Topic: Rare Diseases

2015 Approvals Herald Rapid Progress, Growing Diversity in Precision Medicine

The Personalized Medicine Coalition (PMC) reports that 13 of 45 new molecular entities or therapeutic biologics approved by FDA in 2015, or 28%, were personalized medicines. The number of approved personalized (precision) medicines increased from 9 in 2014. While completing the transition to precision medicine will take years and require addressing major challenges, the momentum… Read more »

Debunking the Instant Myth of FDA as Rubber Stamp

FDA’s high approval rate for New Drug Applications (NDAs) is much in the news, with headlines like the one on a Matthew Herper post at Forbes: The FDA Is Basically Approving Everything. Here’s the Data to Prove It. The data comes from an analysis that Forbes commissioned at BioMedTracker. When FDA approves 23 out of… Read more »

Independent Pre-FDA Draft Guidances from Stakeholders

The introduction to an FDA draft guidance for industry published in June 2015, “Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment,” makes an unusual statement: Development of this guidance was preceded by the submission to FDA of a proposed draft guidance independently prepared by a consortium of stakeholders including patients, parents and caregivers, clinicians,… Read more »