Finding potent drugs for Alzheimer's disease is more important than proving the drugs are disease modifying.
There are many who feel that the achievement of a disease-modifying (DM) designation for a drug is an important goal in Alzheimer disease (AD) therapeutics. Several designs that might prove DM have been proposed, including randomized start, randomized withdrawal, and slope analysis. Using biomarkers such as cerebrospinal fluid assays or imaging features as primary outcome measures have also been considered a way of proving DM. The available methods for establishing DM are fraught with logistical and analytic problems. Unless the drug's benefits are large, attempts to prove DM for a truly effective drug may fail. Therefore, clinical trial designs that are the simplest to conduct and interpret are superior to those more complex approaches intended to prove DM.
Knopman
David
D
Department of Neurology, Mayo Clinic College of Medicine, Rochester, MN 55905, USA. knopman@mayo.edu