This is a hard blog to write, but I’m giving it my best.
The last article in my three part NYT series is concerning Dr. Richard Pazdur, the F.D.A. oncology chief. Dr. Pazdur lost his wife to cancer in November. My sincere condolences, Dr. Pazdur.
When you lose someone close, a new normal for your life begins. The circumstances surrounding their death, whether tragic accident or disease has a lasting affecting on those that survive. Dr. Pazdur is no different. What is different about Dr. Pazdur is that he’s the chief of oncology for the F.D.A. The buck stops with him. He has supreme veto power. For instance, in 2014, he authorized the approval of an ovarian cancer drug that the expert advisory panel voted against. It wasn’t even close, 11 to 2 AGAINST approval, yet Dr. Pazdur vetoed the panel and it was approved.
So the question that hits everyone’s mind: Was there a conflict of interest and did Dr. Pazdur have decision making incapacitation? Is he fit to lead the oncology section of the F.D.A. when his wife was suffering from cancer? Two main points to consider:
Those suffering from any disease would like to light a fire under the F.D.A. giving faster approvals for the new, and even older generic drugs.
Thorough study of drugs in Phase III / IV prevent major catastrophes like that of Thalidomide.
This is an even argument, one that has a delicate balance, BUT> can be stabilized.
What can’t be handled in this balance is the economics of drug approval and the extreme costs of these newer therapeutics, blogged previously this year.
The magnitude of clinical benefit in terms of symptom alleviation, mortality reduction and improvement in quality of life are measured in a standardized way, per unit of cost of the new treatment.
Regulators like Dr. Pazdur should not and cannot subvert health economic imperatives that focus on achieving similar or better cost-effectiveness between the new and older treatment options.
Further, there are mechanisms for the “Hail-Mary” or formally called “Expanded Access” that really is the compassionate use clause. This is the safety net (no pun intended) for those that don’t qualify for inclusion into an appropriate, in progress, Phase III clinical trial.
Yes, even after a patient doesn’t qualify for a Phase III trial, they can still gain access to a novel therapeutic even if that therapeutic is in the earlier Phase II of the approval process.
And here’s the kicker, that compassionate use case is NOT included in any of the trial data, regardless of outcome. Any effect the new therapeutic has will not be considered during the drug approval process. That makes BigPharma happy. Who then regulates “Expanded Access”… the patient’s physician, under the approval of an Institutional Review Board (IRB).
Doesn’t that sound like an effective way to balance? There are excellent, time proven, checks and balances that incorporate individual health, population safety and economics….Further, there’s an excellent mechanism to help those that don’t have the time, literarily, to wait for the proper approvals.
It seems to me that the system is NOT broke and frankly, in my humble opinion, Dr. Pazdur exceeded his powers at least once due to understandable duress. I sincerely hope that is the only time.