter to the FTC that “…we do not oppose the addition of suffixes (e.g., alpha, beta) to the INN name if experts believe this approach is needed to facilitate pharmacovigilance.”

The Hematology/Oncology Pharmacy Association (HOPA) has stated its support for distinguishable naming in a published position paper:  “Health care providers, patients, manufacturers, and regulatory agencies must be able to identify that a product is biosimilar to the original branded medication, and they must be able to associate the medication with the appropriate therapeutic class to assure appropriate prescribing. Naming is important to avoid prescribing and dispensing errors. Further, biosimilars must be able to be easily tracked to monitor safety and quality. Pharmacists are uniquely positioned to understand the important role that naming will have in ensuring appropriate medication substitutions take place when biosimilars are used.”

Nowhere are the issues of safety, efficacy, and traceability more important than to those with Orphan Diseases. And nowhere are biologics more important for treatment. So let’s end this missive with the position of NORD.

In a letter submitted to FDA Commissioner Margaret Hamburg, NORD asks that the agency give serious consideration to the concerns of the rare disease community when setting policy regarding official names for biologics, including biosimilars. NORD President and CEO Peter L. Saltonstall writes,

With over 7,000 rare diseases identified and 30 million Americans affected, the patient population represented by NORD is extraordinarily heterogeneous, the letter notes. Without thoughtful and consistent naming protocols for biologics, there is the potential for significant confusion among treatment options and increased adverse events, both of which could jeopardize patient safety. Distinguishable naming of all biologics is imperative for health care professionals to deliver the degree of customized care that is routinely required for patients with complicated, uncommon and less well-studied diseases. Every patient deserves the care best suited for their medical situation and most likely to give them the best outcomes. Biologics are often the most advanced and effective treatments for patients we represent and everyone in the treatment continuum should be able to readily identify the specific drug product a patient was given.

… and you will know the truth, and the truth will make you free.

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AHIP'S ANTI-PHARMATISM PROPAGANDA DEBUNKED

07/22/2014 06:02 PM |

AHIP’s Deceptions and Distortions About Prescription Costs and Value : Part One



AHIP Deception:  Drug costs are unsustainable. 

Facts:  In 2013 prescription drugs were 8.9% of health care spending.   In 2022 they will be 9.1% of total health expenditures.  

(This and other data was compiled from CMS statistics.  National Health Expenditure (NHE) Amounts by Type of Expenditure and Source of Funds: Calendar Years 1965-2022 in PROJECTIONS format .  The raw numbers are here: NHE Historical and Projections 1965-2022 
The projections are based on the 2011 version of the NHE released in January 2013.)



Fact:  In 2013 prescription drug spending by AHIP was 12 percent of the $962 billion it spent on health care.  In 2022 it will be 12 percent of the $1.6 trillion private insurance will pay out for health care



And finally Rx spending by AHIP as percent of total US healthcare spending remains steady at 4 percent.




If you are going to lie, it has to have some element of truth to be effective.. 


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Can E&C Lift the Off-Label Gag Order?

07/22/2014 10:34 AM |

Today the House Energy & Commerce Committee’s 21^st Century Cures Initiative tackles the FDA and “communications.”

According to the committee’s own white paper:

Communication about how certain treatments are working in certain patients is happening through a multitude of media around the globe. These conversations between and among doctors, patients, researchers, and scientists in academia and industry should be facilitated. This includes the free flow of data, research, and results related to what a therapy or combination of therapies does or does not do well and in what types of patients. We need to harness the power of the Internet and social networks.

That’s a big topic – but perhaps the most important issue within that big tent is that of off-label communications.

Perhaps the first thing to note is that there is distinction between off-label communications and off-label marketing. And is a distinction with a difference. Off-label marketing means sharing information with the intent to impact sales. Off-label communications means sharing information to improve and advance the public health. One well-known moniker for off-label communications is “the free and fair dissemination of scientific data.”

Let’s start here: Facts do not cease to exist because they are ignored.

According to a 2011 notice in the Federal Register:

The Food and Drug Administration (FDA) is announcing the establishment of a docket to assist with our evaluation of our policies on communications and activities related to off-label uses of marketed products, as well as communications and activities related to use of products that are not yet legally marketed for any use, we would like to obtain comments and information related to scientific exchange.  FDA is interested in obtaining comments and information regarding scientific exchange about both unapproved new uses of products already legally marketed (“off-label” use) and use of products not yet legally marketed for any use. 

And the issue of “scientific exchange” comes front and center. According to the FR notice, To assist with our evaluation of our policies on communications and activities related to off-label uses of marketed products, as well as communications and activities related to use of products that are not yet legally marketed for any use, we would like to obtain comments and information related to scientific exchange.

The FR notice puts this request into perspective:

On July 5, 2011, a citizen petition was submitted by Ropes & Gray and Sidley Austin LLP on behalf of seven product manufacturers (Petitioners):  Allergan, Inc.; Eli Lilly and Co.; Johnson & Johnson; Novartis Pharmaceuticals Corp.; Novo Nordisk, Inc.; Pfizer, Inc.; and sanofi-aventis U.S. LLC under 21 CFR 10.30.  The citizen petition requested that FDA clarify its policies for drug products and devices governing certain communications and activities related to off-label uses of marketed products and use of products that are not yet legally marketed for any use.  Specifically, the petition requests clarification in the following areas:

1.  Manufacturer responses to unsolicited requests; 

2.  Scientific exchange; 

3.  Interactions with formulary committees, payers, and similar entities; and

4.  Dissemination of third-party clinical practice guidelines.

For some time, FDA has been considering these issues and is currently evaluating our policies on sponsor or investigator communications and activities related to off-label uses of marketed products and use of products that are not yet legally marketed for any use.  We have been considering what actions to take in the areas specified by the petitioners with respect to manufacturer responses to unsolicited requests; interactions with formulary committees, payors, and similar entities; and the dissemination of third-party clinical practice guidelines.

Specifically, the FDA asks:

• How should FDA define scientific exchange?  

• What types of activities fall under scientific exchange?

• What types of activities do not fall under scientific exchange?

• Are there particular types and quality of data that may indicate that an activity is, or is not, scientific exchange?

• In what types of forums does scientific exchange typically occur?  Should the use of certain forums be given particular significance in determining whether an activity is scientific exchange or an activity that promotes the drug or device?  If so, which forums?

• What are the distinctions between scientific exchange and promotion?  What are the boundaries between scientific exchange and promotion?

•  Generally, who are the speakers involved in scientific exchange, and who is the audience for their communications?

• Should the identity of the participants (either speakers or audience) be given particular significance in determining whether an activity is scientific exchange or an activity that promotes the drug or device?  If so, which participants would be indicative of scientific exchange and which would be indicative of promotion?

• How do companies generally separate scientific roles and promotional roles within their corporate structures?

• How should the Agency treat scientific exchange concerning off-label uses of already approved drugs and new uses of legally marketed devices? Please address whether there should be any distinctions between communications regarding uses under FDA-regulated investigation (to support potential approval) and communications regarding uses that are not under express FDA-regulated investigation.

• How should the Agency treat scientific exchange concerning use of products that are not yet legally marketed (that is, products that cannot be legally distributed for any use outside of an FDA- or institutional review board (IRB)-approved clinical trial)?

• Should investigational new drugs and investigational devices be treated the same with respect to scientific exchange? Why or why not? 

• Under 21 CFR 812.7(b), an investigational device is considered to be “commercialized” if the price charged for it is more than is necessary to recover the costs of manufacture, research, development, and handling.  Similarly, FDA considers charging a price for an investigational drug that exceeds that permitted under its regulations (generally limited to cost recovery) to constitute “commercialization” of the drug (see 74 FR 40872 at 40890, August 13, 2009; 52 FR 19466 at 19467).  What other actions indicate the commercialization of drug and/or device products?  If there are differences in the steps taken to commercialize drug products and the steps taken to commercialize device products, either before or after approval, please explain these differences.

As PhRMA wrote in a June 2014 statement:

To get the best possible health outcome for patients, FDA should revise its regulations to allow companies to share truthful, scientifically accurate, and data-driven information with healthcare professionals to inform treatment decisions.  Some examples of this kind of information include:

Observational data and “real world evidence” – Information on the safety and effectiveness of medicines taken from medical records based on actual use of approved medicines.

Sub-population data – Information on the safety and effectiveness of medicines in sub-populations including gender and race.  Such information can help healthcare professionals tailor their treatment to meet the needs of individual patients.

 Observational and comparative data – Information from the use of a medicine outside of randomized clinical trials, especially comparisons between two or more therapies.

 Pharmacoeconomic information – Healthcare economic data and information on the economic value of medicines can improve the efficiency of patient care.

 Information on medically accepted alternative uses of medicines – Information on new uses of approved medicines that are listed in major compendia and/or routinely reimbursed by the federal government and major payers.  As the National Cancer Institute states, “Often, usual care for a specific type or stage of cancer includes the off-label use of one or more drugs.”^[1] Healthcare professionals help patients by applying new uses of approved drugs in “every specialty of medicine.”^[2]  When patients are being prescribed medicines off-label, they deserve to know that their healthcare professionals have the latest information on these uses.

[1] See National Cancer Institute, Off-Label Drug Use in Cancer Treatment, available at http://www.cancer.gov/cancertopics/druginfo/offlabeldrug.

[2] Christopher M. Wittich, et; al., Ten Common Questions (and Their Answers) About Off-label Drug Use, Mayo Clinic Proceedings, available at http://www.mayoclinicproceedings.org/article/S0025-6196(12)00683-0/fulltext#sec3.

And it’s not just PhRMA – patient groups are keen to weigh in. Some examples:

NORD:

At the same time, the government severely restricts what drug companies can say about new research and about off-label uses, thus cutting off information from the most knowledgeable sources. The Congress should seek new policies that permit drug companies to share appropriate information without fear of enforcement action.

Ovarian Cancer National Alliance

In ovarian cancer, as in many oncology settings, patients receive “off-label” therapies, which are legal and often part of practice guidelines. Access to these therapies is critical to providing patients with the best possible care...

The Alliance is deeply concerned that these revisions will chill off-label use of drugs and the dissemination of scientific information about non-approved uses. We strongly urge FDA to reconsider these changes and remove any language that may curb patient access to medically-accepted and life-saving medications.

And from BIO:

Current law deals with the important question of providing payers and others with meaningful information regarding the pharmacoeconomic benefits of medicines. However, implementation of Section 114 of the Food and Drug Administration Modernization Act of 1997 (FDAMA) has undermined innovators’ ability to meet requests for such information. The committee could evaluate how this important provision could be implemented in a less restrictive way to allow manufacturers to discuss more fully the value to the healthcare system of their innovations.

More broadly, provision of other truthful and non-misleading information to providers, payers, and patients also should not be impeded by unnecessary and cumbersome regulatory restrictions or requirements. Such approaches hinder users of medicines from accessing information that can help them use the medicines most effectively.

A lot of questions and, it seems, a lot of potential regulatory mission creep. All the more reason for both Congressional input .. and oversight.   Read More & Comment...