Latest Drugwonks' Blog
FDA is predicting a sharp drop-off in fee-paying drug and biologic applications in the current fiscal year.
In a Federal Register notice announcing the fiscal 2011 PDUFA fee rates for applications, establishments and products, FDA estimates it will receive 117.5 fee-paying full application equivalents in fiscal 2010. This figure represents a 16.3 percent drop from the 2009 level of 140.3 FAEs.
(The 2010 projection of 117.5 FAEs is based upon receipt of 88.125 fee-paying FAEs through the first nine months of the fiscal year. On average, 25 percent of the applications submitted each year come in the final three months, the agency says, citing data from the last 10 fiscal years.)
That means 2010 would mark the lowest number of applications submitted since 1995, when 112.5 FAEs were received by the agency. The 1995 figure, however, did not include pediatric supplements, which were exempt from application fees until 2002. When pediatric supplements are factored in, 1994 was the next lowest year, with 115.6.
Why? It’s largely due to a 25 percent decline in applications requiring clinical data. FDA had received 59 such applications through June 30, of which 17 were either exempt or fees were waived, bringing the total number of fee-paying applications to 42. In contrast, during the first nine months of fiscal 2009 the agency received 88.75 applications requiring clinical data, 32.75 of which were fee-exempt or waived, for a total of 56 fee-paying applications.
The total number of fee-paying applications not requiring clinical data declined 23 percent through the first nine months, from 11 last year to 8.5, while the number of fee-paying supplements requiring clinical data fell 5 percent, from 39 last year to 37. The number of fee-paying applications withdrawn or refused to file held steady at .625.
PDUFA implications? Sure. But the pure decline speaks to a much more troubling issue: a parse pipeline. Now more than ever we need a well-funded Reagan/Udall Center assist us all down the Critical Path.
(Hat tip to Sweet Sue Sutter @ the Pink Sheet.)
Medical journals are full of potboilers writen by hack writers who today throw together reports designed to fit the media's appetite for stories fitting the anti-Big Pharma narrative...
Case in point:
Outcome Reporting Among Drug Trials Registered in ClinicalTrials.gov
- Florence T. Bourgeois, MD, MPH;
- Srinivas Murthy, MD; and
- Kenneth D. Mandl, MD, MPH
Can you guess what the conclusion of the study was from the following headline?
Review Suggests Bias in Drug Study Reporting
Industry-funded trials more likely to have positive findings than other studies, analysis shows
www.businessweek.com/lifestyle/content/healthday/641567.htmlYou can spend your own $15 to get this potboiler on line or you can read the juicy parts of the hatchet job here.. You just have to suffer through my commentary.
"Results were considered favorable if they were statistically significant (based on P values or CIs) and supported the efficacy or safety of the test drug or not favorable if they were not statistically significant for the efficacy or safety of the test drug (25). For noninferiority trials, if the test drug was equal to the comparison drug, the results were also classified as favorable."
An industry sponsored study showing any benefit even if barely statistically signiifcant is considered positive. An industry sponsored study showing no difference in outcome in treatment compared to another drug or a placebo... that's also called a positive study.
There is also these important findings:
"Trials funded by industry were more likely to be phase 3 or 4 trials (88.7%; P < 0.001 across groups), to use an active comparator in controlled trials (36.8%; P = 0.010 across groups), to be multicenter (89.0%; P < 0.001 across groups), and to enroll more participants (median sample size, 306 participants; P < 0.001 across groups). Government-funded trials were most likely to be placebo-controlled (56.2%), whereas trials funded by nonprofit or nonfederal sources were least likely to be multicenter (24.6%) and tended to have the smallest sample size (median, 50 participants). Industry-funded trials were also most successful at enrolling the anticipated number of participants, with 84.9% of trials enrolling at least 75% of the planned number of participants (P < 0.001 across groups)"
In otherwords, drug companies did more post market studies (increasingly required) and confirmatory trials (always required) that were more diverse and larger. That explains in part the higher percentage of trials showing statistically significant efficacy. Smaller studies that are underfunded and underpowered -- and less likely to enroll the number of patients regarded to achieve a level of confidence that results are reliable: More likely early phase studies looking at other endpoints. No wonder industry sponsored trials are more likely to be "positive." And just to be sure, the researchers toss treatment toss-ups into the positive category. Nothing like creating your own standards. I wonder how many product managers would regard a no-difference result to be "positive."
So the authors twist the obvious into a conspiracy about how industry funding deliberately puts a happy face on otherwise lousy results... But in the world of medical publishing, skewing data to stick to pharma is, dare I say, a positive.
So do the headlines about the study, which I bet few reporters even read.
The Food and Drug Administration proposed shoring up medical-device approval rules that have been criticized as lax and inconsistent by consumer advocates and the agency itself.
The FDA aims to better define what devices can use an approval pathway known as 510(k), under which companies can get an accelerated decision on whether they can market a new product if they can show it is similar to an already approved device. The proposals, which will be open for public comment, will be closely watched by the device industry because more-stringent rules would raise development costs.
The FDA's top device regulator, Jeffrey Shuren, rejected suggestions that the changes would result in fewer devices being eligible for 510(k) approval or raise the need in general for extra medical studies. He said clarity was the main point of the effort.
The FDA began reviewing its medical-device regulations in September after issuing a self-critical report that said top regulators bowed to outside political pressure when approving a ReGen Biologics Inc. knee device in 2008. Articles in The Wall Street Journal had pointed to the political pressure and other irregularities in the ReGen case. ReGen said its device was safe and effective and defended its activities before the FDA.
"There is widespread recognition that there's significant room for improvement in the way we operate," said Dr. Shuren, who directs the FDA Center for Devices and Radiological Health.
The FDA said it wants to develop guidance clarifying when older devices shouldn't be used as a benchmark, or "predicate," for comparison to a new product. The agency is still working on the guidelines, but said there appears to be confusion about the rules.
The agency also proposed creating a subset of Class II devices for which clinical or manufacturing data would be needed to bolster the case that they are substantially equivalent to an already approved product. Class II devices are generally considered to carry moderate risk to patients. Dr. Shuren said drug-infusion pumps, ubiquitous hospital products which the FDA is seeking to improve amid a history of problems, fall into this new "Class IIb" category.
Pump maker Baxter International Inc. is in the process of pulling 200,000 pumps off the U.S. market after the FDA decided it was moving too slowly to fix the devices, whose flaws have included failure to deliver the right amount of medicine.
The device industry's trade group said the proposals included a number of steps that would increase the consistency and predictability of the approval process, but as a whole threatened "significant disruption." Among the industry's complaints are an agency plan to include detailed information about new products, such as design schematics, in a public database. That would give foreign competitors access to proprietary information, potentially hurting U.S. manufacturers, said Stephen J. Ubl, president and chief executive of the Advanced Medical Technology Association.
According to a poll of 802 registered voters by the Pew Charitable Trust's Prescription Project, more than three out of four are confident that prescription drugs made in the USA are free from contamination. However, fewer than one in 10 feel confident about medications made in India or China.
But what “made” represents can be misleading. On the one hand it means “manufactured.” On the other it means “with ingredients from.” And the two aren’t the same thing.
This is an important distinction for a number of reasons – especially when you’re asking the question to a general audience. When you ask someone about their feelings about where a drug is “made,” they are going to assume that “made” means “manufactured.” And while that’s important – it’s only part of the story.
An estimated 80% of the substances used to make or package drugs sold in the United States are made in other countries – and increasingly those “other countries” are India and China. Globally, in 2007, 68% of ingredients of drugs sold worldwide came from India or China, vs. 49% in 2004.
As far as products that are manufactured abroad, it’s important for the American public to understand that the exact same GMP’s are required as a plant in the Lower 48. The Pew poll didn’t share that information. It’s a game changer.
More important is the issue of ingredients sourced from foreign nations. Heparin comes to mind. (In 2007 and 2008, more than 100 patients in the USA died after taking heparin made with a contaminated active ingredient from China.) Attention must be paid and enhanced oversight is essential.
From the legislative standpoint is the Drug Safety and Accountability Act of 2010, to be introduced today by Senator Michael Bennet (D, CO). The bill would empower the FDA to order recalls of unsafe batches of drugs. This recognizes the reality that the concept of “total recall” of a drug is a very 20th century concept.
The "study" is really a restatement of who will get covered when under Obamacare. It duly notes that certain preventive services will not require a copay or deductible and that all health plans will be required to cover pregnancy and maternal care.
The Commonwealth Fund report fails to consider the added cost of buying coverage for such services and ignores the fact that there out of pocket costs associated with maternal care under every health plan. It also fails to note that obtaining ob-gyn coverage under Medicaid is increasingly difficult. In fact, ob-gyn services are hard to find under any circumstances. Just wait till twice as many patients whose care will be reimbursed as one third private insurance rates flood the system.
The report conveys the impression that all these services will be free and widely available. And it does not account for any increase in insurance premiums or efforts to limits coverage of new services to control costs.
tinyurl.com/26wo4br
An FDA advisory committee’s 12-1 vote to remove Avastin’s breast cancer indication is unloved - -and misunderstood – by Senator David Vitter (R, LA) who called the decision “essentially government rationing.”
“I shudder at the thought of a government panel assigning a value to a day of a person’s life,” Vitter said in a statement. “It is sickening to think that care would be withheld from a patient simply because their life is not deemed valuable enough.” In a letter to the FDA cancer division leader, Richard Pazdur, Vitter said the committee’s vote appeared to be based on cost effectiveness, not safety issues.
Not so.
New studies presented to the panel showed more side effects among women being treated with Avastin and no overall survival benefit, though they did show women taking the drug had an extra month to 2.9 months of progression-free survival.
Whether or not you agree with the panel’s viewpoint on the clinical efficacy – they can’t be dinged by making a decision based on cost. That’s just unfair.
While the fear of cost-based care trumping patient-centric medicine is real – the Avastin issue is one of data.
Senator Vitter’s clarion call is accurate – we need to be vigilant to call “comparative effectiveness” what it is – rationing. But this is not the right instance to do so.
A women's health advocacy group that hopes to meet with FDA within the next year to explore potentially switching oral contraceptives to OTC may be hard-pressed to find a pharmaceutical company interested in sponsoring such a venture.
The Oral Contraceptives Over-the-Counter Working Group, coordinated by Ibis Reproductive Health, is "interested in moving oral contraceptives over the counter as a way to increase access to" the drugs, "especially for women who now face barriers to access," such as needing a prescription, said Daniel Grossman, leader of the working group and senior associate at Ibis.
"We are currently in the process of talking with a number of potential partners about what would be needed for a switch, and are hoping to meet with FDA in the next few months to discuss the regulatory pathway," said Grossman, an obstetrician-gynecologist.
One aspect the group needs to explore is finding a drug firm as a partner to sponsor a potential switch application.
Grossman said the working group is not interested in sponsoring a switch application or filing a citizen petition to switch the category.
The Pink Sheet reports that pharmacist groups asked FDA to create a few common templates for risk evaluation and mitigation strategies so they can better anticipate and implement the programs into their practices.
FDA and industry groups spoke in favor of REMS templates July 27, the first day of a two-day agency workshop gathering comments on the program. FDA expects to use the comments to help formulate more guidance for REMS on drugs.
Janet Woodcock said an effort will begin in the fall to develop a standard REMS system.
"Each program is proposed by the drug sponsor and then it's negotiated between the sponsor and the FDA and is uniquely tailored to the risks of that particular drug," Woodcock said. "So we don't have the kind of standardization and consistency of the programs that would be ideal."
Additional REMS changes already have been proposed, including allowing medication guides to be developed outside the REMS process.
Poll shows opposition to health care overhaul declining
Washington Post Staff Writer
Thursday, July 29, 2010; 12:02 AM
Opposition to the landmark health care overhaul declined over the past month, to 35 percent from 41 percent, according to the latest results of a tracking poll, reported Thursday.
Support For Health Law Remains Steady While Opposition Drops
The percentage of people who view the new health bill unfavorably dropped 6 points to 35 percent in the past month, but that has not translated into a significant increase of supporters, according to the July tracking poll from the Kaiser Family Foundation. Overall support remained stable since the June survey, with about half the public expressing a favorable view of the overhaul, the poll found.www.kaiserhealthnews.org/Stories/2010/July/29/health-reform-tracking-poll.aspx
If you find the two ledes strikingly, uh, similar, don't worry. It's not plagiarism or even laziness on the part of the WaPo. It's merely a product of the Kaiser Health News effort to "provide new opportunities for health care journalists to produce in-depth work and a new vehicle to distribute it through collaborations with major news organizations and on this Web site." You see, it's really a jobs program first and a pass through mechanism of pro-government run health care reform "news" and surveys second.
Kaiser Health News, which partners with USA Today, NPR, the New Republic along with many fair-minded types who were on the JournoList before it was circumscribed down to the Cabalist, is a venture of the Kaiser Family Foundation. The foundation provides " strategic guidance". That direction consists largely of ignoring original research critical of health care reform in its current iteration and a slavish devotion to recycling the less than balanced or nuanced coverage of the fight over the implementation of the law. Some guidance.
Case in point is the echo chamber KHN creates by paying for polls and writing articles about the polls that are then essentially reprinted by WaPo and then blogged on by the Journolistas like Ezra Klein, Jonathan Cohn, Kate Steadman, etc. It would be sinister if it weren't so banal and boring...
The KHN and it's effort to consolidate control coverage of health care reporting and the shaping of public opinion about health care should not be set aside lightly. It should be thrown away with great force...
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.
