NorthWest Biotherapeutics Inc (NWBO)

[Lykiri]

"We want the treatment, not the placebo!"

The FDA draft, "Considerations For The Design And Conduct Of Externally Controlled Trials (ECT'S) For Drugs And Biological Products" has been released for public comment! Docket #FDA-2022-D-2983-0019. ACT NOW!https://t.co/lhAxaEgHbT pic.twitter.com/4Ghkn5SiU5

— EndBrainCancer Initiative (@EndBrainCancer) April 26, 2023

Calling ALL: Patients, Caregivers, HCP’s, Researchers, and Companies, etc. - We NEED Your Comments!

"CONSIDERATIONS FOR THE DESIGN AND CONDUCT OF EXTERNALLY CONTROLLED TRIALS (ECT'S) FOR DRUGS AND BIOLOGICAL PRODUCTS"

READ FDA GUIDELINES HERE https://endbraincancer.org/wp-content/uploads/2023/04/FDA-2022-D-2983-0002_attachment_1.pdf

Key points from an EBCI Advocacy Perspective for GBM/rGBM Patients in layman's terms are:

FDA’s new DRAFT Clinical Trial Guidance now suggests that External Data/Info can now be used/blessed in clinical trials. EBCI says “Thank You” to the FDA for considering this as it is inhuman for this patient population to participate in a clinical trial and to not receive the treatment at the beginning of the trial when all data that is needed can be assessed from prior clinical trials/historical data, real-world data (RWD) sources s well as from electronic health records (EHRs) and medical claims.
We applaud the FDA for working towards updating their clinical trial requirements for this patient population. We appreciate the FDA hearing EBCI’s Advocacy voice on this topic AND the voice of the patient, caregiver, family member, and/or general public who has/have been touched with GBM. It is time to acknowledge that Advocacy and this patient population want a change in how clinical trials are required to be run by the FDA and it is time to stop subjecting this patient population to needless randomized trials. This patient population should ALWAYS receive treatment up front and not later in a “cross-over” protocol. It is time to move away from progression free survival (PFS) endpoint. It is beyond time for the data that is available to researchers gleaned from the brave GBM patients who have come before us, to help others. It is time that this patient population is heard and it is time that this patient population eagerly desires to participate in clinical trials instead of not wanting to participate for fear of receiving a placebo. It is time that the FDA heard the patient loud and clear as well as the same message from Advocacy groups like EBCI.
EBCI does not Advocate for Randomized Clinical Trials, which means that some of the patients participating in the clincial trial will receive the treatment and a group of patients will not receive the treatment. When a patient participates in a clinical trial and does not receive the treatment, what they receive is called a “placebo”.
EBCI advocates for updates in clinical trial protocol for this patient population as it relates to primary and secondary endpoints/PFS/OS the patient/cohort that received the treatment and the placebo cohort/patients that did not receive the treatment, who participated in the trial.
EBCI feels that with the deadly nature of GBM and rGBM disease and the patient data that has already been collected over many years from this patient population in past clinical trials/historical control, should be data that can be used in place of a randomized group of patients as it relates to clinical trial development/protocol and FDA approval. Specifically, we feel that the patient population that is participating in a clinical trial should ALWAYS receive the treatment and then be compared to historical patient data from previous clinical trials, therefore, saving this patient population from participating in a trial that is randomized where some of the patients will receive a placebo.
This is a terminal disease, so why does it make sense for this patient population to be subjected to a randomized trial where they will receive a placebo or a "comparison arm”. Why are External Controls such as using data from patients in prior clinical trials for GBM not allowed for this deadly disease? Clinical trials for this patient population should no longer require a randomized set of patients as all patients in the randomized set of patients will likely die. Clinical trials for this patient population should no longer allow randomization that forces some of these patients to receive a placebo vs. treatment.
There is enough historical data from past patients with GBM/rGBM that have participated in clinical trials that were randomized and that were given a placebo instead of treatment that it doesn’t make sense for clincial trials to continue to be developed for this patient population that includes randomization and placebos.
Here’s what we hear consistently from patients with GBM/rGBM: “I do not want to be placed in a clinical trial where I will receive a placebo. I know that I am going to die from this disease so why would I participate in a trial where I do not receive the treatment which may increase my survivorship in addition to providing more patient data to help others in future clinical trials?

Please understand that this Advocacy Task and Request from EBCI is URGENT and the deadline is Friday 4/28 at 12:00 noon Pacific Time/3:00p Eastern Time.

"These Patients are going to die quickly, therefore, it makes no sense to give them a placebo in a clinical trial instead of the treatment, especially when the external patient data already exists and can be used as the control arm w/in the clinical trial design/protocol for rare diseases."

Make Your Voice Count TODAY!

Our GOAL with this urgent ACTION is to strongly encourage the Brain Tumor/Cancer Community to tell their own story as it relates to NOT wanting a Placebo when taking part in a clinical trial. Tell them why it's so important to you and whether or not you would/or have turned down a Clinical Trial simply due to the fact that you aren't sure if you'll be getting the desired protocol or a placebo!

Submitting your comment is EASY! Simply click on the button to be directed to the FDA portal in order to review any information you'd like and submit your comment. It doesn't have to be long and we've provided some snippets of comments below to help make it quick and easy. There is a very tight DEADLINE on this so immediate action is highly encouraged!

https://endbraincancer.org/fda-docket-fda-2022-d-2983fda/