{"id":42169,"date":"2013-12-09T06:30:54","date_gmt":"2013-12-09T11:30:54","guid":{"rendered":"http:\/\/1boringoldman.com\/?p=42169"},"modified":"2013-12-09T06:57:35","modified_gmt":"2013-12-09T11:57:35","slug":"rebel-with-a-cause","status":"publish","type":"post","link":"https:\/\/1boringoldman.com\/index.php\/2013\/12\/09\/rebel-with-a-cause\/","title":{"rendered":"rebel with a cause…"},"content":{"rendered":"\n
There’s a fascinating interview in the BMJ worth anyone’s time to read:<\/div>\n
\n
FDA official: “clinical trial system is broken”<\/a><\/div>\n
by Deborah Cohen, investigations editor<\/div>\n
British Medical Journal<\/font><\/strong>. 2013 347:f6980.<\/div>\n
[full text on-line<\/a>]<\/div>\n<\/blockquote>\n
First, a word about the FDA as a reminder. The FDA was established to insure the safety<\/font><\/strong><\/em> of foods and drugs in 1906 as part of the USDA, and became the FDA officially in 1930. In response to the Thalidomide tragedy, in 1962, proof of "substantial" efficacy<\/font><\/strong><\/em> was added to their charge. The FDA approval does not say a drug is a recommended clinically effective treatment, but only that it is safe, and that it has been shown to have efficacy in Clinical Trials. Efficacy has come to mean proven efficacy in at least two clinical trials. Safety, is evaluated under a more strident standard looking at all trials<\/em> done on the drug. Drugs are approved for a specific use. What that means in practical terms is that the drug companies can only market the drug for the approved use. Actual usage is determined by the medical profession who can prescribe the drugs independent of the FDA approved usage.<\/div>\n

I’ve spent more time than I’d like to admit roaming through the FDA Approvals over the last several years. In spite of being underfunded, I’ve actually been impressed with the quality of the evaluations done by the FDA scientists and the thoroughness of their reports. In my mind, it’s a piece of our government that’s doing its best in a tough circumstance. The place where I’ve been disappointed is not the scientists who evaluate the NDAs [New Drug Applications] and write the reports, it’s in the panel that looks at the reports and makes final approval. There are too many examples where they’ve approved a drug against the recommendations of the FDA scientists on shaky grounds – Zoloft® comes immediately to mind [zoloft: the approval I…<\/a>, zoloft: the approval II…<\/a>, zoloft: the approval III…<\/a>]. The central task of the FDA is safety. Efficacy evaluation is more limited [in spite of what the drug companies would have us believe]. So FDA Approval of an antidepressant says, "this drug has demonstrated antidepressant properties in two short term trials." It says little about clinical relevance or long term usage.<\/p>\n

So back to the BMJ interview with an FDA official, a rebel with a cause<\/font><\/strong><\/em>, Dr. Thomas Marciniak, a veteran investigator at the FDA. He’s less impressed with the FDA process than I am, and has had something of a head-butting career – most recently over Avandia®. Since the interview is there for you to read, I’ll avoid the details. I’ve been trying to think of a way to synopsize what Dr. Marciniak is about, and this is what I came up with. When I started to pore over the FDA reports, I had a preconceived idea of what I might find – sloppiness or maybe even collusion with the pharmaceutical industry. I didn’t find that. I found people being thorough in evaluating the NDAs, checking the findings, questioning confusing parts – in general, doing their jobs as defined. They were neither sloppy nor corrupt. Matter-of-fact, bureaucrats doing what they were supposed to do.<\/p>\n

Dr. Marciniak is not such a person. He looks into the clinical trial design, the details of how these studies have been conducted, he worries about missing data and that it’s on the rise. He worries about the long term and thinks the FDA doesn’t do anywhere near enough post-approval investigation. In short, he’s more like you and me – interested in the nuts and bolts and wanting to be thorough in his evaluations. He goes way beyond the extra mile. He wants to know about hidden data. and talks, at times, more like a data transparency activist that a government bureaucrat just doing his job. He steps beyond the FDA’s concrete assignment and wants to look deeply into both the safety and efficacy of a drug, He’s outspoken<\/em>, or maybe out<\/em>·<\/em>speaking<\/em>, willing to go nose-to-nose with his bosses. <\/p>\n

He strikes me as the kind of investigator we need – someone who is willing to be a pain-in-the-ass in order to keep marginal or dangerous drugs off the market. Like I said, he’s a rebel with a cause<\/font><\/strong><\/em> – and it’s a pretty good cause at that…<\/div>\n","protected":false},"excerpt":{"rendered":"

There’s a fascinating interview in the BMJ worth anyone’s time to read: FDA official: “clinical trial system is broken” by Deborah Cohen, investigations editor British Medical Journal. 2013 347:f6980. [full text on-line] First, a word about the FDA as a reminder. The FDA was established to insure the safety of foods and drugs in 1906 […]<\/p>\n","protected":false},"author":2,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_bbp_topic_count":0,"_bbp_reply_count":0,"_bbp_total_topic_count":0,"_bbp_total_reply_count":0,"_bbp_voice_count":0,"_bbp_anonymous_reply_count":0,"_bbp_topic_count_hidden":0,"_bbp_reply_count_hidden":0,"_bbp_forum_subforum_count":0,"footnotes":""},"categories":[2],"tags":[],"class_list":["post-42169","post","type-post","status-publish","format-standard","hentry","category-politics"],"_links":{"self":[{"href":"https:\/\/1boringoldman.com\/index.php\/wp-json\/wp\/v2\/posts\/42169","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/1boringoldman.com\/index.php\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/1boringoldman.com\/index.php\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/1boringoldman.com\/index.php\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/1boringoldman.com\/index.php\/wp-json\/wp\/v2\/comments?post=42169"}],"version-history":[{"count":16,"href":"https:\/\/1boringoldman.com\/index.php\/wp-json\/wp\/v2\/posts\/42169\/revisions"}],"predecessor-version":[{"id":42185,"href":"https:\/\/1boringoldman.com\/index.php\/wp-json\/wp\/v2\/posts\/42169\/revisions\/42185"}],"wp:attachment":[{"href":"https:\/\/1boringoldman.com\/index.php\/wp-json\/wp\/v2\/media?parent=42169"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/1boringoldman.com\/index.php\/wp-json\/wp\/v2\/categories?post=42169"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/1boringoldman.com\/index.php\/wp-json\/wp\/v2\/tags?post=42169"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}