The regulatory pathway that we have learned from rare diseases is now becoming the paradigm in general, FDA Deputy Commissioner Stephen P. Spielberg, MD, PhD, said this morning in the opening session of Day 2 of the U.S. Conference on Rare Diseases and Orphan Products.

Dr. Spielberg acknowledged the challenges in drug development today — the need to reduce large clinical trials to more efficient, less costly, smaller ones and to cut drug development and review times significantly — but expressed optimism that these challenges may be met, using rare diseases as a guide.  He pointed to the recently approved therapy for cystic fibrosis patients who have a specific mutation as one model.

“I’ve never been so optimistic about the field of medicine,” Dr. Spielberg, a pediatrician, said in describing the pace of scientific discovery today.  Basic, translational, and clinical science are advancing in tandem, he said.  Regulatory pathways need to advance to match the science.

Other speakers at the conference will be focusing on advocacy and how to better harness the power of today’s science so that it translates into better healthcare for patients.  Dr. Spielberg proposed a new paradigm emphasizing communication, breaking down of silos and partnership among all involved.  Thoughts?