Regulatory Capture

As you probably know, the FDA recently approved a monoclonal antibody called aducanumab as a treatment for Alzheimer ' s disease. To make a long story short, there ' s no good evidence that it actually has any effect on dementia; the approval is based on its effectiveness in removing abnormal plaques of a protein called beta-amyloid from the brain. These are associated with Alzheimer ' s disease, but there ' s no evidence that they cause it, or that removing them improves symptoms. Aducanumab can also have some very troubling adverse effects such as brain swelling. Oh yeah -- Biogen plans to charge $56,000/year for the treatment. Now, if you don ' t think this makes a whole lot of sense,you ' ll be even more nonplussed to learn that it ' s actually quite common.  The FDA approved aducanumab through its “accelerated pathway,” a process created in 1992 to hasten approval of “drugs that treat serious conditions, and that fill an unmet medical need.” Such approvals are based on surrogate endpoints, which the agency defines as “a laboratory measurement, radiographic image, physical sign o r other measure that is thought to predict clinical benefit but is not itself a measure of clinical benefit.”Surrogate endpoints stand in contrast to direct measurements of patient outcomes, often referred to as clinical endpoints, such as living longer, symptom relief, or improved quality of life. The FDA now has multiple expedited pathways. Although accelerated approv...
Source: Stayin' Alive - Category: American Health Source Type: blogs