Nidan, precision medicine (or personalized medicine) has been around for decades though none of the AD therapies that are approved fall in that category. As a good old example, treating a Urinary tract infection is personalized -- cultures are grown from the urine and if many colonies form, they are regrown with antibiotics. If nothing grows they know the bacteria is sensitive to that antibiotic and we can prescriber it. If bacteria re resistant to an antibiotic in culture, we know it is unlikely to work in the person. We usually think of precision medicine as picking a treatment based on someone's genes and on cellular markers (especially with cancer). The hope is that outcomes will be better if the right medication is chosen initially. Outside of cancer where it is often used, we now know that a person's genes affect metabolism of many medications such as antidepressants, blood thinners, pain med's and tests are available (though seldom used) to try to personalize treatment. Obviously, Anavax hopes that the DNA screening will match some genes to the better outcomes. The problem, and it is a big problem, is that there are only a few super-responders and no placebo. I don't see how they can get any meaningful result. They may just find markers for slow AD rather than treatment effect. Anavex would have been much better served by a larger phase II with a placebo arm. The FDA is very content to have companies do Precision medicine trials -- however, if they only investigate patients who are XYZ positive then the approval will only be for XYZ patients not for everyone with AD and insurance will not cover those who are not in the genetic category. As a great example, one of the new lung cancer drugs is approved for ALK+ NSCLC, not everyone with NSCLC nor every lung cancer
Real world experience trials are very commonly done in Phase IV and doctors kind of ignore the results as they are uncontrolled and/or nonrandomized and often too small or too variable and we always wonder if patients were selected to be in one group or another based on disease aggressiveness or other factors. As an example, if there was real world nonrandomized comparison of Avonex (weaker) vs Tysabri (stronger), chances are I would put my aggressive MS patients on Tysabri and less aggressive patients on Avonex. This would serve to make the weaker drug look stronger as the bar for success was lower. I just don't see the FDA allowing RWE AD trials since AD is so common and recruitment is not difficult.
One has to be careful changing a protocol mid-study. In Phase III's there are independent data monitoring committees (independent doctors who are not recruiting patients plus statisticians). Let's say a study compared two doses and placebo but the high dose was found to have many dropouts due to toxicity. It would be reasonable for the independent data monitoring committee to pass this info on to the company. They then would likely drop the high dose (and move all high dose patients to the lower dose) and continue the study, and possibly increase the numbers to be enrolled in the now only dose and the placebo. The advantage of adapting is the study goes on and timeframes can be kept but the disadvantage is they now have less likelihood for success (assuming higher dose was more effective). Biogen just adapted their amyloid drug study by adding more patients
News 
Market Data 
Discover 
