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Executive Summary

FDA ORPHAN PRODUCTS OFFICE IS MEETING WITH REVIEW DIVISIONS "to discuss specific opportunities for developing orphan products," the agency said May 8. The meetings are in response to a recently-released report of the National Commission on Orphan Diseases. FDA also noted that its Office of Orphan Products Development plans to develop a "compilation of materials to help sponsors prepare for the labeling, good manufacturing practices, and other regulatory challenges" involved in winning FDA approval. The two planned actions by the agency's orphan products office were included in a list of FDA activities in the orphan drug area that were outlined in a May 8 FDA "Talk Paper." The National Commission on Orphan Diseases released its final report last month ("The Pink Sheet" April 3, T&G-5). In response to the commission's recommendation that all orphan drugs be treated as 1A reviews (new chemical entities representing significant therapeutic advances), FDA said it "does not feel it is appropriate to classify all orphans as 1A." While acknowledging that a 1A classification might allow some orphan drugs to be eligible for a Treatment IND or open protocol, FDA said only that it would "continue to give high priority to those [orphan drugs] with important and moderate potential for therapeutic gains." The agency noted that orphan products for AIDS and other life-threatening or severely debilitating diseases would receive the "extra attention" that FDA has committed to giving products for such conditions. The "Talk Paper" also points out that all orphans are currently eligible to apply for Treatment IND status or for an open "compassionate use" protocol. FDA said it will evaluate "the benefits of orphan products in light of the severity of the diseases being treated." Other actions in the orphan area listed in the "Talk Paper" include: publicizing to orphan drug sponsors the existence of Drug Master Files (DMF) and putting DMF sponsors in touch with developers of new orphan uses; continuing to cosponsor symposiums to attract investigators and encouraging use of Small Business Innovation Research grants; referring orphan grant applications that do not meet FDA's "relevance standards" to the NIH; and routinely including rare disease experts as reviewers of orphan grant applications.

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