Month: June 2017

Dr. Scott Gottlieb was appointed the new commissioner of the Food and Drug Administration (FDA) on May 11, 2017. He is a physician and a cancer survivor, having been successfully treated for Hodgkin’s lymphoma.

Gottlieb has previously commented that the agency’s current focus on statistics rather than good medicine is making it too difficult for drugs, especially orphan drugs, to get approved.

The National Coalition for Cancer Survivorship, for which Gottlieb served as a policy advisor, strongly endorsed his nomination to head the FDA, saying he “understands the human toll cancer takes on individuals and families, during both treatment and long-term survivorship. He is open to a wide range of perspectives, including those of the patients whose lives depend on a strong FDA.”

In a statement, Tom Price, the secretary of Health and Human Services, said Dr. Gottlieb’s “background will be crucial” for maintaining the FDA’s high standards for safe treatments “while advancing new, innovative solutions” to the nation’s public health challenges.

In rare diseases, the evaluation of new treatments is often challenging.  We are hopeful that Dr. Gottlieb will champion the need for the FDA to be efficient and patient-focused in the evaluation of vital new therapies for rare diseases like amyloidosis.