Medicine By Mandarins
Gottlieb, Scott
"Medicine By Mandarins" New drugs are too important to be tested in the closet BY SCOTT GOTTLIEB Fight years ago, when the biotechnology company Bio-4 gen had to make a change in a new drug that...
...Fabry's is a rare, painful, and fatal genetic disorder, in which deficiency of an enzyme causes fat to build up in different organs...
...Biogen had found a better way...
...Opacity was the issue in the early 1990s, when a group of FDA statisticians arbitrarily tightened the metrics for assessing new antibiotics...
...Lift up on their "private" counsel, and regulators will have to show some restraint before ratcheting up the metrics...
...In September, Transkaryotic Therapies announced that it had "agreed to disagree" with the FDA over the clinical data for Replagal, a new drug for patients with Fabry's disease...
...Corporate naivete...
...Name Address City State Apt...
...Signed (Your order will not be processed without signature and date of birth) Birth date —Ma/Day/Year OFFER EXPIRES 2/28/03 • OFFER NOT AVAILABLE TO MINORS • OFFER GOOD ONLY IN THE USA NOVEMBER/DECEMBER 2002 • THE AMERICAN SPECTATOR 23...
...it's a sure bet these decisions were privately coerced during an off-the-record chat with an FDA reviewer...
...The company relied on reduction in pain as a benchmark for success...
...Add an additional $4.95...
...er# desk :At the low low price of ~ ~`' '~ + , v t bu fr ˆ & > $7m soon&t- ~ 'ff a"tp 4er citarr compan yy S nce ~2t ltaya ertioye air i e =?N 19 tlxm ken attictes...
...Only the largest companies are inclined to transcribe their notes from their off-the-record conversations with FDA reviewers,play them back to the agency, and try to hold regulators to their words...
...And the benchmarks for success are similarly moving targets—FDA reviewers rarely commit to explicitly stated endpoints...
...citizens...
...As a result, there's brooding mistrust in the drug industry of anything the FDA says and growing uncertainty over which voice the agency is really speaking through...
...Biogen met the standards, the agency stuck to its promise, and thousands of patients got the new treatment without delay...
...For President Bush's newly minted FDA commissioner, Mark McClellan, transparency and consistency in decision making should be job one...
...Most clinical experience with the drug was accumulated from batches made using an older process...
...ANNOUNCING THE 2003 PHILLIPS FOUNDATION JOURNALISM FELLOWSHIPS WORKING JOURNALISTS ELIGIBLE FOR $50,000 If you are a working print journalist with less than five years of professional experience, a unique opportunity awaits—the chance to apply for a grant to complete a one-year project of your choosing, focusing on journalism supportive of American culture and a free society...
...Every time a drug company The FDA's "whispered guidance" gives unfair advantage to the big pharmaceutical companies...
...Only ©2002 Thompson Cigar & Co., Inc...
...JOHN FARLEY THE PHILLIPS FOUNDATION 7811 Montrose Rd., Potomac, MD 20854 Telephone 301-340-2100 E-mail: jfarley@phillips.com www.thephillipsfoundation.org Deadline: March 1, 2003 My Introductory offer r~1rss~ I)N to New Cigar utomers...
...Simply recruiting enough patients with the relatively rare but often deadly infection would have taken five years, even with participants from Europe added in...
...ImClone, like other hap-less biotech companies before it, misread the agency's body language, believing that regulators had blessed the design of its clinical trial...
...Applications must be postmarked by March 1, 2003...
...The Foundation's fellowship program serves to provide support for journalists who share the Foundation's mission: to advance constitutional principles, a democratic society and a vibrant free enterprise system...
...Drug makers have little choice but to err on the side of caution...
...Regulators say they provide a valuable service by privately counseling drug companies over the design of their clinical trials...
...Dept...
...It°s the complete package for the snooker: twenty-five Thompson handmade, imported cigars, a dependable windproof lighter (may vary), and a•solidly constructed cedar-All this for L linen divided humidor whose French quadrant hinges, humidification systemand (regularly a $79 value),: ; >ty romefel:•make it a veritable vault to protect our.pur .This exquisitely fashioned k 'lx lldltr t$, jarldsame enough to grace any mo...
...Putting the FDA's advice into the public record would force the agency to adhere to a uniform standard...
...The FDA doesn't disclose whispered guidance, and neither do scared companies...
...But since the agency is never held to its word, they can often do more harm than good...
...Those that misread these tea leaves end up as biotech blowups...
...The Phillips Foundation offers $50,000 full-time and $25,000 part-time journalism fellowships...
...Scott Gottlieb, a practicing physician, is author of the Gilder Biotech Report and a resident fellow at the American Enterprise Institute...
...Date By responding to this offer and signing below, I certify that I am a smoker, 21 years of age or older...
...T4029 Fax: 813-882-4605 Est...
...By speaking with so many tongues, the FDA creates an endlessly shifting playing field, favoring those who can interpret its proprietary language, especially the largest drug companies that take multiple shots on goal...
...1915 ' America's Oldest Mail Order Cigar Company P.O...
...Wyeth similarly abandoned testing of an antibiotic for serious infections, when faced with the same statistical tinkering...
...Bristol Myers Squibb was developing a treatment for bacterial meningitis when the FDA moved the goalposts...
...Applicants must be U.S...
...Applications are now being accepted for 2003...
...Because of their long history with the FDA, "Big Pharmas" have the competitive advantage of learning the agency's peculiar lexicon...
...Founded in 1990, the Phillips Foundation is a non-profit organization whose purpose is to advance the cause of objective journalism...
...Opacity was also at issue in thenow-celebrated ImClone debacle, in which the company was blind-sided by an FDA decision to refuse even to consider ImClone's application to market a cancer drug, Erbitux...
...Print all digits) Exp...
...MEDICINE BY MANDARINS New drugs are too important to be tested in the closet BY SCOTT GOTTLIEB F ight years ago, when the biotechnology company Bio-4 gen had to make a change in a new drug that it was developing for the treatment of multiple sclerosis, the company literally bet the farm that the Food and Drug Administration would stick to its word...
...it was tripped up when the FDA said that these endpoints wouldn't be good enough...
...Benchmarks on public display would let doctors and patients judge for themselves whether the approval process is winding on too long...
...The winners will be announced at an awards dinner in Washington in the Spring...
...At issue was the way the drug was manufactured...
...Another biotech blowup in the making, to haunt investors, drug makers, and patients who depend on their products...
...But you'll never hear that...
...Artificial or not, that knowledge gives Big Pharmas a favorable position and becomes a barrier to entry for entrepreneurs...
...Since FDA officials have become accustomed to giving companies ad-lib advice, it's likely that both versions are true...
...Whispered guidance" makes FDA benchmarks mutable, with companies perpetually adjusting the rigor of the drug trials to meet shifting standards conveyed in winks and nods...
...Facing mounting losses, Bristol dropped the program...
...Things have changed since then...
...The setback for Replagal is the result of an agency continuously at pains to exercise its policy prerogatives...
...Vagueness and unpredictability lie at the center of the agency's current woes...
...the FDA says that never happened...
...Charge to my ^ VISA [j MasterCard ^ AmExpress I Discover Printed name as it appears on credit card Credit Card No...
...Regulatory murk carries a high price...
...Small and medium-sized companies worry that such tactics would just tick the mandarins off—and they're right...
...Florida residents add 6% sales tax + appropriate county tax...
...Companies handicap their prospects for clearing new drugs according to which regulator they pull from the pool...
...22 THE AMERICAN SPECTATOR • NOVEMBER/DECEMBER 2002 announces that it has doubled the size of a clinical trial or "pulled a drug application pending more data...
...The fellowships will begin on September 1, 2003...
...Decisions that significantly delay the approval of new drugs and increase the cost of development—for instance, asking companies to double the number of patients on which they need to test new drugs—typically are communicated in the private musings of individual regulators...
...Replagal and Frabrazyme, made by the biotechnology company Genzyme, have already won approval from European regulators...
...They maintain meticulous data-bases that handicap individual regulators...
...Small, innovative biotech companies end up forced into the arms of their larger competitors, simply to have some assurance of navigating the regulatory maze...
...Zip Daytime phone # ( ^ Payment Enclosed...
...The new regimen would have required testing the drug on 700 patients—the largest meningitis trial ever...
...Insiders say that Transkaryotic's management misinterpreted conflicting FDA signals over the suit-ability of some of their data...
...Box 31274 • Tampa, FL 33631-3274 Send me one Classic Combo For $29.95 + $4.95 shipping - (All shipments to AK, HI, Guam, Virgin Islands and Puerto Rico must go priority mail...
...Today, individual regulators at the FDA exert increasing control, making freelance policy decisions, off-the-cuff requests, and promises they have no inclination to keep...
...The seemingly simple change went unnoticed until it began having a huge impact on drug makers, doubling the size and the cost of clinical trials—and ultimately stifling development of promising drugs...
...For applications and more information, visit our website or write: MR...
...One executive at a small biotech company told me recently that her FDA reviewer suggested conducting just a single phase 3 trial—instead of the usual two—for a promising new drug, as long as the results were "good enough?' No one in the company is sure what "good enough" means, but they're following the regulatory nod and conducting a single trial...
...Not surprisingly, companies often misread these signals...
...Both have been under review by our FDA for more than two years...
...FDA officials said that, provided the new process met certain criteria, the agency would still approve the new drug, without forcing the company to start testing all over again...
Vol. 35 • November 2002 • No. 6
