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Abigail Alliance for Better Access to Developmental Drugs. DECELERATED APPROVAL November 8, 2005 Moving Backward for Cancer Patients. March 12, 2003 ODAC Meeting Opening Comments by FDA Dr. Richard Pazdur Accelerated Approval.
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November 8, 2005
Moving Backward for
“Accelerated approvals have been granted with the trial design using single arm trials in refractory populations as stated previously. These trials obviously allow more rapid trial completion and hence expedite drugs to patients with life-threatening diseases.”
“An alternative trial design uses arandomized trialallowing accelerated approval on the basis of aninterim analysisof surrogate endpoints, for example, response rate or time to progression.”
“Randomized trials also may optimize the evaluation of novel cytostatic agents by allowing an assessment of slowing or retarding or preventing tumor progression. This may simply not be possible with single arm trials.”
“Obviously randomized trials are more expensive than single arm trials and take more time. “
“Survival analysis can be complicated and confounded by cross over and subsequent therapy.”
“The mandatory confirmatory trials to demonstrate clinical benefits are equally important as the initial trials demonstrating an effect on a surrogate endpoint leading to that drugs approval.”
March 12, 2003 ODAC MeetingOpening Comments by FDADr. Richard PazdurThe Patients Will Have to WaitDo You Want Your Drug Approved orNot?
“Hence confirmatory trials must be an inherent and integral part of a comprehensive drug development plan and drug development strategy. “
Accelerated Approval ~ Regular Approval
A Clearly Effective Well-Targeted Drug for Myelodysplastic Syndrome – An Almost Universally Fatal Disease – Based on Two Single-Arm, Highly Ethical Phase II Clinical Trials
“On several occasions, as will be mentioned by the FDA reviewer, we have recommended to the sponsor before they began the study, that we look at randomized studies of this drug in MDS to have a better understanding of the disease in relationship either to other therapies or the natural history of the disease.”
Celgene Keeps Its Own CounselProceeds With An Ethical Single-Arm, Phase II Registration Trial Single-Arm Trial Proves Expected An Undeniable Efficacy of the Drug in an Identifiable Subset of MDS Patients
“I want to bring people back to the kind of regulations, and there is a mantra, adequate and well-controlled trials, adequate and well-controlled trials, adequate and well-controlled trials. I am mentioning that three times, because I think that is at the heart of the question here.”
“And why you chose not to do a Phase III trial when you were asked to do that?”
[randomized, placebo-controlled trial]
“We are going to go to Phase III. We are going to be doing a placebo-controlled trial. I have to say that in discussing that trial with the investigators,there is actually reluctance to put patients on placebo for very long based on the benefit that has been seen here.”
“The patients who receive placebo, receive that for 4 months. If they are not responding, and we think that essentially, none of them are likely to respond from what we know, then, they will have the opportunity to go on to lenalidomide and continue on that as long as that seems to be benefiting them.”
The Outcome for RevlimidOn October 3, 2005 FDA Decided to Extend Its Review Timeframe for Three Months to Review the Risk Management Plan Already Deemed Acceptable by ODACThis Safe, Effective and Much Needed Drug Remains Unapproved
Patient Protection?The FDA is Asking for An Unethical, Unnecessary Randomized TrialWhileCelgene Negotiates for Ethical Treatment of Patients
We Have A ProblemThe FDA’s Decelerated Approval Initiative is an Extreme Case of Form Over SubstanceWhere the Substance at IssueIs Life Itself
Banish Mantras – They are Not the Stuff of Open Minds
Reactivate Accelerated Approval and Find Ways to Accelerate It Further