Latest Drugwonks' Blog
After years of focusing the majority of its attention on HIV/AIDS (and to a lesser extent malaria), the global health community is finally waking up to the fact that the biggest health problems now facing developing countries are actually ‘non communicable diseases’ such as diabetes, cancer and cardiovascular disease. And not before time: 80% of global deaths from these diseases occur in developing countries, and already cause double the number of deaths that result from things we more normally associate with these parts of the world, such as infectious diseases, maternal and perinatal conditions, and malnutrition.[1]
Although the UN and its specialised health agency, the WHO, have already started discussing how to respond to this issue, real flesh should be put on the policy bones at a special UN Summit on Non-Communicable Diseases, to be held in New York in September 2011. We can expect whatever global agreement emerges from this gathering to focus on prevention (watch out Big Food and Alcohol), as well as making treatments and diagnostics more readily available in developing countries.
None of this is going to be easy, no matter what is agreed and what funds are committed in New York. The international response to the AIDS crisis in the late 1990s and 2000s highlighted the difficulties of delivering modern medicines and treatments to patients in developing countries, the vast majority of which have dilapidated health infrastructures and demoralised and underfunded health workforces, a large proportion of which has emigrated to better opportunities in countries like the US.
In order to bring some perspective to this issue, CMPI will be discussing this very issue ahead of the NY Summit in Geneva -- the administrative and policy heart of the UN. Our inaugural “Geneva Round Table” event (13th July) will focus on the challenges faced by clinics and hospitals in responding to this epidemiological shift, and we are lucky enough that Dr Eric Roodenbeke, the head of the International Hospitals Federation, has agreed to give his perspective based on his extensive knowledge of hospital policies and health systems approaches.
It should be a great event, and the guest list includes ambassadors, health attaches and other members of the UN health community. If you happen to be in Geneva on 13th July, please do drop by for what should be a most illuminating discussion!
From the pages of Advertising Age:
ANA Weighs Lawsuit With FDA Over Graphic Cigarette Labels
Trade Group: Government 'Can't Put Words in the Mouths of Advertisers'
The Association of National Advertisers says it will fight the federal government's move to slap graphic new warning labels on cigarette packs.
"We are still discussing whether we go directly with a lawsuit or whether we will enter of friend-of-the-court filing," Dan Jaffe, ANA's exec VP for government relations, told Ad Age. But "we will certainly join with others in opposing this proposal." Mr. Jaffe added that the ANA thinks the proposal is unconstitutional because "the government on its own ... can't put words in the mouths of advertisers."
The nine warning labels, unveiled Tuesday by the Food and Drug Administration, are the biggest change to cigarette packaging in more than 25 years. By September 2012, all cigarettes made or sold in the U.S. must display the images, which include graphic depictions of diseased lungs, decaying lips and teeth, a man wearing an oxygen mask and a morgue scene with a message: "Smoking can kill you."
As defenders of advertising, it's no surprise that the ANA is fighting back, as fears swirl that the government might use the momentum to more aggressively target other categories, such as alcohol. Indeed, the ANA is already on record opposing the cigarette labels, filing comments in January after the initial proposal was first unveiled. The association has also sided with six tobacco companies that are challenging in federal court the underlying Tobacco Control Act, passed with bipartisan support in Congress and signed by President Barack Obama in 2009.
But while the ANA is compelled to defend what is sees as free-speech rights, the association has a tricky task of defending Big Tobacco, hardly a sympathetic industry. Former FDA Associate Commissioner Peter Pitts, who served during the Bush administration, said: "It would be very tough for me to defend anybody who believes an anti-tobacco campaign is somehow deleterious to the national good."
He added: "Any image that scares the bejesus out of people away from cigarettes is a step in the right direction. It's been clearly shown by research that visuals are more powerful than words."
The FDA says it chose the images from a list of 36 potential warnings after reviewing scientific literature and reviewing more than 1,700 comments from various interest groups. The warning will be accompanied by a smoking-cessation-help phone number.
"The Tobacco Control Act requires FDA to provide current and potential smokers with clear and truthful information about the risks of smoking -- these warnings do that," FDA Commissioner Margaret Hamburg said in a statement.
But the ANA, in a blog post, said "this is precisely the kind of paternalism that the First Amendment does not permit."
As expected, overturned 6-3.
The ruling can be found here.
Next!
Today’s update on interfering with Non-Interference, paying the value-based piper, and biosimilars at BIO.
Say it ain’t so Jo
Repeating her mistake from last tme around, Representative Jo Ann Emerson (R-Mo.) is co-sponsoring legislation requiring the Medicare to negotiate prescription drug prices.
(The same provision was in the House-passed healthcare reform bill but didn't make it into the final bill as part of “the deal.”)
"The ability to negotiate the cost to taxpayers of prescription drugs purchased through the Medicare program could be a substantial savings at a critical moment," Emerson said in a statement.
Except that’s not true. Politics aside, consider the facts:
"It is not obvious that allowing the government to negotiate with pharmaceutical companies will lead to lower prices than those achieved by private drug plans. Private plans like Kaiser or United are able to negotiate deep discounts with pharmaceutical companies precisely because of the plans' ability to say no – the ability to include some drugs and to exclude others, allowing the market to judge the resulting formulary. On the other hand, when the government negotiates, its hands are tied because there are few drugs it can exclude without facing political backlash from doctors and the Medicare population, a very influential group of voters. Neither economic theory nor historical experience suggests government price negotiation will achieve lower drug prices. Congressional Democrats need to be careful in making the logical leap from market share to bargaining power. Empowering the government to negotiate with pharmaceutical companies is not necessarily equivalent to achieving lower drug prices. In fact, neither economic theory nor historical experience suggests that will be the outcome. Members should think carefully before jumping on the bandwagon – this promise may bring just the opposite of what was ordered."
Stanford Business School's Alain Enthoven and Kyna Fong
And in the words of the American vox populi (aka, USA Today):
"Both the non-partisan Congressional Budget Office and Medicare actuaries have said they doubt the government could negotiate lower costs than the private sector. The theory behind Part D is that market forces and competition among drug plans, overseen by government, can achieve better results than a government-run program. The multitude of plans allows seniors to pick one that best meets their needs. Government price negotiation could leave people without drugs that manufacturers decide aren't sufficiently profitable under the plan. Medicare recipients account for half of all drug prescriptions. With that kind of clout, government might try to dictate prices, not just negotiate them. This could leave people without drugs that manufacturers decide aren't sufficiently profitable under the plan. The VA plan illustrates the point. It offers 1,300 drugs, compared with 4,300 available under Part D, prompting more than one-third of retired veterans to enroll in Medicare drug plans."
Many of the President’s men and women are ready with the following talking point, “Look at how successful direct Federal negotiation works for the Veteran’s Administration,” suggesting that allowing the feds to directly negotiate for Part D is no different from the current VA scenario. But suggesting that the Veteran’s Administration “negotiates” prices for prescription drugs is a false premise.
Under rules set by Congress, to sell drugs to the VA, companies must offer each drug at a price that “represents the same discount off a drug’s list price that the manufacturer offers its most-favored nonfederal customer under comparable terms and conditions.” The medication must be offered “at a discount of at least 24 percent off [the] nonfederal average manufacturer price (NFAMP). An excess inflation rebate is also required, equal to the percentage by which the price increase for [the] drug has exceeded the consumer price index (CPI) in the prior period.” The manufacturer must make all of its drugs available through the Federal Service Schedule for any of its drugs to be eligible for reimbursement under the VA and Defense Department health systems, the Public Health Service (including the Indian Health Service), the Coast Guard, and the various state Medicaid programs.
A study by Professor Frank Lichtenberg of Columbia University found that the majority of the VA formulary’s drugs are more than eight years old and more than 40 percent are 16 years old or more. Just 19 percent of all prescription drugs approved by the FDA since 2000 are available to veterans; only 38 percent approved during the 1990s are.
There’s a big difference between negotiating and mandating – and it’s not a thin line. My fear is that a government negotiated Part D plan is but the first step towards a more strident program of government price controls.
But putting politics aside to advance the public health is, alas, easier said than done. "There's savings to be had by negotiating those drug prices and addressing how Medicare reimbursement takes place for drug benefits," House Minority Leader Nancy Pelosi (D-Calif.) said last month. "This is a rip-off of the taxpayer, to have the pharmaceutical companies make as much money as they do."
Facts? We don’t need no stinkin’ facts.
U.K. provides more color on planned pricing scheme
BioCentury reports that the U.K. Department of Health said that under a planned value-based pricing (VBP) system, the NHS would be mandated to "consistently fund medicines with a value-based price." The government's previously published consultation on VBP had been silent on whether or not the NHS would be required to pay for drugs for which value-based prices had been negotiated. Under the current system, a positive recommendation from the National Institute for Health and Clinical Excellence (NICE) requires the NHS to pay for a drug within three months of publication of the final guidance. The VBP system is slated to be in place by Jan. 1, 2014.
The DoH's statement came in its response to a report by the NHS Future Forum, a group of government and NHS leaders who collected input from professional groups, patients and the general public on the future of the NHS. The forum's report, published last week, recommended changes to the NHS.
FOB GPS
Hope to see you there.
I appreciate you taking the time to respond to my post and email. I will publish your response on my blog without editorializing on my part.
Your analysis did nothing to help patients.. you only raised alarms about conducting smaller studies not randomized to your standards. That's an old saw used by you and others -- along with your assertion that unless you understand the mechanism of action endpoints, even if met, really don't matter. Your assertion that cancer patients might suffer more is a counterfactual argument that frankly is a function of individual response to medicines that, absent biomarkers or algorithms derived from patient level data, cannot be tested. What we do know is that life expectancy is higher from orphan drugs so the fact that you want longer and more expensive clinical trials for orphan drugs translates into you being willing to let sick kids die. And your agenda is not secret: you believe most drugs should not be marketed unless they meet your standards.
Regarding CER, you claim RCT is the gold standard for demonstrating effectiveness. Yet when ALLHAT was all but eviscerated by ACCOMPLISH and other studies, I didn't see you call for a change in clinical practice.
The evidence gathered from smaller trials is not "less valid." Such data does not comport with your cultural and ideological bias against the commercialization of medical information. I have a different bias. And I am not on the Right or the Left when it comes to science.
Finally, regarding BiDil, you should read your own work and what FDA officials have written in response. (See Annals in Internal Medicine, Jan, 2007 vol146 pages 57-62) You did object to it's approval on grounds of political correctness and the mechanism of action argument. In a perfect world we would know the MOA for drugs and diseases. But your view of how to evaluate drugs, RCTs above everything else, will not bring that about.
Best wishes,
Bob Goldberg
Dr. Goldberg –
Our JAMA piece was not even about comparative effectiveness research, or about paying for treatments. We agreed in it that orphan drugs for cancer may sometimes need to be approved with less valid data than is usually required, and called for better understanding how well these drugs work by studying their outcomes once they are on the market. But cancer patients do not benefit – and may well suffer more -- if a drug that does not work is approved and then given to them. We undertook this analysis to help understand how patients with rare diseases could be provided with drugs that actually are going to help them; we certainly did not publish it because of some secret agenda to delay or deny treatment to people. Your making such a wild charge just makes you look silly.
I also do not recall ever having said that BiDil should not be approved because we don’t know its mechanism of action. In any case, since it was just a combination of two widely available generic drugs, the treatment would be readily available whatever FDA did with the application for its approval as a patentable combination.
Reasonable people can (and do) differ about the best way to evaluate new medications, but this statement is so wild that it just looks weird. A flaming, inaccurate diatribe like this reflects badly on its author. Most others on the right have reassessed the wisdom of depicting those with whom they disagree as “malignant presence[s]” who want sick children to “drop dead,” and calling for someone to “pull the plug” on them. Such hate speech just makes those who use it look like they are flailing around in ill-founded rage.
Back in February, Peggy Hamburg made some interesting comments at the Council on Foreign Relations:
* The new reality of food and drug regulation is that it’s global. In fact, it should be a topic for conversation at the next meeting of the G20.
* The recent crises in both food and drug safety will only repeat themselves unless regulatory agencies from around the world work in closer and more regular partnership.
* There is a responsibility on the part of the FDA and other more developed regulatory agencies around the world. (MHRA certainly comes to mind.) to help build “regulatory capacity” for those nation’s that want and need assistance. (Nigeria comes to mind.
* Part of a closer working relationship means a more regular and robust sharing of global intelligence on issues of counterfeiting. (And, for that matter – many other things too.)
* And lastly, “We can’t inspect our way out of this problem."
All good things – progressive things -- but, short of a regulatory Marshall Plan, things that will have to rely (at least initially) on personal relationships between senior officials at various regulatory agencies and a focus on what’s best for global public health writ large is convergent with what’s best for any given nation.
It’s not as easy as it sounds.
Beyond the generally difficult nature of international regulatory harmonization (yes friends, it is a lot harder than it looks) is the profoundly difficult issue of domestic economic interests and trade.
For example, every nation (from the United States to Nigeria) is concerned about counterfeit drugs and unsafe food coming across its borders. That’s obvious. But are all nations equally concerned about controlling the export of counterfeit, unsafe, and substandard products to other nations? That’s trade.
Principles aren’t principles, as my father used to say, until they hurt.
Today the FDA released a new report designed to monitor the rising tide of goods from abroad. It includes planned partnerships with counterpart regulators in other countries and better data-sharing between those regulators.
According to the report, imports of FDA-regulated products are growing at an estimated 15% annual rate.
Peggy Hamburg said it will likely take many years to implement its new approach, which also includes building up its IT capabilities to try to identify risks in the food and drug supplies before they pose serious harm and using public and private third parties to conduct audits and inspections on the agency’s behalf.
Hamburg said that the new approach “will require some up-front investments in terms of dollar resources, human resources and time,” she expects better sharing of data and strategies between regulators in different countries will eventually mean using fewer resources to achieve the same end.
“All of us are confronting the same set of challenges,” said John Taylor, acting principal deputy commissioner of food and drugs.
Not all business travel is arduous. I’ve just returned from the 11th annual France-USA Conference on pharmaceuticals and health policy. The meeting was in Paris. Sometimes being a policy wonk has its perks.
My role was to debate NYU professor, Dr. Victor Rodwin on “The Current State of Obama’s Health Reforms.” It was fun and feisty – especially the parts where we both spoke in French. (Shout out to my high school French teacher, Linda Diaz!)
Dr. Rodwin regularly cited Commonwealth Fund research, ‘Nuff said. I focused on the legality of the individual mandate, the growing “idealism gap” of those who want to improve our healthcare system – but not via the approaches of the Affordable Care Act, the increasing (and increasingly contradictory) roles of comparative effectiveness and patient-centered outcomes research, IPAB (and the creeping fear of Uncle Sam, MD), the dangerous vagaries of academic detailing, and the war against medical innovation (personified by President Obama’s call for a roll-back of biologics patent life from 12 to 7) and the issue of data exclusivity.
My summation – “L’audace, l’audace. Toujours l’audace.”
Another fascinating presentation was offered by Dominique Maraninchi, the relatively new (since the end of February 2011) General Manager of AFSSAPS. That means he’s the French equivalent of the Commissioner of the FDA.
France is going through it’s own “Vioxx Moment,” having recently rescinded the market license for Mediator (a diabetes drug that was widely used off label as a weight loss treatment). “L’Affaire Mediator” is a big deal and has created a public outcry for change and reform. Sound familiar? (FYI – for more detailed information on the Mediator issue, see here.)
Much of what Dr. Maraninchi had to say will sound very familiar to FDA watchers:
AFSSAPS must:
· Reestablish trust
· Enhance pharmacovigilance and do a better job analyzing data and drawing conclusions
· Be more transparent (a truly radical concept for the French bureaucracy)
· Be more accountable
· Offer more information and explanations about its decisions via “bon usage” (safe use)
These might sound rather derivative to a US audience, they are not. While they certainly do share much of what the FDA is trying to accomplish, (1) the French system is very different and these initiatives will require different strategies and tactics, (2) as such, their efforts will deliver different results, and (3) there will be much for the Wizards of White Oak to learn from the AFSSAPS experience.
Another echo of FDA came from Yves Burr (a member of France's National Assembly and chair of that body’s working group on medicines and health products), who asked, “Do we have the courage to put the patient in a role where they can be heard?”
Dr. Maraninchi ended his presentation with a statement of principle that should be widely discussed inside the FDA:
“It is better to make a decision than to have no decision at all.”
Perhaps he should testify at the upcoming Congressional hearings on PDUFA?
Paying doctor as consultants or to speak about products/treatments is problematic when the physician in question is not an expert or not disclosing the payment. And it's no more problematic than a doctor who is paid to write or speak against a product or even does so because of an ideological or cultural belief that any new drug is inherently useless or overhyped. In fact, the latter case is even more troubling since it is shaped not by science or clinical practice but an agenda.
Second, having attended sessions where KOL have spoken (and disclosed support) it is obvious that doctors are not drinking kool-aid. They are curious, skeptical, interested in getting additional information about the risks and benefits of drugs and devices.
Finally, the ongoing effort to ban pharma payments to doctors is part of a larger effort to substitute information provided by or supported by industry by groups that want to impose their views and control all medical information.
Diffusion of innovation is evil to the opponents of pharma supported KOL. So is any research or clinical finding funded by drug companies. This is a purist and paranoid view of science.
Meanwhile, pharmalot is hosting a webinar on a subject every drug company and doctor should be paying attention to. I encourage everyone to tune in.
How Can Pharma Use Social Media for Real-Time Intelligence and Research?
Tuesday, June 21, 2011
2:00 p.m. ET | 11:00 a.m. PT
Webcast Duration: 1 hour
Registration: $59
Register Now - Click Here!
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.
