Latest Drugwonks' Blog
According to executive director Guido Rasi, the European Medicines Agency will issue final guideline on biosimilar monoclonal antibodies in March or April with draft guidelines on the approval process for copies of other drugs to follow in May or June. (These will include low molecular weight heparin and modern analogues of insulin.)
Rasi said he believed biosimilars would play an important role in future European healthcare and also noted that many pharmaceutical companies are developing next-generation versions of medicines.
Rasi, "I don't really have concern about the level of safety. What is a question-mark is efficacy, since many biotechnology products are now being improved."
“Biobetters” anyone?
BioCentury reports:
CDER creating medical policy board
FDA is creating a medical policy board at the Center for Drug Evaluation and Research to enhance the consistency of medical policies, CDER Director Janet Woodcock told BioCentury. Woodcock, who announced the initiative to CDER staff on Friday, said the new board is a response to complaints from drug sponsors that review divisions often take different, sometimes contradictory, approaches to similar issues.
Examples of issues the board will work on include creating a CDER-wide policy for obtaining approval based on a single trial, and the types of evidence that can be used to supplement a single trial. The board also will establish standards for use of the Animal Rule to obtain approvals of bioterrorism countermeasures based on efficacy studies in animals, Woodcock said.
In addition to consistency, the new body will also examine whether approval standards are infeasible, and if they are, establish new standards. The board's conclusions will be published and will be subject to public comment procedures, Woodcock said. It will be headed by Rachel Sherman, director of CDER's Office of Medical Policy
Is the free market the cause or the solution to the problem of drug shortages.?
Yes.
According to a story in today’s Detroit Free Press:
“The most common causes are manufacturing violations, production delays, shipping problems or ingredient shortages … Shortages grow as generic manufacturers have consolidated and fewer plants are left making certain drugs.”
All of these issues can be addressed and resolved through smart free market solutions and the helping hand of economic incentives.
As Scott Gottlieb commented in front of the Senate Finance Committee:
The is a clear correlation between price and availability – with many of the cheapest infused medicines also being the ones that seem most likely to be in shortage. These drugs are often sold for very low prices, sometimes just several dollars for a single dosage vial of a medicine. As a result, the cost of manufacturing ends up comprising a sizable proportion of the overall price of the finished medicine. In some cases, these drugs are being sold at a loss to their manufacturers once all the production and distribution expenses get fully loaded into the cost. The economic problems are widespread, and deeply embedded in the markets for these drugs.
We should consider policy constructs that would give manufacturers a financial incentive to develop intellectual property that improved the manufacturing characteristics of generic medicines even if these changes it didn’t alter the clinical properties of a drug.
Let’s adopt a free-market solution and free American physicians, pharmacists, and patients of the problem of needless drug shortages.
What do Avastin and Plan B have in common?
The answer is that both products received attention from both the FDA and HHS. One was a policy decision, the other a political.
During the FDA’s hearing on Avastin, HHS announce that “The label change will not affect our coverage.” (Actually, he should have said “potential label change,” but neither here nor there.)
That “Yes from HHS” was a policy decision that was also a savvy political one (had CMS withdrawn reimbursement for Avastin’s use in breast cancer, it would have become a front-and-center election issue).
On the other hand, Secretary Sebelius’ override of the FDA’s decision to allow unfettered OTC access for Plan B was a political decision thinly (and awkwardly) couched as a policy decision.
That’s the difference between policy and politics.
The institute is trying to advance the cause of CER without creating a political flashpoint.
By BRETT NORMAN | 12/29/11 9:32 AM EST
So far, the new institute that’s supposed to run comparative effectiveness research has avoided the harsh political rhetoric of health care rationing and “death panels” — but its supporters worry that its luck could be about to change.
The Patient-Centered Outcomes Research Institute, set up by the health care reform law, is walking a fine line, trying to advance the cause of comparative effectiveness research without creating a political flashpoint. And that means getting as much buy-in as possible from stakeholders throughout the health care sector.
In January, PCORI will lay down two markers — its draft research agenda and its national principles — outlining the framework of a national program for comparative effectiveness research. And a lot of people are watching closely.
“They are really operating under the shadow of the death panel crazies,” said Jerry Avorn, professor at Harvard Medical School and author of "Powerful Medicines." “There’s still a lot of fear that even the most reasonable and necessary research — say, the best approach to lowering blood pressure or cholesterol, for instance — will be demagogued and demonized by those on the right.”
But tempers have cooled at least somewhat since the arguments flared in 2009 after the stimulus bill included $1.1 billion for CER. The calmer environment is leading supporters to be guardedly optimistic that PCORI will be able to proceed — albeit cautiously — without excessive political distraction.
“My belief is that much of the storm has subsided — not to say it can’t come back,” said Robert Dubois, chief science officer of the National Pharmaceutical Council. Dubois wrote a paper this month in Health Affairs with advice for how PCORI should prioritize treatments for study — transparently and based on explicit evidence, with input from all stakeholders.
“The combination of the way the legislation was written, the board of governors and the tone that they have set has satisfied most people,” he said.
The point of comparative effectiveness research is to compare two or more different ways of treating the same condition to see which one works best. The idea is that if definitive best practices can be established, they will be widely adopted by providers and may be preferentially reimbursed by payers.
Cheaper treatments that are effective would be favored.
It may sound harmless — like common sense, even, to the uninitiated — but it’s a menacing prospect to some pharmaceutical companies and medical device-makers who are concerned that their products may wind up on the wrong side of the ledger.
For this reason, Michael Cannon, director of health care studies at the Cato Institute, says good comparative effectiveness research is almost suicidal.
“The whole point of [comparative effectiveness research] is to find out what doesn’t work,” Cannon said in an email. “Every time the government has tried to do CER, the guys who provide the stuff found not to work successfully lobby to have the offending agency defunded. I see no reason to think this time will be any different. The moment it produces useful CER, PCORI is toast.”
And that’s just one source of opposition. The other includes general foes of the Affordable Care Act, who have seized on elements like the Independent Payment Advisory Board, and end of life counseling, suggesting that health reform will lead to government “pulling the plug on grandma.”
Betsy McCaughey, former lieutenant governor of New York and author of “Obama Health Law: What It Says and How to Overturn It,” fueled some of the most heated claims in 2009 about threats of government health care rationing.
McCaughey said she won’t prejudge PCORI’s agenda, but that generally “there’s an intense politicization of medical research to support an agenda that wants to save money by denying care to the elderly and sick. … The term ‘comparative effectiveness’ is code for giving care based on age and life expectancy of the patient.”
Supporters say that objection is off base, and that the research is designed to find the most effective treatment for every population.
But even the institute’s name bears the imprint of the red-hot controversy the subject fuels. During the health care reform debate, the very term “comparative effectiveness research” was abandoned in favor of “patient-centered outcomes research.” The latter has an explicit emphasis on the wishes of the patient, rather than hinting that the true goal is cheaper care.
And the ACA specifically states that PCORI will have no authority to mandate coverage or reimbursement, although it doesn’t forbid public or private payers from using the research to inform their coverage decisions, John McDonough, who worked as an aide to Sen. Ted Kennedy, reports in his book "Inside National Health Reform."
PCORI is led by Joe Selby, a widely respected former executive at Kaiser Permanente, who is well aware of the delicate political balance he has to strike.
“Everyone at PCORI, our board and our staff, recognizes the great need for research that compares different prevention, diagnosis or treatment options to see which ones work best for different people with a particular health problem,” Selby said in an emailed response to questions. “That’s at the heart of PCORI’s mission, to provide patients and those who care for them with the information they need to support health care choices.”
After the release of its draft agenda in January, PCORI will award 40 grants totaling $26 million that will fund methodological and agenda-setting research. Selby said PCORI will award another $100 million in primary research funds by the end of the year. The majority of funds will go to comparative effectiveness research studies, he said.
The ACA established a trust fund for PCORI, so it isn’t subject to the annual appropriations process, which provides a measure of security. It’s funded by the government but is independent of it, and so far, in the various debt reduction negotiations this year, it has not emerged as a target.
But laws can change, and even PCORI foes suspect the agency will tread lightly next year.
Robert Goldberg, vice president of The Center for Medicine in the Public Interest, believes that money being spent on PCORI would be better spent elsewhere. “I don’t think there’s any hard evidence that comparative effectiveness actually improves health at a reduced cost,” he said.
He co-authored a paper with John Vernon, an economist at the University of North Carolina, arguing that if a comparative effectiveness standard were built into the FDA’s premarket approval process, the impact on innovation would be severe. “To the extent that it’s just another link in the chain from the time a product is developed 'til the time it’s approved, it’s going to delay access and discourage innovation,” Goldberg says.
Harvard’s Avorn calls this “crying wolf,” because comparative effectiveness has never been a part of FDA review and including it is not in the cards.
And Goldberg acknowledges it is a purely hypothetical scenario — one that can’t be allowed to become reality.
Despite his suspicions, though, he thinks of PCORI as mostly harmless.
“I don’t see PCORI as a real threat. I think they’re more interested in shoving the money out in a neutral way not to upset anybody and see if they can build support for it after 2012,” Goldberg said. “Depending how the elections go, they could be an early target, so they’d like to build some consensus.”
Supporters agree on the last part — the need for consensus — and generally applaud the efforts to bring as many people to the table as possible. Consensus is needed, they believe, not just to keep political foes at bay, but also to advance the cause of comparative effectiveness research beyond the institute.
Large-scale comparative effectiveness studies are extremely expensive. Despite PCORI’s substantial funding, it will never be able to tackle all of the clinical issues that need to be studied.
The goal is to set up a framework that will allow others — insurance companies, research institutes and others — to build on the experience and carry out studies of their own, Dubois said.
Rita Redberg, a comparative effectiveness expert and professor at the University of California San Francisco, said she believes PCORI is doing great work under very trying circumstances.
“I think they’re going to move the needle in terms of what we need to know,” Redberg said. “They’re really doing great with the restrictions they have, but there are a lot of masters to please.”
According to a new report by University of Southern California researchers in the American Journal of Managed Care," "Though some doctors stick to a few highly advertised medications, only a few physicians prescribe them exclusively.”
No surprise there, but useful and timely as we enter into the New Year with the specter of government detailing (aka “academic detailing”) hovering over both patient choice and the overall ability of physicians to practice medicine as they see fit.
"Narrow prescribing patterns may be optimal when one drug is clearly superior to the others, or if all the drugs in the class act in a similar way."
“Superior” in individual patient performance (clinical performance) to be sure.
The researchers also noted “although most physicians have a favorite drug, they are not reluctant to try new therapies.”
Which begs the question – how and from whom are physicians to learn about new treatments?
A tough and important question for 2012. And it’s not, strictly speaking, academic.
Civilization is a limitless multiplication of unnecessary necessities. -- Mark Twain
According to the FDA’s Karen Mahoney, the recent draft guidance regarding Unsolicited Requests for Off-Label Information About Prescription Drugs and Medical Devices is "the first of multiple planned guidances that respond to testimony and comments from the Part 15 public hearing that FDA held in November 2009."
In the meantime, we’ll see if these promised “multiples” will reinvigorate the denizens of inertia who believe regulated industry needs to “wait” for further agency instruction before embracing a more holistic approach to social media.
Here is the source of the FDA quote:
FDA's Social-Media 'Guidelines' Befuddle Big Pharma
After Two-Year Wait, Guidance Addresses Only Off-Label Uses of Drugs
By: Rich Thomaselli
After a two-year wait that included the fanfare of a two-day public hearing, the Food and Drug Administration this week quietly announced new social-media marketing guidelines for pharmaceutical companies.
Well, sort of.
The FDA on Tuesday posted its "Guidance for Industry Responding to Unsolicited Requests for Off-Label Information About Prescription Drugs and Medical Devices" in the Federal Register, an under-the-radar move neither announced by press conference or press release. And the guidance seems to fall a bit short of what a legion of pharma-industry and healthcare-agency marketing execs anticipated.
"What everybody was expecting was actual guidelines around social media," said Jim Dayton, senior director of emerging media for Overland Park, Kan.-based InTouch Solutions, a pharma-centric digital-marketing agency. "I still think it's monumental," he added. "The FDA finally addressed the digital channel in a specific way by mentioning Twitter and YouTube in the document, and those have never been mentioned before. But this is an industry that wants specific instructions and rules, and that didn't happen here."
"We understand the level of interest and wanted to get out what we had available to provide guidance," said FDA spokeswoman Karen Mahoney, who added that this was just "the first of multiple planned guidances that respond to testimony and comments from the Part 15 public hearing that FDA held in November 2009."
The 15-page FDA guidance addresses only off-label information, laying out instructions for pharma companies on how to respond to consumers seeking information for a prescription drug other than what it is indicated for. That is, in a very rudimentary example, if a consumer asked a question on Facebook or called a pharma company's 800 number asking whether it was safe for men over the age of 50 to use Botox, a representative for Botox-maker Allergan could certainly answer the question. However, if the consumer asked whether the use of Botox also helped grow hair on balding men, the representative should refer the consumer to the Allergan website on use and safety information.
"Firms may choose to respond to unsolicited requests for information about off-label uses of their approved or cleared products in a manner other than that recommended in this draft guidance. Such activity would not constitute a per se violation of the law, but could potentially be introduced as evidence of a new intended use," the FDA wrote in its guidance.
"FDA recognizes that it can be in the best interest of public health for a firm to respond to unsolicited requests for information about off-label uses of the firm's products that are addressed to a public forum, as other participants in the forum who offer responses may not provide or have access to the most accurate and up-to-date information about the firm's products. … Statements that promote a drug or medical device for uses other than those approved or cleared by FDA may be used as evidence of a new intended use. Introducing a product into commerce for such a new intended use without FDA approval or clearance would, under these requirements, generally violate the law."
But for some, that still leaves things ….."ambiguous," said one pharma company digital director who asked not to be identified. "It leaves a lot open to interpretation."
Big Pharma, which spent $1 billion in online promotion last year and was expected to reach $1.52 billion in spending by 2014, has been somewhat inhibited by the lack of guidance and ambiguity on social-media use.
But that's the best the industry can expect right now, said Peter Pitts, former FDA associate commissioner and now the president of the Center for Medicine in the Public Interest. "What everybody was looking for was never going to happen. If you're waiting for divine guidance, you're still waiting," said Mr. Pitts.
Both Messrs. Pitts and Dayton said pharma should embrace this first leg of social-media guidelines. "On the face of it, you can look at it and say 'This is nothing,'" Mr. Pitts said. "In reality, I doubt there is ever going to be definitive rules for social media-marketing like there are for TV and print. And there's a reason for that. FDA has made it very clear they were not going to make platform-specific guidelines, like how to use Facebook, how to use Twitter, because social media evolves every day. How can you write guidelines for something that shifts in 90 days? Social media is unpredictable. If industry wants to use social media, they have to embrace ambiguity. The key element here is to use your judgment. If you wouldn't say it offline, don't say it online."
Added Mr. Dayton: "However ambiguous some might see this, I do not think this document should stop anybody from using social media for marketing purposes. We should use this document to show us how the FDA is thinking when it comes to social media. This is indicative of future guidelines around social media. It's going to have to be something we interpret, which is typical of a government agency. We're not ever going to be like Nike or Apple and say whatever we want in social media. The rules haven't changed for us -- it's the same rules this industry has for traditional media."
Mary Pendergast, former Deputy Commissioner) and an architect of FDA's call for emergency contraceptives during the Clinton administration) and I find ourselves in violent agreement over the outrageous decision of HHS Secretary Kathleen Sebelius to overrule the FDA on Plan B. Here are three links to our recent discussion (expertly moderated by Steve Usdin) on BioCentury This Week.
Part 1: Is there a Secret FDA in the Secretary's Office?
BioCentury This Week kicks off the election year dissecting the Obama administration's decision to overturn FDA's plan to authorize OTC access to the Plan B contraceptive to all women, regardless of age. Did HHS compromise FDA's scientific integrity to avoid a campaign fight over the morning-after pill?
Debating the public health and social consequences:
Jeanne Monahan of the Family Research Council
Kirsten Moore of the Reproductive Health Technologies Project
Cynthia Pearson of the National Women's Health Network
Analyzing the political calculus:
Mary Pendergast, former Deputy Commissioner and an architect of FDA's call for emergency contraceptives during the Clinton administration
Peter Pitts of the Center for Medicine in the Public Interest, and former Associate Commissioner at FDA during the Plan B debate in the Bush administration
Watch the Broadcast
8:30 - 9:00 a.m. EST
WUSA Channel 9
in Washington, D.C.
Watch on the Web
www.biocenturytv.com
Continuously available
starting at 9:00 a.m. EST
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.
