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For every 10 percent shaved off the life of patent protection, biotech investment declines by 10 percent. Over the next 15 years, the Obama deal would cut biotech investment, heavily concentrated in the Bay State, by 33 percent. Every biotech dollar invested has a ripple effect in terms of jobs, spending and investment in related businesses. Cut biotech spending by a third and the impact on economic growth will be magnified in reverse.
Then there is the human element. For cancer an increase in the number of biotech drugs iis associated with an increase in both the one-year and the five-year survival rate for all forms of the illness. For orphan disease, it has meant longer lives for people with lupus, cystic fibrosis, MS, Gaucher’s disease, HIV, etc. Less investment, fewer drugs, more deaths sooner. That’s one way to keep costs down and pay for the union tax break.
Finally, Obama wants to save a few million by cutting corners on drug safety. Current proposals require genetic companies to demonstrate patient safety by requiring appropriate and stringent clinical trials and testing. This is necessary because biologic drugs are created from living organisms such as proteins and carbohydrates, and are not as simple to replicate as traditional drugs like aspirin and antihistamines. Even changing the size of the molecule of the same protein can turn of biosimilar from avatar of health into an avalanche of deadly side effects. But Obama wants safety to meet budgetary, not scientific standards.
Having been rebuffed in his own committee on follow-on biologics, Mr. Waxman is trying to roll the President. That’s petty politics and bad public health policy.
These miracle medicines, called biologics, are complex molecules whose healing power has been brought to patients by dynamic biotechnology companies. Such drugs were once a rarity in the medical arsenal, but each day seems to bring new hope from new breakthrough biologics.
Now Congress must consider whether to authorize FDA to accept applications for follow-on versions of these path breaking medicines.
The stakes riding on the answer to this question are enormous, both for patients and for our economy, and the interest among our committee colleagues in this question is intense. One of our colleagues, Senator Clinton, has a proposal to allow FDA to approve follow-on biologics. I look forward to hearing her views on this question, and to receiving the testimony of the legislation’s co-sponsor, Senator Schumer.
Our committee should be guided by three basic principles.
First, we must be led by science. Acceptable legislation on follow-on biologics must not pre-judge science, but should enable the FDA to make the best decisions based on the most complete science reasonably available.
Second, protecting patient safety is essential. Congress must make certain that any drug given to patients – whether a conventional drug, an innovative biologic, or a follow-on product – is safe and effective.
Third, innovation must be valued and promoted. Just as it is essential to help patients afford the medicines of today, so too it is vital to provide incentives for the innovations that will bring the medical miracles of tomorrow.
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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.
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.
