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.

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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.

  Read More & Comment...

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.. 


  Read More & Comment...

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...

Sovaldi and the Price/Value Debate

07/21/2014 07:38 AM |

From today's edition of the New York Post.

Why ‘pricey’ drugs save money — and lives

Expensive new drugs often get fingered as the culprit to rising US health-care costs. The truth is closer to the reverse.

First off, it’s hard to see how pharmaceuticals can be a major driver of costs when they’re just over 11 percent of the total US health-care budget.

But more important is that even extremely pricey drugs still save money if used right.

Consider Sovaldi, which has a 90 percent cure rate for Hepatitis C, a disease affecting over 3 million Americans. A three-month treatment cycle of the new drug costs upward of $84,000. On the market for just a few months, Sovaldi has already clocked in a record-shattering $2.3 billion in sales.

Some are calling foul, accusing the drug’s developer — Gilead Sciences Inc. — of exploitative pricing. “The company in this case is asking for a blank check,” says Karen Ignagni, president of America’s Health Insurance Plans. “It will blow up family budgets, state Medicaid budgets, employer costs and wreak havoc on the federal debt.”

That’s 100 percent wrong — the exact opposite of reality. New, better medications are actually the best and swiftest way for this country to cut down on our health-care expenses. By more effectively combating disease and improving patients’ lives, drugs reduce long-term medical costs and bolster the overall economy.

Consider one pre-Sovaldi “best practice” treatment for Hepatitis C, the drug Pegasys. This requires one injection a week for 48 weeks — and very few patients see the treatment through to completion, so much of that treatment, both physician time and drug cost, is wasted. Nor is it that much cheaper: At about $7,000/month, the full course of treatment is over $70,000 — barely less than cost of the three months needed for Sovaldi to work a cure.

And the price of not using Sovaldi is very high. One in three patients with the Hepatitis C virus eventually develops liver cirrhosis, and managing these patients is costly. A “routine” liver transplant (where the liver is from a cadaver) costs close to $300,000; a “living donor” transplant is even more expensive.

Thanks to Sovaldi, a pill that cures the disease when taken once a day over 12 weeks will eradicate the need, the risks and the costs of liver transplantation. Such radical innovation deserves to be both lauded and rewarded.

And Sovaldi’s costs will come down. The initial price of such breakthrough medications reflects the huge R&D costs required to bring the drug to market, not avarice.

As Food and Drug Administration official Dr. Janet Woodcock noted of the Sovaldi controversy: “We may have to put a big down payment down now to get something really good.”

It’s remarkable that some large insurers have the chutzpah to complain that curing 3 million Americans of hepatitis C will bankrupt health-care systems. Data recently published by the PwC Health Research Institute suggests the reverse. The study shows that the use of Sovaldi will actually drive down overall spending within a decade. According to the authors, “The challenge may lie in targeting the patient most in need of the more expensive course of therapy.”

In short, drugs aren’t the cause of rising health-care costs — they’re the solution. Demonizing new treatments distracts from the real problem in the US biopharmaceutical industry: top-down cost-centric policies that focus on the near-term, short-changing long-term patient outcomes, and so endanger “sustainable innovation” by denying fair reimbursement for high-risk investment in R&D. (Research and development costs big even if a drug never makes it to market — and most don’t.)

New treatments are a bargain. Disease is always much more costly.

Unfortunately, under ObamaCare health plans are sticking more people with a bigger share of drug costs — a strategy designed to discourage use by the people in greatest need and direct outrage away from insurers to drug companies.

Breakthrough drugs could generate huge new savings in the US economy — but only if federal regulators don’t smother them in the womb with expensive and unnecessary legal hurdles. Left unencumbered, domestic medical innovation will generate the new treatments to improve lives, stave off disease and cut down on long-term health-care costs.

If we don’t reward risk-taking on behalf of human health, both will shrink.

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

Patient, treat thyself. Maybe.

07/18/2014 06:52 AM |

Whether it’s allergy medications, treatments for erectile dysfunction or high cholesterol, the issue of Rx-to-OTC switching is complicated, important – and timely. The FDA’s September 2011 draft guidance provides a valuable resource for those thinking about proceeding with OTC switches based on self-selection studies. It's fair to say that support of Rx-to-OTC switches reflects FDA's interest in drilling down for greater insight into consumers' thought processes.

Can a patient self-diagnose and self-dose?  Do symptoms hide another, potentially more serious, underlying condition?  And what of safety concerns?  In considering an Rx-to-OTC switch, the FDA looks to see whether patients can use the product safely without the oversight of a physician or pharmacist. This includes ensuring that the right patients use the drug in the appropriate way ("safe use"). The agency is not evaluating the risk-benefit of the product, since that was done earlier when it was approved as a prescription drug.

The journal SelfCare has published "an essential blueprint for designing and implementing any Rx-to-OTC drug development program," says lead author Bill Soller, professor and executive director of the Center for Consumer Self Care at the University of California, San Francisco, School of Pharmacy.  Well, maybe not essential -- but certainly intriguing.

Soller and his colleagues list questions - "OTC Considerations" - based on switch principles FDA set in 1990 and 1998 and questions to post-2002 advisory committees that evaluated first-in-class switches.

The analysis recommends 11 primary questions about the Rx fundamentals of a drug, its "OTCness" and overall risk vs. benefit.

1- Has the Rx product been on the market for a sufficient time and extent to enable full characterization of the drug's safety profile?

2- Can the condition be adequately self-diagnosed or is there a need for physician diagnosis?

3- Is the minimally effective dose known?

4- Are there efficacy studies needed to support the intended OTC use of the switch candidate?

5- What are the patterns of diagnosing, prescribing and patient use in the Rx setting related to OTC intended use?

6- Are the studies supporting OTCness generalizable to the intended OTC target population?

7-Do consumers understand key communication objectives of the label, relating to directions for use, contraindications, in-use warnings and precautions?

8- Do consumers show they would be likely to be able to assess and take action on the treatment effect (e.g., take appropriate action if the drug is not working, serious side effects emerge, or self-monitoring is needed)?

9- Do consumers demonstrate successful self-selection and de-selection of the product under conditions (or simulated conditions) of actual use?

10- Does the pattern of actual use support that the label can be successfully used in practice?

11- Do the benefits of OTC availability outweigh the risks?

The authors say FDA "uses its discretion to select areas of concentration for advisory committee discussions on switch." Factors influencing the agency's questions for advisory committees include the novelty and uniqueness of a proposed OTC indication or Rx active ingredient; intrinsic and extrinsic toxicity of a switch candidate; and robustness of published and NDA-derived data and worldwide post-marketing surveillance evaluations.

But there are other important factors at play that, while not entirely within the scope of FDA’s regulatory authority, need to be considered within the context of the broader Rx-to-OTC switch conversation. These factors fall under the general headline of “the consumer healthcare continuum.”

Today’s healthcare consumer has access to much more information than ever before. (Some of it is even accurate.) More importantly, they seek choice and empowerment. At the same time healthcare providers are under multiple pressures to further decrease the amount of time spent with their patients. Moving certain products from Rx to OTC would certainly decrease (in a safe and appropriate manner) a provider's patient load, while at the same time obviating the need for a patient (otherwise known as a “consumer”) to spend both the time and money (office co-pay as well as time off from work) required for an office visit -- allowing physicians to focus their limited time on high-risk patients or those with more complex conditions.

And what about the evolving role of pharmacists and the important question (among others) of pharmacy scope of practice (blood tests, etc.). The removal of the Rx designation doesn’t require the removal of a learned intermediary.

There is also good research showing that patients on OTC medications demonstrate higher rates of utilization. A 2013 study indicates that offering OTC forms of a prescription medication increased utilization per drug class by 30 percent, potentially closing treatment gaps.  And, in contrast with many Rx treatments for chronic disease (such as high cholesterol), adherence rates are higher for OTC medications.

Patient, treat thyself. That’s one way of looking at it. Knowledge is power. But is a little knowledge a dangerous thing?

The consumer healthcare continuum continues.

  Read More & Comment...

Biosimilar Saftey and the Real Patient Voice

07/17/2014 07:35 AM |

Desperate too-clever-by-half efforts to derail biosimilar pharmacovigilance are contrary to the public health and require rebuttal.

Some generics manufacturers, payers, and national chain drug stores have interwoven half-truths, out of context comments and just plain misinformation to build a fatuous argument dressed up as support for patient safety. But the actual patient and safety communities are firmly on the polar opposite side – in favor of safety, choice, and transparency.

The insurance-industry driven effort claims that distinguishable names for biologics “could lead to patient and prescriber confusion, increasing the possibility of medication errors.”  Unpacking that a bit, biologics are complex therapies that are made from living cells and prescribed for patients with difficult-to-target, debilitating and life-threatening health conditions such as cancer, diabetes, MS, lupus, Crohn’s disease and rheumatoid arthritis. There are no generics for them but it is expected that the closest thing –  biosimilars – will soon enter the US marketplace. What the insurers are essentially saying is that, a biosimilar that relates to a biologic should have exactly the same name as that biologic. 

In the case of biologics and biosimilars, even minute differences between products can cause individual patients to respond differently even though each product is considered safe and effective. Of greatest concern is immunogenicity, which carries a significant risk for all biologics. Because of the size and complexity of the biologic molecule, patients can experience unwanted immune reactions; these reactions can occur months after a patient begins taking the medicine and it is often difficult to know if it is due to the reaction to the medicine or simply the progression of the patient’s condition.

With all of these considerations, it stands to reason that the name of biologics and biosimilars should be similar, not the same. But rather than taking my word for it, consider these important viewpoints:

“Distinguishable names…will enable the gathering of sufficient data to ultimately allow providers to fully understand how all biologics – including biosimilars – are performing for minorities. This will lessen the inevitable confusion and assure optimum medical care in the use of biosimilars among minority populations.” – From a June 10th letter to FDA signed by 22 of the leading U.S. organizations focused on minority health, including the National Alliance for Hispanic Health, National Hispanic Medical Association and National Medical Association.

“…For millions of female patients, any potential increase or decrease in effectiveness of a biologic, along with side effects and adverse reactions, will only be discovered after the treatment is approved and under active use…As states across the country look to the FDA for guidance on issues surrounding biosimilarity, interchangeability, and therapeutic substitution, the agency’s views on sex and genomic-based differences will be crucial…Distinguishable names will enable the gathering of sufficient data to ultimately allow providers to fully understand how all biologics – including biosimilars – are performing for both men and women.” – From a May 20th letter to FDA spearheaded by the Society for Women’s Health Research and signed by 45 other leading U.S. organizations focused on women’s health.

“Distinguishable names for biologics support the medical community’s vital post-approval learning curve about which medicines are best for their rare disease patients. Health care providers need to know that a prescribed medicine was actually given to the patient and whether a substitution was made and to what alternative product. This can’t be achieved unless biologic products—especially ones with similar therapeutic purposes—cannot be distinguished, tracked and studied.” – From a June 3rd letter to FDA by the National Organization for Rare Disorders (NORD), the umbrella organization representing the interests of the many advocacy groups that serve 30 million patients impacted by nearly 7,000 rare diseases.

By my estimate, the patient-focused organizations that have come out in support of distinguishable naming cover just about every single American family. Fully addressing all of the inaccuracies and agenda-driven language in the insurance-industry driven letter requires an extensive point-by-point rebuttal [link to one], but the key take-away is this:  if you’re for patient safety, you can’t be against distinguishable naming.

As acknowledged by WHO and regulatory bodies of every developed nation, biologics are not chemical compounds (e.g., statins) — they’re infinitely more complicated. When it comes to biosimilars, we need to be extremely thoughtful about how we set policy and strike a balance that promotes health and safety, rather than forcing a binary response that is driven by profits rather than patients.   Read More & Comment...

What Pharma Can Learn From World Cup Soccer

07/15/2014 09:15 PM |

Pharma companies have to take a page from the World Cup champion German soccer team and stay on offense about innovation.. all the time.    As the Wall Street Journal notes:

“After years of timid, low-scoring play, that one word has become the theme of Brazil's World Cup. When the history of this tournament is written, the sport's cognoscenti will likely point to it as an event that changed the game. The finalists, Germany and Argentina, have survived the most offense-oriented tournament of the modern era, a series of games where playing defensively almost guaranteed an early exit.”

Pharma could learn a lot from the World Cup champion German club.   Stay on offense. 

Last night I attended a dinner hosted by Poppy McDonald the publisher of The National Journal to discuss what “value” means to various health care interests.   Attendees included Chris Jennings, who advised both the Clinton and Obama administrations on health care reform, John Rother, the former head of policy for AARP (and now CEO of the National Coalition on Health Care), Nancy Ennis-Davenport who is the CEO of National Patient Advocacy Foundation and Alex Wayne, who writes about health care for Bloomberg.  Julie Rovner led the discussion. 

It was a lively group and the discussions were spirited and friendly.  The conversation quickly turned to and focused on the price of Solvadi and whether it was reasonable or not. 

I was struck by how, for the most part, few at the table thought in terms of what Solvadi would replace, how much money it would save relative to the cost of treating people with liver disease, the horrible side effects of current treatments and how a 12 week cure could and would affect productivity, disability costs, etc., etc. 

And while there was a lot of moaning about drug prices, there was scant discussion about the price of liver transplants, bone marrow grafts, intensive care, etc. 

Innovator drug firms have usually done a lousy job thinking about or making the case for the value of their products.  But even when they are good at it, they are not consistently making the case.  Too often they are on defense, using lobbyists to fend off regulation with apologies and explanations about the price of drugs.

And by playing defense, pharma, more than ever, is in danger of being booed and reviled.  The audience expects both sides to mount an offense.  Thus, “A team that dared to play passively for even the briefest period of a match was guaranteed to hear derisive howls from the Brazilian crowds, no matter how hot and humid it was or how tactically intelligent slowing down the game might have proven.
"Every team has realized you need a balance, that you need to attack and defend," said Avram Grant, the former manager of the English club Chelsea. "If all you do is defend, you lose."

All pharma does is play defense. At it’s peril. That’s what the dinner discussion about Solvadi and the World Cup showed me.  Read More & Comment...

Pet Peeves

07/15/2014 08:57 AM |

Imagine waking up in the morning and finding your beloved pet on the floor convulsing, motionless or dead.

Now stop imagining, because this was the real-life nightmare for horse trainers in both Lexington, Kentucky and Ocala, Florida when they found their horses experiencing seizures and thrashing in their stalls. In total, more than a dozen horses died or were severely injured – in some cases, paralyzed.

The cause of these horrific scenes – illegally compounded medicines.

These horses were prescribed illegal drug concoctions that contained unsafe levels of pyrimethamine. By one account 25 times the amount it was supposed to contain. A deadly overdose for innocent animals.

There is legal and appropriate animal drug compounding.  All legally compounded product starts with an FDA-approved product.  The product may then be modified by a trained pharmacist on the order of a veterinarian to treat the medical needs of an individual or group of animals such as adding flavorings, or turning tablets into an oral liquid for easier administration.

But some animal drug compounding pharmacies go far beyond those parameters – with deadly results.  They mass produce and market drugs that attempt to mimic FDA-approved products, often using untested bulk active ingredients, imported from countries that may not enforce the strict controls on drug manufacturing that FDA requires for approved products. These copies carry a cheaper price tag than approved drugs, but none of the consumer protection.

Why does this black market exist? It’s both expensive and time-consuming to take a drug through the FDA approval process. For animal drugs, this can take up to 10 years and cost up to $100 million.  But it is a process that protects consumers and their animals – by ensuring that the approved drug is both safe and effective at the labeled dose.

FDA permits a limited amount of necessary compounding from bulk ingredients in order to make sure medical needs can be met when there is no approved drug.  But these compounding pharmacies have taken the proverbial inch and run for murderous miles.  Despite their attempts to muddy the waters with lawsuits and rhetoric, the law is clear: FDA and three federal appeals courts have ruled that compounding animal drugs from bulk substances is illegal. Period.

Illegally compounded animal drugs have caused harm to more than just horses. One university veterinary hospital has reported that dogs and cats are brought in for medical care because their medicines aren’t working, only to find they were being treated with illegally compounded drugs.  The literature is full of reports showing these compounded drugs contain far more or far less than the amount of advertised active ingredient.  This is not acceptable.

As a former FDA Associate Commissioner (and a current dog owner), I know the agency has rigorous enforcement authority.  Pharmacies that engage in illegal manufacturing cannot be allowed to ignore the law and put animal health at risk in the name of selling a cheaper product. 

For several approved animal drugs, FDA has sent warning letters to these pharmacies, but often they were simply ignored and the illegal practices continued.  Unfortunately there has been little to no follow up by the agency. The FDA must do more and commit to strong and sustained enforcement to protect animal health.

What can you do? It’s frightening to think a drug given to your pet might not carry the guarantee of safety and effectiveness that comes with FDA approval.  The first step is to talk to your veterinarian about best treatment options for your pet.  Ask if the drug being prescribed is FDA-approved.  If there’s no approved drug and compounding is necessary, make sure the pharmacist preparing the compound has the credentials and license to do it safely and legally. Why that might sound obvious it is the only – and best – way to ensure the prescribed drug is what is best for your pet. If you have concerns about a pharmacy, check with your state Board of Pharmacy.  At the same time, FDA must do its job of protecting our pets and animals by more regularly and aggressively enforcing the law and regulations.    

  Read More & Comment...

The Dangers of Mixing Libertarianism and Access to Experimental Medicines

07/14/2014 07:12 AM |

Per pending state legislation and a patient's riights to experimental medicines -- pure libertarianism isn't in the best interests of the public health.

Here is my commentary in the most recent edition of BioCentury.

Net/Net -- let's not do the wrong thing by trying to do the right thing.  Read More & Comment...

Cracking the Social Media Code

07/11/2014 07:51 AM |

From the pages of the Burrill Report …

Cracking the Social Media Code

The Burrill Report

The U.S. Food and Drug Administration recently issued two long-awaited draft guidances for drug and device makers' use of social media. The first concerns risk and benefit information, and the second addresses correcting misinformation published by others on the web. We spoke to Peter Pitts, president of the Center for Medicine in the Public Interest, about the issues of concern to the agency, why the industry has been slow to embrace social media, and how these new ways to communicate with patients are accelerating broader changes in the healthcare landscape.

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