Guidance on Early Alzheimer’s Drugs Covers Endpoint, Patient Selection

FDA draft guidance on early-stage Alzheimer’s drugs offers tips on endpoint selection, patient selection and showing disease modification. The guidance applies to drugs that treat patients before the onset of overt dementia. Those closest to developing overt dementia may have mild but noticeable impairments in daily functioning, and it is important to show an effect on those impairments in addition to showing an improvement in cognition, the guidance states. It suggests using a composite scale, validated in early-stage patients, to assess both cognition and function as a single primary efficacy outcome measure. The agency recommends the Clinical Dementia Rating – Sum of Boxes score but is open to considering other scales as well.
Washington Drug Letter