ACCELERATED APPROVALS ARE INCREDIBLY IMPORTANT FOR RARE DISEASES and are vastly underutilized today. These pathways have been vital in advancing therapies in cancer and HIV. In the thousands of rare diseases, traditional clinical studies are inappropriate or unfeasible and the only way we will see therapies come to so many in need (many of them children) is by taking measured risks that are outweighed by the potential benefits to patients. The use of biomarkers, for instance, will be of paramount importance for new medicines in rare, devastating diseases. And it’s important to remember that the legislative standard here is an endpoint “reasonably likely” to predict clinical benefit. This is an issue that we at BIO, and I specifically, will be talking a lot more about in the months ahead.
JOHN CROWLEY is chairman and CEO of Amicus Therapeutics.