The way Pharmos is doing their clinical study is required for FDA approval. The FDA requires proof of performance before it will approve a drug for general use. There is usually a comparison with placebo, and sometimes a comparison with whatever the standard treatment is for the condition in question, (especially if the company knows that its product is better).
One of the reasons that there are so many centers is that it is difficult to get subjects for the reasons that you are concerned. If you personalize the issue, one of your loved ones has a serious head trauma injury and needs to be treated, are you willing to allow them to test a new drug on your loved one? For a lot of people the answer is going to be "no", but for some folks the answer will be "yes". Every drug or device that has been approved by the FDA over the years has gone through this type of testing, so people do say "yes".
One of the reasons that there are so many subjects is that the company needs to get a representative data sample, and a statistically significant data sample. The company has to prove that the product not only works, but how it works within gender, age, race, etc. classifications.
The number of subjects required is sort of worked out with the FDA beforehand partly. The FDA does NOT tell a company what the study should be, but the FDA will provide "guidance" or "suggestions" as to what testing they would like to see, (I don't actually know what buzz words they use, but basically the FDA is obviously not going to guarantee anything. They expect the company in question to prove that the product works.)
Phase III studies are extensive studies that are done to prove product claims. Whatever Pharmos' present claims on dexanabinol, they will have to be proven in the clinical study. This is the other part in determining the "proper" number of subjects in clinical studies. If you've ever done experiments of any sort, the larger the number of samples, (provided you have a "representative" sample), the smaller your error bars generally, the "better" your data is or the more you can see in the data. With a large data set, it is easier to see trends in the data, and hopefully justify the claims that one makes for an outcome. On the flip side, the more subjects one runs, the more expensive the study, and Pharmos isn't made of money, (as we shareholders well know).
So, in my not-so-humble-opinion and my experience, I think that Pharmos wanted to do the Phase III studies themselves because they think that dexanabinol has a chance to be a superior product in the treatment of head trauma, and by completing the Phase III trials themselves and proving the drug is superior, they can make more money from the pharmaceutical firm with whom they eventually partner.
The risk is: are the trials planned sufficient to get data that will lead to FDA approval? If you want to see what can happen if not, look at the trials and tribulations of SCIO (Scios) from 1998 to 2001 as a primer. |
