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This shows how stupid our current system is and the need for change. The video in the link below is about families with kids fighting brain tumor who want to get an experimental drug that I believe will be the basis for a cocktail approach that will lead to a major breakthrough as soon as we are allowed to try combinations with it. I get calls and emails from around the world requesting help getting access to it and most patients can not get it. There is really no approved alternatives. Imagine being a parent of a child with an incurable disease and you know there is a treatment available that may help but you can not get it for your child. That is my worst nightmare and keeps me up at night,
At an FDA meeting over 3 years ago, all of the data was presented about this drug - it helps by itself and had no side effect. I asked (at about the one hour mark in the video) the FDA to approve it immediately. They did not - they wanted more trials. Meanwhile, kids who could benefit from this drug are dying. The mission of the USA FDA is "The Food and Drug Administration is responsible for protecting the public health by ensuring the safety, efficacy, and security of human and veterinary drugs". They are too conservative with their approach to terminal diseases. Their policies are destroying families and they are not protecting our kids. The system serves only to prop up the huge expense of drugs and create monoplies by making it so expensive and time consuming to get a drug approved that many good drug candidates never make it into humans and only large companies can even try to get a drug approved. Even worse - drugs that may be part of the ultimate cocktail approach may not get approved because they do not help enough on their own. There are many examples.
It is time for a change. I believe the Promising Pathway Act is our best chance now. It is a proposed law in congress right now. It will create a new pathway to FDA approval for drugs for incurable diseases where the approval is conditional - the drug company has 6 years to prove the drug works to get full approval or loses the conditional approval. The approval will happen as soon as the drug is shown to be relatively safe compared to the currently used treatments, and has a biologic effect such as increasing survival, shrinking tumors on scan, or improving biomarkers. This may mean conditional approval after a small phase 1/2 trial instead of a prolonged phase 3 trial. After conditional approval, the drug can be sold and any doctor can prescribe it. However, every patient who uses such drugs has to be followed in a virtual trial so we can see how the drug does and learn from every patients' experiences. So this law would basically move most of the research from before approval to after approval, Doctors will have access to the ongoing results so they see how the drug does and which combinations are being tried and the results - which would let us home in on the ultimate cocktail quickly. The key is knowledge and research - this plan requires much more research and openness than the current pathways. Under the current pathway to approval, the drug is only tested on highly selected patients (very few minorities, poor people, rural people, people with comorbidities and people who are in bad shape) before approval, then after approval there is no more research. Nobody can get full access to the research. Parts are usually reported but not individual patient details.
The Promising Pathway Act will hold down the cost of drugs for a few reasons: the cost and time of getting FDA approval is reduced by about 90%. There will be more competition as the barriers to approval are reduced, allowing us to try the best new ideas without having to raise a bullion dollars for each one we try.
We need help getting this bill passed. Right now it is stalled in Congress as other more "important" issues take precedence. We tried writing letters. It helps a little and we still encourage it at https://virtualtrials.org/activism.cfm#/1/. It may be time to call and visit your representatives - especially those running for office this year. We have the support of many other foundations - mostly pediatric brain tumor foundations as well as pancreatic, ovarian and ALS foundations but need all brain tumor foundations to work together on this (or I am open if they have other ideas to get access to these drugs and increase research as Promising Pathway Act does.).