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President Obama and Congress should have checked with the country’s physicians before passing a law that relies on our efforts to handle health insurance expansion to more than 30 million more people.
A new on-line survey by the non-profit The Physicians Foundation, one of the largest doctors surveys ever performed, confirms that over two thirds of physicians are pessimistic about the future of medicine, over 84 percent feel that our profession is in decline, and a majority would not recommend it as a career for their children. (The survey was sent to over 600,000 doctors and over 14,000 responded).
If you ask my three children you will find that neither my wife or I (also a physician) are recommending a medical career to them despite the fact that we still manage to find ways to enjoy what we do.
Read the full piece here.
Read More & Comment...
Dear CDER Staff:
On September 6, 2012, CDER Center Director Janet Woodcock informed you that she would be proposing important organizational changes within the Center to sharpen the Center’s focus and strengthen resources around pharmaceutical quality.
As part of these organizational changes, we will also be proposing that the Drug Shortages Staff (DSS) be moved from the Office of New Drugs and be elevated to the Office of the Center Director under the leadership of Deputy Director for Regulatory Programs, Dr. Doug Throckmorton.
On July 9, 2012, President Obama signed into law the Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012. In this new law, Congress provided FDA with new authorities to combat shortages of drug products in the United States and imposed new requirements on manufacturers regarding early notification to FDA of issues that could lead to a potential shortage or disruption in supply of a product.
Drug shortages can occur for many reasons, including manufacturing and quality problems, delays, and discontinuations. They have reached crisis proportions for many healthcare systems and are creating personal crises for many patients. Fortunately, the Agency has been able to prevent a significant number of drug shortages. In 2011, FDA helped prevent 195 drug shortages; as of August 2012, FDA prevented close to 100 shortages.
FDA places tremendous value on the Center’s efforts to curb drug shortages. In addition, with the new authorities brought by FDASIA, we look forward to an ever-increasing focus on this crucial program.
The proposed elevated placement for DSS -- with its greater visibility and prominence within the Center -- reflects how important the work of the DSS is to ensuring that patients in need have critical and life-saving drugs available to them. Undoubtedly, this work is paramount to the Center’s mission to guarantee that safe, effective, and high-quality drugs are available to the American public.
Doug Throckmorton
Deputy Director for Regulatory Programs, Office of the Center Director
John Jenkins
Director, Office of New Drugs
Read More & Comment...Each year, physicians in America conduct over 1.2 billion patient visits, treating illnesses ranging from the minor to the life-threatening.
With A Survey of America’s Physicians, the Physicians Foundation has endeavored to provide a “state of the union” of the medical profession. What are physicians thinking about in the year 2012: about the practice of medicine, about their career plans, and about the current state of the healthcare system?
The survey was sent to over 630,000 physicians – or over 80 percent of physicians in active patient. One thing is clear -- it is a challenging and uncertain time to be a doctor.
The results of the survey reflect uncertainty and should be taken in the context of current events.
Key findings include:
* Over three quarters of physicians – 77.4 percent – are somewhat pessimistic or very pessimistic about the future of the medical profession.
* Over 84 percent of physicians agree that the medical profession is in decline.
* The majority of physicians – 57.9 percent -- would not recommend medicine as a career to their children or other young people.
* Over one third of physicians would not choose medicine if they had their careers to do over.
* Over 52 percent of physicians have limited the access Medicare patients have to their practices or are planning to do so.
* Over 26 percent of physicians have closed their practices to Medicaid patients.
* Over 62 percent of physicians said Accountable Care Organizations (ACOs) are either unlikely to increase healthcare quality and decrease costs or that that any quality/cost gains will not be worth the effort.
* Over 59 percent of physicians indicate passage of the Patient Protection and Affordable Care Act (i.e., “health reform”) has made them less positive about the future of healthcare in America.
What’s wrong with this picture?
The complete study can be found here.
Read More & Comment...A companion’s words of persuasion are effective.
-- Homer
Diagnostic test quality may not be something that pharmaceutical and biotechnology companies have had a lot of experience dealing with, but shortfalls in a diagnostic development program can undercut efforts to streamline clinical trials for personalized medicines.
The Pink Sheet reports that the need for a high-quality diagnostic test development program was a key message delivered by a representative from FDA’s devices center at a September 14th conference on drug/diagnostic co-development.
The conference, sponsored by the Friends of Cancer Research and Alexandria Real Estate Equities Inc., served as a discussion forum for a multi-stakeholder draft proposal aimed at enabling late-stage clinical development of drugs with companion diagnostics by quickly identifying a patient subset most likely to benefit from treatment while minimizing exposure of patients least likely to benefit.
FDA officials were generally receptive to the draft ideas put forward. However, they highlighted the threshold need for a well-characterized, high-quality diagnostic that can accurately distinguish between those patients well-suited for treatment and those unlikely to derive any benefit.
The draft conference paper presented at the meeting was developed with the goal of guiding design of Phase III trials to evaluate a drug and companion diagnostic in situations where prior studies do not provide a clear definition of the diagnostically selected population and sufficient evidence to restrict development.
The draft is intended to fill some of the stakeholder-identified gaps in FDA’s July 2011 draft guidance on vitro companion diagnostic devices. In that 12-page guidance, FDA explained how it defines a companion diagnostic and its expectations for simultaneous development and approval of a drug with the accompanying test.
However, the guidance left many in the drug and device industries wanting more details, such as the regulatory ramifications for drug/diagnostic combinations that are not developed and reviewed in parallel fashion and how laboratory-developed tests fit into the agency’s paradigm for companion diagnostics.
“Patients have to be protected by appropriately balancing the strength of the diagnostic hypothesis with the need for thorough data generation and evaluation,” the draft paper states. “We believe the appropriateness of including marker-negative patients primarily depends on the strength of the science in support of the diagnostic hypothesis (including but not limited to mechanism of action, pre‐clinical efficacy and, if known, class effect), the potential for risk to patients, and clinical data available to date,” the draft paper states.
FDA Office of Hematology and Oncology Products Director Richard Pazdur highlighted several basic principles that FDA considers when evaluating the use of biomarkers in applications submitted to the agency.
The first among these is that the companion diagnostic must be essential for use of the drug, Pazdur said. “It’s not like we’d like to have it or it might be nice to have it or we might in the future need it. It should be essential for the use of the drug when it is licensed.”
Read More & Comment...The U.S. should implement policies that will double the output of new, innovative medicines for important unmet medical needs within 15 years, according to a report released yesterday by the President's Council of Advisors on Science and Technology. The report on "Propelling Innovation in Drug Discovery, Development, and Evaluation" said this goal could be met through increasing funding for basic, translational and regulatory science and creating a broad-based partnership bringing together government, industry, academia and other stakeholders to improve the discovery, development and evaluation of new medicines.
The report recommends creating a new "special medical use" pathway to approve drugs for narrow populations. It also recommends strengthening FDA's postmarketing surveillance capacity by appropriating $40 million per year for the agency's Sentinel electronic safety surveillance system. PCAST said FDA should explore the creation of an adaptive, or progressive, approval system, but it concluded that legislation creating an adaptive approval system "would be premature at this time."
Read More & Comment...The Patient-Centered Outcomes Research Institute’s draft methodology report contains discussions and language choices that could (per Pink Sheet reportage) “tarnish” the institute’s approach to research on pharmaceuticals.
Rust never sleeps.
Both PhRMA and BIO express consternation about what they see as the draft report’s implication that the public should be suspicious of the results of drug industry-sponsored trials.
Their views are contained in recent comments on a draft methodology report written by PCORI’s Methodology Committee. The methodological standards contained in the report will be part of funding review criteria for future applicants for the institute’s research grants.
The idea that manufacturer-sponsored trials may be untrustworthy is mentioned in the report’s introductory chapter, which contains a section labeled “A trust problem.”
PhRMA, in its written comments, responds that the “implication that physicians are willing to subject patients to potential harm as a result of financial interests is troubling and should be deleted.”
BIO’s written comments agree that the passage is “unduly biased against industry-sponsored research” and “presents a skewed view of research conducted by biopharmaceutical companies.”
The Association of Clinical Research Organizations takes an even stronger stance, saying that for the Methodology Committee “to make such assertions of manipulation or bias without any supporting evidence is highly problematic and calls into question the objectivity and integrity of the committee.” It adds that the discussion “impinges on PCORI’s credibility as a research organization by exposing its own potential bias.”
And then there’s the issue of cost in the draft report’s Chapter Five. It says: “The committee’s view is that in the context of PCOR, cost, like other aspects of the health care delivery system, can be a factor in the effectiveness of care if it influences choices made by patients and clinicians. Cost can be an incentive for delivering inappropriate care, not just a barrier to appropriate care. Providers may have incentive to favor more costly treatments under the common belief that ‘more is better’ in healthcare.”
BIO contends that identifying cost as a potential endpoint “is in direct conflict with the authorizing statute’s specific prohibition of PCORI from considering cost effectiveness in studies of comparative effectiveness.”
PhRMA asserts, “While cost undoubtedly can influence the quality and patient-centeredness of care that individuals receive, research that includes cost as an element of analysis or endpoint of measurement is outside the scope of PCORI’s mandate.”
Industry commenters also say the report is trying to do too much by addressing issues that lie outside of research methodology, in particular the report’s discussions of setting research priorities – the topic of an entire chapter – and disseminating research results.
BIO observes that ACA assigns two responsibilities to the methodology report: provide recommendations for PCORI on methodological standards and develop a translation table to guide researchers in applying the standards to specific studies. The “diversity of other subjects the report discusses,” in particular the discussions of the dissemination of research results and developing research priorities, “distracts from the mandated focus on scientifically-derived methodological standards for PCOR and the framework underpinning the development of a translation table,” BIO says.
PhRMA joins in calling for PCORI to cut these non-mandated subjects from the report, saying that given the resources demands of providing comprehensive standards for PCOR, “we recommend PCORI focus its report on standards for research, rather than important but ancillary issues like research priority-setting and results dissemination.”
Read More & Comment...Studies more firmly tie sugary drinks to obesity
By MARILYNN MARCHIONE - AP Chief Medical Writer - Associated PressFriday, September 21, 2012
New research powerfully strengthens the case against soda and other sugary drinks as culprits in the obesity epidemic.
A huge, decades-long study involving more than 33,000 Americans has yielded the first clear proof that drinking sugary beverages interacts with genes that affect weight, amplifying a person's risk of obesity beyond what it would be from heredity alone...
This means that such drinks are especially harmful to people with genes that predispose them to weight gain. And most of us have at least some of these genes.
In addition, two other major experiments have found that giving children and teens calorie-free alternatives to the sugary drinks they usually consume leads to less weight gain.
Collectively, the results strongly suggest that sugary drinks cause people to pack on the pounds, independent of other unhealthy behavior such as overeating and getting too little exercise, scientists say.
That adds weight to the push for taxes, portion limits like the one just adopted in New York City, and other policies to curb consumption of soda, juice drinks and sports beverages sweetened with sugar.
Now what's wrong with the article and the studies that make this claim?
1. The article on genetics and soft drinks did not control for other forms of sugar or interaction with other behaviors. Or other genes for that matter. For instance, there are other genetic mutations that appear regulate obesity which interact with the amount of fiber in one's diet.
2. Even if gene-soft drink association (let's just say sugar) is established, the contribution of the genetic factor may be -- and has been reported to be -- quite small. For instance, most of us have genetic mutations that increase the risk of diabetes or cancer. But the relative contribution of those genes and their mutations to winding up with either disease is very, very small in most of us. Other factors and genetic interactions trigger disease and influence it's progression. Getting a virus or infection, or having high levels of inflammation, etc. can lead to epigenetic changes that shape disease risk. Shame on the report and the researchers for not qualifying their message in this way.
3. Similarly, the reporter asserts that sugary drinks are the biggest source of calories in the American diet. Wrong. The USDA's Dietary Guideline Advisory Committee gives the breakdown for most Americans...
Yeast breads (129 calories per day)
• Chicken and chicken mixed dishes (121 calories per day)
• Soda/energy/sports drinks (114 calories per day)
• Pizza (98 calories per day)
Soft drinks are not even the biggest source of sugar among many age groups. (Snacks are) Nor are they problem. What is? We don't eat enough plant-based fiber, we take in too much saturated fat.. And we don't exercise. The DGAC again:
Several distinct dietary patterns are associated with health benefits, including lower blood
pressure and a reduced risk of CVD and total mortality. A common feature of these diets is an
emphasis on plant foods. Fiber intake is high and saturated fat is typically low. When
total fat intake is high, that is, more than 30 percent of calories, the predominant fats are
monounsaturated and polyunsaturated fats. Carbohydrate intake is typically in the range of 50 to 60
percent of calories, but these often include whole grain products with minimal processing, as well as
cooked dry beans and peas. The totality of evidence documenting a beneficial impact of plant-based
dietary patterns on CVD risk is remarkable and worthy of recommendation.
The so-called 'empty' calories don't come from soft drinks but the food we eat with them. But soft drinks are a sweeter target and feed into the narrative that corporations process food to kill us for the sake of profits. The same capitalism = pollution story that has been applied to energy, chemicals and pharmaceuticals is being applied to food.
When you think about it, the professional critics and their recording secretaries (the media) are attacking the four human innovations that have made life on t his planet better and healthier.
Read More & Comment...
Study Divides Breast Cancer Into Four Distinct Types
By GINA KOLATA
In findings that are fundamentally reshaping the scientific understanding of breast cancer, researchers have identified four genetically distinct types of the cancer. And within those types, they found hallmark genetic changes that are driving many cancers.
These discoveries, published online on Sunday in the journal Nature, are expected to lead to new treatments with drugs already approved for cancers in other parts of the body and new ideas for more precise treatments aimed at genetic aberrations that now have no known treatment.
The study is the first comprehensive genetic analysis of breast cancer, which kills more than 35,000 women a year in the United States. The new paper, and several smaller recent studies, are electrifying the field.
“This is the road map for how we might cure breast cancer in the future,” said Dr. Matthew Ellis of Washington University, a researcher for the study.
Researchers and patient advocates caution that it will still take years to translate the new insights into transformative new treatments. Even within the four major types of breast cancer, individual tumors appear to be driven by their own sets of genetic changes. A wide variety of drugs will most likely need to be developed to tailor medicines to individual tumors.
“There are a lot of steps that turn basic science into clinically meaningful results,” said Karuna Jaggar, executive director of Breast Cancer Action, an advocacy group. “It is the ‘stay tuned’ story.”
The study is part of a large federal project, the Cancer Genome Atlas, to build maps of genetic changes in common cancers. Reports on similar studies of lung and colon cancer have been published recently. The breast cancer study was based on an analysis of tumors from 825 patients.
“There has never been a breast cancer genomics project on this scale,” said the atlas’s program director, Brad Ozenberger of the National Institutes of Health.
The investigators identified at least 40 genetic alterations that might be attacked by drugs. Many of them are already being developed for other types of cancer that have the same mutations. “We now have a good view of what goes wrong in breast cancer,” said Joe Gray, a genetic expert at Oregon Health & Science University, who was not involved in the study. “We haven’t had that before.”
The study focused on the most common types of breast cancer that are thought to arise in the milk duct. It concentrated on early breast cancers that had not yet spread to other parts of the body in order to find genetic changes that could be attacked, stopping a cancer before it metastasized.
The study’s biggest surprise involved a particularly deadly breast cancer whose tumor cells resemble basal cells of the skin and sweat glands, which are in the deepest layer of the skin. These breast cells form a scaffolding for milk duct cells. This type of cancer is often called triple negative and accounts for a small percentage of breast cancer.
But researchers found that this cancer was entirely different from the other types of breast cancer and much more resembles ovarian cancer and a type of lung cancer.
“It’s incredible,” said Dr. James Ingle of the Mayo Clinic, one of the study’s 348 authors, of the ovarian cancer connection. “It raises the possibility that there may be a common cause.”
There are immediate therapeutic implications. The study gives a biologic reason to try some routine treatments for ovarian cancer instead of a common class of drugs used in breast cancer known as anthracyclines. Anthracyclines, Dr. Ellis said, “are the drugs most breast cancer patients dread because they are associated with heart damage and leukemia.”
A new type of drug, PARP inhibitors, that seems to help squelch ovarian cancers, should also be tried in basal-like breast cancer, Dr. Ellis said.
Basal-like cancers are most prevalent in younger women, in African-Americans and in women with breast cancer genes BRCA1 and BRCA2.
Two other types of breast cancer, accounting for most cases of the disease, arise from the luminal cells that line milk ducts. These cancers have proteins on their surfaces that grab estrogen, fueling their growth. Just about everyone with estrogen-fueled cancer gets the same treatment. Some do well. Others do not.
The genetic analysis divided these cancers into two distinct types. Patients with luminal A cancer had good prognoses while those with luminal B did not, suggesting that perhaps patients with the first kind of tumor might do well with just hormonal therapy to block estrogen from spurring their cancers while those with the second kind might do better with chemotherapy in addition to hormonal therapy.
In some cases, genetic aberrations were so strongly associated with one or the other luminal subtype that they appeared to be the actual cause of the cancer, said Dr. Charles Perou of the University of North Carolina, who is the lead author of the study. And he called that “a stunning finding.”
“We are really getting at the roots of these cancers,” he said.
After basal-like cancers, and luminal A and B cancers, the fourth type of breast cancer is what the researchers called HER2-enriched. Breast cancers often have extra copies of a gene, HER2, that drives their growth. A drug, Herceptin, can block the gene and has changed the prognosis for these patients from one of the worst in breast cancer to one of the best.
Yet although Herceptin is approved for every breast cancer patient whose tumor makes too much HER2, the new analysis finds that not all of these tumors are alike. The HER2-enriched should respond readily to Herceptin; the other type might not.
The only way to know is to do a clinical trial, and one is already being planned. Herceptin is expensive and can occasionally damage the heart. “We absolutely only want to give it to patients who can benefit,” Dr. Perou said.
For now, despite the tantalizing possibilities, patients will have to wait for clinical trials to see whether drugs that block the genetic aberrations can stop the cancers. And it could be a vast undertaking to get all the drug testing done. Because there are so many different ways a breast cancer cell can go awry, there may have to be dozens of drug studies, each focusing on a different genetic change.
One of Dr. Ellis’s patients, Elizabeth Stark, 48, has a basal-type breast cancer. She has gone through three rounds of chemotherapy, surgery and radiation over the past four years. Her disease is stable now and Dr. Stark, a biochemist at Pfizer, says she knows it will take time for the explosion of genetic data to produce new treatments that might help her.
“In 10 years it will be different,” she said, adding emphatically, “I know I will be here in 10 years.”
Read More & Comment...A research letter published in JAMA says “closer attention” should be paid to the lack of risk information in advertising for the OTC switch versions of prescription drugs. Research sponsor CVS Caremark says OTC ads should convey the same information as Rx ads.
Facts are facts – Drug Facts, to be precise. And, no doubt, when prescription drugs become available over-the-counter advertisements are less likely to tell consumers about the potential harms and side effects.
But how little is too little – and how much is too much?
To say (as many now are) that OTC ads should carry the same warnings as their Rx brethren is to assume that Rx warnings are useful. When it comes to, for example, the so-called “Brief Summary,” that’s an open question (and that’s being charitable). More of what the consumer doesn’t understand isn’t the solution.
Is it the FDA’s responsibility to figure this out? Before we answer that, perhaps a more direct question is, does the FDA have the social science chops to do so? With all due respect to Kit Aikin and crew – this issue will be best resolved through the joint efforts of industry and agency.
And one size may not fit all.
Beyond BTC, it’ll be interesting to see how various Rx-to-OTC applications address the question of consumer education.
Ladies and Gentlemen of industry – it’s time to step up to the plate.
Read More & Comment...From The New York Post
An ugly way to get insurance
By ROBERT GOLDBERG
Last Updated: 11:23 PM, September 19, 2012
Posted: 11:10 PM, September 19, 2012
The good news: More Americans have health insurance. The bad news: It’s because they don’t have jobs.
ObamaCare supporters hail the drop in the number of uninsured, announced by the Census Bureau last week, as a sign of the new law’s success. In fact, it’s a sign of continued job-market decline and of how many of us have to depend on government programs for far too long.
Nearly 1.4 million more people had health insurance in 2011 than in 2010 — but that includes nearly 800,000 who gained coverage despite not working at all.
In short, the rise comes mainly from more Americans being forced into safety-net programs by declining incomes and reduced job opportunities.
Look at this trend another way: In 2011, the number of people covered by Medicaid jumped 2.3 million, while Medicare saw 2 million new enrollees.
And only 575,000 of those new Medicare cases were people turning 65. Most of the other 1.5 million is associated with the exponential growth in people becoming Medicare-eligible because they’ve filed for Social Security disability coverage.
By comparison, the number of people gaining health insurance via work rose only 730,000.
ObamaCare fans also claim that much of the increase in coverage came from the Obama law’s mandate that young adults can stay on their parents’ health plans until they turn 25.
Indeed, the administration boasted earlier this year that 3 million young adults got insurance that way. But the Census Bureau report shows that health coverage for people in that age bracket rose by only 540,000.
And it’s clear that ObamaCare was not responsible for much of even that increase. Past Census reports show the share of 19- to 25- year-olds on Medicaid or Medicare doubling from 2000 to 2011. That long-term trend — not the ObamaCare mandate — plainly accounts for a good chunk of the rise in coverage of these younger folks.
Slice it another way. The total increase in health coverage for ages 18-24 in 2011 was 825,000. Nearly 331,000 of that was from employer-based coverage. More than 220,000 was from Medicaid enrollment.
So, at most, that’s 247,000 from ObamaCare’s under-25 mandate.
And that gain comes at a hard-to-measure cost: The price of forcing insurers to cover under-25s on their parents’ policies is higher premiums for other people.
Let’s be clear: Medicaid, the Children’s Health Insurance Program and Medicare make health insurance affordable for tens of million of Americans. But that safety net, in place before ObamaCare was enacted, is supposed to be a temporary source of support when we need it, not a permanent solution.
And a jump in the number of Americans who have to use that safety net is nothing to brag about.
Robert Goldberg is vice president of the Center for Medicine in the Public Interest.
http://www.nypost.com/p/news/opinion/opedcolumnists/an_ugly_way_to_get_insurance_etNZJPm74YKgP0euAZJWSM#ixzz271N2B2m9 Read More & Comment...
Drugs elude cookie-cutter approval process
A powerhouse mix of pharmaceutical companies including Abbott, AstraZeneca, Boehringer Ingelheim and Bristol-Myers Squibb has launched a non-profit tasked with making it easier for companies to bring news drugs to market.
The initiative, called TransCelerate BioPharma, is headed up by J&J alum Garry Neil. Its first project focuses on clinical trial execution and will include creating an investigator site, developing clinical data standards and establishing a comparator drug supply model, according to a statement. More tangible details were not forthcoming, other than the comment that the goal is to help speed drugs to market, but the news release quoted FDA Director Janet Woodcock as saying the collaboration “has the promise to lead to new paradigms and cost savings in drug development, all of which would strengthen the industry.”
In other words, it appears to be a project that tries to pin down the very thing that has roiled industry watchers when it comes to the FDA's review process: predictability and its seeming absence. Yet experts have told MM&M in the past that uniform standards won't create an if-then scenario in which the FDA will approve drugs based on meeting the demands of a checklist.
“Predictability assumes that you can approve drugs in a cookie-cutter kind of fashion and you just can't do that,” Dr. Steven Nissen, a frequent member of FDA advisory panels and chairman of the Cleveland Clinic's cardiology department, told MM&M. “It's always going to be a nuanced decision. It's always going to be a careful evaluation of benefit vs. risks,” he added.
The agency has come under a mixed review, with critics saying the FDA is approving drugs without adequately considering a drug's risks. Advocacy group Public Citizen is suing the agency over its approval of the Alzheimer's drug Aricept 23, an approval which its deputy director of the Health Research Group Michael A. Carome told MM&M was made even though the agency acknowledged that the drug didn't meet the standards the regulators demanded even before it came up for review. The drug, at a higher dose of the 5- and 10-mg versions which have gone generic, has been linked to nausea, vomiting and dizziness, but with little benefit, according to Carome and other critics.
“This represents an extreme example of the FDA's failure to properly weigh the evidence and only approve drugs when there's clear evidence [that] benefit outweighs the risk,” Carome said.
Nissen, who was not part of the Aricept 23 review and does not specialize in Alzheimer's, said that the risk-benefit equation had a degree of flex that depends on who's doing the calculation.
“I think the most salient example is cancer drugs. You're dealing with a disorder with often a lethal disorder. These drugs that are used to treat cancer are pretty toxic, but the disease is a pretty lethal disease,” he said.
Former FDA Associate Commissioner Peter Pitts said the FDA gets knocked in part because it doesn't do a very good job at explaining how the drug review and approval process works.
Among the steps is a clinical trial design that the FDA approves before it's kicked off. Pitts noted that hitting all the right design notes doesn't guarantee a panel's endorsement if the results raise questions.
“Those questions need to be answered.” Pitts highlighted two other components of the FDA's decision-making process which includes basing its decisions on “the twin pillars of safety and efficacy,” a balance he said is important because “you can't look at safety independent of benefit.” He also noted that the committees are comprised of experts that see data differently, which can add nuance to discussions.
But nuance or heated argument doesn't always mean pushback. The May debate over the blood thinner Xarelto was a contentious committee hearing, in which panelists took serious issue with the quality of the data presented. The drug passed with a panel endorsement despite the attacks on missing patients and an inability to state just how many patients may have died during the clinical trials.
Adding to the fuzziness is that what constitutes acceptable risk is also subject to change. Recent examples: weight-loss drugs Qsymia and Belviq which failed to clear the FDA the first time they were up to review, which was before the CDC declared obesity an epidemic.
Pitts said one way to provide continuity would be to use the same requirements for drugs that have the same purpose and said that this industry push is not about lower standards, but about making smart investments. He said ambiguity in that regard is "not fair and that's not good science." He said it could also help within the wider context of creating a single standard that can be used for both international and domestic regulatory review.
Such a change will not remove risk, and both Pitts and Nissen said results can't simply be accepted because panelists didn't know what to ask before results roll in. Even when a clinical trial meets expectations and has the FDA's go-ahead, Nissen noted that new risks are bound to surface because clinical trials are little more than real-world approximations. “You have done a series of studies with a drug in a few thousand people and the drug may be used in a million . . . there is no way to be absolutely certain whether a drug is going to be helpful or harmful,” he explained.
Nissen said he understands the drug industry's frustration with the case-by-case approach, but he said it can't be avoided. “It's about their investment and their bottom line and I completely understand that but the FDA has a different mission and that mission is to protect public health.”
Two items for your consideration:
EC Does It
BioCentury reminds us that the House Energy and Commerce Committee is expected to vote this week on a bill that would allow FDA to collect newly enacted generic drug user fees, a committee spokesperson told BioCentury. The bill does not include a provision that would allow FDA to collect new biosimilar user fees.
Last week, the House of Representatives passed a continuing resolution that would extend funding at current levels for government operations but omitted technical language that would have allowed FDA to benefit from an increase in drug and medical device user fees and to spend the newly enacted user fees for biosimilars and generics.
FDA had planned to use $299 million from new generic drug user fees in FY13 to reduce huge application backlogs, increase postmarket safety oversight, and ramp up inspections of generic drug manufacturers outside the U.S.
Occupy Pharmalot
I was pleased to offer a guest op-ed on Ed Silverman’s Pharmalot blog. Here’s a link. Of particular interest is the comments section. Have a look and see how the other 47% lives.
Read More & Comment...The Patient Centered Outcomes Research Institute began accepting proposals for up to $96 million in grants for research projects addressing four of its five priorities.
The fifth element, the one it’s ignoring, however, is the most important.
The four priorities are: comparing alternative prevention, diagnosis and treatment options; improving healthcare systems; comparing communication approaches to providing comparative effectiveness research information; and addressing potential differences in prevention, diagnosis or treatment effectiveness, or preferred clinical outcomes across patient populations.
(Earlier this month, on BioCentury This Week, PCORI Executive Director Joe Selby told that low back pain, uterine fibroids and depression could be early targets for comparative effectiveness research.)
What’s not being addressed, is PCORI’s fifth priority, improving the nation's capacity to conduct patient-centered outcomes research by building data infrastructure, improving analytic methods and training researchers, patients and other stakeholders to participate in the research.
Hello! That’s the most urgent need but, alas the least agenda-driven for those whose ultimate goal is a US NICE. After all, why fund programs to advance a science-based understanding of patient outcomes data when you can fund “research” programs to find the best way to “communicate” about comparative effectiveness.
Say it ain’t so Joe.
Read More & Comment...Since the end of 2011 the FDA has sent 11 warning letters to companies in seven different countries that were exporting drugs to the U.S. after having let their required registration and listing expire.
The warning letter recipients also failed to respond to letters FDA sent them last year notifying them that they were not registered or listed for imports into the U.S., according to nearly identical language in the warning letters.
“FDA has established an ongoing program to identify drug manufacturing firms that have not complied with registration and listing requirements and to notify such firms of their ostensible noncompliance,” the agency said. “FDA has issued warning letters to some of the firms that have not responded to this notification and are out of compliance with registration and listing requirements. These warning letters have targeted firms whose lack of compliance has the greatest potential impact on FDA's regulatory mission.”
FDA’s focus on compliance at foreign drug manufacturing facilities has been sharper since the tainted heparin scandal in 2008, and new provisions in the recently passed user fee legislation is designed to reduce the number of unregistered facilities -- particularly for generic drugs.
But that was before sequestration.
We’ll see.
Read More & Comment...“The convergence of two powerful forces is driving today’s medical innovation to the top of the national agenda: The urgent need for medical breakthroughs and the unprecedented opportunities created by recent advances in science.”
So said El Lilly & Company’s Grand Poobah John Lechleiter at yesterday’s “Advancing Medical Innovation” summit in Washington, DC. (The event was co-sponsored by Lilly and the Washington Post.) I was pleased to attend.
More Lechleiter:
“Our only hope of breaking our of the crisis is through innovation that changes the terms of the trade-offs we must make and expands the scope of what’s possible.”
The Hoosier Honcho then offered a very potent example of what he meant:
“In the early 1950s, the cost of polio care in the US was predicted to be $100 billion by the year 2000 but, thanks to the advent in 1954 of the polio vaccine, the cost of treating polio in the US in the year 2000 was $100 million.”
In other words – the prediction was off by 99.9% because of game changing innovation. Can we do that again? We’d better try – and try hard.
But Lechleiter offered a crucial caveat, “Realizing the benefits of medical innovation requires an ecosystem where innovation can thrive.”
Are we there yet?
Read More & Comment...President Obama is wooing seniors with promises to protect Medicare as they've known it. On the defensive because of the $716 billion his health care law takes from Medicare, Obama assures seniors he's cutting payments to hospitals and other providers, not their benefits.
Don't be bamboozled. It's illogical to think that reducing what a hospital is paid to treat seniors won't harm their care. A mountain of scientific evidence proves the cuts will worsen the chance that an elderly patient survives a hospital stay and goes home. It’s reasonable to conclude that tens of thousands of seniors will die needlessly each year.
Under ObamaaCare, hospitals, hospice care, dialysis centers, and nursing homes will be paid less to care for the same number of seniors than if the health law had not been enacted. Payments to doctors will also be cut.
Read more here.
Politico on the Hatch Act violation by HHS Secretary Sebelius:
INDEPENDENT COUNSEL: SEBELIUS CROSSED THE LINE - Limits on political maneuvering by public officials working on the taxpayers' dime are nebulous, byzantine and often blurred. But the Office of Special Counsel - an independent prosecuting authority - has concluded that HHS Secretary Kathleen Sebelius broke the law when she exhorted a North Carolina crowd last February to reelect her boss, President Barack Obama, during what was supposed to be an official visit. The determination - which OSC said was an isolated incident - creates an immediate headache for the Obama administration on a day that Census data showed a dip in uninsured Americans, partly because of the Affordable Care Act.
--From the OSC report: "[Sebelius] noted that North Carolina is critical in the next election and emphasized that it is 'hugely important to make sure we reelect the president.' These statements were made in Secretary Sebelius's official capacity and therefore violated the Hatch Act's prohibition against using official authority or influence to affect the results of an election."
--Sebelius admitted she shouldn't have injected politics into an official event but pointed to her office's immediate decision to reclassify the event as a campaign stop and to reimburse the U.S. Treasury for her travel. More telling, though, was her explanation for the lapse: "As I have also explained, keeping the roles straight can be a difficult task, particularly on mixed trips that involve both campaign and official stops on the same day."
Read more here.
Read More & Comment...
Much important discussion of late about possible new categories for Rx-to-OTC switching – namely erectile dysfunction medicines and statins. But how does this impact consumer information? Consider the impact of the FDA-to-FTC switch.
The FDA requires prescription drug advertising to provide consumers with a "fair balance" of risks and benefits. The FTC, on the other hand, holds drug advertisements to the same standards as other consumer products, requiring a "reasonable" standard of truthfulness.
What’s the impact on how these products are presented to consumers?
According to a new study, when prescription drugs become available over-the-counter, advertisements for the medications are less likely to tell consumers about the potential harms and side effects. This according to Dr. Jeremy Greene, an associate professor in the history of medicine department and the department of medicine at Johns Hopkins University (study published in the September 12th issue of the Journal of the American Medical Association).
Greene and his colleagues analyzed print and broadcast advertisements for four commonly used drugs that were heavily marketed to consumers as prescription drugs and then approved for sale over-the-counter.
The drugs included loratadine (brand name: Claritin, sold over-the-counter since 2002), omeprazole (brand name: Prilosec, went over-the-counter in 2004), orlistat (brand name: Alli, Xenical, sold over-the-counter since 2007), and cetirizine (brand name: Zyrtec, sold over-the-counter since 2008).
When the drugs were available only by prescription, 70 percent of the ads mentioned potential harms. After the drugs were available over-the-counter, only 11 percent did, the investigators found.
After drugs became available over-the-counter, only about half of print and broadcast advertisements mentioned a drug's generic name, compared to 94 percent of ads when drugs were prescription-only. Knowing a drug's generic name can help consumers make sure they're not taking more than one medication that has it as a component, risking overdose, Greene explained.
Greene believes that the FDA should be given authority to regulate marketing of over-the-counter drugs, or perhaps the FTC should adopt guidelines similar to what the FDA requires.
Rather than asking either underfunded agency to take on more work (for which they are neither staffed nor suited), perhaps OTC advertisers should consider what they could do to better educate consumers. After all, a key FDA consideration in approving an Rx-to-OTC switch is whether consumers understand key communication objectives of the label, relating to directions for use, contraindications, in-use warnings and precautions.
Knowledge is power.
Read More & Comment...
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