NORD lauds FDA's establishment of new position within CDER

Peter L. Saltonstall, president and CEO of the National Organization for Rare Disorders (NORD), today said the Food and Drug Administration's (FDA) announcement that it has created a new position—Associate Director for Rare Diseases—in the agency's Center for Drug Evaluation and Research's (CDER) Office of New Drugs (OND) represents an important advance for patients with rare diseases.

"NORD asked FDA to create a new position within CDER to provide a focal point for the approval of new orphan drugs, and to supplement the support that orphan products receive from the Commissioner's office," Saltonstall said.  "We believe the creation of this new office reaffirms and expands FDA's commitment to facilitate the development and approval of safe and effective drugs for Americans with rare diseases."

Dr. Anne Pariser has been selected as the new Acting Associate Director for Rare Diseases and the agency said it will move quickly to fill the position on a permanent basis.  Dr. Pariser will report to the Director of the Office of New Drugs, Dr. John Jenkins.

"NORD has met with Dr. Pariser and her commitment to advancing the availability of safe and effective new drugs for rare diseases is clear," Saltonstall said.  "I especially want to thank Dr. Janet Woodcock, the Center's director, and Dr. Jenkins for their commitment to orphan products.  NORD also works closely with Dr. Tim Cote, and the staff of FDA's Office of Orphan Products Development, and we appreciate their commitment to the rare disease community."

Drs. Woodcock, Jenkins and Cote participated in NORD's Partners in Progress Summit last May and have met several times with NORD to discuss how to facilitate orphan drug development.

The FDA said that the Associate Director for Rare Diseases will serve as CDER's "focal point to the rare disease drug development community and assist stakeholders and developers of drug and biologic products in navigating the complex regulatory requirements for bringing safe and effective treatments to patients in need."

The Associate Director will also coordinate an initiative to develop CDER policies and procedures for the review and approval of treatments for rare diseases.  An important focus of this new initiative, according to FDA, will be to ensure collaboration among scientists and clinicians throughout CDER to help develop and bring to market new treatments for patients with rare diseases.

"Approximately 15 million Americans currently have rare diseases for which there is no treatment," Saltonstall said.  "One of NORD's primary missions is helping to fine-tune and accelerate the process of developing new treatments and evaluating their safety and efficacy.  We're very happy with FDA's announcement of the establishment of this position, which we view as a direct response to our advocacy."