Latest Drugwonks' Blog
FDA's Endocrine and Metabolic Drugs Advisory Committee unanimously agreed that a retrospective analysis of 23 case studies provided substantial evidence to support approval of Orphan Europe's Carbaglu, but recommended collection of long-term safety data as the drug could be used for years as maintenance therapy.
Did somebody say precedent?
The Web site, “FDA Basics,” features short videos about the agency as well as conversations with FDA leaders.
Must see TV. Maybe it can hosted by Conan O’Brien.
The website is a good idea. A positive step in the right direction. If “the people” don’t know what the FDA does, it’s impossible to build a broader base of support. In that respect, it’s more than just a good idea – it’s a crucial one.
From: A Message from the Commissioner
Sent: Thursday, January 07, 2010 7:12 AM
To: FDA-Wide
Subject: A New Year
The beginning of a new year is a good time to take stock of what we have done over the past 12 months and where the next year will take us. Upon my arrival at FDA in May, I found myself distinctly impressed by this agency’s enormous impact, both as a regulator of so much of the American economy and as an organization upon which so many depend for the safe use of a wide array of products critical to their daily lives.
I recently observed to Secretary Sebelius that I have found FDA’s employees to be a wonderfully talented and dedicated group that I believed, if adequately resourced and supported, could solve virtually any problem that comes your way. The Center directors, ORA, and Commissioner’s staff offices recently shared with me their accomplishments for 2009, and it’s a remarkably impressive list of product reviews, inspections, enforcement activities, rulemaking, outreach to the public and those we regulate, reaction to crises and so many other activities that enable the agency to be an effective public health protector.
All of that was done as a cascade of new challenges were thrust upon us – H1N1 influenza, implementation of the new FDAAA and animal drug legislation, new food contamination and drug registration systems, and an entirely new Center to regulate tobacco for the first time in the nation’s history. I should also note that lurking out there are new requirements in the health care bill moving through Congress (e.g., “follow on” biologics and restaurant menu labeling).
Of course, we launched a number of new things ourselves – new foreign offices, a safe use initiative for drugs, a new food labeling effort, a reexamination of the process for reviewing medical device 510(k)s, new procedures for emergency response, a new policy with regard to antimicrobial resistance for animal drugs, and a rejuvenation and integration of the food safety program, to name just a few. New facilities also came on line in 2009, most notably the medical device office and laboratory complex at White Oak and the Bio-Imaging facility at NCTR.
For my part, I am proud of the new emphasis that Josh Sharfstein and I have placed on ensuring FDA’s reputation as a public health agency, as an organization more transparent to the outside world, and as a regulator intent on its scientific integrity and on enforcing the safety standards we have been charged with implementing.
All of the things that I have mentioned above will, of course, be a priority for 2010 as well. But I also intend to dedicate myself to giving you more and better tools to do your jobs. This will include seeking Administration support to improve our regulatory science, ensuring passage of the food safety legislation now before Congress, seeking new authorities to better regulate imports, and identifying changes in our medical device statute that are needed to ensure that program has 21st century capabilities. I also intend to urge the Administration and Congress to complete the long-awaited consolidation of our headquarters facilities at White Oak and College Park. And, of course, getting you the resources and staffing necessary to be successful will be a constant imperative, despite the demands to reduce Federal spending.
I have gone on long enough, even though I have barely touched upon the hundreds of discreet activities that FDA staff carry out every day. I will close simply by saying that I consider myself privileged to serve as your Commissioner in this great enterprise we are about. I pledge to you the same dedication that you have shown to the American people. I am proud to be associated with you and with the Food and Drug Administration, and that I hope the new year is as filled with accomplishment and progress as the old.
With all best wishes for a happy, healthy and productive new year.
Margaret A. Hamburg, M.D.
Commissioner of Food and Drugs
The Pink Sheet reports on a very urgent ethical question:
FDA's Endocrinologic and Metabolic Drugs Advisory Committee on Jan. 13 will consider whether Orphan Europe can use positive results from patient experience with carglumic acid to demonstrate efficacy of the firm's Carbaglu for treating hyperammonemia associated with NAGS deficiency, given the difficulty of conducting a clinical study in the orphan setting.
A controlled clinical trial in this target patient population "cannot be conducted because the disease has an extremely low incidence, it is life-threatening, severely symptomatic, and hyperammonemic decompensation leads to quick deleterious neurological/psychomotor consequences," Orphan Europe maintains in briefing material for the committee meeting.
A deficiency of N-acetyl-glutamate synthase is one of the rarest of the urea cycle disorders, which have an overall occurrence rate of approximately one per 30,000 live births. It results in hyperammonemia - high blood ammonia levels - that can lead to death or neurological impairment.
Instead of conducting a trial, the sponsor submitted a retrospective review of the effect of carglumic acid on both short-term and long-term plasma ammonia levels in 23 patients diagnosed with NAGS deficiency. Carbaglu is the pharmaceutical grade of carglumic acid, which has been used as a chemical grade product by clinicians. Also submitted in support of the NDA were interim data for three patients in an open-label, Phase II clinical trial of three days duration.
What is the response?
Let Arnold Relman and David Rothman (he who receives his dollars from George Soros) render judgment:
Relman: “But it’s just not a good idea for a profession that says it wants to be independent and trusted, a reliable source of information to the profession and the public about drugs, to take money from the drug company under any conditions.”
Rothman: I will also confess I am surprised Pfizer is doing it,” he added, “and I don’t know how many more times they will give money with no strings attached.” (As opposed to the tight strings attached to everything Soros gives to his sock puppets?)
So doctors who take unrestricted money for any reason under conditions from any company are inherently corrupt?
Too bad the NY TImes continues to give these extremists a platform -- without ask them to disclose their funding sources... as if that mattered.
www.nytimes.com/2010/01/11/business/11drug.html
Harvard Pilgrim Health Care Inc. has won a major grant from the Food and Drug Administration to build a system to monitor the safety of drugs and medical devices after they have gone on the market.
Pilgrim will use a five-year, $72 million grant to coordinate the efforts of 28 health care organizations around the country as they design ways to make use of information they already gather, in electronic medical records or claims data, to provide early warning of potential harm.
“The goal is to be able to do active surveillance, to look for signals that might indicate there is a safety problem and then to be able to follow up to determine whether there is a problem or not,’’ said Dr. Richard Platt, who will lead the effort. He is chairman of population medicine at Harvard Medical School and the Harvard Pilgrim Health Care Institute. “This is part of a major shift on FDA’s part from being reactive, waiting for physicians and patients to tell them about problems, rather than to be looking actively for problems.’’
"We do not support approaches, such as those suggested in the Senate's proposed Independent Payment Advisory Board, that rely too heavily on the Medicare program to achieve cost-containment objectives." So said AARP CEO Barry Rand in a letter sent to Senate Majority Leader Harry Reid and Speaker of the House Nancy Pelosi.
The provisions in the Senate bill task the 15-member board with reducing excess cost growth by recommending cuts in Part D premium subsidies paid by Medicare. Those recommendations mandate fast-track congressional action, required within two-and-a-half months unless the Senate has a three-fifths vote to repeal the board recommendations or any of the savings targets.
Rand continues, "Congress should not abdicate its role of determining benefits and cost-sharing for Medicare.”
Mr. Rand is right. One of the most frightening unintended consequences of such an advisory board is that they will make decisions based on cost rather than care. And it’s an inevitable consequence. Those who owe their jobs to the American voter must always be held ultimately responsible for government reimbursement decisions. And if you don't think this really makes a difference -- just ask the folks at NICE about Sutent.
Make patients' needs a higher priority
The Senate's vision for health care reform - the "Patient Protection and Affordable Care Act" - would create a "Patient-Centered Outcomes Research Institute." This institute would aim to compare the effectiveness of different treatments to "assist patients, clinicians, purchasers, and policy-makers in making informed health decisions." ("Purchasers" refers to public, private and employer-sponsored health insurance.)
As the founding editor of "The Patient - Patient-Centered Outcomes Research" - the first medical journal devoted exclusively to informing the health care system about patients' needs and wants - I have several reservations.
It is unclear how the institute will define, let alone meet, the often-competing interests of patients, clinicians, purchasers and policymakers. While clinicians, purchasers and policymakers have adequate means and resources for advancing their interests, it is questionable whether the patient's voice will be adequately represented in this process. Despite the many patient organizations representing specific diseases, only a few are active in research, and these may be tarnished through funding by pharmaceutical companies. Furthermore, unlike clinicians and purchasers who are bound together by their commercial and professional interests, representing the range of patient perspectives is difficult and expensive.
"Patient-Centered Outcomes Research Institute" sounds like a more politically palatable name for an "Institute for Comparative Effectiveness Research." Like other such agencies internationally, the bill gives the institute the power to determine the "appropriateness of medical treatments." International experience demonstrates that "appropriate" is defined as cost-effective or budget minimizing (although our senators have carefully avoided such terms). Also like its international counterparts, the institute would be placed at arm's reach from government, to ensure lawmakers have plausible deniability with regards to health care rationing.
Finally, similar institutes internationally have been criticized for promoting "one-size-fits-all" medicine by ignoring variations in needs and wants across patient populations. While the institute will consider "variations in patient subpopulations," it is unclear what characteristics will define such subpopulations.
A centralist and paternalistic approach is found throughout bill's description of the institute. Already a number of professional organizations have expressed concern that the institute would control the health care system by controlling the flow of information. Internationally, this occurs explicitly through limitations on the advertising of pharmaceuticals and implicitly by restricting funding of research. While the new institute will have a role in "the dissemination of research findings with respect to the relative health outcomes, clinical effectiveness, and appropriateness of the medical treatments," a truly patient-centered approach would also focus on the patient's perspective.
There is little federally funded research focused on the patient's perspective. Knowing more about what patients think might have resulted in less of an uproar over the recent changes to breast cancer screening recommendations, for example.
It is unclear how the findings of the institute will we implemented. Similar institutes overseas cannot have their decisions challenged in court. In trying to be everything to everybody, the institute will need to make trade-offs that will most likely promote clinician-centered or payer-centered outcomes research - ignoring the disparate needs of patients.
House and Senate negotiators should create a separate institute for the scientific study of the patient's perspective, an "Institute of Patients' Needs and Wants," to conduct and fund real patient-centered outcomes research. Such an institute would not only provide necessary oversight, but more importantly, the input of the patient's perspective and participation in health care decisions, from start to finish.
John F. P. Bridges is an assistant professor in the Department of Health Policy and Research at the Johns Hopkins Bloomberg School of Public Health and a senior fellow at the Center for Medicine in the Public Interest.
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.
