Latest Drugwonks' Blog
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.
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
The ill-considered alliance hyping non-differentiated nomenclature for biosimilars gets smaller and more marginalized every day.
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.
This past April, PhRMA held it’s 14th 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
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.
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.
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
From the pen of our friend and colleague Robert Popovian, Senior Director of Pfizer US Government Relations.
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.
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.
Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.
