NorthWest Biotherapeutics Inc (NWBO)

[flipper44]

Well done Dok,


I agree it does not make sense to "anticipate a problem," but it does seem this was the thread of logic started by some questionable posters on Yahoo.

Instead of "anticipating," like you, I think its best to be prepared. The lawsuit (plus protests) by Dendreon to get the FDA to do the right thing in their case was a very good example. Very good.

(I think Marnix Bosch's topic at the end of January will address how PFS is used appropriately as a primary endpoint.)

Anyway, I am not worried. Here's why.

1. Even though ICT-107 received that letter from the FDA in 2011 suggesting the FDA would not allow approval if PFS was a primary endpoint in IMUC's Phase II trial, that does not apply to NWBO because:

A. NWBO actually received approval from the FDA in 2011 to use PFS as their primary endpoint in NWBO's more highly powered, phase III (not phase II) pivotal trial.

B. DCVAX-L has orphan status for this trial. Many people do not know this gives the FDA more authority to approve a medication without as stringent requirements (powering) as otherwise might be used in a non-orphan status situation. Cramer, for instance, keeps shouting that orphan status only gets companies things like additional patent time protection. This is not true, it also requires the FDA to work closely with the company and reasonably accommodate matters such as trial size and powering in order to help patients with deadly/rare conditions that might not otherwise ever have companies try to work on medications for their diseases. The assignment of orphan status to a disease and to any drugs developed to treat it is a matter of public policy in many countries, and has resulted in medical breakthroughs that may not have otherwise been achieved due to the economics of drug research and development.

Orphan drugs generally follow the same regulatory development path as any other pharmaceutical product, in which testing focuses on pharmacokinetics and pharmacodynamics, dosing, stability, safety and efficacy. However, some statistical burdens are lessened in an effort to maintain development momentum. For example, orphan drug regulations generally acknowledge the fact that it may not be possible to test 500 patients in a phase III clinical trial, as fewer than that number who are eligible for enrollment may be afflicted with the disease in question.

C. Since 2011, the FDA has moved toward using PFS as a primary endpoint, even in non orphan status cases where OS would otherwise take far too much time, too many lives and resources to get a drug approved. The European (EMA) started using this metric (in many cases) as a primary endpoint several years ago.