neuroinvestment & hm,
The other way at looking at this is that we are going to see more powerful preclinical and Phases I and II testing. In other words, the pre-Phase III testing will have a much better chance of picking up rare toxicities and finding the sub-groups in which the candidate works bet. Instead of relying on large numbers of patients to cover all bases, early testing will occur on smaller groups pre-selected to have different genetic characteristics.
In the case of Clozaril, I don't recall when the agranulocytosis side-effect showed up - I believe it was pre-approval, but I'm not sure how early. Paradoxically, if it had showed up very early (e.g., in pre-clinical) it might have been enough to kill the drug entirely before its dramatic efficacy was demonstrated.
So in the short term, we might see more early casualties among early-stage biotech drug candidates. Superficially this might look bad, but of course it is in fact good.
Trying to look at the big valuation picture, failing candidates early clearly makes a big difference. With more and more biotechs financially strong, the pressure to hang onto bad candidates too long should lessen some, although it is always going to be more significant for biotechs than for pharma.
The payoff of course is in smaller, quicker and more powerful Phase III's. If you basically know ahead of time just which patients the drug is most likely to work on, then you will no longer need large trials to show statistical significance. For example, suppose you find a drug that works 80% of the time among a small (say 10%) subset of the general population, in a condition where there is a 20-30% placebo response. A 50 or 100 person trial in the subgroup (I'm just guessing at the statistics here <g) would likely be powered enough to show efficacy, whereas you would likely still get equivocal results from even a few thousand patient trial in the broader population.
Peter |
