NorthWest Biotherapeutics Inc (NWBO)

[flipper44]

Each size company presents a pragmatic issue, hence one of the primary reasons orphan drug designation and accelerated approval exists.

On the front end, if Merck said, we are pulling the plug on developing more orphan drugs because the FDA is denying accelerated approval to therapies we spent hundreds of millions on, even though they reached (albeit it barely in some cases) sufficient scientific proof for AA. Consequently, we can't justify the added expense of what would otherwise be "confirmatory" trials, because no revenue is coming in. The ripple effect of this goes right to our next child, or grandchild, or neighbor's child who gets an orphan disease -- hopelessness, and when it is a company like Merck the exclusion of orphan drug development is felt on a massive scale. Maybe they could afford to complete the drugs they had in the pipeline, but their development program may stop after that.

On the other hand, on the back end, if the FDA states to some small (the size of NWBO) company that they will not grant accelerated approval, even though such company may have met the minimal requirements for statistical significance, safety and the like; and instead forces them to conduct another trial to full approval, the FDA is basically hammering the last nails in a sea of coffins for that product and their patients, because they cannot afford to go forward.

In either case, large company or small, there can be fatal and horrific human ramifications in a world where an FDA chose not to use the scientific benefit of the doubt known as "accelerated approval" for orphan drugs.