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U.S. Department of Health and Human Services


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FDA Expands Commitment to Patients with Rare Diseases

Expanding on its commitment to facilitate the development and approval of safe and effective drugs for Americans with rare diseases, FDA is pleased to announce the newly-created position of Associate Director for Rare Diseases in the Agency’s Center for Drug Evaluation and Research’s (CDER’s) Office of New Drugs (OND).  Dr. Anne Pariser, has been selected as the new Acting Associate Director for Rare Diseases and the Agency will move quickly to fill the position on a permanent basis.  Dr. Pariser will report to the Director of the Office of New Drugs. 

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 for Rare Diseases will also coordinate an initiative to develop CDER policies and procedures for the review and approval of treatments for rare diseases and to ensure appropriate training of CDER staff.  An important focus of this new initiative will be to ensure collaboration among scientists and clinicians throughout CDER, to promote the adoption of new scientific and regulatory innovations that will help facilitate timely development and approval of new treatments for patients with rare diseases.

The OND Associate Director for Rare Diseases will focus on the development and regulatory review of drugs for rare diseases and will complement the work of FDA’s Office of Orphan Products Development (OOPD).  OOPD will continue to exercise its authority under the Orphan Drug Act to recognize products that demonstrate promise for treating rare diseases with the designation of “orphan” status and offer financial incentives to manufacturers to develop and gain approval for these products.  OOPD also administers the Orphan Product Grants Program.  The OND Associate Director for Rare Diseases will work closely with staff in OOPD in applying the regulatory requirements for approval of drugs for rare diseases. 

Dr. Pariser brings a great deal of experience and expertise to this new position.  She has worked at FDA as a Medical Officer and Medical Team Leader in OND for the past 10 years.  For the last seven of these years she has honed her expertise in rare diseases working almost exclusively on the review and regulation of treatments for rare disorders known as Inborn Errors of Metabolism (IEM).  Her responsibilities have included the founding of a review team dedicated to the review of IEM products and education and training of other FDA staff for the study and regulation of IEMs.  Her work includes numerous collaborations with IEM stakeholders, including researchers, patient advocates and other regulatory agencies.