Latest Drugwonks' Blog
According to CDER Office of New Drugs Director John Jenkins, a crucial piece it creating a risk/benefit assessment model is the patient perspective.
"I think it's very important to understand the patients' perspective about how they value the benefits and how they are willing to accept the risk," Jenkins said.
Jenkins continues, "A lot of us are basing these decisions in the abstract. We don't have the disease, we haven't achieved the benefit, and we do not actually have to weigh, personally, that benefit against the risk."
And further, "Regulators and others may not consider those benefits to be very important, but to the patients, they are extremely important and allowed them to go on about their lives.”
Providing people with different therapeutic options is another important aspect of risk-benefit assessment, Jenkins said. "We at FDA consider having a range of choices available to be a very important aspect in the practice of medicine." Even though a drug may not be a best in class, he noted, it may still be the best choice for an individual patient.
There also are various societal expectations about "how we should interpret and apply the standard," he said. He gave the example of when FDA was first criticized for being too slow in approving drugs, and later criticized for being too fast in drug approval, and now it is once again criticized for being too slow.
"So as regulators we have to be aware of the societal expectations of how we interpret our standard and how we make our decisions.”
John Jenkins taking about “societal expectations” within the context of new drug review?
Bravo.
Here is an anecdote about how Medicaid performs today:
Holes in the safety net: Medicaid falls short just as some need it most
As need rises, patients struggle to find doctors who take the insurance plan
By Tom Curry and JoNel Aleccia
msnbc.com
updated 11:13 a.m. ET Aug. 11, 2009
GOSHEN, Ind. - For weeks now, 2-year-old Ashley Soto's hair has been falling out in clumps and bunches.
Doctors at the Maple City Health Care Center, a neighborhood clinic where the toddler's family receives most care, couldn’t diagnose the problem. The child needed to see a specialist, but no local dermatologist would agree to accept Medicaid, the government’s safety net plan. Instead, Antonia Mejorado, 33, has to drive nearly two hours to see a dermatologist willing to treat her daughter's potentially serious illness.
“There is not a doctor around here that takes Medicaid,” said Mejorado, whose husband, Osvaldo Soto, 33, has recently seen his hours cut to almost nothing at a local mechanic shop....
About one in five physicians say they are not accepting any new Medicaid patients, largely because of low payments or delays in reimbursements, according to the Center for Studying Health System Change in Washington, D.C.
‘A lot of things are scary’
Ashley Soto, the 2-year-old with unexplained hair loss, is eligible for Medicaid because she was born in this country. The child’s parents, who are from Mexico, are not. Her mother worries that a serious diagnosis could mean more long days traveling the 75 miles between Goshen and Michigan City, Ind.
“A lot of things are scary — not having a doctor nearby in case something happens to her,” said Mejorado, who is also the mother of three other children ages 17, 14 and 14 months.
http://www.msnbc.msn.com/id/32127373/ns/us_news-the_elkhart_project/
Things are going to get a lot scarier....
From an AEI Report
Medicaid: The Forgotten Issue in Health Reform
By Robert B. Helms
AEI Health Policy Outlook, November 2009
"...the distribution of federal Medicaid expenditures per poor person ranges from a low of $2,014 in Nevada to a high of $7,753 in Maine, an almost fourfold difference. One of the objectives of the original Medicaid legislation was to allow states the flexibility to design a Medicaid program that reflected the preferences of their voters and their willingness to be taxed to support the program. Some states obviously prefer more extensive programs than others, but this choice has been influenced by the availability of federal, open-ended matching. This has had the effect of expanding the program in all states, while expanding it relatively more in higher-income states. The availability of federal funds has also reduced the incentive for the states to design cost-effective programs or to reduce the level of fraud and abuse.
The politics of fixing this conundrum are indeed daunting since it pits one region of the country against another, but to ignore this effect means Medicaid will continue to do a poor job of helping the vast majority of the poor and disabled. Expanding Medicaid eligibility to more adults and to higher-income families with 100 percent federal financing may have a marginal effect to narrow this distribution, but it will not change the basic incentives now built into the system of federal financing."
And efforts to equalize Medicaid spending by "redistributing" resources to the lower cost regions will only hurt the concentration of urban poor in rich states... The same goes for efforts to punish doctors who spend more per patient than 10 percent of the lowest spending docs... (Features in health reform proposals in both houses)
And we are going to increase health care coverage for 50 percent of the uninsured in this fashion?
From the MSNBC report:
"Medicaid reimbursement rates can be as much as 40 percent lower than those for private insurance, according to John Holohan, the director of the Health Policy Research Center at The Urban Institute, a Washington think tank.
Michigan, for example, recently announced a 4 percent cut in payments to doctors, dentists and hospitals who treat Medicaid patients. The move prompted a new exodus of doctors who’ve decided to limit care.
"I love what I do, but I can't keep getting cuts from Medicaid," Dr. John Pfenninger, a family physician in Midland, Mich., told the Associated Press. "It's time to say no. "
Are higher co-pays the answer to controlling medical costs?
NO: Discouraging treatment is shortsighted and leads to more costs.
In the current national health care debate, let’s hope we never hear the words, “As Georgia goes, so goes the nation.”
Since 2005, Georgia politicians have been conducting a dangerous penny-wise, pound-foolish experiment with its state health program by hiking co-pays for brand-name prescription medications.
The results of that policy have been sicker, less productive state employees. These Georgians end up consuming more and costlier health care during the course of their lives, as their neglected conditions worsen.
The lesson here is that higher co-pays discourage patients from getting the treatment they need — especially when they reach upwards of $100.
Just consider what Daniel M. Hartung of Oregon Health & Science University calls the “co-pay effect.”
Professor Hartung and his colleagues analyzed the effect of even a small co-payment — $2 for generic drugs and $3 for brand-name drugs — for those pharmaceuticals that were available to Oregon Medicaid enrollees in 2003.
The co-pay fees were not required for patients who were unable to pay. The researchers examined pharmacy claims data on about 117,000 Medicare enrollees with conditions like depression, schizophrenia, respiratory disease, cardiovascular disease and diabetes.
They found that the patients’ overall use of prescription drugs decreased by about 17 percent after the introduction of the co-pay policy.
It should come as no surprise that any policy that encourages patients to stop taking their prescription drugs is a recipe for disaster.
There is already a growing national trend of Americans not adhering to their prescribed drug regimens.
A study by Wolter Kluwer Health found that fewer and fewer Americans are even bothering to fill their prescriptions.
In fact, during the fourth quarter of 2008, American patients neglected to fill 6.8 percent of their brand-name prescriptions — a 22 percent increase when compared to the previous quarter.
This practice — often known as prescription drug “nonadherence” — can have serious repercussions on a patient’s health.
For example, hypertensive patients who do not take their prescribed medicines as directed suffer 5.4 times as many poor clinical outcomes as those who do.
And poor outcomes are 1.5 times more common for heart disease patients who do not take their meds regularly.
This adds an additional $100 billion to $300 billion in health care costs each year.
The trend has been perpetuated by the fact the Americans with private health insurance have found themselves paying more for prescription drugs in recent years.
Why? Because insurance companies are paying less. In 2000, people under 65 with private health insurance paid 37.2 percent of their prescription drugs costs out of their own pockets.
Many Americans mistakenly believe that this increase in out-of-pocket expenses is the result of higher drug costs. The data reveal otherwise.
In fact, the growth in prescription drug co-payments outpaced the growth rate of prescription drug prices four to one.
It’s easy to see why plans to increase the co-pays for Medicare beneficiaries will also have serious adverse effects on the health of our seniors, as well as on our health care system as a whole.
Unable to afford their prescriptions, many Medicare enrollees will begin treating strict obedience to their drug regimen as a luxury, not a necessity.
As more and more seniors choose to abandon their treatment, health care outcomes will suffer, as prices soar even higher.
Making health care decisions based solely on cost is a losing strategy over the long term for both the state and for the health of its residents.
But maybe those are the kind of shortsighted, budget-driven results you get when cost-over-care bureaucrats run your health plan.
Peter J. Pitts is president of the Center for Medicine in the Public Interest and a former FDA associate commissioner.
- Thomas W. Abrams - Director, Division of Drug Marketing, Advertising, and Communications (DDMAC) - Center for Drug Evaluation, and Research (CDER)
- Kathryn J. Aikin - Social Science Analyst, DDMAC - CDER
- Rachel E. Behrman - Deputy Director, Office of Medical Policy (OMP) - CDER
- Gerald Dal Pan - Director, Office of Surveillance and Epidemiology - CDER
- Kristin Davis - Deputy Director, DDMAC - CDER
- David J. Horowitz - Assistant Commissioner for Policy, Office of Policy, Planning, and Budget - Office of the Commissioner
- Ele Ibarra-Pratt - Branch Chief, Advertising and Promotional Labeling Branch - Center for Biologics Evaluation Research
- Jean-Ah Kang - Special Assistant to the Director, DDMAC - CDER
- Sharon Kapsch - Chief, MDR Policy Branch - Center for Devices and Radiological Health (CDRH)
- Dorothy R. McAdams - Supervisory Veterinary Medical Officer, Division of Surveillance - Center for Veterinary Medicine
- Seth S. Ray - Associate Deputy Chief Counsel for Drugs and Biologics - Office of the Chief Counsel
- Robert Temple - Director, OMP - CDER
- Deborah Wolf - Regulatory Counsel, Office of Compliance - CDRH
Yes, the government will be empowered to redistribute health care spending along geographically areas. Doctors in the 20 percent of counties that have the lowest medicare expenditures will get a 5 percent bonus in the House bill. The House bill also propose to revamp hospital and physician fees to equalize geographical payment levels with the goal of forcing down higher spending areas to the lower spending regions. The impact on innovation, access to care and health will be devastating. Such efforts ultimately fail. Both Canada and the UK have substantially increased health care spending (at a faster rate than we have) and still have wide regional disparties in expenditures and outcomes. In the meantime, health outcomes in the US are better than they are in Canada or the UK for cancer and heart disease, the leading causes of death.
Two new articles on the issue of off-label promotion.
The first, courtesy of Bloomberg.com, is far from courteous or unbiased. Consider this quote from Jerry Avorn, a professor at Harvard Medical School and regular critic of the industry:
“Marketing departments of many drug companies don’t respect any boundaries of professionalism or the law.”
Untrue and unfair. That’s a pretty broad brush – but Dr. Avorn has never worried about the unintended consequences of hyperbole.
The Bloomberg article continues, “The widespread off-label promotion of drugs is yet another manifestation of a health-care system that has become dysfunctional.”
That’s outside of any citation – that’s just the reporter talking. Bad journalism.
And then back to Jerry Avorn:
“It’s an unbearable cost to a system that’s going broke. We can’t even afford to pay for effective, safe therapies.”
Now the article is confusing the issue of off-label promotion with off-label use. And that’s a much clearer articulation of the article’s broader agenda.
The complete Bloomberg article can be found here.
A much better omnibus discussion of off-label promotion can be found in the November issue of Nature Biotechnology. An excerpt:
“… on October 2, Allergan, in Irvine, California, filed a lawsuit against the FDA seeking to challenge off-label regulations. Allergan contends that agency rules stop them from sharing safety information about off-label use of approved drug Botox (Clostridium botulinum toxin). The company contends that Botox is effective for as-yet unapproved uses in spasticity, and given that physicians are already using it for that purpose, it is important to communicate information to reduce the risk of adverse events. Indeed, Allergan claims the regulations violate the right to free speech under the First Amendment of the US Constitution. But it also stands to reason that if the information it seeks to communicate was in the literature, Allergan would not have needed to file the suit.”
The complete Nature Biotechnology article can be found here.
Also quoted in the Bloomberg article newbie FDA employee Peter Lurie, who makes an important point, “Most physicians don’t keep track of FDA-approved uses of drugs.”
All the more reason for a robust and expanded “safe use” program by the FDA.
Label detailing aids anyone?
From today's New York Times:
F.D.A. Fighting False Online Claims About Swine Flu Treatments
You can buy healing gels that “create a barrier between you and the potentially deadly virus now spreading across the globe.” Or “ionic silver” that kills every known pathogen, germ, bacteria, virus or fungus within six minutes. “Spray Swine Flu . . . Gone . . . with ionic silver on your hands,” one ad claims.
Now that the White House has declared swine flu a national emergency, and with the H1N1 vaccine in short supply, many Web sites have been peddling swine flu nostrums.
The Food and Drug Administration has identified 140 different dubious products sold online and has sent letters to 75 manufacturers. It is violation of federal law to market products that claim to prevent or treat H1N1 and that have not been approved by the F.D.A.
The agency has gone after sellers of gloves, inhalers, masks, shampoos, herbal extracts, air fresheners and an array of vitamins that make claims about fighting swine flu. Some of the Web sites were fly-by-night operations that have since closed down.
The complete New York Times story can be found here.
Food for thought for those who continue to foolishy call for risky drug importation schemes.
The first is that, of 12,500 measured patient-doctor conversations in 2008, the research firm found only 23 requests for specific drugs.
That’s somewhat counterintuitive considering all the punditry and political bloviation about DTC advertising driving “unnecessary” prescribing. But it’s not at odds with studies (including those done by the FDA) that show that about 6% of all doctor visits in the United States are a direct result of a patient (also known as a consumer) having seen a DTC ad. Taken together, these results demonstrate that DTC ads drive patients to visit their doctors (a good thing) and that those visits do not result in inappropriate pressure to prescribe.
The other Verilogue item of interest is that the most frequently cited drug by patients was Boniva. The Boniva DTC campaign, features actress – and honest-to-goodness osteoporosis sufferer -- Sally Field.
Supportive data, via IMS Health, shows that prescriptions for Boniva were up 11% last year, and sales of the drug grew 25%, to $675.6 million. The message, according to Richard "Erik" M. Gordon, assistant professor at the University of Michigan's Ross School of Business, is “authenticity.” Gordon says that, "People want to believe a celebrity isn't just doing this because they were paid a pile of money."
Maybe so. But, in the 21st century, solid healthcare communications is a lot more complicated than finding a celebrity sufferer -- it’s about moving from “Gidget” (the role that initially made Ms. Fields a household name) to “Widget” (social media tools that communicate healthcare messages – pharmaceutical and otherwise – in more immediate, trusted, peer-to-peer ways). Friends and neighbors. Now that’s star power.
Bouquets to Boniva for doing it right. And brickbats to Senators, such as Minnesota's Al Franken, who believe that DTC is deliterious to the public health despite mountains of evidence to the contrary.
FDA Unveils Safe Use Initiative that Targets Preventable Harm from Medication Use
Agency also releases drug dosage guidance for measuring devices included with OTC medications
The U.S. Food and Drug Administration today announced the Safe Use Initiative, a program aimed at reducing the likelihood of preventable harm from medication use.
“Too many people suffer unnecessary injuries from avoidable medication misuse, errors and other problems. The FDA is launching the Safe Use Initiative to develop targeted solutions for reducing these injuries,” said FDA Commissioner Margaret A. Hamburg, M.D.
Millions of people are harmed every year from inappropriate medication use. Many injuries occur as a result of incomplete access to information about a drug, a patient, or the patient’s condition.
Other preventable sources of harm include unintentional misuse of medications, medication abuse, and attempts at self harm. Unintended exposure to prescription medications such as opioid drugs can cause harm, even death, in a single dose, if taken by someone other than the patient who was prescribed the medication.
“Only through coordinated interventions across all sectors of the health care system can we substantially reduce preventable injuries from using medications,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research. "All participants in the health care community have a role to play in reducing the risks and preventing injuries from medication use.”
More detailed information on the new program was contained in a report, titled, “FDA’s Safe Use Initiative – Collaborating to Reduce Preventable Harm from Medicines.” The report was released by Drs. Hamburg and Woodcock at FDA’s annual Science Writers Symposium at the agency’s White Oak Campus in Silver Spring, Md.
As outlined in the report, the FDA intends to collaborate with health care professionals and other stakeholders to identify drugs and drug classes that are linked to preventable harm. A list of specific problems, cross-sector interventions for reducing harm from these problems, and the metrics for success will be developed.
The report highlights several risk-reduction projects that may benefit from Safe Use collaborations, including evaluating consumer medication information, communicating about the risk of inadvertent overexposure to acetaminophen, implementing safeguards against surgery fires caused by alcohol-based surgical preps, and avoiding contamination of multiple use medication vials.
To further advance the Safe Use Initiative, the FDA intends to hold a series of public meetings to gather feedback as the candidate list is being developed and will open a public docket to receive comments on the report and proposed candidate cases.
The agency also today made public new FDA guidance for companies that manufacture, market, or distribute over-the-counter liquid medications packaged with dosage delivery devices such as calibrated cups, droppers, syringes and spoons.
The guidance document, titled “Dosage Delivery Devices for OTC Liquid Drug Products,” was posted for advanced viewing in the Federal Register today.
Accidental overdoses can be caused by dosage delivery devices that are unclear or are inconsistent with the labeled dosing instructions.
"This new drug dosage guidance document is an example of steps that can be taken to ensure safer medication use,” said Woodcock. “Many accidental overdoses result from confusion about exactly how much of a drug to take. Better measuring devices will help patients, parents, and other caregivers use the right amount of these medications – the safest and most effective dose – especially for children.”
WHY YOU CAN'T GET SWINE FLU VACCINE
The shortage of swine-flu vaccine results not from drug- company greed or outsize demand but almost entirely from the government's decision to pander to unfounded and unscientific fear.
As The Wall Street Journal reported last week, the US government set out to have the H1N1 vaccine produced largely in single-dose syringes -- a demand that has set back production considerably, because multidose vials are far easier to make.
And the only reason to seek single-dose production was to please people needlessly worried about the preservative thimerasol, which is used to provide multiple doses of the vaccine. The fear -- utterly groundless and repeatedly debunked is that thimerasol can cause autism and other neurological disorders in infants and other young children.
If not for that decision, we'd have more than enough vaccine. Instead, because the government yielded to pressure from antivaccine fringe groups, we're behind the curve on protecting millions of children from swine flu.
It's a tribute to the effectiveness of pandemic planning and the introduction of new technologies that the nation will likely have plenty of H1N1 vaccine within a month.
Flu strains don't show up when expected. H1N1 was supposed to arrive along with the "regular" seasonal flu, but it began three months early. Using genomic technologies, public-health officials and such vaccine developers as Novartis and Sanofi-Pasteur isolated the H1N1 strain and identified antibodies for production in record time.
Yet, despite this technological ability to adapt quickly to a pandemic threat, our system has been hamstrung by pseudoscience evangelists.
The thimerasol scare, disproved time and again, persists in part because the same public-health entities that encourage immunizations have made significant concessions to small, persistent fear-mongering activist groups.
In 1999, the Food and Drug Administration and the Centers for Disease Control called for the maximum removal of thimerasol from vaccines -- even though both stated there was no scientific evidence of side effects.
Beyond pressuring the government, antivaccine activists have had an indirect effect on the private sector's ability to meet the need for vaccines. Lawsuits in the '80s claimed that DTP (diphtheria, pertussis, tetanus) vaccine caused brain damage; the court actions scared off investment in production facilities using the new recombinant technologies.
As a result, the number of firms producing DTP and other vaccines plummeted. In 1980, 18 companies made vaccines; by the end of the decade, only four were left.
The loss of innovation, investment and profitability in the vaccine industry means we have a gene-based development process for the 21st century -- but a production process that hasn't changed in the last 100 years.
We reap the consequences of that antiquated production process today -- visible on signs posted outside local drugstores and clinics across the country: "Out of H1N1 Vaccine."
But production hasn't been the only casualty of antivaccine hysteria. Since the 1980s, many children have been left unprotected against diphtheria, pertussis and tetanus. How many hundreds of thousands of American children have suffered, from pertussis or other diseases, because of a crackpot minority? How many more will suffer during the current shortage?
By bowing to the unfounded concerns of a few, our government has compromised the safety of many. It's done an admirable job, so far, of communicating with the public about the H1N1 threat and boosting immunization rates. But perhaps its most important job moving forward is to properly align policy with established scientific opinion and the public's best interests.
Twisting the canons of science endangers the public's health and exposes our children to real harm.
Robert Goldberg is vice president of the Center for Medicine in the Public Interest. rgoldberg@cmpi.org
Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.
