European regulators ’ views on a wearable-derived performance measurement of Ambulation for Duchenne Muscular Dystrophy regulatory trials
Before new medicines can be placed on the market, medicines ’ developers have to generate and submit evidence to regulators that the benefits outweigh the risks of the new medicine in the targeted indication. A critical part of such evidence is the design of the pivotal clinical trial and the choice of endpoints. For regulators, a primary endpoint in a piv otal trial needs to be accurate, reliable, sensitive to change, reflect the objectives of the trial, and be relevant to patients and clinicians.
Source: Neuromuscular Disorders - Category: Neurology Authors: Marion Haberkamp, Jane Moseley, Dimitrios Athanasiou, Fernando de Andres-Trelles, Andr é Elferink, Mário Miguel Rosa, Armando Magrelli Tags: Short communication Source Type: research