Several weeks ago, I was invited to participate in a discussion focused on how large pharmaceutical companies should communicate with advocacy; how communication needs to be different in rare disease; and more specifically what advocates in rare diseases expect, want, and need from pharmaceutical companies. I was one of…Continue
Wading through policy is no easy task. But imagine policy as the white lines on the road, the boundaries within which FDA operates. Building our case, showing how Duchenne fits into those white lines and demonstrating how and when Accelerated Approval makes perfect sense is the purpose of PPMD’s white paper. …Continue
Two weeks ago, PPMD proudly published Putting Patients First, a white paper outlining recommendations to speed responsible access to new therapies for Duchenne. In that short amount of time, the reaction to this white paper has been phenomenal. From interest by the media to accolades from industry and agencies, we have been truly humbled by the response we have received.
And while the…Continue
April 29-30 in Washington, D.C.
Recently I had the privilege of attending and participating in the Symposium on Best Practices in Clinical Study Design of Rare Diseases in Washington, D.C. The symposium was organized in collaboration with Children’s National Medical Center (CNMC), Clinical and Translational Science Institute of Children’s National (CTSI), The George Washington University, and Research in Pediatric Developmental Pharmacology Centers (RPDP). This…Continue
CLINICAL TRIALS – The path to approval.
Typically we think about the Clinical Trial Process in the context of our kids. We hear about Phase I trials. Depending on the drug/biologic, many of these compounds will be required to be tested in healthy human subjects. Some Phase I trials have inclusion criteria, suggesting they are looking for healthy adults of a certain age to take a single or multiple doses of a compound or biologic. The purpose of the study…Continue