Pagpapalawak sa kanyang pangako upang mapadali ang pag-unlad at pag-apruba ng mga ligtas at mabisang gamot para sa mga Amerikanong may mga bihirang sakit, Ang FDA ay nasisiyahang ipahayag ang mga bagong-nilikha posisyon ng Associate Director para sa Rare Karamdaman sa Center ang Agency para sa Drug Pagsusuri at Research ng (CDER ng) Opisina ng mga Bagong Gamot (Masama).
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, upang itaguyod ang pag-aampon ng mga bagong pang-agham at regulasyon na mga makabagong-likha na makakatulong mapadali napapanahong pag-unlad at pag-apruba ng mga bagong paggamot para sa mga pasyente na may bihirang sakit.
Ang OND Associate Director para sa Rare Karamdaman ay tumutok sa pag-unlad at regulasyon na pagsusuri ng mga gamot para sa mga bihirang sakit at makadagdag ang gawain ng FDA Office of ulila Produkto 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 taon. 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, kabilang ang mga mananaliksik, tagapagtaguyod ng pasyente at iba pang mga regulasyon ahensya.