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Executive Summary

FDA/NIH IMPROVED COORDINATION ON ORPHAN DRUGS COULD SPEED APPROVALS, the National Commission on Orphan Diseases suggested at its eighth annual meeting, July 21-22 in Bethesda, Maryland. A preliminary recommendation emerged from meeting workshops to "enhance coordination between research activities funded by NIH with approval activities of FDA" to accelerate orphan drug approval. UCLA Professor Edward Ritvo, MD, who headed the working group, said one concern is that much of NIH's research is not being accepted by FDA. He suggested that NIH research grants include a protocol consistent with FDA's requirements for NDA approval. The group further recommended to "increase liaison between NIH and FDA on grant submission," and tentatively recommended that "the commissioner of the FDA impose New Drug Approval authority with the chief officer of orphan products for all designated orphan products." The National Commission on Orphan Diseases is working on a report that will be submitted to Congress by Feb. 1, 1989. A draft of the final report, which will be based on surveys of physicians, researchers, federal agencies and foundations involved with orphan diseases, is scheduled for completion by Oct. 15. The commission will meet in August and again in September or October to make its final recommendations. FDA Director of Orphan Products Development Marlene Haffner, MD, said that FDA is already trying to improve coordination with NIH through meetings between Center of Drug Evaluation and Review Director Carl Peck, MD, and institute heads at NIH. Haffner indicated that she was in general agreement with the recommendations but hesitated at the idea of greatly expanding FDA's role in the evaluation of NIH grants. "We are funding about 15% of our grants this year," she said. "If I get that many more [grant applications], I'm going to have that many less [grant approvals]." PMA Commission on Rare Diseases President George Goldstein suggested an "NIH-FDA axis" similar to PMA's ongoing regulatory relationship with FDA. This sort of arrangement, Goldstein said, would give the NIH investigator "an idea early on of what is needed, of what will or won't fly . . . so that those that can fly can be worked on faster, and those that won't can be told what they need to do to give them lift."

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