Saturday, April 19, 2014 6:03:08 PM
"As a member of the "Jumpstarting Brain Tumor Drug Development Coalition" which is based on the National Brain Tumor Society’s Clinical Trial Endpoints Initiative, I recently had the honor of speaking with the FDA about this issue. The FDA is on our side - we all want to get effective drugs to the people who need them as quickly as possible, while maintaining safety. The bottom line is that the FDA said that although they prefer the overall survival endpoint, they would accept progression free survival as an endpoint for approval of a treatment - IF there was a significant benefit to the treatment.
This particular trial is special. You need a brain tumor surgery to get a tumor sample which is used to make a custom made vaccine to your tumor. Everyone in the trial gets the vaccine made as well as gets the standard of care. 2/3 of the patients get the vaccine and 1/3 get a placebo. When the patients in the placebo group have progression of the tumor, they are allowed to cross over into the treatment group and get the vaccine. This is the most humane way to design a randomized trial; however, it also makes it impossible to judge the treatment on the basis of overall survival. If the treatment actually works, the overall survivals of both groups would be the same - because both groups get the same treatment so you are comparing the treatment against itself- and the better it works the less chance it would get approved based on overall survival. The progression free survival endpoint is ideal in this case and is really the only endpoint possible. If they were forced to use overall survival, they couldn’t let the patients cross over, and the custom made vaccine would go to waste – sentencing the patient to an unnecessary death just to make the statistics look better.
The question became how much of an improvement is needed in progression free survival to justify approval. The FDA wouldn't give us a specific answer, but said that if it works, they will approve it. Historically, the FDA will approve a new treatment for incurable diseases if it is just a little better than existing treatments, or the same results as existing treatments with less side effects. The bar is not that high."
Recent NWBO News
- Form NT 10-K - Notification of inability to timely file Form 10-K 405, 10-K, 10-KSB 405, 10-KSB, 10-KT, or 10-KT405 • Edgar (US Regulatory) • 03/01/2024 10:04:38 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 12/02/2023 01:31:35 AM
- Form 8-K - Current report • Edgar (US Regulatory) • 11/16/2023 10:11:54 PM
- Epazz, Inc. (OTC Pink: EPAZ) ZenaDrone Demonstration to Defense Departments of UAE and Saudi Arabia • InvestorsHub NewsWire • 11/15/2023 12:19:31 PM
- Form 10-Q - Quarterly report [Sections 13 or 15(d)] • Edgar (US Regulatory) • 11/09/2023 09:30:39 PM
- Epazz, Inc. (OTC Pink: EPAZ) US Navy Collaboration ZenaDrone 1000 • InvestorsHub NewsWire • 11/09/2023 01:00:34 PM
- Epazz, Inc. (OTC Pink: EPAZ) US Navy Collaboration ZenaDrone 1000 Extreme Weather Demo • InvestorsHub NewsWire • 11/07/2023 12:29:43 PM
- Form 10-Q - Quarterly report [Sections 13 or 15(d)] • Edgar (US Regulatory) • 08/09/2023 08:36:14 PM
NanoViricides Reports that the Phase I NV-387 Clinical Trial is Completed Successfully and Data Lock is Expected Soon • NNVC • May 2, 2024 10:07 AM
ILUS Files Form 10-K and Provides Shareholder Update • ILUS • May 2, 2024 8:52 AM
Avant Technologies Names New CEO Following Acquisition of Healthcare Technology and Data Integration Firm • AVAI • May 2, 2024 8:00 AM
Bantec Engaged in a Letter of Intent to Acquire a Small New Jersey Based Manufacturing Company • BANT • May 1, 2024 10:00 AM
Cannabix Technologies to Deliver Breath Logix Alcohol Screening Device to Australia • BLO • Apr 30, 2024 8:53 AM
Hydromer, Inc. Reports Preliminary Unaudited Financial Results for First Quarter 2024 • HYDI • Apr 29, 2024 9:10 AM