Biogen’s decision to return to experimental trials with a drug that had failed to meet therapeutic, statistical significance using various populations and different doses is a reversal of many longstanding drug development procedures.
Other companies in the industry are watching closely, as the approach signals changes in study designs that will be praised and damned by other researchers.
The holy grail is a vaccination, of course. The next best thing is a drug that slows down the course of dementia and there are two or three theories for the mechanism of disease that vary widely. The skeptics among us will scoff at three competing theories. The believers will endorse the likelihood that there may be several disease mechanisms. The familial strain in south America is unquestionably genetic.
That something so monumental can hang on tests of statistical significance of such minor magnitude is disturbing. When we discover the grail, we want fireworks and the best Champagne, not an improvement of 0.001 (one person out of a thousand scoring one point higher, for example).
That’s not a blockbuster for the drug company, nor a reason for hope among the afflicted families.
Development of novel antibiotics, cardiac, kidney, MS and many oncology syndromes have fireworks effects. In the best cases, the research stops early because the success is so apparent. These are game changes, quality-of-life changers and the re-opening of the Biogen trials holds little promise to join the pantheon of drug all-stars.