Latest Drugwonks' Blog
A good name is better than precious ointment.
-- Ecclesiastes vii. 1.
U.S. approval of biosimilars promise to be a very good thing, but the devil is on the details. Unfortunately, we’re seeing a disturbing trend relating to one of those key details – naming nomenclature.
Advocates for nonproprietary names across innovator reference product biologics and the biosimilars associated with them dangerously miss the mark on the pivotal issues relating to naming. A new editorial in Nature Biotechnology demonstrates such misguided thinking. Wither their usual good sense?
Obviously much education remains to be done on this issue in the time between now and when FDA issues final draft guidance on naming. Because where FDA winds up on this issue -- nonproprietary names, nonproprietary names + identifier codes, unique names or somewhere in between – will significantly impact patients, providers, manufacturers, pharmacists, safety experts and others. We need to all side firmly with what's best for patients.
If you’re for patient safety, you can’t be against distinguishable naming. The WHO established the International Nonproprietary Names (INN) system in 1953 before biologics were a figment of anyone’s imagination. Through the INN system, innovators and generics that share the same active ingredient also share the same generic name, also called the INN. It’s worked pretty well for chemical compounds but, as has been acknowledged by WHO and regulatory bodies of every developed nation, biologics are not chemical compounds – they’re infinitely more complicated.
We need to learn from these market-based experiences of nonproprietary names in the EU and Thailand, and distinguishable names in places like Japan and Australia. We can also take valuable lessons from how approaches specific to naming of biologics lend themselves to more effective safety monitoring, pharmacovigilence, data collection, clarity and transparency.
While the U.S. National Drug Code system will continue to serve a purpose for both small and large molecules, we can’t count on it to be the be-all-end-all solution for safety monitoring for biologics. Not even close. Payers don’t universally use NDC codes, they are rarely present in patient records and they are often inaccurately entered when they are. Distinguishable names provide a necessary safeguard to maximize safety and credibility. It’s really that simple.
The FTC is holding a hearing on the topic of biosimilar naming on Tuesday. They have stacked the deck (with exceptions) and no one is expecting anything other than the susual cost-centric care-verse-patient-safety drivel. I’ll be there all the same trying (from the audience) to interject occasional bouts of patient-centric sanity.
When it comes to biosimilars, we need to be extremely thoughtful about how we set policy relating to these promising medicines and strike a balance that promotes health and safety, rather than forcing a binary response that is driven by profits rather than patients.
Here’s a non-biosimilar quote (with apologies to Mr. Shakespeare):
He that filches from me my good nameRobs me, enriches him,
And makes patients poor indeed
Pfizer has announced an update of its clinical trial data access policy that will “simplify and broaden access to information gathered in Pfizer-sponsored clinical trials. The updated policy builds upon and expands the company’s established methods of clinical trial information sharing, including Pfizer’s long track record of submitting for publication results from all interventional clinical trials in patients and its pioneering efforts to provide clinical trial results and data to study participants.”
Key elements of Pfizer’s expanded policy (effective January 1, 2014) are:
* Pfizer’s INSPIIRE public web portal for investigator-initiated research (iirsubmission.pfizer.com) will offer qualified researchers a standard form and process for requesting access to anonymized patient-level data from Pfizer-sponsored trials of approved (or discontinued) products/indications posted on clinicaltrials.gov that have been complete for 24 months.
* An external Independent Review Panel will consider all requests denied or only partially approved by Pfizer and make a final decision.
* Pfizer will publish (on Pfizer.com) synopses of clinical study reports (CSRs) filed with regulatory agencies for approved products for which basic results are posted in the clinicaltrials.gov registry (dating back to September 2007). These CSR synopses will include summary results for all primary and secondary endpoints; any data that could be used to identify individual patients will be removed.
* Pfizer will produce and distribute lay-language summaries of clinical trial results to trial participants who wish to receive them, starting with trials that begin enrolling in 2014, in countries where regulations permit.
* Pfizer is piloting the use of “Blue Button™” technology (launched by the U.S. Departments of Veterans Affairs and Health and Human Services) to enable Pfizer trial participants to download their own electronic clinical data collected in the trial.
According to Pfizer, their expanded clinical data access policy “also reinforces the company’s current practice of submitting for publication manuscripts for all interventional clinical trials in patients, regardless of outcomes, within 18 months of study completion, and the company’s commitment to register and post summary results for interventional human clinical trials to clinicaltrials.gov in the United States and to registries outside the United States as required.”
The full version of Pfizer’s expanded policy, the clinical data request form, the searchable CSR database, the Independent Review Panel membership roster and charter, and more information, including Frequently Asked Questions, are available at http://www.pfizer.com/TrialDataandResults.
According to the American Medical Association, cost estimates of inefficient health care claims processing, payment and reconciliation are between $21 and $210 billion. In the physician practice, the claims management revenue cycle consumes an unsustainable 10–14 percent of practice revenue.
The current system is all too often manual. It must be replaced by automated, transparent, unambiguous, real-time health care transactions.
The full AMA white paper on this issue can be found here.
A new article by Robert Oscar, RPh,President and CEO of RxEOB, furthers that argument. According to Oscar:
Prior authorization (PA) is a complex process that is often daunting and monotonous for medical practice managers. It is also costly in terms of economics and human life, in particular when PA requirements lead to patient medication needs “falling through the cracks,” as some patients abandon their prescriptions due to the confusion and delay of the approval process.
Some other important points from Oscar:
The original goal of PA was to save money, requiring physicians to justify to health plans the need for medications, diagnostic tests and procedures, but it has led to pharmacists having to spend an average of five hours per week handling PA requests. This is non-reimbursable time that is better served on direct patient care.
A nationwide physician survey indicates that more than 69 percent of physicians typically wait several days to receive a PA from an insurer for a prescribed drug, while 10 percent wait more than a week. While more than 52 percent of office-based prescribers utilize electronic prescribing methods, most of them continue to use paper-based methods for obtaining PA of medications from health plans, causing unnecessary delays for patients.
The good news is that a growing number of physicians and office managers are taking proactive steps to solve these issues by taking advantage of electronic PA (ePA), a technology that enables them to submit an authorization request through hand-held devices or via a web portal prior to pharmacy adjudication.
ePA speeds up health insurer response time, minimizes resources associated with manual processes, and helps to enhance the quality and utility of the PA process.
For physicians and office practice managers who want to reduce manual PA workflow, improve the quality of the PA enforcement process, automatically document all activities, reduce the PA approval response time, and increase their understanding of lower-cost therapeutic alternatives, ePA technology is the way to go. The best solution can enable physicians and their staff assistants to submit the PA request online via EMR or e-prescribing workflow, thereby mitigating unnecessary delays, improving the quality of patient care and enhancing the patient’s overall experience with care delivery process.
Oscar’s complete analysis can be found here.
Yesterday I participated in a conference on Content Marketing. (The complete program agenda can be found here.)
Content marketing?
As my sister (a mental health professional) asked me, “Content marketing? As opposed to what, bullshit marketing?”
Not a question I was prepared for over the Thanksgiving table but, from someone who we generically refer to as “a provider,” an honest and relevant one.
Rhetoric counts. Maybe a better phrase is “content management” – because then we can compare it to something more tangible – such as “financial management.” We certainly know what that is. You take a certain about of money (the “content”) and through a savvy understanding of the marketplace and using the legal tools and compliant instruments you seek to increase the value of your portfolio.
When it comes to healthcare, is it content marketing or content management – or is it yet something else?
Maybe a better way to ask the question is, if it’s content marketing, what is the content and to whom are we marketing it – and why?
Content marketing, as a business proposition, is about maximizing awareness, reputation, sales, market share – and the advancement of the public health. (And it needn’t be in that order.) Indeed, the purpose of content marketing is to maximize the potential of important, accurate and timely information.
So maybe we should be talking about content maximization – and a more three-dimensional agenda – that is to say, beyond sales acquisition to driving patient outcomes.
(Another reason to alter the nomenclature is to help recalibrate the corporate compass.)
Some important questions:
How can content maximization address the adherence/compliance quandary?
How can content maximization help healthcare communicators advance the use of new platforms and media?
Most importantly, how can content maximization help define healthcare communications in a post-blockbuster environment, specifically as it pertains to orphan diseases and the rise of personalized medicine?
Let’s consider how the goal of content maximization through the strategy of content management and the tactics of content marketing can help advance sales, corporate, and public health goals. (And, again, not necessarily in that sequence.)
The heart of content marketing is story telling. Savvy healthcare marketers need to move from ABC (“Always Be Closing”) to ABT (Always Be Telling).
In the world of 21st Century healthcare, companies must share their content from their own mouths – because in the ultra-transparent world of social media, you can't separate the story from the storyteller. And we shouldn't even want to.
Rhetoric counts. As Kurt Vonnegut wrote, we need to “transcend the bullshit.”
Amen.
It’s fast becoming an n of 1 world. A world where every disease is an orphan disease and success is measured by individual outcomes rather than large population studies such as CATIE or ALLHAT or the multitude of programs being funded by PCORI.
Small is the new Big means that we must also think differently about pharmacovigilance. While we must continue to capture adverse event data (and do a better job at that through social media channels and mobile apps), we must also strive to capture Substandard Pharmaceutical Events (SPEs). SPEs occur when a pharmaceutical product does not perform as expected – perhaps because of API or excipient issues. SPEs can arise because of an issue related to therapeutic interchangeability. When it comes to 21st century pharmacovigilance, we have to both broaden and narrow our views about bioequivalence to the patient level. Small is the new Big.
And when it comes to drug development, adaptive clinical trials and companion diagnostics further define the outsized urgency of small-scale thinking. Demonstrating outcomes on an n of 1 level is crucial not just for 21st century healthcare technology assessment (think “value-based reimbursement”) but also for physician pay-for-performance measures and, last but not least, for the benefit of actual patients
There’s a lot of lip service paid to the comment that “the era of the blockbuster is over.” Now consider that statement from the perspective of another industry – in the 21st Century would you rather be Blockbuster or Netflix?
Small is the new Big. And that means a focus on individual patient outcomes, which means a focus on the individual patient rather than the general population and on long term care rather than short term cost.
And it’s about time.CDER Staff:
Today, FDA announced it will require the removal of certain prescribing and use restrictions for the diabetes drug, Avandia (rosiglitazone), to reflect new safety information regarding Avandia’s cardiovascular risk. The changes include modifications to the drug label about cardiovascular safety, changes to the Risk Evaluation and Mitigation Strategy (REMS), and the removal of a requirement for the drug’s maker to do another study of the drug.
In 2007, concerns had been raised about an elevated risk of heart attacks and strokes, and related deaths associated with Avandia. FDA issued safety recommendations to the health care community and initiated an intensive look into the cardiovascular risk of the drug. In 2010, GlaxoSmithKline (GSK) released a long-term study called the Rosiglitazone Evaluated for Cardiovascular Outcomes and Regulation of Glycemia in Diabetes (RECORD) trial, which evaluated whether patients are at greater risk of heart attack or death when taking Avandia, as compared to standard-of-care diabetes drugs. As a result of this study, GSK said it could find no potential elevated risk.
Questions continued to be raised both internally and externally about Avandia’s safety and the RECORD study. In light of the inconclusive scientific evidence, I made the decision that Avandia could remain on the market but with restricted access, until we had further data about the cardiovascular risk of this drug. An independent analysis, or readjudication, of the RECORD trial was conducted by the Duke Clinical Research Institute (DCRI) and confirmed the original findings by GSK, lending stronger evidence about the safety profile for Avandia. In June of this year, we convened two advisory committee panels to discuss the results of the DCRI review of the RECORD trial. The majority of the advisory committee panelists concurred with the findings and voted in favor of easing the 2010 restrictions on the drug. Our action today is consistent with the advice of those expert panelists.
I believe the Center’s work on Avandia is noteworthy for at least three reasons, as it:
1) Exemplifies our lifecycle approach to drug regulation, and our ongoing work to monitor the safety and effectiveness of medications once they are on the market, and make necessary adjustments when the risk/benefit balance of a drug changes over time
2) Underscores the complexity of our decision-making processes, and how as regulators, we must make decisions about regulated products -- even in the face of uncertainty or in the absence of complete information about a drug’s safety or effectiveness
3) Reinforces the importance of our commitment to Equal Voice and our efforts to encourage viewpoints from our expert disciplines across CDER, and even when there is disagreement, to respect the views of each discipline and strive to make the best decisions possible based on the scientific evidence available and in the best interest of patients
A wide range of scientific disciplines and many people from a number of CDER offices have been involved in our efforts to characterize and communicate to the public the cardiovascular safety profile for Avandia. These offices include the Office of Surveillance and Epidemiology, Office of New Drugs, Office of Executive Programs’ Division of Advisory Committee and Consultant Management, Office of Communications, and statisticians in the Office of Translational Sciences. I greatly appreciate the work and professionalism of so many of our staff throughout this process and believe it can be instructive going forward in how we approach and attempt to resolve very difficult drug safety issues.
For more information about the changes announced today, visit FDA requires removal of certain restrictions on the diabetes drug AvandiaJanet Woodcock
Mom, Apple Pie, and NIH Funding
Is NIH funding really the be-all/end-all of healthcare innovation?
Mari Serebrov, Washington Editor of BioWorld writes, “With the sequestration blade set to indiscriminately shave federal programs again in January, a bipartisan group of senators is urging congressional budget negotiators to spare research dollars at the National Institutes of Health (NIH).”
But is NIH really the only game in town.
Such a singular focus on the basic research funded by NIH ignores the fact that other government agencies are doing important health care research, Peter Pitts, president of the Center for Medicine in the Public Interest, told BioWorld Today. That research also is getting nicked by the sequester razor.”
For instance, the FDA funds research into regulatory science and personalized medicine, despite a “very limited budget,” Pitts said. While regulatory science research may not seem as sexy as basic research into cancer cures, it’s necessary research, he added.
“Basic research is important,” Pitts said, “but it’s not the beginning, middle and end” of all health care research.
The concerns also ignore new government funding for other types of research. Pitts described the current U.S. funding of public health-related research as “a bigger pie with more people with knives and forks.” More research is taking place, but the NIH slice of the pie isn’t getting any larger.
A prime example of that growth was the creation of the Patient-Centered Outcomes Research Institute (PCORI) in 2010. Set up as a nonprofit under the Affordable Care Act, PCORI was charged with funding comparative-effectiveness research. While Congress began shaving NIH funding close to the jaw line, it mandated that $10 million be set aside in fiscal 2010 to fund PCORI’s activities, $50 million in fiscal 2011 and $150 million in fiscal 2012.
To date, PCORI, which is funded through transfers from two Medicare trust funds rather than general tax dollars like the NIH, has approved 197 research awards totaling more than $273.5 million. It also is committing more than $1 billion to research funding over the next two fiscal years. But that money will be used to “study other people’s research, rather than the basic research NIH promotes,” Pitts said.
Noting that all research is subjective, Pitts said it isn’t Congress’ job to approve individual research projects, but Congress makes the choices of how federal research dollars are spent in general.
The complete BioWorld article can be found here.
According to Peggy Hamburg ...
The difference between science and science fiction is a line that seems ever harder to distinguish, thanks in part to a host of astonishing advances in medical science that are helping to create a new age of promise and possibility for patients.
Today cancer drugs are increasingly twinned with a diagnostic device that can determine whether a patient will respond to the drug based on their tumor’s genetic characteristics; medical imaging can be used to identify the best implantable device to treat a specific patient with clogged coronary arteries; and progress in regenerative medicine and stem cell therapy using a patient’s own cells could lead to the replacement or regeneration of their missing or damaged tissues. Given these trends, the future of medicine is rapidly approaching the promising level of care and cure once imagined by Hollywood in futuristic dramas like Star Trek.
But these examples are not science fiction. They are very real achievements that demonstrate the era of “personalized medicine” where advances in the science of drug development, the study of genes and their functions, the availability of increasingly powerful computers and other technologies, combined with our greater understanding of the complexity of disease, makes it possible to tailor treatments to the needs of an individual patient. We now know that patients with similar symptoms may have different diseases with different causes. Individual patients who may appear to have the same disease may respond differently (or not at all) to treatments of that disease.
FDA has been playing a critical role in the growth of this new era for a number of years. Even before I became FDA Commissioner the agency was creating the organizational infrastructure and putting in place the regulatory processes and policies needed to meet the challenges of regulating these complex products and coordinating their review and oversight. It has been my pleasure to serve at FDA during this next exciting period and to help ensure that the agency continues to prioritize this evolution by anticipating, responding to, and encouraging scientific advancements.
I am very pleased to be able to present a new report by FDA as part of our ongoing efforts in this field. Paving the Way for Personalized Medicine: FDA’s Role in a New Era of Medical Product Development describes many of the exciting developments and looming advances in personalized medicine, lays out the historical progress in this field, and examines FDA’s regulatory role: from ensuring the availability of safe and effective diagnostic devices, to addressing the challenges of aligning a drug with a diagnostic device, to post-market surveillance.
Outside collaboration and information sharing is essential for this field to flourish. On Tuesday, the American Association for Cancer Research and AdvaMedDX held a fruitful daylong conversation on personalized medicine to treat cancer. I was one of the speakers, participating in a conversation with Dr. Francis Collins, the head of the National Institutes of Health. Our discussion focused in part on current status of drug and diagnostic co-development and the challenges and potential of whole genome sequencing, where data can be collected on a patient’s entire genetic makeup at a reasonable cost in a reasonable amount of time.
FDA is committed to fostering these cooperative efforts, as it will require the full force of government, private industry, academia and other concerned stakeholders to maximize our efforts and fully realize the promise of personalized medicine. Our new report outlines that commitment, and helps chart the way forward so that more people can live long and prosper.
Via the New Republic:
It would be an understatement to say that pertussis and other formerly conquered childhood diseases like measles and mumps are making a resurgence. Pertussis, specifically, has come roaring back. From 2011 to 2012, reported pertussis incidences rose more than threefold in 21 states. (And that’s just reported cases. Since we’re not primed to be on the look-out for it, many people may simply not realize they have it.) In 2012, the CDC said that the number of pertussis cases was higher than at any point in 50 years. That year, Washington state declared an epidemic; this year, Texas did, too. Washington, D.C. has also seen a dramatic increase. This fall, Cincinnati reported a 283 percent increase in pertussis. It’s even gotten to the point that pertussis has become a minor celebrity cause: NASCAR hero Jeff Gordon and Sarah Michelle Gellar are now encouraging people to get vaccinated.
How responsible are these non-vaccinating parents for my pertussis? Very. A study recently published in the journal Pediatrics indicated that outbreaks of these antediluvian diseases clustered where parents filed non-medical exemptions—that is, where parents decided not to vaccinate their kids because of their personal beliefs. The study found that areas with high concentrations of conscientious objectors were 2.5 times more likely to have an outbreak of pertussis. (To clarify: I was vaccinated against pertussis as a child, but the vaccine wears off by adulthood, which, until recently, was rarely a problem because the disease wasn't running rampant because of people not vaccinating their kids.)
So thanks a lot, anti-vaccine parents. You took an ethical stand against big pharma and the autism your baby was not going to get anyway, and, by doing so, killed some babies and gave me, an otherwise healthy 31-year-old woman, the whooping cough in the year 2013. I understand your wanting to raise your own children as you see fit, science be damned, but you're selfishly jeopardizing more than your own children. Carry your baby around in a sling, feed her organic banana mash while you drink your ethical coffee, fine, but what gives you denialists the right to put my health at risk—to cause me to catch a debilitating, humiliating, and frightening cough that, two months after I finished my last course of antibiotics (how’s that for supporting big pharma?), still makes me convulse several times a day like some kind of tragic nineteenth-century heroine?
If you have an answer, I’ll be here, whooping, while I wait.
Read the full article here.
According to a new letter to the British Medical Journal, “Doctors may not report adverse events or speak up when they witness poor care because of fear of punitive action or lack of confidence that reporting will change anything.”
Whose responsibility is it to build confidence in both the pharmacovigilance process as well as the urgent importance of the proposition? I believe it’s the job of the regulatory body that oversees the both the procedures and the actions that derive from post-marketing reports. That means (in the US), the FDA.
Would it be nice if the FDA could get additional dollars to ramp up pharmacovigilance beyond MedWatch and Sentinel? Sure, but let’s get real – that ain’t gonna happen. What the FDA does have at its disposal is the bully pulpit. It’s time for Janet, Peggy, and Gerald to get up on their soapboxes and start preaching the urgency and importance of pharmacovigilance.
Such renewed efforts are called for since the focus is now increasingly on patient outcomes. If we can’t measure it, it doesn’t count – or counts for less. Perhaps it’s time for the FDA to create an advisory committee of pharmacovigilance issues – and hold meetings to focus on process improvement, greater stakeholder involvement (not just with physicians, but with pharmacists, hospitals and patients), and better ways to share post-marketing data in a transparent and timely fashion – and not just adverse events and label updates but also Substandard Pharmaceutical Events (SPEs), when patient’s don’t respond as they should when their therapy has been impacted by therapeutic switching or interchangeability/bioequivalence issues.
Can you say, “biosimilars?” It's an adverse event horizon.
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.
