We have not had much progress in the treatments of Glioblastomas. Ever. Of every phase 3 clinical trial that was ever done for Glioblastomas, only a handful were considered a success, and the best of them, the Optune EF-14 trial, only increased median survival by 5 months. We tried this methodology for about 70 years and it did not work. Time to try something new.
The current system is too rigid and slow. A trial is designed, then has to run out for 3-15 years to get a result. Then they run another trial based on those results. The cost and time are so significant that most of the promising treatments in the lab never get into human trials.
This proposal will turn the system upside down. It would minimize phase 3 trials. Once a treatment is tested in a small group of patients and considered safe (compared to other approved treatments) and has a biologic effect, it can get a conditional approval. All patients who use the drugs approved under this pathway have to go through an informed consent process as if they are going into a clinical trial and be made aware of the risks of using a drug not fully approved. They all have to be tracked in a virtual trial so we can learn from every patient's experience. The net results is way more research than the traditional pathway - as only about 10% of less of adult Glioblastoma participate in clinical trials. Instead of each iteration of a new combinations taking 3-15 years and millions of dollars to test, multiple Virtual Trials of combinations can be run at the same time, and can take a year or less and virtually no cost to the researchers and clinicians who create these cocktails.
These researchers and clinicians will have access to the live data so they can see which drugs and combinations have been used, how safe they were, how effective they were, and what combinations were used and how they worked out. They can propose a virtual trial of any number of combinations, and limit those to patients with specific markers. Bad drugs will be weeded out quickly instead of lingering for decades as they do now.
And finally, prices should be kept under control. The payment part of the bill is still being hammered out but as it stands now, insurance should pay for the drugs. At first that may increase costs as a lot of new drugs come out and they are used in combinations, but over time, the cost of developing drugs will plummet as will the time needed to do so, which should drop prices. Then the free market system should prevail, with many treatments with similiar results competing based on prices. Furthermore, nonprofits like the Musella Foundation will create drugs ourselves, as nonprofits, at a reasonable price with proceeds going to the next generation of drug. We know of many candidate drugs that did great in the lab but never made it to human trials. We could create an assembly line of pre-clinical testing and phase 1 trials, and take the best of the phase 1 results and move those onto phase 2 trials and try to get those approved. We will see progress continuously instead of now where there is a small bump up every few years.
Posted on: 07/30/2021
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