Today, PPMD President Pat Furlong has the distinct honor of participating on a panel at the Engelberg Center for Health Care Reform at Brookings.
The topic will be: Enhancing the development of and use of patient reported outcomes in drug development.
Patient reported outcomes are of critical importance in the Duchenne community. While the six minute walk test is a sensitive measure and we all agree 30 meters is meaningful, there are other things that matter and should be considered as drugs are approved. We need to consider activities of daily living such as eating, combing hair, or rolling over in bed. Activities that indicate energy and endurance.
This invitation only workshop will help to inform a FDA sponsored public meeting in Spring, 2015.