Latest Drugwonks' Blog
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
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.
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.
