Replies to post #93963 on NorthWest Biotherapeutics Inc (NWBO)

[flipper44]

GoodGuyBill, I don't want to get people too excited, but:

If it is clear during development that a product is intended to be approved under accelerated approval on the basis of a surrogate endpoint or an intermediate clinical endpoint, confirmatory trial(s) should be underway at the time the marketing application is submitted. If it is not clear until shortly before or after submission of a marketing application that a surrogate endpoint or an intermediate clinical endpoint will be the proposed basis for accelerated approval, there should be agreement on the design and conduct of such trial(s) before approval.

Generally, the confirmatory trial would evaluate a clinical endpoint that directly measures clinical benefit. For example, the confirmatory trial population would ordinarily be the same disease population that was studied to support accelerated approval. In some cases, however, the commercial availability of a drug following accelerated approval may make it difficult to enroll patients in the same disease population. In these cases, a confirmatory trial may be conducted in a different but related population that is capable of verifying the predicted clinical benefit. -- U.S. Department of Health and Human Services Food and Drug Administration
Center for Drug Evaluation and Research (CDER) Center for Biologics Evaluation and Research (CBER)
May 2014 Procedural