IDENTIFICATION OF RARE DISEASES, AND DEVELOPMENT FIRMS

Executive Summary

IDENTIFICATION OF RARE DISEASES, AND DEVELOPMENT FIRMS can be aided by pharmacists, maintained American Pharmaceutical Association (APhA) Scientific Affairs Director Arthur Horowitz, PhD, at a Dec. 2 meeting of HHS' Orphan Products Board. APhA proposes "greater individual and group participation in the identification of those rare diseases requiring new drug development" and in "solicitation of sponsor commitments," Horowitz said. Pharmacists, pharmaceutical scientists, and pharmacy students represent "an important resource not fully utilized by the Orphan Products Board," Horowitz contended. "It is generally the pharmacist who has the most frequent contact with patients with rare diseases and who can counsel the patient regarding therapies. The network of many pharmaceutical scientists can identify additional sponsor commitments or provide specific expertise in the manufacture of a certain type of product." APhA also urged greater efforts to publicize the availability of orphan products to patients and to health care providers. The board should "work with all health care providers associations to guarantee continued publicity for newly approved orphan drugs," Horowitz said. For example, HHS should take advantage of "the over 150,000 pharmacists to inform the general public" about the National Information Center for Orphan Drugs and Rare Diseases and its toll-free telephone hotline. The department should also "develop consumer-oriented spot television and radio advertisements to inform the public on the availability of new orphan drugs," he continued. In addition, lists of FDA-approved orphan drugs and products being developed should be published in the FDA Bulletin and the FDA Electronic Bulletin Board.