Unregistered

Jacqueline, and all
I don't think it's an issue of they "did not follow the rules". There are in fact Phase III studies underway. As well as multiple Phase II studies. With a totally new investigational therapy they first need to do Phase I (safety) then Phase II (continued safety and efficacy) studies. These are necessary prerequisites to a Phase III.

Consider the difficulty of recruiting patients before these steps are taken: Here, you can try the approved therapy which we know works for some people and might work for you or you can try this other therapy that we have no idea if it will be safe, or work at all.

True, to get really strong data you need to have valid, large trials. But you can't get to valid, large trials without some smaller steps first. And all these take many years. Plus the many many years it takes to develop the drug itself - TDM1 has been in development for over 10 years.

I do not see "big Pharma" as the villain - or at least if they are, there are many others complicit including the FDA. True, they are in it to make money, but not ONLY to make money and internally they struggle mightily to keep sight of their mission to save lives.

I think too often they all lose perspective - for example to me it is ludicrous to prevent access to not-yet-approved therapies to patients who's only other option is to die quickly from their disease, in the interest of "protecting the patient". Certain death from cancer is a pretty serious "adverse event".

Or, worst of all, lets say that someone actually does invent a "magic bullet" - even a less than 100% one such as TDM1 which has an extraordinary clinical benefit rate considering the heavily treated population that has been studied so far. How many people died or had metastatic recurrence waiting for Herceptin??? How many is too many?

What does BCA or the FDA have to say to the families of all the people who die waiting for "proof"? Oops, sorry about the wait but your mom/sister/daughter/father died for the greater good of validating the data?

Sorry, I'll climb down off my soapbox, but in my opinion BCA should have as part of their "strategic mission" supporting efforts to (as Steph notes) speed up the process by improving the applicability of the science.
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