FDA Reform in the New Congress: A Death Sentence for Orphan Drugs?

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  • 02/28/2007
I received this email from a friend of mine...

"I saw Dr. Scott Gottlieb again today at a meeting of the Food and Drug Law Institute (FDLI). The topic was "Whose Life is it anyway?" and they talked about the Abigail Alliance's lawsuit for earlier access to experimental drugs. Scott gave an appalling example of regulatory creep where the clinical trials for a drug for Hunter's syndrome (one of the mucopolysaccharide diseases) required 100 patients and randomized controlled trial with endpoints being abilty to walk and abilty to breathe! Since it is very rare, he said that the trial likely had to enroll a portion of the world's population in the trial to meet the numbers! He contrasted this with the agency's approval of Cerezyme last decade with about 20 patients! I left the meeting feeling very passionate about the need to do something about regulatory creep and bring more rational clinical trial design to the agency - all of which Scott espoused."

I hope it is not too late...

This latest outrage is just a taste of what's to come under Kennedy-Enzi, Dingell, Stupak, Markey, Waxman, Grassley, Graham, IOM and the coverage thereof of 12 liver problems regarding Ketek hath wrought.... slower cures, fewer drugs, Steve Nissen's god-posturing on cardiovascular problems associated with ADHD drugs,claims that schizophrenia drugs are the next Ketek (meaning the next show trials, which means the next decline in use of drugs for mental illness which will mean another spike in suicides)..

Do any of these preening posturing types care about those who are dying? No, they are too busy trying to explain how watered down biologic standards will produce lifesaving medicines while slowing down orphan drug approvals won't. There are few in official Washington with any passion on this issue. Janet Woodcock of the FDA, Greg Simon of Faster Cures, Frank Burroughs of the Abigail Alliance, Ellen Sigal of Friends of Cancer...The rest could just as well be names drawn from the DC phone directory or in the case of NORD, just a big fat obstacle to change. In fact, NORD should stand for No Orphan Research and Development....

Sorry, just a bad morning.

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.

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