FDA Tackles Neurological Disorders in Five Guidances, Flags Patient Input

The FDA released five guidances on treatments for a variety of neurological disorders including Duchenne muscular dystrophy and Alzheimer’s disease, offering suggestions for trial designs and methods of measuring effectiveness.

The guidances “signal how modernization of the new drug regulatory program includes an enhanced focus on incorporation of patient input into our thinking,” said FDA Commissioner Scott Gottlieb.

A final guidance on Duchenne muscular dystrophy (DMD) and related dystrophinopathies said that endpoints that measure change of function in a wide range of deficits may offer advantages in the development of drugs that treat diseases like DMD and Becker muscular dystrophy, in addition to increasing the number of patients eligible for enrollment.