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My bags are packed. I’m ready to go.
As many of us head out to San Diego for BIO, it’s important to put the gathering in the proper perspective. It’s not just about business. It’s not just about networking. It’s about advancing the public health. It’s about protecting and advancing innovation.
Attached is a letter that Pfizer honcho Ian Read just sent to Representative Fred Upton. Mr. Upton, as you are likely aware, is the Chairman of the House Committee on Energy & Commerce and has been leading a series of hearings on 21st Century Cures.
Ian also attached a more complete thought piece on a series of innovation priorities. Innovation? Read all about.
While at BIO I’ll be participating in two panels.
The first: Will Government and Payor Policies Encourage Innovation and Create Incentives for the Continued Development of Abuse Deterrent Formulations and Improvements to Existing Technologies in the Regulatory Review and Approval Track?
Peter Pitts, Moderator, President and co-founder, Center for Medicine in the Public Interest
Douglas Throckmorton, MD, Deputy Director of Regulatory Programs, FDA’s Center for Drug Evaluation and Research
Richard C. Dart, MD, PhD, Director of the Rocky Mountain Poison and Drug Center
J. David Haddox, DDS, MD, Vice President of Health Policy for Purdue Pharma, L.P.
WHERE: San Diego Convention Center Room 31 B
WHEN: Tuesday, June 24 2014, 9:00 am –10:00 am
David Schull, president of Russo Partners
Penny Daniels, strategic communications consultant at 3D Communications
Craig Audet, Ph.D., senior vice president, operations and head of global regulatory affairs with Arena Pharmaceuticals, Inc.
Peter Pitts, president and co-founder of the Center for Medicine in the Public Interest and former FDA associate commissioner of external affairs
Silvia Taylor, senior vice president, investor relations, public relations, and corporate communications at Sucampo Pharmaceuticals, Inc.
WHERE: San Diego Convention Center Room 31B
WHEN: Tuesday, June 24 2014, 3:00PM – 4:00PM
In addition, the Center for Medicine in the Public Interest will have a booth. It’s #5642. And we’ll be talking about innovation.
Hope to see you there.Read More & Comment...
Urban legend attributes the following quote to Albert Shanker, the very outspoken past president of the American Federation of Teachers:
“When school children start paying union dues, that’s when I’ll start representing the interests of children.”
As always, it’s a question of priorities – and whose priorities. Today that same issue has arisen in the debate over biosimilar nomenclature.
The issue for drug compendia stakeholders (among other things) is interoperability.
According to Inside Health Policy:
“While some say an easy solution is to simply add a suffix to distinguish a biosimilar from the innovator product, the compendia group told FDA any change is a change, and regardless of the simplicity of the change, the associated coding would also have to shift. First data bank and two other databanks are sold to stakeholders who assemble them and put overlays on them and then the data are used for different purposes, including for reimbursement.”
Yes, well unfortunately change is not always easy. But as management guru W. Edwards Deming warned, “Change is not required. Survival is not mandatory.”
Assigning differential naming to biosimilar products will certainly prove challenging to the various constituents of the drug compendia community – but it will be crucial for pharmacovigilance. In the real world, how can we not have separate names when there are going to be four categories of products?
Based on comparative analytical data, FDA will characterize its assessment of biosimilarity into one of four levels -- not similar, similar, highly similar or highly similar with a fingerprint-like similarity -- depending on the type, nature and extent of any structural and functional differences revealed.
Additional pharmacologic studies would be required to show that the identified difference is "within an acceptable range to consider the proposed biosimilar product to be highly similar to the reference product." FDA said only products in the top two tiers would meet the statutory requirement for analytical similarity under the Biologics Price Competition and Innovation Act of 2009. Products in the top two tiers would then only require "targeted and selective animal and/or clinical studies to resolve residual uncertainties" to demonstrate biosimilarity. In addition, these data could be used to extrapolate clinical data for additional indications.
Giving credit where credit is due, the good people at the USP understand that interoperability is important, but that naming is more of a philosohpical issue. That's why they support differentiation via discrete numerical suffix.
Per Inside Health Policy:
The drug compendia stakeholders told a group of 13 FDA drug policy experts, including drug center chief Janet Woodcock, that changing the traditional naming process would require that each piece of the compendia process be individually rebuilt in order to ensure patient safety and restore functionality to the system. Doing so, while possible, would be difficult and could lead to confusion, errors and misunderstanding, creating a "very real risk to patients," according to slides presented at the May 2 meeting and a June 6 follow-up letter, obtained by Inside Health Policy.
The argument that differential naming is bad for patient safety is pure Orwellian Newspeak -- specifically “blackwhite,” The ability to accept whatever "truth" the party puts out, no matter how absurd it may be.
When it comes to biosimilar nomenclature, it’s urgent that we keep our priorities straight. And that means keeping patient safety, not interoperability challenges, at the top of the agenda.
Fortunately, White Oak trumps blackwhite.Read More & Comment...
Per FDASIA: By July 9, 2014, FDA must issue guidance describing FDA policy regarding Internet promotion, including social media, of medical products regulated by FDA.
Well, ahead of schedule we now have two draft guidances:
First let’s discuss “Social Media Platforms with Character Space Limitations.
Perhaps the biggest leap forward for the agency is that is beginning to differentiate between social media platforms, recognizing that different rules must apply for different types of platforms. Obviously, when it comes to platforms such as Twitter the main issue is character limitations. The agency also points out that the same is true for advertising venues such as banner ads on Google (shades of the FDA’s first batch of warning letters from 2009).
So, to the FDA’s credit, it only took the agency about five years to develop draft guidance. Not a badge of honor.
Still, there are some interesting things to be gleaned from the draft guidance. The agency writes:
Risk information should be comparable in content and prominence to benefit claims within the product promotion (i.e., a balanced presentation). Achieving a balanced presentation requires firms to carefully consider the desired benefit claims and risk profiles for their products when choosing a promotional platform.
That’s good advice – choose the platform that best suits any given communications need. But, it’s not really any communications need – it’s any promotional program. That’s important because promotional speech is regulated speech. And that’s not just regulatory rhetoric. And this begs a crucial question not covered in the draft guidance – What it the intent of the communications?
Don’t wait for the FDA to offer guidance on that. They will not and should not. That’s a question only the communicator can answer. And it needs to be done honestly and early in the communications development process.
The agency writes:
FDA acknowledges that Internet/social media platforms associated with character space limitations may pose challenges for firms in providing a balanced presentation of both risks and benefits of medical products, as discussed above … The firm should also provide a mechanism to allow direct access to a more complete discussion of the risks associated with its product.
In a likely unintended nod to self-deprecation, the agency offers potential examples of how this could be done for the fictitious product NoFocus.
The agency writes:
A firm is considering promotion of its prescription drug NoFocus on Twitter … NoFocus is indicated for mild to moderate memory loss.
The draft guidance suggests that the tweet contain the following 40 characters:
NoFocus for mild to moderate memory loss
The agency writes:
The benefit information for NoFocus that is communicated within the first 40 character spaces of this tweet is accurate and non-misleading and includesmaterial facts about the indication and limitations to the use of NoFocus.
So, the agency is also suggesting that these 40 characters be the first 40.
The agency then proceeds to discuss paid search advertising for the fictitious product Headhurtz. (This is likely the product in development for legal and regulatory review departments at pharmaceutical and medical device companies.)
The agency suggests that information on limitations and appropriate use as well as a link to the product website be included. Specifically:
Headhurtz [www.headhurtz.com] For severe headache from traumatic brain injury
It’s not Brief Summary, it’s Tweet Summary.
The agency’s most important advice appears on Page 12 of the draft guidance:
In communicating risk information on Internet/social media platforms with character space limitations, firms should consider the following points:
1. Risk information should be presented together with benefit information within each individual character-space-limited communication (e.g., each individual message or tweet).
2. The content of risk information presented within each individual character - space-limited communication should, at a minimum, include the most serious risks associated with the product.
3. A mechanism, such as a hyperlink, should also be provided within each individual character-space-limited communication to allow direct access to a more complete discussion of risk information about the product … the Agency recommends that a direct hyperlink to a landing page that is devoted exclusively to comprehensive risk information about the product be initially included within the original character-space-limited communication.
Not rocket science, but urgently important to remember when considering both the design and appropriateness of a limited-character platform. Also, the use of hyperlinks is crucial – and will be even more so as we move on to a discussion of the second of the agency’s newly minted draft guidances. (Note – the agency makes it clear that a link’s name should also be considered. Specifically, it shouldn’t be a claim (i.e., www.bestcancercuredrug.com).
Per the new “Tweet Summary,” the agency writes:
Since benefit information was provided within the tweet (example 1A), the firm should communicate, at a minimum, the most serious risks associated with NoFocus within the same tweet. The firm should also include within the same tweet a direct hyperlink to a more complete discussion of risk information about NoFocus. Additionally, the prominence of risk information should be comparable to the benefit information contained within the tweet, taking into consideration any formatting capabilities that may be available. The firm considers including the following benefit and risk information within the tweet:
NoFocus for mild to moderate memory loss; may cause seizures in patients with a seizure disorder www.nofocus.com/risk
And, as a further build:
… the firm should also communicate the brand and established names within the tweet. The firm considers including the following product information, together with benefit and risk information about NoFocus, within the tweet:
NoFocus (rememberine HCl) for mild to moderate memory loss-May cause seizures in patients with a seizure disorder www.nofocus.com/risk
As it turns out, the Tweet Summary takes up quite a bit of the available characters – and this is for a non-Black Box product. For those of you keeping count, it’s now up to 134 characters – leaving only six characters for Hi Mom.
Later on Pages 12-13 the agency spells out how verbiage and hyperlinks can be compliantly used for promotional speech such as sponsored Google links. Its well a well thought out and developed template. And it’s more restrictive – as it should be. After all, there are rules for advertising. Five years in the making. Headhurtz indeed!
Now let’s move on to a far more important public health issue: Correcting Independent Third-Party Misinformation About Prescription Drugs and Medical Devices.
For those of you not currently on NoFocus therapy, you will remember the 2009 Part 15 hearing on social media – the “SuperBowl of FDA Part 15 hearings.” One of the key questions the agency wanted to discuss was how to correct erroneous product information. All they got were blank stares and request for FDA guidance. Five years later – voila!
The agency writes:
This draft guidance is intended to describe FDA’s current thinking about how manufacturers, packers, and distributors (firms) of prescription human and animal drugs (drugs) and medical devices for human use (devices) should respond, if they choose to respond, to misinformation related to a firm’s own FDA-approved or -cleared products when that information is created or disseminated by independent third parties on the Internet or through social media or other technological venues (Internet/social media), regardless of whether that misinformation appears on a firm’s own forum or an independent third-party forum or website.
Key take-away is that the decision to correct errors is purely voluntary. (But, then again, technically so are all product recalls.) Where that becomes more complicated is how companies may choose to cherry-pick where they interceded for corrective purposes. This is not an issue addressed by the FDA in this draft guidance. One thing this all means is more work for legal and regulatory review.
This draft guidance is all about user-generated content. The agency writes:
The Internet and Internet-based technologies have made it easier for third parties who are independent of firms to disseminate information about drugs and devices. Information created by third parties (which for purposes of this guidance is user-generated content (UGC)) might appear on an interactive portion of a firm controlled website or other interactive Internet/social media platform, or information might appear on a website or other Internet/social media platform that is independent of, or not under the control or influence of, a firm.
Many Internet/social media platforms allow for real-time and continuous communications and interactions (e.g., blogs, microblogs, social networks, online communities, and live podcasts) while other platforms do not provide a means for interactive content to be posted. Whether a forum is interactive may affect the means by which a firm is able to respond to information.
And, the agency stipulates, “UGC might not always be accurate and may be dangerous or harmful to the public health.” Well, yes. What’s a manufacturer to do?
Five years later, some helpful advice from the good people at White Oak.
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.
If a firm chooses to respond to misinformation about its products using non-truthful or misleading information or in a manner other than that recommended in this draft guidance, however, FDA may object if the information provided by the firm does not comply with applicable regulatory requirements related to labeling or advertising, if any.
Certainly. But what this also harkens back to is the agency’s previous guidance where it warns against using company-generated (sponsored) UGC relative to other companies’ products. That 12/11 guidance warns:
If a firm chooses to respond to public unsolicited requests for off-label information, the firm should respond only when the request pertains specifically to its own named product (and is not solely about a competitor's product).
Forewarned is Forearmed. Don’t get to cute.
This draft guidance isn’t about sponsored communications. The agency writes:
This draft guidance does not apply when a firm is responsible for the product communication that contains misinformation. A firm is responsible for communications that are owned, controlled, created, or influenced, or affirmatively adopted or endorsed, by, or on behalf of, the firm.
A firm is thus responsible for communications on the Internet and Internet-based platforms, such as social media, made by its employees or any agents acting on behalf of the firm to promote the firm’s product, and these communications must comply with any applicable regulatory requirements.
… this draft guidance applies when a firm is not responsible for a product-related communication that appears on the firm’s own forum, an independent third- party website, or through social media, and the firm chooses to correct misinformation about its own product contained in that communication. In such cases, we recommend that the firm do so as described in this draft guidance.
More detail is provided here relative to the nuance of “control” and “sponsorship (pages 7-8) and are worth reading.
Here are the agency’s eight ground-rules for “appropriate corrective information;”
* Be relevant and responsive to the misinformation;
* Be limited and tailored to the misinformation;
* Be non-promotional in nature, tone, and presentation;
* Be accurate;
* Be consistent with the FDA-required labeling for the product;
* Be supported by sufficient evidence, including substantial evidence, when appropriate, for prescription drugs;
* Either be posted in conjunction with the misinformation in the same area or forum (if posted directly to the forum by the firm), or should reference the misinformation and be intended to be posted in conjunction with the misinformation (if provided to the forum operator or author);
* Disclose that the person providing the corrective information is affiliated with the firm that manufactures, packs, or distributes the product.
Truthful. Accurate. Transparent. Three good rules of thumb. But what about “timeliness?” No advice from the agency on this one – but something crucial for manufacturers to consider since the goal here is to protect the public health.
Again, the agency offers important nuance on how to correct. For example, don’t just correct one mistake when multiples ones appear. The draft guidance also offers absolutely critical commentary relative to the depth and breadth of responsibility when correcting one piece non-sponsored UGC, “The firm is not expected to correct misinformation that appears on other webpages of the website.”
Those sweet sixteen words address a world regulatory angst.
Once a firm undertakes the correction of misinformation, FDA does not expect the firm to continue to monitor the website or communication that previously included UGC containing misinformation.
This does, however, point to the need for individual companies to develop their own policies in this regard. More work for legal/regulatory review teams!
And, for those of you who are thinking a little too hard, consider this important caveat:
… if a firm chooses to correct only misinformation that portrays its product in a negative light in a third-party communication but does not address misinformation that overstates the benefits of its product in that same clearly defined portion of the communication, the firm’s actions do not meet the recommendations in this draft guidance.
Voluntary corrective action isn’t just about “bad” information.
FDA recognizes that a firm cannot control whether an independent third party refuses to correct the misinformation, or corrects only a portion of the misinformation even though the firm provided complete corrective information, or declines to include the respective required labeling, or declines to remove misinformation, or does not correct all the misinformation in one clearly defined part (if the firm sought to correct more than one piece of misinformation). Accordingly, FDA will not hold a firm accountable for an independent third party’s subsequent actions or lack thereof.
The perfect shall not impede the public good.
FDA does not expect firms to submit corrections to the Agency when correcting misinformation pursuant to this draft guidance; however, FDA recommends that firms keep records to assist in responding to questions that may come from the Agency. The records should include, for example, the content of the misinformation, where it appeared, the date it appeared or was located, the corrective information that was provided, and the date the corrective information was provided.
Good guidance has been provided. Now it’s time for industry to do the right thing.Read More & Comment...
U.S. Healthcare: Most Expensive and Worst Performing
In a new international ranking, the United Kingdom ranks first, while the U.S. performs poorly across almost all health metrics.
This study -- or rather a version of it conducted by the World Health Organization -- has been thoroughly shredded by Scott Atlas in his Commentary article: The Worst Study Ever?
As Atlas points out: "researchers divided aspects of health care into subjective categories and tailored the definitions to suit their political aims. They allowed fundamental flaws in methodology, large margins of error in data, and overt bias in data analysis, and then offered conclusions despite enormous gaps in the data they did have."
All to make the case for single payer health systems.
In this case, the Commonwealth Fund measures everything except the quality of care. It measures lots of process measures that are easily skewed by bias. So for instance, in measuring "efficiency" CW ranks countries based on percentage of GDP spent on health and percent of health care spent on administrative services. Of course the US is destined to be the worst.
Next, it ranks countries on access. Again the US is trashed. And the UK is number 1. Don't laugh. CW used "having access to basic medical care the same or next day." Apparently wait times for cancer care, hip replacements, by-pass surgeries, etc. don't count. Indeed, CW ignores the explosion in outpatient same day surgery and walk-in clinics in the US.
The US is also slammed for unfairness. CW claims -- surprise! -- that the UK does the best on providing the same great care to everyone rich and poor. Apparently, despite the exhaustive research CW did, the group failed to come across the Marmot report calling health inequality in the UK a ticking time bomb. Here's the link in case CW wants to update it's study:
Meanwhile, CW ignores the following (courtesy of Scott Atlas again!):
"American cancer patients, both men and women, have superior survival rates for all major cancers. Removing “lead-time bias,” where simply detecting cancer earlier might falsely demonstrate longer survival, death rates from prostate and breast cancer from the early 1980’s to 2005 declined much faster in the US than in the 15 other OECD nations studied (Australia, Austria, Canada, Finland, France, Germany, Greece, Italy, Japan, the Netherlands, Norway, Spain, Sweden, Switzerland, and UK)."
What about heart disease?
"A comparison of the US to ten Western European nations (Austria, Denmark, France, Germany, Greece, Italy, Netherlands, Spain, Sweden, and Switzerland) showed that 60.7 percent of Americans diagnosed with heart disease were actually receiving medication for it, while only 54.5 percent of Western Europeans were treated (a statistically significant difference)."
"Another comparison study showed that fewer Americans than UK residents die (per capita) from heart attack despite the far higher burden of risk factors in Americans for these fatal events. In fact, the heart disease mortality rate in England was 36 percent higher than that in the US. These superior outcomes from US medical care are particularly impressive, considering that American patients have far more risk factors (diabetes, obesity, chronic kidney disease) that worsen outcomes and death rates after heart attack and after heart surgery.
The US shows a far greater reduction in death rates from stroke, the third leading cause of death and the leading cause of disability in adults in the US and most Western European nations, than almost all Western European nations and the European Union overall."
So why did CW ignore outcomes like mortality and survival and focus on income inequality and how universal a health care system was.
You have to take into account CW's frame of reference. If someone told you time again that the VA health system is a model of excellence, would you take their study on health care performance seriously?
And boy does Commonwealth love the VA.
Here's CW president Stephen Shoenbaum gushing over the VA in an interview with Managed Care magazine:
MC What parts of the health care system are working?
SCHOENBAUM: There are some examples of excellence within the U.S. health system. If we pick any given area, such as quality or driving down costs, we find examples of excellence. Some large systems are doing very interesting things. Kaiser Permanente and the Veterans Administration are managed systems that are trying to improve performance and reduce costs by using a primary care-based system and using information technology creatively. Another example is Geisinger Health System in Pennsylvania.
And here's a CW study of the VA, which it claims is a high performing health system. I take that to mean it meets the standards CW set in the report dumping on US health care. The study celebrates how the Memphis and Lincoln, Nebraska VA systems shortened wait times, reduced emergecny room vistis and reduced blood sugar levels in some patients.
Hmm.. Memphis VA.. where have I heard that before..
VA Hospital Axed Veteran Programs While Approving $1 Million In Bonuses
The Memphis Veteran Administration (VA) Medical Center approved over $1 million in bonuses months before closing a therapeutic aquatic pool citing a lack of funds.
The Memphis VA Medical Center handed out $1,005,644 in bonuses for its approximately 2,000 employees in fiscal year 2010, according to data provided to The Daily Caller by Sandra Glover, the communications officer for Veteran Integrated Services Network (VISN) 9, which includes the Memphis VA Medical Center.."
Memphis Veterans Affairs Medical Center Flagged in Audit
The CW study is an exercise in confirmation bias, which is the " tendency to search for or interpret information in a way that confirms one's preconceptions, leading to statistical errors. Confirmation bias is a type of cognitive bias and represents an error of inductive inference toward confirmation of the hypothesis under study."
It was designed to reinforce the meme that goverment run health systems are better, fairer and cheaper. This blog was a modest effort to speak truth to bias.
Read More & Comment...
Here's a story from The Pharma Times on how NICE is searching for multiple ways to say "no". That will of course lead to delays in access, during which time people will die. Now that's a way of reducing the "drain on healthcare resources."
NICE asks for more data on Sovaldi
WORLD NEWS | JUNE 17, 2014Tweet
NICE asks for more data on Sovaldi
Cost regulators for National Health Service treatments in England and Wales have raised an eyebrow or two by asking Gilead for more information on its hepatitis C wonder drug Sovaldi (sofosbuvir).
In draft guidelines the National Institute for Health and Care Excellence (NICE) said it is minded not to recommend Sovaldi, despite the drug being linked with cure rates of over 90% in just 12 weeks.
While the Institute conceded that available evidence shows Sovaldi is an effective treatment for chronic hepatitis C in certain patients, "evidence is lacking for some subgroups of patients with chronic hepatitis C, and there are also substantial uncertainties in the evidence base presented by the manufacturer", it said, explaining its current stance.
"The Committee has therefore requested further information from the manufacturer before it can decide whether sofosbuvir is a cost-effective use of NHS resources".
The move came as somewhat of a surprise given that, in April this year, NHS England approved funding for Sovaldi in England and Wales significantly at risk of dying or needing a liver transplant, while the Scottish Medicines Consortium cleared its restricted use on NHS Scotland.
The drug was approved for use in Europe in January this year, but has been in the spotlight globally for some time because of its widely-perceived high price tag as much as its stellar effectiveness. With a 12-week course costing just shy of £35,000 in the UK, there are concerns that the drug could prove to be a devastating drain on healthcare resources.
On the other hand, the price of not using Sovaldi is could run much much higher; one in three patients injected with HCV eventually developing liver cirrhosis and managing these patients is costly.
Charles Gore, chief executive of the Hepatitis C Trust, said the drug's high price "should not put off commissioners because price is not supposed to be the determinant – cost-effectiveness is".
"If NICE OK Sovaldi, it should be prescribed to anyone who wants it. We have the Pharmaceutical Price Regulation Scheme (PPRS) to cap the drugs budget," he noted.
The closing date for comments on the draft guidance is 4 July 2014.
New combo shows 100% response rates
Meanwhile, Gilead also unveiled Phase III data showing that the fixed-dose combination of the NS5A inhibitor ledipasvir and sofosbuvir achieved 100% sustained virology response in hepatitis C patients in Japan.
The cure rates observed "are impressive because they were achieved without the need for interferon or ribavirin, both of which involve more complex dosing requirements and may be associated with significant side effects,” said Norbert Bischofberger, Gilead’s Chief Scientific Officer.
The results suggest that the once-daily pill "has the potential to be an efficacious and well-tolerated regimen" for many patients in Japan, he added. Read More & Comment...
Recently Allison Bell, a blogger for LifeHealthPro.com, a newsletter focusing on how insurance companies can maximize revenue posted on CMPI's effort to focus on the value of new cancer treatments from the patient's perspective.. Here's her post and my reply.
On the Third Hand: Drugs Opinion
You raise important questions that need to be answered.. unfortunately until now no one has wanted to check the math.
Here's some math: According to IMS the number of targeted has increased from 11 percent of all cancer drugs to 46 percent of all drugs over the past decade. The average price of drugs has doubled. Meanwhile, cancer drugs as a percent of healthcare spending has actually declined. The rate of hospitalization for cancer has fallen by 20 percent during the same time period. Cancer mortality rates have declined 20 percent. Productivity gains of people living cancer free are conservatively estimated at $68 billion a year, or 2.5 times more than what was spent on new treatments.
New drugs and diagnostics are responsible for 90 percent of these gains.
I would argue that the way to make a system sustainable is to increase productivity (which leads to more people paying premiums and taxe) and reduce the use of more labor intensive services like hospitals. Without new cancer drugs there would be 6 milion fewer people alive today. Without 'expensive' HIV drugs that are also being rationed by health plans, there would be 3-5 milion fewer people alive today. If you cut the work force and tax base by 20- 30 percent by denying access in ways AHIP wants, what happens to the financial support everyone seems to worry about.
The greatest gains will come when more cancer care is personalized and is matched the right time, the first time to patient tumor variability. Yet the ASCO and AHIP plan guidelines discourage and delay the adoption of such innovations.
The fact is, no one with ASCO or AHIP has discussed this math. I offered several times to be part of such discussions. No response. Lowell Schnipper who runs the ASCO value task force has written it’s not worth treating patients if it ‘only’ adds three months of life because “it is not a large enough benefit to trump the greater benefits to many that would have to be foregone to provide it.” Indeed, he believes the problem (of wanting to live longer) “ may be a particularly American one; other cultures do not seem to view the postponement of death by a few months” as important as Americans.
Has anyone covered the fact that the Wellpoint bonus program for cancer doctors measures value in the same way ASCO will measure it: efficacy, toxicity and cost. No mention of quality of life from the patient's perspective. Or how about that both Lee Newcomer and Schnipper said that the way to herd people into pathways that save money for health plans is to have the doctors develop the guidelines so that the plans are not accused of being self-serving (Schnipper and Newcomer's words, not mine). Shouldn't this be a subject of journalistic, if not congressional investigation? Shouldn't such cooperation -- with patients left on the sidelines -- be a source of concern?
Also, ask yourself: Should we take AHIP and critics of drug prices at their word because they don't get funding from pharma and ignore what I and others say because we get support from pharma? Does that mean that groups getting money from AHIP or health plans are to be believed? Is truth a function of where you get funding? Paul Offit did work for Merck on the rotavirus vaccine.. Does that mean that what he said about vaccines NOT causing autism should be discounted whereas Jenny McCarthy should be accepted.
When people attack where CMPI gets it support rather than engaging on the substance, it's a sign of desperation. I expect more of the same as the untenable and discriminatory assault on chronically ill patients continues. Read More & Comment...
"One of the major drawbacks of this type of treatment is that it’s extremely expensive. Insurance doesn’t pay for off-label use of cancer drugs—even if Champions’s scientists are able to prove they will work. Rose and Shapiro wouldn’t say how much they paid Champions to find the perfect drug. “Let’s just say it was a lot of money and I felt lucky that I have the resources to be able to do it,” says Rose. “There’s no doubt that part of me feels a little guilty that others don’t have the ability to do it.”
"It has been said that the best way to survive cancer is to hold off dying long enough that a new treatment can be invented to save your life. That seems to be true now more than ever. Of course, for many cancer is still a death sentence. But Rose is a clear example that it doesn’t have to be. Through seven years of toxic chemotherapies and countless surgeries, he has never left his job (in fact, today he has three jobs—he’s the founder of San Francisco design firm SITELAB urbanstudio, a professor of practice in the Department of City and Regional Planning at the University of Pennsylvania and is running Urban Design+, another design firm, based in New York City). During his treatment, he and Josslyn even had a son, Ryder, now three and a half years old.
In many ways, Rose is living with cancer the way someone with HIV/AIDs would have shocked the world by doing so 10 years ago. The disease, once thought to be a debilitating death sentence, has become something that just needs regular care. According to Shapiro, “Before, when we were told there was only palliative care and they told us the best they could do was manage his disease, I thought that was a failure. But actually, it’s not a failure. You can live with disease in your body.”
Health plans, led by AHIP's Karen Ignagni are not only refusing to pay for cancer survival. And ASCO is enabling such rationing by developing an algorithm that determines 3 months of additional life is not worth spending money on. Or as Lowell Schipper, the head of the ASCO Value Task Force puts it, three months of life (using a $10000 cancer treatment) “it is not a large enough benefit to trump the greater benefits to many that would have to be foregone to provide it.”
It should be pointed out that in 2011, Schnipper remodeled his Maine summer home. The renovation cost $200,000 and took seven months.
When oncologists believe fixing up a second home is more valuable than 3 additional months of life, we have a real problem. Apparently the life of Evan Rose is worth less than Schnipper's hardwood floors.
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The "FACTS" excreted by AHIP are in bold. The reality is in italics.
FACT: Astronomical prices for specialty drugs will blow up Medicare Part D budgets and force higher premiums for seniors
An analysis in Health Affairs last week found that the price of the important hepatitis C drug Sovaldi could increase the cost of Medicare Part D and premiums for seniors by 8%. From this one drug alone, seniors on Part D could see an 8% premium hike.
IN FACT the CBO and several economists have shown that $1 of new drug spending saves $7 in other costs. As for Solvadi, the $84000 (not $100K) treatment is a cure that saves 1 million lives and will reduce spending on liver transplants, hospital stays as well as reduce disability. National Viral Hepatitis Roundtable estimates that without any major changes in current testing practices, the cost of HCV care and treatment to Medicare will increase five-fold over the next 20 years, from $5 billion to $30 billion.
This one-time test, in combination with other targeted treatments, would result in identifying more than 800,000 cases and avoiding as many as 121,000 deaths
Assuming $100,000 value for each additional life per year, that's worth $1.5 million for every individual, meaning every healthcare dollar spent curing Hep C generates $17 of value to each patient, the cost is outweighed by the value of reducing the impact of this disease.
FACT: Astronomical prices for specialty drugs will devastate state Medicaid budgets and displace important priorities like education and infrastructure
One recent analysis highlighted on Vox illustrated that, because Sovaldi is so expensive, California could potentially spend more administering the drug for people on Medicaid than it does for K-12 and secondary education combined. Yes, you read that correctly.
In FACT : California spends $110 BILLION per year on K-12 and secondary education. There are 530,000 people living with hepatitis C in California. Even if all of these people received Solvadi (at a Medicaid discounted price of $60K) that would be a little over $3 billion.
FACT: Astronomical prices for specialty drugs put upward pressure on premiums for all consumers
At its core, the cost of health insurance is a reflection of the cost of health care. The skyrocketing prices that drugmakers are charging has a ripple effect throughout the system, raising premiums and increasing health care costs for individuals, families, and employers.
IN FACT: According to a Milliman study, stopping the shift of up to half the cost of new drugs for cancer, RA, MS, etc would cost AHIP companies a whopping 50 cents per member per month. http://publications.milliman.com/research/health-rr/pdfs/parity-oral-intravenous-injected.pdf
FACT: Health plans offer consumers a range of coverage options, including policies with lower cost-sharing
To distract from their unjustifiable pricing, drugmakers have latched onto distorted coverage comparisons that ignore the range of cost-sharing options consumers can choose from. Hey, anything beats talking about the actual price.
IN FACT.. See above. And if you don't believe me, read the lawsuit AIDS patients and the Naational Health Law Program filed against 4 of the largest AHIP insurers.
FACT: Consumers have out-of-pocket limits that mean health plans and state and federal governments rather than patients are paying the vast majority of the cost of these stratospherically priced drugs
Pharmaceutical companies know that consumers’ out-of-pocket costs are capped under the Affordable Care Act, allowing them to ask for what amounts to a blank check from insurers and government programs. Not surprisingly, drugmakers are making the law work for them.
IN FACT: Many drugs once covered by AHIP plans are not covered under Obamacare if they are prescribed by out of network doctors. Also, many drugs are now NOT covered which means consumers -- the sickest consumers -- have to pay full freight. Yet AHIP could solve the problem with 50 cents per person.
BONUS FACT: Drugmakers have no straight-face explanation to justify the increasingly astronomical prices they have been charging for their medications
BONUS RESPONSE TO LIE: IMS data shows that new drug costs have risen about 5 percent a year. Co-pays and co-insurance costs for consumers have climbed to 40 percent in over half of Obamacare plans according to an Avalere Study.
AHIP is hysterical over the fact that 35 states have required their members to cover new drugs at the same level they do old drugs. And now states are limiting the ability of AHIP plans to force patients to fail first on old medicines (translation: get sicker before we treat you and then talk about hospice instead) instead of using precise, personailzed treatments that work best the first time.
AHIP's pursuit of short term profits is understandable. It has tried to shift the cost on to consumers and blame onto drug companies. Why is AHIP trying to avoid paying for new medicines that save lives, reduce cost and increases productivity? Because it can save money short term by 'redlining' chronically ill patients and avoid having to take on such consumers altogether. Read More & Comment...
It wasn't supposed to work this way, but since the Affordable Care Act took effect in January, Norton Hospital has seen its packed emergency room become even more crowded, with about 100 more patients a month.
That 12 percent spike in the number of patients — many of whom aren't actually facing true emergencies — is spurring the hospital to convert a waiting room into more exam rooms.
"We're seeing patients who probably should be seen at our (immediate-care centers)," said Lewis Perkins, the hospital's vice president of patient care and chief nursing officer. "And we're seeing this across the system."
That's just the opposite of what many people expected under Obamacare, particularly because one of the goals of health reform was to reduce pressure on emergency rooms by expanding Medicaid and giving poor people better access to primary care.
Read the full story.
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Hi-Yo Silver, away!
A new analysis from Avalere Health finds that consumers in exchanges receiving federal assistance to reduce their out-of-pocket costs may experience inconsistent reductions in spending depending on the plan they choose.
Specifically, the data show broad differences in how issuers amend plan designs to meet cost- sharing requirements for those receiving cost -sharing reductions (CSR). While almost all plans reduce deductibles and out-of-pocket caps in CSR plans, many plans do not lower cost-sharing for other treatments and services, particularly specialty drugs.
“Many people assume that the lowest-income exchange enrollees will have reduced cost-sharing across all services, but the reality is quite different,” said Caroline Pearson, vice president at Avalere. “While all plans must have reduced out-of-pocket limits for individuals earning less than 250 percent of poverty, how consumers will reach those limits differ significantly. For example, consumers may not experience reduced cost-sharing amounts for drugs or physician visits in many plans.”
All carriers that offer silver plans on the exchange must also offer silver CSR variations that have higher actuarial values (AV) for people who qualify for cost-sharing assistance. Individuals with incomes between 100% and 150% of the federal poverty line (FPL) receive the biggest reductions in likely out-of-pocket costs. Beyond requirements to lower maximum out-of-pocket caps , plans have broad discretion in how they meet the AV targets for CSR plans.
Notably, the vast majority—96% of 94% and 87% AV CSR plans—reduced deductibles to help meet new requirements. However, wide variation even exists among carriers that lowered deductibles. For example, the highest deductible ($700) among 94% AV plans analyzed is three times greater than the average ($220).
By contrast, a far smaller percentage of plans—including only 5% of 73% variations—reduced cost sharing for Tier 4 medications (specialty drugs). Indeed, a quarter of the 94% AV plans (the plans providing the highest level of cost-sharing assistance) had coinsurance of greater than 20% on the specialty tier.
Notably, Avalere also found that average maximum out-of-pocket limits in CSR plans are lower than those required by the Affordable Care Act. Specifically, among 87% AV CSR plans, the average out-of-pocket limit is $450 (or 20%) lower than required, while among 94% AV CSR plans, the average out-of-pocket maximum is $1,140 (or 22%) less.
“Issuers will continue to have flexibility in how they design cost sharing reduction plans in 2015,” said Dan Mendelson, CEO and founder of Avalere Health. “Looking ahead, consumers who qualify for cost-sharing reductions should look closely at how the plan benefit is structured because it could have a major impact on their actual out-of-pocket costs.”
This analysis was funded by PhRMA. Avalere maintained editorial control over the content of the analysis and release.
Avalere analyzed the 11th version of the Department of Health and Human Services (HHS) Landscape file available on HealthCare.gov. The file contains details on individual and family premiums and benefit designs for plans across the 34 states in the Federal Facilitated Exchange (FFE). The 34 states in the federally-facilitated marketplace include: Alaska, Alabama, Arkansas, Arizona, Delaware, Florida, Georgia, Iowa, Illinois, Indiana, Kansas, Louisiana, Maine, Michigan, Missouri, Mississippi, Montana, North Carolina, North Dakota, Nebraska, New Hampshire, New Jersey, Ohio, Oklahoma, Pennsylvania, South Carolina, South Dakota, Tennessee, Texas, Utah, Virginia, Wisconsin, West Virginia, and Wyoming. This analysis focuses solely on this data file and, therefore, does not reflect plans offered in any state-based exchange.
The file contains 5,800 total silver plans, including standard silver plans as well as the required “silver plan variations.” Drug coverage data in the HHS Landscape file are structured into four formulary tiers; therefore, for plans that have fewer or more than four formulary tiers, the data in this file may not align with the plan’s true formulary structure. The accuracy of all analysis is limited by the accuracy of the data included in the Landscape file itself.
Although Avalere analyzed and included a number of details about deductibles, we did not consider whether a person needs to meet a deductible when analyzing cost-sharing categories to conduct our analysis (e.g., if coinsurance is 10% after meeting a $1,000 deductible, when analyzing costs for the service, Avalere used the 10% coinsurance amount). Plans that noted that there was no charge, or no charge after the deductible was met were excluded. Amounts are rounded to the nearest dollar or percent.
A substantial portion of silver plans indicated in the Landscape file that their benefit design included a $0 drug deductible. An examination of a subset of the Summary of Benefits and Coverage (SBC) documents from these plans confirmed that two-thirds of the sample of plans actually did not require drugs to be subject to a deductible. The remaining third could not be validated with the data from the plan’s SBC.
View Avalere's full report here.Read More & Comment...
WASHINGTON DC, June 4, 2014 – In a letter submitted today to the U.S. Food and Drug Administration (FDA), the National Organization for Rare Disorders (NORD) urged Commissioner Margaret Hamburg to give serious consideration to the concerns of the rare disease community when setting policy regarding official names for biologics, including biosimilars.
With over 7,000 rare diseases identified and 30 million Americans affected, the patient population represented by NORD is extraordinarily heterogeneous, the letter notes. Without thoughtful and consistent naming protocols for biologics, there is the potential for significant confusion among treatment options and increased adverse events, both of which could jeopardize patient safety. Distinguishable naming of all biologics is imperative for health care professionals to deliver the degree of customized care that is routinely required for patients with complicated, uncommon and less well-studied diseases.
“Every patient deserves the care best suited for their medical situation and most likely to give them the best outcomes,” said NORD President and CEO Peter L. Saltonstall. “Biologics are often the most advanced and effective treatments for patients we represent and everyone in the treatment continuum should be able to readily identify the specific drug product a patient was given.”
The letter raises a number of concerns specific to rare disease patients. Rare disease patients usually have unique treatment courses and, while similar medications might work equally well, such a judgment can only be made through tracking of outcomes made possible through distinguishable names.
According to the letter, distinguishable names for biologics would:
* Support the medical community’s vital post-approval learning curve regarding which medicines are best for their rare disease patients;
* Support surveillance and tracking of adverse events given that rare disease patients often do not respond to medications in the same way as other individuals might; and
* Reinforce a critical distinction in the biosimilars law between biosimilars (similar not identical) and interchangeable biosimilars (similar but demonstrated to have comparable clinical results).
Next up: Vote at the AMA House of Delegates.
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The FDA has publicly released a huge amount of data on drug side effects that it hopes will lead to new applications and research.
Under its open FDA project, the agency has released more than 3 million reports on adverse drug events and medication errors recorded between 2004 and 2013. These kinds of reports were only available before through lengthy Freedom of Information Act requests.
Before releasing the information, the agency removed patient identification and other sensitive information. It has also been formatted to let researchers, mobile applications and Web developers easily analyze the data and present it in a way that can help improve how people take drugs.
Eventually the agency plans to release more reports on recalls and labeling, as well as other information it thinks can be useful to developers and researchers.
The move to make medical data more transparent isn’t limited to the FDA. Several drug makers working with the National Institutes of Health have begun releasing clinical trial data in a limited fashion to help find new uses for old drugs.
Obvious by its absence is any mention of greater clinical trial transparency. But that's a whole other can of worms. (For more on this issue, see "Cry Havoc, and let slip the Dogs of Data Transparency.")
A good next step would be for the FDA to be the coordinating body for all relevant e-tized information from both public and private sources. But that’ll take resources. It would be money well spent.
Speaking of transparency – how about an OpenFDA program for the agency’s risk/benefit decision-making process?Read More & Comment...
There’s a difference between off-label communications and off-label marketing – and it’s more than a finesse. It’s one of those 800-pound gorilla issues we’ve been pussyfooting around for too long. And now, at long last, it’s time for a serious conversation.
Here’s a bit of outreach send out by PhRMA. It’s well said. It’s brief. And it's a call to action. Take it seriously.
FDA Restrictions on Medical Communications Can Negatively Impact Patient Care
Some of the regulations and guidances of the Food and Drug Administration (FDA) have a more direct impact on patient care than others. The FDA’s restrictions on biopharmaceutical companies’ ability to share authoritative, regulated data about prescription medicines limits healthcare professionals’ access to information that can help them make informed decisions based on their patients’ individual healthcare needs and preferences.
Biopharmaceutical companies have the most complete and up-to-date information about the medicines that they research, develop and manufacture for use by patients. However, companies are often unable to proactively share valuable information about their medicines, especially for information that is not contained in the FDA-approved prescribing information (the package insert you often receive with a prescription), with physicians and other healthcare providers.
To get the best possible health outcome for patients, FDA should revise its regulations to allow companies to share truthful, scientifically accurate, and data-driven information with healthcare professionals to inform treatment decisions. Some examples of this kind of information include:
· Observational data and “real world evidence” – Information on the safety and effectiveness of medicines taken from medical records based on actual use of approved medicines.
· Sub-population data – Information on the safety and effectiveness of medicines in sub-populations including gender and race. Such information can help healthcare professionals tailor their treatment to meet the needs of individual patients.
· Observational and comparative data – Information from the use of a medicine outside of randomized clinical trials, especially comparisons between two or more therapies.
· Pharmacoeconomic information – Healthcare economic data and information on the economic value of medicines can improve the efficiency of patient care.
· Information on medically accepted alternative uses of medicines – Information on new uses of approved medicines that are listed in major compendia and/or routinely reimbursed by the federal government and major payers. As the National Cancer Institute states, “Often, usual care for a specific type or stage of cancer includes the off-label use of one or more drugs.” Healthcare professionals help patients by applying new uses of approved drugs in “every specialty of medicine.” When patients are being prescribed medicines off-label, they deserve to know that their healthcare professionals have the latest information on these uses.
You Can Help. Patients need their healthcare professionals to have timely, authoritative, FDA-regulated information about available medical treatments. Currently, the House Energy and Commerce Committee is soliciting comments from patients, providers and other stakeholders about how they learn about new treatments and cures. If you believe that physicians and other healthcare professionals should have access to sound, evidence-based information that biopharmaceutical companies have about their medicines in order to help patients make informed healthcare decisions, please send a comment to firstname.lastname@example.org by June 13, 2014.
 See National Cancer Institute, Off-Label Drug Use in Cancer Treatment, available at http://www.cancer.gov/cancertopics/druginfo/offlabeldrug.
 Christopher M. Wittich, et; al., Ten Common Questions (and Their Answers) About Off-label Drug Use, Mayo Clinic Proceedings, available at http://www.mayoclinicproceedings.org/article/S0025-6196(12)00683-0/fulltext#sec3.
 SeeRead More & Comment...
It seems that every day there’s another headline about a serious issue involving generic drug quality. And how to improve the agency’s evaluation of these products drugs was a top priority when the agency recently compiled its regulatory science goals.
In a new Broad Agency Announcement (BAA), the FDA has announced a roadmap that will be funded from a pot of up to $50 million, and it reveals some of the difficulties facing the agency as it works to get a handle on an industry riddled with unanswered questions as companies start to produce more-and-more complex products.
In the BAA, the FDA lays out five broad areas (Post-market Evaluation of Generic Drugs; Equivalence of Complex Products; Equivalence of Locally Acting Products; Therapeutic Equivalence Evaluation and Standards; Computational and Analytical Tools) in which it wants to improve its generic evaluation capabilities and invites organizations to submit proposals detailing how they can help. Many of the specific goals focus on the equivalence between generics and their innovator forbearers. The FDA is particularly interested in methods to compare the bioavailability of complex and locally-acting products. Questions about the bioavailability of generic drugs and its impact on therapeutic effects are as old as the industry itself, but the industry's expansion into new dosage forms--such as sustained release injections and inhaled products--throws up fresh concerns.
Per a report in Fierce Pharma, the FDA is also looking at more human aspects of switching from innovative to generic drugs, with the agency keen to know what patients think about the quality and effectiveness of copycats and how tablet size affects substitution. High-profile quality failings at Ranbaxy, Sun Pharma and Wockhardt could have tainted the public's perception of generics, a possibility the FDA wants to understand while also working to get better at spotting problems early.
Other, less generic-focused aspects of the roadmap also deal with this goal. The FDA wants to develop sensitive, rapid, high-throughput methods to spot microbes, and assess ways other than sterilization to remove such contaminants. The technology is one of several ways the FDA wants to retool its capabilities and those of the industry, with the document also calling for investigations into continuous manufacturing, process analytical technologies and Quality-by-Design.
21st century pharmacovigilance must also include tighter and more regularly monitored post-approval bioequivalence measures. Recent recalls of products such as Budeprion (Wellbutrin) and Metoprolol (Toprol) offer vivid examples. "We are losing control over what people are swallowing," said Dr. Harry Lever, a cardiologist at the Cleveland Clinic. It’s a new and difficult task and calls for better validated methodologies for both data collection and signal prioritization.
Another key regulatory question is the appropriate role of regulators in coordinating input from crucial partners such as physicians, nurses, pharmacists, disease organizations, patients, and pharmaceutical manufacturers. “Real World” event monitoring must become as specific and informing as in a clinical trial environment. To borrow a term from the nuclear disarmament discussion, 21st century pharmacovigilance must work with its various colleagues to “trust, but verify.”Read More & Comment...
From the pages of the Orange County Register:
Foreign meds, quality control
In the past year, several major foreign pharmaceutical companies have been forced to recall thousands of medications sold in the United States. The problems with their products were myriad, including packaging defects, dosage errors, and bacterial contamination. In one especially egregious instance, epilepsy medications were found in a bottle of diabetes drugs, resulting in the recall of more than 2,000 bottles produced by a company based in India.
Given these dangerous errors, it's little wonder that American physicians are growing increasingly worried about the safety of prescription medications imported from foreign pharmaceutical companies. Compromised medications jeopardize the health of their patients.
We must strengthen America's "pharmacovigilance." The federal government should bulk up its drug import oversight apparatus and implement a tracking system that monitors the entire life cycle of medications.
Right now, 40 percent of America's over-the-counter and generic drugs come from Indian suppliers. The World Health Organization has estimated that around 20 percent of the drugs manufactured in that country are counterfeit.
As the New York Times recently noted, at one hospital in Kashmir, fake drugs likely lead to the deaths of hundreds of infants. In January, the Indian health ministry identified at least 32 medicines being sold in Indian stores that didn't meet basic safety and quality standards. Earlier this year, the FDA's commissioner, Margaret Hamburg, expressed her dismay about "recent lapses in quality at a handful of [Indian] pharmaceutical firms.
To protect against dangerous drug imports, the FDA has established the Office of Pharmaceutical Quality, which will more meticulously monitor the quality of brand-name, generic, and over-the-counter drugs.
Congress also enacted legislation in 2012 mandating that foreign pharmaceutical manufacturers be subjected to more intense scrutiny. This bill lead to 160 FDA-conducted inspections at Indian drug manufacturers in 2013, an enormous increase over the number of site visits in previous years.
What American inspectors discovered was often profoundly disturbing.
For example, at one plant owned by the Indian pharmaceutical manufacturer Ranbaxy, the FDA found flies "too numerous to count." Ranbaxy has since been hit with half a billion dollars in fines and has plead guilty to felony charges.
These developments are all the more discouraging once situated within the broader context of the global economy. Pharmaceutical manufacturing is a $14 billion industry in India. When its firms are permitted to push sub-par medications into the U.S. health care market, they often end up displacing American companies.
Such displacement can hurt our economy. Drug firms currently support 810,000 jobs throughout the United States. More than 20 percent of the sector's domestic sales are directed back into local research and development projects.
The American pharmaceutical industry has already proven its commitment to ensuring the quality and safety of the medicines it produces. It has vigorously pushed for the federal adoption of a track-and-trace system, which would monitor a drug's journey from manufacturer to packager to distributer to pharmacy and, ultimately, to patients.
Such a system would significantly strengthen the security of America's drug supply chain.
In contrast, India is simply shrugging off its gross failures in drug production. The Indian Medical Association's secretary-general recently said: "Our drugs are being sold in many countries and being accepted, so we have no issues. How do I know that Western drugs are better than our drugs?"
That casual attitude is unacceptable. American consumers are being put at direct risk by India's malfeasance. If Indian authorities won't take responsibility, the United States must step in and ensure that proper vigilance is in place throughout the entire life cycle of a drug.
A global pharmaceutical market benefits consumers only when the appropriate safeguards are in place. The recent spate of recalls should prompt renewed dedication to pharmacovigilence.
For the sake of both American drug companies and American patients, federal regulators must work harder to ensure that all drug imports meet basic standards for quality and safety.
Peter Pitts, a former FDA Associate Commissioner, is president of the Center for Medicine in the Public Interest.
According to report in BioCentury, the U.S. District Court for the District of Columbia vacated HHS' July 2013 final rule that discounted Orphan drugs when used in non-Orphan indications under Medicare's 340B program. The court said HHS lacked authority to make the rule.
The 340B program requires manufacturers to deeply discount outpatient drugs to hospitals and clinics bearing the brunt of healthcare for low income and other special populations. In 2010, Congress amended 340B to exclude Orphan drugs from discounting, but the law was unclear on whether the exclusion applied when the drugs were used for non-Orphan indications. In 2013, HHS issued the final rule saying the drugs were to be discounted when used in non-Orphan indications, and the Pharmaceutical Research and Manufacturers of America subsequently sued to challenge it.
In the decision, the court said that while the rule "seems like the most reasonable way for implementing the orphan drug exclusion, unfortunately Congress did not delegate to HHS broad rulemaking authority as a means of doing so," noting Congress limited HHS's 340B rulemaking authority to three purposes -- establishing a dispute resolution process, setting methodology for price calculations and imposing monetary civil sanctions. Plaintiff PhRMA said in a statement it is "extremely pleased" with the ruling.
The 340B program has been scrutinized for expanding margins. In June, HHS' Health Resources and Services Administration (HRSA) plans to publish a proposed rule addressing eligibility criteria. HRSA declined to comment on implications of the decision on that rule.Read More & Comment...
Part two of my interview from Context Matters:
In 2010, Representative Henry Waxman voiced concern about non-inferiority trials – but when he was confronted with the science he backed down. That’s a good thing. When politicians start to dabble in science, they generally get badly burned.
Today the FDA is trying to expedite reviews of anti-infectives. The need for these products and that in itself changes the benefit / risk profile. And that will allow for approvals based on less data – and in an expedited fashion.
There are multiple ways to get an important new therapy to market quicker – but that has to be based on a public health need, not a marketing strategy.
Pediatric trials bring both public health and IP benefits, but they are hard to design, hard to recruit for, and hard to field. They’re especially important for anti-depressants and anti-psychotics. We also will be seeing more precise clinical trials in minority and genotype populations. From a perspective of companion diagnostics we’re developing more medicines for small, more precisely identifiable patient populations – clearly the wave of the present and this will continue for the foreseeable future. It’s all about personalized medicine.
The best way to address benefit/risk profile is to make sure that the right patient gets the right medicine in the right dose at the right time. That will result in more positive clinical outcomes and huge cost savings. That’s where you’re going to see the greatest increase in adaptive clinical trials, whether in children, pregnant women, certain genotypes, women, men, African-Americans, Asians, etc.
The benefit/risk profile of a product that’s proven to be more beneficial within an identifiable sub-population is a key for expedited review, because you can more precisely define who it works for and, just as importantly, who should not be using it.
As far as so-called “conditional approval” goes, I wouldn’t look for it any time soon in the US. The last time I spoke with senior members of the FDA, I heard comments like, “What does that even mean?” And, then again, do drug developers really want conditional approval? You invest a lot of time and money to get a conditional approval and then the agency decides to take the product off the market? Is that something to roll the dice on?
Unless and until the FDA can ramp up its pharmacovigilance prowess, any kind of provisional approvals will remain problematic. At the moment, the FDA doesn’t have an eye in the sky.
And there’s the issue of Congress politicizing the FDA process. Plan B, Avandia, Zohydro. As Mark McClellan used to say, “If people are saying we’re approving drugs too slowly, and others are saying we’re approving drugs too quickly. We must be doing something right.”
That’s my story… and I’m sticking to it!Read More & Comment...
Facts that don’t reinforce your cognitive mapping are pesky things.
When it comes to the value of innovative medicines, keywords such as welfare impact, cost-effectiveness, innovative drugs, economic evaluation, quality-adjusted life year are often used to explain why both licensure and reimbursement is deferred or denied.
The opposite is true.
In fact, those very key words are tagged in a new Frontiers in Public Health article, Estimating the potential annual welfare impact of innovative drugs in use in Switzerland
In this new study, Swiss academics have estimated that the introduction of innovative pharmaceuticals provided substantial welfare gains to Swiss patients and the health system.
The authors find that "The introduction of innovative pharmaceuticals since 2000 onward to the Swiss market led to a potential welfare gain of about CHF 781 million in the year 2010."
Here’s the abstract:
Expenditure of health care systems are increasing from year to year. Therefore, this study aimed to estimate the difference in costs and benefits of innovative pharmaceuticals launched 2000 onward compared to standard treatment on the national economy of Switzerland in 2010. The approach and formula described in the pilot study by Tsiachristas et al., which analyzed the situation of welfare effects in the Netherlands, served as a model for our own calculations. A literature search was performed to identify cost–utility or cost-effectiveness studies of drugs launched 2000 onward compared to standard treatment. All parameters required for the calculation of welfare effects were derived from these analyses. The base-case threshold value of a quality-adjusted life year was set to CHF 100,000. Overall, 31 drugs were included in the welfare calculations.
The introduction of innovative pharmaceuticals since 2000 onward to the Swiss market led to a potential welfare gain of about CHF 781 million in the year 2010. Univariate sensitivity analysis showed that results were robust. Probably because of the higher benefits of new drugs on health and quality of life compared to standard treatment, these drugs are worth the higher costs. The literature search revealed that there is a lack of information about the effects of innovative pharmaceuticals on the overall economy of Switzerland.
Our study showed that potential welfare gains in 2010 by introducing innovative pharmaceuticals to the Swiss market were substantial. Considering costs and benefits of new drugs is important.
Nations such as South Africa (where the Medicines Control Council is taking 4-5 years to approve medicines that are globally commercialized) should take notice. If innovative medicines aren’t even licensed within months of their globalization, talking about prices is just a hypothetical exercise.
Imagine all the mortality, morbidity and … costs that could be avoided if people had access to innovative medicines.Facts do not cease to exist because they are ignored. -- Aldous Huxley Read More & Comment...
Study Design and the Drug Development Process
To the Editor
A Viewpoint by Dr Djulbegovic and colleagues1 claimed not only that randomized clinical trials (RCTs) aremore ethical but that greater use of randomized designs throughout the drug development process would “improve the efficiency, ie, enable faster development ofnew, successful treatments.” However,RCTs are outdated for several reasons. First,RCTs are inadequate to evaluate cancer therapies. Genomic analysis is uncovering the tremendous heterogeneity of what previously were considered single diseases. Genomic analysis of cancers of individual patients is disclosing the large number of mutations, and thus targets, within one person. Developing RCTs for targeted therapies would be difficult and timeconsuming, prolonging the wait for effective treatments.
Second, RCTs are a less efficient and accurate method of establishing which treatments work.
A recent study demonstrated that RCTs fail to predict or improve outcomes when evaluating multicausal diseasenetworks or treatments orwhen assessing which particular interactions are relevant.2 Rather than increase the use of RCTs, we propose an increase in the use of N-of-1 studies, which are based on simulations that rapidly sort through billions of possible interactions
at the clinical, genomic, and biological levels to arrive at predictive models of multicausality. This approach has identified candidate markers, which have been successfully used in clinical trials.3 These studies also have predicted the most effective treatments for patients based on a particular genotype
and medical history when RCTs have not.4 Increasing the use of RCTs would add to the cost and time required to developand use new products. Other analytic methods more quickly and precisely match patients to treatment.
1. Djulbegovic B, Hozo I, Ioannidis JPA. Improving the drug development
process: more not less randomized trials.JAMA. 2014;311(4):355-356.
2. Eppstein MJ, Horbar JD, Buzas JS, Kauffman SA. Searching the clinical fitness
landscape.PLoS One. 2012;7(11):e49901.
3. Wu C-C, D’Argenio D, Asgharzadeh S, Triche T. TARGETgene: a tool for
identification of potential therapeutic targets in cancer.PLoS One.
4. Garnett MJ, Edelman EJ, Heidorn SJ, et al. Systematic identification of
genomic markers of drug sensitivity in cancer cells.Nature.
2012;483(7391):570-575. Read More & Comment...