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Medical errors are a real problem. I won’t deny that.
It was bad enough when the often-quoted Institute of Medicine figure that 98,000 deaths per year in the US are caused by medical errors was in vogue, but now a paper in the Journal of Patient Safety states that adverse medical events result in 210,000 to 400,000 deaths per year and 10 to 20 times those numbers of serious harms.
Since the paper disparages the medical profession, it has received a lot of media attention.
Most articles about it simply regurgitate the dismal estimates without any real attempt to dig into the paper’s methods.
Let’s take a closer look.
As is true of many papers, the abstract is a bit sketchy when describing how the paper arrived at its conclusion.
The full text of the paper reveals the author found four studies that looked at what are described as preventable adverse events in US hospitals within the last seven years. All four used the Global Trigger Tool which involves the screening of records for adverse events by nurses or pharmacists and a secondary review by physicians.
Based on opinions by “experts,” the author made a key, but erroneous, assumption that all adverse events are preventable.
Read the full blog here.
Read More & Comment...
- House Speaker Nancy Pelosi in February 2010
USA Today reports:
Hospitals, a reliable source of employment growth in the recession and its aftermath, are starting to cut thousands of jobs amid falling insurance payments and inpatient visits.
The payroll cuts are surprising because the Affordable Care Act (ACA), whose implementation took a big step forward this month, is eventually expected to provide health coverage to as many as 30 million additional Americans.
"While the rest of the U.S. economy is stabilizing or improving, health care is entering into a recession," says John Howser, assistant vice chancellor of Vanderbilt University Medical Center.
Health care providers announced more layoffs than any other industry last month — 8,128 — largely because of reductions by hospitals, according to outplacement firm Challenger Gray and Christmas. So far this year, the health care sector has announced 41,085 layoffs, the third-most behind financial and industrial companies.
Read the full article here.
Read More & Comment...
Ice T?
Testosterone gels – do they provide an important treatment option? Is “Low T” a real disease or just a marketing opportunity? Do these two options need to be mutually exclusive? (The F.D.A. has approved testosterone gels “for use in men who either no longer produce the male sex hormone testosterone or produce it in very low amounts.”)
And what does this have to do with asthma inhalers and DTC?
Interesting article in today’s New York Times (see here).
One error that needs immediate correction. After reporting on the amount of money companies such as Eli Lilly and AbbVie spend on advertising their testosterone gel products, the Gray Lady reports that,
In response to an article Sunday in The New York Times on prescription drug costs for asthma medicines in the United States, a number of readers complained about the high price of inhalers, and that the costs were inflated by the millions of dollars pharmaceutical companies spend on advertising for them.
Except that assumption is not true. Whether or not you agree with the style or substance of pharmaceutical direct-to-consumer advertising, one thing that’s just an economic fact is that advertising budgets do not impact the list price of the product.
Study after study after study done by charitable foundations, the federal government and industry all show that the price of a drug does not correlate to the amount spent on advertising.
In other words, if you look at four medicines that treat cholesterol and compare their advertising budgets, the one that spends the most is not necessarily the most expensive -- and often the reverse is true.
Consider this: According to Tufts University it costs about one billion dollars to bring a new medicine to market. That's one billion per drug -- and those are the ones that make it to market.
Now compare that to the $2.5 billion per year the industry spends on advertising and you have a somewhat better perspective. Reducing direct-to-consumer drug advertising would not reduce the price of drugs.
A reduction in advertising wouldn't reduce the price of medicines, but it would most certainly reduce the number of people visiting their doctors, and I cannot imagine that anyone thinks that is a good thing.
Here's some data from an FDA research study: Doctors do not prescribe medicines just because their patients ask for them after seeing an advertisement. They're prescribing medicines because their patients need them. That's called progress.
Read More & Comment...An excellent article from BioWorld on the continuing saga on biosimilars from Sacramento to Singapore.
Some snippets …
The U.S. Battle of Biosimilars Continues on Multiple Fronts
By Mari Serebrov, Washington Editor
While the first biosimilars to hit the U.S. are still in development, the battle over how and when the follow-on biologics should be marketed continues to rage on both the local and global fronts.
A skirmish in California ended Saturday when Gov. Jerry Brown vetoed a bill, overwhelmingly passed by state lawmakers, that would limit automatic biosimilar substitution to those the FDA deems “interchangeable.” SB 598 also would have required pharmacists to notify physicians when an interchangeable is substituted for a prescribed biologic.
“Obviously, the governor gave himself a little wiggle room here,” Peter Pitts, president of the Center for Medicine in the Public Interest, told BioWorld Today.
Pitts disagreed with the governor about the timing of the bill. “It’s never too early to say we need to educate doctors and patients about what’s going on,” he said, stressing the importance of promoting safety and pharmacovigilance in the use of biosimilars.
“The untrained ear hears ‘identical’” when biosimilars are mentioned, Pitts said, but the follow-ons are not the same as generic drugs. Noting that even generics, which are supposed to be identical to the reference drug, can have variants that could affect an individual patient’s response, he pointed out that due to their complexity, biosimilars could produce more variances.
Global Battle
While the substitution battle continues on the state level, another biosimilar war is raging on the global front as the countries negotiating the Trans-Pacific Partnership (TPP) push to finalize the trade talks this year. Despite efforts to have the agreement reflect the 12 years of data protection that biologic innovators enjoy in the U.S., the issue hasn’t made it to the 3-year-old negotiating table yet.
Opposition to including the data protection in the TPP is framed, once again, as an issue of increased access. But it really comes down to some of the partners wanting to maximize their own opportunities, Pitts said.
He noted that the 100 most essential drugs identified by the World Health Organization are all off patent, which means they’re available as generics. But people in many countries still don’t have access to those essential small-molecule drugs, so Pitts questioned the assumption that reducing or removing biologic data exclusivity would increase global access to biosimilars, which are more complex and costly than generics.
The complete article can be found here.
Read More & Comment...California Governor Jerry brown has vetoed SB 598, a bill (passed by both houses of the legislature with overwhelming bipartisan support) that would have allowed biosimilars to be substituted by pharmacists if the F.D.A. deemed the biosimilar “interchangeable” with the reference product.
The California bill would allow substitution of a biosimilar for an innovator product only if FDA declared the biosimilar interchangeable for the specific use; the prescriber had not expressly prohibited use of a biosimilar; the substitution was communicated to patients; the cost to the patient was the same or less than the innovator product; and the pharmacist notified the prescribing physician within five days. The requirement for physician notification would sunset after three years Additionally, the California State Board of Pharmacy would maintain a list of biosimilar products FDA determines to be interchangeable on its website
Patient information? Pharmacist empowerment? Who could be against such a significant public health double play?
According to Governor Brown,
“Doctors with whom I have spoken would welcome this information. CALPERS and other large purchasers warn that the requirement itself would cast doubt on the safety and desirability of more cost-effective alternatives to biologics.”
(CALPERS is the California Public Employees’ Retirement System, which provides health benefits to more than 1.3 million people.)
“Case doubt on the safety …”
Perhaps the Governor should be made aware of what bioequivalence really means.
Per a report in the New York Times, “Ralph G. Neas, president of the Generic Pharmaceutical Association, celebrated the veto, saying that Mr. Brown had “demonstrated compassion for millions of patients and strong fiscal stewardship for the state of California.”
Nothing like celebrating the victory of cost-centric medicine over patient-centric care.
For shame.
Read More & Comment...The Washington Post reports that Senator Joe Manchin (D/WV),
... is calling for an investigation of “pay to play” allegations regarding a scientific panel that shaped the Food and Drug Administration’s thinking on painkillers and that, according to the organizer’s e-mails, was funded by major pharmaceutical companies that put up as much $25,000 to attend a meeting.
FDA responds that the sessions,
… amounted to what one agency officials described as “an essential collaborative effort.”
And, yes -- collaboration is essential. FDA must be both regulator and colleague. But should money have been charged (by a private, for-profit organization) for industry to have a seat at the table? That’s a good question.
It’s also important to clarify that these were not (repeat – not) advisory committee meetings.
Read More & Comment...Sent: Thursday, October 10, 2013 02:33 PM
To: FDA-CDER-wide
Subject: Retirement Rumors
CDER Staff:
I want to assure you that I am not planning to retire as erroneously reported in the media today. In fact, quite the opposite is true. I am becoming more deeply involved in many of the Center’s issues, including the proposed reorganizations of the Office of Pharmaceutical Quality and the Office of Generic Drugs.
The inaccuracy of the media has unnecessarily raised concerns among Center staff, and even among my own family. My daughter emailed me this morning to ask if I’m retiring! I continue to be fully committed to the important work CDER does and to its staff who work so diligently to protect the health of the American public.
Janet Woodcock Read More & Comment...
In July 2013, the Oncology Nursing Society (ONS) held its annual Leadership Weekend. Each year, the organization collects information about oncology nurses, their interests, and their needs. This year, they asked about their membership’s experiences helping patients manage adherence to oral therapies and about their interest in non-CNE training on the topic. The results of the survey, completed by 127 nurses, show that:
• Oncology nurses are experiencing patient non-adherence to oral therapies
• Oncology nurses want and need specific clinical practice assistance to help improve adherence
• Oncology nurses want and will attend educational programs on adherence to oral therapies.
Some findings of interest and importance:
Most Nurses Observe Problems with Adherence
There is evidence that most nurses are observing problems with adherence. Forty-one respondents (32%) said that 25%–50% of their patients have trouble, and another 26 (20%) said that 51%–75% of their patients do. In other words, more than half the respondents said that at least 25% of their patients do not adhere properly to their regimens.
Nurses Highlight Most Common Barriers to Adherence
The nurses ranked various potential causes of non-adherence by their importance to their patients. Cost was viewed as very important by 73 (57%) and important by 31 (24%). Thus, of the 127 total respondents, 104 find that cost, including reimbursement issues and high copays, is a significant barrier to adherence to oral therapies.
Another barrier appears to be side-effect management. Fifty-nine percent said this is an important problem (45 [35%]) or very important (31 [24%]). Cognitive difficulties following instructions are another potential barrier, but perhaps less significant. Although forty-eight (38%) ranked cognitive difficulties as 3 on a 1-5 scale, or of modest importance, another 51 (40%) viewed cognitive difficulties as important or very important in interfering with adherence to oral therapies. Difficulty swallowing seemed to be the least significant barrier, with 55 (43%) suggesting that it was not important or of minimal importance. Twenty-eight (22%) said it was mildly important, and 41 said it was important or very important. Thus, although most of the nurses did not view swallowing difficulty as a critical barrier, it does affect a fair number of patients.
What Do Nurses Need to Help Manage Adherence?
The participating nurses were offered a number of options for possible practical tools or specific aid. They are listed below in order of popularity with the respondents.
• Co-pay and reimbursement assistance was the most positively received form of help with adherence to oral therapies. Here, 119 (94%) of the respondents said they would find the help useful or very useful (70%).
• Side-effect management guides/tear sheets were seen as useful or very useful by another 116 nurses (91%).
• Patient journals/calendars were seen to be useful or very useful by 103 (81%) of the respondents.
• Online resources for both nurses and patients were very positively received, with 100 (79%) respondents viewing them as useful or very useful.
• Patient refrigerator magnets with significant information were also popular, with 72 (57%) indicating they would be useful or very useful.
• Telephone or email follow-up services by drug company or pharmacy were viewed as useful or very useful by 73 nurses (58%).
• Tablet or smartphone apps might be an emerging area of opportunity for drug companies or pharmacies. Fifty-seven (45%) thought they would be useful or very useful.
It’s interesting that the current outreach efforts by pharmaceutical companies and pharmacies rank only one percentage point higher than refrigerator magnets. They’re both 20th century technologies.
What’s also important is that the research differentiated between “online resources” and apps. While both are 21st century technologies, “online” is the substrate -- apps are the wave of the present.
Apps remind, cajole, educate, praise, incentivize, and assist patients in their quest for better health. Apps are at the nexus of safe use, treatment outcomes, and patient satisfaction. And it’s not science fiction.
At present, there are some 17,828 healthcare and fitness apps and 14,558 that can be deemed “medical.” While some are better than others, these numbers tell us one thing – this is not a fad or a trend. It is reality.
I have been working with the Mobile Health Library to help develop their safe use and outcomes support platform. It’s delivered as an app (viable on all tablet and smart phone systems) specifically to address many of the issues called out specifically by the ONS study – facilitating co-pay cards, patient diary functionality, side-effect management, direct physician office communications (via phone, e-mail, and text) and, perhaps most importantly, providing educational services via app-enabled printed materials, website links, and video).
Will our socio-economic “technology gap” lead to a more pronounced “adherence/compliance gap?” It’s an important question. That’s why it’s crucial we remember there is no one-size-fits all solution. But that mustn’t mean we disregard the reality of the growth and pervasiveness of apps, mobile apps. Let’s face it, when it comes to mobile phones, any gap is rather narrow – and it’s getting narrower all the time, literally every day.
The ONS research refers to apps as an “emerging opportunity.” And that’s true. But maybe a better way to phrase it is as an “emergent opportunity.”
Apps are here now. People are using them – more every day, and that includes healthcare providers, patients, and care-givers.
And as Philip K. Dick wrote, “Reality is that which, when you stop believing in it, doesn’t go away.”
Read More & Comment...The only thing that dies harder than a bad idea is a bad idea with political resonance.
The Wall Street Journal reports:
AUGUSTA, Maine—The hunt for cheaper prescription drugs long has led consumers to reach beyond U.S. borders, but under a Maine law set to take effect Wednesday, their search now will have the state's blessing.
The law, the first of its kind, sanctions the direct purchase of mail-order drugs from some foreign pharmacies. It has ignited a court battle with the pharmaceutical industry and set the stage for a broader fight over access to less-costly medication.
Vendor of choice – CanaRx.
This is particularly appalling since the drugs being sent to U.S. customers from CanaRX are most certainly not “the same drugs Canadians get.” That bit of rhetoric is just plain wrong. CanaRX – by their own admission – sources their drugs from the European Union. And while they may say their drugs come from the United Kingdom, let’s not conveniently forget that 20% of all the medicines sold in the UK are parallel imported from other nations in the EU – like Spain, Greece, Portugal, and Lithuania.
PS/ The drugs CanaRX sells to Americans aren’t even legal for sale in Canada.
Once again -- a brief primer on why drug importation is a bad idea
(1) It doesn’t save money.
(2) The drugs being sent to U.S. customers from Canadian Internet pharmacies are not “the same drugs Canadians get.”
(3) The state experience has been dismal and politically embarrassing.
(4) National Security concerns.
Let’s look at each of these four items.
(1) It won’t save any money. Let’s not forget the non-partisan CBO study that showed that such policy would reduce our nation’s spending on prescription medicines a whopping 0.1% -- and that’s not including the millions of dollars the FDA would need to set up a monitoring system.
(2) The drugs being sent to U.S. customers from Canadian internet pharmacies are not “the same drugs Canadians get.” That bit of rhetoric is just plain wrong. In fact, drugs sold to Americans by Canadian Internet pharmacies aren’t even legal for sale in Canada. This isn’t about the quality of Canadian drug regulation. Canadian Internet pharmacies – by their own admission – are sourcing the drugs they're sending to the United States from outside of Canada. And while they may say their drugs come from Great Britain, let’s not conveniently forget that 20% of all the medicines sold in the UK are parallel imported from other nations in the EU – like Spain, Greece, Portugal, and Lithuania.
And the important political point here is that when Americans are asked if they want drugs from nations other than Canada – the answer is a resounding “no thank you.”
(3) The state experience has been dismal and politically embarrassing. Remember Illinois’ high profile “I-Save-RX”program? Over 19 months of operation, a grand total of 3,689 Illinois residents used the program -- which equals approximately .02% of the population.
And what of Minnesota’s RxConnect program? According to its latest statistics, Minnesota RxConnect fills about 138 prescriptions a month. That's for the whole state. Minnesota population: 5,167,101.
That's not a surprise considering that Minnesota, state officials observed Canadian Internet pharmacies engaging in dangerous practices.
One pharmacy had its pharmacists check 100 new prescriptions or 300 refill prescriptions per hour, a volume so high that there is no way to assure safety.
One pharmacy failed to label its products and several others failed to send any patient drug information to patients receiving prescription drugs.
Drugs requiring refrigeration were being shipped un-refrigerated with no evidence that the products would remain stable.
One pharmacy had no policy in place for drug recalls. Representatives of the pharmacy allegedly said that the patient could contact the pharmacy about a recall "if they wished."
The FDA launched an investigation, confiscating thousands of drug shipments headed for the United States. Some of them were headed for Minnesotans who ordered them over the state's Web site.
When opened, nearly half claimed to be of Canadian origin, but "85 percent of them were from 27 other countries including Iran, Ecuador and China." And 30 of them were counterfeit.
One Minnesota resident discovered that one of his "Canadian" drugs came from Greece, and another came from Vanuatu, a small island in the South Pacific. "I never heard of the place," he said.
Wisconsin also has an importation program, modeled on the one in Minnesota. It too hawks its promise and hides its dangers. All of the legalese buries the fact that the state doesn't accept any responsibility for the safety or effectiveness of any medicines bought on the state's Web site.
The state won't even guarantee that the drugs ordered are what the customer will receive. Not only that, but the state also says that it will not accept any legal responsibility or liability should any of the drugs cause a problem.
And remember Springfield, MA and “the New Boston Tea Party?” Well the city of Springfield is now out of the drugs from Canada business.
(4) National Security concerns. According to a report from the federal Joint Terrorism Task Force, a global terrorist ring with ties to Hezbollah, is importing counterfeit drugs into America by way of Canada. They are doing so for profit today - but could just as easily do so for more nefarious and deadly purposes. And legalizing importation would only facilitate such actions.
And then there are those politically pesky safety issues.
Adding fuel to the reality is a new by the European Alliance for Access to Safe Medicines. The title says it all, “The Counterfeiting Superhighway.”
The report reveals the scope of the unregulated trade of fake pharmaceuticals. Through extensive research and examination of over 100 online pharmacies and over 30 commonly purchased prescription-only medicines, the report makes one thing very clear – we’re not winning the battle.
Key findings from this report
* 62% of medicines purchased online are fake or substandard (including medicines indicated to treat serious conditions such as cardiovascular and respiratory disease, neurological disorders, and mental health conditions).
* 95.6% of online pharmacies researched are operating illegally.
* 94% of websites do not have a named, verifiable pharmacist.
* Over 90% of websites supply prescription-only medicines without a prescription.
* 78.8 of websites violate intellectual property.
My favorite anecdote is the report’s example of an Internet pharmacy whose products came wrapped in pages from the Mumbai Daily News. The most frightening fact, though, is most of the fake medicines “were delivered in seemingly authentic boxes, accompanied by patient information leaflets in good condition and ostensibly trustworthy blister packs.”
"Those who cannot learn from history are doomed to repeat it."
Read More & Comment...Another way the Republic of South Africa is trying to reinvent itself is in the way it regulates and reimburses for medicines. “BE” means not only “Black Empowerment” (in the post-apartheid sense) but also “bioequivalence.”
I’ve just returned from the “New Developments in Drug Regulation” conference in Pretoria, and there was a lot of serious discussion as to how government regulators can do a better job in a uniquely South African situation.
Some memorable moments …
Tomas Salmonson (Chair of the EMA’s CHMP) told the audience that, while he is a strong believer in transparency, he is not in favor of patient representatives on decision panels. Such representation, he said, “would be like having an elected parliament and then additional members.” He also does not believe that these meetings (akin in many ways to an FDA advisory committee meeting) should be open to the public. I pointed out that having patient reps and open adcomm meetings was a crucial part of the FDA process. Salmonson commented, “I know.”
Transparency translates in different ways. Salmonson noted that one way the EMA promulgates transparency is through its EPARS (European Public Assessment Reports System). The European Medicines Agency publishes an EPAR for every medicine granted a central marketing authorization by the European Commission. EPARs are full scientific assessment reports of medicines authorized at a European Union level.
Salmonson also made the point that a transparent process behind benefit/risk allows consumers and healthcare providers to trust the regulator. FDA – attention must be paid.
Peter Bachmann, head of the Germany’s BfArM (Federal Institute for Drugs and Medical Devices) Coordination Group Unit, spoke to the EMA’s philosophy of being “united in diversity” (a theme that certainly resonated in Pretoria). He also discussed the EMA’s program of mutual recognition, detailing the agency’s methodology of “work-sharing” – a process of cross-national regulatory peer review that should be studied by FDA policy panjandrums for many reasons, not the least of which is quality control.
Ngokoana Khomo, Vice Chair of South Africa’s MCC (Medicines Control Council – the South African version of the FDA) honestly spoke of the tension between “the policy and the practice” of medicines regulation in South Africa. Unspoken was the obvious under-current of a third “p” – politics. And perhaps a fourth – “Potemkin Regulation.” She spoke of the MCC’s mission as “access, equity, efficiency, quality, and sustainability.” Dr. Khomo wisely said that “transparency takes away all suspicions.” But saying and doing is not always the same thing. She also spoke about the rational use of medicine. After all, you can’t spell Ngokoana without NGO.
Ekkehard Baader (Senior Director, Head of EU Regulatory Affairs at Teva) addressed the importance of (and distinctions between) consultation, communications, and cooperation.
Speaking of Teva – there was no discussion of generic drugs during the conference. Not a single mention of API sourcing and quality, not a word about excipients. Disturbing considering the venue and the reality.
BfArM’s Birka Lehmann (a member of PDCO, the EMA’s Paediatric Committee) spoke about issues surrounding informed consent for children (“What if the baby is crying?”) as well as the need for a Paediatric Investigation Plan (PIP) at end of phase 1. Clearly an area ripe for FDA harmonization conversations. Clearly the pediatric train is moving – but EMA and FDA should be on the same track.
At this point you may be wondering what any of this has to do with regulatory reform in the Republic of South Africa. That question was also on the minds of many in the audience who asked questions like, “how much does all of this cost?” and “how many people do you need?” and “how do you train staff?”
Day 2 began with a focus on biosimilars and the successes and failures of the EMA experience – as well as a nod to the work being done by the WHO.
The presentation by Chris Holloway (Group Director of Regulatory Affairs & Chief Scientific Officer at the ERA Consulting Group) was the first person to use the word “quality” – and “process.” He spoke about “balancing needs against expectations.” And it was a welcome addition to the conversation. A breath of real-world reality vs. regulatory theory.
Elwyn Griffiths (WHO) likened his organization’s attempts to develop criteria for biosimilars to the League of Nations – but in a good way.
Haile Selassie, call your office.
Max Wegner (VP, Head of Global Regulatory Affairs, General Medicine at Bayer AG) addressed the unintended consequences of the EMA’s PRAC (Pharmacovigilance Risk Assessment Committee), specifically the impact of public reports of adverse events that had yet to be investigated. I added that the FDA had similar issues with Early Safety Signal Communications and the resultant unintended (but not entirely unsurprising) over reaction by the media and the ensuing leap in non-adherence.
Finally, Marc Blockman of the host Medicines Control Council (MCC) spoke on the issues surrounding both the importance of and difficulties with pharmacoviginance in South Africa. Two of his comments stand out: “Pharmacovigilance is a public service” Bravo. And, “Yes, we have a yellow card – but the form is as much the problem as it is the process.” Sounds familiar.
The conference was, as Pierre Abélard might have put it, a “sic et non” experience. Lots of good advice – but not a lot of applicable next steps. What makes this all the more urgent is the South African position on medicines reimbursement and intellectual property – which shares many philosophical underpinnings with that other bastion of IP protection – India.
Just as there are issues relative to South Africa’s need for regulatory capacity building, so too is capacity building and investment important for the future of intellectual property issues. One frightening passage in South Africa’s proposal states, “The Draft Policy proposes adopting strict patenting rules to “exclude diagnostic, therapeutic and surgical methods from patentability, including new uses of known products, as is the case under the TRIPS Agreement.” Not a good start for a nation that wants to be the “s” added to BRICS.
Further, South African law currently provides no regulatory data protection and the Draft Policy does not include any. Moreover, it explicitly rejects the utility of “blanket data protection” for innovator data and emphasizes the importance of “access to knowledge.” Uh oh. And, of course, there’s a magic rain dance call for compulsory licensing.
The Draft Policy encourages the use of parallel importation to improve access to medicines. Been there. Done that. A clear and present risk to patients.
And these are only a few examples. It gets worse.
The policy disconnect is profound. In April, South Africa sent a sizable delegation to the mega BIO convention with the message that they were “open for business,” and aspires to be a player in global life sciences.
At the same time, local generics manufacturers and the usual suspect activists are whipping up frenzy over intellectual property issues. The same old song. And a dangerous one considering that Scientific American ranks South Africa in the bottom 20% of its life science/innovation index.
(PS/ Under the current regulatory regime, it takes the MCC between 3-5 years after EMA or FDA approval t bring innovative therapeutics to South African patients – the slowest among “advanced” MEA nations. What’s wrong with this picture?)
Rather than looking to emulate the EMA, perhaps South Africa should adopt a reference basket of maybe five to six countries. A good model is Singapore. Everyone’s favorite city-state reviews seven countries—USA, Canada, Australia, NZ, Japan, Switzerland and the EMA. If any two of the seven have approved, then approval is basically a formality.
Talk is cheap.
There is nothing like returning to a place that remains unchanged to find the ways in which you yourself have altered. – Nelson Mandela
Read More & Comment...
At CMPI’s recent Capital Hill conference ,Personalized Medicine and Responsible Access to Pain Medication, Dr. Charles Inturrisi, professor of pharmacology at the Weill Cornell Medical College, laid down the gauntlet:
I want to make a distinction that really does make a difference. And this is the distinction between efficacy and effectiveness. We know that opioids can provide analgesia for some chronic pain patients. We don’t know what percentage but we know that some, and you’ve heard from them this morning, at least one of them. But we also know the treatment outcomes with opioids are variable and not predictable. And this is the take home message if you have to leave. At present, there are no well-validated means of identifying optimal candidates for effective long-term chronic opioid therapy. That’s the problem. That’s the gap in our knowledge. That’s the gap in our evidence base.
We need to learn who will experience good analgesic effectiveness at stable dosages with limited side effects and low risk of abuse. So the critical question there is are there phenotypic or endo-genotypic characteristics that we can associate with better or worse outcomes that will help us to predict which patients might benefit and so that the cost-benefit ratio will be favorable rather than unfavorable.
Now I’m going to talk about personalized medicine in general and in particular. This refers to this emerging concept approach that uses patient-related factors including the phenotype, that is what information you can observe about the patient and a lot of that information now is contained in the electronic medical record. Also genotypic information that you can gain by collecting a sample and it can be either a sample of blood, or in some cases even a sample of saliva and by going through and looking at snips of DNA and creating biomarkers that select optimum medication and dosage for individual patients. It’s been estimated, on average, that prescription drugs are effective for only about half of those who take them. And for some drugs like anticancer drugs and antidepressants, the so-called non-responder rate is even higher.
Personalized medicine can reduce the non-responder rate because you can focus in on individuals who are highly associated with being respondersand you can eliminate the trial and error inefficiencies that inflate healthcare cost.
An audio recording on Dr. Inturrisi’s full comments can be found here (at the 1:03 mark) and the panel discussion that followed here.
On Friday CMS issued its final decision on beta amyloid imaging. It’s similar to their draft decision in that they ignored the recommendations of the medical community that was calling for full coverage for the Appropriate Use Criteria (AUC) population and imposed Coverage with Evidence Development (CED). CMS will only pay for Alzheimer's imaging tests used in clinical research or to exclude Alzheimer's disease when diagnosing patients in narrow circumstances. There are some slight changes from the initial draft, but from a patient access perspective, we are in the same boat as we were previously, which is non-coverage.
The Alzheimer's Association noted that, in the past, it has taken as long as seven years for CMS to move from a CED designation for new medical technologies to full coverage.
CMS’ move is just the latest example of cost-based thinking trumping patient-centric care
And, as per a recent article in BioCentury, there are significant unintended consequences that will impact the future of personalized medicine.
The cost of demonstrating clinical utility, along with the lack of clear or consistent standards, is killing a business model that had made it possible for small companies to commercialize molecular diagnostics quickly and cheaply. The fate of these laboratory-developed molecular diagnostics companies, and especially the conclusions investors draw about the viability of the space, could shape the future of personalized medicine.
According to research by the Tufts Center for the Study of Drug Development, without clinically useful diagnostics, personalized medicine growth will occur at a relatively slow pace.
And personalized medicine represents the future of healthcare around the world.
Let’s cut to the chase, if we are going to take meaningful strides both in addressing Alzheimer’s Disease specifically and in personalized medicine more broadly, we should not rely on Coverage with Evidence Development (CED) criteria in cases where the FDA’s approval process has expressly evaluated and endorsed the use of a drug or biologic in a specific patient population.
Fact: The evidence on amyloid imaging supports coverage for the population as identified by the Amyloid Imaging Task Force through Appropriate Use Criteria (AUC). A task force, convened by the Alzheimer’s Association and the Society of Nuclear Medicine and Molecular Imaging, recommends coverage in this population based on a comprehensive review of the literature and expert consensus.
Fact: CMS currently covers similar PET technologies to aid in the diagnosis of Alzheimer’s Disease and other forms of cognitive decline. The agency has not previously required evidence of health outcome improvement as a condition of such coverage.
Fact: Using CED alone will deny Medicare beneficiaries adequate and rapid access to this technology, as the path to implementation is unclear. Such uncertainty in the reimbursement process strongly dis-incentivizes future investments in research and development. And without innovation there will not be advances in personalized medicine.
Wither “sustainable innovation?”
Why even bother with expedited review and similar FDA pathways? Clearly closer FDA/CMS coordination is required to address both the will of Congress – and the future of American healthcare.
In the absence of either an FY 2014 appropriation or a Continuing Resolution for FDA, beginning on October 1 and continuing until the date of enactment of an FY 2014 appropriation or Continuing Resolution ("lapse period"), agency operations will be limited to the following:
- Emergency work involving the safety of human life or the protection of property;
- Criminal law enforcement work; and
- Activities funded by carryover user fee balances, including user fee balances under the Prescription Drug User Fee Act (PDUFA), Generic Drug User Fee Amendments (GDUFA), Medical Device User Fee Amendments (MDUFA), Animal Drug User Fee Act (ADUFA), Animal Generic Drug User Fee Act (AGDUFA), and Family Smoking Prevention and Tobacco Control Act. Carryover user fee balances will only be spent on activities for which the fees are authorized under the Federal Food, Drug, and Cosmetic Act (FD&C Act).
With respect to medical product user fees, during the lapse period, FDA will not have legal authority to accept user fees assessed for FY 2014 until an FY 2014 appropriation for FDA is enacted. This will mean that FDA will not be able to accept any regulatory submissions for FY 2014 that require a fee payment and that are submitted during the lapse period.
Read More & Comment...If you’re following the multifaceted and global debate over biosimilars, Steve Usdin’s article, Biosimilar battlefronts, is a must read. Authored by BioCentury's Steve Usdin, it’s opening paragraphs set the tone,
Biosimilars developers, manufacturers of originator biologics, payers and consumer groups are battling in state capitals, at FDA headquarters, and at the World Health Organization in Geneva over the rules that will shape perceptions and prescribing practices for copycat biologics in the U.S. and around the world.
Separately, none of the conflicts will have a decisive effect on either biosimilars developers or manufacturers of the products they hope to replace. But collectively, state laws on interchangeable biosimilar substitution, national and international naming practices, and FDA's labeling policies could have powerful effects on the market penetration of biosimilars.
The article covers many issues, both foreign and domestic – and one of the most important and most contentious is the issue of the nonproprietary names assigned to biosimilar and interchangeable biologics.
Usdin writes,
Proponents of distinctive non-proprietary names, including some of the biggest biotech companies, say patient safety and possibly the viability of whole classes of biologic drugs would be threatened by a failure to adopt their recommendations.
However, European regulators say distinct non-proprietary names are unnecessary, and some biosimilars companies argue such a naming scheme would put a dark cloud over biosimilar products that would substantially reduce sales.
A WHO group will meet on Oct. 22 to debate biosimilars naming principles.
The basic disagreement: one camp is arguing for biosimilars to keep the same international non-proprietary name (INN) as the reference product; a second camp argues for distinct INNs.
Supporters of distinct INNs say the ability of regulators to track and trace biologic drugs is essential for patient safety and the commercial viability of biologics — original and biosimilar. Opponents of distinct INNs say they are intended to confuse and scare physicians, patients and payers by drawing unnecessary distinctions between original biologics and biosimilars.
According to Geoffrey Eich, executive director of R&D policy at Amgen, “It seems counterintuitive, like sticking your head in the sand, to not want to include a distinguishing feature that allows us to aggregate info when appropriate and disaggregate when appropriate.”
Amgen supports the use of the reference product’s root INN, with the addition of a unique suffix or prefix for each biosimilar. “We believe having some kind of a distinguishable feature is absolutely essential to patient welfare, for pharmacovigilance,” Amgen’s Eich told BioCentury.
A WHO working group will meet Oct. 22-24 to discuss the creation of voluntary international standards for non-proprietary names for biosimilars.
WHO isn’t likely to adopt a final naming policy at the October meeting. Whatever policy it eventually adopts will not be binding, but it is likely to be adopted by developing countries, and, if it includes a distinctive naming system, could intensify pressure on European countries to adopt new naming policies.
Without casting any aspersions, “quality” and “pharmacovigilance” in developing countries takes on an entirely different meaning than similar concepts in more developed regulatory regimes.
At a meeting in Pretoria last week Tomas Salmonson, Chair of the EMA’s Committee for Medicinal Products for Human Use (CHMP), said that the working group is “split down the middle” on the issue of naming.
Stay tuned.
We do what we must, and call it by the best names. -- Ralph Waldo Emerson
Read More & Comment...BioCentury This Week is must see TV – especially if you work in the FDA’s government affairs office.
On this weekend’s program Representative Dr. Mike Burgess (R, TX) said that the OMB misinterpreted the law – and that user fees shouldn't have been sequestered.
Dr. Burgess’ comments can be found here.
Read More & Comment...Dr. Charles Inturrisi is Professor of Pharmacology at Weill Cornell Medical College. He also has appointments in the Neuroscience Program at WCMC and with the Pain and Palliative Care Service, of the Memorial Sloan-Kettering Cancer Center and with the Drug Abuse Center at The Rockefeller University.
Dr. Inturrisi's current research is measuring the long term outcomes of treatments for chronic cancer and noncancer pain received by patients at four hospital-based outpatient Pain Clinics. He continues to have an interest the role of glutamate receptors in injury-induced pain and opioid tolerance, dependence and addictive behaviors. His research is directed at the discovery of new treatments for pain and drug addiction.
Dr. Inturrisi has received the John J. Bonica award of the Eastern Pain Association, a Distinguished Alumnus award from the University of Connecticut and the Excellence in Mentoring Award, Weill Cornell Medical College Postdoctoral Association. In 2008, Dr. Inturrisi received the first Graduate Dean’s Award for Excellence in Teaching and Mentoring of Graduate Students, presented by the Weill Cornell Graduate School of Medical Sciences.
He served as the president of the American Pain Society from 2008 to 2010 and in 2013 received the Distinguished Service Award from APS.
He was a member of the Institute of Medicine Committee that prepared the 2011 Report entitled “Relieving Pain in America”.
You can listen to Dr. Inturrisi’s presentation here (at the 1:03 mark) and the panel discussion that followed here.
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Judy Foreman is a nationally syndicated health columnist whose “Health Sense” columns have appeared regularly in the Boston Globe, Los Angeles Times, Dallas Morning News and other national and international outlets. For years, she also wrote the Globe’s popular short feature, “Health Answers.”
She graduated Phi Beta Kappa from Wellesley College, served in the Peace Corps in Brazil for three years, then got a Master’s degree from the Harvard Graduate School of Education. From 2001 to 2004, she was a Lecturer on Medicine at Harvard Medical School and, for most of this time, was a scholar at the Brandeis Women’s Research Center. She has also been the host of a weekly, call-in radio show on Healthtalk.com and has won more than 50 journalism awards.
Her book on chronic pain, “A Nation in Pain – Healing Our Biggest Health Problem,” is due out in January, 2014 from Oxford University Press.
Steve Usdin has been Washington Editor of BioCentury since 1993, and has spent the past 20 years in the nation's capital covering political and policy issues affecting the life sciences sector. He also is the host of BioCentury This Week, BioCentury's weekly public affairs television program, as well as BioCentury Senior Editor responsible for coverage of social issues involving biotechnology. Steve’s reporting about biotechnology and biomedical policy has been cited in The Economist, The Wall Street Journal, the Washington Post, New Scientist and other publications. In 2012, the FDA Alumni Association named Steve the Harvey W. Wiley Lecturer, making him the first journalist to receive the Wiley Award. His book, “Engineering Communism: How Two Americans Spied for Stalin and Founded the Soviet Silicon Valley,” was published in 2005 by Yale University Press.
You can listen to Judy Foreman’s presentation and the panel discussion moderated by Peter Pitts here.
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India's Protectionist Prescription
At the recent Group of 20 Summit, Indian Prime Minister Manmohan Singh implored American policymakers to recommit to encouraging robust growth in India and other emerging markets. It was a surprising request from a leader who has spent the past year overseeing a barrage of damaging reforms that threaten the American economy — and India’s prosperity.
India has been systematically shutting out foreign goods in an effort to prop up domestic industries. Indian officials have recklessly ignored basic intellectual-property protections, unfairly bolstering their own businesses at the expense of improved public health. America’s leaders need to make clear that a fair and open relationship benefits both India and the United States. If India wants to remain a valued economic partner, this kind of crude protectionism won’t be tolerated.
In recent months, India’s policymakers have pulled out all the stops to give homegrown companies an unfair advantage. Since the beginning of last year, the government has raised customs duties on high-end cars from 75 percent to 100 percent. In April, leaders mandated that all cosmetic products be registered with the Indian government prior to marketing there — a bureaucratic hurdle designed to obstruct foreign firms. Yet another new regulation requires that procurements by the country’s military give priority to Indian defense firms.
All together, the country has enacted 33 potentially trade-restricting measures since October 2008, according to a recent report from the European Commission. Where India’s self-serving policies have been especially irresponsible, however, is in the area of intellectual property, particularly for cutting-edge pharmaceuticals.
The country’s blatant disregard for intellectual-property rights was made clear in April, when India’s Supreme Court denied patent protection for a new form of the cancer drug Gleevec. In doing so, the court gave India’s $22 billion domestic drug industry free rein to sell copycat versions of the treatment. Indian officials were quick to declare the decision a victory for their poorest citizens, applauding the court for helping to make sophisticated medicines more affordable.
This claim, however, entirely misrepresents the decision and its consequences. Officials strategically neglected to mention that 95 percent of Indians who rely on Gleevec already receive the drug free of charge, thanks to a program supported by the drug’s manufacturer, Novartis. The Gleevec case was never about improving pharmaceutical access; the real intent was always to benefit India’s drug manufacturers.
By unfairly boosting the country’s generic industry, the court announced to health care research firms around the world that India is no friend to cutting-edge treatments — and the repercussions of such a message could be severe.
A breakthrough drug such as Gleevec requires, on average, more than a decade of trial and error to create and costs firms more than $1 billion. If India isn’t willing to protect the basic intellectual-property protections companies need to earn back some of that investment, drug firms won’t be able to offer their products to Indian patients, much less give them away for free. Considering India is home to the largest population of people who lack access to essential drugs, Indian leaders can ill afford to shun foreign pharmaceutical companies and the advanced medicines they offer.
Additionally, there is the direct threat India’s policies pose to the American economy. U.S. exports to India totaled $33 billion in 2011, an increase of more than 12 percent from the year before. As Rep. John B. Larson, Connecticut Democrat, and Rep. Erik Paulsen, Minnesota Republican, noted in a recent letter to fellow lawmakers in Congress, no fewer than 75 U.S. industries depend on intellectual-property protections. All told, companies in these fields are responsible for roughly 40 million American jobs.
America’s technology, agriculture and information-technology industries — among other sectors — are already feeling the effects of India’s current policies. If India continues to erect trade barriers, more and more U.S. firms will be denied a valuable and growing market for their goods.
What’s more, if our leaders don’t reverse this protectionist shift, many other nations may follow suit. In fact, many already have: The European Commission found that between May 2012 and May 2013, 154 new trade-restricting measures were adopted around the world, while only 18 were lifted.
As Mr. Singh made clear at the G-20 summit, America continues to have considerable influence on India’s economic future. It’s time our leaders use this clout to pressure India’s government to abandon their destructive trade policies. Officials in Washington must demonstrate to India — and the world — that fair economic partnerships are mutually beneficial and that protectionist tactics won’t be tolerated.
Peter J. Pitts, a former associate commissioner at the Food and Drug Administration, is president of the Center for Medicine in the Public Interest.
Read More & Comment...Another biologics nomenclature Citizen Petition was filed before this (see www.biopharmacopeia.com). The GPhA petition concerns the issue of established/official nonproprietary names (useable for marketing and prescription filling), particularly, whether these should be unique or not (generic). The earlier petition was filed in June by the Biotechnology Information Institute (R. Rader). It requests FDA assign both unofficial unique and biosimilar/(bio)generic-like (or class) names (and/or other identifiers) upon biologics approvals, along with public disclosures of needed basic agent/product descriptive information, including regarding supplemental approvals (product drift). These names (and the nomenclature system) need to be coherent science/entity/product-based, i.e., totally new and with no connection or carry-over from INN/USAN, which as a legacy pre-recombinant nomenclature system is simply not workable with modern biologics.
Thank you.
Ronald A. Rader
President
Biotechnology Information Institute
Read More & Comment...
CMPI president Peter Pitts’ opening remarks at the conference can be found here.
In the next audio clips, Steve Usdin of BioCentury moderates a panel discussion addressing the role of patients and manufacturers as it concerns access to pain medications.
The panelists are listed below:
Cindy Steinberg, National Director of Policy and Advocacy, US Pain Foundation
Bob Twillman, Director of Policy and Advocacy, American Academy of Pain Management
Stuart Kim, Associate General Counsel, Regulatory Affairs, Mallinckrodt Pharmaceuticals
You can listen to Cindy Steinberg’s presentation and the panel discussion moderated by Steve Usdin here.
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