SMG 1141.12

FDA STAFF MANUAL GUIDES, VOLUME I - ORGANIZATIONS AND FUNCTIONS

FOOD AND DRUG ADMINISTRATION

OFFICE OF MEDICAL PRODUCTS AND TOBACCO

OFFICE OF SPECIAL MEDICAL PROGRAMS

OFFICE OF ORPHAN PRODUCTS DEVELOPMENT

Effective Date: 07/08/2011

[PDF Version]

1. OFFICE OF ORPHAN PRODUCTS DEVELOPMENT (DKKAB)

A. Manages the implementation of the provisions of the Orphan Drug Act and its amendments as well as implementation of provisions of statute related to humanitarian devices and pediatric devices and manages a program to encourage the development of drugs and devices of limited commercial value for use in rare or common diseases and conditions

B. Develops and communicates Agency policy and makes decisions on approval of sponsor requests and incentives under the Federal Food, Drug, and Cosmetic Act, including orphan drug protocol assistance per section 525, orphan drug designation per section 526, orphan drug exclusivity per section 527, orphan drug grants and contracts to support clinical research, humanitarian devices, pediatric device consortia grants and other areas of Agency policy related to the development of products for rare disorders

C. Represents the Commissioner or serves as the Agency’s principal authority and spokesperson to governmental committees, industry, foreign regulatory bodies, professional, patient advocates and consumer associations requesting Agency participation in orphan product development activities

D. Reviews investigational new drug and biologics applications and investigational device exemptions to locate the existence of products under investigational study that show promise for effectiveness for rare or common diseases but lack commercial sponsorship. Assists sponsors, researchers, and investigators in communicating with Agency regulatory officials and expediting solutions to problems in obtaining investigational or market approval status

E. Manages extramural programs of clinical research and consortia to evaluate safety and effectiveness of orphan products by funding grants and contracts, requesting applications for funding, organizing peer review of applications, monitoring and guiding investigators, and evaluating study results

2. AUTHORITY AND EFFECTIVE DATE

The functional statements for this Office were approved by the Secretary of Health and Human Services, effective July 8, 2011.

STATUS (I, R, C)	DATE APPROVED	LOCATION OF CHANGE HISTORY	CONTACT	APPROVING OFFICIAL
Initial	07/08/2011	N/a	OO/OM	Secretary of Health and Human Services