Aggregated News
The Food and Drug Administration was more than likely correct to reject Biohaven Pharmaceuticals’ treatment for spinocerebellar ataxia, a rare and debilitating neurodegenerative disease. At the very least, the decision announced Tuesday night was not a surprise to anyone paying attention. Approval was a coin flip, at best.
The complete response letter has not yet been posted to the FDA website, but according to Biohaven, the agency rejected its drug, called troriluzole, due to issues that can be “inherent to real-world evidence and external control studies, including potential bias, design flaws, law of pre-specification and unmeasured confounding factors.”
Biohaven has said the FDA was copacetic with the construction of the external control database and the statistical analysis plan used as the comparator arm in its clinical trial. The company is now blasting the FDA for changing its mind.
Fair criticism, except it’s important to remember that agreeing to review a drug application based on a study with an external control arm is not the same thing as concluding the data are sufficiently convincing to grant approval.
It is entirely possible...