This article raises the question about
clinical trials and how effective they are. Have a read:www.nytimes.com/2013/07/14/opinion/sunday/do-clinical-trials-work.html?pagewanted=all&_r=0
Just a few quick comments about
clinical trials and the expectations a patient should have. Some trials are rather mundane, for example right now there is a trial to be randomized into surgery or radiation. In either direction, the patient will benefit with a local therapy and a trial like this cannot use a "placebo" so all will be treated. But when it comes to advanced therapies it should be noted that the only truly effective trials are the randomized prospective, and if able double blind where neither the doctor nor the patient know if the drug us being used or the placebo.
Here are some thoughts about
trials and studies:
1> RCT's (Randomized Clinical Trials) are not a therapy. They are an established protocol to answer a question.
2> Level III Studies and their conclusions and stand 11% accurate when they are run as a Level 1 clinical trial. Meaning that what we typically see in a level III study is wrong 8 out of 9 times.
3> Level I RCT's are facts. But that does not mean that these facts are constant. For example, the TAX 327 trial completed in 2004 gave us Taxotere as the first life extending chemotherapy drug for prostate cancer. The benefit in the trial was 19 months over the placebo arm. Meaning some men did not have a response to the drug, and some men had extensive response to the drug. 19 months as a mean survival does not sound like a long time but given the conditions it was a great breakthrough.
4> RCT's are very expensive to run. In addition for every trial that brings us a new drug, hundreds of trials were either cancelled due to lack of participation, issues in the protocol, or simply proving ineffective. You wonder why Zytiga costs so much? Currently I am working on a long term trial for metastatic disease and early use Zytiga. It will take years to complete and there are literally hundreds of physicians involved in the research. The drug company is footing the bill. If this proves effective, and it likely will, then the costs will need to be paid with the current on-patent drugs. Once a drug goes off patent, then trials become far more difficult. For example, dutasteride, and its effectiveness as a "stabilizer" when on AS or when on vacation from HT could take years to run the trial. But since the drug is off label, the costs are generally absorbed by the CDMRP for research for these types of drugs.
5> A failed completed trial is still a success. While patients might not benefit, having the correct answer about
drugs that don't pan out in RCT's is still valuable information. This can improve patient care by wide margins by targeting when and if a drug can be most effective or not effective at all.
6> A trial that gets pulled is a researchers worst nightmare. Some of the reason's to pull a trial are that accrual is too low to be statistically significant, an error in the protocol must be corrected, adverse effects are overwhelmingly in need of changing the trial, etc. There are more reasons, but when a trial comes down without completion it's back to the drawing board and whatever time in the trial no matter how long the trial ~ is lost.
I hope this gives some food for thought in trials. In recent years we have added quite a few new options in advanced PCa and more are headed our way. To all those that have been on trials ~ thank you for paving the way for others.
Post Edited (TC-LasVegas) : 7/16/2013 9:58:11 AM (GMT-6)