There has been a prolonged years long debate among scientists, researchers, doctors and perhaps most strongly from patients about the phased clinical process by which drugs are approved by the FDA. Most agree that the methods by which these drugs and now other biologics like stem cells are antiquated but there is not a lot of agreement about solutions.
A little over a year ago, Andrew Von Eschenbach, a former commissioner of the FDA pled for progress in a well-read Wall Street Journal article (http://online.wsj.com/article/SB10001424052702303815404577331673917964962.html) where he explained the landscape of problems that exist preventing quicker translation of stem cells and drugs into treatments that are available to patients.
Paul Knoepfler, a well regarded associate professor at UC Davis and one of the most prolific stem cell bloggers (http://www.ipscell.com) who I have addressed here before, has posted a four part series with Dr. Arthur Caplan, the man who actually named MSCs. In Paul's interview with him, Dr. Caplan states "The FDA is inhibitory to translation of stem cell therapies to patients. There is no sense in regulating MSCs as a drug." And kudos to Paul for publishing something that pushes against that which he has been supporting--greater regulations albeit far from the most conservative position. By using the link to Paul's blog which you can subscribe to, you can read the entire four part series.
Dr. Caplan rightly points out the various stakeholders in this process and we can see what some of the issues are. However, to further point out the antiquated process the FDA in the U.S. uses, I will cite two brief and simple examples of the absurdity involved.
Phase l trials are basically for safety. Phase ll are for efficacy. When testing patients for safety, it would be difficult to not be able to notice whether they are improving. However, those observations are not allowed to used in the Phase ll portion of the trial.
Another example is if there are several arms and one is working far better than the others, rather than being able to decrease the size of one of the lesser effective arms and increase the focus on the more effective arm, basically that portion of the trial needs to be started all over. There are adaptive trials now beginning to take place in other countries that allow this flexibility but the U.S. remains rigid in its rules.
My biggest problem however, lies with those who are terminally ill or have no other possibilities for cures to their illness. Even with complete patient consent and stem cells that have been produced under the highest standards, The U.S. FDA will not approve the compassionate use of stem cells even as an experimental process. In other words, someone is dying and the FDA is protecting them from something that might harm them and this is in light of so much research such as the meta trial on MSCs that I blogged about in an earlier post.
This doesn't seem prudent. In fact, it seems like cruel and unusual punishment.
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