In Phase 1 trials, healthy people, often students, are offered money to test new drugs for safety. In a Phase 2 trial, people with the targeted disease are enrolled and may experience benefits – only to be denied access at the end of the trial. For terminal diseases like ALS that is a death sentence!
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The FDA still adheres to a 1962 clinical trial protocol that is fixated on an arbitrary statistical number. This outdated protocol has created a multi-billion dollar clinical trial industry and approvals that takes an average of 12 years at a cost of up to $1 billion for a new treatment. The FDA has not uniformly adopted current state-of-the-art science applications for trials. Unlike 1962, today we have computers to analyze large databases, strategically populate trials, utilize complex predictive algorithms, and detect small changes and patterns in clinical data.
*A surrogate endpoint is typically a biomarker intended to substitute for a clinical endpoint to save time and money. It is most useful in slow-moving diseases where measuring the progression of clinical endpoints (e.g. patient function or survival) could take years. The ALS research community has not yet concluded there is a verifiable biomarker for ALS. However, forced vital capacity (FVC), can and should be regarded as both a clinical and a surrogate endpoint.
Amyotrophic Lateral Sclerosis (ALS) or Motor Neuron Disease (MND) is a degenerative neurological disease that affects neurons in both the brain and spinal cord, causing voluntary muscles throughout the body to wither. It is always fatal, on average within 3 years. It afflicts people of all races, genders and ages. People with ALS (PALS) lose the ability to eat, drink, speak, walk, use their arms and hands, and ultimately breathe. Up to 6,000 U.S. citizens are diagnosed with ALS each year, and up to 35,000 have the disease at any given time.
There are experimental drugs showing promise that may improve quality of life, and slow or even stop the progression of this horrific disease – BUT WE ARE DENIED ACCESS TO THEM UNDER OUR CURRENT SYSTEM.