Dr. Avorn:
I appreciate you taking the time to respond to my post and email. I will publish your response on my blog without editorializing on my part.
Your analysis did nothing to help patients.. you only raised alarms about conducting smaller studies not randomized to your standards. That's an old saw used by you and others -- along with your assertion that unless you understand the mechanism of action endpoints, even if met, really don't matter. Your assertion that cancer patients might suffer more is a counterfactual argument that frankly is a function of individual response to medicines that, absent biomarkers or algorithms derived from patient level data, cannot be tested. What we do know is that life expectancy is higher from orphan drugs so the fact that you want longer and more expensive clinical trials for orphan drugs translates into you being willing to let sick kids die. And your agenda is not secret: you believe most drugs should not be marketed unless they meet your standards.
Regarding CER, you claim RCT is the gold standard for demonstrating effectiveness. Yet when ALLHAT was all but eviscerated by ACCOMPLISH and other studies, I didn't see you call for a change in clinical practice.
The evidence gathered from smaller trials is not "less valid." Such data does not comport with your cultural and ideological bias against the commercialization of medical information. I have a different bias. And I am not on the Right or the Left when it comes to science.
Finally, regarding BiDil, you should read your own work and what FDA officials have written in response. (See Annals in Internal Medicine, Jan, 2007 vol146 pages 57-62) You did object to it's approval on grounds of political correctness and the mechanism of action argument. In a perfect world we would know the MOA for drugs and diseases. But your view of how to evaluate drugs, RCTs above everything else, will not bring that about.
Best wishes,
Bob Goldberg
I appreciate you taking the time to respond to my post and email. I will publish your response on my blog without editorializing on my part.
Your analysis did nothing to help patients.. you only raised alarms about conducting smaller studies not randomized to your standards. That's an old saw used by you and others -- along with your assertion that unless you understand the mechanism of action endpoints, even if met, really don't matter. Your assertion that cancer patients might suffer more is a counterfactual argument that frankly is a function of individual response to medicines that, absent biomarkers or algorithms derived from patient level data, cannot be tested. What we do know is that life expectancy is higher from orphan drugs so the fact that you want longer and more expensive clinical trials for orphan drugs translates into you being willing to let sick kids die. And your agenda is not secret: you believe most drugs should not be marketed unless they meet your standards.
Regarding CER, you claim RCT is the gold standard for demonstrating effectiveness. Yet when ALLHAT was all but eviscerated by ACCOMPLISH and other studies, I didn't see you call for a change in clinical practice.
The evidence gathered from smaller trials is not "less valid." Such data does not comport with your cultural and ideological bias against the commercialization of medical information. I have a different bias. And I am not on the Right or the Left when it comes to science.
Finally, regarding BiDil, you should read your own work and what FDA officials have written in response. (See Annals in Internal Medicine, Jan, 2007 vol146 pages 57-62) You did object to it's approval on grounds of political correctness and the mechanism of action argument. In a perfect world we would know the MOA for drugs and diseases. But your view of how to evaluate drugs, RCTs above everything else, will not bring that about.
Best wishes,
Bob Goldberg
