NorthWest Biotherapeutics Inc (NWBO)

[Doktornolittle]

I don't see anything disturbing about calling out subgroups. It de-risks the trial if you somehow compensate for the alpha spend and increase the total patient numbers to allow SS at an obtainable median efficacy for each of those subgroups. And they did increase the patient numbers. I think they might have compensated for the alpha spend by not doing any efficacy looks until the trial reaches the primary endpoint.

The cost of that de-risking is that the trial becomes a little more expensive and the total $ gained for that trial might be a little less, but maybe not as much less as you think. When you narrow approval to subgroups you increase efficacy for the approved patient pool. Since reimbursement is based on efficacy, at least to some extent, the reduced future patient numbers are offset by increased cost per patient. Approval by subgroup is also the more ethical way to go about this. I am certain of that.

If you made the commitment to build a super expensive manufacturing infrastructure for a drug in trials, expecting an enormous market on or soon after approval, you might just chose to de-risk that investment at the relatively small costs described above.